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Sample records for patient-reported outcomes measurement

  1. [Patient-reported outcomes: definition and measurement].

    PubMed

    Botturi, Davide; Rodella, Stefania

    2014-06-01

    The concept of "patient-reported outcomes" have been proposed by the Food and Drug Administration in the year 2000, in order to describe one of the different and potential sources of information on the drug's safety and effectiveness. It represents an "umbrella" term, which covers a multiplicity of meanings and primarily identifies a conceptual approach and a methodology specifically oriented to the patients' point of view on outcomes, instead of the traditional clinical and professional perspective. The patient-reported outcomes measures are frequently self-completed questionnaires. The measures can be classified in general and specific. The first one, general, relates to the assessment of the quality of life or the health status, in the general population or in subgroups with particular health problems (eg. SF-36 Health Survey, EQ-5D). The second one, specific, mainly relates to the assessment of particular types of symptoms (eg. pain, anxiety, fear, depression) and functions (eg. daily living activities), in population's subgroups with definite health problems, undergoing or not to a healthcare procedure (eg. Adult Asthma Quality of Life Questionnaire, Kidney Disease Quality of Life Instrument, Oxford Hip Score, Oxford Knee Score). For the selection of an instrument a series of criteria needs to be taken into account, among which the psychometric properties, the expert judgement, the interpretability, the acceptability, and the feasibility of the entire process. PMID:25002284

  2. Patient reported outcome measures in septorhinoplasty surgery

    PubMed Central

    Fraser, LR; Ward, MJ; Sunkaraneni, VS; Harries, PG; Salib, RJ

    2015-01-01

    Introduction Surgical procedures incorporating a cosmetic element such as septorhinoplasty and otoplasty are currently under threat in the National Health Service (NHS) as they are deemed to be procedures of ‘limited clinical benefit’ by many primary care providers. Patient reported outcome measures (PROMs), which assess the quality of care delivered from the patients’ perspective, are becoming increasingly important in documenting the effectiveness of such procedures. Methods The Rhinoplasty Outcomes Evaluation (ROE) questionnaire, a validated PROM tool, was used to assess patient satisfaction in 141 patients undergoing septorhinoplasty surgery over a 90-month period at the University Hospital Southampton NHS Foundation Trust. Results Overall, 100 patients with a mean follow-up period of 36 months completed the study. The mean ROE score was 73.3%. In addition, 75% of patients questioned were happy with the final result of their operation and 83% would undergo the procedure again if required. These benefits occurred irrespective of age, sex and primary versus revision surgery, and were maintained for up to 71 months following surgery. Conclusions This study has shown that patients are generally satisfied with their functional and cosmetic outcomes following septorhinoplasty surgery. These results help support the case for septorhinoplasty surgery to continue being funded as an NHS procedure. PMID:25519270

  3. Patient reported outcome measures in neurogenic bladder

    PubMed Central

    Clark, Roderick

    2016-01-01

    Many interventions for neurogenic bladder patients are directed towards improving quality of life (QOL). Patient reported outcome measures (PROMs) are the primary method of evaluating QOL, and they provide an important quantification of symptoms which can’t be measured objectively. Our goal was to review general measurement principles, and identify and discuss PROMs relevant to neurogenic bladder patients. We identify two recent reviews of the state of the literature and updated the results with an additional Medline search up to September 1, 2015. Using the previous identified reviews, and our updated literature review, we identified 16 PROMs which are used for the assessment of QOL and symptoms in neurogenic bladder patients. Several are specifically designed for neurogenic bladder patients, such as the Qualiveen (for neurogenic bladder related QOL), and the Neurogenic Bladder Symptom Score (NBSS) (for neurogenic bladder symptoms). We also highlight general QOL measures for patients with multiple sclerosis (MS) and spinal cord injury (SCI) which include questions about bladder symptoms, and incontinence PROMs which are commonly used, but not specifically designed for neurogenic bladder patients. It is essential for clinicians and researchers with an interest in neurogenic bladder to be aware of the current PROMs, and to have a basic understanding of the principals of measurement in order to select the most appropriate one for their purpose. PMID:26904409

  4. Patient-Reported Outcome Measures in Systemic Sclerosis (Scleroderma).

    PubMed

    Pellar, Russell E; Tingey, Theresa M; Pope, Janet Elizabeth

    2016-05-01

    Scleroderma (systemic sclerosis) is a rare autoimmune connective tissue disease that can damage multiple organs and reduce quality of life. Patient-reported outcome measures capture the patient's perspective. Some measures are specific to systemic sclerosis and others are general. Patient-reported outcomes in systemic sclerosis are important to aid in understanding the impact of systemic sclerosis on patients. PMID:27133491

  5. Advances in Patient-Reported Outcomes: The NIH PROMIS® Measures

    PubMed Central

    Broderick, Joan E.; DeWitt, Esi Morgan; Rothrock, Nan; Crane, Paul K.; Forrest, Christopher B.

    2013-01-01

    Patient-reported outcomes (PRO) are questionnaire measures of patients’ symptoms, functioning, and health-related quality of life. They are designed to provide important clinical information that generally cannot be captured with objective medical testing. In 2004, the National Institutes of Health launched a research initiative to improve the clinical research enterprise by developing state-of-the-art PROs. The NIH Patient-Reported Outcomes Measurement System (PROMIS) and Assessment Center are the products of that initiative. Adult, pediatric, and parent-proxy item banks have been developed by using contemporary psychometric methods, yielding rapid, accurate measurements. PROMIS currently provides tools for assessing physical, mental, and social health using short-form and computer-adaptive testing methods. The PROMIS tools are being adopted for use in clinical trials and translational research. They are also being introduced in clinical medicine to assess a broad range of disease outcomes. Recent legislative developments in the United States support greater efforts to include patients’ reports of health experience in order to evaluate treatment outcomes, engage in shared decision-making, and prioritize the focus of treatment. PROs have garnered increased attention by the Food and Drug Administration (FDA) for evaluating drugs and medical devices. Recent calls for comparative effectiveness research favor inclusion of PROs. PROs could also potentially improve quality of care and disease outcomes, provide patient-centered assessment for comparative effectiveness research, and enable a common metric for tracking outcomes across providers and medical systems. PMID:25848562

  6. Patient-reported outcome measures in inflammatory bowel disease

    PubMed Central

    El-Matary, Wael

    2014-01-01

    Patient-reported outcome measures (PROMs) are increasingly used in both research and clinical health settings. With the recent development of United States Food and Drug Administration guidance on PROMs, more attention is being devoted to their role and importance in health care. Several methodological challenges in the development, validation and implementation of PROMs must be resolved to ensure their appropriate utilization and interpretation. The present review discusses recent developments and updates in PROMs, with specific focus on the area of inflammatory bowel disease. PMID:25390615

  7. Patient-reported outcome measures in arthroplasty registries.

    PubMed

    Rolfson, Ola; Eresian Chenok, Kate; Bohm, Eric; Lübbeke, Anne; Denissen, Geke; Dunn, Jennifer; Lyman, Stephen; Franklin, Patricia; Dunbar, Michael; Overgaard, Søren; Garellick, Göran; Dawson, Jill

    2016-07-01

    The International Society of Arthroplasty Registries (ISAR) Steering Committee established the Patient-Reported Outcome Measures (PROMs) Working Group to convene, evaluate, and advise on best practices in the selection, administration, and interpretation of PROMs and to support the adoption and use of PROMs for hip and knee arthroplasty in registries worldwide. The 2 main types of PROMs include generic (general health) PROMs, which provide a measure of general health for any health state, and specific PROMs, which focus on specific symptoms, diseases, organs, body regions, or body functions. The establishment of a PROM instrument requires the fulfillment of methodological standards and rigorous testing to ensure that it is valid, reliable, responsive, and acceptable to the intended population. A survey of the 41 ISAR member registries showed that 8 registries administered a PROMs program that covered all elective hip or knee arthroplasty patients and 6 registries collected PROMs for sample populations; 1 other registry had planned but had not started collection of PROMs. The most common generic instruments used were the EuroQol 5 dimension health outcome survey (EQ-5D) and the Short Form 12 health survey (SF-12) or the similar Veterans RAND 12-item health survey (VR-12). The most common specific PROMs were the Hip disability and Osteoarthritis Outcome Score (HOOS), the Knee injury and Osteoarthritis Outcome Score (KOOS), the Oxford Hip Score (OHS), the Oxford Knee Score (OKS), the Western Ontario and McMaster Universities Arthritis Index (WOMAC), and the University of California at Los Angeles Activity Score (UCLA). PMID:27168175

  8. Patient-reported outcome measures in arthroplasty registries

    PubMed Central

    Bohm, Eric; Franklin, Patricia; Lyman, Stephen; Denissen, Geke; Dawson, Jill; Dunn, Jennifer; Eresian Chenok, Kate; Dunbar, Michael; Overgaard, Søren; Garellick, Göran; Lübbeke, Anne

    2016-01-01

    Abstract — The International Society of Arthroplasty Registries (ISAR) Patient-Reported Outcome Measures (PROMs) Working Group have evaluated and recommended best practices in the selection, administration, and interpretation of PROMs for hip and knee arthroplasty registries. The 2 generic PROMs in common use are the Short Form health surveys (SF-36 or SF-12) and EuroQol 5-dimension (EQ-5D). The Working Group recommends that registries should choose specific PROMs that have been appropriately developed with good measurement properties for arthroplasty patients. The Working Group recommend the use of a 1-item pain question (“During the past 4 weeks, how would you describe the pain you usually have in your [right/left] [hip/knee]?”; response: none, very mild, mild, moderate, or severe) and a single-item satisfaction outcome (“How satisfied are you with your [right/left] [hip/knee] replacement?”; response: very unsatisfied, dissatisfied, neutral, satisfied, or very satisfied). Survey logistics include patient instructions, paper- and electronic-based data collection, reminders for follow-up, centralized as opposed to hospital-based follow-up, sample size, patient- or joint-specific evaluation, collection intervals, frequency of response, missing values, and factors in establishing a PROMs registry program. The Working Group recommends including age, sex, diagnosis at joint, general health status preoperatively, and joint pain and function score in case-mix adjustment models. Interpretation and statistical analysis should consider the absolute level of pain, function, and general health status as well as improvement, missing data, approaches to analysis and case-mix adjustment, minimal clinically important difference, and minimal detectable change. The Working Group recommends data collection immediately before and 1 year after surgery, a threshold of 60% for acceptable frequency of response, documentation of non-responders, and documentation of incomplete or

  9. Advances in Patient-Reported Outcomes: The NIH PROMIS(®) Measures.

    PubMed

    Broderick, Joan E; DeWitt, Esi Morgan; Rothrock, Nan; Crane, Paul K; Forrest, Christopher B

    2013-01-01

    Patient-reported outcomes (PRO) are questionnaire measures of patients' symptoms, functioning, and health-related quality of life. They are designed to provide important clinical information that generally cannot be captured with objective medical testing. In 2004, the National Institutes of Health launched a research initiative to improve the clinical research enterprise by developing state-of-the-art PROs. The NIH Patient-Reported Outcomes Measurement System (PROMIS) and Assessment Center are the products of that initiative. Adult, pediatric, and parent-proxy item banks have been developed by using contemporary psychometric methods, yielding rapid, accurate measurements. PROMIS currently provides tools for assessing physical, mental, and social health using short-form and computer-adaptive testing methods. The PROMIS tools are being adopted for use in clinical trials and translational research. They are also being introduced in clinical medicine to assess a broad range of disease outcomes. Recent legislative developments in the United States support greater efforts to include patients' reports of health experience in order to evaluate treatment outcomes, engage in shared decision-making, and prioritize the focus of treatment. PROs have garnered increased attention by the Food and Drug Administration (FDA) for evaluating drugs and medical devices. Recent calls for comparative effectiveness research favor inclusion of PROs. PROs could also potentially improve quality of care and disease outcomes, provide patient-centered assessment for comparative effectiveness research, and enable a common metric for tracking outcomes across providers and medical systems. PMID:25848562

  10. Patient-Reported Shoulder Outcome Measures Utilized in Breast Cancer Survivors: A Systematic Review

    PubMed Central

    Harrington, Shana; Michener, Lori A; Kendig, Tiffany; Miale, Susan; George, Steven Z

    2014-01-01

    Objective 1) To identify English Language published patient-reported upper extremity outcome measures used in breast cancer research and 2) To examine construct validity and responsiveness in patient-reported upper extremity outcome measures used in breast cancer research. Data Sources PubMed, CINAHL and ProQuest MEDLINE® databases were searched up to February 5, 2013. Study Selection Studies were included if a patient-reported upper extremity outcome measure was administered, the participants were diagnosed with breast cancer, and published in English. Data Extraction Eight hundred and sixty-five articles were screened. Fifty-nine full text articles were assessed for eligibility. A total of 46 articles met the initial eligibility criteria for aim 1. Eleven of these articles reported mean and standard deviations for the outcome scores, and included a comparison group analysis for aim 2. Data Synthesis Construct validity was evaluated by calculating effect sizes for known group differences in 6 studies using the Disabilities of Arm, Shoulder and Hand (DASH), Penn Shoulder Score, Shoulder Disability Questionnaire-Dutch, and 10 Questions by Wingate (Wingate). Responsiveness was analyzed comparing a treatment and control group by calculating the coefficient of responsiveness in 5 studies for the DASH and Wingate. Conclusions Eight different patient-reported upper extremity outcome measures have been reported in the peer-review literature for women with breast cancer, some (n=3) were specifically developed for breast cancer survivors and others that were not (n=5). Based on the current evidence we recommend administering the DASH to assess patient-reported upper extremity function in breast cancer survivors because the DASH had most consistently large effects sizes for construct validity and responsiveness. Future large studies are needed for more definitive recommendations. PMID:23932969

  11. Using Patient Reported Outcome Measures to Improve Service Effectiveness (UPROMISE): Training clinicians to Use Outcome Measures in Child Mental Health.

    PubMed

    Edbrooke-Childs, Julian; Wolpert, Miranda; Deighton, Jessica

    2016-05-01

    Patient reported outcome measures (PROMs) are prevalent in child mental health services. In this point of view, we discuss our experience of training clinicians to use PROMs and to interpret and discuss feedback from measures. Findings from pre-post observational data from clinicians who attended either a 1- or 3-day training course showed that clinicians in both courses had more positive attitudes and higher levels of self-efficacy regarding administering measures and using feedback after training. We hope that this special issue will lead the way for future research on training clinicians to use outcome measures so that PROMs may be a source of clinically useful practice based evidence. PMID:25331446

  12. Challenges and Opportunities in Using Patient-reported Outcomes in Quality Measurement in Rheumatology.

    PubMed

    Wahl, Elizabeth R; Yazdany, Jinoos

    2016-05-01

    Use of patient-reported outcome measures (PROs) in rheumatology research is widespread, but use of PRO data to evaluate the quality of rheumatologic care delivered is less well established. This article reviews the use of PROs in assessing health care quality, and highlights challenges and opportunities specific to their use in rheumatology quality measurement. It first explores other countries' experiences collecting and evaluating national PRO data to assess quality of care. It describes the current use of PROs as quality measures in rheumatology, and frames an agenda for future work supporting development of meaningful quality measures based on PROs. PMID:27133495

  13. Longitudinal evaluation of patient-reported outcomes measurement information systems measures in pediatric chronic pain.

    PubMed

    Kashikar-Zuck, Susmita; Carle, Adam; Barnett, Kimberly; Goldschneider, Kenneth R; Sherry, David D; Mara, Constance A; Cunningham, Natoshia; Farrell, Jennifer; Tress, Jenna; DeWitt, Esi Morgan

    2016-02-01

    The Patient-Reported Outcomes Measurement Information System (PROMIS) initiative is a comprehensive strategy by the National Institutes of Health to support the development and validation of precise instruments to assess self-reported health domains across healthy and disease-specific populations. Much progress has been made in instrument development, but there remains a gap in the validation of PROMIS measures for pediatric chronic pain. The purpose of this study was to investigate the construct validity and responsiveness to change of 7 PROMIS domains for the assessment of children (ages: 8-18) with chronic pain--Pain Interference, Fatigue, Anxiety, Depression, Mobility, Upper Extremity Function, and Peer Relationships. The PROMIS measures were administered at the initial visit and 2 follow-up visits at an outpatient chronic pain clinic (CPC; N = 82) and at an intensive amplified musculoskeletal pain day-treatment program (N = 63). Aim 1 examined construct validity of PROMIS measures by comparing them with corresponding "legacy" measures administered as part of usual care in the CPC sample. Aim 2 examined sensitivity to change in both CPC and amplified musculoskeletal pain samples. Longitudinal growth models showed that PROMIS' Pain Interference, Anxiety, Depression, Mobility, Upper Extremity, and Peer Relationship measures and legacy instruments generally performed similarly with slightly steeper slopes of improvement in legacy measures. All 7 PROMIS domains showed responsiveness to change. Results offered initial support for the validity of PROMIS measures in pediatric chronic pain. Further validation with larger and more diverse pediatric pain samples and additional legacy measures would broaden the scope of use of PROMIS in clinical research. PMID:26447704

  14. The Promise of Patient-Reported Outcomes Measurement Information System-Turning Theory into Reality: A Uniform Approach to Patient-Reported Outcomes Across Rheumatic Diseases.

    PubMed

    Witter, James P

    2016-05-01

    PROMIS, the Patient-Reported Outcomes Measurement Information System, is opening new possibilities to explore and learn how patient (or proxy) self-report of core symptoms and health-related quality of life can meaningfully advance clinical research and patient care. PROMIS leverages Item Response Theory to agnostically assess, across diseases and conditions or clinical settings, numerous universally applicable core "domains" of health (symptoms and functioning) from the patient perspective. Importantly, PROMIS is enabling the testing and adoption of computerized adaptive testing, which holds great potential to minimize patient burden while maximizing accuracy. PMID:27133496

  15. Measuring and Predicting Patient Dissatisfaction after Anterior Urethroplasty Using Patient Reported Outcomes Measures

    PubMed Central

    Bertrand, Laura A.; Voelzke, Bryan B.; Elliott, Sean P.; Myers, Jeremy B.; Breyer, Benjamin N.; Vanni, Alex J.; McClung, Christopher D.; Tam, Christopher A.; Warren, Gareth J.; Erickson, Bradley A.

    2016-01-01

    Purpose Subjective measures of success after urethroplasty have become increasingly valuable in postoperative monitoring. We examined patient reported satisfaction following anterior urethroplasty using objective measures as a proxy for success. Materials and Methods Men 18 years old or older with urethral strictures undergoing urethroplasty were prospectively enrolled in a longitudinal, multi-institutional urethroplasty outcomes database. Preoperative and postoperative assessment included questionnaires to assess lower urinary tract symptoms, pain, satisfaction and sexual health. Analyses controlling for stricture recurrence (defined as the inability to traverse the reconstructed urethra with a flexible cystoscope) were performed to determine independent predictors of dissatisfaction. Results At a mean followup of 14 months we found a high 89.4% rate of overall postoperative satisfaction in 433 patients and a high 82.8% rate in those who would have chosen the operation again. Men with cystoscopic recurrence were more likely to report dissatisfaction (OR 4.96, 95% CI 2.07–11.90) and men reporting dissatisfaction had significantly worse uroflowmetry measures (each p <0.02). When controlling for recurrence, multivariate analysis revealed that urethra and bladder pain (OR 1.71, 95% CI 1.05–2.77 and OR 2.74, 95% CI 1.12–6.69, respectively), a postoperative decrease in sexual activity (OR 4.36, 95% CI 2.07–11.90) and persistent lower urinary tract symptoms (eg straining to urinate OR 3.23, 1.74-6.01) were independent predictors of dissatisfaction. Conclusions Overall satisfaction after anterior urethroplasty is high and traditional measures of surgical success strongly correlate with satisfaction. However, independently of the anatomical appearance of the reconstructed urethra, postoperative pain, sexual dysfunction and persistent lower urinary tract symptoms were predictors of patient dissatisfaction. PMID:26907509

  16. Development and Validation of the Keele Musculoskeletal Patient Reported Outcome Measure (MSK-PROM)

    PubMed Central

    2015-01-01

    Objective To develop and validate a patient report outcome measure (PROM) for clinical practice that can monitor health status of patients with a range of musculoskeletal (MSK) disorders. Methods Constructs for inclusion in the MSK-PROM were identified from a consensus process involving patients with musculoskeletal conditions, clinicians, purchasers of healthcare services, and primary care researchers. Psychometric properties of the brief tool, including face and construct validity, repeatability and responsiveness were assessed in a sample of patients with musculoskeletal pain consulting physiotherapy services in the United Kingdom (n=425). Results The consensus process identified 10 prioritised domains for monitoring musculoskeletal health status: pain intensity, quality of life, physical capacity, interference with social/leisure activities, emotional well-being, severity of most difficult thing, activities and roles, understanding independence, and overall impact. As the EuroQol (EQ-5D-5L) is a widely adopted PROMs tool and covers the first four domains listed, to reduce patient burden to a minimum the MSK-PROM was designed to capture the remaining six prioritised domains which are not measured by the EQ-5D-5L. The tool demonstrated excellent reliability, construct validity, responsiveness and acceptability to patients and clinicians for use in clinical practice. Conclusion We have validated a brief patient reported outcome measure (MSK-PROM) for use in clinical practice to measure musculoskeletal health status and monitor outcomes over time using domains that are meaningful to patients and sensitive to change. Further work will establish whether the MSK-PROM is useful in other musculoskeletal healthcare settings. PMID:25928807

  17. The measurement of patient-reported outcomes of refractive surgery: the refractive status and vision profile.

    PubMed Central

    Schein, O D

    2000-01-01

    PURPOSE: To develop a questionnaire, the Refractive Status and Vision Profile (RSVP), to assess health-related quality of life associated with refractive error and its correction. METHODS: The published literature on patient report of visual and overall function was reviewed, and the RSVP was self-administered by 550 participants with refractive error. Cross-sectional validation was performed using standard psychometric techniques. The responsiveness of the RSVP to surgical intervention was assessed prospectively in a subset of 176 patients. The principal outcomes measures were scores on the overall RSVP scale (S) and on 8 RSVP subscales (functioning, driving, concern, expectations, symptoms, glare, optical problems, problems with corrective lenses). RESULTS: The RSVP (S) and its subscales demonstrated very good internal consistency (Cronbach's alpha, 0.70-0.93). S and several subscale scores were independently associated with satisfaction with vision and were more correlated with satisfaction with vision than with either visual acuity or refractive error. Higher refractive error was associated with lower scores on S and on 5 subscales. In the prospective surgical cohort, 15% of patients had some worsening in their total RSVP score; however, substantial variation was seen in the individual subscales where worsening ranged from 7% (problems with corrective lenses) to 41% (driving). The effect size (measure of responsiveness) of the RSVP and most of its subscales was very high. Approximately 14% of patients had significant worsening in 3 or more subscales, and this outcome was found to be independently associated with being dissatisfied with vision following surgery (OR, 5.84; 95% CI, 1.88, 8.13). CONCLUSIONS: The RSVP has been validated as a questionnaire that measures patient-reported quality of life related to refractive error and its correction. It is responsive to surgical intervention and provides important information regarding patient outcomes not available

  18. Patient-Reported Outcome Measures-What Data Do We Really Need?

    PubMed

    Lyman, Stephen; Hidaka, Chisa

    2016-06-01

    The Center for Medicaid and Medicare Services has recently announced the inclusion of several patient-reported outcome measures (PROMs), including the abbreviated Hip Disability and Osteoarthritis Outcome Score and Knee Injury and Osteoarthritis Outcome Score for joint replacement (HOOS, JR and KOOS, JR) for the purpose of quality assessment in total hip and total knee replacement (THR and TKR). Historically, Center for Medicaid and Medicare Services and other agencies have used measures of process (eg, % vaccinated) or adverse events (eg, infection rates, readmission rates) for quality assessment. However, the use of PROMs has become a priority based on stated goals by the National Quality Strategy and Institute of Medicine for a more patient-centered approach. Here, we review several general health and joint-specific PROMs, which have been extensively used in research to assess treatment efficacy and discuss their relevance to the new criteria for quality assessment, particularly for THR and TKR. Although we expect HOOS, JR and KOOS, JR to yield much useful information in the near term, these surveys are likely an interim solution. In the future, we anticipate that novel measurement platforms, such as wearable technologies or patient-specific surveys, may open new and exciting avenues of research to discover which types of data-perhaps not previously available-best represent patient quality of life and satisfaction after THR, TKR, or other orthopedic procedures. PMID:27157278

  19. Patient reported outcome measures for cardiac ablation procedures: a multicentre pilot to develop a new questionnaire

    PubMed Central

    Withers, Kathleen L.; White, Judith; Carolan-Rees, Grace; Patrick, Hannah; O'Callaghan, Peter; Murray, Stephen; Cunningham, David; Wood, Kathryn A.; Lencioni, Mauro; Griffith, Michael

    2014-01-01

    Aim To assess the feasibility of administering Patient Reported Outcomes Measures (PROMs) in patients treated with ablation for cardiac arrhythmias, and to conduct the first stage of development and testing of a new PROM tool. Methods and results A new tool was developed by a multidisciplinary team and tested alongside an adaptation of the patient perception of arrhythmia questionnaire (PPAQ) and EQ-5D-5L in a multicentre retrospective audit involving 791 consecutive cardiac arrhythmia patients treated with catheter ablation at three UK centres over 13 months. Data were recorded in the National Cardiac Rhythm Management Database, part of the National Institute for Cardiovascular Outcomes Research. The response rate was 71.9% (n = 569). Patients reported significant improvements across all outcomes and impacts, with reductions in symptoms of 51.7% (heart racing), 33.9% (fatigue) 31.8% (heart flutters), 43.5% (dizziness), 38.6% (breathlessness), 44.2% (chest pressure), 33.1% (trouble concentrating), 15.9% (headache), 28.3% (neck pressure), and 23.4% (fainting) (P < 0.001). The mean number of social days affected reduced by 7.49 days/month (P < 0.001); mean work/school days affected/month reduced by 6.26 (P < 0.001); mean GP/hospital visits reduced by 1.36 days/month (P < 0.001). The procedure met patient expectations in 72% of responders. Conclusions The high response rate suggests that the use of PROMs in this patient group is feasible, with rates equalling those of the National PROMs Programme. The results showed that patients experienced significant improvements in their quality of life following ablation, while feedback allowed the tools to be improved. Further work is required to validate these tools; however, the findings suggest that PROMs could be useful in the audit of ablation techniques. PMID:24627541

  20. Beyond FEV1 in COPD: a review of patient-reported outcomes and their measurement

    PubMed Central

    Jones, Paul; Miravitlles, Marc; van der Molen, Thys; Kulich, Karoly

    2012-01-01

    Patients with chronic obstructive pulmonary disease (COPD) present with a variety of symptoms and pathological consequences. Although primarily viewed as a respiratory disease, COPD has both pulmonary and extrapulmonary effects, which have an impact on many aspects of physical, emotional, and mental well-being. Traditional assessment of COPD relies heavily on measuring lung function, specifically forced expiratory volume in 1 second (FEV1). However, the evidence suggests that FEV1 is a relatively poor correlate of symptoms such as breathlessness and the impact of COPD on daily life. Furthermore, many consequences of the disease, including anxiety and depression and the ability to perform daily activities, can only be described and reported reliably by the patient. Thus, in order to provide a comprehensive view of the effects of interventions in clinical trials, it is essential that spirometry is accompanied by assessments using patient-reported outcome (PRO) instruments. We provide an overview of patient-reported outcome concepts in COPD, such as breathlessness, physical functioning, and health status, and evaluate the tools used for measuring these concepts. Particular attention is given to the newly developed instruments emerging in response to recent regulatory guidelines for the development and use of PROs in clinical trials. We conclude that although data from the development and validation of these new PRO instruments are emerging, to build the body of evidence that supports the use of a new instrument takes many years. Furthermore, new instruments do not necessarily have better discriminative or evaluative properties than older instruments. The development of new PRO tools, however, is crucial, not only to ensure that key COPD concepts are being reliably measured but also that the relevant treatment effects are being captured in clinical trials. In turn, this will help us to understand better the patient’s experience of the disease. PMID:23093901

  1. Toward Ensuring Health Equity: Readability and Cultural Equivalence of OMERACT Patient-reported Outcome Measures

    PubMed Central

    Petkovic, Jennifer; Epstein, Jonathan; Buchbinder, Rachelle; Welch, Vivian; Rader, Tamara; Lyddiatt, Anne; Clerehan, Rosemary; Christensen, Robin; Boonen, Annelies; Goel, Niti; Maxwell, Lara J.; Toupin-April, Karine; De Wit, Maarten; Barton, Jennifer; Flurey, Caroline; Jull, Janet; Barnabe, Cheryl; Sreih, Antoine G.; Campbell, Willemina; Pohl, Christoph; Duruöz, Mehmet Tuncay; Singh, Jasvinder A.; Tugwell, Peter S.; Guillemin, Francis

    2016-01-01

    Objective The goal of the Outcome Measures in Rheumatology (OMERACT) 12 (2014) equity working group was to determine whether and how comprehensibility of patient-reported outcome measures (PROM) should be assessed, to ensure suitability for people with low literacy and differing cultures. Methods The English, Dutch, French, and Turkish Health Assessment Questionnaires and English and French Osteoarthritis Knee and Hip Quality of Life questionnaires were evaluated by applying 3 readability formulas: Flesch Reading Ease, Flesch-Kincaid grade level, and Simple Measure of Gobbledygook; and a new tool, the Evaluative Linguistic Framework for Questionnaires, developed to assess text quality of questionnaires. We also considered a study assessing cross-cultural adaptation with/without back-translation and/or expert committee. The results of this preconference work were presented to the equity working group participants to gain their perspectives on the importance of comprehensibility and cross-cultural adaptation for PROM. Results Thirty-one OMERACT delegates attended the equity session. Twenty-six participants agreed that PROM should be assessed for comprehensibility and for use of suitable methods (4 abstained, 1 no). Twenty-two participants agreed that cultural equivalency of PROM should be assessed and suitable methods used (7 abstained, 2 no). Special interest group participants identified challenges with cross-cultural adaptation including resources required, and suggested patient involvement for improving translation and adaptation. Conclusion Future work will include consensus exercises on what methods are required to ensure PROM are appropriate for people with low literacy and different cultures. PMID:26077410

  2. An Introduction to Item Response Theory for Patient-Reported Outcome Measurement

    PubMed Central

    Nguyen, Tam H.; Han, Hae-Ra; Kim, Miyong T.

    2015-01-01

    The growing emphasis on patient-centered care has accelerated the demand for high-quality data from patient-reported outcome (PRO) measures. Traditionally, the development and validation of these measures has been guided by classical test theory. However, item response theory (IRT), an alternate measurement framework, offers promise for addressing practical measurement problems found in health-related research that have been difficult to solve through classical methods. This paper introduces foundational concepts in IRT, as well as commonly used models and their assumptions. Existing data on a combined sample (n = 636) of Korean American and Vietnamese American adults who responded to the High Blood Pressure Health Literacy Scale and the Patient Health Questionnaire-9 are used to exemplify typical applications of IRT. These examples illustrate how IRT can be used to improve the development, refinement, and evaluation of PRO measures. Greater use of methods based on this framework can increase the accuracy and efficiency with which PROs are measured. PMID:24403095

  3. An introduction to item response theory for patient-reported outcome measurement.

    PubMed

    Nguyen, Tam H; Han, Hae-Ra; Kim, Miyong T; Chan, Kitty S

    2014-01-01

    The growing emphasis on patient-centered care has accelerated the demand for high-quality data from patient-reported outcome (PRO) measures. Traditionally, the development and validation of these measures has been guided by classical test theory. However, item response theory (IRT), an alternate measurement framework, offers promise for addressing practical measurement problems found in health-related research that have been difficult to solve through classical methods. This paper introduces foundational concepts in IRT, as well as commonly used models and their assumptions. Existing data on a combined sample (n = 636) of Korean American and Vietnamese American adults who responded to the High Blood Pressure Health Literacy Scale and the Patient Health Questionnaire-9 are used to exemplify typical applications of IRT. These examples illustrate how IRT can be used to improve the development, refinement, and evaluation of PRO measures. Greater use of methods based on this framework can increase the accuracy and efficiency with which PROs are measured. PMID:24403095

  4. Interviewing to develop Patient-Reported Outcome (PRO) measures for clinical research: eliciting patients’ experience

    PubMed Central

    2014-01-01

    Patient-reported outcome (PRO) measures must provide evidence that their development followed a rigorous process for ensuring their content validity. To this end, the collection of data is performed through qualitative interviews that allow for the elicitation of in-depth spontaneous reports of the patients’ experiences with their condition and/or its treatment. This paper provides a review of qualitative research applied to PRO measure development. A clear definition of what is a qualitative research interview is given as well as information about the form and content of qualitative interviews required for developing PRO measures. Particular attention is paid to the description of interviewing approaches (e.g., semi-structured and in-depth interviews, individual vs. focus group interviews). Information about how to get prepared for a qualitative interview is provided with the description of how to develop discussion guides for exploratory or cognitive interviews. Interviewing patients to obtain knowledge regarding their illness experience requires interpersonal and communication skills to facilitate patients’ expression. Those skills are described in details, as well as the skills needed to facilitate focus groups and to interview children, adolescents and the elderly. Special attention is also given to quality assurance and interview training. The paper ends on ethical considerations since interviewing for the development of PROs is performed in a context of illness and vulnerability. Therefore, it is all the more important that, in addition to soliciting informed consent, respectful interactions be ensured throughout the interview process. PMID:24499454

  5. Patient-reported outcome measures in psoriasis: the good, the bad and the missing!

    PubMed

    Kitchen, H; Cordingley, L; Young, H; Griffiths, C E M; Bundy, C

    2015-01-01

    As a long-term condition, psoriasis demands significant personal and professional input for optimal self-management. Low levels of well-being and high levels of psychological distress in patients with psoriasis are associated with reduced resources for self-care. Patient-reported outcome (PRO) measures can be used to assess physical, social and psychological functioning in order to guide treatment. In this article, we systematically reviewed the development and validation of existing PRO measures. PubMed (Medline), PsycINFO and CINAHL were searched systematically using predefined search terms. The search was limited to articles in the English language relating to human subjects. Articles were selected for full review through explicit inclusion/exclusion criteria. PRO measures were critically reviewed in accordance with the published guidelines and theory on the development and validation of PROs. The search identified 967 abstracts; 71 of these articles met the criteria for full review. In these 71 articles, 45 PRO measures were found: 16 were specific to psoriasis, 21 assessed other dermatological conditions and eight were developed for generic nondermatological health conditions. The review revealed several limitations of the existing measures, including: (i) a composite structure assessing multiple, poorly-defined concepts; (ii) a lack of evidence for face and content validity; (iii) a failure to include both patient and clinician perspectives and requirements and (iv) a lack of evidence regarding the feasibility and acceptability for patients and physicians. No single PRO measure with adequate evidence of validity, reliability and sensitivity to change captures patient well-being in psoriasis. A valid, sensitive, specific and acceptable PRO that assesses the full impact of psoriasis on well-being is needed for the comprehensive clinical management of psoriasis. PMID:25677764

  6. Patient-reported outcome measures in nonmelanoma skin cancer of the face: a systematic review.

    PubMed

    Bates, A S; Davis, C R; Takwale, A; Knepil, G J

    2013-06-01

    Nonmelanoma skin cancer (NMSC) is the most common malignancy in the western world, with an incidence of 98,000 in the U.K. Since 2009 the Department of Health (DoH) has collected patient-reported outcome measure (PROM) data following four common surgical procedures. However, a DoH PROM for NMSC does not exist. A systematic review of questionnaires published on patient concerns due to NMSC of the face was conducted. Keywords relevant to PROMs, NMSC and the facial region were comprehensively searched in medical databases. Inclusion criteria stipulated that questionnaires from relevant papers recruited patients with NMSC for both the item formulation and subsequent validation. Questionnaires were then discussed by a multispecialty skin cancer research team. Initially 2548 papers were found; after exclusion criteria were applied, 73 articles were retrieved. Four patient questionnaires for NMSC featured adequate development and validation according to the inclusion criteria. The Facial Skin Cancer Index (FSCI) was the only PROM specific to facial NMSC. Additional questionnaires identified included the Skin Cancer Quality of Life Impact Tool, Skindex, and Dermatology Life Quality Index. There is a scarcity of data relating to NMSC PROMs and appearance concerns. Only one questionnaire--the FSCI--was specific to patients with facial NMSC. We recommend nationally standardized data collection from patients with NMSC in order to create an evidence-based validated PROM for patients with facial skin cancer. PMID:23387431

  7. Pragmatic Characteristics of Patient-Reported Outcome Measures are Important for Use in Clinical Practice

    PubMed Central

    Kroenke, Kurt; Monahan, Patrick O.; Kean, Jacob

    2015-01-01

    Objective Measures for assessing patient-reported outcomes (PROs) that may have initially been developed for research are increasingly being recommended for use in clinical practice as well. While psychometric rigor is essential, this paper focuses on pragmatic characteristics of PROs that may enhance uptake into clinical practice. Methods Three sources were drawn upon in identifying pragmatic criteria for PROs: 1) selected literature review including recommendations by other expert groups; 2) key features of several model public domain PROs; 3) the author' experience in developing practical PROs. Results Eight characteristics of a practical PRO include: 1) actionability (i.e., scores guide diagnostic or therapeutic actions/decision-making); 2) appropriateness for the relevant clinical setting; 3) universality (i.e., for screening, severity assessment, and monitoring across multiple conditions); 4) self-administration; 5) item features (number of items and bundling issues); 6) response options (option number and dimensions, uniform vs. varying options, timeframe, intervals between options); 7) scoring (simplicity, interpretability); and 8) accessibility (nonproprietary, downloadable, available in different languages and for vulnerable groups, incorporated into electronic health records) Conclusion Balancing psychometric and pragmatic factors in the development of PROs is important for accelerating the incorporation of PROs into clinical practice. PMID:25962972

  8. Use of an electronic patient-reported outcome measurement system to improve distress management in oncology

    PubMed Central

    Smith, Sophia K.; Rowe, Krista; Abernethy, Amy P.

    2014-01-01

    Objective Management of patient distress is a critical task in cancer nursing and cancer practice. Here we describe two examples of how an electronic patient-reported outcome (ePRO) measurement system implemented into routine oncology care can practically aid clinical and research tasks related to distress management. Methods Tablet personal computers were used to routinely complete a standardized ePRO review of systems surveys at point of care during every encounter in the Duke Oncology outpatient clinics. Two cases of use implementation are explored: (1) triaging distressed patients for optimal care, and (2) psychosocial program evaluation research. Results Between 2009 and 2011, the ePRO system was used to collect information during 17,338 Duke Oncology patient encounters. The system was used to monitor patients for psychosocial distress employing an electronic clinical decision support algorithm, with 1,952 (11.3%) referrals generated for supportive services. The system was utilized to examine the efficacy of a psychosocial care intervention documenting statistically significant improvements in distress, despair, fatigue, and quality of life (QOL) in 50 breast cancer patients. Significance of results ePRO solutions can guide best practice management of cancer patient distress. Nurses play a key role in implementation and utilization. PMID:24128592

  9. The case for an international patient-reported outcomes measurement information system (PROMIS®) initiative.

    PubMed

    Alonso, Jordi; Bartlett, Susan J; Rose, Matthias; Aaronson, Neil K; Chaplin, John E; Efficace, Fabio; Leplège, Alain; Lu, Aiping; Tulsky, David S; Raat, Hein; Ravens-Sieberer, Ulrike; Revicki, Dennis; Terwee, Caroline B; Valderas, Jose M; Cella, David; Forrest, Christopher B

    2013-01-01

    Patient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often resulting in improved measurement precision and responsiveness. Here we describe and discuss the case for developing an international core set of PROs building from the US PROMIS® network.PROMIS is a U.S.-based cooperative group of research sites and centers of excellence convened to develop and standardize PRO measures across studies and settings. If extended to a global collaboration, PROMIS has the potential to transform PRO measurement by creating a shared, unifying terminology and metric for reporting of common symptoms and functional life domains. Extending a common set of standardized PRO measures to the international community offers great potential for improving patient-centered research, clinical trials reporting, population monitoring, and health care worldwide. Benefits of such standardization include the possibility of: international syntheses (such as meta-analyses) of research findings; international population monitoring and policy development; health services administrators and planners access to relevant information on the populations they serve; better assessment and monitoring of patients by providers; and improved shared decision making.The goal of the current PROMIS International initiative is to ensure that item banks are translated and culturally adapted for use in adults and children in as many countries as possible. The process includes 3 key steps: translation/cultural adaptation, calibration, and validation. A universal translation, an approach focusing on commonalities, rather than differences across versions developed in regions or countries speaking the same language, is proposed to ensure conceptual equivalence for all items. International item

  10. Patient-Reported Outcome Measures in Safety Event Reporting: PROSPER Consortium guidance.

    PubMed

    Banerjee, Anjan K; Okun, Sally; Edwards, I Ralph; Wicks, Paul; Smith, Meredith Y; Mayall, Stephen J; Flamion, Bruno; Cleeland, Charles; Basch, Ethan

    2013-12-01

    The Patient-Reported Outcomes Safety Event Reporting (PROSPER) Consortium was convened to improve safety reporting by better incorporating the perspective of the patient. PROSPER comprises industry, regulatory authority, academic, private sector and patient representatives who are interested in the area of patient-reported outcomes of adverse events (PRO-AEs). It has developed guidance on PRO-AE data, including the benefits of wider use and approaches for data capture and analysis. Patient-reported outcomes (PROs) encompass the full range of self-reporting, rather than only patient reports collected by clinicians using validated instruments. In recent years, PROs have become increasingly important across the spectrum of healthcare and life sciences. Patient-centred models of care are integrating shared decision making and PROs at the point of care; comparative effectiveness research seeks to include patients as participatory stakeholders; and industry is expanding its involvement with patients and patient groups as part of the drug development process and safety monitoring. Additionally, recent pharmacovigilance legislation from regulatory authorities in the EU and the USA calls for the inclusion of patient-reported information in benefit-risk assessment of pharmaceutical products. For patients, technological advancements have made it easier to be an active participant in one's healthcare. Simplified internet search capabilities, electronic and personal health records, digital mobile devices, and PRO-enabled patient online communities are just a few examples of tools that allow patients to gain increased knowledge about conditions, symptoms, treatment options and side effects. Despite these changes and increased attention on the perceived value of PROs, their full potential has yet to be realised in pharmacovigilance. Current safety reporting and risk assessment processes remain heavily dependent on healthcare professionals, though there are known limitations such

  11. Item Banks for Measuring Emotional Distress From the Patient-Reported Outcomes Measurement Information System (PROMIS®): Depression, Anxiety, and Anger

    PubMed Central

    Pilkonis, Paul A.; Choi, Seung W.; Reise, Steven P.; Stover, Angela M.; Riley, William T.; Cella, David

    2011-01-01

    The authors report on the development and calibration of item banks for depression, anxiety, and anger as part of the Patient-Reported Outcomes Measurement Information System (PROMIS®). Comprehensive literature searches yielded an initial bank of 1,404 items from 305 instruments. After qualitative item analysis (including focus groups and cognitive interviewing), 168 items (56 for each construct) were written in a first person, past tense format with a 7-day time frame and five response options reflecting frequency. The calibration sample included nearly 15,000 respondents. Final banks of 28, 29, and 29 items were calibrated for depression, anxiety, and anger, respectively, using item response theory. Test information curves showed that the PROMIS item banks provided more information than conventional measures in a range of severity from approximately −1 to +3 standard deviations (with higher scores indicating greater distress). Short forms consisting of seven to eight items provided information comparable to legacy measures containing more items. PMID:21697139

  12. Application of the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS®) to Mental Health Research

    PubMed Central

    Riley, William T.; Pilkonis, Paul; Cella, David

    2013-01-01

    Background The Patient-Reported Outcomes Measurement Information System (PROMIS) is a National Institutes of Health initiative to develop item banks measuring patient-reported outcomes (PROs) and to create and make available a computerized adaptive testing system (CAT) that allows for efficient and precise assessment of PROs in clinical research and practice. Aims of the Study Based on the presentation from a symposium on “Evidence-based Outcomes in Psychiatry: Updates on Measurement Using Patient-Reported Outcomes (PRO)” at the 2011 American Psychiatry Association Convention, this paper provides an overview of PROMIS and its application to mental health research. Methods The PROMIS methodology for item bank development and testing is described, with a focus on the implications of this work for mental health research. Results Utilizing qualitative item review and state-of-the-art applications of item response theory (IRT), PROMIS investigators have developed, tested, and released item banks measuring physical, mental, and social health components. Ongoing efforts continue to add new item banks and further validate existing banks. Discussion PROMIS provides item banks measuring several domains of interest to mental health researchers including emotional distress, social function, and sleep. PROMIS methodology also provides a rigorous standard for the development of new mental health measures. Implications for Health Care Provision Web-based CAT or administration of short forms derived from PROMIS item banks provide efficient and precise dimensional estimates of clinical outcomes that can be utilized to monitor patient progress and assess quality improvement. Implications for Future Research Use of the dimensional PROMIS metrics (and co-calibration of the PROMIS item banks with existing PROs) will allow comparisons of mental health and related health outcomes across disorders and studies. PMID:22345362

  13. Measurement properties of patient-reported outcome measures (PROMs) in adults with obstructive sleep apnea (OSA): A systematic review.

    PubMed

    Abma, Inger L; van der Wees, Philip J; Veer, Vik; Westert, Gert P; Rovers, Maroeska

    2016-08-01

    This systematic review summarizes the evidence regarding the quality of patient-reported outcome measures (PROMs) validated in patients with obstructive sleep apnea (OSA). We performed a systematic literature search of all PROMs validated in patients with OSA, and found 22 measures meeting our inclusion criteria. The quality of the studies was assessed using the consensus-based standards for the selection of health status measurement instruments (COSMIN) checklist. The results showed that most of the measurement properties of the PROMs were not, or not adequately, assessed. For many identified PROMs there was no involvement of patients with OSA during their development or before the PROM was tested in patients with OSA. Positive exceptions and the best current candidates for assessing health status in patients with OSA are the sleep apnea quality of life index (SAQLI), Maugeri obstructive sleep apnea syndrome (MOSAS) questionnaire, Quebec sleep questionnaire (QSQ) and the obstructive sleep apnea patient-oriented severity index (OSAPOSI). Even though there is not enough evidence to fully judge the quality of these PROMs as outcome measure, when interpreted with caution, they have the potential to add value to clinical research and clinical practice in evaluating aspects of health status that are important to patients. PMID:26433776

  14. Systematic review of measurement properties of patient-reported outcome measures used in patients undergoing hip and knee arthroplasty

    PubMed Central

    Harris, Kristina; Dawson, Jill; Gibbons, Elizabeth; Lim, Chris R; Beard, David J; Fitzpatrick, Raymond; Price, Andrew J

    2016-01-01

    Objectives To identify patient-reported outcome measures (PROMs) that have been developed and/or used with patients undergoing hip or knee replacement surgery and to provide a shortlist of the most promising generic and condition-specific instruments. Methods A systematic review of the literature was performed to identify measures used in patients undergoing hip and knee replacement and extract and evaluate information on their methodological quality. Results Thirty-two shortlisted measures were reviewed for the quality of their measurement properties. On the basis of the review criteria, the measures with most complete evidence to date are the Oxford Hip Score (OHS) (for patients undergoing hip replacement surgery) and the Oxford Knee Score (OKS), with OKS-Activity and Participation Questionnaire (for patients undergoing knee replacement surgery). Conclusion A large number of these instruments lack essential evidence of their measurement properties (eg, validity, reliability, and responsiveness) in specific populations of patients. Further research is required on almost all of the identified measures. The best-performing condition-specific PROMs were the OKS, OHS, and Western Ontario and McMaster Universities Osteoarthritis Index. The best-performing generic measure was the Short Form 12. Researchers can use the information presented in this review to inform further psychometric studies of the reviewed measures. PMID:27524925

  15. TBI-QOL: Development and Calibration of Item Banks to Measure Patient Reported Outcomes Following Traumatic Brain Injury

    PubMed Central

    Tulsky, David S.; Kisala, Pamela A.; Victorson, David; Carlozzi, Noelle; Bushnik, Tamara; Sherer, Mark; Choi, Seung W.; Heinemann, Allen W.; Chiaravalloti, Nancy; Sander, Angelle M.; Englander, Jeffrey; Hanks, Robin; Kolakowsky-Hayner, Stephanie; Roth, Elliot; Gershon, Richard; Rosenthal, Mitchell; Cella, David

    2016-01-01

    Objective: To use a patient-centered approach or participatory action research design combined with advanced psychometrics to develop a comprehensive patient-reported outcomes (PRO) measurement system specifically for individuals with traumatic brain injury (TBI). This TBI Quality-of-Life (TBI-QOL) measurement system expands the work of other large PRO measurement initiatives, that is, the Patient-Reported Outcomes Measurement Information System and the Neurology Quality-of-Life measurement initiative. Setting: Five TBI Model Systems centers across the United States. Participants: Adults with TBI. Design: Classical and modern test development methodologies were used. Qualitative input was obtained from individuals with TBI, TBI clinicians, and caregivers of individuals with TBI through multiple methods, including focus groups, individual interviews, patient consultation, and cognitive debriefing interviews. Item pools were field tested in a large multisite sample (n = 675) and calibrated using item response theory methods. Main Outcomes Measures: Twenty-two TBI-QOL item banks/scales. Results: The TBI-QOL consists of 20 independent calibrated item banks and 2 uncalibrated scales that measure physical, emotional, cognitive, and social aspects of health-related quality of life. Conclusions: The TBI-QOL measurement system has potential as a common data element in TBI research and to enhance collection of health-related quality-of-life and PRO data in rehabilitation research and clinical settings. PMID:25931184

  16. Day-to-day measurement of patient-reported outcomes in exacerbations of chronic obstructive pulmonary disease

    PubMed Central

    Kocks, Jan Willem H; van den Berg, Jan Willem K; Kerstjens, Huib AM; Uil, Steven M; Vonk, Judith M; de Jong, Ynze P; Tsiligianni, Ioanna G; van der Molen, Thys

    2013-01-01

    Background Exacerbations of chronic obstructive pulmonary disease (COPD) are a major burden to patients and to society. Little is known about the possible role of day-to-day patient-reported outcomes during an exacerbation. This study aims to describe the day-to-day course of patient-reported health status during exacerbations of COPD and to assess its value in predicting clinical outcomes. Methods Data from two randomized controlled COPD exacerbation trials (n = 210 and n = 45 patients) were used to describe both the feasibility of daily collection of and the day-to-day course of patient-reported outcomes during outpatient treatment or admission to hospital. In addition to clinical parameters, the BORG dyspnea score, the Clinical COPD Questionnaire (CCQ), and the St George’s Respiratory Questionnaire were used in Cox regression models to predict treatment failure, time to next exacerbation, and mortality in the hospital study. Results All patient-reported outcomes showed a distinct pattern of improvement. In the multivariate models, absence of improvement in CCQ symptom score and impaired lung function were independent predictors of treatment failure. Health status and gender predicted time to next exacerbation. Five-year mortality was predicted by age, forced expiratory flow in one second % predicted, smoking status, and CCQ score. In outpatient management of exacerbations, health status was found to be less impaired than in hospitalized patients, while the rate and pattern of recovery was remarkably similar. Conclusion Daily health status measurements were found to predict treatment failure, which could help decision-making for patients hospitalized due to an exacerbation of COPD. PMID:23766644

  17. The VVSymQ® instrument: Use of a new patient-reported outcome measure for assessment of varicose vein symptoms

    PubMed Central

    Turner-Bowker, Diane M; Elash, Celeste A; Wright, David

    2015-01-01

    Introduction No existing patient-reported outcome instrument focuses solely on assessment of varicose veins symptoms that are bothersome to patients. Methods The VVSymQ® instrument is a five-item patient-reported outcome that assesses symptoms most important to patients with varicose veins (heaviness, achiness, swelling, throbbing and itching). This paper describes how the VVSymQ® instrument was incorporated into an electronic daily diary to monitor key outcomes over time and capture treatment benefit in two randomized, controlled, phase 3 clinical trials. Results Patients were highly compliant in completing the electronic daily diary, and the VVSymQ® instrument demonstrated ability to detect overall change and ability to detect change that is meaningful to patients. Conclusion The VVSymQ® instrument is a reliable, valid instrument responsive to measuring change in the patient experience of varicose vein symptoms pre- and post-intervention, and is uniquely focused on patient-reported symptoms compared with other widely used questionnaires completed by clinicians. PMID:26183669

  18. Using Patient-Reported Outcome Measures to Capture the Patient's Voice in Research and Care of Juvenile Idiopathic Arthritis.

    PubMed

    Hersh, Aimee O; Salimian, Parissa K; Weitzman, Elissa R

    2016-05-01

    Patient-reported outcome (PRO) measures provide a valuable window into how patients with juvenile idiopathic arthritis and their parents perceive their functioning, quality of life, and medication side effects in the context of their disease and treatment. Momentum behind adoption of PRO measures is increasing as these patient-relevant tools capture information pertinent to taking a patient-centered approach to health care and research. This article reviews the clinical and research utility of obtaining PROs across domains applicable to the experience of juvenile idiopathic arthritis and summarizes available self-report and parent-proxy PRO measures. Current challenges and limitations of PRO usage are discussed. PMID:27133493

  19. Patient-Reported Outcomes in Rheumatoid Arthritis.

    PubMed

    van Tuyl, Lilian H D; Michaud, Kaleb

    2016-05-01

    Patient-reported outcomes (PROs) and their measures have a long and important history for determining the status and treatment of patients with rheumatoid arthritis (RA). This article describes the history and evolution of PROs for RA and the current state of the field, with key examples of accepted and widely used measures, and offers some reflection on the roles of PROs for the study and management of RA. PMID:27133486

  20. A Qualitative Exploration of Patient and Clinician Views on Patient Reported Outcome Measures in Child Mental Health and Diabetes Services.

    PubMed

    Wolpert, Miranda; Curtis-Tyler, Katherine; Edbrooke-Childs, Julian

    2016-05-01

    Patient Reported Outcome Measures (PROMs) are increasingly being recommended for use in both mental and physical health services. The present study is a qualitative exploration of the views of young people, mothers, and clinicians on PROMs. Semi-structured interviews were conducted with a sample of n = 10 participants (6 young people, 4 clinicians) from mental health services and n = 14 participants (4 young people, 7 mothers, 3 clinicians) from a diabetes service. For different reasons, young people, mothers, and clinicians saw feedback from PROMs as having the potential to alter the scope of clinical discussions. PMID:25179754

  1. Patient-reported Outcomes in Cystic Fibrosis

    PubMed Central

    Goss, Christopher H.; Quittner, Alexandra L.

    2007-01-01

    Over the past 20 years, there has been tremendous progress in the area of patient-reported outcomes (PROs). A PRO instrument is defined as any measure of a patient's health status that is elicited directly from the patient and assesses how the patient “feels or functions with respect to his or her health condition.” The advances seen in clinical research regarding PROs has been mirrored in research in cystic fibrosis (CF). A large number of instruments have been used for both therapeutic and nontherapeutic clinical research for many chronic conditions. This review will summarize a history of the development of PROs and how PROs are viewed by the U.S. Food and Drug Administration. We will then review the current state of the art of patient-reported outcomes in CF, specifically addressing the evaluation of different PRO instruments in terms of their reliability and validity. Finally, we will delineate further areas for development of PROs in CF. We believe that the future of CF research will incorporate a more diverse selection of PRO outcome measures; these outcome measures ultimately may be incorporated into clinical care to standardize symptom assessment and provide information regarding the need for specific clinical interventions to improve the quality of care delivered to these patients. PMID:17652505

  2. Item Banks for Measuring Emotional Distress from the Patient-Reported Outcomes Measurement Information System (PROMIS[R]): Depression, Anxiety, and Anger

    ERIC Educational Resources Information Center

    Pilkonis, Paul A.; Choi, Seung W.; Reise, Steven P.; Stover, Angela M.; Riley, William T.; Cella, David

    2011-01-01

    The authors report on the development and calibration of item banks for depression, anxiety, and anger as part of the Patient-Reported Outcomes Measurement Information System (PROMIS[R]). Comprehensive literature searches yielded an initial bank of 1,404 items from 305 instruments. After qualitative item analysis (including focus groups and…

  3. Patient-Reported Outcomes in Psoriatic Arthritis.

    PubMed

    Orbai, Ana-Maria; Ogdie, Alexis

    2016-05-01

    Patient-reported outcome (PRO) measures are an important component to assessing disease impact and therapy response in patients with psoriatic arthritis (PsA). Overall, there are few PsA-specific PROs. Most PROs used in PsA are borrowed from other diseases (eg, rheumatoid arthritis and ankylosing spondylitis) or general population PROs. PROs are used in PsA clinical trials and in the clinical management of PsA. In this review, we discuss the most commonly used PRO in PsA, including their inclusion in composite measures. Future studies may be helpful to determine the best performing PROs in patients with PsA. PMID:27133489

  4. U.S. Dental School Deans' Views on the Value of Patient-Reported Outcome Measures in Dentistry.

    PubMed

    Rosen, Evan B; Donoff, R Bruce; Riedy, Christine A

    2016-06-01

    There has historically been limited development and utilization of patient-reported outcome measures (PROMs) in clinical dentistry. However, in recent years PROMs have been recognized by other health care fields as valuable in the comprehensive assessment of patient outcomes. The aim of this study was to survey deans of U.S. dental schools to better understand their vision for the role of PROMs in the field of dentistry. A 13-question online survey was emailed to the deans of the 64 accredited U.S. dental schools at the time to gather their opinions about the value of patient-reported outcomes in dentistry. The survey consisted of questions in 12 domains such as treatment planning, perceived success/complications of surgery, identification/management of dental pain, psychological and oral function, and insurance payment/reimbursement. Of the 64 deans, 33 responses were received (51.5% response rate), but three surveys were excluded due to incomplete answers, resulting in a final response rate of 46.8%. All respondents reported there was value in utilization of PROMs for understanding a patient's satisfaction of a procedure, a patient's perceived success of dental surgery, identifying dental pain, and managing dental pain. However, there was disagreement among the respondents about utilization of PROMs for the purpose of determining insurance payment and/or reimbursement. Additional steps should be taken to develop clinically appropriate PROMs for dentistry and to determine the appropriate situations in which to use dental PROMs. This study suggests that PROMs should be incorporated into dental school curricula as they will likely play a role in future comprehensive treatment assessment. PMID:27251354

  5. Patient-Reported Outcomes Measurement Information System (PROMIS): efficient, standardized tools to measure self-reported health and quality of life.

    PubMed

    Bevans, Margaret; Ross, Alyson; Cella, David

    2014-01-01

    All nurses are interested in the effects of diseases and treatments on individuals. Patient-reported outcome (PRO) measures are used to obtain self-reported information about symptoms, function, perceptions, and experiences. However, there are challenges to their use, including multiple measures of the same concept, widely varying quality, excessive length and complexity, and difficulty comparing findings across studies and conditions. To address these challenges, the National Institutes of Health funded the Patient-Reported Outcomes Measurement Information System (PROMIS), a web-based repository of valid and reliable PRO measures of health concepts relevant to clinician and researchers. Through the PROMIS Assessment Center, clinicians and researchers can access PRO measures, administer computerized adaptive tests, collect self-report data, and report instant health assessments. The purpose of this article was to summarize the development and validation of the PROMIS measures and to describe its current functionality as it relates to nursing science. PMID:25015409

  6. Physiotherapists use of and perspectives on the importance of patient-reported outcome measures for shoulder dysfunction

    PubMed Central

    Michener, Lori A

    2014-01-01

    Background Many patient-reported outcome measures (PROs) for shoulder dysfunctions have acceptable psychometric properties. The present study examined current PRO usage and perceived importance. Methods Delegates at the 2010 International Congress of Shoulder and Elbow Therapists were invited to participate in this cross-sectional observational study. Research Electronic Data Capture (REDCap) web-based tools were used to design an online questionnaire, which was e-mailed to participants. Results Participants (n = 101) reflected an 84% response rate. PRO use was considered ‘extremely’ or ‘very’ important by the majority of clinicians (76%) and researchers (98%). Most commonly used as a primary outcome by clinicians and researchers, respectively, were the Disabilities of the Arm Shoulder Hand Questionnaire (DASH) (40%, 44%) and the Oxford Shoulder Scale (OSS) (36%, 22%) and, as secondary outcomes, the DASH (33%, 28%), OSS (17%, 8%), the Shoulder Pain and Disability Index (SPADI) (8%,18%), and American Shoulder and Elbow Surgeons standardized assessment form (ASES) (8%, 13%). Psychometric properties were rated as ‘extremely’ or ‘very’ important by 86% to 96% of participants. Conclusions The majority of shoulder therapists consider PRO use to be very important and psychometric properties to be critical in PRO selection. The DASH, OSS, SPADI and ASES are most commonly used in clinical practice and research studies.

  7. Development of an Online Library of Patient-Reported Outcome Measures in Gastroenterology: The GI-PRO Database

    PubMed Central

    Khanna, Puja; Agarwal, Nikhil; Khanna, Dinesh; Hays, Ron D.; Chang, Lin; Bolus, Roger; Melmed, Gil; Whitman, Cynthia B.; Kaplan, Robert M.; Ogawa, Rikke; Snyder, Bradley; Spiegel, Brennan M.R.

    2014-01-01

    OBJECTIVES Because gastrointestinal (GI) illnesses can cause physical, emotional, and social distress, patient-reported outcomes (PROs) are used to guide clinical decision making, conduct research, and seek drug approval. It is important to develop a mechanism for identifying, categorizing, and evaluating the over 100 GI PROs that exist. Here we describe a new, National Institutes of Health (NIH)-supported, online PRO clearinghouse—the GI-PRO database. METHODS Using a protocol developed by the NIH Patient-Reported Outcome Measurement Information System (PROMIS®), we performed a systematic review to identify English-language GI PROs. We abstracted PRO items and developed an online searchable item database. We categorized symptoms into content “bins” to evaluate a framework for GI symptom reporting. Finally, we assigned a score for the methodological quality of each PRO represented in the published literature (0–20 range; higher indicates better). RESULTS We reviewed 15,697 titles (κ > 0.6 for title and abstract selection), from which we identified 126 PROs. Review of the PROs revealed eight GI symptom “bins”: (i) abdominal pain, (ii) bloat/gas, (iii) diarrhea, (iv) constipation, (v) bowel incontinence/soilage, (vi) heartburn/reflux, (vii) swallowing, and (viii) nausea/vomiting. In addition to these symptoms, the PROs covered four psychosocial domains: (i) behaviors, (ii) cognitions, (iii) emotions, and (iv) psychosocial impact. The quality scores were generally low (mean 8.88±4.19; 0 (min)−20 (max)). In addition, 51% did not include patient input in developing the PRO, and 41% provided no information on score interpretation. CONCLUSIONS GI PROs cover a wide range of biopsychosocial symptoms. Although plentiful, GI PROs are limited by low methodological quality. Our online PRO library (www.researchcore.org/gipro/) can help in selecting PROs for clinical and research purposes. PMID:24343547

  8. Use of patient-reported outcomes to measure symptoms and health related quality of life in the clinic.

    PubMed

    Gilbert, Alexandra; Sebag-Montefiore, David; Davidson, Susan; Velikova, Galina

    2015-03-01

    There is increasing interest in the use of patient-reported outcomes (PROs) in routine practice in cancer care to measure symptoms and health related quality of life (HRQOL). PROs are designed to capture the patient's perspective of their care and treatment, and complement the traditional clinical outcomes of survival and toxicity assessment. Integrating routine collection and feedback of PROs has been found to improve care for patients on both an individual level, through improved communication and management of symptoms, and at an organizational level, by enabling aggregation of data to compare performance. This article reviews the benefits and challenges of introducing patient-reported assessments into routine clinical practice. Methods for choosing a questionnaire; collection and presentation of results; timing and frequency of administration as well as clinician training methods to aid the ability of clinicians to integrate the use of PROs into their own practice are described. Electronic PRO capture and integration with electronic health records seems to provide the most effective method for seamless integration into existing patient care pathways. Case studies from our own practice illustrate the issues raised. Electronic methods enabling immediate collection, scoring and interpretation of the data, as well as real-time data capture, email alert systems and individualized, online self-management advice may enable severe symptoms to be managed in a more timely manner. Evaluation methods are described to establish the effectiveness of the PRO intervention. Engaging stakeholders throughout the process of initial consultation and development, during delivery and evaluation is key to success. Future work needs to focus on the effectiveness of PROs in longer-term follow-up of patients in routine care and the relationship between the PRO severity grading and clinician severity grading using the Common Terminology Criteria of Adverse Events (CTCAE). PMID:25448486

  9. Patient-reported outcomes in multiple sclerosis: Relationships among existing scales and the development of a brief measure.

    PubMed

    Chua, Alicia S; Glanz, Bonnie I; Guarino, Anthony J; Cook, Sandra L; Greeke, Emily E; Little, Grace E; Chitnis, Tanuja; Healy, Brian C

    2015-11-01

    Several patient-reported outcome (PRO) measures are commonly used in multiple sclerosis (MS) research, but the relationship among items across measures is uncertain. We proposed to evaluate the associations between items from a standard battery of PRO measures used in MS research and to develop a brief, reliable and valid instrument measure by combining these items into a single measure. Subjects (N = 537) enrolled in CLIMB complete a PRO battery that includes the Center for Epidemiologic Studies Depression Scale, Medical Outcomes Study Modified Social Support Survey, Modified Fatigue Impact Scale and Multiple Sclerosis Quality of Life-54. Subjects were randomly divided into two samples: calibration (n = 269) and validation (n = 268). In the calibration sample, an Exploratory Factor Analysis (EFA) was used to identify latent constructs within the battery. The model constructed based on the EFA was evaluated in the validation sample using Confirmatory Factor Analysis (CFA), and reliability and validity were assessed for the final measure. The EFA in the calibration sample revealed an eight factor solution, and a final model with one second-order factor along with the eight first-order factors provided the best fit. The model combined items from each of the four parent measures, showing important relationships among the parent measures. When the model was fit using the validation sample, the results confirmed the validity and reliability of the model. A brief PRO for MS (BPRO-MS) that combines MS-related psychosocial and quality of life domains can be used to assess overall functioning in mildly disabled MS patients. PMID:26590669

  10. The Cervical Dystonia Impact Profile (CDIP-58): Can a Rasch developed patient reported outcome measure satisfy traditional psychometric criteria?

    PubMed Central

    Cano, Stefan J; Warner, Thomas T; Thompson, Alan J; Bhatia, Kailash P; Fitzpatrick, Ray; Hobart, Jeremy C

    2008-01-01

    Background The United States Food and Drug Administration (FDA) are currently producing guidelines for the scientific adequacy of patient reported outcome measures (PROMs) in clinical trials, which will have implications for the selection of scales used in future clinical trials. In this study, we examine how the Cervical Dystonia Impact Profile (CDIP-58), a rigorous Rasch measurement developed neurologic PROM, stands up to traditional psychometric criteria for three reasons: 1) provide traditional psychometric evidence for the CDIP-58 in line with proposed FDA guidelines; 2) enable researchers and clinicians to compare it with existing dystonia PROMs; and 3) help researchers and clinicians bridge the knowledge gap between old and new methods of reliability and validity testing. Methods We evaluated traditional psychometric properties of data quality, scaling assumptions, targeting, reliability and validity in a group of 391 people with CD. The main outcome measures used were the CDIP-58, Medical Outcome Study Short Form-36, the 28-item General Health Questionnaire, and Hospital and Anxiety and Depression Scale. Results A total of 391 people returned completed questionnaires (corrected response rate 87%). Analyses showed: 1) data quality was high (low missing data ≤ 4%, subscale scores could be computed for > 96% of the sample); 2) item groupings passed tests for scaling assumptions; 3) good targeting (except for the Sleep subscale, ceiling effect = 27%); 4) good reliability (Cronbach's alpha ≥ 0.92, test-retest intraclass correlations ≥ 0.83); and 5) validity was supported. Conclusion This study has shown that new psychometric methods can produce a PROM that stands up to traditional criteria and supports the clinical advantages of Rasch analysis. PMID:18684327

  11. Use of SMS and tablet computer improves the electronic collection of elective orthopaedic patient reported outcome measures

    PubMed Central

    Bradley, B; Williams, D

    2014-01-01

    Introduction Electronic patient reported outcome measures (PROMs) enable real time reporting back to the patient and medical team, comparison between similar patient cohorts and long-term cost effective outcome measurement. The primary objective of this three-phase pilot study was to measure uptake using a web-based PROM system following the introduction of two separate process improvements. Methods Eighty consecutive new elective orthopaedic patients in a single surgeon’s practice were recruited for the study. Patients in Group 1 (n=26) received only a letter reminding them to complete a symptom score. Those in Group 2 (n=31) also received a reminder SMS (short message service) message via their mobile or home telephone and those in Group 3 (n=23) also had access to a tablet computer in clinic. Results The mean patient age in Group 1 was 55 years (range: 24–80 years), in Group 2 it was 60 years (range: 23–85 years) and in Group 3 it was 58 years (range: 37–78 years) (p>0.05). Overall, 79% of patients had internet access, and 35% of Group 1, 55% of Group 2 and 74% of Group 3 recorded an electronic PROM score (p=0.02). In Group 3, 94% of patients listed for an operation completed an electronic PROM score (p=0.006). Conclusions Collecting PROM data effectively in everyday clinical practice is challenging. Electronic collection should meet that challenge and improve healthcare delivery but it is in its infancy. This pilot study shows that the combination of an SMS reminder and access to a Wi-Fi enabled tablet computer in the clinic setting enabled 94% of patients listed for an operation to complete a score on a web-based clinical outcomes system. Additional staff training and telephone call reminders may further improve uptake. PMID:24992417

  12. The effect of surgical factors on early patient-reported outcome measures (PROMS) following total knee replacement.

    PubMed

    Baker, P N; Deehan, D J; Lees, D; Jameson, S; Avery, P J; Gregg, P J; Reed, M R

    2012-08-01

    Patient-reported outcome measures (PROMs) are increasingly being used to assess functional outcome and patient satisfaction. They provide a framework for comparisons between surgical units, and individual surgeons for benchmarking and financial remuneration. Better performance may bring the reward of more customers as patients and commissioners seek out high performers for their elective procedures. Using National Joint Registry (NJR) data linked to PROMs we identified 22,691 primary total knee replacements (TKRs) undertaken for osteoarthritis in England and Wales between August 2008 and February 2011, and identified the surgical factors that influenced the improvements in the Oxford knee score (OKS) and EuroQol-5D (EQ-5D) assessment using multiple regression analysis. After correction for patient factors the only surgical factors that influenced PROMs were implant brand and hospital type (both p < 0.001). However, the effects of surgical factors upon the PROMs were modest compared with patient factors. For both the OKS and the EQ-5D the most important factors influencing the improvement in PROMs were the corresponding pre-operative score and the patient's general health status. Despite having only a small effect on PROMs, this study has shown that both implant brand and hospital type do influence reported subjective functional scores following TKR. In the current climate of financial austerity, proposed performance-based remuneration and wider patient choice, it would seem unwise to ignore these effects and the influence of a range of additional patient factors. PMID:22844046

  13. Minimally important differences for Patient Reported Outcomes Measurement Information System pain interference for individuals with back pain

    PubMed Central

    Amtmann, Dagmar; Kim, Jiseon; Chung, Hyewon; Askew, Robert L; Park, Ryoungsun; Cook, Karon F

    2016-01-01

    Background The minimally important difference (MID) refers to the smallest change that is sufficiently meaningful to carry implications for patients’ care. MIDs are necessary to guide the interpretation of scores. This study estimated MID for the Patient Reported Outcomes Measurement Information System (PROMIS) pain interference (PI). Methods Study instruments were administered to 414 people who participated in two studies that included treatment with low back pain (LBP; n=218) or depression (n=196). Participants with LBP received epidural steroid injections and participants with depression received antidepressants, psychotherapy, or both. MIDs were estimated for the changes in LBP. MIDs were included only if a priori criteria were met (ie, sample size ≥10, Spearman correlation ≥0.3 between anchor measures and PROMIS-PI scores, and effect size range =0.2–0.8). The interquartile range (IQR) of MID estimates was calculated. Results The IQR ranged from 3.5 to 5.5 points. The lower bound estimate of the IQR (3.5) was greater than mean of standard error of measurement (SEM) both at time 1 (SEM =2.3) and at time 2 (SEM =2.5), indicating that the estimate of MID exceeded measurement error. Conclusion Based on our results, researchers and clinicians using PROMIS-PI can assume that change of 3.5 to 5.5 points in comparisons of mean PROMIS-PI scores of people with LBP can be considered meaningful. PMID:27175093

  14. Construct validity of the Patient Reported Outcomes Measurement Information System (PROMIS®) Gastrointestinal Symptom Scales in Systemic Sclerosis

    PubMed Central

    Nagaraja, Vivek; Hays, Ron D.; Khanna, Puja P.; Spiegel, Brennan M.R.; Chang, Lin; Melmed, Gil Y.; Bolus, Roger; Khanna, Dinesh

    2014-01-01

    Objective Gastrointestinal (GI) involvement is common in patients with systemic sclerosis (SSc). The Patient-Reported Outcomes Measurement Information System (PROMIS®) GI Symptom item bank captures upper and lower GI symptoms (reflux, disrupted swallowing, nausea/vomiting, belly pain, gas /bloating /flatulence, diarrhea, constipation, and fecal incontinence). The objective of this study was to evaluate the construct validity of the PROMIS-GI bank in SSc. Methods 167 patients with SSc were administered the PROMIS GI bank and the UCLA Scleroderma Clinical Trials Consortium Gastrointestinal Scale (GIT 2.0) instrument. GIT 2.0 is a multi-item instrument that measures SSc-associated GI symptoms. Product-moment correlations and a multitrait-multimethod analysis of the PROMIS GI scales with the GIT 2.0 symptom scales were used to evaluate convergent and discriminant validity. Results Patients with SSc GI involvement had PROMIS GI scale scores 0.2–0.7 SD worse than US population. Correlations among scales measuring the same domains for the PROMIS GI and GIT 2.0 measures were large, ranging from 0.61 to 0.87 (average r = 0.77). The average correlation between different symptom scales was 0.22, supporting discriminant validity. Conclusion This study provides support for the construct validity of the PROMIS GI scales in SSc. Future research is needed to assess the responsiveness to change of these scales in patients with SSc. PMID:24692332

  15. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy.

    PubMed

    Vinik, Etta J; Vinik, Aaron I; Paulson, James F; Merkies, Ingemar S J; Packman, Jeff; Grogan, Donna R; Coelho, Teresa

    2014-06-01

    The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire is an instrument to assess QOL in diabetic polyneuropathy. The objective of this observational, cross-sectional study in 61 patients with V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) and 16 healthy volunteers was to validate the Norfolk QOL-DN for assessment of QOL in TTR-FAP. Comparisons were conducted to identify the best items to discriminate disease stages and assess which individual Norfolk domains (symptoms, large fiber, small fiber, autonomic, and activities of daily living) would be most affected by disease stage. Analysis of individual items revealed a significant pattern of discrimination among disease stages (p < 0.001). Total QOL scores increased (indicating worsening) with duration of symptoms, with a steeper increase observed earlier in the course of disease. Significant correlations were observed between each Norfolk domain and other measures of neurological function. Limitations include cross-sectional study design, low patient numbers in this rare disease, and the ordinal-based character of the metric used; future areas to explore include item response theory approaches such as Rasch analysis. These results suggest the Norfolk QOL-DN is a reliable indicator of the impact of disease severity on QOL in patients with TTR-FAP. PMID:24738700

  16. Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).

    PubMed

    Kluetz, Paul G; Chingos, Diana T; Basch, Ethan M; Mitchell, Sandra A

    2016-01-01

    Systematic capture of the patient perspective can inform the development of new cancer therapies. Patient-reported outcomes (PROs) are commonly included in cancer clinical trials; however, there is heterogeneity in the constructs, measures, and analytic approaches that have been used making these endpoints challenging to interpret. There is renewed effort to identify rigorous methods to obtain high-quality and informative PRO data from cancer clinical trials. In this setting, PROs are used to address specific research objectives, and an important objective that spans the product development life cycle is the assessment of safety and tolerability. The U.S. Food and Drug Administration's (FDA) Office of Hematology and Oncology Products (OHOP) has identified symptomatic adverse events (AEs) as a central PRO concept, and a systematic assessment of patient-reported symptomatic AEs can provide data to complement clinician reporting. The National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is being evaluated by multiple stakeholders, including the FDA, and is considered a promising tool to provide a standard yet flexible method to assess symptomatic AEs from the patient perspective. In this article, we briefly review the FDA OHOP's perspective on PROs in cancer trials submitted to the FDA and focus on the assessment of symptomatic AEs using PRO-CTCAE. We conclude by discussing further work that must be done to broaden the use of PRO-CTCAE as a method to provide patient-centered data that can complement existing safety and tolerability assessments across cancer clinical trials. PMID:27249687

  17. Patient-reported outcome measures in arthroplasty registries Report of the Patient-Reported Outcome Measures Working Group of the International Society of Arthroplasty Registries Part II. Recommendations for selection, administration, and analysis.

    PubMed

    Rolfson, Ola; Bohm, Eric; Franklin, Patricia; Lyman, Stephen; Denissen, Geke; Dawson, Jill; Dunn, Jennifer; Eresian Chenok, Kate; Dunbar, Michael; Overgaard, Søren; Garellick, Göran; Lübbeke, Anne

    2016-07-01

    - The International Society of Arthroplasty Registries (ISAR) Patient-Reported Outcome Measures (PROMs) Working Group have evaluated and recommended best practices in the selection, administration, and interpretation of PROMs for hip and knee arthroplasty registries. The 2 generic PROMs in common use are the Short Form health surveys (SF-36 or SF-12) and EuroQol 5-dimension (EQ-5D). The Working Group recommends that registries should choose specific PROMs that have been appropriately developed with good measurement properties for arthroplasty patients. The Working Group recommend the use of a 1-item pain question ("During the past 4 weeks, how would you describe the pain you usually have in your [right/left] [hip/knee]?"; response: none, very mild, mild, moderate, or severe) and a single-item satisfaction outcome ("How satisfied are you with your [right/left] [hip/knee] replacement?"; response: very unsatisfied, dissatisfied, neutral, satisfied, or very satisfied). Survey logistics include patient instructions, paper- and electronic-based data collection, reminders for follow-up, centralized as opposed to hospital-based follow-up, sample size, patient- or joint-specific evaluation, collection intervals, frequency of response, missing values, and factors in establishing a PROMs registry program. The Working Group recommends including age, sex, diagnosis at joint, general health status preoperatively, and joint pain and function score in case-mix adjustment models. Interpretation and statistical analysis should consider the absolute level of pain, function, and general health status as well as improvement, missing data, approaches to analysis and case-mix adjustment, minimal clinically important difference, and minimal detectable change. The Working Group recommends data collection immediately before and 1 year after surgery, a threshold of 60% for acceptable frequency of response, documentation of non-responders, and documentation of incomplete or missing data. PMID

  18. Validating English- and Spanish-language patient-reported outcome measures in underserved patients with rheumatic disease

    PubMed Central

    2011-01-01

    Introduction Rheumatic diseases are among the most common and debilitating health problems in the United States. These diseases are chronic, can result in severe decrements of physical and psychosocial functioning and affect patients' overall quality of life. A consensus regarding the best patient outcomes to be measured in randomized, controlled trials and prospective natural history studies is essential to provide best estimates of efficacy and safety of interventions across diverse patient populations. Methods Face-to-face English- and Spanish-language cognitive interviews were conducted among urban Hispanic and African American patients with rheumatic disease to develop a questionnaire booklet. Six measures validating patient-reported outcomes were included: the Arthritis Self-Efficacy Scale, the Stanford Health Assessment Questionnaire Disability Index, the Wong-Baker Faces Pain Scale, the Short Acculturation Scale, the Center for Epidemiologic Studies Depression Scale and the Inventory of Complementary and Alternative Medicine Practices. A sample of patients (n = 15) attending the National Institute of Arthritis and Musculoskeletal and Skin Diseases Community Health Center participated in the initial interviews. Revised measures were further tested for reliability in a separate sample of patients (n = 109) upon enrollment at the health center. Results Cognitive interviews provided feedback for questionnaire modifications and methods to enhance content validity and data quality, including discarding redundant questions, providing visual aids and concrete examples when appropriate and increasing the use of racially and ethnically concordant interviewers. The cognitive interviews further elucidated that some contextual assumptions and language usage in the original questionnaires may not have taken each respondent's environmental and sociocultural context into consideration. Internal reliability for previously tested measures remained high (Cronbach's α = 0

  19. Using the bootstrap to establish statistical significance for relative validity comparisons among patient-reported outcome measures

    PubMed Central

    2013-01-01

    Background Relative validity (RV), a ratio of ANOVA F-statistics, is often used to compare the validity of patient-reported outcome (PRO) measures. We used the bootstrap to establish the statistical significance of the RV and to identify key factors affecting its significance. Methods Based on responses from 453 chronic kidney disease (CKD) patients to 16 CKD-specific and generic PRO measures, RVs were computed to determine how well each measure discriminated across clinically-defined groups of patients compared to the most discriminating (reference) measure. Statistical significance of RV was quantified by the 95% bootstrap confidence interval. Simulations examined the effects of sample size, denominator F-statistic, correlation between comparator and reference measures, and number of bootstrap replicates. Results The statistical significance of the RV increased as the magnitude of denominator F-statistic increased or as the correlation between comparator and reference measures increased. A denominator F-statistic of 57 conveyed sufficient power (80%) to detect an RV of 0.6 for two measures correlated at r = 0.7. Larger denominator F-statistics or higher correlations provided greater power. Larger sample size with a fixed denominator F-statistic or more bootstrap replicates (beyond 500) had minimal impact. Conclusions The bootstrap is valuable for establishing the statistical significance of RV estimates. A reasonably large denominator F-statistic (F > 57) is required for adequate power when using the RV to compare the validity of measures with small or moderate correlations (r < 0.7). Substantially greater power can be achieved when comparing measures of a very high correlation (r > 0.9). PMID:23721463

  20. The level of association between functional performance status measures and patient-reported outcomes in cancer patients: a systematic review

    PubMed Central

    Andreotti, Charissa F.; Roberts, Kailey E.; Saracino, Rebecca M.; Hernandez, Marisol; Basch, Ethan

    2016-01-01

    Purpose The process of assessing patient symptoms and functionality using patient-reported outcomes (PROs) and functional performance status (FPS) is an essential aspect of patient-centered oncology research and care. However, PRO and FPS measures are often employed separately or inconsistently combined. Thus, the purpose of this study was to conduct a systematic review of the level of association between PRO and FPS measures to determine their differential or combined utility. Methods A systematic search was conducted using five databases (1966 to February 2014) to identify studies that described an association between PRO and FPS. Studies were excluded if they were non-cancer specific, did not include adults aged 18 or older, or were review articles. Publications were selected for review by consensus among two authors, with a third author arbitrating as needed. Results A total of 18 studies met inclusion criteria. FPS was primarily assessed by clinicians using the ECOG Performance Status or Karnofsky Performance Status measures. PROs were captured using a variety of measures, with numerous domains assessed (e.g., pain, fatigue, and general health status). Concordance between PROs and FPS measures was widely variable, falling in the low to moderate range (0.09–0.72). Conclusions Despite consistency in the method of capture of PROs or FPS, domain capture varied considerably across reviewed studies. Irrespective of the method of capturing PROs or FPS, the quantified level of association between these two areas was moderate at best, providing evidence that FPS and PRO assessments offer unique information to assist clinicians in their decision-making. PMID:26314706

  1. Cognitive Interviewing in the Evaluation of Fatigue Items: Results from the Patient-Reported Outcomes Measurement Information System (PROMIS)

    PubMed Central

    Christodoulou, Christopher; Junghaenel, Doerte U.; DeWalt, Darren A.; Rothrock, Nan; Stone, Arthur A.

    2010-01-01

    Objectives Cognitive Interviewing (CI) is a technique increasingly used to obtain respondent feedback on potential items during questionnaire development. No standard guidelines exist by which to incorporate CI feedback in deciding to retain, revise, or eliminate potential items. We used CI in developing fatigue items for the National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Roadmap initiative. Our aims were to describe the CI process, formally evaluate the utility of decisions made on the basis of CI, and offer suggestions for future research. Methods Participants were 22 patients with a diverse range of chronic health conditions. During CI, each participant provided feedback on a series of items. We then reviewed the CI data and decided whether to retain, revise, or eliminate each potential item. Following this, we developed or adopted three quantitative methods to compare retained versus eliminated items. Results Retained items raised fewer serious concerns, were less likely to be viewed as non-applicable, and were less likely to display problems with clarity or to make incorrect assumptions about respondents. Conclusions CI was useful in developing the PROMIS fatigue items and the methods used to judge CI for the present item set may be useful for future investigations. PMID:18850327

  2. Addressing Missing Data in Patient-Reported Outcome Measures (PROMS): Implications for the Use of PROMS for Comparing Provider Performance.

    PubMed

    Gomes, Manuel; Gutacker, Nils; Bojke, Chris; Street, Andrew

    2016-05-01

    Patient-reported outcome measures (PROMs) are now routinely collected in the English National Health Service and used to compare and reward hospital performance within a high-powered pay-for-performance scheme. However, PROMs are prone to missing data. For example, hospitals often fail to administer the pre-operative questionnaire at hospital admission, or patients may refuse to participate or fail to return their post-operative questionnaire. A key concern with missing PROMs is that the individuals with complete information tend to be an unrepresentative sample of patients within each provider and inferences based on the complete cases will be misleading. This study proposes a strategy for addressing missing data in the English PROM survey using multiple imputation techniques and investigates its impact on assessing provider performance. We find that inferences about relative provider performance are sensitive to the assumptions made about the reasons for the missing data. © 2015 The Authors. Health Economics Published by John Wiley & Sons Ltd. PMID:25740592

  3. Integrating Patient-Reported Outcome Measures into Routine Cancer Care: Cancer Patients’ and Clinicians’ Perceptions of Acceptability and Value

    PubMed Central

    Stover, Angela; Irwin, Debra E.; Chen, Ronald C.; Chera, Bhishamjit S.; Mayer, Deborah K.; Muss, Hyman B.; Rosenstein, Donald L.; Shea, Thomas C.; Wood, William A.; Lyons, Jessica C.; Reeve, Bryce B.

    2015-01-01

    Introduction: Despite growing interest in integrating patient-reported outcome (PRO) measures of symptoms and functional status into routine cancer care, little attention has been paid to patients’ and clinicians’ perceptions of acceptability and value. Methods: A two-phase qualitative study was conducted to develop a web-based PRO screening system with 21 items assessing symptoms (e.g., nausea) and functional status. Phase 1 involved cognitive interviews with 35 cancer outpatients (n=9 breast chemotherapy, radiation for prostate (n=8) or head and neck cancer (n=10), and n=8 bone marrow transplant [BMT]). In Phase 2, we evaluated the acceptability and perceived value of reviewing a PRO measure during real-time clinical encounters with 39 additional outpatients (n=10 breast, n=9 head and neck, n=10 prostate, n=10 BMT) and 12 clinicians (n=3 breast, n=2 head and neck, n=4 prostate, n=3 BMT). At least 20% of patients were ≥60 years, African American, or ≤ high school. Results: Patients felt that their PRO summary of symptoms and functional status was helpful in discussing health issues with clinicians (92%), wanted to review their results with clinicians during future visits (82%), and would recommend it to other patients (87%). Clinicians found the PRO summary to be easy to interpret (83%), most helpful for documenting the Review of Symptoms (92%), and would recommend it to future patients (92%). Over 90% of clinicians reported that consultation time did not increase. Conclusion: Both cancer patients and clinicians reported that discussing a PRO summary of symptoms and functional status during an outpatient visit was useful, acceptable, and feasible. PMID:26557724

  4. Surgeon’s experiences of receiving peer benchmarked feedback using patient-reported outcome measures: a qualitative study

    PubMed Central

    2014-01-01

    Background The use of patient-reported outcome measures (PROMs) to provide healthcare professionals with peer benchmarked feedback is growing. However, there is little evidence on the opinions of professionals on the value of this information in practice. The purpose of this research is to explore surgeon’s experiences of receiving peer benchmarked PROMs feedback and to examine whether this information led to changes in their practice. Methods This qualitative research employed a Framework approach. Semi-structured interviews were undertaken with surgeons who received peer benchmarked PROMs feedback. The participants included eleven consultant orthopaedic surgeons in the Republic of Ireland. Results Five themes were identified: conceptual, methodological, practical, attitudinal, and impact. A typology was developed based on the attitudinal and impact themes from which three distinct groups emerged. ‘Advocates’ had positive attitudes towards PROMs and confirmed that the information promoted a self-reflective process. ‘Converts’ were uncertain about the value of PROMs, which reduced their inclination to use the data. ‘Sceptics’ had negative attitudes towards PROMs and claimed that the information had no impact on their behaviour. The conceptual, methodological and practical factors were linked to the typology. Conclusion Surgeons had mixed opinions on the value of peer benchmarked PROMs data. Many appreciated the feedback as it reassured them that their practice was similar to their peers. However, PROMs information alone was considered insufficient to help identify opportunities for quality improvements. The reasons for the observed reluctance of participants to embrace PROMs can be categorised into conceptual, methodological, and practical factors. Policy makers and researchers need to increase professionals’ awareness of the numerous purposes and benefits of using PROMs, challenge the current methods to measure performance using PROMs, and reduce

  5. Estimating recruitment rates for routine use of patient reported outcome measures and the impact on provider comparisons

    PubMed Central

    2014-01-01

    Background The routine use of patient reported outcome measures (PROMs) aims to compare providers as regards the clinical need of their patients and their outcome. Simple methods of estimating recruitment rates based on aggregated data may be inaccurate. Our objectives were to: use patient-level linked data to evaluate these estimates; produce revised estimates of national and providers’ recruitment rates; and explore whether or not recruitment bias exists. Methods Case study based on patients who were eligible to participate in the English National PROMs Programme for elective surgery (hip and knee replacement, groin hernia repair, varicose vein surgery) using data from pre-operative questionnaires and Hospital Episode Statistics. Data were linked to determine: the eligibility for including operations; eligibility of date of surgery; duplicate questionnaires; cancelled operations; correct assignment to provider. Influence of patient characteristics on recruitment rates were investigated. Results National recruitment rates based on aggregated data over-estimated the true rate because of the inclusion of ineligible operations (from 1.9% - 7.0% depending on operation) and operations being cancelled (1.9% - 3.6%). Estimates of national recruitment rates using inclusion criteria based on patient-level linked data were lower than those based on simple methods (eg hip replacement was 73% rather than 78%). Estimates of provider’s recruitment rates based on aggregated data were also adversely affected by attributing patients to the wrong provider (2.4% - 4.9%). Use of linked data eliminated all estimates of over 100% recruitment, though providers still showed a wide range of rates. While the principal determinant of recruitment rates was the provider, some patients’ socio-demographic characteristics had an influence on non-recruitment: non-white (Adjusted Odds Ratio 1.25-1.67, depending on operation); most deprived socio-economic group (OR 1.11-1.23); aged over 75

  6. Psychometric Characteristics of a Patient Reported Outcome Measure on Ego-Integrity and Despair among Cancer Patients

    PubMed Central

    Kleijn, Gitta; Post, Lenneke; Witte, Birgit I.; Bohlmeijer, Ernst T.; Westerhof, Gerben J.; Cuijpers, Pim; Verdonck-de Leeuw, Irma M.

    2016-01-01

    Purpose To evaluate psychometric characteristics of a questionnaire (the Northwestern Ego-integrity Scale (NEIS)) on ego-integrity (the experience of wholeness and meaning in life, even in spite of negative experiences) and despair (the experience of regret about the life one has led, and feelings of sadness, failure and hopelessness) among cancer patients. Methods Cancer patients (n = 164) completed patient reported outcome measures on ego-integrity and despair (NEIS), psychological distress, anxiety and depression (Hospital Anxiety and Depression Scale (HADS)), and quality of life (EORTC QLQ-C30 (cancer survivors, n = 57) or EORTC QLQ-C15-PAL (advanced cancer patients, n = 107)). Confirmatory Factor Analysis was used to assess construct validity. Cronbach’s alpha was used to assess internal consistency. Convergent validity was tested based on a priori defined hypotheses: a higher level of ego-integrity was expected to be related to a higher level of quality of life, and lower levels of distress, depression and anxiety; a higher level of despair was expected to be related to a lower level of quality of life, and higher levels of distress, depression and anxiety. Results The majority of all items (94.5%) of the NEIS were completed by patients and single item missing rate was below 2%. The two subscales, labeled as Ego-integrity (5 items) and Despair (4 items) had acceptable internal consistency (Cronbach’s alpha .72 and .61, respectively). The Ego-integrity subscale was not significantly associated with quality of life, distress, anxiety, or depression. The Despair subscale correlated significantly (p <.001) with quality of life (r = -.29), distress (r = .44), anxiety (r = .47) and depression (r = .32). Conclusion The NEIS has good psychometric characteristics to assess ego-integrity and despair among cancer patients. PMID:27195750

  7. Gaining the Patient Reported Outcomes Measurement Information System (PROMIS) Perspective in Chronic Kidney Disease: a Midwest Pediatric Nephrology Consortium study

    PubMed Central

    Selewski, David T.; Massengill, Susan F.; Troost, Jonathan P.; Wickman, Larysa; Messer, Kassandra L.; Herreshoff, Emily; Bowers, Corinna; Ferris, Maria E.; Mahan, John D.; Greenbaum, Larry A.; MacHardy, Jackie; Kapur, Gaurav; Chand, Deepa H.; Goebel, Jens; Barletta, Gina Marie; Geary, Denis; Kershaw, David B.; Pan, Cynthia G.; Gbadegesin, Rasheed; Hidalgo, Guillermo; Lane, Jerome C.; Leiser, Jeffrey D.; Song, Peter X.; Thissen, David; Liu, Yang; Gross, Heather E.; DeWalt, Darren A.; Gipson, Debbie S.

    2014-01-01

    Background and Objectives Chronic kidney disease is a persistent chronic health condition commonly seen in pediatric nephrology programs. Our study aims to evaluate the sensitivity of the Patient Reported Outcomes Measurement Information System (PROMIS) pediatric instrument to indicators of disease severity and activity in pediatric chronic kidney disease. Methods This cross sectional study included 233 children 8–17 years old with chronic kidney disease from 16 participating institutions in North America. Disease activity indicators, including hospitalization in the previous 6 months, edema, and number of medications consumed daily, as well as disease severity indicators of kidney function and coexisting medical conditions were captured. PROMIS domains, including depression, anxiety, social-peer relationships, pain interference, fatigue, mobility, and upper extremity function, were administered via web-based questionnaires. Absolute effect sizes (AES) were generated to demonstrate the impact of disease on domain scores. Four children were excluded because of missing GFR estimations. Results 221 of the 229 children included in the final analysis completed the entire PROMIS questionnaire. Unadjusted PROMIS domains were responsive to chronic kidney disease activity indicators and number of coexisting conditions. PROMIS domain scores were worse in the presence of recent hospitalizations (depression AES 0.33, anxiety AES 0.42, pain interference AES 0.46, fatigue AES 0.50, mobility AES 0.49), edema (depression AES 0.50, anxiety AES 0.60, pain interference AES 0.77, mobility AES 0.54) and coexisting medical conditions (social peer-relationships AES 0.66, fatigue AES 0.83, mobility AES 0.60, upper extremity function AES 0.48). Conclusions The PROMIS pediatric domains of depression, anxiety, social-peer relationships, pain interference, and mobility were sensitive to the clinical status of children with chronic kidney disease in this multi-center cross sectional study

  8. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance

    PubMed Central

    2006-01-01

    This guidance describes how the FDA evaluates patient-reported outcome (PRO) instruments used as effectiveness endpoints in clinical trials. It also describes our current thinking on how sponsors can develop and use study results measured by PRO instruments to support claims in approved product labeling (see appendix point 1). It does not address the use of PRO instruments for purposes beyond evaluation of claims made about a drug or medical product in its labeling. By explicitly addressing the review issues identified in this guidance, sponsors can increase the efficiency of their endpoint discussions with the FDA during the product development process, streamline the FDA's review of PRO endpoint adequacy, and provide optimal information about the patient's perspective of treatment benefit at the time of product approval. A PRO is a measurement of any aspect of a patient's health status that comes directly from the patient (i.e., without the interpretation of the patient's responses by a physician or anyone else). In clinical trials, a PRO instrument can be used to measure the impact of an intervention on one or more aspects of patients' health status, hereafter referred to as PRO concepts, ranging from the purely symptomatic (response of a headache) to more complex concepts (e.g., ability to carry out activities of daily living), to extremely complex concepts such as quality of life, which is widely understood to be a multidomain concept with physical, psychological, and social components. Data generated by a PRO instrument can provide evidence of a treatment benefit from the patient perspective. For this data to be meaningful, however, there should be evidence that the PRO instrument effectively measures the particular concept that is studied. Generally, findings measured by PRO instruments may be used to support claims in approved product labeling if the claims are derived from adequate and well-controlled investigations that use PRO instruments that reliably

  9. The History of Patient-Reported Outcomes in Rheumatology.

    PubMed

    Callahan, Leigh F

    2016-05-01

    The rheumatology community began incorporating patient-reported outcomes in the early 1980s, helping shift the care of chronic diseases from a narrower biomedical model to a broader biopsychosocial model of health. Early efforts were focused primarily in clinical trials and clinical research, but over the last decade there has been increasing use in routine rheumatology clinical care. More than 250 valid and reliable scales to assess domains of importance to patients with rheumatic conditions have been developed. The approach to measurement continues to be refined. Rheumatology has much to be proud of in contributions to the important field of patient-reported outcomes. PMID:27133485

  10. Patient-reported outcomes in obsessive-compulsive disorder

    PubMed Central

    Subramaniam, Mythily; Soh, Pauline; Ong, Clarissa; Esmond Seow, Lee Seng; Picco, Louisa; Vaingankar, Janhavi Ajit; Chong, Siow Ann

    2014-01-01

    The purpose of the article was to provide an overview of patient-reported outcomes (PROs) and related measures that have been examined in the context of obsessive-compulsive disorder (OCD). The current review focused on patient-reported outcome measures (PROMs) that evaluated three broad outcome domains: functioning, health-related quality of life (HRQoL), and OCD-related symptoms. The present review ultimately included a total of 155 unique articles and 22 PROMs. An examination of the PROs revealed that OCD patients tend to suffer from significant functional disability, and report lower HRQoL than controls. OCD patients report greater symptom severity than patients with other mental disorders and evidence indicates that PROMs are sensitive to change and may be even better than clinician-rated measures at predicting treatment outcomes. Nonetheless, it should be noted that the measures reviewed lacked patient input in their development. Future research on PROMs must involve patient perspectives and include rigorous psychometric evaluation of these measures. PMID:25152661

  11. Electronic capture of patient-reported and clinician-reported outcome measures in an elective orthopaedic setting: a retrospective cohort analysis

    PubMed Central

    Malhotra, Karan; Buraimoh, Olatunbosun; Thornton, James; Cullen, Nicholas; Singh, Dishan; Goldberg, Andrew J

    2016-01-01

    Objectives To determine whether an entirely electronic system can be used to capture both patient-reported outcomes (electronic Patient-Reported Outcome Measures, ePROMs) as well as clinician-validated diagnostic and complexity data in an elective surgical orthopaedic outpatient setting. To examine patients' experience of this system and factors impacting their experience. Design Retrospective analysis of prospectively collected data. Setting Single centre series. Outpatient clinics at an elective foot and ankle unit in the UK. Participants All new adult patients attending elective orthopaedic outpatient clinics over a 32-month period. Interventions All patients were invited to complete ePROMs prior to attending their outpatient appointment. At their appointment, those patients who had not completed ePROMs were offered the opportunity to complete it on a tablet device with technical support. Matched diagnostic and complexity data were captured by the treating consultant during the appointment. Outcome measures Capture rates of patient-reported and clinician-reported data. All information and technology (IT) failures, language and disability barriers were captured. Patients were asked to rate their experience of using ePROMs. The scoring systems used included EQ-5D-5L, the Manchester-Oxford Foot Questionnaire (MOxFQ) and the Visual Analogue Scale (VAS) pain score. Results Out of 2534 new patients, 2176 (85.9%) completed ePROMs, of whom 1090 (50.09%) completed ePROMs at home/work prior to their appointment. 31.5% used a mobile (smartphone/tablet) device. Clinician-reported data were captured on 2491 patients (98.3%). The mean patient experience score of using Patient-Reported Outcome Measures (PROMs) was 8.55±1.85 out of 10 and 666 patients (30.61%) left comments. Of patients leaving comments, 214 (32.13%) felt ePROMs did not adequately capture their symptoms and these patients had significantly lower patient experience scores (p<0.001). Conclusions This study

  12. How to routinely collect data on patient-reported outcome and experience measures in renal registries in Europe: an expert consensus meeting.

    PubMed

    Breckenridge, Kate; Bekker, Hillary L; Gibbons, Elizabeth; van der Veer, Sabine N; Abbott, Denise; Briançon, Serge; Cullen, Ron; Garneata, Liliana; Jager, Kitty J; Lønning, Kjersti; Metcalfe, Wendy; Morton, Rachael L; Murtagh, Fliss E M; Prutz, Karl; Robertson, Susan; Rychlik, Ivan; Schon, Steffan; Sharp, Linda; Speyer, Elodie; Tentori, Francesca; Caskey, Fergus J

    2015-10-01

    Despite the potential for patient-reported outcome measures (PROMs) and experience measures (PREMs) to enhance understanding of patient experiences and outcomes they have not, to date, been widely incorporated into renal registry datasets. This report summarizes the main points learned from an ERA-EDTA QUEST-funded consensus meeting on how to routinely collect PROMs and PREMs in renal registries in Europe. In preparation for the meeting, we surveyed all European renal registries to establish current or planned efforts to collect PROMs/PREMs. A systematic review of the literature was performed. Publications reporting barriers and/or facilitators to PROMs/PREMs collection by registries were identified and a narrative synthesis undertaken. A group of renal registry representatives, PROMs/PREMs experts and patient representatives then met to (i) share any experience renal registries in Europe have in this area; (ii) establish how patient-reported data might be collected by understanding how registries currently collect routine data and how patient-reported data is collected in other settings; (iii) harmonize the future collection of patient-reported data by renal registries in Europe by agreeing upon preferred instruments and (iv) to identify the barriers to routine collection of patient-reported data in renal registries in Europe. In total, 23 of the 45 European renal registries responded to the survey. Two reported experience in collecting PROMs and three stated that they were actively exploring ways to do so. The systematic review identified 157 potentially relevant articles of which 9 met the inclusion criteria and were analysed for barriers and facilitators to routine PROM/PREM collection. Thirteen themes were identified and mapped to a three-stage framework around establishing the need, setting up and maintaining the routine collection of PROMs/PREMs. At the consensus meeting some PROMs instruments were agreed for routine renal registry collection (the generic SF

  13. How to routinely collect data on patient-reported outcome and experience measures in renal registries in Europe: an expert consensus meeting

    PubMed Central

    Breckenridge, Kate; Bekker, Hillary L.; Gibbons, Elizabeth; van der Veer, Sabine N.; Abbott, Denise; Briançon, Serge; Cullen, Ron; Garneata, Liliana; Jager, Kitty J.; Lønning, Kjersti; Metcalfe, Wendy; Morton, Rachael L.; Murtagh, Fliss E.M.; Prutz, Karl; Robertson, Susan; Rychlik, Ivan; Schon, Steffan; Sharp, Linda; Speyer, Elodie; Tentori, Francesca; Caskey, Fergus J.

    2015-01-01

    Despite the potential for patient-reported outcome measures (PROMs) and experience measures (PREMs) to enhance understanding of patient experiences and outcomes they have not, to date, been widely incorporated into renal registry datasets. This report summarizes the main points learned from an ERA-EDTA QUEST-funded consensus meeting on how to routinely collect PROMs and PREMs in renal registries in Europe. In preparation for the meeting, we surveyed all European renal registries to establish current or planned efforts to collect PROMs/PREMs. A systematic review of the literature was performed. Publications reporting barriers and/or facilitators to PROMs/PREMs collection by registries were identified and a narrative synthesis undertaken. A group of renal registry representatives, PROMs/PREMs experts and patient representatives then met to (i) share any experience renal registries in Europe have in this area; (ii) establish how patient-reported data might be collected by understanding how registries currently collect routine data and how patient-reported data is collected in other settings; (iii) harmonize the future collection of patient-reported data by renal registries in Europe by agreeing upon preferred instruments and (iv) to identify the barriers to routine collection of patient-reported data in renal registries in Europe. In total, 23 of the 45 European renal registries responded to the survey. Two reported experience in collecting PROMs and three stated that they were actively exploring ways to do so. The systematic review identified 157 potentially relevant articles of which 9 met the inclusion criteria and were analysed for barriers and facilitators to routine PROM/PREM collection. Thirteen themes were identified and mapped to a three-stage framework around establishing the need, setting up and maintaining the routine collection of PROMs/PREMs. At the consensus meeting some PROMs instruments were agreed for routine renal registry collection (the generic SF

  14. Initial Adult Health Item Banks and First Wave Testing of the Patient-Reported Outcomes Measurement Information System (PROMIS™) Network: 2005–2008

    PubMed Central

    Cella, David; Riley, William; Stone, Arthur; Rothrock, Nan; Reeve, Bryce; Yount, Susan; Amtmann, Dagmar; Bode, Rita; Buysse, Daniel; Choi, Seung; Cook, Karon; DeVellis, Robert; DeWalt, Darren; Fries, James F.; Gershon, Richard; Hahn, Elizabeth A.; Lai, Jin-Shei; Pilkonis, Paul; Revicki, Dennis; Rose, Matthias; Weinfurt, Kevin; Hays, Ron

    2010-01-01

    Objective Patient-reported outcomes (PROs) are essential when evaluating many new treatments in health care, yet current measures have been limited by a lack of precision, standardization and comparability of scores across studies and diseases. The Patient-Reported Outcomes Measurement Information System (PROMIS™) provides item banks that offer the potential for PRO measurement that is efficient (minimizes item number without compromising reliability) flexible (enables optional use of interchangeable items), and precise (has minimal error in estimate) measurement of commonly-studied PROs. We report results from the first large-scale testing of PROMIS items. Study Design and Setting Fourteen item pools were tested in the U.S. general population and clinical groups using an online panel and clinic recruitment. A scale-setting sub-sample was created reflecting demographics proportional to the 2000 U.S. census. Results Using item response theory (graded response model), 11 item banks were calibrated on a sample of 21,133, measuring components of self-reported physical, mental and social health, along with a 10-item global health scale. Short forms from each bank were developed and compared to the overall bank as well as with other well-validated and widely accepted (“legacy”) measures. All item banks demonstrated good reliability across the majority of the score distributions. Construct validity was supported by moderate to strong correlations with legacy measures. Conclusion PROMIS item banks and their short forms provide evidence they are reliable and precise measures of generic symptoms and functional reports comparable to legacy instruments. Further testing will continue to validate and test PROMIS items and banks in diverse clinical populations. PMID:20685078

  15. Patient-reported outcomes in borderline personality disorder

    PubMed Central

    Hasler, Gregor; Hopwood, Christopher J.; Jacob, Gitta A.; Brändle, Laura S.; Schulte-Vels, Thomas

    2014-01-01

    Patient-reported outcome (PRO) refers to measures that emphasize the subjective view of patients about their health-related conditions and behaviors. Typically, PROs include self-report questionnaires and clinical interviews. Defining PROs for borderline personality disorder (BPD) is particularly challenging given the disorder's high symptomatic heterogeneity, high comorbidity with other psychiatric conditions, highly fluctuating symptoms, weak correlations between symptoms and functional outcomes, and lack of valid and reliable experimental measures to complement self-report data. Here, we provide an overview of currently used BPD outcome measures and discuss them from clinical, psychometric, experimental, and patient perspectives. In addition, we review the most promising leads to improve BPD PROs, including the DSM-5 Section III, the Recovery Approach, Ecological Momentary Assessments, and novel experimental measures of social functioning that are associated with functional and social outcomes. PMID:25152662

  16. Towards Tailored Patient's Management Approach: Integrating the Modified 2010 ACR Criteria for Fibromyalgia in Multidimensional Patient Reported Outcome Measures Questionnaire

    PubMed Central

    El Miedany, Yasser; El Gaafary, Maha; Youssef, Sally; Ahmed, Ihab

    2016-01-01

    Objectives. To assess the validity, reliability, and responsiveness to change of a patient self-reported questionnaire combining the Widespread Pain Index and the Symptom Severity Score as well as construct outcome measures and comorbidities assessment in fibromyalgia patients. Methods. The PROMs-FM was conceptualized based on frameworks used by the WHO Quality of Life tool and the PROMIS. Initially, cognitive interviews were conducted to identify item pool of questions. Item selection and reduction were achieved based on patients as well as an interdisciplinary group of specialists. Rasch and internal consistency reliability analyses were implemented. The questionnaire included the modified ACR criteria main items (Symptom Severity Score and Widespread Pain Index), in addition to assessment of functional disability, quality of life (QoL), review of the systems, and comorbidities. Every patient completed HAQ and EQ-5D questionnaires. Results. A total of 146 fibromyalgia patients completed the questionnaire. The PROMs-FM questionnaire was reliable as demonstrated by a high standardized alpha (0.886–0.982). Content construct assessment of the functional disability and QoL revealed significant correlation (p < 0.01) with both HAQ and EQ-5D. Changes in functional disability and QoL showed significant (p < 0.01) variation with diseases activity status in response to therapy. There was higher prevalence of autonomic symptoms, CVS risk, sexual dysfunction, and falling. Conclusions. The developed PROMs-FM questionnaire is a reliable and valid instrument for assessment of fibromyalgia patients. A phased treatment regimen depending on the severity of FMS as well as preferences and comorbidities of the patient is the best approach to tailored patient management. PMID:27190648

  17. Patient-Reported Outcome Measures and Risk Factors in a Quality Registry: A Basis for More Patient-Centered Diabetes Care in Sweden

    PubMed Central

    Borg, Sixten; Palaszewski, Bo; Gerdtham, Ulf-G; Ödegaard, Fredrik; Roos, Pontus; Gudbjörnsdottir, Soffia

    2014-01-01

    Diabetes is one of the chronic diseases that constitute the greatest disease burden in the world. The Swedish National Diabetes Register is an essential part of the diabetes care system. Currently it mainly records clinical outcomes, but here we describe how it has started to collect patient-reported outcome measures, complementing the standard registry data on clinical outcomes as a basis for evaluating diabetes care. Our aims were to develop a questionnaire to measure patient abilities and judgments of their experience of diabetes care, to describe a Swedish diabetes patient sample in terms of their abilities, judgments, and risk factors, and to characterize groups of patients with a need for improvement. Patient abilities and judgments were estimated using item response theory. Analyzing them together with standard risk factors for diabetes comorbidities showed that the different types of data describe different aspects of a patient’s situation. These aspects occasionally overlap, but not in any particularly useful way. They both provide important information to decision makers, and neither is necessarily more relevant than the other. Both should therefore be considered, to achieve a more complete evaluation of diabetes care and to promote person-centered care. PMID:25431875

  18. [Portuguese-language cultural adaptation of the Items Banks of Anxiety and Depression of the Patient-Reported Outcomes Measurement Information System (PROMIS)].

    PubMed

    Castro, Natália Fontes Caputo de; Rezende, Carlos Henrique Alves de; Mendonça, Tânia Maria da Silva; Silva, Carlos Henrique Martins da; Pinto, Rogério de Melo Costa

    2014-04-01

    The Patient-Reported Outcome Measurement Information System (PROMIS), structured in Itens Banks, provides a new tool for evaluating results that apply to various chronic diseases through advanced statistical techniques (TRI) and computerized adaptive testing (CAT). The aim of this study was to culturally adapt the Items Banks of Anxiety and Depression of PROMIS to the Portuguese language. The process followed the recommendations of PROMIS through the advanced translation, reconciliation, back-translation, FACIT review, independent review, finalization, pre-test, and incorporation of the results from the pre-test. The translated version was pre-tested in ten patients, and items 3, 46, and 53 of the Bank of Anxiety and item 46 of the bank of Depression had to be changed. Changes affected equivalence of meaning, and the final version was consistent with the Brazilian population's linguistic and cultural skills. In conclusion, for the Brazilian population the translated version proved semantically and conceptually equivalent to the original. PMID:24896064

  19. Patient-reported outcomes in lupus clinical trials with biologics.

    PubMed

    Annapureddy, N; Devilliers, H; Jolly, M

    2016-09-01

    Therapeutic advances in systemic lupus erythematosus (SLE) are greatly needed. Despite advances in our knowledge of pathogenesis of the disease and targets, treatment remains a significant challenge. Finding effective and relatively safe medications remains one of the top priorities. SLE significantly impairs quality of life (QoL), and patient-reported outcomes (PROs) measure a unique aspect of the disease not captured by disease activity. Inclusion of PRO measurements is encouraged in SLE clinical trials, as they allow capturing benefits of a proposed intervention in language patients can relate to and in areas deemed pertinent and important to and by patients. Availability of patient-reported and patient-centric clinical trials data may facilitate patients in informed and shared decision making, and allow for comparative cost-effectiveness evaluation for future resource allocation and reimbursements. Herein we review clinical trials with biologic therapies wherein PRO tools were included in the study design. PMID:27497256

  20. Prevalence and impact of premature ejaculation in outpatients complaining of ejaculating prematurely: using the instruments of intravaginal ejaculatory latency time and patient-reported outcome measures.

    PubMed

    Gao, J; Zhang, X; Su, P; Shi, K; Tang, D; Hao, Z; Zhou, J; Liang, C

    2014-01-01

    This study was designed to assess the impact of premature ejaculation (PE) on a large population of men and their female partners using the instruments of intravaginal ejaculatory latency time (IELT) and patient-reported outcome (PRO) measures. A non-interventional, observational and cross-sectional field survey enrolled 2704 men with self-reported PE and their female partners from January 2010 to January 2012. PE was diagnosed by the International Society for Sexual Medicine (ISSM) criterion. IELT and sexual dysfunction were measured using a stopwatch and PRO measures, respectively. The incidence of PE in this study was 19.27%. PE negatively impacted on subjects and their partners, including reduced ejaculatory control and sexual satisfaction, and increased personal distress and interpersonal difficulty (P<0.001 for all). The severity of PE was considered worse by subjects than by their female partners (P<0.001). Reduced ejaculatory control and sexual satisfaction were considered the central themes of PE. Furthermore, a correlation was observed among the outcomes of IELT and PRO measures for subjects and their partners (absolute correlation coefficient ranged from 0.33 to 0.67). This in-depth qualitative study provides valuable insight into the PE status in Chinese men. Further research is needed to confirm and extend these results. PMID:24384563

  1. Patient-Reported Outcomes and Fibromyalgia.

    PubMed

    Williams, David A; Kratz, Anna L

    2016-05-01

    Fibromyalgia (FM) is classified as a chronic pain condition accompanied by symptoms of fatigue, sleep problems, problems with cognition, negative mood, limited functional status, and the presence of other chronic overlapping pain conditions. Comprehensive assessment of all of these components can be challenging. This paper provides an overview of patient-reported approaches that can be taken to assess FM in the contexts of diagnosis, symptom monitoring, phenotyping/characterization, and for purposes of clinical trials. PMID:27133492

  2. Improving patient reported outcome measures (PROMs) in total knee replacement by changing implant and preserving the infrapatella fatpad: a quality improvement project

    PubMed Central

    Partridge, Thomas; Carluke, Ian; Emmerson, Kevin; Partington, Paul; Reed, Mike

    2016-01-01

    Patient reported outcome measures (PROMs) were introduced in 2009 to allow patient perspectives to potentially influence change and improvement. In collaboration with the national joint registry (NJR), PROMs data has been examined on a national basis to compare surgical factors in total knee replacement (TKR). Initial results demonstrated there were statistically significant differences in Oxford Knee Score (OKS) when using different brands of implant. Preservation of the infrapatella fatpad (IFP) has also been shown improve outcomes. This led Northumbria Healthcare NHS Foundation Trust to make a mass move to the Zimmer Nexgen TKR and later change surgeons' routine practice to preserving the IFP. The PROMs were recorded pre and six months post operation to obtain improvement scores. The baseline improvement in OKS was 14.0. After changing implant to the Zimmer Nexgen in Plan-Do-Study-Act (PDSA) cycle 1 the average improvement score was 16.7. After implementing default preservation of the IFP in PDSA cycle 2 the average OKS improvement score was 17.3. The results from this project demonstrate a significant improvement in local services after implementing changes based on national and local evaluations. This initiative is an excellent example of improvement by evidence based practice and success of the English National Health Service PROMs scheme. PMID:27239301

  3. Validation of the German version of the extended ALS functional rating scale as a patient-reported outcome measure.

    PubMed

    Abdulla, Susanne; Vielhaber, Stefan; Körner, Sonja; Machts, Judith; Heinze, Hans-Jochen; Dengler, Reinhard; Petri, Susanne

    2013-09-01

    The revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a well-established rating instrument to assess the functional status of ALS patients. A recent innovation was the addition of three further items designed to improve its sensitivity at lower levels of physical function (ALSFRS-Extension, ALSFRS-EX). Neither the ALSFRS-R nor the ALSFRS-EX has been validated in German yet. The aim of the present study was the validation of the German version of a self-administered form of the ALSFRS-EX. Seventy-six patients participated in the study. Psychometric analysis included reliability assessment and factorial analysis. To evaluate convergent validity, correlations between ALSFRS-EX items and the MRC score, spasticity, tongue movement, pulmonary function, ALSAQ-40 and Borg dyspnoea scales (upright and supine) were performed. Internal consistency as measured by Cronbach's alpha (total scale 0.868, subscales 0.690-0.938) and corrected item to total correlations (all above 0.50) was high. Test-retest reliability assessed by Spearman's rho (0.882-0.972) and Cohen's Kappa (0.63-0.92) was also high. Principal component analysis with varimax rotation yielded a four-factor solution accounting for approximately 79% of the variance. Clinical parameters were strongly correlated with respective items and subscores of the ALSFRS-EX (muscle strength 0.568-0.833 p < 0.01; spasticity -0.236 to -0.376 p < 0.05; tongue movement 0.437-0.818 p < 0.01; pulmonary function 0.485-0.577 p < 0.01). ALSAQ-40 and Borg score correlated highly with the corresponding ALSFRS-EX items. The German self-report version of the ALSFRS-EX possesses very good psychometric properties similar to the original scale including high internal consistency and test-retest reliability as well as excellent convergent validity. PMID:23736973

  4. Difference in method of administration did not significantly impact item response: an IRT-based analysis from the Patient-Reported Outcomes Measurement Information System (PROMIS) initiative

    PubMed Central

    Rose, Matthias; Gandek, Barbara; Stone, Arthur A.; Junghaenel, Doerte U.; Ware, John E.

    2013-01-01

    Purpose To test the impact of method of administration (MOA) on the measurement characteristics of items developed in the Patient-Reported Outcomes Measurement Information System (PROMIS). Methods Two non-overlapping parallel 8-item forms from each of three PROMIS domains (physical function, fatigue, and depression) were completed by 923 adults (age 18–89) with chronic obstructive pulmonary disease, depression, or rheumatoid arthritis. In a randomized crossover design, subjects answered one form by interactive voice response (IVR) technology, paper questionnaire (PQ), personal digital assistant (PDA), or personal computer (PC) on the Internet, and a second form by PC, in the same administration. Structural invariance, equivalence of item responses, and measurement precision were evaluated using confirmatory factor analysis and item response theory methods. Results Multigroup confirmatory factor analysis supported equivalence of factor structure across MOA. Analyses by item response theory found no differences in item location parameters and strongly supported the equivalence of scores across MOA. Conclusions We found no statistically or clinically significant differences in score levels in IVR, PQ, or PDA administration as compared to PC. Availability of large item response theory-calibrated PROMIS item banks allowed for innovations in study design and analysis. PMID:23877585

  5. Mapping the Content of the Patient Reported Outcomes Measurement Information System (PROMIS®) Using the International Classification of Functioning, Health and Disability

    PubMed Central

    Tucker, Carole A; Escorpizo, Reuben; Cieza, Alarcos; Lai, Jin Shei; Stucki, Gerold; Ustun, T. Bedirhan; Kostanjsek, Nenad; Cella, David; Forrest, Christopher B.

    2014-01-01

    Background The Patient Reported Outcomes Measurement Information System (PROMIS®) is a U.S. National Institutes of Health initiative that has produced self-reported item banks for physical, mental, and social health. Objective To describe the content of PROMIS at the item level using the World Health Organization’s International Classification of Functioning, Disability and Health (ICF). Methods All PROMIS adult items (publicly available as of 2012) were assigned to relevant ICF concepts. The content of the PROMIS adult item banks were then described using the mapped ICF code descriptors. Results The 1006 items in the PROMIS instruments could all be mapped to ICF concepts at the second level of classification, with the exception of 3 items of global or general health that mapped across the first-level classification of ICF activity and participation component (d categories). Individual PROMIS item banks mapped from 1 to 5 separate ICF codes indicating one-to-one, one-to-many and many-to-one mappings between PROMIS item banks and ICF second level classification codes. PROMIS supports measurement of the majority of major concepts in the ICF Body Functions (b) and Activity & Participation (d) components using PROMIS item banks or subsets of PROMIS items that could, with care, be used to develop customized instruments. Given the focus of PROMIS is on measurement of person health outcomes, concepts in body structures (s) and some body functions (b), as well as many ICF environmental factor have minimal coverage in PROMIS. Discussion The PROMIS-ICF mapped items provide a basis for users to evaluate the ICF related content of specific PROMIS instruments, and to select PROMIS instruments in ICF based measurement applications. PMID:24760532

  6. A systematic review of the impact of routine collection of patient reported outcome measures on patients, providers and health organisations in an oncologic setting

    PubMed Central

    2013-01-01

    Background Despite growing interest and urges by leading experts for the routine collection of patient reported outcome (PRO) measures in all general care patients, and in particular cancer patients, there has not been an updated comprehensive review of the evidence regarding the impact of adopting such a strategy on patients, service providers and organisations in an oncologic setting. Methods Based on a critical analysis of the three most recent systematic reviews, the current systematic review developed a six-method strategy in searching and reviewing the most relevant quantitative studies between January 2000 and October 2011 using a set of pre-determined inclusion criteria and theory-based outcome indicators. The Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) system was used to rate the quality and importance of the identified publications, and the synthesis of the evidence was conducted. Results The 27 identified studies showed strong evidence that the well-implemented PROs improved patient-provider communication and patient satisfaction. There was also growing evidence that it improved the monitoring of treatment response and the detection of unrecognised problems. However, there was a weak or non-existent evidence-base regarding the impact on changes to patient management and improved health outcomes, changes to patient health behaviour, the effectiveness of quality improvement of organisations, and on transparency, accountability, public reporting activities, and performance of the health care system. Conclusions Despite the existence of significant gaps in the evidence-base, there is growing evidence in support of routine PRO collection in enabling better and patient-centred care in cancer settings. PMID:23758898

  7. Ecological Validity and Clinical Utility of Patient-Reported Outcomes Measurement Information System (PROMIS®) instruments for detecting premenstrual symptoms of depression, anger, and fatigue

    PubMed Central

    Junghaenel, Doerte U.; Schneider, Stefan; Stone, Arthur A.; Christodoulou, Christopher; Broderick, Joan E.

    2014-01-01

    Objective This study examined the ecological validity and clinical utility of NIH Patient Reported-Outcomes Measurement Information System (PROMIS®) instruments for anger, depression, and fatigue in women with premenstrual symptoms. Methods One-hundred women completed daily diaries and weekly PROMIS assessments over 4 weeks. Weekly assessments were administered through Computerized Adaptive Testing (CAT). Weekly CATs and corresponding daily scores were compared to evaluate ecological validity. To test clinical utility, we examined if CATs could detect changes in symptom levels, if these changes mirrored those obtained from daily scores, and if CATs could identify clinically meaningful premenstrual symptom change. Results PROMIS CAT scores were higher in the pre-menstrual than the baseline (ps < .0001) and post-menstrual (ps < .0001) weeks. The correlations between CATs and aggregated daily scores ranged from .73 to .88 supporting ecological validity. Mean CAT scores showed systematic changes in accordance with the menstrual cycle and the magnitudes of the changes were similar to those obtained from the daily scores. Finally, Receiver Operating Characteristic (ROC) analyses demonstrated the ability of the CATs to discriminate between women with and without clinically meaningful premenstrual symptom change. Conclusions PROMIS CAT instruments for anger, depression, and fatigue demonstrated validity and utility in premenstrual symptom assessment. The results provide encouraging initial evidence of the utility of PROMIS instruments for the measurement of affective premenstrual symptoms. PMID:24630180

  8. Introducing the Concept of the Minimally Important Difference to Determine a Clinically Relevant Change on Patient-Reported Outcome Measures in Patients with Intermittent Claudication

    SciTech Connect

    Conijn, Anne P.; Jonkers, Wilma; Rouwet, Ellen V.; Vahl, Anco C.; Reekers, Jim A.; Koelemay, Mark J. W.

    2015-10-15

    PurposeThe minimally important difference (MID) represents the smallest change in score on patient-reported outcome measures that is relevant to patients. The aim of this study was to introduce the MID for the Vascular Quality of Life Questionnaire (VascuQol) and the walking impairment questionnaire (WIQ) for patients with intermittent claudication (IC).MethodsIn this multicenter study, we recruited 294 patients with IC between July and October 2012. Patients completed the VascuQol, with scores ranging from 1 to 7 (worst to best), and the WIQ, with scores ranging from 0 to 1 (worst to best) at first visit and after 4 months follow-up. In addition, patients answered an anchor-question rating their health status compared to baseline, as being improved, unchanged, or deteriorated. The MID for improvement and deterioration was calculated by an anchor-based approach, and determined with the upper and lower limits of the 95 % confidence interval of the mean change of the group who had not changed according to the anchor-question.ResultsFor the MID analyses of the VascuQol and WIQ, 163 and 134 patients were included, respectively. The MID values for the VascuQol (mean baseline score 4.25) were 0.87 for improvement and 0.23 for deterioration. For the WIQ (mean baseline score 0.39), we found MID values of 0.11 and −0.03 for improvement and deterioration, respectively.ConclusionIn this study, we calculated the MID for the VascuQol and the WIQ. Applying these MID facilitates better interpretation of treatment outcomes and can help to set treatment goals for individual care.

  9. Capturing Patient-Reported Outcome (PRO) Data Electronically: The Past, Present, and Promise of ePRO Measurement in Clinical Trials.

    PubMed

    Coons, Stephen Joel; Eremenco, Sonya; Lundy, J Jason; O'Donohoe, Paul; O'Gorman, Hannah; Malizia, William

    2015-08-01

    Patient-reported outcomes (PROs) are an important means of evaluating the treatment benefit of new medical products. It is recognized that PRO measures should be used when assessing concepts best known by the patient or best measured from the patient's perspective. As a result, there is growing emphasis on well defined and reliable PRO measures. In addition, advances in technology have significantly increased electronic PRO (ePRO) data collection capabilities and options in clinical trials. The movement from paper-based to ePRO data capture has enhanced the integrity and accuracy of clinical trial data and is encouraged by regulators. A primary distinction in the types of ePRO platforms is between telephone-based interactive voice response systems and screen-based systems. Handheld touchscreen-based devices have become the mainstay for remote (i.e., off-site, unsupervised) PRO data collection in clinical trials. The conventional approach is to provide study subjects with a handheld device with a device-based proprietary software program. However, an emerging alternative for clinical trials is called bring your own device (BYOD). Leveraging study subjects' own Internet-enabled mobile devices for remote PRO data collection (via a downloadable app or a Web-based data collection portal) has become possible due to the widespread use of personal smartphones and tablets. However, there are a number of scientific and operational issues that must be addressed before BYOD can be routinely considered as a practical alternative to conventional ePRO data collection methods. Nevertheless, the future for ePRO data collection is bright and the promise of BYOD opens a new chapter in its evolution. PMID:25300613

  10. Patient reported outcomes in hip arthroplasty registries.

    PubMed

    Paulsen, Aksel

    2014-05-01

    -retest). I found that the translated PRO had good feasibility, an excellent response rate, no floor effect, but a high ceiling effect (as was expected with our postoperative patients) and few patients missed too many items to calculate a sum score. The translated PRO had high test-retest reliability and very high internal consistency, and appears to be a valid and reliable tool for outcome studies on THA patients in a hip registry setting. The MCII and PASS study included 1,335 patients, and I estimated that one year after THA, an improvement of 38-55% from mean baseline PRO score and absolute follow-up scores of 57-91% of the maximum score correspond to a minimal important improvement and acceptable symptom state, respectively. PMID:24814747

  11. [Patient-reported and patient-weighted outcomes in ophthalmology].

    PubMed

    Scheibler, F; Finger, R P; Grosselfinger, R; Dintsios, C-M

    2010-03-01

    Considering patients' values and preferences in comparative effectiveness research (CER) is one of the main challenges in ophthalmology (value-based medicine). This article defines core terms in CER. The concept of patient-relevant (or patient-important) outcomes is distinguished from patient-reported outcomes (PRO) by means of examples in the field of ophthalmology. In order to be able to give a consistant recommendation if an intervention leads to conflicting results for different outcomes (trade-off), a ranking of outcomes will be necessary. Examples of studies in glaucoma patients are provided that demonstrate the possibilities of ranking of outcomes based on patient preferences. PMID:20024566

  12. Occurrences and sources of Differential Item Functioning (DIF) in patient-reported outcome measures: Description of DIF methods, and review of measures of depression, quality of life and general health

    PubMed Central

    Teresi, Jeanne A.; Ramirez, Mildred; Lai, Jin-Shei; Silver, Stephanie

    2009-01-01

    Examination of the equivalence of measures involves several levels, including conceptual equivalence of meaning, as well as quantitative tests of differential item functioning (DIF). The purpose of this review is to examine DIF in patient-reported outcomes. Reviewed were measures of self-reported depression, quality of life (QoL) and general health. Most measures of depression contained large amounts of DIF, and the impact of DIF at the scale level was typically sizeable. The studies of QoL and health measures identified a moderate amount of DIF; however, many of these studies examined only one type of DIF (uniform). Relative to DIF analyses of depression measures, less analysis of the impact of DIF on QoL and health measures was performed, and the authors of these analyses generally did not recommend remedial action, with one notable exception. While these studies represent good beginning efforts to examine measurement equivalence in patient-reported outcome measures, more cross-validation work is required using other (often larger) samples of different ethnic and language groups, as well as other methods that permit more extensive analyses of the type of DIF, together with magnitude and impact. PMID:20165561

  13. Validity and reliability of patient reported outcomes used in Psoriasis: results from two randomized clinical trials

    PubMed Central

    Shikiar, Richard; Bresnahan, Brian W; Stone, Stephen P; Thompson, Christine; Koo, John; Revicki, Dennis A

    2003-01-01

    Background Two Phase III randomized controlled clinical trials were conducted to assess the efficacy, safety, and tolerability of weekly subcutaneous administration of efalizumab for the treatment of psoriasis. Patient reported measures of psoriasis-related functionality and health-related quality of life and of psoriasis-related symptom assessments were included as part of the trials. Objective To assess the reliability, validity, and responsiveness of the patient reported outcome measures that were used in the trials – the Dermatology Life Quality Index (DLQI), the Psoriasis Symptom Assessment (PSA) Scale, and two itch measures, a Visual Analog Scale (VAS) and the National Psoriasis Foundation (NPF) itch measure. Methods Subjects aged 18 to 70 years with moderate to severe psoriasis for at least 6 months were recruited into the two clinical trials (n = 1095). Internal consistency reliability was evaluated for all patient reported outcomes at baseline and at 12 weeks. Construct validity was evaluated by relations among the different patient reported outcomes and between the patient reported outcomes and the clinical assessments (Psoriasis Area and Severity Index; Overall Lesion Severity Scale; Physician's Global Assessment of Change) assessed at baseline and at 12 weeks, as was the change over the course of the 12 week portion of the trial. Results Internal consistency reliability ranged from 0.86 to 0.95 for the patient reported outcome measures. The patient reported outcome measures were all shown to have significant construct validity with respect to each other and with respect to the clinical assessments. The four measures also demonstrated significant responsiveness to change in underlying clinical status of the patients over the course of the trial, as measured by the independently assessed clinical outcomes. Conclusions The DLQI, the PSA, VAS, and the NPF are considered useful tools for the measurement of dermatology-related limitations of functional

  14. Interpreting and reporting results based on patient-reported outcomes.

    PubMed

    Revicki, Dennis A; Erickson, Pennifer A; Sloan, Jeff A; Dueck, Amylou; Guess, Harry; Santanello, Nancy C

    2007-01-01

    This article deals with the incorporation of patient-reported outcomes (PROs) into clinical trials and focuses on issues associated with the interpretation and reporting of PRO data. The primary focus and context of this information relates to the evidentiary support and reporting for a labeling or advertising claim of a PRO benefit for a new or approved pharmaceutical product. This manuscript focuses on issues associated with assessing clinical significance and common pitfalls to avoid in presenting results related to PROs. Specifically, the questions addressed by this manuscript involve: What are the best methods to assess clinical significance for PROs? How should investigators present PRO data most effectively in a Food and Drug Administration (FDA) application? In labeling or in a scientific publication? Guidelines for interpreting clinical significance of PROs and for comprehensively reporting on the methods, measures and results of clinical trials that incorporate PROs are important for clinicians, regulatory agencies, and most of all to patients. Clear specifications for considering a finding on a PRO measure, as clinically meaningful, need to be determined by instrument developers and psychometricians; they need to be reported for all clinical trials involving PRO end points. Clinical trial reports need to be comprehensive, clear, and sufficient to enable any reader to understand the methods, PRO measures, statistical analysis, and results. PMID:17995470

  15. 'The patient': at the center of patient-reported outcomes.

    PubMed

    Awad, A George

    2015-01-01

    The recent emphasis of including patient reports in their own care management is reviewed in terms of the factors that contributed to its popularity. The role change of patients as being active participants in their own care as a result of the rising consumerism and advocacy has led to increased pressures for including patients in the therapeutic decision-making process. As consumers of clinical services, their perspectives and attitudes towards health and illness acquired more importance. The rising cost of healthcare has added another dimension in cost containment by empowering patients and sharing responsibility in their recovery, which hopefully can improve outcomes. Challenges in the development and implementation of patient-reported outcomes in psychiatry are reviewed and include the still unresolved subjective/objective dichotomy, identification of the most appropriate and relevant patient-reported outcomes. Few outcomes are identified and include: subjective tolerability of medication, self-reported health-related quality of life, preferences, patients' attitudes towards health and illness, satisfaction with medication and overall satisfaction of quality of care, and functional state, with particular focus on social functioning. PMID:26289737

  16. Patient-reported outcomes: instrument development and selection issues.

    PubMed

    Turner, Ralph R; Quittner, Alexandra L; Parasuraman, Bhash M; Kallich, Joel D; Cleeland, Charles S

    2007-01-01

    At its most elemental, patient-reported outcomes (PRO) assessment involves asking the patients questions and evaluating their answers. Instrument developers need to be clear about what they want to know, from whom they want to know it and why, whether what they learned is credible, and whether they can interpret what they learned in the context of the research objectives. Because credible instrument development is neither inexpensive nor technically trivial, researchers must first determine that no available measure meets their research objectives. We suggest that the tasks of either reviewing current instruments or developing new ones originate from the same basic premise: PRO assessment requires a well-articulated conceptual framework. Once defined in the context of the research objectives, the conceptual framework needs to be adapted to the population of interest. We discuss how qualitative methods enrich the conceptual framework and facilitate the technical measurement tasks of item development, testing, and reduction. We recognize that PRO assessment stands at a technological crossroads with the increasingly frequent application of "modern" psychometric methods and discuss how innovations such as item banks and computer-adaptive testing will influence PRO instrument development. Although items are the essential building blocks for instruments, scales are the primary unit of analysis for PRO assessment, and we discuss methods for scoring and combining them. Finally, PRO assessment is meaningless if the key figure chooses not to cooperate. We consider how respondent burden influences the quality of PRO assessment. PMID:17995478

  17. Use of Patient-Reported Outcome (PRO) Measures at Group and Patient Levels: Experiences From the Generic Integrated PRO System, WestChronic

    PubMed Central

    Larsen, Louise Pape; Biering, Karin; Johnsen, Soren Paaske; Riiskjær, Erik; Schougaard, Liv Marit

    2014-01-01

    Background Patient-reported outcome (PRO) measures may be used at a group level for research and quality improvement and at the individual patient level to support clinical decision making and ensure efficient use of resources. The challenges involved in implementing PRO measures are mostly the same regardless of aims and diagnostic groups and include logistic feasibility, high response rates, robustness, and ability to adapt to the needs of patient groups and settings. If generic PRO systems can adapt to specific needs, advanced technology can be shared between medical specialties and for different aims. Objective We describe methodological, organizational, and practical experiences with a generic PRO system, WestChronic, which is in use among a range of diagnostic groups and for a range of purposes. Methods The WestChronic system supports PRO data collection, with integration of Web and paper PRO questionnaires (mixed-mode) and automated procedures that enable adherence to implementation-specific schedules for the collection of PRO. For analysis, we divided functionalities into four elements: basic PRO data collection and logistics, PRO-based clinical decision support, PRO-based automated decision algorithms, and other forms of communication. While the first element is ubiquitous, the others are optional and only applicable at a patient level. Methodological and organizational experiences were described according to each element. Results WestChronic has, to date, been implemented in 22 PRO projects within 18 diagnostic groups, including cardiology, neurology, rheumatology, nephrology, orthopedic surgery, gynecology, oncology, and psychiatry. The aims of the individual projects included epidemiological research, quality improvement, hospital evaluation, clinical decision support, efficient use of outpatient clinic resources, and screening for side effects and comorbidity. In total 30,174 patients have been included, and 59,232 PRO assessments have been collected

  18. Validation of the National Institutes of Health Patient-Reported Outcomes Measurement Information System Survey as a Quality-of-Life Instrument for Patients with Malignant Brain Tumors and Their Caregivers.

    PubMed

    Romero, Melissa M; Flood, Lisa Sue; Gasiewicz, Nanci K; Rovin, Richard; Conklin, Samantha

    2015-12-01

    At present there is a lack of well-validated surveys used to measure quality of life in patients with malignant brain tumors and their caregivers. The main objective of this pilot study was to validate the National Institutes of Health Patient-Reported Outcomes Measurement Information System (NIH PROMIS) survey for use as a quality-of-life measure in this population. This article presents the rationale for using the NIH PROMIS instrument as a quality-of-life measure for patients with malignant brain tumors and their caregivers. PMID:26596656

  19. Mediators of Atherosclerosis in South Asians Living in America: Use of Web-Based Methods for Follow-Up and Collection of Patient-Reported Outcome Measures

    PubMed Central

    Puri-Taneja, Ankita; Victorson, David E; Dave, Swapna S; Kanaya, Alka M; Huffman, Mark D

    2016-01-01

    Background A key challenge for longitudinal cohort studies is follow-up and retention of study participants. Participant follow-up in longitudinal cohort studies is costly and time-consuming for research staff and participants. Objective This study determined the feasibility and costs of using Web-based technologies for follow-up and collection of patient-reported outcomes in the Mediators of Atherosclerosis in South Asians Living in America (MASALA) study. Methods The MASALA study is a community-based cohort of 906 South Asians in the United States. Since the baseline in-person visits (2010-2013), a yearly telephone follow-up survey was used to assess participants’ health status and incidence of cardiovascular disease. A Web-based version of the follow-up survey was developed using the REDCap (Research Electronic Data Capture) Web app. Participants from the Chicago field center who were due for their annual follow-up and who had a valid email address were sent an email link to a secure online portal where they could complete the survey. Telephone follow-up was used with nonresponders. Results A link to the Web survey was emailed to 285 participants (February to October 2014) and the overall completion rate was 47.7% (136/285). One-third of participants who were unresponsive (n=36) to annual telephone follow-up completed the Web survey. Web responders were younger, more likely to be married, and to have higher education and income compared (P<.05) to telephone-only responders. Web survey development involved 240 hours of research staff time. Since launching, the Web-based survey has required 3 hours per week of staff time. Conclusions Although electronic follow-up will not be a panacea for cohort operations, it will serve as an adjunctive strategy to telephonic follow-up for maximizing cohort retention with lower costs. PMID:27278905

  20. Athletic groin pain (part 1): a prospective anatomical diagnosis of 382 patients—clinical findings, MRI findings and patient-reported outcome measures at baseline

    PubMed Central

    Falvey, É C; King, E; Kinsella, S; Franklyn-Miller, A

    2016-01-01

    Background Athletic groin pain remains a common field-based team sports time-loss injury. There are few reports of non-surgically managed cohorts with athletic groin pain. Aim To describe clinical presentation/examination, MRI findings and patient-reported outcome (PRO) scores for an athletic groin pain cohort. Methods All patients had a history including demographics, injury duration, sport played and standardised clinical examination. All patients underwent MRI and PRO score to assess recovery. A clinical diagnosis of the injured anatomical structure was made based on these findings. Statistical assessment of the reliability of accepted standard investigations undertaken in making an anatomical diagnosis was performed. Result 382 consecutive athletic groin pain patients, all male, enrolled. Median time in pain at presentation was (IQR) 36 (16–75) weeks. Most (91%) played field-based ball-sports. Injury to the pubic aponeurosis (PA) 240 (62.8%) was the most common diagnosis. This was followed by injuries to the hip in 81 (21.2%) and adductors in 56 (14.7%) cases. The adductor squeeze test (90° hip flexion) was sensitive (85.4%) but not specific for the pubic aponeurosis and adductor pathology (negative likelihood ratio 1.95). Analysed in series, positive MRI findings and tenderness of the pubic aponeurosis had a 92.8% post-test probability. Conclusions In this largest cohort of patients with athletic groin pain combining clinical and MRI diagnostics there was a 63% prevalence of PA injury. The adductor squeeze test was sensitive for athletic groin pain, but not specific individual pathologies. MRI improved diagnostic post-test probability. No hernia or incipient hernia was diagnosed. Clinical trial registration number NCT02437942. PMID:26626272

  1. The Importance of Hand Appearance as a Patient-Reported Outcome in Hand Surgery

    PubMed Central

    Johnson, Shepard P.; Sebastin, Sandeep J.; Rehim, Shady A.

    2015-01-01

    Summary: Hand appearance is meaningful to patients because hands are an essential part of human interactions, communication, and social integration. Recent literature indicates that hand aesthetics is an important, measurable patient-reported outcome. In hand surgery, several outcome instruments exist that accurately measure functional outcomes, but aesthetics is often overlooked or imprecisely measured. This makes comparison of disease burden and effectiveness of therapies, as they pertain to aesthetics, difficult. This special topic article outlines the aesthetic features of the hand, how literature is evaluating the appearance of the hand in outcomes research, and proposes a novel approach to assessing hand aesthetics. PMID:26893977

  2. Patient-Reported Outcome Measures (PROMS) in patients undergoing heart valve surgery: why should we measure them and which instruments should we use?

    PubMed Central

    Holmes, Charlotte; Briffa, Norman

    2016-01-01

    Objective As the population ages, the incidence of heart valve disease (HVD) is increasing. The aim of treatment is to improve prognosis and quality of life. Standard surgical treatment is being superseded by new catheter-based treatments, many of which are as yet unproven. The need for appropriate instruments to measure quality of life in patients receiving treatment for HVD has therefore never been greater. Methods In this prospective observational study, a generic instrument, Euroqol, and a disease-specific one (Minnesota Living with Heart Failure Questionnaire—MLHFQ) were, for the first time, formally tested before and after surgery in 84 patients with HVD who completed their treatment. Patients were interviewed on the night before surgery and 6–12 weeks after being discharged. Instruments were tested for validity, reliability, responsiveness, sensitivity and interpretability. Results Both Euroqol and MLHFQ registered significant improvements in patients' health. Tests for validity were significantly positive for both Euroqol and MLHFQ. Tests for reliability and responsiveness were very positive for MLHFQ, less so for EQ-5D. There was a moderate ceiling effect in the postoperative Index scores of Euroqol and a moderate floor effect in MLHFQ. Conclusions Both instruments together performed very well in assessing the health of patients undergoing surgical treatment of HVD. As the incidence of HVD increases and therapeutic options increase, measurement of PROMS using these two instruments should become a matter of routine. PMID:27175284

  3. Is Real-Time Feedback of Burn-Specific Patient-Reported Outcome Measures in Clinical Settings Practical and Useful? A Pilot Study Implementing the Young Adult Burn Outcome Questionnaire.

    PubMed

    Ryan, Colleen M; Lee, Austin F; Kazis, Lewis E; Shapiro, Gabriel D; Schneider, Jeffrey C; Goverman, Jeremy; Fagan, Shawn P; Wang, Chao; Kim, Julia; Sheridan, Robert L; Tompkins, Ronald G

    2016-01-01

    Long-term follow-up care of survivors after burn injuries can potentially be improved by the application of patient-reported outcome measures (PROMs). PROMs can inform clinical decision-making and foster communication between the patient and provider. There are no previous reports using real-time, burn-specific PROMs in clinical practice to track and benchmark burn recovery over time. This study examines the feasibility of a computerized, burn-specific PROM, the Young Adult Burn Outcome Questionnaire (YABOQ), with real-time benchmarking feedback in a burn outpatient practice. The YABOQ was redesigned for formatting and presentation purposes using images and transcribed to a computerized format. The redesigned questionnaire was administered to young adult burn survivors (ages 19-30 years, 1-24 months from injury) via an ipad platform in the office before outpatient visits. A report including recovery curves benchmarked to a nonburned relatively healthy age-matched population and to patients with similar injuries was produced for the domains of physical function and social function limited by appearance. A copy of the domain reports as well as a complete copy of the patient's responses to all domain questions was provided for use during the clinical visit. Patients and clinicians completed satisfaction surveys at the conclusion of the visit. Free-text responses, included in the satisfaction surveys, were treated as qualitative data adding contextual information about the assessment of feasibility. Eleven patients and their providers completed the study for 12 clinical visits. All patients found the ipad survey and report "easy" or "very easy" to use. In nine instances, patients "agreed" or "strongly agreed" that it helped them communicate their situation to their doctor/nurse practitioner. Patients "agreed" or "strongly agreed" that the report helped them understand their course of recovery in 10 visits. In 11 visits, the patients "agreed" or "strongly agreed" that

  4. Application of Bother in patient reported outcomes instruments across cultures

    PubMed Central

    2014-01-01

    Background The objective of this study was to determine the applicability of the term bother, as used in Patient Reported Outcomes (PRO) instruments that will be translated into foreign languages from English for the United States. Bother is versatile in English for the U.S., in that it can describe negative mental states and physical sensations, as well as social disturbances. Bother has many different meanings across cultures, due to this versatility. Alternatives for bother were explored for future PRO instrument development. Methods A PRO instrument used to evaluate the degree of bother resulting from psoriasis was analyzed. This disease can negatively impact patients physically, emotionally and socially. Translations of bother were analyzed to determine its meaning when translated into other languages. Cognitive debriefing was conducted on psoriasis patients with the instrument containing bother. Following cognitive debriefing, a questionnaire was distributed to linguists and cognitive debriefing subjects to collect definitions of bother in each target language, and detail any difficulty with translation. To establish alternatives to bother and demonstrate the breakdown of concepts within bother, translations of the Dermatology Quality of Life Index (DLQI) were analyzed. This instrument was selected for its focus on psoriasis and use of terminology that lacks the ambiguity of bother. Results An analysis of back-translations revealed that bother yielded a back-translation that was conceptually different from the source 20% of the time (5/26). Analysis of alternative terminology found in the DLQI revealed much greater conceptual equivalence when translated into other languages. Conclusion When developing the wording of PRO instruments, the terminology chosen should be applicable across languages to allow for international pooling and comparison of data. While all linguists and subjects of cognitive debriefing understood bother to have a negative connotation, a

  5. Patient-reported outcomes following a physiotherapy rehabilitation programme for atraumatic posterior shoulder subluxation

    PubMed Central

    Mackie, Ann; Wallace, W Angus

    2014-01-01

    Background There is a paucity of research that describes the patient-reported benefits of physiotherapy rehabilitation for atraumatic posterior instability despite non-operative treatment being considered the initial treatment of choice. This retrospective case series review describes the patient-reported outcomes following a physiotherapy rehabilitation programme for atraumatic posterior shoulder instability. Methods Nineteen consecutive patients with a clinical diagnosis of atraumatic posterior shoulder subluxation completed our physiotherapy programme. All patients completed Oxford Instability Shoulder scores (OISS) and Western Ontario Shoulder Instability Index (WOSI) scores before and after physiotherapy intervention. Results Patients reported a statistically significant clinical improvement in the main outcome measures following physiotherapy intervention. The OISS showed a mean improvement of 18.6 points, whereas the WOSI score showed a mean improvement of 37.2%. Following physiotherapy rehabilitation, all patients reported that their shoulder did not prevent them from performing their work/studies or their chosen hobbies/sports. Conclusions Our results support the view that specialized physiotherapy rehabilitation is a valuable treatment option for atraumatic posterior shoulder instability and reveal significant clinically important improvements in patient-reported outcomes.

  6. Assessment of Patient Reported Outcomes (PROs) in Melanoma Patients

    PubMed Central

    Cormier, Janice N.; Askew, Robert L.

    2010-01-01

    SYNOPSIS Assessment of patient reported outcomes (PROs) has been shown to provide important information to assist with clinical decision-making. There has been significant progress in the field of PROs over the last two decades with the introduction of a variety of validated disease- and symptom-specific instruments. The Functional Assessment of Cancer Therapy-Melanoma (FACT-M) is a melanoma-specific module to accompany the FACT-General which has been validated to assess health-related quality of life (HRQOL) for patients with all stages of melanoma. Melanoma-specific health state utilities, which are essential for calculating quality adjusted life years and performing cost-effectiveness studies, have also been reported from a number of studies. Assessment of PROs should be incorporated into routine clinical practice to inform clinicians and researchers of the patient perspective for clinical decision-making and to evaluate the effects of psychosocial and medical interventions. PMID:21111967

  7. Patients’ attitudes and perceptions of two health-related quality-of-life questionnaires used to collect patient-reported outcome measures in the English National Health Service: A qualitative study of patients undergoing cardiac interventions

    PubMed Central

    Hinder, Susan; Steele, Sharon; Gibbons, Elizabeth; Jackson, Mark

    2013-01-01

    Objectives: To explore patients’ views on the EuroQol-5D and Coronary Revascularisation Outcome Questionnaire, tools currently used for collecting patient-reported outcome measures in the English National Health Service. The key questions were as follows: (1) whether patients consider them sensitive enough to detect change in their health after cardiovascular disease interventions and (2) whether they consider the health-related quality-of-life questions as meaningful. Methods: Data were collected on patients’ views using focus groups. We held four focus groups selecting participants on the basis of their baseline and follow-up EuroQol-5D scores. Data were analysed using framework analysis and grounded theory. Results: Focus group participants confirmed that they had derived substantial health benefits from their cardiac interventions despite the lack of measurable effects on the EuroQol-5D scores. Participants felt that the EuroQol-5D questionnaire was limited because of the following reasons: Their health fluctuates from day to day. They had difficulty assessing their general health status on the visual analogue scale. They felt that the Coronary Revascularisation Outcome Questionnaire was limited because of the following reasons: They did not understand the clinical terms used. The impact of tiredness on their quality of life was not captured. They were unable to distinguish between the effects of their heart condition and other health issues. Additionally, neither questionnaire considers the adjustments people have made to their domestic arrangements to improve their health-related quality of life. Conclusion: This study provides evidence that the two questionnaires do not capture some aspects of health that patients consider important. Furthermore, the presence of co-morbidities masks the symptoms relating to the heart disease and the effect of their cardiac interventions. Future work on patient-reported outcome measures should consider developing new

  8. Electronic patient-reported outcome systems in oncology clinical practice.

    PubMed

    Bennett, Antonia V; Jensen, Roxanne E; Basch, Ethan

    2012-01-01

    Patient-reported outcome (PRO) questionnaires assess topics a patient can report about his or her own health. This includes symptoms (eg, nausea, fatigue, diarrhea, pain, or frequent urination), physical functioning (eg, difficulty climbing stairs or difficulty fastening buttons), and mental health (eg, anxiety, fear, or worry). Electronic PRO (ePRO) systems are used in oncology clinical care because of 1) their ability to enhance clinical care by flagging important symptoms and saving clinicians time; 2) the availability of standardized methods for creating and implementing PROs in clinics; and 3) the existence of user-friendly platforms for patient self-reporting like tablet computers and automated telephone surveys. Many ePRO systems can provide actionable links to clinical care such as summary reports in a patient's electronic medical record and real-time e-mail alerts to providers when patients report acute needs. This review presents 5 examples of ePRO systems currently in use in oncology practice. These systems support multiple clinical activities, including assessment of symptoms and toxicities related to chemotherapy and radiation, postoperative surveillance, and symptom management during palliative care and hospice. Patient self-reporting is possible both at clinical visits and between visits over the Internet or by telephone. The implementation of an ePRO system requires significant resources and expertise, as well as user training. ePRO systems enable regular monitoring of patient symptoms, function, and needs, and can enhance the efficiency and quality of care as well as communication with patients. PMID:22811342

  9. Developing core outcomes sets: methods for identifying and including patient-reported outcomes (PROs)

    PubMed Central

    2014-01-01

    Background Synthesis of patient-reported outcome (PRO) data is hindered by the range of available PRO measures (PROMs) composed of multiple scales and single items with differing terminology and content. The use of core outcome sets, an agreed minimum set of outcomes to be measured and reported in all trials of a specific condition, may improve this issue but methods to select core PRO domains from the many available PROMs are lacking. This study examines existing PROMs and describes methods to identify health domains to inform the development of a core outcome set, illustrated with an example. Methods Systematic literature searches identified validated PROMs from studies evaluating radical treatment for oesophageal cancer. PROM scale/single item names were recorded verbatim and the frequency of similar names/scales documented. PROM contents (scale components/single items) were examined for conceptual meaning by an expert clinician and methodologist and categorised into health domains. A patient advocate independently checked this categorisation. Results Searches identified 21 generic and disease-specific PROMs containing 116 scales and 32 single items with 94 different verbatim names. Identical names for scales were repeatedly used (for example, ‘physical function’ in six different measures) and others were similar (overlapping face validity) although component items were not always comparable. Based on methodological, clinical and patient expertise, 606 individual items were categorised into 32 health domains. Conclusion This study outlines a methodology for identifying candidate PRO domains from existing PROMs to inform a core outcome set to use in clinical trials. PMID:24495582

  10. Evaluating and Quantifying User and Carer Involvement in Mental Health Care Planning (EQUIP): Co-Development of a New Patient-Reported Outcome Measure

    PubMed Central

    2016-01-01

    International and national health policy seeks to increase service user and carer involvement in mental health care planning, but suitable user-centred tools to assess the success of these initiatives are not yet available. The current study describes the development of a new reliable and valid, interval-scaled service-user and carer reported outcome measure for quantifying user/carer involvement in mental health care planning. Psychometric development reduced a 70-item item bank to a short form questionnaire using a combination of Classical Test, Mokken and Rasch Analyses. Test-retest reliability was calculated using t-tests of interval level scores between baseline and 2–4 week follow-up. Items were worded to be relevant to both service users and carers. Nine items were removed following cognitive debriefing with a service user and carer advisory group. An iterative process of item removal reduced the remaining 61 items to a final 14-item scale. The final scale has acceptable scalability (Ho = .69), reliability (alpha = .92), fit to the Rasch model (χ2(70) = 97.25, p = .02), and no differential item functioning or locally dependent items. Scores remained stable over the 4 week follow-up period, indicating good test-retest reliability. The ‘Evaluating the Quality of User and Carer Involvement in Care Planning (EQUIP)’ scale displays excellent psychometric properties and is capable of unidimensional linear measurement. The scale is short, user and carer-centred and will be of direct benefit to clinicians, services, auditors and researchers wishing to quantify levels of user and carer involvement in care planning. PMID:26963252

  11. Evaluating and Quantifying User and Carer Involvement in Mental Health Care Planning (EQUIP): Co-Development of a New Patient-Reported Outcome Measure.

    PubMed

    Bee, Penny; Gibbons, Chris; Callaghan, Patrick; Fraser, Claire; Lovell, Karina

    2016-01-01

    International and national health policy seeks to increase service user and carer involvement in mental health care planning, but suitable user-centred tools to assess the success of these initiatives are not yet available. The current study describes the development of a new reliable and valid, interval-scaled service-user and carer reported outcome measure for quantifying user/carer involvement in mental health care planning. Psychometric development reduced a 70-item item bank to a short form questionnaire using a combination of Classical Test, Mokken and Rasch Analyses. Test-retest reliability was calculated using t-tests of interval level scores between baseline and 2-4 week follow-up. Items were worded to be relevant to both service users and carers. Nine items were removed following cognitive debriefing with a service user and carer advisory group. An iterative process of item removal reduced the remaining 61 items to a final 14-item scale. The final scale has acceptable scalability (Ho = .69), reliability (alpha = .92), fit to the Rasch model (χ2(70) = 97.25, p = .02), and no differential item functioning or locally dependent items. Scores remained stable over the 4 week follow-up period, indicating good test-retest reliability. The 'Evaluating the Quality of User and Carer Involvement in Care Planning (EQUIP)' scale displays excellent psychometric properties and is capable of unidimensional linear measurement. The scale is short, user and carer-centred and will be of direct benefit to clinicians, services, auditors and researchers wishing to quantify levels of user and carer involvement in care planning. PMID:26963252

  12. Utility of Patient-Reported Outcome Instruments in Heart Failure.

    PubMed

    Kelkar, Anita A; Spertus, John; Pang, Peter; Pierson, Renee F; Cody, Robert J; Pina, Ileana L; Hernandez, Adrian; Butler, Javed

    2016-03-01

    Patient-reported outcomes (PRO) are defined as reports coming directly from patients about how they feel or function in relation to a health condition and its therapy. Although there are numerous compelling reasons why PRO could be an important help in clinical care, they have not evolved into clinical tools integrated into care. The purpose of this review is to assess existing PRO instruments for heart failure with respect to their psychometric properties and potential for use in clinical care. We performed a systematic search of articles published between July 2008 and January 2015 within the MEDLINE, PROMIS, PROQOLID, and Cochrane Library databases. Included instruments had to be developed and tested for heart failure and have had their development processes and psychometric properties described. A total of 31 instruments were identified, 9 of which met all inclusion criteria. After evaluating each remaining instrument in terms of psychometric and clinical criteria and symptom coverage, only 2 instruments-Minnesota Living with Heart Failure and Kansas City Cardiomyopathy questionnaire-met all evaluation criteria. Although clinically useful PRO instruments exist, increasing education to providers on the value and interpretability of PRO instruments, as well as a more streamlined approach to their implementation in the clinical setting is necessary. A clinical trial comparing the routine use of disease-specific PRO with clinical care could further support their incorporation into practice. PMID:26874386

  13. Designing of Intelligent Multilingual Patient Reported Outcome System (IMPROS)

    PubMed Central

    Pourasghar, Faramarz; Partovi, Yeganeh

    2015-01-01

    Background: By self-reporting outcome procedure the patients themselves record disease symptoms outside medical centers and then report them to medical staff in specific periods of time. One of the self-reporting methods is the application of interactive voice response (IVR), in which some pre-designed questions in the form of voice tracks would be played and then the caller responses the questions by pressing phone’s keypad bottoms. Aim: The present research explains the main framework of such system designing according to IVR technology that is for the first time designed and administered in Iran. Methods: Interactive Voice Response system was composed by two main parts of hardware and software. Hardware section includes one or several digital phone lines, a modem card with voice playing capability and a PC. IVR software on the other hand, acts as an intelligent control center, records call information and controls incoming data. Results: One of the main features of the system is its capability to be administered in common PCs, utilizing simple and cheap modems, high speed to take responses and it’s appropriateness to low literate patients. The system is applicable for monitoring chronic diseases, cancer and also in psychological diseases and can be suitable for taking care of elders and Children who require long term cares. Other features include user-friendly, decrease in direct and indirect costs of disease treatment and enjoying from high level of security to access patients’ profiles. Conclusions: Intelligent multilingual patient reported outcome system (IMPROS) by controlling diseases gives the opportunity to patients to have more participation during treatment and it improves mutual interaction between patient and medical staff. Moreover it increases the quality of medical services, Additional to empowering patients and their followers. PMID:26635441

  14. Comparison of Provider-Assessed and Patient-Reported Outcome Measures of Acute Skin Toxicity During a Phase III Trial of Mometasone Cream Versus Placebo During Breast Radiotherapy: The North Central Cancer Treatment Group (N06C4)

    SciTech Connect

    Neben-Wittich, Michelle A.; Atherton, Pamela J.; Schwartz, David J.; Sloan, Jeff A.; Griffin, Patricia C.; Deming, Richard L.; Anders, Jon C.; Loprinzi, Charles L.; Burger, Kelli N.; Martenson, James A.; Miller, Robert C.

    2011-10-01

    Purpose: Considerable interobserver variability exists among providers and between providers and patients when measuring subjective symptoms. In the recently published Phase III N06C4 trial of mometasone cream vs. placebo to prevent radiation dermatitis, the primary provider-assessed (PA) endpoint, using the Common Toxicity Criteria for Adverse Events (CTCAE), was negative. However, prospectively planned secondary analyses of patient-reported outcomes (PROs), using the Skindex-16 and Skin Toxicity Assessment Tool (STAT), were positive. This study assesses the relationship between PA outcomes and PROs. Methods and Materials: Pearson correlation coefficients were calculated to compare the three tools. Statistical correlations were defined as follows: <0.5, mild; 0.5-0.7, moderate; and >0.7, strong. Results: CTCAE dermatitis moderately correlated with STAT erythema, and CTCAE pruritus strongly correlated with STAT itching. CTCAE pruritus had a moderate correlation with Skindex-16 itching. Comparing the 2 PRO tools, Skindex-16 itching correlated moderately with STAT itching. Skindex-16 burning, hurting, irritation, and persistence all showed the strongest correlation with STAT burning; they showed moderate correlations with STAT itching and tenderness. Conclusions: The PRO Skindex-16 correlated well with the PRO portions of STAT, but neither tool correlated well with CTCAE. PROs delineated a wider spectrum of toxicity than PA measures and provided more information on rash, redness, pruritus, and annoyance measures compared with CTCAE findings of rash and pruritus. PROs may provide a more complete measure of patient experience than single-symptom, PA endpoints in clinical trials assessing radiation skin toxicity.

  15. [Health-related quality of life instruments and other patient-reported outcomes].

    PubMed

    Valderas, José María; Ferrer, Montse; Alonso, Jordi

    2005-12-01

    In the last few years, many different instruments assessing health-related quality of life and the broader concept of Patient-Reported Outcomes (PRO) have been developed. Following the recommendations of the Scientific Advisory Committee of the Medical Outcomes Trust, the ePRO checklist provides a modular assessment for PRO instruments based on eight key attributes: conceptual and measurement model, reliability, validity, responsiveness, interpretability, respondent and administrative burden, alternative forms, and cross-cultural adaptation. Selected criteria describing each attribute in detail are also listed. The availability of explicit guidelines is expected to promote the development of better PRO measurement instruments. PMID:16464429

  16. Prevalence of swallowing and speech problems in daily life after chemoradiation for head and neck cancer based on cut-off scores of the patient-reported outcome measures SWAL-QOL and SHI.

    PubMed

    Rinkel, Rico N; Verdonck-de Leeuw, Irma M; Doornaert, Patricia; Buter, Jan; de Bree, Remco; Langendijk, Johannes A; Aaronson, Neil K; Leemans, C René

    2016-07-01

    The objective of this study is to assess swallowing and speech outcome after chemoradiation therapy for head and neck cancer, based on the patient-reported outcome measures Swallowing Quality of Life Questionnaire (SWAL-QOL) and Speech Handicap Index (SHI), both provided with cut-off scores. This is a cross-sectional study. Department of Otolaryngology/Head and Neck Surgery of a University Medical Center. Sixty patients, 6 months to 5 years after chemoradiation for head and neck squamous cell carcinoma. Swallowing Quality of Life Questionnaire (SWAL-QOL) and SHI, both validated in Dutch and provided with cut-off scores. Associations were tested between the outcome measures and independent variables (age, gender, tumor stage and site, and radiotherapy technique, time since treatment, comorbidity and food intake). Fifty-two patients returned the SWAL-QOL and 47 the SHI (response rate 87 and 78 %, respectively). Swallowing and speech problems were present in 79 and 55 %, respectively. Normal food intake was noticed in 45, 35 % had a soft diet and 20 % tube feeding. Patients with soft diet and tube feeding reported more swallowing problems compared to patients with normal oral intake. Tumor subsite was significantly associated with swallowing outcome (less problems in larynx/hypopharynx compared to oral/oropharynx). Radiation technique was significantly associated with psychosocial speech problems (less problems in patients treated with IMRT). Swallowing and (to a lesser extent) speech problems in daily life are frequently present after chemoradiation therapy for head and neck cancer. Future prospective studies will give more insight into the course of speech and swallowing problems after chemoradiation and into efficacy of new radiation techniques and swallowing and speech rehabilitation programs. PMID:26071622

  17. Is the use of antidepressants associated with patient-reported outcomes following total hip replacement surgery?

    PubMed

    Greene, Meridith E; Rolfson, Ola; Gordon, Max; Annerbrink, Kristina; Malchau, Henrik; Garellick, Göran

    2016-10-01

    Background and purpose - Patients with anxiety and/or depression tend to report less pain reduction and less satisfaction with surgical treatment. We hypothesized that the use of antidepressants would be correlated to patient-reported outcomes (PROs) 1 year after total hip replacement (THR), where increased dosage or discontinuation would be associated with worse outcomes. Patients and methods - THR cases with pre- and postoperative patient-reported outcome measures (PROMs) were selected from the Swedish Hip Arthroplasty Register (n = 9,092; women: n = 5,106). The PROMs were EQ-5D, visual analog scale (VAS) for pain, Charnley class, and VAS for satisfaction after surgery. These cases were merged with a national database of prescription purchases to determine the prevalence of antidepressant purchases. Regression analyses were performed where PROs were dependent variables and sex, age, Charnley class, preoperative pain, preoperative health-related quality of life (HRQoL), patient-reported anxiety/depression, and antidepressant use were independent variables. Results - Antidepressants were used by 10% of the cases (n = 943). Patients using antidepressants had poorer HRQoL and higher levels of pain before and after surgery and they experienced less satisfaction. Preoperative antidepressant use was independently associated with PROs 1 year after THR regardless of patient-reported anxiety/depression. Interpretation - Antidepressant usage before surgery was associated with reduced PROs after THR. Cases at risk of poorer outcomes may be identified through review of the patient's medical record. Clinicians are encouraged to screen for antidepressant use preoperatively, because their use may be associated with PROs after THR. PMID:27482877

  18. Patient-reported outcomes in post-traumatic stress disorder Part I: Focus on psychological treatment

    PubMed Central

    d'Ardenne, Patricia; Heke, Sarah

    2014-01-01

    Since 2000, patient reports have contributed significantly to the widening diagnostic criteria for post-traumatic stress disorder, notably with the inclusion of complex, repeated, and indirect threat to people who develop symptoms. This paper describes and explains why patient reports matter, through worldwide mental health users' movements and the human rights movement. It looks at 46 recent patient-reported outcomes of preferred psychological treatments in clinical research and practice, and compares them with clinician-reported outcomes, using rating scales that diagnose and measure therapeutic gains. Attention is given to one qualitative study of survivors of the London bombings as an example of patients' personal traumatic experiences. Understanding patients' views and their limitations can help increase success in trauma-focused therapy outcomes, particularly where patients fail to engage with or complete treatment, where they doubt the validity of the treatment, or do not see it as culturally appropriate, or fear of revisiting the past. Specific recommendations are made for a more collaborative approach with patients in psychiatric and community care and clinical research. PMID:25152659

  19. Patient-reported outcomes in post-traumatic stress disorder. Part I: focus on psychological treatment.

    PubMed

    d'Ardenne, Patricia; Heke, Sarah

    2014-06-01

    Since 2000, patient reports have contributed significantly to the widening diagnostic criteria for post-traumatic stress disorder, notably with the inclusion of complex, repeated, and indirect threat to people who develop symptoms. This paper describes and explains why patient reports matter, through worldwide mental health users' movements and the human rights movement. It looks at 46 recent patient-reported outcomes of preferred psychological treatments in clinical research and practice, and compares them with clinician-reported outcomes, using rating scales that diagnose and measure therapeutic gains. Attention is given to one qualitative study of survivors of the London bombings as an example of patients' personal traumatic experiences. Understanding patients' views and their limitations can help increase success in trauma-focused therapy outcomes, particularly where patients fail to engage with or complete treatment, where they doubt the validity of the treatment, or do not see it as culturally appropriate, or fear of revisiting the past. Specific recommendations are made for a more collaborative approach with patients in psychiatric and community care and clinical research. PMID:25152659

  20. COMPARISON OF PROVIDER-ASSESSED AND PATIENT-REPORTED OUTCOME MEASURES OF ACUTE SKIN TOXICITY DURING A PHASE III TRIAL OF MOMETASONE CREAM VERSUS PLACEBO DURING BREAST RADIOTHERAPY: THE NORTH CENTRAL CANCER TREATMENT GROUP (N06C4)

    PubMed Central

    Neben-Wittich, Michelle A.; Atherton, Pamela J.; Schwartz, David J.; Sloan, Jeff A.; Griffin, Patricia C.; Deming, Richard L.; Anders, Jon C.; Loprinzi, Charles L.; Burger, Kelli N.; Martenson, James A.; Miller, Robert C.

    2012-01-01

    Purpose Considerable interobserver variability exists among providers and between providers and patients when measuring subjective symptoms. In the recently published Phase III N06C4 trial of mometasone cream vs. placebo to prevent radiation dermatitis, the primary provider–assessed (PA) endpoint, using the Common Toxicity Criteria for Adverse Events (CTCAE), was negative. However, prospectively planned secondary analyses of patient-reported outcomes (PROs), using the Skindex-16 and Skin Toxicity Assessment Tool (STAT), were positive. This study assesses the relationship between PA outcomes and PROs. Methods and Materials Pearson correlation coefficients were calculated to compare the three tools. Statistical correlations were defined as follows: <0.5, mild; 0.5–0.7, moderate; and >0.7, strong. Results CTCAE dermatitis moderately correlated with STATerythema, and CTCAE pruritus strongly correlated with STAT itching. CTCAE pruritus had a moderate correlation with Skindex-16 itching. Comparing the 2 PRO tools, Skindex-16 itching correlated moderately with STAT itching. Skindex-16 burning, hurting, irritation, and persistence all showed the strongest correlation with STAT burning; they showed moderate correlations with STAT itching and tenderness. Conclusions The PRO Skindex-16 correlated well with the PRO portions of STAT, but neither tool correlated well with CTCAE. PROs delineated a wider spectrum of toxicity than PA measures and provided more information on rash, redness, pruritus, and annoyance measures compared with CTCAE findings of rash and pruritus. PROs may provide a more complete measure of patient experience than single-symptom, PA endpoints in clinical trials assessing radiation skin toxicity. PMID:20888137

  1. Patient-reported outcomes in stuttering treatment: conceptual framework.

    PubMed

    Franic, Duska M; Bothe, Anne K

    2008-04-01

    Evaluation of: Yaruss JS, Quesal RW. Overall Assessment of the Speaker's Experience of Stuttering (OASES): documenting multiple outcomes in stuttering treatment. J. Fluency Disord. 31(2), 90-115 (2006) [1] . These authors presented the first complete instrument intended to measure the impact of stuttering in adults who stutter (Overall Assessment of the Speaker's Experience of Stuttering; [OASES]). OASES is a 100-item self-report metric with four sections: general information, reactions to stuttering, communication in daily situations and quality of life. Its conceptual framework includes historic views of the influence of emotional and cognitive variables on stuttering; the WHO's International Classification of Impairments, Disabilities and Handicaps (ICIDH); and the WHO's International Classification of Functioning, Disability and Health (ICF). However, both this conceptual framework and the psychometric data presented to support the OASES are problematic in ways that clinicians and researchers in areas well-beyond stuttering may find informative as they consider their own applications. PMID:20528401

  2. International Society for Quality of Life Research commentary on the draft European Medicines Agency reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies.

    PubMed

    Kyte, Derek; Reeve, Bryce B; Efficace, Fabio; Haywood, Kirstie; Mercieca-Bebber, Rebecca; King, Madeleine T; Norquist, Josephine M; Lenderking, William R; Snyder, Claire; Ring, Lena; Velikova, Galina; Calvert, Melanie

    2016-02-01

    In 2014, the European Medicines Agency (EMA) released for comment a draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies. A twelve-member International Society for Quality of Life Research (ISOQOL) taskforce was convened to coordinate the ISOQOL response. Twenty-one ISOQOL members provided detailed comments and suggestions on the paper: 81 % from academia and 19 % from industry. Taskforce members consolidated and further refined these comments and shared the recommendations with the wider ISOQOL membership. A final response was submitted to the EMA in November 2014. The impending publication of the EMA reflection paper presents a valuable opportunity for ISOQOL to comment on the current direction of EMA PRO guidance and strategy. The EMA paper, although focused on cancer, could serve as a model for using PROs in other conditions, as it provides a useful update surrounding some of the design issues common to all trial research including PRO endpoints. However, we believe there are a number of additional areas in need of greater consideration. The purpose of this commentary is therefore to highlight the strengths of this timely and potentially useful document, but also to outline areas that may warrant further discussion. PMID:26275979

  3. Integrating Patient Reported Outcomes With Clinical Cancer Registry Data: A Feasibility Study of the Electronic Patient-Reported Outcomes From Cancer Survivors (ePOCS) System

    PubMed Central

    Jones, Helen; Thomas, James; Newsham, Alex; Downing, Amy; Morris, Eva; Brown, Julia; Velikova, Galina; Forman, David; Wright, Penny

    2013-01-01

    Background Routine measurement of Patient Reported Outcomes (PROs) linked with clinical data across the patient pathway is increasingly important for informing future care planning. The innovative electronic Patient-reported Outcomes from Cancer Survivors (ePOCS) system was developed to integrate PROs, collected online at specified post-diagnostic time-points, with clinical and treatment data in cancer registries. Objective This study tested the technical and clinical feasibility of ePOCS by running the system with a sample of potentially curable breast, colorectal, and prostate cancer patients in their first 15 months post diagnosis. Methods Patients completed questionnaires comprising multiple Patient Reported Outcome Measures (PROMs) via ePOCS within 6 months (T1), and at 9 (T2) and 15 (T3) months, post diagnosis. Feasibility outcomes included system informatics performance, patient recruitment, retention, representativeness and questionnaire completion (response rate), patient feedback, and administration burden involved in running the system. Results ePOCS ran efficiently with few technical problems. Patient participation was 55.21% (636/1152) overall, although varied by approach mode, and was considerably higher among patients approached face-to-face (61.4%, 490/798) than by telephone (48.8%, 21/43) or letter (41.0%, 125/305). Older and less affluent patients were less likely to join (both P<.001). Most non-consenters (71.1%, 234/329) cited information technology reasons (ie, difficulty using a computer). Questionnaires were fully or partially completed by 85.1% (541/636) of invited participants at T1 (80 questions total), 70.0% (442/631) at T2 (102-108 questions), and 66.3% (414/624) at T3 (148-154 questions), and fully completed at all three time-points by 57.6% (344/597) of participants. Reminders (mainly via email) effectively prompted responses. The PROs were successfully linked with cancer registry data for 100% of patients (N=636). Participant feedback

  4. Impact of natalizumab on patient-reported outcomes in multiple sclerosis: a longitudinal study

    PubMed Central

    2012-01-01

    Background Natalizumab (Tysabri, Biogen Idec and Elan Pharmaceuticals) significantly reduces the relapse rate and disability progression, and improves health-related quality of life (HRQoL), in patients with relapsing-remitting multiple sclerosis. We investigated the impact of natalizumab on patient-reported outcomes (PROs) in a real-world setting. Methods PRO data were collected from patients enrolled in a longitudinal real-world study using validated measures administered as surveys before the patients initiated natalizumab treatment and after the 3rd, 6th, and 12th monthly infusion. HRQoL, ability to carry out daily activities, disability level, and impact on cognitive functioning and fatigue were assessed. Results A total of 333 patients completed 12 months of assessments. After 12 months of natalizumab treatment, 69% to 88% of patients reported a positive outcome (either an improvement or no further decline) in all PRO measures assessed. Significant improvements in general and disease-specific HRQoL were observed after three infusions, both with physical (p < .01) and psychological (p < .001) measures, and were sustained after 12 infusions (all p < .001). The impact of multiple sclerosis on cognitive functioning and fatigue was significantly reduced (both p < .001 after 3 and 12 infusions). Conclusions PRO measures were improved with natalizumab in a real-world setting. The improvements were observed as early as after 3 months and sustained over a 12-month period. The improvements in PROs show that, in clinical practice, the clinical benefits of natalizumab are translated into patient-reported benefits. PMID:23270428

  5. A Comparison of Magnetic Resonance Imaging Muscle Fat Content in the Lumbar Paraspinal Muscles with Patient-Reported Outcome Measures in Patients with Lumbar Degenerative Disk Disease and Focal Disk Prolapse.

    PubMed

    Bhadresha, Ashwin; Lawrence, Owen John; McCarthy, Michael J H

    2016-06-01

    Study Design Retrospective study. Objectives To assess the fatty atrophy of the lumbar paraspinal muscles (LPMs) as determined using magnetic resonance imaging in patients with lumbar degenerative disk disease (DDD) and focal disk herniation and to determine if fatty atrophy is associated with patient-reported outcome measures (PROMS). Methods One hundred sixty-five patients with lumbar DDD were identified from a PROMS database of >1,500 patients. These patients were divided into two study groups: DDD alone (n = 58) and DDD with disk herniation (n = 107). A grid was randomly applied to the axial scans at the L3-L4, L4-L5, and L5-S1 levels. The muscle-to-fat ratio of the LPMs was recorded and compared with PROMS data. Subcutaneous fat thickness at each level was also measured. Results This study found no difference in the muscle-to-fat ratio between the DDD and disk herniation groups. There was no association between the muscle-to-fat ratio and PROMS data in either group. There was significantly more subcutaneous fat at all levels in the DDD group as compared with the disk prolapse group. In DDD and disk prolapses, subcutaneous fat was thicker in women (p = 0.013 and 0.001). In patients with DDD, more subcutaneous fat was associated with disability (p < 0.001). Muscle content of erector spinae and multifidus negatively correlated with increasing age in both groups at the L3-L4 level. Conclusions Muscle fat content in the LPM does not appear to relate to PROMS. Muscle content decreases with age. Those with low back pain (DDD) have greater subcutaneous fat thickness. PMID:27190744

  6. A Comparison of Magnetic Resonance Imaging Muscle Fat Content in the Lumbar Paraspinal Muscles with Patient-Reported Outcome Measures in Patients with Lumbar Degenerative Disk Disease and Focal Disk Prolapse

    PubMed Central

    Bhadresha, Ashwin; Lawrence, Owen John; McCarthy, Michael J. H.

    2016-01-01

    Study Design Retrospective study. Objectives To assess the fatty atrophy of the lumbar paraspinal muscles (LPMs) as determined using magnetic resonance imaging in patients with lumbar degenerative disk disease (DDD) and focal disk herniation and to determine if fatty atrophy is associated with patient-reported outcome measures (PROMS). Methods One hundred sixty-five patients with lumbar DDD were identified from a PROMS database of >1,500 patients. These patients were divided into two study groups: DDD alone (n = 58) and DDD with disk herniation (n = 107). A grid was randomly applied to the axial scans at the L3–L4, L4–L5, and L5–S1 levels. The muscle-to-fat ratio of the LPMs was recorded and compared with PROMS data. Subcutaneous fat thickness at each level was also measured. Results This study found no difference in the muscle-to-fat ratio between the DDD and disk herniation groups. There was no association between the muscle-to-fat ratio and PROMS data in either group. There was significantly more subcutaneous fat at all levels in the DDD group as compared with the disk prolapse group. In DDD and disk prolapses, subcutaneous fat was thicker in women (p = 0.013 and 0.001). In patients with DDD, more subcutaneous fat was associated with disability (p < 0.001). Muscle content of erector spinae and multifidus negatively correlated with increasing age in both groups at the L3–L4 level. Conclusions Muscle fat content in the LPM does not appear to relate to PROMS. Muscle content decreases with age. Those with low back pain (DDD) have greater subcutaneous fat thickness. PMID:27190744

  7. The Association Between Patient-Reported and Objective Oral Anticancer Medication Adherence Measures: A Systematic Review

    PubMed Central

    Atkinson, Thomas M.; Rodríguez, Vivian M.; Gordon, Mallorie; Avildsen, Isabelle K.; Emanu, Jessica C.; Jewell, Sarah T.; Anselmi, Kimberly A.; Ginex, Pamela K.

    2016-01-01

    Problem Identification Oral anticancer medication (OAM) use has been steadily increasing, leading to several patient benefits. A notable challenge for nurses is accurate monitoring of patient OAM regimens because nonadherence is associated with poor health outcomes and decreased survival. Currently, no gold standard measure of OAM adherence exists. The authors conducted a systematic review of the association between objective and patient-reported measures of OAM adherence. Literature Search A systematic electronic literature search was conducted using PubMed, EMBASE, Scopus, PsycINFO®, Cochrane Library, Web of Science, and CINAHL® databases through November 2014. Data Evaluation Articles were independently reviewed to determine whether they included an original characterization of the level of association between objective and patient-reported measures of OAM adherence. Synthesis From a total of 11,135 articles retrieved, eight studies met inclusion criteria. Objective adherence was primarily assessed using pill counts or Medication Event Monitoring System (MEMSCap™). Patient-reported adherence was most commonly assessed using study-specific questionnaires. Significant positive correlations were observed between objective and patient-reported adherence across most studies, with three studies reporting higher rates of adherence via patient reporting. Conclusions Despite variation in the OAMs and measures used, patient-reported adherence rates were equal to or higher than objective adherence measures across studies. Social desirability bias may be a concern; however, given the significant concordance observed, using patient-reported methods in future studies of OAM adherence may be justified. Implications for Nursing This review provides evidence to support nursing use of patient-reported measures to accurately monitor OAM adherence and potentially improve the quality of patient–provider communication. PMID:27541550

  8. Incorporating Patient-Reported Outcomes Into Health Care To Engage Patients And Enhance Care.

    PubMed

    Lavallee, Danielle C; Chenok, Kate E; Love, Rebecca M; Petersen, Carolyn; Holve, Erin; Segal, Courtney D; Franklin, Patricia D

    2016-04-01

    The provision of patient-centered care requires a health care environment that fosters engagement between patients and their health care team. One way to encourage patient-centered care is to incorporate patient-reported outcomes into clinical settings. Collecting these outcomes in routine care ensures that important information only the patient can provide is captured. This provides insights into patients' experiences of symptoms, quality of life, and functioning; values and preferences; and goals for health care. Previously embraced in the research realm, patient-reported outcomes have started to play a role in successful shared decision making, which can enhance the safe and effective delivery of health care. We examine the opportunities for using patient-reported outcomes to enhance care delivery and outcomes as health care information needs and technology platforms change. We highlight emerging practices in which patient-reported outcomes provide value to patients and clinicians and improve care delivery. Finally, we examine present and future challenges to maximizing the use of patient-reported outcomes in the clinic. PMID:27044954

  9. What is important for you? A qualitative interview study of living with diabetes and experiences of diabetes care to establish a basis for a tailored Patient-Reported Outcome Measure for the Swedish National Diabetes Register

    PubMed Central

    Johansson, Unn-Britt; Gudbjörnsdottir, Soffia

    2016-01-01

    Objectives There is a growing emphasis on the perspective of individuals living with diabetes and the need for a more person-centred diabetes care. At present, the Swedish National Diabetes Register (NDR) lacks patient-reported outcome measures (PROMs) based on the perspective of the patient. As a basis for a new PROM, the aim of this study was to describe important aspects in life for adult individuals with diabetes. Design Semistructured qualitative interviews analysed using content analysis. Setting Hospital-based outpatient clinics and primary healthcare clinics in Sweden. Participants 29 adults with type 1 diabetes mellitus (DM) (n=15) and type 2 DM (n=14). Inclusion criteria: Swedish adults (≥18 years) living with type 1 DM or type 2 DM (duration ≥5 years) able to describe their situation in Swedish. Purposive sampling generated heterogeneous characteristics. Results To live a good life with diabetes is demanding for the individual, but experienced barriers can be eased by support from others in the personal sphere, and by professional support from diabetes care. Diabetes care was a crucial resource to nurture the individual's ability and knowledge to manage diabetes, and to facilitate life with diabetes by supplying support, guidance, medical treatment and technical devices tailored to individual needs. The analysis resulted in the overarching theme ‘To live a good life with diabetes’ constituting the two main categories ‘How I feel and how things are going with my diabetes’ and ‘Support from diabetes care in managing diabetes’ including five different categories. Conclusions Common aspects were identified including the experience of living with diabetes and support from diabetes care. These will be used to establish a basis for a tailored PROM for the NDR. PMID:27013595

  10. Feasibility of Frequent Patient-Reported Outcome Surveillance in Patients Undergoing Hematopoietic Cell Transplantation

    PubMed Central

    Wood, William A.; Deal, Allison M.; Abernethy, Amy; Basch, Ethan; Battaglini, Claudio; Kim, Yoon Hie; Whitley, Julia; Shatten, Charlotte; Serody, Jon; Shea, Thomas; Reeve, Bryce B.

    2012-01-01

    Patient-reported outcomes (PROs), including symptoms and health-related quality of life (HRQOL), provide a patient-centered description of hematopoietic cell transplantation (HCT)-related toxicity. These data characterize the patient experience after HCT and may have prognostic usefulness for long-term outcomes after HCT. We conducted a study of 32 patients after HCT (10 autologous HCT recipients, 11 full-intensity conditioning allogeneic HCT recipients, and 11 reduced-intensity conditioning allogeneic HCT recipients) to determine the feasibility of weekly electronic PRO collection from HCT until day (D) + 100. We used questions from the PRO version of the Common Terminology Criteria for Adverse Events to capture symptoms, and the Patient-Reported Outcomes Measurement Information System Global Health scale to measure physical and mental HRQOL. The vast majority (94%) of patients used the electronic PRO system, with only 6% opting for paper-and-pencil only. The median weekly percentage of participants who completed the surveys was 100% in all cohorts through hospital discharge, and remained 100% for the autologous HCT and reduced-intensity allogeneic HCT cohorts through D+100. Patients were satisfied with the electronic system, giving high marks for readability, comfort, and questionnaire length. Symptom severity varied by absolute level and type of symptom across the 3 cohorts, with the full-intensity allogeneic HCT cohort exhibiting the greatest median overall symptom severity, peaking at D+7. Median physical health HRQOL scores decreased with time in the 3 cohorts, and HRQOL was generally correlated with overall symptom severity. Our results demonstrate the feasibility of frequent electronic PROs in the early post-HCT period. Future studies in larger populations to explore predictive models using frequent PRO data for outcomes, including long-term HRQOL and survival, are warranted. PMID:23253558

  11. Prediction of Patient-Reported Outcome After Single-Bundle ACL Reconstruction

    PubMed Central

    Kowalchuk, Deborah A.; Harner, Christopher D.; Fu, Freddie H.; Irrgang, James J.

    2010-01-01

    Purpose To identify pre-operative and intra-operative factors that predict patient-oriented outcome as measured by the IKDC Subjective Knee Form after ACL reconstruction. Methods We identified 402 subjects who had undergone primary single-bundle arthroscopic ACL reconstruction at a mean follow-up of 6.3 years (range 2-15 years). The International Knee Documentation Committee Subjective Knee Form (IKDC) was used to measure patient-reported outcome and was dichotomized as above or below the patient-specific age and gender matched population average. Potential predictor variables included subject demographics, activity level prior to surgery, previous meniscectomy, and surgical variables. Multivariate logistic regression analysis was performed to identify the best subset of predictors for determining the likelihood that the IKDC score was better than the age- and sex-matched population average. Results The dichotomized IKDC score was associated with BMI, smoking status, education, previous medial meniscectomy, and medial chondrosis at the time of ACL reconstruction. The multivariate model containing only factors known before surgery included BMI and smoking status. Subjects with a BMI > 30 had 0.35 times the odds of success than subjects with a normal BMI. Subjects who smoked had 0.36 times the odds of success as subjects who did not smoke. A model including medial chondrosis at the time of surgery had a slightly higher discriminatory power (area under the ROC curve 0.65 versus 0.61) and negative predictive value (71.4 versus 60.0), but similar positive predictive power (86.3 versus 85.9). Conclusions Lower patient-reported outcome following ACL reconstruction was strongly associated with obesity, smoking, and severe chondrosis at time of surgery. PMID:19409302

  12. Framework and guidance for implementing patient-reported outcomes in clinical practice: evidence, challenges and opportunities.

    PubMed

    Porter, Ian; Gonçalves-Bradley, Daniela; Ricci-Cabello, Ignacio; Gibbons, Chris; Gangannagaripalli, Jaheeda; Fitzpatrick, Ray; Black, Nick; Greenhalgh, Joanne; Valderas, Jose M

    2016-08-01

    Patient-reported outcomes (PROs) are reports of the status of a patient's health condition that come directly from the patient. While PRO measures are a well-developed technology with robust standards in research, their use for informing healthcare decisions is still poorly understood. We review relevant examples of their application in the provision of healthcare and examine the challenges associated with implementing PROs in clinical settings. We evaluate evidence for their use and examine barriers to their uptake, and present an evidence-based framework for the successful implementation of PROs in clinical practice. We discuss current and future developments for the use of PROs in clinical practice, such as individualized measurement and computer-adaptive testing. PMID:27427277

  13. Patient-reported outcome and quality of life instruments database (PROQOLID): frequently asked questions.

    PubMed

    Emery, Marie-Pierre; Perrier, Laure-Lou; Acquadro, Catherine

    2005-01-01

    The exponential development of Patient-Reported Outcomes (PRO) measures in clinical research has led to the creation of the Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID) to facilitate the selection process of PRO measures in clinical research. The project was initiated by Mapi Research Trust in Lyon, France. Initially called QOLID (Quality of Life Instruments Database), the project's purpose was to provide all those involved in health care evaluation with a comprehensive and unique source of information on PRO and HRQOL measures available through the Internet.PROQOLID currently describes more than 470 PRO instruments in a structured format. It is available in two levels, non-subscribers and subscribers, at http://www.proqolid.org. The first level is free of charge and contains 14 categories of basic useful information on the instruments (e.g. author, objective, original language, list of existing translations, etc.). The second level provides significantly more information about the instruments. It includes review copies of over 350 original instruments, 120 user manuals and 350 translations. Most are available in PDF format. This level is only accessible to annual subscribers. PROQOLID is updated in close collaboration with the instruments' authors on a regular basis. Fifty or more new instruments are added to the database annually.Today, all of the major pharmaceutical companies, prestigious institutions (such as the FDA, the NIH's National Cancer Institute, the U.S. Veterans Administration), dozens of universities, public institutions and researchers subscribe to PROQOLID on a yearly basis. More than 800 users per day routinely visit the database. PMID:15755325

  14. Composite Pain Index (CPI): Reliability, Validity, and Sensitivity of a Patient-Reported Outcome for Research

    PubMed Central

    Wilkie, Diana J.; Molokie, Robert E.; Suarez, Marie L.; Ezenwa, Miriam O.; Wang, Zaijie J.

    2015-01-01

    Objective A single score that represents the multidimensionality of pain would be an innovation for patient-reported outcomes. Our aim was to determine the reliability, validity, and sensitivity of the Composite Pain Index. Design Methodological analysis of data from a randomized controlled, pretest/posttest education-based intervention study. Setting The study was conducted in outpatient oncology clinics. Subjects The 176 subjects had pain, were 52 ± 12.5 years on average, 63% were female, and 46% had stage IV cancers. Methods We generated the Composite Pain Index from pain location, intensity, quality, and pattern scores measured with an electronic version of Melzack’s McGill Pain Questionnaire. Results The internal consistency values for the individual scores comprising the Composite Pain Index were adequate (.71 baseline, .69 posttest). Principal components analysis extracted one factor with an eigenvalue of 2.17 with explained variance of 54% at baseline and replicated the one factor with an eigenvalue of 2.11 at posttest. The factor loadings for location, intensity, quality, and pattern were .65, .71, .85, and .71 respectively (baseline) and .59, .81, .84, and .63 respectively (posttest). The Composite Pain Index was sensitive to an education intervention effect. Conclusions Findings support the Composite Pain Index as a score that integrates the multidimensional pain experience in people with cancer. It could be used as a patient-reported outcome measure to quantify the complexity of pain in clinical research and population studies of cancer pain and studied for relevance in other pain populations. PMID:25712169

  15. Patient-reported outcomes as end points in clinical trials in rheumatoid arthritis

    PubMed Central

    Gossec, Laure; Dougados, Maxime; Dixon, William

    2015-01-01

    There is a growing interest in patient-reported outcomes (PROs) in rheumatology, which goes with a global trend for more ‘patient-centred care’. This review considers the use of PROs in trials, including their strengths and limitations. In rheumatoid arthritis (RA) trials, the most frequently used PROs to assess treatments include pain, patient global assessment, assessment of functional status, but also health-related quality of life and less commonly fatigue. Other aspects of importance for patients, such as sleep, psychological well-being or ability to cope, are rarely assessed. PROs as outcome measures in RA trials have strengths as well as limitations. PROs have face validity, they are reproducible and sensitive to change and they bring additional information beyond joint counts or acute phase reactants. However, their predictive validity for later outcomes has been little explored, some PROs show redundancy (they bring similar information) and, due to the apparently moderate link between some PROs such as fatigue and the disease process, the use of some PROs to inform treatment choices has been questioned. We suggest the choice of PROs for trials depends on the study objective and on the viewpoint of the stakeholder. There needs to be agreed prioritisation across all stakeholders about what is most important to collect in a trial, which is why a prioritisation and selection process is necessary. Trials in RA will continue to include PROs and their interpretation will become easier as our knowledge progresses. PMID:26509052

  16. Overcoming barriers to implementing patient-reported outcomes in an electronic health record: a case report.

    PubMed

    Harle, Christopher A; Listhaus, Alyson; Covarrubias, Constanza M; Schmidt, Siegfried Of; Mackey, Sean; Carek, Peter J; Fillingim, Roger B; Hurley, Robert W

    2016-01-01

    In this case report, the authors describe the implementation of a system for collecting patient-reported outcomes and integrating results in an electronic health record. The objective was to identify lessons learned in overcoming barriers to collecting and integrating patient-reported outcomes in an electronic health record. The authors analyzed qualitative data in 42 documents collected from system development meetings, written feedback from users, and clinical observations with practice staff, providers, and patients. Guided by the Unified Theory on the Adoption and Use of Information Technology, 5 emergent themes were identified. Two barriers emerged: (i) uncertain clinical benefit and (ii) time, work flow, and effort constraints. Three facilitators emerged: (iii) process automation, (iv) usable system interfaces, and (v) collecting patient-reported outcomes for the right patient at the right time. For electronic health record-integrated patient-reported outcomes to succeed as useful clinical tools, system designers must ensure the clinical relevance of the information being collected while minimizing provider, staff, and patient burden. PMID:26159464

  17. Financial Hardship and Patient-Reported Outcomes after Hematopoietic Cell Transplantation.

    PubMed

    Abel, Gregory A; Albelda, Randy; Khera, Nandita; Hahn, Theresa; Salas Coronado, Diana Y; Odejide, Oreofe O; Bona, Kira; Tucker-Seeley, Reginald; Soiffer, Robert

    2016-08-01

    Although hematopoietic cell transplantation (HCT) is the only curative therapy for many advanced hematologic cancers, little is known about the financial hardship experienced by HCT patients nor the association of hardship with patient-reported outcomes. We mailed a 43-item survey to adult patients approximately 180 days after their first autologous or allogeneic HCT at 3 high-volume centers. We assessed decreases in household income; difficulty with HCT-related costs, such as need to relocate or travel; and 2 types of hardship: hardship_1 (reporting 1 or 2 of the following: dissatisfaction with present finances, difficulty meeting monthly bill payments, or not having enough money at the end of the month) and "hardship_2" (reporting all 3). Patient-reported stress was measured with the Perceived Stress Scale-4, and 7-point scales were provided for perceptions of overall quality of life (QOL) and health. In total, 325 of 499 surveys (65.1%) were received. The median days since HCT was 173; 47% underwent an allogeneic HCT, 60% were male, 51% were > 60 years old, and 92% were white. Overall, 46% reported income decline after HCT, 56% reported hardship_1, and 15% reported hardship_2. In multivariable models controlling for income, those reporting difficulty paying for HCT-related costs were more likely to report financial hardship (odds ratio, 6.9; 95% confidence interval, 3.8 to 12.3). Hardship_1 was associated with QOL below the median (odds ratio, 2.9; 95% confidence interval, 1.7 to 4.9), health status below the median (odds ratio, 2.2; 95% confidence interval, 1.3 to 3.6), and stress above the median (odds ratio, 2.1; 95% confidence interval, 1.3 to 3.5). In this sizable cohort of HCT patients, financial hardship was prevalent and associated with worse QOL and higher levels of perceived stress. Interventions to address patient financial hardship-especially those that ameliorate HCT-specific costs-are likely to improve patient-reported outcomes. PMID:27184627

  18. The importance of patient-reported outcomes: a call for their comprehensive integration in cardiovascular clinical trials.

    PubMed

    Anker, Stefan D; Agewall, Stefan; Borggrefe, Martin; Calvert, Melanie; Jaime Caro, J; Cowie, Martin R; Ford, Ian; Paty, Jean A; Riley, Jillian P; Swedberg, Karl; Tavazzi, Luigi; Wiklund, Ingela; Kirchhof, Paulus

    2014-08-01

    Patient-reported outcomes (PROs), such as symptoms, health-related quality of life (HRQOL), or patient perceived health status, are reported directly by the patient and are powerful tools to inform patients, clinicians, and policy-makers about morbidity and 'patient suffering', especially in chronic diseases. Patient-reported outcomes provide information on the patient experience and can be the target of therapeutic intervention. Patient-reported outcomes can improve the quality of patient care by creating a holistic approach to clinical decision-making; however, PROs are not routinely used as key outcome measures in major cardiovascular clinical trials. Thus, limited information is available on the impact of cardiovascular therapeutics on PROs to guide patient-level clinical decision-making or policy-level decision-making. Cardiovascular clinical research should shift its focus to include PROs when evaluating the efficacy of therapeutic interventions, and PRO assessments should be scientifically rigorous. The European Society of Cardiology and other professional societies can take action to influence the uptake of PRO data in the research and clinical communities. This process of integrating PRO data into comprehensive efficacy evaluations will ultimately improve the quality of care for patients across the spectrum of cardiovascular disease. PMID:24904027

  19. Patient-reported outcomes as primary end points in clinical trials of inflammatory bowel disease.

    PubMed

    Williet, Nicolas; Sandborn, William J; Peyrin-Biroulet, Laurent

    2014-08-01

    The Food and Drug Administration (FDA) is moving from the Crohn's Disease Activity Index to patient-reported outcomes (PROs) and objective measures of disease, such as findings from endoscopy. PROs will become an important aspect of assessing activity of inflammatory bowel disease (IBD) and for labeling specific drugs for this disease. PROs always have been considered in the management of patients with rheumatoid arthritis or multiple sclerosis, and have included measurements of quality of life, disability, or fatigue. Several disease-specific scales have been developed to assess these PROs and commonly are used in clinical trials. Outcomes reported by patients in clinical trials of IBD initially focused on quality of life, measured by the Short-Form 36 questionnaire or disease-specific scales such as the Inflammatory Bowel Disease Questionnaire or its shorter version. Recently considered factors include fatigue, depression and anxiety, and work productivity, as measured by the Functional Assessment Chronic Illness Therapy-Fatigue, the Hospital Anxiety Depression, and the Work Productivity Activity Impairment Questionnaire, respectively. However, few data are available on how treatment affects these factors in patients with IBD. Although disability generally is recognized in patients with IBD, it is not measured. The international IBD disability index currently is being validated. None of the PROs currently used in IBD were developed according to FDA guidance for PRO development. PROs will be a major primary end point of future trials. FDA guidance is needed to develop additional PROs for IBD that can be incorporated into trials, to better compare patients' experience with different therapies. PMID:24534550

  20. Patient-Reported Outcomes in Metastatic Breast Cancer: A Review of Industry-Sponsored Clinical Trials

    PubMed Central

    Krohe, Meaghan; Hao, Yanni; Lamoureux, Roger E.; Galipeau, Nina; Globe, Denise; Foley, Catherine; Mazar, Iyar; Solomon, Jeffrey; Shields, Alan L.

    2016-01-01

    INTRODUCTION Patient-reported outcome (PRO) measures serve to capture vital patient information not otherwise obtained by primary study endpoints. This paper examines how PROs are utilized as endpoints in industry-sponsored metastatic breast cancer clinical trials. METHODS A search was conducted in the clinicaltrials.gov web site for trials involving common treatments for metastatic breast cancer. Thirty-eight clinical trials were identified which included a PRO endpoint in the study, and data were extracted and summarized. RESULTS Overall, 17 unique PRO questionnaires and 14 concepts of measurement were identified as secondary or exploratory endpoints. The Functional Assessment of Cancer Therapy—Breast was the most frequently utilized questionnaire, commonly implemented to assess quality of life. The EORTC QLQ-C30 was also frequently used to measure quality of life or pain. CONCLUSION This review shares insights into the role of PROs in trials for metastatic breast cancer from which treatment developers and other stakeholders can enhance successful implementation of the patient voice into future trials. PMID:27441001

  1. The Impact of Waiting Time on Health Gains from Surgery: Evidence from a National Patient-reported Outcome Dataset.

    PubMed

    Nikolova, Silviya; Harrison, Mark; Sutton, Matt

    2016-08-01

    Reducing waiting times has been a major focus of the English National Health Service for many years, but little is known about the impact on health outcomes. The collection of data on patient-reported outcome measures for all patients undergoing four large-volume procedures facilitates analysis of the impact of waiting times on patient outcomes. The availability of patient-reported outcome measures before and after surgery allows us to estimate the impact of waiting times on the effectiveness of treatment, controlling for pre-surgery health and the endogeneity of waiting times caused by prioritisation with respect to pre-intervention health. We find that waiting time has a negative and statistically significant impact on the health gain from hip and knee replacement surgery and no impact on the effectiveness of varicose vein and hernia surgery. The magnitude of this effect at patient level is small, 0.1% of the outcome measure range for each additional week of waiting. However, the value of this effect is substantially larger than existing estimates of the disutility experienced during the waiting period. The health losses associated with an additional week of waiting for annual populations of hip and knee replacement patients are worth £11.1m and £11.5m, respectively. Copyright © 2015 John Wiley & Sons, Ltd. PMID:26013773

  2. Patient-Reported Outcomes Are Changing the Landscape in Oncology Care: Challenges and Opportunities for Payers

    PubMed Central

    Zagadailov, Erin; Fine, Michael; Shields, Alan

    2013-01-01

    Background A patient-reported outcome (PRO) is a subjective report that comes from a patient without interpretation by a clinician. Because of the increasingly significant role of PROs in the development and evaluation of new medicines, the US Food and Drug Administration (FDA) issued a formal guidance to describe how PRO instruments will be reviewed and evaluated with respect to claims in approved medical product labeling. Meanwhile, PROs continue to appear in oncology clinical trials more frequently; however, it is unclear how payers and policymakers can use PRO data in the context of decision-making for cancer treatments. Objective The objective of this article is to discuss the challenges and opportunities of incorporating oncology-related PRO data into payer decision-making. Discussion Payer concerns with PRO instruments are often related to issues regarding measurement, relevance, quality, and interpretability of PROs. Payers may dismiss PROs that do not independently predict improved outcomes. The FDA guidance released in 2009 demonstrates, as evidenced by the case of ruxolitinib, how PRO questionnaires can be generated in a relevant, trustworthy, and meaningful way, which provides an opportunity for payers and policy decision makers to focus on how to use PRO data in their decision-making. This is particularly relevant in oncology, where a recent and sizable number of clinical trials include PRO measures. Conclusion As an increasing number of oncology medications enter the market with product labeling claims that contain PRO data, payers will need to better familiarize themselves with the opportunities associated with PRO questionnaires when making coverage decisions. PRO measures will continue to provide valuable information regarding the risk–benefit profile of novel agents. As such, PRO measures may provide evidence that should be considered in payers' decisions and discussions; however, the formal role of PROs and the pertinence of PROs in decision

  3. The effectiveness of providing peer benchmarked feedback to hip replacement surgeons based on patient-reported outcome measures—results from the PROFILE (Patient-Reported Outcomes: Feedback Interpretation and Learning Experiment) trial: a cluster randomised controlled study

    PubMed Central

    Boyce, Maria B; Browne, John P

    2015-01-01

    Objective To test whether providing surgeons with peer benchmarked feedback about patient-reported outcomes is effective in improving patient outcomes. Design Cluster randomised controlled trial. Setting Secondary care—Ireland. Participants Surgeons were recruited through the Irish Institute of Trauma and Orthopaedic Surgery, and patients were recruited in hospitals prior to surgery. We randomly allocated 21 surgeons and 550 patients. Intervention Surgeons in the intervention group received peer benchmarked patient-reported outcome measures (PROMs) feedback and education. Main outcome variable Postoperative Oxford Hip Score (OHS). Results Primary outcome data were available for 11 intervention surgeons with responsibility for 230 patients and 10 control surgeons with responsibility for 228 patients. The mean postoperative OHS for the intervention group was 40.8 (95% CI 39.8 to 41.7) and for the control group was 41.9 (95% CI 41.1 to 42.7). The adjusted effect estimate was −1.1 (95% CI −2.4 to 0.2, p=0.09). Secondary outcomes were the Hip Osteoarthritis Outcome Score (HOOS), EQ-5D and the proportion of patients reporting a problem after surgery. The mean postoperative HOOS for the intervention group was 36.2 and for the control group was 37.1. The adjusted effect estimate was −1.1 (95% CI −2.4 to 0.3, p=0.1). The mean postoperative EQ-5D for the intervention group was 0.85 and for the control group was 0.87. The adjusted effect estimate was −0.02 (95% CI −0.05 to 0.008, p=0.2). 27% of intervention patients and 24% of control patients reported at least one complication after surgery (adjusted OR=1.2, 95% CI 0.6 to 2.3, p=0.6). Conclusions Outcomes for patients operated on by surgeons who had received peer benchmarked PROMs data were not statistically different from the outcomes of patients operated on by surgeons who did not receive feedback. PROMs information alone seems to be insufficient to identify opportunities for quality improvement. Trial

  4. Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody–associated Vasculitis Using the OMERACT Process

    PubMed Central

    Robson, Joanna C.; Milman, Nataliya; Tomasson, Gunnar; Dawson, Jill; Cronholm, Peter F.; Kellom, Katherine; Shea, Judy; Ashdown, Susan; Boers, Maarten; Boonen, Annelies; Casey, George C.; Farrar, John T.; Gebhart, Don; Krischer, Jeffrey; Lanier, Georgia; McAlear, Carol A.; Peck, Jacqueline; Sreih, Antoine G.; Tugwell, Peter; Luqmani, Raashid A.; Merkel, Peter A.

    2016-01-01

    Objective Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of linked multisystem life- and organ-threatening diseases. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group has been at the forefront of outcome development in the field and has achieved OMERACT endorsement of a core set of outcomes for AAV. Patients with AAV report as important some manifestations of disease not routinely collected through physician-completed outcome tools; and they rate common manifestations differently from investigators. The core set includes the domain of patient-reported outcomes (PRO). However, PRO currently used in clinical trials of AAV do not fully characterize patients’ perspectives on their burden of disease. The OMERACT vasculitis working group is addressing the unmet needs for PRO in AAV. Methods Current activities of the working group include (1) evaluating the feasibility and construct validity of instruments within the PROMIS (Patient-Reported Outcome Measurement Information System) to record components of the disease experience among patients with AAV; (2) creating a disease-specific PRO measure for AAV; and (3) applying The International Classification of Functioning, Disability and Health to examine the scope of outcome measures used in AAV. Results The working group has developed a comprehensive research strategy, organized an investigative team, included patient research partners, obtained peer-reviewed funding, and is using a considerable research infrastructure to complete these interrelated projects to develop evidence-based validated outcome instruments that meet the OMERACT filter of truth, discrimination, and feasibility. Conclusion The OMERACT vasculitis working group is on schedule to achieve its goals of developing validated PRO for use in clinical trials of AAV. (First Release September 1 2015; J Rheumatol 2015;42:2204–9; doi:10.3899/jrheum.141143) PMID:26329344

  5. Usefulness of Patients-Reported Outcomes in Rheumatoid Arthritis Focus Group

    PubMed Central

    Amaya-Amaya, Jenny; Botello-Corzo, Diana; Calixto, Omar-Javier; Calderón-Rojas, Rolando; Domínguez, Aura-Maria; Cruz-Tapias, Paola; Montoya-Ortiz, Gladis; Mantilla, Ruben-Dario; Anaya, Juan-Manuel; Rojas-Villarraga, Adriana

    2012-01-01

    Objective. Patient-reported outcomes (PROs) have become an essential part of the assessment of patients with rheumatoid arthritis (RA). We aimed to evaluate the agreement and correlation between PROs and the physician's measurements. Methods. This was a cross-sectional analytical study in which 135 patients with RA were clinically evaluated during two different sessions of focus group interviews. Rheumatologist recorded 28 swollen (SJCs) and tender joint counts (TJCs). The patients filled out the PROs instruments (MDHAQ, RADAI, RAPID3, 4, and 5 and self-report articular index (SAI) diagram for pain and joint swelling). DAS28 was calculated (C-reactive protein). An adjusted multiple lineal regression model was done (DAS28 as dependent variable). Results. Highly significant agreements were found between SJC and TJC registered by the physician and patient. There was moderate correlation between DAS28 with patient SJC (r = 0.52), patient TJC (r = 0.55), RADAI (r = 0.56), RAPID3 (r = 0.52), RAPID4 (r = 0.56), RAPID5 (r = 0.66), and VAS-Global (r = 0.51). Likewise, we found moderate to high correlations between CDAI and SDAI with all variable measurements done by the patients. The resulting predictive equation was DAS28(CRP) = 2.02 + 0.037 × RAPID4 + 0.042× patient SJC. Conclusion. PROs applied in focus groups interview are a useful tool for managing patients with RA regardless of gender, educational level, and duration of disease. PMID:23097701

  6. Toward a better understanding of patient-reported outcomes in clinical practice.

    PubMed

    Bitton, Asaf; Onega, Tracy; Tosteson, Anna N A; Haas, Jennifer S

    2014-04-01

    Current shifts toward patient-centered healthcare and accountable payment options point to the more personalized production of better health, not just healthcare, as a next organizational paradigm. Transformation to a system geared toward promoting health requires us to think broadly about what it means to engage patients meaningfully, to give them a voice in their health and care, and to capture more of their varied experience and attitudes beyond the provider visit. The collection and use of patient-reported outcome data into electronic health records represents an important step forward for the transition to a more patient-centered health system. We set out an agenda for better understanding how and when patient-reported outcomes may improve patient health and care experience. PMID:24884859

  7. Patient-reported outcomes and considerations in the management of COPD: focus on aclidinium

    PubMed Central

    Lopez-Campos, Jose Luis; Calero, Carmen; Lopez-Ramirez, Cecilia; Asensio-Cruz, Maria Isabel; Márquez-Martín, Eduardo; Ortega-Ruiz, Francisco

    2015-01-01

    Chronic obstructive pulmonary disease (COPD) is a complex heterogeneous disease, in which several factors combine to give the final clinical expression. Both early and more recent studies have shown that forced expiratory volume in one second (FEV1), despite being an extremely important parameter to predict the progression of the disease, is a poor surrogate marker for symptoms perception. Accordingly, patient-reported outcomes (PROs) have gained popularity as a measure of the impact of treatment from the patients’ perspective, since they represent the individuals’ perception of their health status, beyond any physiological limitations. Several such PROs, therefore, are currently included in multidimensional COPD evaluation. This multidimensional approach helps identify different patient types and individualize, up to a certain point, pharmacological treatment. In this multidimensional approach it is important to highlight the importance of long-acting bronchodilators in COPD treatment strategies. Long-acting bronchodilators are cost-effective and have been shown to achieve the greatest functional and clinical improvements in COPD. As a result, long-acting bronchodilators are now the main pharmacological treatment for COPD at all stages of the disease. Until recently, tiotropium was the leading bronchodilator for the treatment of COPD. The clinical development of this medication, unprecedented in inhaled therapy, involved tens of thousands of patients and yielded consistent outcomes in terms of lung function, symptoms, quality of life, exacerbations, and prognosis. However, new long-acting bronchodilators have recently been developed or are currently under development. In this review, we evaluate the effects of aclidinium bromide, a novel long-acting bronchodilator, on PROs in COPD. Aclidinium is a novel long-acting muscarinic antagonist with a good safety profile for the treatment of COPD, and has proven efficacy in both objective functional measurements and

  8. The Impact of Different Surgical Modalities for Hysterectomy on Satisfaction and Patient Reported Outcomes

    PubMed Central

    2014-01-01

    Background There is an ongoing debate regarding the cost-benefit of different surgical modalities for hysterectomy. Studies have relied primarily on evaluation of clinical outcomes and medical expenses. Thus, a paucity of information on patient-reported outcomes including satisfaction, recovery, and recommendations exists. Objective The objective of this study was to identify differences in patient satisfaction and recommendations by approach to a hysterectomy. Methods We recruited a large, geographically diverse group of women who were members of an online hysterectomy support community. US women who had undergone a benign hysterectomy formed this retrospective study cohort. Self-reported characteristics and experiences were compared by surgical modality using chi-square tests. Outcomes over time were assessed with the Jonkheere-Terpstra trend test. Logistic regression identified independent predictors of patient satisfaction and recommendations. Results There were 6262 women who met the study criteria; 41.74% (2614/6262) underwent an abdominal hysterectomy, 10.64% (666/6262) were vaginal, 27.42% (1717/6262) laparoscopic, 18.94% (1186/6262) robotic, and 1.26% (79/6262) single-incision laparoscopic. Most women were at least college educated (56.37%, 3530/6262), and identified as white, non-Hispanic (83.17%, 5208/6262). Abdominal hysterectomy rates decreased from 68.2% (152/223) to 24.4% (75/307), and minimally invasive surgeries increased from 31.8% (71/223) to 75.6% (232/307) between 2001 or prior years and 2013 (P<.001 all trends). Trends in overall patient satisfaction and recommendations showed significant improvement over time (P<.001).There were differences across the surgical modalities in all patient-reported experiences (ie, satisfaction, time to walking, driving and working, and whether patients would recommend or use the same technique again; P<.001). Significantly better outcomes were evident among women who had vaginal, laparoscopic, and robotic

  9. Collecting, Integrating, and Disseminating Patient-Reported Outcomes for Research in a Learning Healthcare System

    PubMed Central

    Harle, Christopher A.; Lipori, Gloria; Hurley, Robert W.

    2016-01-01

    Introduction: Advances in health policy, research, and information technology have converged to increase the electronic collection and use of patient-reported outcomes (PROs). Therefore, it is important to share lessons learned in implementing PROs in research information systems. Case Description: The purpose of this case study is to describe a novel information system for electronic PROs and lessons learned in implementing that system to support research in an academic health center. The system incorporates freely available and commercial software and involves clinical and research workflows that support the collection, transformation, and research use of PRO data. The software and processes that comprise the system serve three main functions, (i) collecting electronic PROs in clinical care, (ii) integrating PRO data with non-patient generated clinical data, and (iii) disseminating data to researchers through the institution’s research informatics infrastructure, including the i2b2 (Informatics for Integrating Biology and the Bedside) system. Strategies: Our successful design and implementation was driven by three overarching strategies. First, we selected and implemented multiple interfaced technologies to support PRO collection, management, and research use. Second, we aimed to use standardized approaches to measuring PROs, sending PROs between systems, and disseminating PROs. Finally, we focused on using technologies and processes that aligned with existing clinical research information management strategies within our organization. Conclusion: These experiences and lessons may help future implementers and researchers enhance the scale and sustainable use of systems for research use of PROs. PMID:27563683

  10. Patient-reported outcomes and the evolution of adverse event reporting in oncology.

    PubMed

    Trotti, Andy; Colevas, A Dimitrios; Setser, Ann; Basch, Ethan

    2007-11-10

    Adverse event (AE) reporting in oncology has evolved from informal descriptions to a highly systematized process. The Common Terminology Criteria for Adverse Events (CTCAE) is the predominant system for describing the severity of AEs commonly encountered in oncology clinical trials. CTCAE clinical descriptors have been developed empirically during more than 30 years of use. The method of data collection is clinician based. Limitations of the CTC system include potential for incomplete reporting and limited guidance on data analysis and presentation methods. The Medical Dictionary for Regulatory Activities (MedDRA) is a comprehensive medical terminology system used for regulatory reporting and drug labeling. MedDRA does not provide for severity ranking of AEs. CTC-based data presentations are the primary method of AE data reporting used in scientific journals and oncology meetings. Patient-reported outcome instruments (PROs) cover the subjective domain of AEs. Exploratory work suggests PROs can be used with a high degree of patient engagement and compliance. Additional studies are needed to determine how PROs can be used to complement current AE reporting systems. Potential models for integrating PROs into AE reporting are described in this review. AE reporting methods will continue to evolve in response to changing therapies and growing interest in measuring the impact of cancer treatment on health status. Although integration of PROs into AE reporting may ultimately improve the comprehensiveness and quality of collected data, it may also increase the administrative burden and cost of conducting trials. Therefore, care must be used when developing health outcomes and safety data collection plans. PMID:17991931

  11. Patient-Reported Outcome Assessments as Endpoints in Studies in Infectious Diseases.

    PubMed

    Powers, John H; Howard, Kellee; Saretsky, Todd; Clifford, Sarah; Hoffmann, Steve; Llorens, Lily; Talbot, George

    2016-08-15

    The goal of administering medical interventions is to help patients live longer or live better. In keeping with this goal, there has been increasing interest in taking the "voice" of the patient into account during the development process, specifically in the evaluation of treatment benefits of medical interventions, and use of patient-centered outcome data to justify reimbursement. Patient-reported outcomes (PROs) are outcome assessments (OAs) used to define endpoints that can provide direct evidence of treatment benefit on how patients feel or function. When PROs are appropriately developed, they can increase the efficiency and clinical relevance of clinical trials. Several PROs have been developed for OA in specific infectious diseases indications, and more are under development. PROs also hold promise for use in evaluating adherence, adverse effects, satisfaction with care, and routine clinical practice. PMID:27481954

  12. Clinical and Patient-reported Outcomes of a Zirconia Oral Implant: Three-year Results of a Prospective Cohort Investigation.

    PubMed

    Spies, B C; Balmer, M; Patzelt, S B M; Vach, K; Kohal, R J

    2015-10-01

    The objective of this study was to determine the clinical, radiographic, and patient-reported outcomes of a 1-piece alumina-toughened zirconia implant restored with single crowns (SCs) or 3-unit fixed dental prostheses (FDPs) after 3 y of observation. Forty patients received 53 implants, placed in a 1-stage operation with immediate temporization. Finally, 50 implants were restored with 24 SCs and 13 FDPs. To evaluate peri-implant bone loss, standardized radiographs were taken at implant insertion, at final restoration delivery, and after 1 and 3 y. Additionally, several soft tissue parameters and patient-reported outcome measures were evaluated. Linear mixed models with random intercept for each patient and patients as clusters were used to compare subgroups. Three patients did not receive a SC due to early implant loss, and 1 patient died. As a result, 36 patients with 49 implants were followed-up for 3 y, giving a cumulative survival rate of 94.2%. The average marginal bone loss amounted to 0.79 mm (SCs, 0.47 mm; FDPs, 1.07 mm; P < 0.001). After the delivery of the final prosthetic restoration, further bone loss was not statistically significant (0.09 mm; P = 0.700). Probing depth, clinical attachment level, and modified bleeding index increased significantly at the implant sites, whereas gingival recession decreased significantly. Compared with the pretreatment questionnaires, the patient-reported outcome measures showed a permanently improved perception of function, aesthetics, sense, speech and self-esteem. The survival rate of the investigated ceramic implant system seems to be comparable to reported survival rates of titanium implants when immediately restored. The recorded parameters suggest its potential for clinical utilization. PMID:26232388

  13. The role of expectations in patients' reports of post-operative outcomes and improvement following therapy.

    PubMed

    Flood, A B; Lorence, D P; Ding, J; McPherson, K; Black, N A

    1993-11-01

    Outcomes research typically focuses on the technical capabilities associated with treatment that predicts patients' post-therapy outcomes adjusting for health-related factors. Research on the ability of placebo therapy to alter outcomes suggests that a patient's expectations about therapy can also influence outcomes. Few studies have examined the effects of expectations and their implications for assessing outcomes. This study followed 348 patients who had surgery for benign prostatic hyperplasia. Four hypotheses are tested: whether positive expectations about improvement influence: 1) patients' postoperative reports of symptoms; 2) their belief that they have improved; 3) their overall health after treatment; and 4) whether these effects persist during the year following treatment. Using step-wise regression to control for sociodemographic and clinical factors, we found positive expectations did not appear to strongly influence a patient's report of postoperative symptoms or their overall health. However, we found strong support for positive expectations increasing the likelihood of reporting they felt better after surgery, even after controlling for symptom changes. This effect persisted throughout the postoperative year. We conclude that positive expectations result in a more optimistic view of improvement after surgery rather than altering reports of outcomes or health. PMID:7694013

  14. Eczema Trials: Quality of Life Instruments Used and Their Relation to Patient-reported Outcomes. A Systematic Review.

    PubMed

    Heinl, Daniel; Chalmers, Joanne; Nankervis, Helen; Apfelbacher, Christian J

    2016-06-15

    It is unclear which quality of life instruments have thus far been used in eczema trials. Therefore, we aimed to identify these instruments. We searched the Global Resource of Eczema Trials (GREAT) database for reports of randomized controlled trials. Information on patient-reported outcomes, particularly quality of life, was extracted from eligible studies. 287 full texts reporting on 303 trials and 72 abstracts were included. 63/303 studies (20.8%) assessed quality of life and used 18 named and 4 unnamed instruments. The Dermatology Life Quality Index (DLQI), the Children's Dermatology Life Quality Index (CDLQI), the Infant's Dermatitis Quality of Life Index (IDQOL), and the Dermatitis Family Impact (DFI) were the most common measures in adults, children, infants, and caregivers, respectively. In conclusion, only about one fifth of eczema trials include a quality of life measure as outcome. Many different instruments are used, limiting the possibilities of comparing and synthesising individual trials' findings. PMID:26676847

  15. Non-dystrophic myotonia: prospective study of objective and patient reported outcomes

    PubMed Central

    Bundy, Brian; Statland, Jeffrey; Salajegheh, Mohammad; Rayan, Dipa Raja; Venance, Shannon L.; Wang, Yunxia; Fialho, Doreen; Matthews, Emma; Cleland, James; Gorham, Nina; Herbelin, Laura; Cannon, Stephen; Amato, Anthony; Griggs, Robert C.; Hanna, Michael G.; Barohn, Richard J.

    2013-01-01

    .6% of sodium channel participants, which increased post-cooling to 57.6% in sodium channel mutations. In evaluation of patients with clinical and electrical myotonia, despite considerable phenotypic overlap, the presence of eye closure myotonia, paradoxical myotonia, and an increase in short exercise test sensitivity post-cooling suggest sodium channel mutations. Outcomes designed to measure stiffness or the electrophysiological correlates of stiffness may prove useful for future clinical trials, regardless of underlying mutation, and include patient-reported stiffness, bedside manoeuvres to evaluate myotonia, muscle specific quality of life instruments and short exercise testing. PMID:23771340

  16. Patient-Reported Outcomes Following Total Hip Arthroplasty Stratified by Body Mass Index.

    PubMed

    Wu, Eddie S; Cherian, Jeffrey J; Jauregui, Julio J; Robinson, Kristin; Harwin, Steven F; Mont, Michael A

    2016-05-01

    Obese patients undergoing total hip arthroplasty have been shown to have less functional recovery. This study prospectively compared temporal trends in patient-reported outcomes and activity levels between patients with a body mass index (BMI) of less than 30, 30 to 35, and 35 to 40 kg/m(2) after total hip arthroplasty. Patients were evaluated via the Harris Hip Score, Lower Extremity Activity Scale, and Short Form-12 physical and mental components. The results suggest that patients with BMIs of 35 to 40 kg/m(2) might have poorer functional outcomes preoperatively, with function returning more slowly or poor function being sustained and their not reaching other cohorts' levels. Surgeons must counsel these patients regarding functional expectations and the potential for slower functional returns. [Orthopedics. 2016; 39(3):e572-e577.]. PMID:27064775

  17. Web-based patient-reported outcomes in drug safety and risk management: challenges and opportunities?

    PubMed

    Banerjee, Anjan K; Ingate, Simon

    2012-06-01

    Patient-reported outcomes (PROs) from web-based sources are becoming increasingly important, providing opportunities for industry and regulators to understand the benefits and risks of medicines in a real-world context. Although some guidance exists for the use of adverse event (AE) reports from company-sponsored social network sites, this does not cover non-company-sponsored sites. Additionally, there are concerns about the validity of data from social media sources. Techniques for the collection, analysis and reporting of safety data from patients should be defined, and guidelines agreed, to cover PROs and patient-reported adverse drug-related events from more organized sources of patient outcomes. This review considers drivers for web-based PRO adoption in drug safety, the current regulatory framework and potential methodologies, and concludes that there is an urgent unmet need for guidelines on web-based PRO AEs. Stakeholders for the development of any such guidance should include industry, patients, regulators, academic groups and prescribers. PMID:22551007

  18. Programmatic assessment of a university-based implant training program using patient-reported outcomes.

    PubMed

    Al-Sabbagh, Mohanad; Jenkins, Diane W; de Leeuw, Reny; Nihill, Patricia; Robinson, Fonda G; Thomas, Mark V

    2014-11-01

    The University of Kentucky College of Dentistry (UKCD) established an implant training program that provides training in the use of a single implant system, evidence-based diagnostic and treatment protocols (standardized work practices), and a total quality management system (Implant Quality Assurance Program). The aim of this study was to assess the programmatic effectiveness of the UKCD implant training program by reporting the success and survival of implants placed, using patient-reported outcomes and comparing them to previously established benchmarks. A total of 415 patients (963 implants) were interviewed, approximately 50 percent of all qualified patients. The implant survival rate was 97 percent, and 88 percent of the implants were considered successful (as determined by patient-centric criteria). These outcomes were consistent with the program's previously established benchmarks of 90 percent. These results suggest that work standardization (in the form of specific treatment protocols) and the use of a formal, incremental learning system can result in positive patient outcomes. Clinical outcomes should be monitored in academic dental settings as part of clinical process improvement, and these outcomes can provide a means of assessing the effectiveness of the training program. PMID:25362695

  19. Regression models for patient-reported measures having ordered categories recorded on multiple occasions

    PubMed Central

    Preisser, J. S.; Phillips, C.; Perin, J.; Schwartz, T. A.

    2011-01-01

    Objectives The article reviews proportional and partial proportional odds regression for ordered categorical outcomes, such as patient-reported measures, that are frequently used in clinical research in dentistry. Methods The proportional odds regression model for ordinal data is a generalization of ordinary logistic regression for dichotomous responses. When the proportional odds assumption holds for some but not all of the covariates, the lesser known partial proportional odds model is shown to provide a useful extension. Results The ordinal data models are illustrated for the analysis of repeated ordinal outcomes to determine whether the burden associated with sensory alteration following a bilateral sagittal split osteotomy procedure differed for those patients who were given opening exercises only following surgery and those who received sensory retraining exercises in conjunction with standard opening exercises. Conclusions Proportional and partial proportional odds models are broadly applicable to the analysis of cross-sectional and longitudinal ordinal data in dental research. PMID:21070317

  20. Lack of patient-reported outcomes assessment in phase III breast cancer studies: a missed opportunity for informed decision making.

    PubMed

    Blinder, Victoria S

    2014-01-01

    A phase III study comparing capecitabine monotherapy to combination treatment with capecitabine and sunitinib in patients with metastatic breast cancer failed to demonstrate a benefit in terms of progression-free or overall survival. Both regimens were reasonably well tolerated with some differences noted in the specific toxicity profiles. However, the study failed to incorporate an assessment of patient-reported outcomes (PROs) such as self-reported pain, quality of life, or employment outcomes. This is a missed opportunity. If more clinical trials included such measures, they would provide valuable information to patients and clinicians choosing from a wide array of available and otherwise similarly effective systemic therapies for metastatic breast cancer. PMID:25841482

  1. Correlation of Patient-reported Symptom Outcomes and Treadmill Test Outcomes after Treatment for Aortoiliac Claudication

    PubMed Central

    Murphy, Timothy P.; Reynolds, Matthew R.; Cohen, David J.; Regensteiner, Judith G.; Massaro, Joseph M.; Cutlip, Donald E.; Mohler, Emile R.; Cerezo, Joselyn; Oldenburg, Niki C.; Thum, Claudia C.; Goldberg, Suzanne; Hirsch, Alan T.

    2016-01-01

    Purpose To examine the relationship between objective treadmill test outcomes and subjective symptom outcomes among patients with claudication treated with stent revascularization (ST) compared with supervised exercise (SE). Materials and Methods Five scales of the Peripheral Artery Questionnaire and Walking Impairment Questionnaire were correlated with peak walking time and treadmill claudication onset time. Results The correlation between change in disease-specific quality of life (QOL) and change in peak walking time differed according to treatment group, with statistically significant correlations for all five scales for the ST group and weaker trends for the SE group, only one of which was statistically significant. In contrast, improvements in disease-specific QOL correlated well with increases in claudication onset time, with no significant interaction with treatment group for any of the five scales. Conclusions Disease-specific QOL results at 6 months in the CLEVER (Claudication: Exercise Vs. Endoluminal Revascularization) study show that improved maximal treadmill walking in patients with claudication treated with SE correlated poorly with self-reported symptom relief. Conversely, patients treated with ST showed good correlation between improved maximal treadmill walking and self-reported symptom improvement. The correlation between claudication onset time and self-reported symptom relief was good across treatment groups. This finding indicates that traditional objective treadmill test outcomes may not correlate well with symptom relief in patients with claudication. Future studies should investigate these data and improve understanding of patient relevance of traditional objective treadmill-based treatment outcomes. PMID:23906799

  2. Power and Sample Size Determination for the Group Comparison of Patient-Reported Outcomes with Rasch Family Models

    PubMed Central

    Blanchin, Myriam; Hardouin, Jean-Benoit; Guillemin, Francis; Falissard, Bruno; Sébille, Véronique

    2013-01-01

    Background Patient-reported outcomes (PRO) that comprise all self-reported measures by the patient are important as endpoint in clinical trials and epidemiological studies. Models from the Item Response Theory (IRT) are increasingly used to analyze these particular outcomes that bring into play a latent variable as these outcomes cannot be directly observed. Preliminary developments have been proposed for sample size and power determination for the comparison of PRO in cross-sectional studies comparing two groups of patients when an IRT model is intended to be used for analysis. The objective of this work was to validate these developments in a large number of situations reflecting real-life studies. Methodology The method to determine the power relies on the characteristics of the latent trait and of the questionnaire (distribution of the items), the difference between the latent variable mean in each group and the variance of this difference estimated using Cramer-Rao bound. Different scenarios were considered to evaluate the impact of the characteristics of the questionnaire and of the variance of the latent trait on performances of the Cramer-Rao method. The power obtained using Cramer-Rao method was compared to simulations. Principal Findings Powers achieved with the Cramer-Rao method were close to powers obtained from simulations when the questionnaire was suitable for the studied population. Nevertheless, we have shown an underestimation of power with the Cramer-Rao method when the questionnaire was less suitable for the population. Besides, the Cramer-Rao method stays valid whatever the values of the variance of the latent trait. Conclusions The Cramer-Rao method is adequate to determine the power of a test of group effect at design stage for two-group comparison studies including patient-reported outcomes in health sciences. At the design stage, the questionnaire used to measure the intended PRO should be carefully chosen in relation to the studied

  3. Patient-reported outcomes of parenteral somatostatin analogue injections in 195 patients with acromegaly

    PubMed Central

    Strasburger, Christian J; Karavitaki, Niki; Störmann, Sylvère; Trainer, Peter J; Kreitschmann-Andermahr, Ilonka; Droste, Michael; Korbonits, Márta; Feldmann, Berit; Zopf, Kathrin; Sanderson, Violet Fazal; Schwicker, David; Gelbaum, Dana; Haviv, Asi; Bidlingmaier, Martin; Biermasz, Nienke R

    2016-01-01

    Background Long-acting somatostatin analogues delivered parenterally are the most widely used medical treatment in acromegaly. This patient-reported outcomes survey was designed to assess the impact of chronic injections on subjects with acromegaly. Methods The survey was conducted in nine pituitary centres in Germany, UK and The Netherlands. The questionnaire was developed by endocrinologists and covered aspects of acromegaly symptoms, injection-related manifestations, emotional and daily life impact, treatment satisfaction and unmet medical needs. Results In total, 195 patients participated, of which 112 (57%) were on octreotide (Sandostatin LAR) and 83 (43%) on lanreotide (Somatuline Depot). The majority (>70%) of patients reported acromegaly symptoms despite treatment. A total of 52% of patients reported that their symptoms worsen towards the end of the dosing interval. Administration site pain lasting up to a week following injection was the most frequently reported injection-related symptom (70% of patients). Other injection site reactions included nodules (38%), swelling (28%), bruising (16%), scar tissue (8%) and inflammation (7%). Injection burden was similar between octreotide and lanreotide. Only a minority of patients received injections at home (17%) and 5% were self-injecting. Over a third of patients indicated a feeling of loss of independence due to the injections, and 16% reported repeated work loss days. Despite the physical, emotional and daily life impact of injections, patients were satisfied with their treatment, yet reported that modifications that would offer major improvement over current care would be ‘avoiding injections’ and ‘better symptom control’. Conclusion Lifelong injections of long-acting somatostatin analogues have significant burden on the functioning, well-being and daily lives of patients with acromegaly. PMID:26744896

  4. Considerations for Developing Interfaces for Collecting Patient-Reported Outcomes That Allow the Inclusion of Individuals With Disabilities

    PubMed Central

    Harniss, Mark; Amtmann, Dagmar; Cook, Debbie; Johnson, Kurt

    2010-01-01

    PROMIS (Patient-Reported Outcome Measurement Information System) is developing a set of tools for collecting patient reported outcomes, including computerized adaptive testing that can be administered using different modes, such as computers or phones. The user interfaces for these tools will be designed using the principles of universal design to ensure that it is accessible to all users, including those with disabilities. We review the rationale for making health assessment instruments accessible to users with disabilities, briefly review the standards and guidelines that exist to support developers in the creation of user interfaces with accessibility in mind, and describe the usability and accessibility testing PROMIS will conduct with content experts and users with and without disabilities. Finally, we discuss threats to validity and reliability presented by universal design principles. We argue that the social and practical benefits of interfaces designed to include a broad range of potential users, including those with disabilities, seem to outweigh the need for standardization. Suggestions for future research are also included. PMID:17443119

  5. The rationale for patient-reported outcomes surveillance in cancer and a reproducible method for achieving it.

    PubMed

    Smith, Tenbroeck G; Castro, Kathleen M; Troeschel, Alyssa N; Arora, Neeraj K; Lipscomb, Joseph; Jones, Shelton M; Treiman, Katherine A; Hobbs, Connie; McCabe, Ryan M; Clauser, Steven B

    2016-02-01

    Patient-reported outcomes (PROs) measure quality of life, symptoms, patient functioning, and patient perceptions of care; they are essential for gaining a full understanding of cancer care and the impact of cancer on people's lives. Repeatedly captured facility-level and/or population-level PROs (PRO surveillance) could play an important role in quality monitoring and improvement, benchmarking, advocacy, policy making, and research. This article describes the rationale for PRO surveillance and the methods of the Patient Reported Outcomes Symptoms and Side Effects Study (PROSSES), which is the first PRO study to use the American College of Surgeons Commission on Cancer's Rapid Quality Reporting System to identify patients and manage study data flow. The American Cancer Society, the National Cancer Institute, the Commission on Cancer, and RTI International collaborated on PROSSES. PROSSES was conducted at 17 cancer programs that participated in the National Cancer Institute Community Cancer Centers Program among patients diagnosed with locoregional breast or colon cancer. The methods piloted in PROSSES were successful as demonstrated by high eligibility (93%) and response (61%) rates. Differences in clinical and demographic characteristics between respondents and nonrespondents were mostly negligible, with the exception that non-white individuals were somewhat less likely to respond. These methods were consistent across cancer centers and reproducible over time. If repeated and expanded, they could provide PRO surveillance data from patients with cancer on a national scale. PMID:26619031

  6. Patient-Reported Outcomes and Total Health Care Expenditure in Prediction of Patient Satisfaction: Results From a National Study

    PubMed Central

    Zhang, Weiping; Chen, Wei; Bounsanga, Jerry; Cheng, Christine; Franklin, Jeremy D; Crum, Anthony B; Voss, Maren W; Hon, Shirley D

    2015-01-01

    Background Health care quality is often linked to patient satisfaction. Yet, there is a lack of national studies examining the relationship between patient satisfaction, patient-reported outcomes, and medical expenditure. Objective The aim of this study is to examine the contribution of physical health, mental health, general health, and total health care expenditures to patient satisfaction using a longitudinal, nationally representative sample. Methods Using data from the 2010-2011 Medical Expenditure Panel Survey, analyses were conducted to predict patient satisfaction from patient-reported outcomes and total health care expenditures. The study sample consisted of adult participants (N=10,157), with sampling weights representative of 233.26 million people in the United States. Results The results indicated that patient-reported outcomes and total health care expenditure were associated with patient satisfaction such that higher physical and mental function, higher general health status, and higher total health care expenditure were associated with higher patient satisfaction. Conclusions We found that patient-reported outcomes and total health care expenditure had a significant relationship with patient satisfaction. As more emphasis is placed on health care value and quality, this area of research will become increasingly needed and critical questions should be asked about what we value in health care and whether we can find a balance between patient satisfaction, outcomes, and expenditures. Future research should apply big data analytics to investigate whether there is a differential effect of patient-reported outcomes and medical expenditures on patient satisfaction across different medical specialties. PMID:27227131

  7. Patient-reported Outcomes of Acupuncture for Symptom Control in Cancer.

    PubMed

    Thompson, Lora M A; Osian, Sarah Rausch; Jacobsen, Paul B; Johnstone, Peter A S

    2015-06-01

    Acupuncture is increasingly offered as a treatment option for managing cancer-related symptoms. In addition to randomized controlled trials, patient-reported outcomes may be needed to establish treatment effectiveness. This study retrospectively examined the symptoms and the satisfaction ratings of 90 patients receiving acupuncture at an integrative oncology clinic. At least two acupuncture sessions were completed by 72% of the sample. The prevalence rates of fatigue, pain, anxiety, physical distress, emotional distress, and poor quality of life before acupuncture were > 62%. Paired t tests revealed a significant reduction in symptoms from baseline until after the first acupuncture session and after the last session (p < 0.05). Fewer (21%) patients reported nausea, which was significantly reduced after the first session, but not the last session. Reductions represented clinically meaningful differences in 33-41% of patients after the first session and in 41-53% of patients after the last session for all symptoms, except nausea. A small subset of patients (0-8%) reported worsening symptoms after acupuncture. The majority were satisfied with the service. The results of this study suggest that acupuncture may be useful as an adjunct treatment for cancer symptom management. While high-quality trials are still needed to establish the treatment's efficacy, patients may benefit from these primarily safe, low-cost services. PMID:26100066

  8. Patient-reported outcomes before and after treatment of major depressive disorder.

    PubMed

    IsHak, Waguih William; Mirocha, James; Pi, Sarah; Tobia, Gabriel; Becker, Bret; Peselow, Eric D; Cohen, Robert M

    2014-06-01

    Patient reported outcomes (PROs) of quality of life (QoL), functioning, and depressive symptom severity are important in assessing the burden of illness of major depressive disorder (MDD) and to evaluate the impact of treatment. We sought to provide a detailed analysis of PROs before and after treatment of MDD from the large Sequenced Treatment Alternatives to Relieve Depression (STAR*D) study. This analysis examines PROs before and after treatment in the second level of STAR*D. The complete data on QoL, functioning, and depressive symptom severity, were analyzed for each STAR*D level 2 treatment. PROs of QoL, functioning, and depressive symptom severity showed substantial impairments after failing a selective serotonin reuptake inhibitor trial using citalopram (level 1). The seven therapeutic options in level 2 had positive statistically (P values) and clinically (Cohen's standardized differences [Cohen's d]) significant impact on QoL, functioning, depressive symptom severity, and reduction in calculated burden of illness. There were no statistically significant differences between the interventions. However, a substantial proportion of patients still suffered from patient-reported QoL and functioning impairment after treatment, an effect that was more pronounced in nonremitters. PROs are crucial in understanding the impact of MDD and in examining the effects of treatment interventions, both in research and clinical settings. PMID:25152656

  9. Potential of patient-reported outcomes as nonprimary endpoints in clinical trials

    PubMed Central

    2013-01-01

    Background The purpose of this research was to fully explore the impact of endpoint type (primary vs. nonprimary) on decisions related to patient-reported outcome (PRO) labeling claims supported by PRO measures and to determine if nonprimary PRO endpoints are being fully optimized. This review examines the use of PROs as both primary and nonprimary endpoints in support of demonstration of treatment benefit of new molecular entities (NMEs) and biologic license applications (BLAs) in the United States in the years 2000 to 2012. Methods All NMEs and BLAs approved by the Food and Drug Administration (FDA) between January 2000 and June 2012 were identified using the FDA Drug Approval Reports Web page. Generic products granted tentative approvals were excluded. For all identified products, medical review sections from publicly available drug approval packages were reviewed to identify PRO endpoint status. Product labels (indication, clinical trials sections) were reviewed to determine the number and type of PRO claim. Results A total of 308 NMEs/BLAs were identified. Of these, 70 NMEs/BLAs (23%) were granted PRO claims. The majority of product claims were for disease- or condition-specific signs and symptoms. Of the 70 products with PRO claims, a PRO was a primary endpoint for the vast majority (57 [81%]). A total of 19 of the 70 products were granted a PRO claim based on a nonprimary endpoint. While nonprimary endpoints were used most often to support claims of improved signs or symptoms, nonprimary endpoints were much more likely to support claims of higher order impacts. Conclusions Successful PRO labeling claims are typically based on primary endpoints assessing signs and symptoms. Based on this research, studies with PROs as primary endpoints are far more likely to facilitate positive regulatory review and acceptance of PROs in support of labeling claims. Although inclusion of PROs as nonprimary endpoints in clinical trials has its challenges, recent PRO labels

  10. The BODY-Q: A Patient-Reported Outcome Instrument for Weight Loss and Body Contouring Treatments

    PubMed Central

    Cano, Stefan J.; Alderman, Amy; Soldin, Mark; Thoma, Achilles; Robson, Sam; Kaur, Manraj; Papas, Athanasios; Van Laeken, Nancy; Taylor, Valerie H.; Pusic, Andrea L.

    2016-01-01

    Background: Body contouring performed for cosmetic purposes, or after weight loss, has the potential to improve body image and health-related quality of life (HRQL). The BODY-Q is a new patient-reported outcome (PRO) instrument designed to measure patient perceptions of weight loss and/or body contouring. In this article, we describe the psychometric properties of the BODY-Q scales after an international field-test. Methods: Weight loss and body contouring patients from Canada, United States, and United Kingdom were recruited between November 2013 and February 2015. Data were collected using an iPad directly into a web-based application or a questionnaire booklet. Rasch measurement theory analysis was used for item reduction and to examine reliability, validity, and ability to detect change. Results: The sample included 403 weight loss and 331 body contouring patients. Most BODY-Q items had ordered thresholds (134/138) and good item fit. Scale reliability was acceptable, ie, Person separation index >0.70 for 16 scales, Cronbach α ≥0.90 for 18 of 18 scales, and Test–retest ≥0.87 for 17 of 18 scales. Appearance and HRQL scores were lower in participants with more obesity-related symptoms, higher body mass index, and more excess skin and in those pre- versus postoperative body contouring. The 134 weight loss patients who completed the BODY-Q twice, either 6 weeks (weight loss/nonsurgical body contouring program) or 6 months (bariatric program) later, improved significantly on 7 appearance and 4 HRQL scales. Conclusion: The BODY-Q is a clinically meaningful and scientifically sound patient-reported outcome instrument that can be used to measure outcomes in patients who undergo weight loss and/or body contouring. PMID:27200241

  11. Implementation of an ODM and HL7 Compliant Electronic Patient-Reported Outcome System.

    PubMed

    Soto-Rey, Iñaki; Dugas, Martin; Storck, Michael

    2016-01-01

    Interoperability is one of the biggest issues in health informatics despite of the huge effort invested to solve it. Clinical Data Interchange Standards Consortium (CDISC) and Health Level 7 (HL7) are two of the most recognized institutions working on this field. Several systems are becoming compliant with their standards; however, the process to accomplish it is not always straightforward. In this manuscript, we present the successful implementation of the CDISC ODM and HL7 import and export functions for "MoPat", a web-based multi-language electronic patient-reported outcomes system. The system has been evaluated and tested and is currently being used for clinical study and routine data collection, including more than 10.000 patient encounters. PMID:27577417

  12. Patient-reported outcomes after neoadjuvant therapy for rectal cancer: a systematic review.

    PubMed

    Gavaruzzi, Teresa; Lotto, Lorella; Giandomenico, Francesca; Perin, Alessandro; Pucciarelli, Salvatore

    2014-08-01

    Neoadjuvant therapy followed by total mesorectal excision is standard of care for locally advanced rectal cancer. However, this approach has been previously shown to be associated with high rate of morbidity and it may have a negative effect on patients' reported outcomes (PROs). In order to summarize findings on the effect of the neoadjuvant approach on PROs, we systematically reviewed articles published in the last five years. Thirty-five articles met the inclusion criteria. Ten articles compared the effect of surgery with and without neoadjuvant therapy, six articles compared different neoadjuvant therapies, ten articles reported on patients who were all treated with neoadjuvant therapy, and nine articles examined the effect of neoadjuvant therapy in the analyses. The results are summarized by function investigated and critically commented. PMID:24745308

  13. The Equivalence of Remote Electronic and Paper Patient Reported Outcome (PRO) Collection.

    PubMed

    Griffiths-Jones, W; Norton, M R; Fern, E D; Williams, D H

    2014-11-01

    Individual patient level Patient Reported Outcomes (PROs) are increasingly important in clinical practice. Web-based collection enables clinicians to remotely collect scores at regular intervals, away from the clinic setting. In this randomized crossover study, 47 patients, having undergone hip surgery, were allocated to two groups. Group 1 completed the web-based scores followed by the paper equivalents one week later; Group 2 completed the scores the other way around. The Intraclass Correlation Coefficient (ICC) for the Oxford Hip Score was 0.99, 0.98 to 0.99 (ICC, 95% CI) and the ICCs for the other scores were between 0.95 and 0.97. We conclude that remote ePRO collection using this web-based system reveals excellent equivalence to paper PRO collection of the Oxford Hip, McCarthy, UCLA and howRu scores. PMID:25123605

  14. Patient-reported outcomes of brentuximab vedotin in Hodgkin lymphoma and anaplastic large-cell lymphoma

    PubMed Central

    Chen, Robert; Allibone, Suzanne; Bartlett, Nancy L; Brice, Pauline; Chen, Andy; Pose, Katrina; Rich, Lynn; Bonthapally, Vijay; Garfin, Phillip M; Fanale, Michelle

    2016-01-01

    Background Patients with relapsed/refractory (R/R) Hodgkin lymphoma (HL) or R/R systemic anaplastic large-cell lymphoma (sALCL) treated with brentuximab vedotin (BV) experienced high remission rates in two Phase II trials. With increased response rates and survival times, patient-reported outcomes (PROs) and health-related quality of life (HRQoL) are becoming increasingly important and can help inform treatment decisions to enhance care of cancer patients. Objective The objective was to qualitatively assess HRQoL in long-term survivors treated with BV. Methods An eight-question survey assessing PRO-related aspects was developed and fielded to a subset of patients with HL or sALCL who remained in long-term follow-up after completing BV treatment in the two pivotal studies. Results The survey was completed by 25 of 38 patients (12 with HL, 13 with sALCL). The majority of patients reported that their energy level, outlook on life, difficulties with daily activities, ability to participate in physical activities, and overall HRQoL improved compared to those before BV treatment. Limitations Small sample size and lack of a baseline questionnaire or validated assessment instrument limit broad applicability of these findings to large populations of patients with HL or sALCL. Conclusion This is the first report of BV PRO data in R/R HL and sALCL. Given the patients’ poor prognostic outcomes before stem cell transplant, these encouraging results warrant formal evaluation of PRO end points in BV trials. PMID:27103829

  15. The Establishment of the GENEQOL Consortium to Investigate the Genetic Disposition of Patient-Reported Quality-of-Life Outcomes

    PubMed Central

    Sprangers, Mirjam A. G.; Sloan, Jeff A.; Veenhoven, Ruut; Cleeland, Charles S.; Halyard, Michele Y.; Abertnethy, Amy P.; Baas, Frank; Barsevick, Andrea M.; Bartels, Meike; Boomsma, Dorret I.; Chauhan, Cynthia; Dueck, Amylou C.; Frost, Marlene H.; Hall, Per; Klepstad, Pål; Martin, Nicholas G.; Miaskowski, Christine; Mosing, Miriam; Movsas, Benjamin; Van Noorden, Cornelis J. F.; Patrick, Donald L.; Pedersen, Nancy L.; Ropka, Mary E.; Shi, Quiling; Shinozaki, Gen; Singh, Jasvinder A.; Yang, Ping; Zwinderman, Ailko H.

    2010-01-01

    To our knowledge, no comprehensive, interdisciplinary initiatives have been taken to examine the role of genetic variants on patient-reported quality-of-life outcomes. The overall objective of this paper is to describe the establishment of an international and interdisciplinary consortium, the GENEQOL Consortium, which intends to investigate the genetic disposition of patient-reported quality-of-life outcomes. We have identified five primary patient-reported quality-of-life outcomes as initial targets: negative psychological affect, positive psychological affect, self-rated physical health, pain, and fatigue. The first tangible objective of the GENEQOL Consortium is to develop a list of potential biological pathways, genes and genetic variants involved in these quality-of-life outcomes, by reviewing current genetic knowledge. The second objective is to design a research agenda to investigate and validate those genes and genetic variants of patient-reported quality-of-life outcomes, by creating large datasets. During its first meeting, the Consortium has discussed draft summary documents addressing these questions for each patient-reported quality-of-life outcome. A summary of the primary pathways and robust findings of the genetic variants involved is presented here. The research agenda outlines possible research objectives and approaches to examine these and new quality-of-life domains. Intriguing questions arising from this endeavor are discussed. Insight into the genetic versus environmental components of patient-reported quality-of-life outcomes will ultimately allow us to explore new pathways for improving patient care. If we can identify patients who are susceptible to poor quality of life, we will be able to better target specific clinical interventions to enhance their quality of life and treatment outcomes. PMID:19456223

  16. Patient Reported Outcomes for Rotator Cuff Disease - Which PRO Should You Use?

    PubMed Central

    Makhni, Eric Chugh; Hamamoto, Jason Taizo; Higgins, John; Patterson, Taylor; Romeo, Anthony A.; Verma, Nikhil N.

    2016-01-01

    Objectives: Patient reported outcomes (PRO) are important clinical and research tools that are utilized by orthopedic surgeons in order to assess health outcomes following treatment. This is particularly so in the setting of rotator cuff pathology, in which several different validated patient reported outcomes exist. However, multiple recent studies have demonstrated a lack of standardization in the utilization of these scores. Moreover, many of these scores contain numerous components, thereby making them difficult to administer in a busy ambulatory setting. The goal of this study was to quantitatively assess the commonly used PRO for rotator cuff disease in order to identify the most efficient and comprehensive ones available for clinicians. Methods: Fifteen different PROs commonly used for rotator cuff pathology were selected for review. These outcome tools were assessed by the study team and reviewed for comprehensiveness with regards to assessment of pain, strength, activity, motion, and quality of life. The comprehensiveness and efficiency of each tool was evaluated by inclusion of questions addressing each domain. PROs were also evaluated with a focus of pain criteria (night pain, baseline/general pain, pain during activities of daily living, pain during sport, and pain during work). Finally, all PROs were assessed with regards to comprehensiveness in assessing activity scores (motion/stiffness, activities of daily living, sport, and work). Comprehensiveness scores were calculated by dividing the number of domains or subdomains present by the total domains or subdomains possible. Efficiency was calculated by dividing the number of domains present by the number of questions contained in each PRO. Results: The UCLA, Western Ontario Rotator Cuff Index (WORC), Disabilities of the Arm, Shoulder, and Hand (DASH), PENN, Shoulder Rating Questionnaire (SRQ), and Korean Shoulder Score (KSS) had an overall comprehensiveness score of 1.00 indicating all domains were

  17. Birmingham Hip Resurfacing--Patient reported outcomes pre and post 'Metal-on-Metal' media attention.

    PubMed

    Barke, Samuel; Malagelada, Francesc; Stafford, Giles; McMinn, Derek; Field, Richard

    2016-03-01

    We have investigated whether patient reported outcomes provided by patients with Birmingham Hip Resurfacing (BHR) changed after negative media coverage of metal-on-metal (MOM) hip replacement. We also investigated whether patients whose procedures were performed by a designer surgeon behaved differently to those performed elsewhere. 1178 consecutive BHR procedures performed between January 2002 and December 2006, by one of the designer surgeons in his private practice, were reviewed. We also reviewed 402 BHRs undertaken by two non-designer surgeons in both their NHS and private practice. 150 of the latter cohort were undertaken at an NHS hospital and 252 at an independent private hospital. All patients had annual Oxford Hip Scores (OHS) collected. We chose 2007 as pre-"media attention" and compared scores from this year against subsequent years. We found no clinically significant change in OHS between 2007 and subsequent years, at all centres. We conclude that negative media reporting does not appear to have had an impact on patients' perceived outcome after BHR. In consequence, patients who have undergone this type of hip resurfacing and show deterioration should be investigated. PMID:26984649

  18. Setting the vision: Applied patient reported outcomes and smart, connected digital healthcare systems to improve patient-centered outcomes prediction in critical illness

    PubMed Central

    Wysham, Nicholas G.; Abernethy, Amy P.; Cox, Christopher E.

    2014-01-01

    Purpose of review Prediction models in critical illness are generally limited to short-term mortality and uncommonly include patient-centered outcomes. Current outcome prediction tools are also insensitive to individual context or evolution in healthcare practice, potentially limiting their value over time. Improved prognostication of patient-centered outcomes in critical illness could enhance decision making quality in the ICU. Recent Findings Patient-reported outcomes (PROs) have emerged as precise methodological measures of patient-centered variables and have been successfully employed using diverse platforms and technologies, enhancing the value of research in critical illness survivorship and in direct patient care. The learning health system is an emerging ideal characterized by integration of multiple data sources into a smart and interconnected health information technology infrastructure with the goal of rapidly optimizing patient care. We propose a vision of a smart, interconnected learning health system with integrated electronic PROs (ePRO) to optimize patient-centered care including critical care outcome prediction. Summary A learning health system infrastructure integrating ePROs may aid in the management of critical illness associated conditions and yield tools to improve prognostication of patient-centered outcomes in critical illness. PMID:25159475

  19. A review of the most common patient-reported outcomes in COPD--revisiting current knowledge and estimating future challenges.

    PubMed

    Cazzola, Mario; Hanania, Nicola Alexander; MacNee, William; Rüdell, Katja; Hackford, Claire; Tamimi, Nihad

    2015-01-01

    Patient-reported outcome (PRO) measures that quantify disease impact have become important measures of outcome in COPD research and treatment. The objective of this literature review was to comprehensively evaluate psychometric properties of available PRO instruments and the ability of each of them to characterize pharmaceutical treatment effects from published clinical trial evidence. Identified in this study were several PRO measures, both those that have been used extensively in COPD clinical trials (St George's Respiratory Questionnaire and Chronic Respiratory Questionnaire) and new instruments whose full value is still to be determined. This suggests a great need for more information about the patient experience of treatment benefit, but this also may pose challenges to researchers, clinicians, and other important stakeholders (eg, regulatory agencies, pharmaceutical companies) who develop new treatment entities and payers (including but not limited to health technology assessment agencies such as the National Institute for Health and Care Excellence and the Canadian Agency for Drugs and Technologies in Health). The purpose of this review is to enable researchers and clinicians to gain a broad overview of PRO measures in COPD by summarizing the value and purpose of these measures and by providing sufficient detail for interested audiences to determine which instrument may be the most suitable for evaluating a particular research purpose. PMID:25897216

  20. Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods.

    PubMed

    Shields, Alan; Coon, Cheryl; Hao, Yanni; Krohe, Meaghan; Yaworsky, Andrew; Mazar, Iyar; Foley, Catherine; Globe, Denise

    2015-01-01

    This paper describes ways to approach the conceptual and practical challenges associated with interpreting the clinical meaning of scores produced by patient reported outcome (PRO) questionnaires, particularly when used to inform efficacy decisions for regulatory approval for oncology products. Score interpretation estimates are not inherent to PRO questionnaires per se, instead, vary dependent upon sample and study design characteristics. Scores from PRO measures can be interpreted at the individual and group level, and each carries its own set of statistics for evaluating differences. Oncology researchers have a variety of methods and data analytic strategies available to support their score interpretation needs, which should be considered in the context of their a priori knowledge of the target patient population, the hypothesized effects of treatment, the study design and assessment schedule, and the inferences and decisions to be made from the PRO data. PMID:26594897

  1. The Role of Technical Advances in the Adoption and Integration of Patient-Reported Outcomes in Clinical Care

    PubMed Central

    Jensen, Roxanne E.; Rothrock, Nan E.; DeWitt, Esi Morgan; Spiegel, Brennan; Tucker, Carole A.; Crane, Heidi M.; Forrest, Christopher B.; Patrick, Donald L.; Fredericksen, Rob; Shulman, Lisa M.; Cella, David; Crane, Paul K.

    2016-01-01

    Background Patient-reported outcomes (PROs) are gaining recognition as key measures for improving the quality of patient care in clinical care settings. Three factors have made the implementation of PROs in clinical care more feasible: increased use of modern measurement methods in PRO design and validation, rapid progression of technology (e.g., touch screen tablets, Internet accessibility, and electronic health records (EHRs)), and greater demand for measurement and monitoring of PROs by regulators, payers, accreditors, and professional organizations. As electronic PRO collection and reporting capabilities have improved, the challenges of collecting PRO data have changed. Objectives To update information on PRO adoption considerations in clinical care, highlighting electronic and technical advances with respect to measure selection, clinical workflow, data infrastructure, and outcomes reporting. Methods Five practical case studies across diverse healthcare settings and patient populations are used to explore how implementation barriers were addressed to promote the successful integration of PRO collection into the clinical workflow. The case studies address selecting and reporting of relevant content, workflow integration, pre-visit screening, effective evaluation, and EHR integration. Conclusions These case studies exemplify elements of well-designed electronic systems, including response automation, tailoring of item selection and reporting algorithms, flexibility of collection location, and integration with patient health care data elements. They also highlight emerging logistical barriers in this area, such as the need for specialized technological and methodological expertise, and design limitations of current electronic data capture systems. PMID:25588135

  2. Patient-reported outcomes 3 months after spine surgery: is it an accurate predictor of 12-month outcome in real-world registry platforms?

    PubMed

    Parker, Scott L; Asher, Anthony L; Godil, Saniya S; Devin, Clinton J; McGirt, Matthew J

    2015-12-01

    OBJECT The health care landscape is rapidly shifting to incentivize quality of care rather than quantity of care. Quality and outcomes registry platforms lie at the center of all emerging evidence-driven reform models and will be used to inform decision makers in health care delivery. Obtaining real-world registry outcomes data from patients 12 months after spine surgery remains a challenge. The authors set out to determine whether 3-month patient-reported outcomes accurately predict 12-month outcomes and, hence, whether 3-month measurement systems suffice to identify effective versus noneffective spine care. METHODS All patients undergoing lumbar spine surgery for degenerative disease at a single medical institution over a 2-year period were enrolled in a prospective longitudinal registry. Patient-reported outcome instruments (numeric rating scale [NRS], Oswestry Disability Index [ODI], 12-Item Short Form Health Survey [SF-12], EQ-5D, and the Zung Self-Rating Depression Scale) were recorded prospectively at baseline and at 3 months and 12 months after surgery. Linear regression was performed to determine the independent association of 3- and 12-month outcome. Receiver operating characteristic (ROC) curve analysis was performed to determine whether improvement in general health state (EQ-5D) and disability (ODI) at 3 months accurately predicted improvement and achievement of minimum clinical important difference (MCID) at 12 months. RESULTS A total of 593 patients undergoing elective lumbar surgery were included in the study. There was a significant correlation between 3-month and 12-month EQ-5D (r = 0.71; p < 0.0001) and ODI (r = 0.70; p < 0.0001); however, the authors observed a sizable discrepancy in achievement of a clinically significant improvement (MCID) threshold at 3 versus 12 months on an individual patient level. For postoperative disability (ODI), 11.5% of patients who achieved an MCID threshold at 3 months dropped below this threshold at 12 months; 10

  3. Collecting Patient-Reported Outcomes: Lessons from the California Joint Replacement Registry

    PubMed Central

    Chenok, Kate; Teleki, Stephanie; SooHoo, Nelson F.; Huddleston, James; Bozic, Kevin J.

    2015-01-01

    Context: While patient-reported outcomes (PROs) have long been used for research, recent technology advancements make it easier to collect patient feedback and use it for patient care. Despite the promise and appeal of PROs, substantial barriers to widespread adoption remain—including challenges in interpreting privacy regulations, educating patients and physicians about the power that PRO collection can provide to patient-centered care. Case Description: This article describes lessons learned from the California Joint Replacement Registry’s (CJRR) five-year effort to collect PROs from patients undergoing total hip and total knee replacement surgeries. CJRR is a voluntary, multi-institutional registry in California that collects clinical and device information, as well as PROs from patients undergoing total hip arthroplasty (THA) and total knee arthroplasty (TKA) surgeries. Proposed Solutions: The CJRR encountered and developed solutions to overcome several key issues: (1) limitations of electronic PRO collection, (2) challenges in patient recruitment and tracking, (3) challenges in encouraging patients to complete PRO surveys, (4) real and perceived administrative burden to clinic and hospital staff, (5) surgeon engagement, and (6) survey costs. Conclusion: The CJRR’s field experience can inform growing numbers of providers and researchers who seek to more fully understand the impact of care from the patient’s perspective. In addition, the authors believe that these challenges can best be addressed through a combination of policy changes and increased incentives. PMID:26793737

  4. Introduction to patient-reported outcome item banks: issues in minority aging research.

    PubMed

    Templin, Thomas N; Hays, Ron D; Gershon, Richard C; Rothrock, Nan; Jones, Richard N; Teresi, Jeanne A; Stewart, Anita; Weech-Maldonado, Robert; Wallace, Steve

    2013-04-01

    Pre-Conference Workshop in conjunction with the Annual Meeting of the Geriatrics Society of America San Diego Convention Center, San Diego, CA, USA, 14 November 2012 In 2004, the NIH awarded contracts to initiate the development of high-quality psychological and neuropsychological outcome measures for the improved assessment of health-related outcomes. The workshop introduced these measurement development initiatives, the measures created and the NIH-supported resource (Assessment Center) for internet or tablet-based test administration and scoring. Presentations covered item response theory and assessment of test bias, construction of item banks and computerized adaptive testing, and the different ways in which qualitative analyses contribute to the definition of construct domains and the refinement of outcome constructs. The panel discussion included questions about representativeness of samples and the assessment of cultural bias. PMID:23570428

  5. Why Measure Outcomes?

    PubMed

    Kuhn, John E

    2016-01-01

    The concept of measuring the outcomes of treatment in health care was promoted by Ernest Amory Codman in the early 1900s, but, until recently, his ideas were generally ignored. The forces that have advanced outcome measurement to the forefront of health care include the shift in payers for health care from the patient to large insurance companies or government agencies, the movement toward assessing the care of populations not individuals, and the effort to find value (or cost-effective treatments) amid rising healthcare costs. No ideal method exists to measure outcomes, and the information gathered depends on the reason the outcome information is required. Outcome measures used in research are best able to answer research questions. The methods for assessing physician and hospital performance include process measures, patient-experience measures, structure measures, and measures used to assess the outcomes of treatment. The methods used to assess performance should be validated, be reliable, and reflect a patient's perception of the treatment results. The healthcare industry must measure outcomes to identify which treatments are most effective and provide the most benefit to patients. PMID:27049223

  6. Effect of Sleep Disturbances on Quality of Life, Diabetes Self-Care Behavior, and Patient-Reported Outcomes.

    PubMed

    Chasens, Eileen R; Luyster, Faith S

    2016-02-01

    IN BRIEF Poor sleep quality and sleep disorders, particularly insomnia, obstructive sleep apnea, and restless legs syndrome, are prevalent among people with type 2 diabetes. Evidence suggests that coexisting diabetes and sleep disturbances are associated with decreases in quality of life, diabetes self-care behaviors, and patient-reported outcomes. Additional research is required to determine the effect of treatment of sleep disorders on patient-centered outcomes in people with type 2 diabetes. PMID:26912961

  7. Income and patient-reported outcomes (PROs) after primary total knee arthroplasty

    PubMed Central

    2013-01-01

    Background To assess whether income is associated with patient-reported outcomes (PROs) after primary total knee arthroplasty (TKA). Methods We used prospectively collected data from the Mayo Clinic Total Joint Registry to assess the association of income with index knee functional improvement, moderate to severe pain and moderate to severe activity limitation at 2-year and 5-year follow-up after primary TKA using multivariable-adjusted logistic regression analyses. Results There were 7, 139 primary TKAs at 2 years and 4, 234 at 5 years. In multivariable-adjusted analyses, at 2-year follow-up, compared to income > US$45, 000, lower incomes of ≤ US$35, 000 and > US$35, 000 to 45, 000 were associated (1) significantly with moderate to severe pain with an odds ratio (OR) 0.61 (95% CI 0.40 to 0.94) (P = 0.02) and 0.68 (95% CI 0.49 to 0.94) (P = 0.02); and (2) trended towards significance for moderate to severe activity limitation with OR 0.78 (95% CI 0.60 to 1.02) (P = 0.07) and no significant association with OR 0.96 (95% CI 0.78 to 1.20) (P = 0.75), respectively. At 5 years, odds were not statistically significantly different by income, although numerically they favored lower income. In multivariable-adjusted analyses, overall improvement in knee function was rated as 'better' slightly more often at 2 years by patients with income in the ≤ US$35, 000 range compared to patients with income > US$45, 000, with an OR 1.9 (95% CI 1.0 to 3.6) (P = 0.06). Conclusions We found that patients with lower income had better pain outcomes compared to patients with higher income. There was more improvement in knee function, and a trend towards less overall activity limitation after primary TKA in lower income patients compared to those with higher incomes. Insights into mediators of these relationships need to be investigated to understand how income influences outcomes after TKA. PMID:23497272

  8. Patient-reported outcomes: pathways to better health, better services, and better societies.

    PubMed

    Black, N; Burke, L; Forrest, C B; Sieberer, U H Ravens; Ahmed, S; Valderas, J M; Bartlett, S J; Alonso, J

    2016-05-01

    While the use of PROs in research is well established, many challenges lie ahead as their use is extended to other applications. There is consensus that health outcome evaluations that include PROs along with clinician-reported outcomes and administrative data are necessary to inform clinical and policy decisions. The initiatives presented in this paper underline evolving recognition that PROs play a unique role in adding the patient perspective alongside clinical (e.g., blood pressure) and organizational (e.g., admission rates) indicators for evaluating the effects of new products, selecting treatments, evaluating quality of care, and monitoring the health of the population. In this paper, we first explore the use of PRO measures to support drug approval and labeling claims. We critically evaluate the evidence and challenges associated with using PRO measures to improve healthcare delivery at individual and population levels. We further discuss the challenges associated with selecting from the abundance of measures available, opportunities afforded by agreeing on common metrics for constructs of interest, and the importance of establishing an evidence base that supports integrating PRO measures across the healthcare system to improve outcomes. We conclude that the integration of PROs as a key end point within individual patient care, healthcare organization and program performance evaluations, and population surveillance will be essential for evaluating whether increased healthcare expenditure is translating into better health outcomes. PMID:26563251

  9. Measuring Course Learning Outcomes

    ERIC Educational Resources Information Center

    Keshavarz, Mohsen

    2011-01-01

    Accreditation criteria of programs require effective learning outcomes, assessment with documented procedures, tools, results, and actions to close the assessment loop with broad faculty involvement. This article describes a methodology for providing quantitative measurement of a course's learning outcomes. The methodology uses a linkage matrix…

  10. The relationship between pain severity and patient-reported outcomes among patients with chronic low back pain in Japan

    PubMed Central

    Montgomery, William; Vietri, Jeffrey; Shi, Jing; Ogawa, Kei; Kariyasu, Sawako; Alev, Levent; Nakamura, Masaya

    2016-01-01

    Objective The aim of this study was to quantify the impact of pain severity on patient-reported outcomes among individuals diagnosed with chronic low back pain in Japan. Methods Data were provided by the 2012 Japan National Health and Wellness Survey (N=29,997), a web-based survey of individuals in Japan aged ≥18 years. This analysis included respondents diagnosed with low back pain of ≥3-month duration. Measures included the revised Medical Outcomes Study 36-Item Short-Form Survey Instrument, the Patient Health Questionnaire-9, the Generalized Anxiety Disorder-7 scale, the Work Productivity and Activity Impairment: General Health questionnaire, and self-reported all-cause health care visits (6 months). Generalized linear models were used to assess the relationship between outcomes and severity of pain in the past week as reported on a numeric rating scale ranging from 0 (no pain) to 10 (pain as bad as you can imagine), controlling for length of diagnosis, sociodemographics, and general health characteristics. Results A total of 290 respondents were included in the analysis; mean age was 56 years, 41% were females, and 56% were employed. Pain severity was 3/10 for the first quartile, 5/10 for the median, and 7/10 for the third quartile of this sample. Increasing severity was associated with lower scores for mental and physical component summaries and Short-Form 6D health utility, higher depression (Patient Health Questionnaire-9) and anxiety (Generalized Anxiety Disorder-7) scores, greater absenteeism and presenteeism, greater activity impairment, and more health care provider visits (all P<0.0001). Conclusion The impact of chronic low back pain on health-related quality of life, depression and anxiety symptoms, impairment to work and daily activities, and health care use increases with the severity of pain. Interventions reducing the severity of pain may improve numerous health outcomes even if the pain cannot be eliminated. PMID:27330326

  11. Current Trends in the Use of Patient-Reported Outcome Instruments in Degenerative Cervical Spine Surgery.

    PubMed

    Ueda, Haruki; Cutler, Holt S; Guzman, Javier Z; Cho, Samuel K

    2016-05-01

    Study Design Bibliometric analysis. Objective To determine trends, frequency, and distribution of patient-reported outcome instruments (PROIs) in degenerative cervical spine surgery literature over the past decade. Methods A search was conducted via PubMed from 2004 to 2013 on five journals (The Journal of Bone and Joint Surgery, The Bone and Joint Journal, The Spine Journal, European Spine Journal, and Spine), which were chosen based on their impact factors and authors' consensus. All abstracts were screened and articles addressing degenerative cervical spine surgery using PROIs were included. Articles were then analyzed for publication date, study design, journal, level of evidence, and PROI trends. Prevalence of PROIs and level of evidence of included articles were analyzed. Results From 19,736 articles published, 241 articles fulfilled our study criteria. Overall, 53 distinct PROIs appeared. The top seven most frequently used PROIs were: Japanese Orthopaedic Association score (104 studies), visual analog scale for pain (100), Neck Disability Index (72), Short Form-36 (38), Nurick score (25), Odom criteria (21), and Oswestry Disability Index (15). Only 11 PROIs were used in 5 or more articles. Thirty-three of the PROIs were appeared in only 1 article. Among the included articles, 16% were of level 1 evidence and 32% were of level 4 evidence. Conclusion Numerous PROIs are currently used in degenerative cervical spine surgery. A consensus on which instruments to use for a given diagnosis or procedure is lacking and may be necessary for better communication and comparison, as well as for the accumulation and analysis of vast clinical data across multiple studies. PMID:27099815

  12. Current Trends in the Use of Patient-Reported Outcome Instruments in Degenerative Cervical Spine Surgery

    PubMed Central

    Ueda, Haruki; Cutler, Holt S.; Guzman, Javier Z.; Cho, Samuel K.

    2015-01-01

    Study Design Bibliometric analysis. Objective To determine trends, frequency, and distribution of patient-reported outcome instruments (PROIs) in degenerative cervical spine surgery literature over the past decade. Methods A search was conducted via PubMed from 2004 to 2013 on five journals (The Journal of Bone and Joint Surgery, The Bone and Joint Journal, The Spine Journal, European Spine Journal, and Spine), which were chosen based on their impact factors and authors' consensus. All abstracts were screened and articles addressing degenerative cervical spine surgery using PROIs were included. Articles were then analyzed for publication date, study design, journal, level of evidence, and PROI trends. Prevalence of PROIs and level of evidence of included articles were analyzed. Results From 19,736 articles published, 241 articles fulfilled our study criteria. Overall, 53 distinct PROIs appeared. The top seven most frequently used PROIs were: Japanese Orthopaedic Association score (104 studies), visual analog scale for pain (100), Neck Disability Index (72), Short Form-36 (38), Nurick score (25), Odom criteria (21), and Oswestry Disability Index (15). Only 11 PROIs were used in 5 or more articles. Thirty-three of the PROIs were appeared in only 1 article. Among the included articles, 16% were of level 1 evidence and 32% were of level 4 evidence. Conclusion Numerous PROIs are currently used in degenerative cervical spine surgery. A consensus on which instruments to use for a given diagnosis or procedure is lacking and may be necessary for better communication and comparison, as well as for the accumulation and analysis of vast clinical data across multiple studies. PMID:27099815

  13. Comparative Effectiveness of Etanercept and Adalimumab in Patient Reported Outcomes and Injection-Related Tolerability

    PubMed Central

    Navarro-Millán, Iris; Herrinton, Lisa J.; Chen, Lang; Harrold, Leslie; Liu, Liyan; Curtis, Jeffrey R.

    2016-01-01

    Objective To describe patient preferences in selecting specific biologics and compare clinical response using patient reported outcomes (PROs) among patients with rheumatoid arthritis (RA) started on different anti-tumor necrosis factor (TNF) therapies. Methods Participants were enrollees in Kaiser Permanente Northern California. Patients with RA who had at least two provider visits and started a new anti-TNF therapy from 10/2010–8/2011, were eligible for participation in this longitudinal study. Using a telephone survey, patient preferences in biologic selection and RAPID3, MDHAQ, and SF-12 scores were collected at baseline and at 6 months. Patient scores rating injection/infusion-site burning and stinging (ISBS) were collected at 6 months. Results In all, 267 patients with RA responded to the baseline survey, of whom 57% preferred an injectable biologic, 22% preferred an infused biologic, and 21% had no preference. Motivation for injectable biologics was convenience (92%) and for infusion therapy was dislike or lack of self-efficacy for self-injection (16%). After 6 months of treatment with anti-TNF, 70% of the 177 patients who answered the ISBS question reported ISBS with the last dose; on a scale of 1 (none) to 10 (worst), 41% of these reported a score of 2–5; and 29% reported a score of 6–10. Adalimumab users experienced 3.2 times (95% confidence interval 1.2–8.6) the level of ISBS that etanercept users experienced. There were no significant differences in RAPID3, MDHAQ, or SF-12 scores between etanercept or adalimumab initiators. Conclusion Convenience and fear of self-injection were important considerations to patients selecting a biologic drug. Although more convenient, adalimumab associated with more ISBS than did etanercept, and this rate was higher than reported in clinical trials. At 6 months, PROs did not differ between etanercept and adalimumab users. PMID:27007811

  14. Review of Electronic Patient-Reported Outcomes Systems Used in Cancer Clinical Care

    PubMed Central

    Jensen, Roxanne E.; Snyder, Claire F.; Abernethy, Amy P.; Basch, Ethan; Potosky, Arnold L.; Roberts, Aaron C.; Loeffler, Deena R.; Reeve, Bryce B.

    2014-01-01

    Purpose: The use of electronic patient-reported outcomes (PRO) systems is increasing in cancer clinical care settings. This review comprehensively identifies existing PRO systems and explores how systems differ in the administration of PRO assessments, the integration of information into the clinic workflow and electronic health record (EHR) systems, and the reporting of PRO information. Methods: Electronic PRO (e-PRO) systems were identified through a semistructured review of published studies, gray literature, and expert identification. System developers were contacted to provide detailed e-PRO system characteristics and clinical implementation information using a structured review form. Results: A total of 33 unique systems implemented in cancer clinical practice were identified. Of these, 81% provided detailed information about system characteristics. Two system classifications were established: treatment-centered systems designed for patient monitoring during active cancer treatment (n = 8) and patient-centered systems following patients across treatment and survivorship periods (n = 19). There was little consensus on administration, integration, or result reporting between these system types. Patient-centered systems were more likely to provide user-friendly features such as at-home assessments, integration into larger electronic system networks (eg, EHRs), and more robust score reporting options. Well-established systems were more likely to have features that increased assessment flexibility (eg, location, automated reminders) and better clinical integration. Conclusion: The number of e-PRO systems has increased. Systems can be programmed to have numerous features that facilitate integration of PRO assessment and routine monitoring into clinical care. Important barriers to system usability and widespread adoption include assessment flexibility, clinical integration, and high-quality data collection and reporting. PMID:24301843

  15. Communicating the results of randomized clinical trials: do patients understand multidimensional patient-reported outcomes?

    PubMed

    McNair, Angus G K; Brookes, Sara T; Davis, Christopher R; Argyropoulos, Miltiadis; Blazeby, Jane M

    2010-02-10

    PURPOSE Evidence suggests that patient-reported outcomes (PROs) from randomized trials in oncology may not influence clinical decision making and patient choice. Reasons for this are currently unclear and little is known about patients' interpretation of PROs. This study assessed patients' understanding of multidimensional PROs in a graphical format. PATIENTS AND METHODS Semistructured interviews in which patients interpreted a series of graphs depicting simple, then multiple different hypothetical PROs associated with two treatments with identical chances of survival were audio recorded. The interviewer and a blinded observer (listening to audio recordings) scored patients' understanding of the graphs. Logistic regression examined the associations between patient understanding of the graphs and clinical and sociodemographic details. Results One hundred thirty-two patients with esophageal and gastric cancer were interviewed and 115 understood the first two graphs depicting different PROs of two treatments (87%; 95% CI,81 to 93). Simultaneous interpretation of adverse and beneficial treatment effects was achieved by 74 (66%; 95% CI, 57 to 75). Graphs showing complex, longitudinal data were correctly interpreted by 97 (73%; 95% CI, 66 to 81) and 108 (81%; 95% CI, 75 to 88), respectively. Univariable analyses demonstrated associations between patient understanding and patient age, educational level, and cancer site (P < or = .02 for all); however, in a multivariable model each of these associations was attenuated. CONCLUSION Most patients understand graphical multidimensional PROs, although a smaller majority were able to interpret more complex, or simultaneous, presentations. Additional work is needed to define methods for communicating clinical and PRO data from trials to allow patients to make informed treatment choices. PMID:20065187

  16. Developing core patient-reported outcomes in maternity: PRO-Maternity.

    PubMed

    Mahmud, A; Morris, E; Johnson, S; Ismail, K M

    2014-09-01

    Women-centred maternity PRO measures can generate outcome measures that will allow benchmarking of service delivery using meaningful outcomes that will drive service improvement to ensure commitment to a culture of openness, patient satisfaction, patient safety and the delivery of clinically effective care. Moreover, they can generate new insights into women’s perspectives of healthcare delivery, allowing changes in attitude, practice and organisational services. Once developed, the adoption of PRO measures within units is likely to come at additional cost. However, the expected benefits in improved woman and family satisfaction, reduced legal claims and the presence of an early indicator of compromised quality of care will have the potential to save a significant amount of money for individual units and the wider health economy. PMID:25236628

  17. Stigma among patients with lung cancer: A patient-reported measurement model

    PubMed Central

    Hamann, Heidi A.; Ostroff, Jamie S.; Marks, Emily G.; Gerber, David E.; Schiller, Joan H.; Craddock Lee, Simon J.

    2014-01-01

    Objective Although stigma may have negative psychosocial and behavioral outcomes for patients with lung cancer, its measurement has been limited. A conceptual model of lung cancer stigma and a patient-reported outcome (PRO) measure is needed to mitigate these sequelae. This study identified key stigma-related themes to provide a blueprint for item development through thematic analysis of semi-structured interviews and focus groups with lung cancer patients. Methods Participants were recruited from two outpatient oncology clinics and included: a) 42 lung cancer patients who participated in individual interviews and, b) 5 focus groups (inclusive of 23 new lung cancer patients). Never smokers, long-term quitters, recent quitters, and current smokers participated. Individual interviews facilitated theme development and a conceptual model of lung cancer stigma, whereas subsequent focus groups provided feedback on the conceptual model. Qualitative data analyses included iterative coding and validation with existing theory. Results Two main thematic elements emerged from interviews with lung cancer patients: perceived (felt) stigma and internalized (self) stigma. Discussions of perceived stigma were pervasive, while internalized stigma was more commonly endorsed among current and recently quit smokers. Participants also discussed maladaptive (e.g., decreased disclosure) and adaptive (e.g., increased advocacy) stigma-related consequences. Conclusions Results indicate widespread acknowledgment of perceived stigma among lung cancer patients, but varying degrees of internalized stigma and associated consequences. Next steps for PRO measure development are item consolidation, item development, expert input, and cognitive interviews before field testing and psychometric analysis. Future work should address stigma-related consequences and interventions for reducing lung cancer stigma. PMID:24123664

  18. Patient-reported outcomes of caries prophylaxis among Swedish caries active adults in a long-term perspective.

    PubMed

    Flink, Håkan; Tegelberg, Åke; Arnetz, Judy; Birkhed, Dowen

    2016-01-01

    The aim of this study was to measure patient-reported outcomes of caries prophylaxis and to compare them with previously documented efforts in dental offices. A questionnaire was mailed to 134 caries active (CA) and 40 caries inactive (CI) adult patients treated at a Swedish public dental service clinic. The overall response rate was 69%. The questionnaire included items regarding patient perceived caries prophylaxis in relation to: 1) treatment and recommendations given by the dental personnel, 2) performed self-care and 3) perceived and expected effects.The responses were studied for their association to clinical data, extracted retrospectively from the patients' dental records.The mean follow up time was > 16 years. Information about caries prophylaxis (p = 0.01) and recommendations for self-care (p = 0.04) were given more often to the CA group than to the CI group. Supplementary examinations and recommendations of self-care risk treatments were more frequent in the CA group (p < 0.001). CA patients also made more frequent extra efforts at home to avoid caries by changing their eating habits (p < 0.001), improving their oral hygiene (p = 0.04) and using extra fluoride (p = 0.001). In the CA group, 60% did not considerthat the extra prophylaxis efforts had made them caries inactive, and 40% were not satisfied with the outcome. Most patients (> 90%) hoped that the outcome of caries prophylactics would be a reduced number of cavities.The patient-perceived experiences of caries prophylaxis-were in concordance with dental records. Both the dentists and the caries active middle-aged Swedish adults were aware of the need for extra prophylaxis.The caries active patients perceived having made extra home care efforts, but had not experienced that they had become free from caries. PMID:27464386

  19. Power and sample size determination for group comparison of patient-reported outcomes using polytomous Rasch models.

    PubMed

    Hardouin, Jean-Benoit; Blanchin, Myriam; Feddag, Mohand-Larbi; Le Néel, Tanguy; Perrot, Bastien; Sébille, Véronique

    2015-07-20

    The analysis of patient-reported outcomes or other psychological traits can be realized using the Rasch measurement model. When the objective of a study is to compare groups of individuals, it is important, before the study, to define a sample size such that the group comparison test will attain a given power. The Raschpower procedure (RP) allows doing so with dichotomous items. The RP is extended to polytomous items. Several computational issues were identified, and adaptations have been proposed. The performance of this new version of RP is assessed using simulations. This adaptation of RP allows obtaining a good estimate of the expected power of a test to compare groups of patients in a large number of practical situations. A Stata module, as well as its implementation online, is proposed to perform the RP. Two versions of the RP for polytomous items are proposed (deterministic and stochastic versions). These two versions produce similar results in all of the tested cases. We recommend the use of the deterministic version, when the measure is obtained using small questionnaires or items with a few number of response categories, and the stochastic version elsewhere, so as to optimize computing time. PMID:25787270

  20. Patient-reported Outcomes in Asian Patients With Chronic Hepatitis C Treated With Ledipasvir and Sofosbuvir

    PubMed Central

    Younossi, Zobair M.; Stepanova, Maria; Chan, Henry L.Y.; Lee, Mei H.; Yu, Ming-Lung; Dan, Yock Y.; Choi, Moon S.; Henry, Linda

    2016-01-01

    Abstract Prevalence of chronic hepatitis C (CH-C) infection in patients of Asian ancestry ranges between 1% and 20%. Interferon (IFN)- and ribavirin (RBV)-containing regimens for CH-C have a negative impact on patient-reported outcomes (PROs) during treatment. The aim of this study was to assess the impact of IFN-free RBV-free sofosbuvir (SOF)-based regimens on PROs in CH-C patients of Asian ancestry. In this observational retrospective study, the PRO data from 12 multicenter multinational phase 3 clinical trials (2012–2015, conducted in Europe, North America, Australia, and New Zealand) of SOF-based regimens with and without IFN, ledipasvir (LDV), and/or RBV were used. At baseline, during treatment, and post-treatment, patients completed 4 validated PRO questionnaires (SF-36, CLDQ-HCV, FACIT-F, and WPAI:SHP). The resulting PROs in Asian patients were compared across the treatment regimens. Of 4485 of the trials’ participants, 106 patients were of Asian ancestry (55.7% male, 69.8% treatment-naïve, 17.0% cirrhotic). In comparison with other patients, the Asian CH-C cohort was younger, had lower BMI, and lower rates of pre-treatment psychiatric comorbidities (anxiety, depression, sleep disorders) (all P < .05). At baseline, Asian patients also had lower SF-36 physical functioning scores (on average, by −5.6% on a normalized 0–100% PRO scale, P = .001). During treatment, Asian CH-C patients experienced a decline in their PRO scores while receiving IFN and/or RBV-containing regimens (up to −19.6%, P < .001). In contrast, patients receiving LDV/SOF experienced no PRO decrement and improvement of some PRO scores during treatment (+9.0% in general health of SF-36, P = .03). After achieving SVR-12, some of the PRO scores in Asian patients improved regardless of the regimen (up to +9.3%, P < .001). In multivariate analysis of Asian patients, the use of LDV/SOF was independently associated with higher PRO scores during and soon after the end of

  1. Management of Patient-Reported Outcome (PRO) Alerts in Clinical Trials: A Cross Sectional Survey.

    PubMed Central

    Kyte, Derek; Ives, Jonathan; Draper, Heather; Calvert, Melanie

    2016-01-01

    Background Assessment of patient-reported outcomes (PROs) provides valuable information to inform patient-centered care, but may also reveal ‘PRO alerts’: psychological distress or physical symptoms that may require an immediate response. Ad-hoc management of PRO alerts in clinical trials may result in suboptimal patient care or potentially bias trial results. To gain greater understanding of current practice in PRO alert management we conducted a national survey of personnel involved in clinical trials with a PRO endpoint. Methods and Findings We conducted a national cross-sectional survey of 767 UK-based research nurses, data managers/coordinators, trial managers and chief/principal investigators involved in clinical trials using PROs. Respondents were self-selected volunteers from a non-randomised sample of eligible individuals recruited via 55 UK Clinical Research Collaboration Registered Clinical Trials Units and 19 Comprehensive Local Research Networks. Questions centred on the proportion of trial personnel encountering alerts, how staff responded to PRO alerts and whether current guidance was deemed sufficient to support research personnel. We undertook descriptive analyses of the quantitative data and directed thematic analysis of free-text comments. 20% of research nurses did not view completed PRO questionnaires and were not in a position to discover alerts, 39–50% of the remaining respondent group participants reported encountering PRO alerts. Of these, 83% of research nurses and 54% of data managers/trial coordinators reported taking action to assist the trial participant, but less than half were able to record the intervention in the trial documentation. Research personnel reported current PRO alert guidance/training was insufficient. Conclusions Research personnel are intermittently exposed to PRO alerts. Some intervene to help trial participants, but are not able to record this intervention in the trial documentation, risking co

  2. Patient-reported Outcomes in Asian Patients With Chronic Hepatitis C Treated With Ledipasvir and Sofosbuvir.

    PubMed

    Younossi, Zobair M; Stepanova, Maria; Chan, Henry L Y; Lee, Mei H; Yu, Ming-Lung; Dan, Yock Y; Choi, Moon S; Henry, Linda

    2016-03-01

    Prevalence of chronic hepatitis C (CH-C) infection in patients of Asian ancestry ranges between 1% and 20%. Interferon (IFN)- and ribavirin (RBV)-containing regimens for CH-C have a negative impact on patient-reported outcomes (PROs) during treatment.The aim of this study was to assess the impact of IFN-free RBV-free sofosbuvir (SOF)-based regimens on PROs in CH-C patients of Asian ancestry.In this observational retrospective study, the PRO data from 12 multicenter multinational phase 3 clinical trials (2012-2015, conducted in Europe, North America, Australia, and New Zealand) of SOF-based regimens with and without IFN, ledipasvir (LDV), and/or RBV were used. At baseline, during treatment, and post-treatment, patients completed 4 validated PRO questionnaires (SF-36, CLDQ-HCV, FACIT-F, and WPAI:SHP). The resulting PROs in Asian patients were compared across the treatment regimens.Of 4485 of the trials' participants, 106 patients were of Asian ancestry (55.7% male, 69.8% treatment-naïve, 17.0% cirrhotic). In comparison with other patients, the Asian CH-C cohort was younger, had lower BMI, and lower rates of pre-treatment psychiatric comorbidities (anxiety, depression, sleep disorders) (all P < .05). At baseline, Asian patients also had lower SF-36 physical functioning scores (on average, by -5.6% on a normalized 0-100% PRO scale, P = .001). During treatment, Asian CH-C patients experienced a decline in their PRO scores while receiving IFN and/or RBV-containing regimens (up to -19.6%, P < .001). In contrast, patients receiving LDV/SOF experienced no PRO decrement and improvement of some PRO scores during treatment (+9.0% in general health of SF-36, P = .03). After achieving SVR-12, some of the PRO scores in Asian patients improved regardless of the regimen (up to +9.3%, P < .001). In multivariate analysis of Asian patients, the use of LDV/SOF was independently associated with higher PRO scores during and soon after the end of treatment (betas +15

  3. The Impact of Obesity on Patient Reported Outcomes Following Stereotactic Body Radiation Therapy for Prostate Cancer

    PubMed Central

    Cyr, Robyn; Feng, Li Rebekah; Bae, Edward; Danner, Malika T; Ayoob, Marilyn; Yung, Thomas M; Lei, Siyuan; Collins, Brian T; Saligan, Leorey; Simeng, Suy; Kumar, Deepak; Collins, Sean P

    2016-01-01

    Objectives The relationship between obesity (Body Mass Index ­>30 kg/m2) and quality of life (QoL) following prostate cancer (PCa) radiation therapy (RT) is unknown. Excess abdominal fat may compromise the precise delivery of radiation, putting surrounding organs at risk for greater radiation exposure. Stereotactic body radiation therapy (SBRT) utilizes a real-time tracking system that provides updated prostate position information and allows for correction of the therapeutic beam during treatment with high accuracy. In this study, we evaluate the impact of obesity on patient reported outcomes following SBRT for prostate cancer. Materials and methods Between February 2008 and April 2012, 88 obese and 178 non-obese patients with PCa were treated with SBRT at Georgetown University Hospital, Washington, DC. Health-related quality of life (HRQol) was assessed via the expanded prostate cancer index composite (EPIC)-26 at baseline, 6, 12, 18, and 24 months after 5-fraction delivery of 35-36.25 Gy with the CyberKnife. Patients who received androgen deprivation therapy (ADT) were excluded from this analysis due to its known negative impact on HRQoL. Results Pretreatment characteristics of obese and non-obese patient groups were similar except that obese patients had lower total testosterone levels. Urinary and bowel function and bother scores between the two patient cohorts were comparable at baseline and subsequent follow-ups. Sexual function and bother were also similar at baseline between both groups. Bother was defined by displeasure patients may experience from functional decline. At 24 months post-SBRT, obese men experienced borderline clinically significant decrease in sexual function and greater sexual bother compared to non-obese patients. Fatigue was significantly higher in obese patients compared to non-obese patients at 18 months post-SBRT. Conclusions Prostate SBRT affects obese and non-obese patients similarly in total HRQoL scores and majority of its

  4. Patient experiences with oily skin: The qualitative development of content for two new patient reported outcome questionnaires

    PubMed Central

    Arbuckle, Robert; Atkinson, Mark J; Clark, Marci; Abetz, Linda; Lohs, Jan; Kuhagen, Ilka; Harness, Jane; Draelos, Zoe; Thiboutot, Diane; Blume-Peytavi, Ulrike; Copley-Merriman, Kati

    2008-01-01

    Objective To develop the content for two new patient reported outcome (PRO) measures to: a) assess the severity of symptoms; and b) the impact of facial skin oiliness on emotional wellbeing using qualitative data from face to face, and internet focus groups in Germany and the US. Methods Using input from initial treatment satisfaction focus groups (n = 42), a review of relevant literature and expert clinicians (n = 3), a discussion guide was developed to guide qualitative inquiry using Internet focus groups (IFGs). IFGs were conducted with German (n = 26) and US (n = 28) sufferers of oily skin. Questionnaire items were generated using coded transcript data from the focus groups. Cognitive debriefing was conducted online with 42 participants and face to face with an additional five participants to assess the comprehension of the items. Results There were equal numbers of male and female participants; mean age was 35.4 (SD 9.3) years. On average, participants had had oily skin for 15.2 years, and 74% (n = 40) reported having mild-moderate acne. Participants reported using visual, tactile and sensory (feel without touching their face) methods to evaluate the severity of facial oiliness. Oily facial skin had both an emotional and social impact, and was associated with feelings of unattractiveness, self-consciousness, embarrassment, irritation and frustration. Items were generated for a measure of oily skin severity (Oily Skin Self-Assessment Scale) and a measure of the impact of oily skin on emotional well-being (Oily Skin Impact Scale). Cognitive debriefing resulted in minor changes to the draft items and confirmed their face and content validity. Conclusion The research provides insight into the experience of having oily skin and illustrates significant difficulties associated with the condition. Item content was developed for early versions of two PRO measures of the symptoms and emotional impact of oily facial skin. The psychometric validation of these measures

  5. Patient-reported outcomes (PROs) in dental clinical trials and product development: introduction to scientific and regulatory considerations.

    PubMed

    Gwaltney, Chad J

    2010-06-01

    Although typically measured with objective assessments, treatment efficacy and disease progression may also be examined using reports directly from the patient (patient-reported outcomes or PROs). PROs can enhance clinician and researcher understanding of an illness and its treatment in multiple ways, ranging from an assessment of core signs and symptoms to assessment of impairments and impacts across a variety of domains (eg, physical, social, occupational, emotional). Regulated drug and medical device development programs are increasingly using PROs as endpoints to support label claims. PRO instruments are commonly implemented in dental clinical trials. Concepts such as dental pain, tooth sensitivity, eating discomfort, and speech impairments may all be assessed via PROs. Although PROs are commonly implemented in trials, it is not clear that the development, use, and interpretation of PROs are consistent with scientific and regulatory best practices. The goals of this article were to introduce the concept of PROs and to provide an introduction to PRO scientific and regulatory principles. These principles can be used as a blueprint for using PROs in clinical research and for evaluating trials that have implemented PROs. When used appropriately, PROs may be useful in understanding the patient's perspective on illness and interventions and this perspective may be critical in fully evaluating the efficacy of dental treatments. PMID:20466315

  6. Systematic Review of Radiation Therapy Toxicity Reporting in Randomized Controlled Trials of Rectal Cancer: A Comparison of Patient-Reported Outcomes and Clinician Toxicity Reporting

    SciTech Connect

    Gilbert, Alexandra; Ziegler, Lucy; Martland, Maisie; Davidson, Susan; Efficace, Fabio; Sebag-Montefiore, David; Velikova, Galina

    2015-07-01

    The use of multimodal treatments for rectal cancer has improved cancer-related outcomes but makes monitoring toxicity challenging. Optimizing future radiation therapy regimens requires collection and publication of detailed toxicity data. This review evaluated the quality of toxicity information provided in randomized controlled trials (RCTs) of radiation therapy in rectal cancer and focused on the difference between clinician-reported and patient-reported toxicity. Medline, EMBASE, and the Cochrane Library were searched (January 1995-July 2013) for RCTs reporting late toxicity in patients treated with regimens including preoperative (chemo)radiation therapy. Data on toxicity measures and information on toxicity reported were extracted using Quantitative Analyses of Normal Tissue Effects in the Clinic recommendations. International Society for Quality of Life Research standards on patient-reported outcomes (PROs) were used to evaluate the quality of patient-reported toxicity. Twenty-one RCT publications met inclusion criteria out of 4144 articles screened. All PRO studies reported higher rates of toxicity symptoms than clinician-reported studies and reported on a wider range and milder symptoms. No clinician-reported study published data on sexual dysfunction. Of the clinician-reported studies, 55% grouped toxicity data related to an organ system together (eg “Bowel”), and 45% presented data only on more-severe (grade ≥3) toxicity. In comparison, all toxicity grades were reported in 79% of PRO publications, and all studies (100%) presented individual symptom toxicity data (eg bowel urgency). However, PRO reporting quality was variable. Only 43% of PRO studies presented baseline data, 28% did not use any psychometrically validated instruments, and only 29% of studies described statistical methods for managing missing data. Analysis of these trials highlights the lack of reporting standards for adverse events and reveals the differences between clinician and

  7. Patient Reported Outcomes (PROs) in Clinical Trials: Is ‘In-Trial’ Guidance Lacking? A Systematic Review

    PubMed Central

    Kyte, Derek G.; Draper, Heather; Ives, Jonathan; Liles, Clive; Gheorghe, Adrian; Calvert, Melanie

    2013-01-01

    Background Patient reported outcomes (PROs) are increasingly assessed in clinical trials, and guidelines are available to inform the design and reporting of such trials. However, researchers involved in PRO data collection report that specific guidance on ‘in-trial’ activity (recruitment, data collection and data inputting) and the management of ‘concerning’ PRO data (i.e., data which raises concern for the well-being of the trial participant) appears to be lacking. The purpose of this review was to determine the extent and nature of published guidelines addressing these areas. Methods and Findings Systematic review of 1,362 articles identified 18 eligible papers containing ‘in-trial’ guidelines. Two independent authors undertook a qualitative content analysis of the selected papers. Guidelines presented in each of the articles were coded according to an a priori defined coding frame, which demonstrated reliability (pooled Kappa 0.86–0.97), and validity (<2% residual category coding). The majority of guidelines present were concerned with ‘pre-trial’ activities (72%), for example, outcome measure selection and study design issues, or ‘post-trial’ activities (16%) such as data analysis, reporting and interpretation. ‘In-trial’ guidelines represented 9.2% of all guidance across the papers reviewed, with content primarily focused on compliance, quality control, proxy assessment and reporting of data collection. There were no guidelines surrounding the management of concerning PRO data. Conclusions The findings highlight there are minimal in-trial guidelines in publication regarding PRO data collection and management in clinical trials. No guidance appears to exist for researchers involved with the handling of concerning PRO data. Guidelines are needed, which support researchers to manage all PRO data appropriately and which facilitate unbiased data collection. PMID:23560103

  8. Informative value of Patient Reported Outcomes (PRO) in Health Technology Assessment (HTA)

    PubMed Central

    Brettschneider, Christian; Lühmann, Dagmar; Raspe, Heiner

    2011-01-01

    Background “Patient-Reported Outcome” (PRO) is used as an umbrella term for different concepts for measuring subjectively perceived health status e. g. as treatment effects. Their common characteristic is, that the appraisal of the health status is reported by the patient himself. In order to describe the informative value of PRO in Health Technology Assessment (HTA) first an overview of concepts, classifications and methods of measurement is given. The overview is complemented by an empirical analysis of clinical trials and HTA-reports on rheumatoid arthritis and breast cancer in order to report on type, frequency and consequences of PRO used in these documents. Methods For both issues systematic reviews of the literature have been performed. The search for methodological literature covers the publication period from 1990 to 2009, the search for clinical trials of rheumatoid arthritis and breast cancer covers the period 2005 to 2009. Both searches were performed in the medical databases of the German Institute of Medical Documentation and Information (DIMDI). The search for HTA-reports and methodological papers of HTA-agencies was performed in the CRD-Databases (CRD = Centre for Reviews and Dissemination) and by handsearching the websites of INAHTA member agencies (INAHTA = International Network of Agencies for Health Technology Assessment). For all issues specific inclusion and exclusion criteria were defined. The methodological quality of randomized controlled trials (RCT) was assessed by a modified version of the Cochrane Risk of Bias Tool. For the methodological part information extraction from the literature is structured by the report’s chapters, for the empirical part data extraction sheets were constructed. All information is summarized in a qualitative manner. Results Concerning the methodological issues the literature search retrieved 158 documents (87 documents related to definition or classification, 125 documents related to operationalisation of

  9. Evaluating more naturalistic outcome measures

    PubMed Central

    Bove, Riley; White, Charles C.; Giovannoni, Gavin; Glanz, Bonnie; Golubchikov, Victor; Hujol, Johnny; Jennings, Charles; Langdon, Dawn; Lee, Michelle; Legedza, Anna; Paskavitz, James; Prasad, Sashank; Richert, John; Robbins, Allison; Roberts, Susan; Weiner, Howard; Ramachandran, Ravi; Botfield, Martyn

    2015-01-01

    Objective: In this cohort of individuals with and without multiple sclerosis (MS), we illustrate some of the novel approaches that smartphones provide to monitor patients with chronic neurologic disorders in their natural setting. Methods: Thirty-eight participant pairs (MS and cohabitant) aged 18–55 years participated in the study. Each participant received an Android HTC Sensation 4G smartphone containing a custom application suite of 19 tests capturing participant performance and patient-reported outcomes (PROs). Over 1 year, participants were prompted daily to complete one assigned test. Results: A total of 22 patients with MS and 17 cohabitants completed the entire study. Among patients with MS, low scores on PROs relating to mental and visual function were associated with dropout (p < 0.05). We illustrate several novel features of a smartphone platform. First, fluctuations in MS outcomes (e.g., fatigue) were assessed against an individual's ambient environment by linking responses to meteorological data. Second, both response accuracy and speed for the Ishihara color vision test were captured, highlighting the benefits of both active and passive data collection. Third, a new trait, a person-specific learning curve in neuropsychological testing, was identified using spline analysis. Finally, averaging repeated measures over the study yielded the most robust correlation matrix of the different outcome measures. Conclusions: We report the feasibility of, and barriers to, deploying a smartphone platform to gather useful passive and active performance data at high frequency in an unstructured manner in the field. A smartphone platform may therefore enable large-scale naturalistic studies of patients with MS or other neurologic diseases. PMID:26516627

  10. Relationship between clinical and patient-reported outcomes in a phase 3 trial of tofacitinib or MTX in MTX-naïve patients with rheumatoid arthritis

    PubMed Central

    Fleischmann, Roy; Strand, Vibeke; Wilkinson, Bethanie; Kwok, Kenneth; Bananis, Eustratios

    2016-01-01

    Objective To compare the relationship between clinical measures and patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) treated with tofacitinib or methotrexate (MTX). Methods In a phase 3 randomised controlled trial, patients (N=956) who were MTX-naïve or had received ≤3 doses were randomised and received tofacitinib 5 or 10 mg twice daily or MTX titrated to 20 mg/week. Outcomes included: per cent of patients achieving American College of Rheumatology 70% responses (ACR70), ACR50, low disease activity (LDA) by Simplified Disease Activity Index (SDAI ≤11) and Clinical Disease Activity Index (CDAI ≤10), remission by SDAI (≤3.3) and CDAI (≤2.8), patient-reported Health Assessment Questionnaire-Disability Index (HAQ-DI scores <0.5), pain and global assessment of disease activity. Results At month 6, most patients who achieved LDA/remission by one definition achieved LDA/remission with others; however, discordance between measures was greater with MTX than with tofacitinib. As expected, concordance between CDAI and SDAI responses was high. Overall, patients achieving LDA or ACR50 responses reported less improvement in PROs (HAQ-DI, pain and patient global assessment) compared with clinical measures (tender and swollen joint counts). Conclusions Variability in levels of responses between clinical outcomes and PROs should be considered when setting treat-to-target goals in patients with RA. Trial registration number NCT01039688; Post-results. PMID:27175296

  11. [Patient evaluation and outcome measures].

    PubMed

    Nieto Pol, Enrique

    2014-01-01

    Both the initial evaluation and follow-up of patients with osteoarthritis require systematic evaluation of the indicators that provide information on the degree of involvement of the disease and allow its quantification. Reliable measures of disease progression help decision-making by clinicians and provide valid information on treatment response and the effectiveness of the distinct therapeutic interventions. The instruments recommended in research, as outcome measures in osteoarthritis, are pain evaluation, assessment of physical function, and self-reported global evaluation. In studies lasting more than 1 year, structural changes are evaluated through simple X-ray. Self-reported quality of life assessment and physician global assessment are also recommended as options. These indicators should be incorporated into routine clinical practice for adequate evaluation and correct follow-up of patients with osteoarthritis. The recommended pain evaluation method for use in clinical practice is the visual analog scale (VAS). The best instrument to evaluate physical function in patients with hip or knee osteoarthritis is the WOMAC scale (Western Ontario and McMaster Universities Osteoarthritis Index). For patient-reported global assessment in routine practice, the recommended scales are VAS or the SF-12 (12-item short-form health survey). PMID:24467959

  12. A systematic review of patient-reported outcome instruments of dermatologic adverse events associated with targeted cancer therapies

    PubMed Central

    Chan, Alexandre; Cameron, Michael C.; Garden, Benjamin; Boers-Doets, Christine B.; Schindler, Katja; Epstein, Joel B.; Choi, Jennifer; Beamer, Laura; Roeland, Eric; Russi, Elvio G.; Bensadoun, René-Jean; Teo, Yi Ling; Chan, Raymond J.; Shih, Vivianne; Bryce, Jane; Raber-Durlacher, Judith; Gerber, Peter Arne; Freytes, César O.; Rapoport, Bernardo; LeBoeuf, Nicole; Sibaud, Vincent; Lacouture, Mario E.

    2016-01-01

    Purpose Dermatologic adverse events (dAE) in cancer treatment are frequent with use of targeted therapies. These dAEs have been shown to have significant impact on health-related quality of life (HRQoL). While standardized assessment tools have been developed for physicians to assess severity of dAEs, there is a discord between objective and subjective measures. The identification of patient-reported outcome (PRO) instruments useful in the context of targeted cancer therapies is therefore important in both the clinical and research settings for the overall evaluation of dAEs and their impact on HRQoL. Methods A comprehensive, systematic literature search of published articles was conducted by two independent reviewers in order to identify PRO instruments previously utilized in patient populations with dAEs from targeted cancer therapies. The identified PRO instruments were studied to determine which HRQoL issues relevant to dAEs were addressed, as well as the process of development and validation of these instruments. Results Thirteen articles identifying six PRO instruments met the inclusion criteria. Four instruments were general dermatology (Skindex-16©, Skindex-29©, Dermatology Life Quality Index [DLQI], and DIELH-24), and two were symptom-specific (Functional Assessment of Cancer Therapy-Epidermal Growth Factor Receptor Inhibitors-18 [FACT-EGFRI-18] and Hand-Foot Syndrome 14 [HFS-14]). Conclusions While there are several PRO instruments that have been tested in the context of targeted cancer therapy, additional work is needed to develop new instruments and to further validate the instruments identified in this study in patients receiving targeted therapies. PMID:25564221

  13. Asthma outcome measures.

    PubMed

    Watkins, K D

    1999-08-01

    Annually, asthma accounts for 10.4 million physician visits, 468,000 hospitalizations, 1.8 million emergency room visits, and 10 million missed school days. According to the Centers for Disease Control and Prevention, the number of asthma deaths has increased progressively since 1978 form 1,800 to 5,400 per year. It is an expensive disease, accounting for about 1% of all health expenditure in the United States in 1990. Asthma has a significant impact on the patient and the community at large and the assessment of the clinical, physiologic, humanistic and economic outcomes will be measured. PMID:10563275

  14. Tofacitinib or adalimumab versus placebo: patient-reported outcomes from a phase 3 study of active rheumatoid arthritis

    PubMed Central

    Strand, Vibeke; van Vollenhoven, Ronald F.; Lee, Eun Bong; Fleischmann, Roy; Zwillich, Samuel H.; Gruben, David; Koncz, Tamas; Wilkinson, Bethanie

    2016-01-01

    Objective. To evaluate effects of tofacitinib or adalimumab on patient-reported outcomes (PROs) in patients with moderate to severe RA and inadequate responses to MTX. Methods. In this 12-month, phase 3, randomized controlled trial (ORAL Standard), patients (n = 717) receiving background MTX were randomized to tofacitinib 5 or 10 mg twice daily (BID), adalimumab 40 mg once every 2 weeks or placebo. PROs included HAQ-Disability Index, Patient Global Assessment of Arthritis, Patient Assessment of Arthritis Pain, health-related quality of life (Short Form-36 [SF-36]), fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue) and sleep (Medical Outcomes Study-Sleep). Results. At month 3, tofacitinib 10 mg BID treatment resulted in significant changes from baseline vs placebo across all PROs, sustained to month 12, with the highest number of patients reporting improvements ⩾minimum clinically important differences vs placebo (P < 0.05). Changes from baseline at month 3 with tofacitinib 5 mg BID and adalimumab were similar and statistically significant vs placebo across most PROs, excluding SF-36 Mental Component Score and Social Functioning, Role Emotional, and Mental Health domains, with significantly more patients reporting improvements ⩾minimum clinically important differences. Numbers Needed to Treat were lowest for tofacitinib 10 mg BID and similar between tofacitinib 5 mg BID and adalimumab. Conclusion. Patients with moderate to severe RA and inadequate responses to MTX reported improvements across a broad range of PROs with tofacitinib 5 and 10 mg BID and adalimumab that were significantly superior to placebo. PMID:26929445

  15. The OnyCOE-t™ questionnaire: responsiveness and clinical meaningfulness of a patient-reported outcomes questionnaire for toenail onychomycosis

    PubMed Central

    Potter, Lori P; Mathias, Susan D; Raut, Monika; Kianifard, Farid; Tavakkol, Amir

    2006-01-01

    Background This research was conducted to confirm the validity and reliability and to assess the responsiveness and clinical meaningfulness of the OnyCOE-t™, a questionnaire specifically designed to measure patient-reported outcomes (PRO) associated with toenail onychomycosis. Methods 504 patients with toenail onychomycosis randomized to receive 12 weeks of terbinafine 250 mg/day with or without target toenail debridement in the IRON-CLAD® trial completed the OnyCOE-t™ at baseline, weeks 6, 12, 24, and 48. The OnyCOE-t™ is composed of 6 multi-item scales and 1 single-item scale. These include a 7-item Toenail Symptom assessment, which comprises both Symptom Frequency and Symptom Bothersomeness scales; an 8-item Appearance Problems scale; a 7-item Physical Activities Problems scale; a 1-item Overall Problem scale; a 7-item Stigma scale; and a 3-item Treatment Satisfaction scale. In total, 33 toenail onychomycosis-specific items are included in the OnyCOE-t™. Clinical data, in particular the percent clearing of mycotic involvement in the target toenail, and OnyCOE-t™ responses were used to evaluate the questionnaire's reliability, validity, responsiveness, and the minimally clinical important difference (MCID). Results The OnyCOE-t™ was shown to be reliable and valid. Construct validity and known groups validity were acceptable. Internal consistency reliability of multi-item scales was demonstrated by Cronbach's alpha > .84. Responsiveness was good, with the Treatment Satisfaction, Symptom Frequency, Overall Problem, and Appearance Problem scales demonstrating the most responsiveness (Guyatt's statistic of 1.72, 1.31, 1.13, and 1.11, respectively). MCID was evaluated for three different clinical measures, and indicated that approximately an 8.5-point change (on a 0 to 100 scale) was clinically meaningful based on a 25% improvement in target nail clearing. Conclusion The OnyCOE-t™ questionnaire is a unique, toenail-specific PRO questionnaire that can be

  16. Assessing Patient-Reported Outcomes Following Orthognathic Surgery and Osseous Genioplasty.

    PubMed

    Schwitzer, Jonathan A; Albino, Frank P; Mathis, Ryan K; Scott, Amie M; Gamble, Laurie; Baker, Stephen B

    2015-11-01

    Primary outcomes for orthognathic surgery and genioplasty patients include satisfaction with appearance, improved motor function, and enhanced quality of life. The goal of this study was to assess outcomes among patients undergoing these procedures, and to highlight the potential use of FACE-Q instrument for use in patients with dentofacial deformities. A total of 56 patients presenting for orthognathic surgery and/or osseous genioplasty completed the FACE-Q during preoperative and/or at postoperative visits. FACE-Q scores increased following surgery in satisfaction with facial appearance overall (+24.5, P < 0.01), satisfaction with lower face and jawline (+40.7, P < 0.01), and in all satisfaction with chin items (profile, prominence, shape, and overall). Patients also demonstrated increased social confidence (+8.9, P = 0.29). There was no improvement in psychologic well-being (-0.8, P = 0.92). All 3 surgical groups of patients experienced gains in satisfaction with appearance following surgery. Patients who underwent orthognathic surgery either alone or in combination with genioplasty demonstrated statistically significant improvements in satisfaction with facial appearance overall (P < 0.01 for both groups), whereas patients who underwent genioplasty alone did not (P = 0.13). In addition, patients who underwent orthognathic surgery combined with genioplasty demonstrated greater improvement in satisfaction with chin than patients who underwent genioplasty alone. In conclusion, patients who underwent orthognathic surgery and/or genioplasty demonstrated improvement in appearance and social confidence. The use of this model supports the successful outcomes possible for patients undergoing these procedures. PMID:26501967

  17. Stakeholder perspectives on implementing the National Cancer Institute's patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).

    PubMed

    Bruner, Deborah Watkins; Hanisch, Laura J; Reeve, Bryce B; Trotti, Andy M; Schrag, Deborah; Sit, Laura; Mendoza, Tito R; Minasian, Lori; O'Mara, Ann; Denicoff, Andrea M; Rowland, Julia H; Montello, Michael; Geoghegan, Cindy; Abernethy, Amy P; Clauser, Steven B; Castro, Kathleen; Mitchell, Sandra A; Burke, Laurie; Trentacosti, Ann Marie; Basch, Ethan M

    2011-03-01

    The National Cancer Institute (NCI) is developing a patient-reported version of its Common Terminology Criteria for Adverse Events, called the "PRO-CTCAE." The PRO-CTCAE consists of a library of patient-reported items which can be administered in clinical trials to directly capture the patient experience of adverse events during cancer treatment, as well as a software platform for administering these items via computer or telephone. In order to better understand the impressions of stakeholders involved in cancer clinical research about the potential value of the PRO-CTCAE approach to capturing adverse event information in clinical research, as well as their perspectives about barriers and strategies for implementing the PRO-CTCAE in NCI-sponsored cancer trials, a survey was conducted. A survey including structured and open-ended questions was developed to elicit perceptions about the use of patient-reported outcomes (PROs) for adverse event reporting, and to explore logistical considerations for implementing the PRO-CTCAE in cancer trials. The survey was distributed electronically and by paper to a convenience sample of leadership and committee members in the NCI's cooperative group network, including principal investigators, clinical investigators, research nurses, data managers, patient advocates, and representatives of the NCI and Food and Drug Administration. Between October, 2008 through February, 2009, 727 surveys were collected. Most respondents (93%) agreed that patient reporting of adverse symptoms would be useful for improving understanding of the patient experience with treatment in cancer trials, and 88%, 80%, and 76%, respectively, endorsed that administration of PRO-CTCAE items in clinical trials would improve the completeness, accuracy, and efficiency of symptom data collection. More than three fourths believed that patient reports would be useful for informing treatment dose modifications and towards FDA regulatory evaluation of drugs. Eighty

  18. Poor patient-reported outcome after hip replacement, related to poor perception of perioperative information, commoner in immigrants than in non-immigrants.

    PubMed

    Krupic, Ferid; Rolfson, Ola; Nemes, Szilard; Kärrholm, Johan

    2016-06-01

    Background and purpose - In preparing patients for total hip replacement surgery, providing thorough information helps to reduce anxiety, manage postoperative pain, prevent complications, and better engage patients in their rehabilitation. However, patient characteristics may have an influence on the ability to comprehend and assimilate the information given. We investigated differences in patients born in Sweden and those born outside Sweden regarding how they perceived the information given before THR, and if this was associated with different patient-reported outcomes one year after surgery. Patients and methods - From Sahlgrenska University Hospital, we recruited 150 patients born in Sweden and 50 patients born outside Sweden who were to undergo THR. We retrieved routinely collected data from the Swedish Hip Arthroplasty Register including basic demographic variables and patient-reported outcome measures, both preoperatively and at 1-year follow-up. In a separate survey carried out 1-2 weeks after surgery, patients were asked about the information provided in connection with the operation. Results - Patients born outside Sweden more frequently reported that they were poorly informed about possibilities to treat pain and about the operation itself. 1 year after the operation, patients born outside Sweden who, 1-2 weeks after the operation, had reported that they were poorly informed also reported having worse outcomes. Poorer results were found for the questions self-care and anxiety/depression in the EQ-5D questionnaire, pain on a visual analog scale (VAS), EQVAS, and EQ-5D index compared to those patients born in Sweden who had received at least some information of acceptable quality. Interpretation - One quarter of the patients were not satisfied with the information provided before and after THR. These patients more commonly reported perioperative anxiety and they were more often born outside Sweden. Poorly informed patients who had come from countries

  19. Poor patient-reported outcome after hip replacement, related to poor perception of perioperative information, commoner in immigrants than in non-immigrants

    PubMed Central

    Krupic, Ferid; Rolfson, Ola; Nemes, Szilard; Kärrholm, Johan

    2016-01-01

    Background and purpose In preparing patients for total hip replacement surgery, providing thorough information helps to reduce anxiety, manage postoperative pain, prevent complications, and better engage patients in their rehabilitation. However, patient characteristics may have an influence on the ability to comprehend and assimilate the information given. We investigated differences in patients born in Sweden and those born outside Sweden regarding how they perceived the information given before THR, and if this was associated with different patient-reported outcomes one year after surgery. Patients and methods From Sahlgrenska University Hospital, we recruited 150 patients born in Sweden and 50 patients born outside Sweden who were to undergo THR. We retrieved routinely collected data from the Swedish Hip Arthroplasty Register including basic demographic variables and patient-reported outcome measures, both preoperatively and at 1-year follow-up. In a separate survey carried out 1–2 weeks after surgery, patients were asked about the information provided in connection with the operation. Results Patients born outside Sweden more frequently reported that they were poorly informed about possibilities to treat pain and about the operation itself. 1 year after the operation, patients born outside Sweden who, 1–2 weeks after the operation, had reported that they were poorly informed also reported having worse outcomes. Poorer results were found for the questions self-care and anxiety/depression in the EQ-5D questionnaire, pain on a visual analog scale (VAS), EQVAS, and EQ-5D index compared to those patients born in Sweden who had received at least some information of acceptable quality. Interpretation One quarter of the patients were not satisfied with the information provided before and after THR. These patients more commonly reported perioperative anxiety and they were more often born outside Sweden. Poorly informed patients who had come from countries outside

  20. Having a Family Doctor is Associated with Some Better Patient-Reported Outcomes of Primary Care Consultations

    PubMed Central

    Lam, Cindy L. K.; Yu, Esther Y. T.; Lo, Yvonne Y. C.; Wong, Carlos K. H.; Mercer, Stewart M.; Fong, Daniel Y. T.; Lee, Albert; Lam, Tai Pong; Leung, Gabriel M.

    2014-01-01

    Background: Hong Kong (HK) has pluralistic primary care that is provided by a variety of doctors. The aim of our study was to assess patient-reported outcomes of primary care consultations in HK and whether having a family doctor (FD) made any difference. Methods: We interviewed by telephone 3148 subjects from 5174 contacted households (response rate 60.8%) randomly selected from the general population of HK about the experience of their last primary care consultations in September 2007 and April 2008. We compared the patient-reported outcomes (PRO) and patient-centered process of care in those with a FD, those with other types of regular primary care doctors (ORD) and those without any regular primary care doctor (NRD). PRO included patient enablement, global improvement in health, overall satisfaction, and likelihood of recommending their doctors to family and friends. Patient-centered process of care indicators was explanations about the illness, and address of patient’s concerns. Results: One thousand one hundred fifty, 746, and 1157 reported to have FD, ORD, and NRD, respectively. Over 80% of those with FD consulted their usual primary care doctors in the last consultation compared with 27% of those with NRD. Compared with subjects having ORD or NRD, subjects with FD reported being more enabled after the consultation and were more likely to recommend their doctors to family and friends. Subjects with FD and ORD were more likely than those having NRD to report a global improvement in health and satisfaction. FD group was more likely than the other two groups to report receiving an explanation on the diagnosis, nature, and expected course of the illness, and having their concerns addressed. Patient enablement was associated with explanation of diagnosis, nature, and expected course of illness, and address of patient’s concerns. Conclusion: People with a regular FD were more likely to feel being enabled and to experience patient-centered care in consultations

  1. Feasibility and Value of PatientViewpoint: A Web System for Patient-Reported Outcomes Assessment in Clinical Practice

    PubMed Central

    Snyder, Claire F.; Blackford, Amanda L.; Wolff, Antonio C.; Carducci, Michael A.; Herman, Joseph M.; Wu, Albert W.

    2012-01-01

    OBJECTIVE The PatientViewpoint website collects patient-reported outcomes (PROs) and links them with the electronic health record to aid patient management. This pilot-test evaluated PatientViewpoint’s use, usefulness, and acceptability to patients and clinicians. METHODS This was a single-arm prospective study that enrolled breast and prostate cancer patients undergoing treatment and the clinicians who managed them. Patients completed PROs every two weeks, and clinicians could access the results for patient visits. Scores that were poor relative to norms or substantially worse than the previous assessment were highlighted. After three on-study visits, we assessed patient and clinician perspectives on PatientViewpoint using close-ended and open-ended questions. RESULTS 11/12 eligible clinicians (92%) and 52/76 eligible patients (68%) enrolled. Patients completed a median of 71% of assigned questionnaires; clinicians reported using the information for 79% of patients, most commonly as a source of additional information (51%). At the median, score reports identified 3 potential issues, of which 1 was discussed during the visit. Patients reported the system was easy to use (92%), useful (70%), aided recall of symptoms/side effects (72%), helped them feel more in control of their care (60%), improved discussions with their provider (49%), and improved care quality (39%). Patients and clinicians desired more information on score interpretation and minor adjustments to site navigation. CONCLUSIONS These results support the feasibility and value of PatientViewpoint. An ongoing study is using a continuous quality improvement approach to further refine PatientViewpoint. Future studies will evaluate its impact on patient care and outcomes. PMID:22544513

  2. Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008-2012).

    PubMed

    Bansal, Dipika; Bhagat, Anil; Schifano, Fabrizio; Gudala, Kapil

    2015-12-01

    The present study aimed at systematically reviewing the role and extent of patient-reported outcomes (PROs) usage within the package of scientific evidence considered for marketing authorization (MA). All regulatory information published by the European Medicines Agency (EMA) for products authorized between January 2008 and December 2012 and appearing in the European Public Assessment Report (EPAR) database was examined for efficacy endpoints. The endpoints here considered included: PROs, clinician reported outcomes (CROs), and laboratory reported outcomes (LROs). LROs were the most frequently reported endpoints. Out of the 180 products here selected, 99 (55%), 67 (37%), and 30 (17%), respectively, used LROs, CROs and PROs as primary endpoints (PEs). PROs as any endpoints were used in 82 (46%) products. Out of these, PROs were documented as PE in 30 (37%), with 27 (33%) products having used PROs both as primary and non-PEs. PRO usage was most frequently identified with nervous system and antineoplastic agents. During the study period, the use of all the three types of endpoints appeared to be static. Both the regulatory bodies and the industry should ensure complete and clear reporting of all endpoints used, including PROs, to improve transparency. PMID:26031612

  3. Statistical approaches to analyse patient-reported outcomes as response variables: an application to health-related quality of life.

    PubMed

    Arostegui, Inmaculada; Núñez-Antón, Vicente; Quintana, José M

    2012-04-01

    Patient-reported outcomes (PRO) are used as primary endpoints in medical research and their statistical analysis is an important methodological issue. Theoretical assumptions of the selected methodology and interpretation of its results are issues to take into account when selecting an appropriate statistical technique to analyse data. We present eight methods of analysis of a popular PRO tool under different assumptions that lead to different interpretations of the results. All methods were applied to responses obtained from two of the health dimensions of the SF-36 Health Survey. The proposed methods are: multiple linear regression (MLR), with least square and bootstrap estimations, tobit regression, ordinal logistic and probit regressions, beta-binomial regression (BBR), binomial-logit-normal regression (BLNR) and coarsening. Selection of an appropriate model depends not only on its distributional assumptions but also on the continuous or ordinal features of the response and the fact that they are constrained to a bounded interval. The BBR approach renders satisfactory results in a broad number of situations. MLR is not recommended, especially with skewed outcomes. Ordinal methods are only appropriate for outcomes with a few number of categories. Tobit regression is an acceptable option under normality assumptions and in the presence of moderate ceiling or floor effect. The BLNR and coarsening proposals are also acceptable, but only under certain distributional assumptions that are difficult to test a priori. Interpretation of the results is more convenient when using the BBR, BLNR and ordinal logistic regression approaches. PMID:20858689

  4. The natural history of untreated dorsal wrist ganglia and patient reported outcome 6 years after intervention.

    PubMed

    Dias, J J; Dhukaram, V; Kumar, P

    2007-10-01

    We have evaluated the long-term outcome of excision, aspiration and no treatment of dorsal wrist ganglia prospectively in 236 (83%) of 283 patients who responded to a postal questionnaire at a mean of 70 months. The resolution of symptoms was similar between the treatment groups (p>0.3). Pain and unsightliness improved in all three treatment groups. The prevalence of weakness and stiffness altered only slightly in all three treatment groups. More patients with a recurrent, or persistent ganglion complained of pain, stiffness and unsightliness (p<0.0001). Patient satisfaction was higher after surgical excision (p<0.0001), even if the ganglion recurred. Twenty-three of 55 (58%) untreated ganglia resolved spontaneously. The recurrence rate was 58% (45/78) and 39% (40/103) following aspiration and excision, respectively. Eight out of 103 patients had complications following surgery. In this study, neither excision nor aspiration provided significant long-term benefit over no treatment. PMID:17950209

  5. Underlying diagnosis predicts patient-reported outcomes after revision total knee arthroplasty

    PubMed Central

    Lewallen, David G.

    2014-01-01

    Objective. To assess the association of underlying diagnosis with outcomes after revision total knee arthroplasty (TKA). Methods. For this cohort study we used prospectively collected data from the Mayo Clinic Total Joint Registry on all revision TKA patients from 1993 to 2005 with 2- or 5-year response to a validated knee questionnaire that assesses pain and function. We used logistic regression to assess the odds of moderate–severe activities of daily living (ADL) limitations and moderate–severe index knee pain 2 and 5 years after revision TKA. Odds ratios (ORs) and 95% CIs are presented. Results. The underlying diagnosis for the 2- and 5-year cohorts was loosening, wear or osteolysis in 73% and 75%; dislocation, bone or prosthesis fracture, instability or non-union in 17% and 15%; and failed prior arthroplasty with components removed or infection in 11% and 11%, respectively. In multivariable adjusted analyses that included preoperative status, compared with patients with loosening/wear/osteolysis, patients with dislocation/fracture/instability/non-union had an OR of 2.1 for moderate–severe ADL limitation (95% CI 1.3, 3.1, P < 0.001) and those with failed prior arthroplasty/infection had an OR of 1.1 (95% CI 0.6, 1.8, P = 0.4). At 5 years, differences were no longer significant. In multivariable adjusted analyses, compared with patients with loosening/wear/osteolysis, patients with dislocation/fracture/instability/non-union had an OR of 2.0 for moderate–severe pain (95% CI 1.3, 3.1, P < 0.01) at 2 years and an OR of 2.1 (95% CI 1.3, 3.8, P = 0.01) at 5 years. Failed prior arthroplasty/infection was not significantly different than the reference category. Conclusion. Underlying diagnosis is independently associated with ADL limitations and pain after revision TKA. This information can help patients have realistic expectations of outcomes. PMID:24196389

  6. Similar patient-reported outcomes and performance after total knee arthroplasty with or without patellar resurfacing.

    PubMed

    Ali, Abdulemir; Lindstrand, Anders; Nilsdotter, Anna; Sundberg, Martin

    2016-06-01

    Background and purpose - Knee pain after total knee arthroplasty (TKA) is not uncommon. Patellar retention in TKA is one cause of postoperative knee pain, and may lead to secondary addition of a patellar component. Patellar resurfacing in TKA is controversial. Its use ranges from 2% to 90% worldwide. In this randomized study, we compared the outcome after patellar resurfacing and after no resurfacing. Patients and methods - We performed a prospective, randomized study of 74 patients with primary osteoarthritis who underwent a Triathlon CR TKA. The patients were randomized to either patellar resurfacing or no resurfacing. They filled out the VAS pain score and KOOS questionnaires preoperatively, and VAS pain, KOOS, and patient satisfaction 3, 12, and 72 months postoperatively. Physical performance tests were performed preoperatively and 3 months postoperatively. Results - We found similar scores for VAS pain, patient satisfaction, and KOOS 5 subscales at 3, 12, and 72 months postoperatively in the 2 groups. Physical performance tests 3 months postoperatively were also similar in the 2 groups. No secondary resurfacing was performed in the group with no resurfacing during the first 72 months Interpretation - Patellar resurfacing in primary Triathlon CR TKA is of no advantage regarding pain, physical performance, KOOS 5 subscales, or patient satisfaction compared to no resurfacing. None of the patients were reoperated with secondary addition of a patellar component within 6 years. According to these results, routine patellar resurfacing in primary Triathlon TKA appears to be unnecessary. PMID:27212102

  7. Similar patient-reported outcomes and performance after total knee arthroplasty with or without patellar resurfacing

    PubMed Central

    Ali, Abdulemir; Lindstrand, Anders; Nilsdotter, Anna; Sundberg, Martin

    2016-01-01

    Background and purpose Knee pain after total knee arthroplasty (TKA) is not uncommon. Patellar retention in TKA is one cause of postoperative knee pain, and may lead to secondary addition of a patellar component. Patellar resurfacing in TKA is controversial. Its use ranges from 2% to 90% worldwide. In this randomized study, we compared the outcome after patellar resurfacing and after no resurfacing. Patients and methods We performed a prospective, randomized study of 74 patients with primary osteoarthritis who underwent a Triathlon CR TKA. The patients were randomized to either patellar resurfacing or no resurfacing. They filled out the VAS pain score and KOOS questionnaires preoperatively, and VAS pain, KOOS, and patient satisfaction 3, 12, and 72 months postoperatively. Physical performance tests were performed preoperatively and 3 months postoperatively. Results We found similar scores for VAS pain, patient satisfaction, and KOOS 5 subscales at 3, 12, and 72 months postoperatively in the 2 groups. Physical performance tests 3 months postoperatively were also similar in the 2 groups. No secondary resurfacing was performed in the group with no resurfacing during the first 72 months Interpretation Patellar resurfacing in primary Triathlon CR TKA is of no advantage regarding pain, physical performance, KOOS 5 subscales, or patient satisfaction compared to no resurfacing. None of the patients were reoperated with secondary addition of a patellar component within 6 years. According to these results, routine patellar resurfacing in primary Triathlon TKA appears to be unnecessary. PMID:27212102

  8. Validating a Patient-Reported Comorbidity Measure with Respect to Quality of Life in End-Stage Renal Disease

    PubMed Central

    Robinski, Maxi; Strich, Franz; Mau, Wilfried; Girndt, Matthias

    2016-01-01

    Purpose Medical record-derived comorbidity measures such as the Charlson Comorbidity Index (CCI) do not predict functional limitations or quality of life (QoL) in the chronically ill. Although these shortcomings are known since the 1980s, they have been largely ignored by the international literature. Recently, QoL has received growing interest as an end-point of interventional trials in Nephrology. The aim of this study is to compare a patient-reported comorbidity measure and the CCI with respect to its validity regarding QoL. Methods The German Self-Administered Comorbidity Questionnaire (SCQ-G) was completed by 780 adult end-stage renal disease-patients recruited from 55 dialysis units throughout Germany. Acceptance was evaluated via response rates. Content validity was examined by comparing the typical comorbidity pattern in dialysis patients and the pattern retrieved from our data. Convergent validity was assessed via kappa statistics. Data was compared to the CCI. Linear associations with QoL were examined (criterion validity). Results The SCQ-G was very well accepted by dialysis patients of all ages (response rate: 99%). Content validity can be interpreted as high (corresponding comorbidity items: 73.7%). Convergent validity was rather weak (.27≤ρ≤.29) but increased when comparing only concordant items (.39≤ρ≤.43). With respect to criterion validity, the SCQ-G performed better than the CCI regarding the correlation with QoL (e.g., SF-12-physical: SCQ-G total score: ρ = -.49 vs. CCI: ρ = -.36). Conclusions The patient-reported measure proved to be more valid than the external assessment when aiming at insights on QoL. Due to the inclusion of subjective limitations, the SCQ-G is more substantial with respect to patient-centered outcomes and might be used as additional measure in clinical trials. PMID:27294867

  9. Qualitative approach to patient-reported outcomes in oncology: protocol of a French study

    PubMed Central

    Orri, Massimiliano; Sibeoni, Jordan; Labey, Mathilde; Bousquet, Guilhem; Verneuil, Laurence; Revah-Levy, Anne

    2015-01-01

    Introduction The past decade has been characterised by movement from a doctor-centred to a patient-centred approach to treatment outcomes, in which doctors try to see the illness through their patients’ eyes. Patients, family members and doctors are the three participants in cancer care, but their perspectives about what have been helpful during cancer treatment have never simultaneously and explicitly compared in the same qualitative study. The aim of this study project is to explore patients’ perspectives about the care they receive, as well as families’ and doctors’ perspectives about what have been helpful for the patient. These three points of view will be compared and contrasted in order to analyse the convergences and divergences in these perspectives. Methods and analysis This is a national multicentre qualitative study. Participants will be constituted by three different subsamples: (1) patients with cancer (skin, breast, urological and lung cancers), (2) their relatives, and (3) their referring physicians. Recruitment will follow the purposive sample technique, and the final sample size will be determined by data saturation. Data will be collected through open-ended semistructured interviews and independently analysed with NVivo V.10 software by three researchers according to the principles of Interpretative Phenomenological Analysis. Ethics and dissemination The research protocol received approval from the University Paris Descartes review board (IRB number: 20140600001072), and participants will provide written consent. To the best of our knowledge, this is the first study to focus on the simultaneous exploration of the separate points of view of patients, families and doctors about the care received during the cancer care journey. We expect that our findings will help to improve communication and relationships between doctors, patients and families. Comparison of these three points of view will provide information about the convergences and

  10. Different patient-reported outcomes in immigrants and patients born in Sweden

    PubMed Central

    Krupic, Ferid; Garellick, Göran; Gordon, Max; Kärrholm, Johan

    2014-01-01

    Background and purpose Some patients have persistent symptoms after total hip arthroplsty (THA). We investigated whether the proportions of inferior clinical results after total hip arthroplasty—according to the 5 dimensions in the EQ-5D form, and pain and satisfaction according to a visual analog scale (VAS)—are the same in immigrants to Sweden as observed in those born in Sweden. Methods Records of total hip arthroplasties performed between 1992 and 2007 were retrieved from the Swedish Hip Arthroplasty Register (SHAR) and cross-matched with data from the National Board of Health and Welfare and also Statistics, Sweden. 18,791 operations (1,451 in immigrants, 7.7%) were eligible for analysis. Logistic and linear regression models including age, sex, diagnosis, type of fixation, comorbidity, surgical approach, marital status, and education level were analyzed. Outcomes were the 5 dimensions in EQ-5D, EQ-VAS, VAS pain, and VAS satisfaction. Preoperative data and data from 1 year postoperatively were studied. Results Preoperatively (and after inclusion of covariates in the regression models), all immigrant groups had more negative interference concerning self-care. Immigrants from the Nordic countries outside Sweden and Europe tended to have more problems with their usual activities and patients from Europe and outside Europe more often reported problems with anxiety/depression. Patients born abroad showed an overall tendency to report more pain on the VAS than patients born in Sweden. After the operation, the immigrant groups reported more problems in all the EQ-5D dimensions. After adjustment for covariates including the preoperative baseline value, most of these differences remained except for pain/discomfort and—concerning immigrants from the Nordic countries—also anxiety/depression. After the operation, pain according to VAS had decreased substantially in all groups. The immigrant groups indicated more pain than those born in Sweden, both before and

  11. Integrating Patient-Reported Outcomes into Spine Surgical Care through Visual Dashboards: Lessons Learned from Human-Centered Design

    PubMed Central

    Hartzler, Andrea L.; Chaudhuri, Shomir; Fey, Brett C.; Flum, David R.; Lavallee, Danielle

    2015-01-01

    Introduction: The collection of patient-reported outcomes (PROs) draws attention to issues of importance to patients—physical function and quality of life. The integration of PRO data into clinical decisions and discussions with patients requires thoughtful design of user-friendly interfaces that consider user experience and present data in personalized ways to enhance patient care. Whereas most prior work on PROs focuses on capturing data from patients, little research details how to design effective user interfaces that facilitate use of this data in clinical practice. We share lessons learned from engaging health care professionals to inform design of visual dashboards, an emerging type of health information technology (HIT). Methods: We employed human-centered design (HCD) methods to create visual displays of PROs to support patient care and quality improvement. HCD aims to optimize the design of interactive systems through iterative input from representative users who are likely to use the system in the future. Through three major steps, we engaged health care professionals in targeted, iterative design activities to inform the development of a PRO Dashboard that visually displays patient-reported pain and disability outcomes following spine surgery. Findings: Design activities to engage health care administrators, providers, and staff guided our work from design concept to specifications for dashboard implementation. Stakeholder feedback from these health care professionals shaped user interface design features, including predefined overviews that illustrate at-a-glance trends and quarterly snapshots, granular data filters that enable users to dive into detailed PRO analytics, and user-defined views to share and reuse. Feedback also revealed important considerations for quality indicators and privacy-preserving sharing and use of PROs. Conclusion: Our work illustrates a range of engagement methods guided by human-centered principles and design

  12. Patient-reported outcomes of cancer survivors in England 1–5 years after diagnosis: a cross-sectional survey

    PubMed Central

    Glaser, Adam W; Fraser, Lorna K; Corner, Jessica; Feltbower, Richard; Morris, Eva J A; Hartwell, Greg; Richards, Mike

    2013-01-01

    Objectives To determine the feasibility of collecting population-based patient-reported outcome measures (PROMs) in assessing quality of life (QoL) to inform the development of a national PROMs programme for cancer and to begin to describe outcomes in a UK cohort of survivors. Design Cross-sectional postal survey of cancer survivors using a population-based sampling approach. Setting English National Health Service. Participants 4992 breast, colorectal, prostate and non-Hodgkin's lymphoma (NHL) survivors 1–5 years from diagnosis. Primary and secondary outcome measures Implementation issues, response rates, cancer-specific morbidities utilising items including the EQ5D, tumour-specific subscales of the Functional Assessment of Cancer Therapy and Social Difficulties Inventory. Results 3300 (66%) survivors returned completed questionnaires. The majority aged 85+ years did not respond and the response rates were lower for those from more deprived area. Response rates did not differ by gender, time since diagnosis or cancer type. The presence of one or more long-term conditions was associated with significantly lower QoL scores. Individuals from most deprived areas reported lower QoL scores and poorer outcomes on other measures, as did those self-reporting recurrent disease or uncertainty about disease status. QoL scores were comparable at all time points for all cancers except NHL. QoL scores were lower than those from the general population in Health Survey for England (2008) and General Practice Patient Survey (2012). 47% of patients reported fear of recurrence, while 20% reported moderate or severe difficulties with mobility or usual activities. Bowel and urinary problems were common among colorectal and prostate patients. Poor bowel and bladder control were significantly associated with lower QoL. Conclusions This method of assessing QoL of cancer survivors is feasible and acceptable to most survivors. Routine collection of national population-based PROMs will

  13. Cognitive interviewing of the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

    PubMed Central

    Hay, Jennifer L.; Atkinson, Thomas M.; Reeve, Bryce B.; Mitchell, Sandra A.; Mendoza, Tito R.; Willis, Gordon; Minasian, Lori M.; Clauser, Steven B.; Denicoff, Andrea; O’Mara, Ann; Chen, Alice; Bennett, Antonia V.; Paul, Diane B.; Gagne, Joshua; Rogak, Lauren; Sit, Laura; Viswanath, Vish; Schrag, Deborah; Basch, Ethan

    2013-01-01

    Purpose The National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is a library of question items that enables patient reporting of adverse events (AEs) in clinical trials. This study contributes content validity evidence of the PRO-CTCAE by incorporating cancer patient input of the relevance and comprehensiveness of the item library. Methods Cognitive interviews were conducted among patients undergoing chemotherapy or radiation therapy at multiple sites to evaluate comprehension, memory retrieval, judgment, and response mapping related to AE terms (e.g., nausea); attribute terms (regarding frequency, severity, or interference); response options; and recall period. Three interview rounds were conducted with ≥20 patients completing each item per round. Items were modified and retested if ≥3 patients exhibited cognitive difficulties or if experienced by ≤25% patients. Results 127 patients participated (35% ≤high school; 28% non-white; 59% female). Most AE terms (63/80) generated no cognitive difficulties. The remaining 17 were modified without further difficulties by Round 3. Terms were comprehended regardless of education level. Attribute terms and response options required no modifications. Patient adherence to recall period (7-days) was improved when the reference period was incorporated. Conclusions This study provides evidence confirming comprehension of the U.S. English language versions of items in the PRO-CTCAE library for measuring symptomatic AEs from the patient perspective within the context of cancer treatment. Several minor changes were made to the items to improve item clarity, comprehension, and ease of response judgment. This study helps establish the content validity of PRO-CTCAE items for patient reporting of AEs during cancer treatment. PMID:23868457

  14. Quality of life: patient-reported outcomes after total replacement of the temporomandibular joint.

    PubMed

    Kunjur, J; Niziol, R; Matthews, N S

    2016-09-01

    Since publication of the UK guidelines on total replacement of the temporomandibular joint (TMJ) in 2008 by the British Association of Oral and Maxillofacial Surgeons (BAOMS), pain scores, mouth opening, and diet have been used as markers of success. We have looked at quality of life (QoL) as another. We analysed the data from a single surgeon on patients who had had joints replaced and devised a questionnaire to find out about the subjective, functional, psychological, and social aspects of TMJ disease. A total of 18 patients who had the same operation were included (mean (range) age 50 (33 - 73) years, mean (range) follow up 30 (18 - 48) months). Jaw function and facial aesthetics had improved, and patients needed less analgesia. Overall, they reported a better QoL with improvements in mood and social interaction, and the activities of daily life were easier. The NHS uses QoL questionnaires to measure success in fields such as orthopaedic surgery, but currently we know of no nationally accepted questionnaire that measures success after total replacement of the TMJ. PMID:27209222

  15. Conceptual and methodological advances in child-reported outcomes measurement

    PubMed Central

    Bevans, Katherine B; Riley, Anne W; Moon, JeanHee; Forrest, Christopher B

    2011-01-01

    Increasingly, clinical, pharmaceutical and translational research studies use patient-reported outcomes as primary and secondary end points. Obtaining this type of information from children themselves is now possible, but effective assessment requires developmentally sensitive conceptual models of child health and an appreciation for the rapid change in children’s cognitive capacities. To overcome these barriers, outcomes researchers have capitalized on innovations in modern measurement theory, qualitative methods for instrument development and new computerized technologies to create reliable and valid methods for obtaining self-reported health data among 8–17-year-old children. This article provides a developmentally focused framework for selecting child-report health assessment instruments. Several generic health-related quality of life instruments and the assessment tools developed by the NIH-sponsored Patient-Reported Outcome Measurement Information System network are discussed to exemplify advances in the measurement of children’s self-reported health, illness, wellbeing and quality of life. PMID:20715916

  16. Patient-reported depression measures in cancer: a meta-review.

    PubMed

    Wakefield, Claire E; Butow, Phyllis N; Aaronson, Neil A; Hack, Thomas F; Hulbert-Williams, Nicholas J; Jacobsen, Paul B

    2015-07-01

    The patient-reported depression measures that perform best in oncology settings have not yet been identified. We did a meta-review to integrate the findings of reviews of more than 50 depression measures used in adults with, or recovering from, any type of cancer. We searched Medline, PsycINFO, Embase, and grey literature from 1999 to 2014 to identify 19 reviews representing 372 primary studies. 11 reviews were rated as being of high quality (defined as meeting at least 20 criteria in the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement). The Hospital Anxiety Depression Scale (HADS) was the most thoroughly evaluated measure, but was limited by cutpoint variability. The HADS had moderate screening utility indices and was least recommended in advanced cancer or palliative care. The Beck Depression Inventory was more generalisable across cancer types and disease stages, with good indices for screening and case finding. The Center for Epidemiologic Studies Depression Scale was the best-weighted measure in terms of responsiveness. This meta-review provides a comprehensive overview of the strengths and limitations of available depression measures. It can inform the choice of the best measure for specific settings and purposes. PMID:26303561

  17. Relationship between Arm Morbidity and Patient-Reported Outcomes Following Surgery in Women with Node-Negative Breast Cancer: NSABP Protocol B-32

    PubMed Central

    Kopec, Jacek A.; Colangelo, Linda H.; Land, Stephanie R.; Julian, Thomas B.; Brown, Ann M.; Anderson, Stewart J.; Krag, David N.; Ashikaga, Takamaru; Costantino, Joseph P.; Wolmark, Norman; Ganz, Patricia A.

    2012-01-01

    Background The impact of arm morbidity following breast cancer surgery on patient-observed changes in daily functioning and health-related quality of life (HRQoL) have not been well-studied. Objective To examine the association of objective measures such as range of motion (ROM) and lymphedema, with patient-reported outcomes (PROs) in the arm and breast, upper extremity function, activities, and HRQoL. Methods The National Surgical Adjuvant Breast and Bowel Project Protocol B-32 was a randomized trial comparing sentinel node resection (SNR) with axillary dissection (AD) in women with node-negative breast cancer. ROM and arm volume were measured objectively. PROs included symptoms; arm function; limitations in social, recreational, occupational, and other regular activities; and a global index of HRQoL. Statistical methods included cross-tabulations and multivariable linear regression models. Results In all, 744 women provided at least 1 postsurgery assessment. About one-third of the patients experienced arm mobility restrictions. A similar number of patients avoided the use of the arm 6 months after surgery. Limitations in work and other regular activities were reported by about a quarter of the patients. In this multivariable analysis, arm mobility and sensory neuropathy were predictors of patient-reported arm function and overall HRQoL. Predictors for activity limitations also included side of surgery (dominant vs nondominant). Edema was not significant after adjustment for sensory neuropathy and ROM. Limitations Arm mobility and edema were measured simultaneously only once during the follow-up (6 months). Conclusion Clinical measures of sensory neuropathy and restrictions in arm mobility following breast cancer surgery are associated with self-reported limitations in activity and reductions in overall HRQoL. PMID:22951047

  18. Can we use social media to support content validity of patient-reported outcome instruments in medical product development?

    PubMed

    Rothman, Margaret; Gnanaskathy, Ari; Wicks, Paul; Papadopoulos, Elektra J

    2015-01-01

    We report a panel designed to open a dialog between pharmaceutical sponsors, regulatory reviewers, and other stakeholders regarding the use of social media to collect data to support the content validity of patient-reported outcome instruments in the context of medical product labeling. Multiple stakeholder perspectives were brought together to better understand the issues encountered in pursuing social media as a form of data collection to support content validity. Presenters represented a pharmaceutical sponsor of clinical trials, a regulatory reviewer from the Food and Drug Administration, and an online data platform provider. Each presenter shared its perspective on the advantages and disadvantages of using social media to collect this type of information. There was consensus that there is great potential for using social media for this purpose. There remain, however, unanswered questions that need to be addressed such as identifying which type of social media is most appropriate for data collection and ensuring that participants are representative of the target population while maintaining the advantages of anonymity provided by online platforms. The use of social media to collect evidence of content validity holds much promise. Clarification of issues that need to be addressed and accumulation of empirical evidence to address these questions are essential to moving forward. PMID:25595228

  19. Nonpharmacological Treatments of Insomnia for Long-Term Painful Conditions: A Systematic Review and Meta-analysis of Patient-Reported Outcomes in Randomized Controlled Trials

    PubMed Central

    Tang, Nicole K.Y.; Lereya, S. Tanya; Boulton, Hayley; Miller, Michelle A.; Wolke, Dieter; Cappuccio, Francesco P.

    2015-01-01

    Study Objectives: Insomnia is a debilitating comorbidity of chronic pain. This study evaluated the effect of nonpharmacological sleep treatments on patient-reported sleep quality, pain, and well-being in people with long-term cancer and non-cancer (e.g., back pain, arthritis, fibromyalgia) pain conditions. Design: We systematically searched Cochrane CENTRAL, MEDLINE, Embase, and PsychINFO for relevant studies. Search period was set to inception of these databases to March 2014. Studies were included if they were: original randomized controlled trials (RCTs); testing a nonpharmacological intervention; that targets sleep; in adults; with painful health conditions; that has a control group; includes a measure of sleep quality; and at least one other health and well-being outcome. Measurement and Findings: Means and standard deviations of sleep quality, pain, fatigue, depression, anxiety, physical and psychological functioning were extracted for the sleep treatment and control groups at baseline, posttreatment and final follow-up. Methodological details concerning the treatment, participants, and study design were abstracted to guide heterogeneity and subgroup analyses. Eleven RCTs involving 1,066 participants (mean age 45–61 years) met the criteria for the meta-analysis. There was no systematic evidence of publication bias. Nonpharmacological sleep treatments in chronic pain patients were associated with a large improvement in sleep quality (standardized mean difference = 0.78, 95% Confidence Interval [0.42, 1.13]; P < 0.001), small reduction in pain (0.18 [0, 0.36] P < 0.05), and moderate improvement in fatigue (0.38 [0.08, 0.69]; P < 0.01) at posttreatment. The effects on sleep quality and fatigue were maintained at follow-up (up to 1 year) when a moderate reduction in depression (0.31, [0.09, 0.53]; P < 0.01) was also observed. Both cancer and non-cancer pain patients benefited from nonpharmacological sleep treatments. Face-to-face treatments achieved better

  20. Measuring outcomes in craniofacial and pediatric plastic surgery.

    PubMed

    Wong, Karen W Y; Forrest, Christopher R; Goodacre, Tim E E; Klassen, Anne F

    2013-04-01

    This article discusses the measurement of outcomes in craniofacial and pediatric plastic surgery, using examples of craniosynostosis and cleft lip and/or palate (CLP). The challenges in measuring the standard outcomes of function, aesthetics, and health-related quality of life are discussed, along with the importance of developing evidence and studying quality improvement in this specialty. The need to define specific and comprehensive goals is discussed with a focus on patient-reported outcomes (PROs). Examples from the development of the CLEFT-Q, a PRO instrument for patients with CLP, are provided to support the need to seek the patient perspective. PMID:23506771

  1. Electronic Patient-Reported Outcomes: The Time Is Ripe for Integration Into Patient Care and Clinical Research.

    PubMed

    Schwartzberg, Lee

    2016-01-01

    In the emerging team-based approach to delivering cancer care, collecting patient-reported outcomes (PROs) provides longitudinal monitoring of treatment adverse effects, disease complications, functional statuses, and psychological states throughout the cancer continuum for all providers to use. Electronic systems offer added capabilities, including easy quantitation of individual symptom items and aggregated scales, standardization, and longitudinal tracking of patient surveys for trend analysis over time. An ideal electronic PRO (ePRO) platform is clinically relevant, validated, and reliable and would offer patient usability. Additionally, it should allow for automated responses to and from patients, have scheduling functionality, and send real-time alerts to site personnel and patients. Clinical interfaces should be easy to read and integrated into the electronic medical record. Multiple ePRO systems, often using electronic tablets, have been created and are beginning to be widely deployed. The Patient Care Monitor is one example of a system that has evolved into a comprehensive patient engagement platform, with a complete review of systems survey and capabilities for mobile health usage. Recent clinical trials have established ePRO systems as an effective method of providing information, which aids improved patient outcomes, including reduced health resource utilization and longer time on therapy. ePROs are also increasingly incorporated into clinical trials, where they can provide more thorough reporting of adverse events than can be captured by alternative methods. Mobile devices have the potential to become the method by which all members of the provider team communicate with patients both at the point-of-care and between clinic visits to optimize care delivery. PMID:27249775

  2. Test-Retest Reliability of Two Patient-Report Measures for Use in Adults with ADHD

    ERIC Educational Resources Information Center

    Matza, Louis S.; Van Brunt, David L.; Cates, Charlotte; Murray, Lindsey T.

    2011-01-01

    Aims: Childhood attention-deficit/hyperactivity disorder (ADHD) frequently persists into adulthood and continues to impair health-related quality of life (HRQL). Thus, it is important to have validated symptom and HRQL measures for assessing treatment outcomes in this population. The purpose of the current analysis was to assess test-retest…

  3. Functional Status Assessment of Patients With COPD: A Systematic Review of Performance-Based Measures and Patient-Reported Measures.

    PubMed

    Liu, Yang; Li, Honghe; Ding, Ning; Wang, Ningning; Wen, Deliang

    2016-05-01

    Presently, there is no recommendation on how to assess functional status of chronic obstructive pulmonary disease (COPD) patients. This study aimed to summarize and systematically evaluate these measures.Studies on measures of COPD patients' functional status published before the end of January 2015 were included using a search filters in PubMed and Web of Science, screening reference lists of all included studies, and cross-checking against some relevant reviews. After title, abstract, and main text screening, the remaining was appraised using the Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) 4-point checklist. All measures from these studies were rated according to best-evidence synthesis and the best-rated measures were selected.A total of 6447 records were found and 102 studies were reviewed, suggesting 44 performance-based measures and 14 patient-reported measures. The majority of the studies focused on internal consistency, reliability, and hypothesis testing, but only 21% of them employed good or excellent methodology. Their common weaknesses include lack of checks for unidimensionality, inadequate sample sizes, no prior hypotheses, and improper methods. On average, patient-reported measures perform better than performance-based measures. The best-rated patient-reported measures are functional performance inventory (FPI), functional performance inventory short form (FPI-SF), living with COPD questionnaire (LCOPD), COPD activity rating scale (CARS), University of Cincinnati dyspnea questionnaire (UCDQ), shortness of breath with daily activities (SOBDA), and short-form pulmonary functional status scale (PFSS-11), and the best-rated performance-based measures are exercise testing: 6-minute walk test (6MWT), endurance treadmill test, and usual 4-meter gait speed (usual 4MGS).Further research is needed to evaluate the reliability and validity of performance-based measures since present studies failed to provide convincing

  4. The Aphasia Communication Outcome Measure (ACOM): Dimensionality, Item Bank Calibration, and Initial Validation

    ERIC Educational Resources Information Center

    Hula, William D.; Doyle, Patrick J.; Stone, Clement A.; Hula, Shannon N. Austermann; Kellough, Stacey; Wambaugh, Julie L.; Ross, Katherine B.; Schumacher, James G.; St. Jacque, Ann

    2015-01-01

    Purpose: The purpose of this study is to investigate the structure and measurement properties of the Aphasia Communication Outcome Measure (ACOM), a patient-reported outcome measure of communicative functioning for persons with aphasia. Method: Three hundred twenty-nine participants with aphasia responded to 177 items asking about communicative…

  5. Patient-reported satisfaction and cosmesis outcomes following laparoscopic adrenalectomy: Laparoendoscopic single-site adrenalectomy vs. conventional laparoscopic adrenalectomy

    PubMed Central

    Inoue, Shogo; Ikeda, Kenichiro; Kobayashi, Kanao; Kajiwara, Mitsuru; Teishima, Jun; Matsubara, Akio

    2014-01-01

    Introduction: We evaluate patient-reported satisfaction and cosmesis of laparoendoscopic single-site adrenalectomy (LESS-A) in comparison with that of conventional laparoscopic adrenalectomy (CLA). Methods: A total of 19 and 104 patients who respectively underwent LESS-A and CLA between May 1996 and June 2011 were included in the study. Questionnaires inquiring about scar pain (0: not painful, 10: very painful), satisfaction (0: not satisfied, 10: very satisfied) and cosmesis (0: very unsightly, 10: very beautiful) on the basis of a visual analogue scale were sent to patients postoperatively. Results: The respondents consisted of 11 and 54 patients who underwent LESS-A and CLA, respectively. There was no significant inter-group difference in age, sex, affected side or body mass index. No significant differences were observed in operative time or estimated blood loss. There were also no significant differences in pain (0.67 vs. 0.57, p = 0.393), satisfaction (8.92 vs. 8.46, p = 0.453), or cosmesis score (8.58 vs. 8.00, p = 0.487) between the LESS-A and CLA groups overall. In female patients, the satisfaction score was significantly higher in the LESS-A group than in the CLA group (10.0 vs. 8.72, p = 0.049). In young patients (<50 years old), the satisfaction score was also significantly higher in the LESS-A group than in the CLA group (9.17 vs. 6.38, p = 0.036). Conclusions: Young patients and female patients who had received LESS-A adrenal surgery were more satisfied with the scar outcomes than were the young patients and female patients who had received CLA. We suggest that this patient subset most values the cosmetic benefits of LESS-A. PMID:24454596

  6. Symptoms and Distress in Children With Advanced Cancer: Prospective Patient-Reported Outcomes From the PediQUEST Study

    PubMed Central

    Wolfe, Joanne; Orellana, Liliana; Ullrich, Christina; Cook, E. Francis; Kang, Tammy I.; Rosenberg, Abby; Geyer, Russ; Feudtner, Chris; Dussel, Veronica

    2015-01-01

    Purpose Thousands of children are living with advanced cancer; yet patient-reported outcomes (PROs) have rarely been used to describe their experiences. We aimed to describe symptom distress in 104 children age 2 years or older with advanced cancer enrolled onto the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) Study (multisite clinical trial evaluating an electronic PRO system). Methods Symptom data were collected using age- and respondent-adapted versions of the PediQUEST Memorial Symptom Assessment Scale (PQ-MSAS) at most once per week. Clinical and treatment data were obtained from medical records. Individual symptom scores were dichotomized into high/low distress. Determinants of PQ-MSAS scores were explored using linear mixed-effects models. Results During 9 months of follow-up, PQ-MSAS was administered 920 times: 459 times in teens (99% self-report), 249 times in children ages 7 to 12 years (96% child/parent report), and 212 times in those ages 2 to 6 years (parent reports). Common symptoms included pain (48%), fatigue (46%), drowsiness (39%), and irritability (37%); most scores indicated high distress. Among the 73 PQ-MSAS surveys administered in the last 12 weeks of life, pain was highly prevalent (62%; 58% with high distress). Being female, having a brain tumor, experiencing recent disease progression, and receiving moderate- or high-intensity cancer-directed therapy in the prior 10 days were associated with worse PQ-MSAS scores. In the final 12 weeks of life, receiving mild cancer-directed therapy was associated with improved psychological PQ-MSAS scores. Conclusion Children with advanced cancer experience high symptom distress. Strategies to promote intensive symptom management are indicated, especially with disease progression or administration of intensive treatments. PMID:25918277

  7. Patient reported outcomes in head and neck cancer: selecting instruments for quality of life integration in clinical protocols

    PubMed Central

    2010-01-01

    Background Health Related Quality of Life has been used in medical research for more than twenty years, being progressively accepted during the last decade as an important patient reported outcome. Considering the multidimensional approach involved in Health Related Quality of Life assessment, instrument applicability and cultural adaptation must be tested for each population. In order to select the most appropriate instrument for Head and Neck cancer patients, two major Health Related Quality of Life specific questionnaires for Head and Neck cancer patients were compared. Conceptual differences, psychometric characteristics, scores, reliability, construct validity and sensitivity to symptomatology, tumour location, tumour size were analyzed. Methods 102 consecutive Head and Neck cancer patients completed two different Health Related Quality of Life questionnaires: EORTC QLQ-C30 and its specific head and neck module QLQ-H&N35 and the Functional Assessment of Cancer Therapy Scales (FACT-H&N). Patients completed the questionnaires, immediately before consultation as a part of the routine evaluation. Results A greater variability was always found in the EORTC QLC-C30 questionnaire's scores for all comparable domains. Both instruments revealed a good internal consistency and demonstrated to be good tools to distinguish symptomatic patients. The EORTC questionnaires still demonstrated sensitivity to distinguish T3 and T4 staging. Conceptual differences and the psychometric characteristics are discussed. Our results suggest that these two instruments assess different aspects of Health Related Quality of Life - the questionnaires should be used separately and chosen according to the study objectives and methodology. Conclusions This study emphases the importance in selecting the appropriate tool as a critical success factor in implementing routine Health Related Quality of Life assessment in clinical practice. This decision assumes particularly importance when utilization

  8. Effect of Ruxolitinib Therapy on Myelofibrosis-Related Symptoms and Other Patient-Reported Outcomes in COMFORT-I: A Randomized, Double-Blind, Placebo-Controlled Trial

    PubMed Central

    Mesa, Ruben A.; Gotlib, Jason; Gupta, Vikas; Catalano, John V.; Deininger, Michael W.; Shields, Alan L.; Miller, Carole B.; Silver, Richard T.; Talpaz, Moshe; Winton, Elliott F.; Harvey, Jimmie H.; Hare, Thomas; Erickson-Viitanen, Susan; Sun, William; Sandor, Victor; Levy, Richard S.; Kantarjian, Hagop M.; Verstovsek, Srdan

    2013-01-01

    Purpose To assess the effects of ruxolitinib on symptom burden and quality of life (QoL) and to evaluate the ability of the modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 to measure meaningful changes in myelofibrosis-related symptoms in patients with myelofibrosis. Patients and Methods COMFORT-I (Controlled Myelofibrosis Study With Oral JAK Inhibitor Treatment–I) is a double-blind, placebo-controlled phase III study evaluating ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis. Exploratory analyses were conducted on the following patient-reported outcomes (PROs) assessments: modified MFSAF v2.0 (individual symptoms and Total Symptom Score [TSS]), European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale, and Patient Global Impression of Change (PGIC). Results Patients receiving ruxolitinib experienced improvements in individual myelofibrosis-related symptoms, although patients receiving placebo experienced worsening (P < .001). The majority (91%) of ruxolitinib-treated patients designated as ≥ 50% TSS responders (≥ 50% TSS improvement) self-reported their condition as either “Much improved” or “Very much improved” on the PGIC. These patients achieved significant improvements in the EORTC QLQ-C30 functional domains and Global Health Status/QoL versus patients receiving placebo, who experienced worsening on these measures (P ≤ .0135). Ruxolitinib-treated patients with a lesser degree of symptom improvement (< 50% TSS responders) also achieved improvements over placebo on these measures. The degree of spleen volume reduction with ruxolitinib correlated with improvements in TSS, PGIC, PROMIS Fatigue Scale, and EORTC Global Health Status/QoL. Ruxolitinib-treated patients who achieved a ≥ 35% reduction in spleen volume experienced the greatest improvements in these PROs. Conclusion

  9. The Computer-based Health Evaluation Software (CHES): a software for electronic patient-reported outcome monitoring

    PubMed Central

    2012-01-01

    Background Patient-reported Outcomes (PROs) capturing e.g., quality of life, fatigue, depression, medication side-effects or disease symptoms, have become important outcome parameters in medical research and daily clinical practice. Electronic PRO data capture (ePRO) with software packages to administer questionnaires, storing data, and presenting results has facilitated PRO assessment in hospital settings. Compared to conventional paper-pencil versions of PRO instruments, ePRO is more economical with regard to staff resources and time, and allows immediate presentation of results to the medical staff. The objective of our project was to develop software (CHES – Computer-based Health Evaluation System) for ePRO in hospital settings and at home with a special focus on the presentation of individual patient’s results. Methods Following the Extreme Programming development approach architecture was not fixed up-front, but was done in close, continuous collaboration with software end users (medical staff, researchers and patients) to meet their specific demands. Developed features include sophisticated, longitudinal charts linking patients’ PRO data to clinical characteristics and to PRO scores from reference populations, a web-interface for questionnaire administration, and a tool for convenient creating and editing of questionnaires. Results By 2012 CHES has been implemented at various institutions in Austria, Germany, Switzerland, and the UK and about 5000 patients participated in ePRO (with around 15000 assessments in total). Data entry is done by the patients themselves via tablet PCs with a study nurse or an intern approaching patients and supervising questionnaire completion. Discussion During the last decade several software packages for ePRO have emerged for different purposes. Whereas commercial products are available primarily for ePRO in clinical trials, academic projects have focused on data collection and presentation in daily clinical practice and

  10. Efficacy of strength and aerobic exercise on patient-reported outcomes and structural changes in patients with knee osteoarthritis: study protocol for a randomized controlled trial

    PubMed Central

    2013-01-01

    weeks, 1 year and 2 years post-randomization. The interventions will be a 14 weeks exercise program. Discussion Although exercise therapy has been found to be effective in knee osteoarthritis, the knowledge of the underlying mechanisms for why exercise works is lacking. This study will contribute with knowledge on the efficacy of strength exercise versus cycling on patient-reported outcomes, cartilage quality and cost-effectiveness. Trial registration Clinicaltrial.gov Identifier: NCT01682980. PMID:24028201

  11. Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review: Perspectives From Industry, the Food and Drug Administration, and the Patient.

    PubMed

    Basch, Ethan; Geoghegan, Cindy; Coons, Stephen Joel; Gnanasakthy, Ari; Slagle, Ashley F; Papadopoulos, Elektra J; Kluetz, Paul G

    2015-06-01

    Data reported directly by patients about how they feel and function are rarely included in oncology drug labeling in the United States, in contrast to Europe and to nononcology labeling in the United States, where this practice is more common. Multiple barriers exist, including challenges unique to oncology trials, and industry's concerns regarding cost, logistical complexities, and the Food and Drug Administration's (FDA's) rigorous application of its 2009 guidance on the use of patient-reported outcome (PRO) measures. A panel consisting of representatives of industry, FDA, the PRO Consortium, clinicians, and patients was assembled at a 2014 workshop cosponsored by FDA to identify practical recommendations for overcoming these barriers. Key recommendations included increasing proactive encouragement by FDA to clinical trial sponsors for including PROs in drug development programs; provision of comprehensive PRO plans by sponsors to FDA early in drug development; promotion of an oncology-specific PRO research agenda; development of an approach to existing ("legacy") PRO measures, when appropriate (focused initially on symptoms and functional status); and increased FDA and industry training in PRO methodology. FDA has begun implementing several of these recommendations. PMID:26181187

  12. Patient-reported outcome results in patients with type 2 diabetes treated with once-weekly dulaglutide: data from the AWARD phase III clinical trial programme.

    PubMed

    Yu, M; Van Brunt, K; Varnado, O J; Boye, K S

    2016-04-01

    We evaluated patient-reported outcome (PRO) measures from the Assessment of Weekly AdministRation of LY2189265 (dulaglutide) in Diabetes (AWARD) clinical trial programme for dulaglutide (1.5 mg and 0.75 mg) in patients with type 2 diabetes (T2D). The Impact of Weight on Self-Perception (IW-SP), Impact of Weight on Ability to Perform Physical Activities of Daily Living (APPADL), Impact of Weight on Quality of Life-Lite, EQ-5D, Diabetes Treatment Satisfaction Questionnaire (DTSQ), Diabetes Symptom Checklist-Revised and Adult Low Blood Sugar Survey were administered and analysed for changes from baseline in one or more AWARD studies. Significant within-group changes from baseline to the primary time point were observed for several PRO measures across all studies. Compared with insulin glargine, significantly greater improvements in the IW-SP score were observed with dulaglutide 1.5 mg and with both dulaglutide doses in the APPADL score. Both dulaglutide doses resulted in significantly greater improvement in DTSQ scores (all subscales) compared with exenatide. Dulaglutide 1.5 mg also resulted in significantly greater improvement on the DTSQ hyperglycaemia subscale compared with metformin. Overall, these PRO results suggest that dulaglutide is beneficial in the treatment of T2D. PMID:26691396

  13. Cultural adaptation: translatability assessment and linguistic validation of the patient-reported outcome instrument for irritable bowel syndrome with diarrhea

    PubMed Central

    Delgado-Herrera, Leticia; Lasch, Kathryn; Popielnicki, Ana; Nishida, Akito; Arbuckle, Rob; Banderas, Benjamin; Zentner, Susan; Gagainis, Ingrid; Zeiher, Bernhardt

    2016-01-01

    Background and objective Following a 2009 US Food and Drug Administration guidance, a new patient-reported outcome (PRO) instrument was developed to support end points in multinational clinical trials assessing irritable bowel syndrome with diarrhea (IBS-D) symptom severity. Our objective was to assess the translatability of the IBS-D PRO instrument into ten languages, and subsequently perform a cultural adaptation/linguistic validation of the questionnaire into Japanese and US Spanish. Materials and methods Translatability assessments of the US English version of the IBS-D PRO were performed by experienced PRO translators who were native speakers of each target language and currently residing in target-language countries. Languages were Chinese (People’s Republic of China), Dutch (the Netherlands), French (Belgium), German (Germany), Japanese (Japan), Polish (Poland), Portuguese (Brazil), Russian (Russia), Spanish (Mexico), and Spanish (US). The project team assessed the instrument to identify potential linguistic and/or cultural adaptation issues. After the issues identified were resolved, the instrument was translated into Spanish (US) and Japanese through a process of two forward translations, one reconciled translation, and one backward translation. The project team reviewed the translated versions before the instruments were evaluated by cognitive debriefing interviews with samples of five Spanish (US) and five Japanese IBS-D patients. Results Linguistic and cultural adaptation concerns identified during the translatability assessment required minor revisions, mainly the presentation of dates/times and word structure. During the cognitive debriefing interviews, two of five Spanish respondents misunderstood the term “bowel movement” to mean only diarrhea in the Spanish version. Consequently, the term was changed from “movimiento intestinal” to “evacuaciones”. None of the Japanese respondents identified issues with the Japanese version. Conclusion

  14. Patient-reported Outcomes in Randomised Controlled Trials of Prostate Cancer: Methodological Quality and Impact on Clinical Decision Making

    PubMed Central

    Efficace, Fabio; Feuerstein, Michael; Fayers, Peter; Cafaro, Valentina; Eastham, James; Pusic, Andrea; Blazeby, Jane

    2014-01-01

    Context Patient-reported outcomes (PRO) data from randomised controlled trials (RCTs) are increasingly used to inform patient-centred care as well as clinical and health policy decisions. Objective The main objective of this study was to investigate the methodological quality of PRO assessment in RCTs of prostate cancer (PCa) and to estimate the likely impact of these studies on clinical decision making. Evidence acquisition A systematic literature search of studies was undertaken on main electronic databases to retrieve articles published between January 2004 and March 2012. RCTs were evaluated on a predetermined extraction form, including (1) basic trial demographics and clinical and PRO characteristics; (2) level of PRO reporting based on the recently published recommendations by the International Society for Quality of Life Research; and (3) bias, assessed using the Cochrane Risk of Bias tool. Studies were systematically analysed to evaluate their relevance for supporting clinical decision making. Evidence synthesis Sixty-five RCTs enrolling a total of 22 071 patients were evaluated, with 31 (48%) in patients with nonmetastatic disease. When a PRO difference between treatments was found, it related in most cases to symptoms only (n = 29, 58%). Although the extent of missing data was generally documented (72% of RCTs), few reported details on statistical handling of this data (18%) and reasons for dropout (35%). Improvements in key methodological aspects over time were found. Thirteen (20%) RCTs were judged as likely to be robust in informing clinical decision making. Higher-quality PRO studies were generally associated with those RCTs that had higher internal validity. Conclusions Including PRO in RCTs of PCa patients is critical for better evaluating the treatment effectiveness of new therapeutic approaches. Marked improvements in PRO quality reporting over time were found, and it is estimated that at least one-fifth of PRO RCTs have provided sufficient

  15. Rasch-family models are more valuable than score-based approaches for analysing longitudinal patient-reported outcomes with missing data.

    PubMed

    de Bock, Elodie; Hardouin, Jean-Benoit; Blanchin, Myriam; Le Neel, Tanguy; Kubis, Gildas; Bonnaud-Antignac, Angélique; Dantan, Etienne; Sébille, Véronique

    2013-12-16

    The objective was to compare classical test theory and Rasch-family models derived from item response theory for the analysis of longitudinal patient-reported outcomes data with possibly informative intermittent missing items. A simulation study was performed in order to assess and compare the performance of classical test theory and Rasch model in terms of bias, control of the type I error and power of the test of time effect. The type I error was controlled for classical test theory and Rasch model whether data were complete or some items were missing. Both methods were unbiased and displayed similar power with complete data. When items were missing, Rasch model remained unbiased and displayed higher power than classical test theory. Rasch model performed better than the classical test theory approach regarding the analysis of longitudinal patient-reported outcomes with possibly informative intermittent missing items mainly for power. This study highlights the interest of Rasch-based models in clinical research and epidemiology for the analysis of incomplete patient-reported outcomes data. PMID:24346165

  16. A Prospective Study Assessing Complication Rates and Patient-Reported Outcomes in Breast Reconstructions Using a Novel, Deep Dermal Human Acellular Dermal Matrix

    PubMed Central

    Vu, Michael M.; De Oliveira, Gildasio S.; Mayer, Kristen E.; Blough, Jordan T.

    2015-01-01

    Abstract Background: The value proposition of an acellular dermal matrix (ADM) taken from the deep dermis is that the allograft may be more porous, allowing for enhanced integration and revascularization. In turn, this characteristic may attenuate complications related to foreign body reactions, seromas, and infection. However, this is juxtaposed against the potential loss of allograft structural integrity, with subsequent risk of malposition and extrusion. Despite the active use of novel, deep dermal ADMs, the clinical outcomes of this new technology has not been well studied. Methods: This is a prospective study to evaluate surgical and patient-reported outcomes using a deep dermal ADM, FlexHD Pliable. Surgical outcomes and BREAST-Q patient-reported outcomes were evaluated postoperatively at 2- and 6-month time points. Results: Seventy-two breasts (41 patients) underwent reconstruction. Complication rate was 12.5%, including 2 hematomas and 7 flap necroses. One case of flap necrosis led to reconstructive failure. Notably, there were no cases of infection, seroma, or implant extrusion or malposition. Average BREAST-Q scores were satisfaction with outcome (70.13 ± 23.87), satisfaction with breasts (58.53 ± 20.00), psychosocial well being (67.97 ± 20.93), sexual well being (54.11 ± 27.72), and physical well being (70.45 ± 15.44). Two-month postoperative BREAST-Q scores decreased compared with baseline and returned to baseline by 6 months. Postoperative radiation therapy had a negative effect on satisfaction with breasts (P = 0.004) and sexual well being (P = 0.006). Conclusions: Deep dermal ADM is a novel modification of traditional allograft technology. Use of the deep dermal ADM yielded acceptably low complication rates and satisfactory patient-reported outcomes. PMID:26894010

  17. Patient reported outcomes in a trial of combination therapy with etanercept and methotrexate for rheumatoid arthritis: the TEMPO trial

    PubMed Central

    van der Heijde, D; Klareskog, L; Singh, A; Tornero, J; Melo‐Gomes, J; Codreanu, C; Pedersen, R; Freundlich, B; Fatenejad, S

    2006-01-01

    Objective To compare patient reported measures of function, health related quality of life (QoL), and satisfaction with medication among patients with rheumatoid arthritis (RA) treated with methotrexate (MTX), etanercept, or both for up to 1 year. Methods In a 52 week, double blind, clinical trial, patients with active RA were randomised to receive etanercept 25 mg twice weekly, methotrexate up to 20 mg weekly, or combination therapy. The Health Assessment Questionnaire (HAQ) disability index, EuroQoL health status visual analogue scale (EQ‐5D VAS), patient global assessment, and patient general health VAS were administered at baseline and weeks 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, and 52. Satisfaction with the medication was compared at 52 weeks. Results Of 682 enrolled patients, 522 completed 52 weeks of treatment. Mean improvement from baseline in HAQ score was 0.65, 0.70, and 1.0 for MTX, etanercept, and the combination, respectively. The mean percentage and absolute improvement in the HAQ was significantly higher (p<0.01) for combination therapy than for either of the monotherapies. Combination therapy produced significantly more rapid achievement of HAQ ⩽0.5 sustained for 6 months than either of the monotherapies (p<0.01). Compared with patients receiving monotherapy, those receiving combination therapy achieved a significantly better (p<0.05) health state as measured by the EQ‐5D VAS (mean (SD) 63.7 (3.2), 66.8 (3.2), 72.7 (3.1) for MTX, etanercept, and the combination, respectively). Results were similar for other assessments (p<0.01). Patients in combination and etanercept groups were significantly more likely (p<0.0001, p = 0.0009, respectively) to report satisfaction with the medication. Conclusions Combination therapy with etanercept and methotrexate improved function, QoL, and satisfaction with the medication significantly more than monotherapy. PMID:16079172

  18. The Gait Deviation Index Is Associated with Hip Muscle Strength and Patient-Reported Outcome in Patients with Severe Hip Osteoarthritis—A Cross-Sectional Study

    PubMed Central

    Rosenlund, Signe; Holsgaard-Larsen, Anders; Overgaard, Søren; Jensen, Carsten

    2016-01-01

    Background The Gait Deviation Index summarizes overall gait ‘quality’, based on kinematic data from a 3-dimensional gait analysis. However, it is unknown which clinical outcomes may affect the Gait Deviation Index in patients with primary hip osteoarthritis. The aim of this study was to investigate associations between Gait Deviation Index as a measure of gait ‘quality’ and hip muscle strength and between Gait Deviation Index and patient-reported outcomes in patients with primary hip osteoarthritis. Method Forty-seven patients (34 males), aged 61.1 ± 6.7 years, with BMI 27.3 ± 3.4 (kg/m2) and with severe primary hip osteoarthritis underwent 3-dimensional gait analysis. Mean Gait Deviation Index, pain after walking and maximal isometric hip muscle strength (flexor, extensor, and abductor) were recorded. All patients completed the ‘Physical Function Short-form of the Hip disability and Osteoarthritis Outcome Score (HOOS-Physical Function) and the Hip disability and Osteoarthritis Outcome Score subscales for pain (HOOS-Pain) and quality-of-life (HOOS-QOL). Results Mean Gait Deviation Index was positively associated with hip abduction strength (p<0.01, r = 0.40), hip flexion strength (p = 0.01, r = 0.37), HOOS-Physical Function (p<0.01, r = 0.41) HOOS-QOL (p<0.01, r = 0.41), and negatively associated with HOOS-Pain after walking (p<0.01, r = -0.45). Adjusting the analysis for walking speed did not affect the association. Conclusion Patients with the strongest hip abductor and hip flexor muscles had the best gait ‘quality’. Furthermore, patients with higher physical function, quality of life scores and lower pain levels demonstrated better gait ‘quality’. These findings indicate that interventions aimed at improving hip muscle strength and pain management may to a moderate degree improve the overall gait ‘quality’ in patients with primary hip OA. PMID:27065007

  19. Routine outcome measures in Canada.

    PubMed

    Kisely, Steve; Adair, Carol E; Lin, Elizabeth; Marriott, Brian

    2015-01-01

    Canada is a federal country of 10 provinces and three territories. High level information on mental health conditions and service use has mostly been generated from administrative data collected by provinces and territories. These include four major types - hospital admissions and discharges, physician billings, ambulatory care services, and drug databases. At the national level, the Canadian Institute for Health Information brings together this information to produce indicators of outcome. Although these data provide information on patient and health system characteristics, they do not capture the full spectrum of formal and informal mental healthcare. These include changes in health status, functioning, community integration and quality of life. As a result, some jurisdictions have begun to implement more standardized measures of outcome such as the clinician-rated Health of the Nation Outcome Scales or the inpatient Resident Assessment Instrument - Mental Health. In this paper we provide an overview of mental-health-related data sources in Canada, highlight some of the more progressive practices beginning to emerge, and conclude with some thoughts about how the routine measurement and reporting of mental health outcomes in Canada might be advanced including efforts at engaging both clinicians and decision-makers. PMID:25738745

  20. Issues and Challenges With Integrating Patient-Reported Outcomes in Clinical Trials Supported by the National Cancer Institute–Sponsored Clinical Trials Networks

    PubMed Central

    Bruner, Deborah Watkins; Bryan, Charlene J.; Aaronson, Neil; Blackmore, C. Craig; Brundage, Michael; Cella, David; Ganz, Patricia A.; Gotay, Carolyn; Hinds, Pamela S.; Kornblith, Alice B.; Movsas, Benjamin; Sloan, Jeff; Wenzel, Lari; Whalen, Giles

    2016-01-01

    Purpose The objective of this report is to provide a historical overview of and the issues and challenges inherent in the incorporation of patient-reported outcomes (PROs) into multinational cancer clinical trials in the cancer cooperative groups. Methods An online survey of 12 cancer cooperative groups from the United States, Canada, and Europe was conducted between June and August of 2006. Each of the cooperative groups designated one respondent, who was a member of one of the PRO committees within the cooperative group. Results There was a 100% response rate, and all of the cancer clinical trial cooperative groups reported conducting PRO research. PRO research has been conducted in the cancer cooperative groups for an average of 15 years (range, 6 to 30 years), and all groups had multidisciplinary committees focused on the design of PRO end points and the choice of appropriate PRO measures for cancer clinical trials. The cooperative groups reported that 5% to 50% of cancer treatment trials and an estimated 50% to 75% of cancer control trials contained PRO primary and secondary end points. There was considerable heterogeneity among the cooperative groups with respect to the formal and informal policies and procedures or cooperative group culture towards PROs, investigator training/mentorship, and resource availability for the measurement and conduct of PRO research within the individual cooperatives. Conclusion The challenges faced by the cooperative groups to the incorporation of PROs into cancer clinical trials are varied. Some common opportunities for improvement include the adoption of standardized training/mentorship mechanisms for investigators for the conduct of PRO assessments and data collection and the development of minimal criteria for PRO measure acceptability. A positive cultural shift has occurred in most of the cooperative groups related to the incorporation of PROs in clinical trials; however, financial and other resource barriers remain and need

  1. Effect of Certolizumab Pegol on Multiple Facets of Psoriatic Arthritis as Reported by Patients: 24-Week Patient-Reported Outcome Results of a Phase III, Multicenter Study

    PubMed Central

    Gladman, D; Fleischmann, R; Coteur, G; Woltering, F; Mease, P J

    2014-01-01

    Objective To examine the effect of certolizumab pegol (CZP) on patient-reported outcomes (PROs) in psoriatic arthritis (PsA) patients with and without prior tumor necrosis factor (TNF) inhibitor exposure. Methods The ongoing phase III RAPID-PsA trial was double blind and placebo controlled to week 24. Patients were randomized 1:1:1 to placebo every 2 weeks or CZP 400 mg at weeks 0, 2, and 4, followed by either CZP 200 mg every 2 weeks or CZP 400 mg every 4 weeks. PRO measures evaluated were the Health Assessment Questionnaire (HAQ) disability index (DI), health status (measured by the Short Form 36 [SF-36] health survey), Psoriatic Arthritis Quality of Life (PsAQOL), Fatigue Assessment Scale, patient assessment of pain (visual analog scale), and Dermatology Life Quality Index (DLQI). Post hoc analyses of PROs in patients with and without prior TNF inhibitor exposure were conducted. Change from baseline for all PROs was analyzed for the randomized population using analysis of covariance with last observation carried forward imputation. Results A total of 409 patients were randomized. Twenty percent had received a prior TNF inhibitor. Baseline demographics were similar between the treatment groups. At week 24, clinically meaningful differences in HAQ DI, SF-36, PsAQOL, fatigue, pain, and DLQI were observed in both CZP arms versus placebo (P < 0.001), irrespective of prior TNF inhibitor exposure. More CZP-treated patients reached SF-36 general population norms than placebo-treated patients. Conclusion Both CZP dosing schedules provided rapid improvements in PROs across multiple disease aspects in patients with PsA. The benefits of CZP treatment for health-related quality of life were seen across generic, PsA-specific, and dermatology-specific measures and were observed in patients regardless of prior TNF inhibitor exposure. PMID:24339179

  2. Development of the National Cancer Institute's patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE).

    PubMed

    Basch, Ethan; Reeve, Bryce B; Mitchell, Sandra A; Clauser, Steven B; Minasian, Lori M; Dueck, Amylou C; Mendoza, Tito R; Hay, Jennifer; Atkinson, Thomas M; Abernethy, Amy P; Bruner, Deborah W; Cleeland, Charles S; Sloan, Jeff A; Chilukuri, Ram; Baumgartner, Paul; Denicoff, Andrea; St Germain, Diane; O'Mara, Ann M; Chen, Alice; Kelaghan, Joseph; Bennett, Antonia V; Sit, Laura; Rogak, Lauren; Barz, Allison; Paul, Diane B; Schrag, Deborah

    2014-09-01

    The standard approach for documenting symptomatic adverse events (AEs) in cancer clinical trials involves investigator reporting using the National Cancer Institute's (NCI's) Common Terminology Criteria for Adverse Events (CTCAE). Because this approach underdetects symptomatic AEs, the NCI issued two contracts to create a patient-reported outcome (PRO) measurement system as a companion to the CTCAE, called the PRO-CTCAE. This Commentary describes development of the PRO-CTCAE by a group of multidisciplinary investigators and patient representatives and provides an overview of qualitative and quantitative studies of its measurement properties. A systematic evaluation of all 790 AEs listed in the CTCAE identified 78 appropriate for patient self-reporting. For each of these, a PRO-CTCAE plain language term in English and one to three items characterizing the frequency, severity, and/or activity interference of the AE were created, rendering a library of 124 PRO-CTCAE items. These items were refined in a cognitive interviewing study among patients on active cancer treatment with diverse educational, racial, and geographic backgrounds. Favorable measurement properties of the items, including construct validity, reliability, responsiveness, and between-mode equivalence, were determined prospectively in a demographically diverse population of patients receiving treatments for many different tumor types. A software platform was built to administer PRO-CTCAE items to clinical trial participants via the internet or telephone interactive voice response and was refined through usability testing. Work is ongoing to translate the PRO-CTCAE into multiple languages and to determine the optimal approach for integrating the PRO-CTCAE into clinical trial workflow and AE analyses. It is envisioned that the PRO-CTCAE will enhance the precision and patient-centeredness of adverse event reporting in cancer clinical research. PMID:25265940

  3. Development of the National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

    PubMed Central

    Reeve, Bryce B.; Mitchell, Sandra A.; Clauser, Steven B.; Minasian, Lori M.; Dueck, Amylou C.; Mendoza, Tito R.; Hay, Jennifer; Atkinson, Thomas M.; Abernethy, Amy P.; Bruner, Deborah W.; Cleeland, Charles S.; Sloan, Jeff A.; Chilukuri, Ram; Baumgartner, Paul; Denicoff, Andrea; St. Germain, Diane; O’Mara, Ann M.; Chen, Alice; Kelaghan, Joseph; Bennett, Antonia V.; Sit, Laura; Rogak, Lauren; Barz, Allison; Paul, Diane B.; Schrag, Deborah

    2014-01-01

    The standard approach for documenting symptomatic adverse events (AEs) in cancer clinical trials involves investigator reporting using the National Cancer Institute’s (NCI’s) Common Terminology Criteria for Adverse Events (CTCAE). Because this approach underdetects symptomatic AEs, the NCI issued two contracts to create a patient-reported outcome (PRO) measurement system as a companion to the CTCAE, called the PRO-CTCAE. This Commentary describes development of the PRO-CTCAE by a group of multidisciplinary investigators and patient representatives and provides an overview of qualitative and quantitative studies of its measurement properties. A systematic evaluation of all 790 AEs listed in the CTCAE identified 78 appropriate for patient self-reporting. For each of these, a PRO-CTCAE plain language term in English and one to three items characterizing the frequency, severity, and/or activity interference of the AE were created, rendering a library of 124 PRO-CTCAE items. These items were refined in a cognitive interviewing study among patients on active cancer treatment with diverse educational, racial, and geographic backgrounds. Favorable measurement properties of the items, including construct validity, reliability, responsiveness, and between-mode equivalence, were determined prospectively in a demographically diverse population of patients receiving treatments for many different tumor types. A software platform was built to administer PRO-CTCAE items to clinical trial participants via the internet or telephone interactive voice response and was refined through usability testing. Work is ongoing to translate the PRO-CTCAE into multiple languages and to determine the optimal approach for integrating the PRO-CTCAE into clinical trial workflow and AE analyses. It is envisioned that the PRO-CTCAE will enhance the precision and patient-centeredness of adverse event reporting in cancer clinical research. PMID:25265940

  4. Routine outcome measurement in Australia.

    PubMed

    Burgess, Philip; Pirkis, Jane; Coombs, Tim

    2015-08-01

    Australia has been implementing routine outcome measurement in its specialized public sector mental health services for over a decade. It uses a range of clinician-rated and consumer-rated measures that are administered at set times during episodes of inpatient, ambulatory and community residential episodes of care. Routine outcome measurement is now embedded in service delivery, and data are made available in a variety of ways to different audiences. These data are used by policy-makers and planners to inform decisions about system-wide reforms, by service managers to monitor quality and effectiveness, and by clinicians to guide clinical decision-making and to promote dialogue with consumers. Consumers, carers and the general community can use these data to ensure that services are accountable for the care they deliver. This paper describes the status quo in Australia with respect to routine outcome measurement, discusses the factors that led to its successful implementation, and considers the steps that are necessary for its continued development. PMID:25768326

  5. Patient-Reported Outcome Results From the Open-Label Phase III AURELIA Trial Evaluating Bevacizumab-Containing Therapy for Platinum-Resistant Ovarian Cancer

    PubMed Central

    Stockler, Martin R.; Hilpert, Felix; Friedlander, Michael; King, Madeleine T.; Wenzel, Lari; Lee, Chee Khoon; Joly, Florence; de Gregorio, Nikolaus; Arranz, José Angel; Mirza, Mansoor Raza; Sorio, Roberto; Freudensprung, Ulrich; Sneller, Vesna; Hales, Gill; Pujade-Lauraine, Eric

    2014-01-01

    Purpose To determine the effects of bevacizumab on patient-reported outcomes (PROs; secondary end point) in the AURELIA trial. Patients and Methods Patients with platinum-resistant ovarian cancer were randomly assigned to chemotherapy alone (CT) or with bevacizumab (BEV-CT). PROs were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–Ovarian Cancer Module 28 (EORTC QLQ-OV28) and Functional Assessment of Cancer Therapy–Ovarian Cancer symptom index (FOSI) at baseline and every two or three cycles (8/9 weeks) until disease progression. The primary PRO hypothesis was that more patients receiving BEV-CT than CT would achieve at least a 15% (≥ 15-point) absolute improvement on the QLQ-OV28 abdominal/GI symptom subscale (items 31-36) at week 8/9. Patients with missing week 8/9 questionnaires were included as unimproved. Questionnaires from all assessments until disease progression were analyzed using mixed-model repeated-measures (MMRM) analysis. Sensitivity analyses were used to determine the effects of differing assumptions and methods for missing data. Results Baseline questionnaires were available from 89% of 361 randomly assigned patients. More BEV-CT than CT patients achieved a ≥ 15% improvement in abdominal/GI symptoms at week 8/9 (primary PRO end point, 21.9% v 9.3%; difference, 12.7%; 95% CI, 4.4 to 20.9; P = .002). MMRM analysis covering all time points also favored BEV-CT (difference, 6.4 points; 95% CI, 1.3 to 11.6; P = .015). More BEV-CT than CT patients achieved ≥ 15% improvement in FOSI at week 8/9 (12.2% v 3.1%; difference, 9.0%; 95% CI, 2.9% to 15.2%; P = .003). Sensitivity analyses gave similar results and conclusions. Conclusion Bevacizumab increased the proportion of patients achieving a 15% improvement in patient-reported abdominal/GI symptoms during chemotherapy for platinum-resistant ovarian cancer. PMID:24687829

  6. Adjuvant exemestane versus tamoxifen in premenopausal women with early breast cancer undergoing ovarian suppression: patient-reported outcomes in the TEXT and SOFT randomised trials

    PubMed Central

    Bernhard, Jürg; Luo, Weixiu; Ribi, Karin; Colleoni, Marco; Burstein, Harold J.; Tondini, Carlo; Pinotti, Graziella; Spazzapan, Simon; Ruhstaller, Thomas; Puglisi, Fabio; Pavesi, Lorenzo; Parmar, Vani; Regan, Meredith M.; Pagani, Olivia; Fleming, Gini F.; Francis, Prudence A.; Price, Karen N.; Coates, Alan S.; Gelber, Richard D.; Goldhirsch, Aron; Walley, Barbara A.

    2015-01-01

    Background Efficacy analysis of the combined IBCSG TEXT and SOFT trials showed a significant disease-free survival benefit for exemestane plus ovarian function suppression (OFS) compared with tamoxifen +OFS. We present patient-reported outcomes from these trials. Methods Between 7 November 2003 and 7 April 2011, 4717 premenopausal patients with hormone-receptor positive breast cancer were enrolled in TEXT or SOFT to receive unblinded adjuvant treatment with 5 years of exemestane+OFS or tamoxifen+OFS. Chemotherapy use was optional. Randomization was performed via IBCSG’s internet-based system with the use of permuted blocks and was stratified by chemotherapy use and lymph nodes status. Patients completed a quality of life (QoL) form including several global and symptom-specific indicators at baseline, every 6 months for 24 months, then annually during years 3 to 6. Differences in change of QoL from baseline between the two treatments were tested at short-, mid-, and long-term using mixed-models for repeated measures, for each trial with and without chemotherapy and overall. The analysis was intention-to-treat using treatment as randomly assigned. At the time of analysis, the median follow-up was 5·7 years (IQR, 3·7 to 6·9 years), with treatment and follow-up of patients ongoing. Findings Patients reported considerable worsening from baseline in key endocrine symptoms. Those on tamoxifen+OFS were more affected by hot flushes and sweats over five years than those on exemestane+OFS, although these symptoms improved. Patients on exemestane+OFS reported more vaginal dryness, greater loss of sexual interest, and difficulties becoming aroused. These differences persisted over time. An increase in bone/joint pain was more pronounced, particularly in the short-term, in patients on exemestane+OFS. Changes of global QoL indicators from baseline were small and similar between treatments over the whole treatment period. Interpretation Overall, from a QoL perspective, there

  7. Imputation by the mean score should be avoided when validating a Patient Reported Outcomes questionnaire by a Rasch model in presence of informative missing data

    PubMed Central

    2011-01-01

    Background Nowadays, more and more clinical scales consisting in responses given by the patients to some items (Patient Reported Outcomes - PRO), are validated with models based on Item Response Theory, and more specifically, with a Rasch model. In the validation sample, presence of missing data is frequent. The aim of this paper is to compare sixteen methods for handling the missing data (mainly based on simple imputation) in the context of psychometric validation of PRO by a Rasch model. The main indexes used for validation by a Rasch model are compared. Methods A simulation study was performed allowing to consider several cases, notably the possibility for the missing values to be informative or not and the rate of missing data. Results Several imputations methods produce bias on psychometrical indexes (generally, the imputation methods artificially improve the psychometric qualities of the scale). In particular, this is the case with the method based on the Personal Mean Score (PMS) which is the most commonly used imputation method in practice. Conclusions Several imputation methods should be avoided, in particular PMS imputation. From a general point of view, it is important to use an imputation method that considers both the ability of the patient (measured for example by his/her score), and the difficulty of the item (measured for example by its rate of favourable responses). Another recommendation is to always consider the addition of a random process in the imputation method, because such a process allows reducing the bias. Last, the analysis realized without imputation of the missing data (available case analyses) is an interesting alternative to the simple imputation in this context. PMID:21756330

  8. Patient-Reported Outcomes Following Breast Reconstruction Surgery and Therapeutic Mammoplasty: Prospective Evaluation 1 Year Post-Surgery with BREAST-Q Questionnaire.

    PubMed

    Shekhawat, Laxmi; Busheri, Laleh; Dixit, Santosh; Patel, Chaula; Dhar, Upendra; Koppiker, Chaitanyanand

    2015-12-01

    Breast Cancer (BC) treatment leads to mutilation and destruction of breast shape with negative effects on body image and self-esteem.One of the main goals of reconstructive and oncoplastic breast surgery is to satisfy patients and improve their quality of life (QoL).Therefore, it is important to assess the patient experience post-surgery by means of patient-reported outcome measures (PROMs) that focus on the patient's perception of the surgery and surgical care, as well as psychosocial well-being and physical functioning. The objective of the current study was to identify predictors of patient satisfaction such as breast appearance including implant type in a selective sample of women who underwent breast reconstruction surgery using implants. Participants in this prospective study were women, (age 26-75 years) that were newly diagnosed with breast carcinoma. All consecutive patients who underwent breast reconstruction between January 2013 and October 2014 were asked to complete the BREAST-Q questionnaire 1 year after surgery. 120 patients underwent unilateral breast reconstruction using implant. While 38 patients underwent reconstruction with opposite breast reduction symmertization, 27 patients underwent therapeutic mammoplasty. The response rate for BREAST-Q questionnaire completion was 98 % with 147 out of 150 study participants completed the questionnaire. From the data collected from 147 patients, the responses could be distributed into 4 distinct groups based on the reconstruction outcomes namely "very much satisfied" (93 %) or "definitely and mostly satisfied" (94 %) or "satisfied" with the outcome (88 %) or "definitely agree on having reconstruction rather than the alternative of having no breast "(91 %).The results showed significant improvement in all four areas that were evaluated after surgery namely satisfaction with the appearance of the breasts, psychosocial, sexual and physical well-being. While the reconstruction surgery had an overall

  9. Patient-reported outcomes among patients using exenatide twice daily or insulin in clinical practice in six European countries: the CHOICE prospective observational study

    PubMed Central

    2013-01-01

    Background Improvements in the clinical condition of patients with type 2 diabetes are often accompanied by improvements in health-related quality of life and other patient-reported outcomes (PROs), but data assessing injectable treatment initiation from the patient’s perspective in routine clinical practice are lacking. We examined PROs in patients initiating injectable treatment in the CHOICE (CHanges to treatment and Outcomes in patients with type 2 diabetes initiating InjeCtablE therapy) study. Methods CHOICE was a 24-month, prospective observational study conducted in six European countries. Patients initiated exenatide twice daily (BID) or insulin based on a physician’s clinical judgement. Clinical and PRO data were collected at baseline (injectable therapy initiation) and after approximately 3, 6, 12, 18 and 24 months. The two treatment cohorts had different baseline characteristics; therefore, no statistical comparisons of endpoints between main cohorts were conducted. Results There were 2388 patients eligible for analysis (exenatide BID cohort, n = 1114; insulin cohort, n = 1274). Mean positive changes in Impact of Weight on Quality of Life-Lite (IWQOL-Lite) total score and EuroQoL5-Dimension (EQ-5D) index and visual analogue scale (VAS) scores were observed in both cohorts with most changes observed during the first 6 months after injectable therapy initiation. Patients who experienced weight loss (≥1 kg) at 24 months appeared to have higher mean improvements in IWQOL-Lite total score than did patients with weight gain or no weight change. Patients who met the composite clinical endpoint of glycated haemoglobin (HbA1c) <7.0%, no weight gain (≤1 kg) and no hypoglycaemia generally experienced higher mean improvements in EQ-5D index and VAS scores (compared with patients who did not meet this endpoint) and Diabetes Health Profile-18 scores (versus the main cohorts). High levels of missing data were observed for all PRO measures in both

  10. Developing IntegRATE: a fast and frugal patient-reported measure of integration in health care delivery

    PubMed Central

    Elwyn, Glyn; Thompson, Rachel; John, Roshen; Grande, Stuart W

    2015-01-01

    Background Efforts have been made to measure integration in health care delivery, but few existing instruments have adopted a patient perspective, and none is sufficiently generic and brief for administration at scale. We sought to develop a brief and generic patient-reported measure of integration in health care delivery. Methods Drawing on both existing conceptualisations of integrated care and research on patients’ perspectives, we chose to focus on four distinct domains of integration: information sharing, consistent advice, mutual respect and role clarity. We formulated candidate items and conducted cognitive interviews with end users to further develop and refine the items. We then pilot-tested the measure. Results Four rounds of cognitive interviews were conducted (n = 14) and resulted in a four-item measure that was both relevant and understandable to end users. The pilot administration of the measure (n = 15) further confirmed the relevance and interpretability of items and demonstrated that the measure could be completed in less than one minute. Conclusions This new measure, IntegRATE, represents a patient-reported measure of integration in health care delivery that is conducive to use in both routine performance monitoring and research. The psychometric properties of the measure will be assessed in the next stage of development. PMID:26034467

  11. Improving treatment satisfaction and other patient-reported outcomes in people with type 2 diabetes: the role of once-daily insulin glargine.

    PubMed

    Bradley, C; Gilbride, C J B

    2008-07-01

    Insulin therapy becomes essential for many people with type 2 diabetes. After starting insulin, people with diabetes that is poorly controlled with oral agents typically report improved well-being and treatment satisfaction. However, healthcare professionals and people with type 2 diabetes are often reluctant to begin insulin treatment, citing concerns such as time/resources needed to educate patients, increased risks of hypoglycaemia and fear of injections, which lead them to focus on intensifying conventional oral therapy. Insulin glargine, which offers people with diabetes a once-a-day injection regimen with low risk of hypoglycaemia, is more likely to overcome such initial barriers than other more complex insulin regimens. Once-daily insulin glargine, in combination with modern glucose-dependent oral agents that do not need to be chased with food to prevent hypoglycaemia, does not require the fixed mealtimes and set amounts of carbohydrates necessary with twice-daily injection mixes and older sulphonylureas. We know that it is such dietary restrictions that cause the most damage to quality of life (QoL). To avoid damaging QoL unnecessarily and to ensure optimal satisfaction with treatment, it is important to evaluate the effects of treatment on QoL, treatment satisfaction and other patient-reported outcomes (PROs) using questionnaires validated for this purpose, such as the widely used Diabetes Treatment Satisfaction Questionnaire and the Audit of Diabetes-Dependent Quality of Life measure. A systematic electronic literature search identified reports of studies evaluating PROs associated with insulin glargine in comparison with other treatments. The studies show that insulin glargine is usually associated with greater improvements in treatment satisfaction and other PROs compared with intensifying oral therapy or alternative insulin regimens. PMID:18577157

  12. Long-term results of oncoplastic breast surgery with latissimus dorsi flap reconstruction: a pilot study of the objective cosmetic results and patient reported outcome

    PubMed Central

    Kim, Kyeong-Deok; Kim, Zisun; Kuk, Jung Cheol; Jeong, Jaehong; Choi, Kyu Sung; Hur, Sung Mo; Jeong, Gui Ae; Chung, Jun Chul; Cho, Gyu Seok; Shin, Eung Jin; Kim, Hyung Chul; Kang, Sang-Gue; Lee, Min Hyuk

    2016-01-01

    Purpose The goal of oncoplastic breast surgery is to restore the appearance of the breast and improve patient satisfaction. Thus, the assessment of cosmetic results and patient-reported outcomes (PROs) using appropriately constructed and validated instruments is essential. The aim of the present study was to assess the long-term objective cosmetic results and corresponding PROs after oncoplastic breast surgery. Methods Cosmetic results were assessed by the patients, a medical panel, and a computer program (BCCT.core). PROs were assessed using BREAST-Q, a questionnaire that measures the perception of patients having breast surgery. The cosmetic results and PROs were analyzed in patients who underwent quadrantectomy and partial breast reconstruction utilizing the latissimus dorsi flap. Results The mean duration of the follow-up period was 91.6 months (range, 33.3–171.0 months), and mean age of the patients was 51 years old (range, 33–72 years). The mean tumor size was 2.1 cm (range, 0.9–5.5 cm). There was fair agreement between the medical panel and BCCT.core score (K = 0.32, P < 0.001), and a statistically significant correlation between the BCCT.core score and medical panel cosmetic results was identified (r = 0.606, P < 0.001). A better BCCT.core result was related to a higher PRO of each BREAST-Q domain—satisfaction with breasts (R2 = 0.070, P = 0.039), satisfaction with outcome (R2 = 0.087, P = 0.021), psychosocial well-being (R2 = 0.085, P = 0.023), sexual well-being (R2 = 0.082, P = 0.029), and satisfaction with information (R2 = 0.064, P = 0.049). Conclusion Our long-term results of oncoplastic surgery achieved a high level of patient satisfaction with good cosmetic results. The medical panel and BCCT.core results correlated well with the PROs of the patients using valid, reliable, and procedure-specific measures. PMID:26942154

  13. Understanding the Recovery Phase of Breast Reconstructions: Patient Reported Outcomes Correlated to Type and Timing of Reconstruction

    PubMed Central

    Weichman, Katie E.; Hamill, Jennifer B.; Kim, Hyungjin Myra; Chen, Xiaoxue; Wilkins, Edwin G.; Pusic, Andrea L.

    2016-01-01

    Summary Introduction During preoperative discussions with breast reconstruction patients, questions often arise about what to expect during the recovery period. However, there is a paucity of data elucidating post breast reconstruction pain, fatigue and physical morbidity. This information is important to patient and physician understanding of reconstructive choices and the postoperative recovery process. We sought to evaluate how recovery may vary for patients based on the timing and type of reconstruction. Materials and Methods Patients were recruited as part of the Mastectomy Reconstruction Outcomes Consortium (MROC) Study, a prospective, multi-centered NIH-funded study (1RO1CA152192). Here, patients completed the Numerical Pain Rating Scale (NPRS), McGill Pain Questionnaire, and Breast-Q preoperatively and at one week and three months postoperatively. Pain, fatigue, and upper body morbidity were evaluated by type and timing of reconstruction. Results A total of 2,013 MROC study participants had completed 3-month follow-up and therefore were included for analysis. 1,583 patients (78.6%) completed surveys at one-week post-reconstruction, 1,517 patients (75.3%) at three months post-reconstruction. Across all procedure groups, fatigue and physical well-being scores did not return to preoperative levels by three months. At three months, pain measured by the NPRS differed across procedure types (p=0.01), with tissue expander/implant (TE/I) having more pain than direct to implant (p<0.01). Similarly, at three months, chest and upper body physical morbidity, as measured by BREAST-Q, differed by procedure types (p<0.001), with generally less morbidity for autologous reconstruction as compared to TE/Is. Conclusions For all reconstructive procedure groups, patients have not fully recovered at three months post-surgery. Additionally, postoperative pain and upper body physical morbidity vary significantly by reconstructive procedure with patients undergoing TE/I reporting

  14. Establishing a population-based patient-reported outcomes study (PROMs) using national cancer registries across two jurisdictions: the Prostate Cancer Treatment, your experience (PiCTure) study

    PubMed Central

    Drummond, F J; Kinnear, H; Donnelly, C; O'Leary, E; O'Brien, K; Burns, R M; Gavin, A; Sharp, L

    2015-01-01

    Objective To establish an international patient-reported outcomes (PROMs) study among prostate cancer survivors, up to 18 years postdiagnosis, in two countries with different healthcare systems and ethical frameworks. Design A cross-sectional, postal survey of prostate cancer survivors sampled and recruited via two population-based cancer registries. Healthcare professionals (HCPs) evaluated patients for eligibility to participate. Questionnaires contained validated instruments to assess health-related quality of life and psychological well-being, including QLQ-C30, QLQ-PR25, EQ-5D-5L, 21-question Depression, Anxiety and Stress Scale (DASS-21) and the Decisional Regret Scale. Setting Republic of Ireland (RoI) and Northern Ireland (NI). Primary outcome measures Registration completeness, predictors of eligibility and response, data missingness, unweighted and weighted PROMs. Results Prostate cancer registration was 80% (95% CI 75% to 84%) and 91% (95% CI 89% to 93%) complete 2 years postdiagnosis in NI and RoI, respectively. Of 12 322 survivors sampled from registries, 53% (n=6559) were classified as eligible following HCP screening. In the multivariate analysis, significant predictors of eligibility were: being ≤59 years of age at diagnosis (p<0.001), short-term survivor (<5 years postdiagnosis; p<0.001) and from RoI (p<0.001). 3348 completed the questionnaire, yielding a 54% adjusted response rate. 13% of men or their families called the study freephone with queries for assistance with questionnaire completion or to talk about their experience. Significant predictors of response in multivariate analysis were: being ≤59 years at diagnosis (p<0.001) and from RoI (p=0.016). Mean number of missing questions in validated instruments ranged from 0.12 (SD 0.71; EQ-5D-5L) to 3.72 (SD 6.30; QLQ-PR25). Weighted and unweighted mean EQ-5D-5L, QLQ-C30 and QLQ-PR25 scores were similar, as were the weighted and unweighted prevalences of depression, anxiety and

  15. Current Status of Outcome Measure Development in Vasculitis

    PubMed Central

    Merkel, Peter A.; Aydin, Sibel Z.; Boers, Maarten; Cornell, Christina; Direskeneli, Haner; Gebhart, Don; Hatemi, Gulen; Luqmani, Raashid; Matteson, Eric L.; Milman, Nataliya; Robson, Joanna; Seo, Philip; Tomasson, Gunnar

    2015-01-01

    The conduct of randomized controlled trials for vasculitis, especially for the antineutrophil cytoplasmic antibody-associated vasculitides [AAV, granulomatosis with polyangiitis (Wegener’s) and microscopic polyangiitis], has been greatly advanced by the development, use, and acceptance of validated outcome measures. Trials have subsequently provided the opportunity to validate and refine reliable, valid outcome measures for these multisystemic and relapsing rare diseases. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group was formed in 2004 to foster development of validated and widely accepted outcomes in vasculitis using data-driven analyses, a dedication to building consensus, and adherence to, and guidance by, the principles of the OMERACT approach. This work led to the endorsement by OMERACT of the core set of domains and associated outcome measures for AAV. Next steps for the study of existing outcome tools in AAV include better definition of response criteria through development of more data-driven weighting of the elements of activity and damage assessment. The Working Group is now also embarking on a series of linked projects to develop validated patient-reported outcomes for use in clinical research in vasculitis. Additionally, the Working Group is studying how current methods of disease assessment and plans for new outcomes can be informed by the conceptual framework of the International Classification of Function of the World Health Organization. The success of the Group’s work in AAV has also led to a formal process for developing outcomes for the large vessel vasculitides (Takayasu arteritis and giant cell arteritis) and Behçet disease. PMID:24429177

  16. Measurement outcomes from hip simulators.

    PubMed

    de Villiers, Danielle; Shelton, Julia C

    2016-05-01

    Simulation of wear in total hip replacements has been recognised as an important factor in determining the likelihood of clinical success. However, accurate measurement of wear can be problematic with factors such as number and morphology of wear particles produced as well as ion release proving more important in the biological response to hip replacements than wear volume or wear rate alone. In this study, hard-on-hard (CoCr alloy, AgCrN coating) and hard-on-soft (CoCr alloy and CrN coating on vitamin E blended highly cross-linked polyethylene) bearing combinations were tested in an orbital hip simulator under standard and some adverse conditions. Gravimetric wear rates were determined for all bearings, with cobalt and where applicable, silver release determined throughout testing. Isolation of wear particles from the lubricating fluid was used to determine the influence of different bearing combinations and wear conditions on particle morphology. It was found that cobalt and silver could be measured in the lubricating fluid even when volumetric wear was not detectable. In hard-on-hard bearings, Pearson's correlation of 0.98 was established between metal release into the lubricating fluid and wear volume. In hard-on-soft bearings, coating the head did not influence the polyethylene wear rates measured under standard conditions but did influence the cobalt release; the diameter influenced both polyethylene wear and cobalt release, and the introduction of adverse testing generated smaller polyethylene particles. While hip simulators can be useful to assess the wear performance of a new material or design, measurement of other outcomes may yield greater insight into the clinical behaviour of the bearings in vivo. PMID:26888886

  17. Treatment adherence and other patient-reported outcomes as cost determinants in multiple sclerosis: a review of the literature

    PubMed Central

    Lizán, Luis; Comellas, Marta; Paz, Silvia; Poveda, José Luis; Meletiche, Dennis M; Polanco, Carlos

    2014-01-01

    Background Treatment adherence is one of the key factors for achieving optimal clinical outcomes. In order to assess costs related to adherence to, and persistence and compliance with, disease-modifying therapies (DMTs) in patients with multiple sclerosis (MS), a narrative review of the literature was performed. Satisfaction with and preference for DMTs and their delivery devices were also assessed, as both can have an influence on patients’ adherence and persistence. Methods Electronic databases (MEDLINE, PubMed, Google Scholar, congress proceedings) were searched to identify publications analyzing MS costs related to adherence, persistence, satisfaction, and preferences for MS treatments. Bibliographic references were hand searched. English or Spanish studies published between January 2007 and January 2013 were selected. Results A total of 398 titles were identified, of which 12 met the inclusion criteria. Six studies evaluated the impact of adherence, persistence, and compliance on treatment costs; four publications analyzed satisfaction with DMTs; and two assessed treatment preferences based on attributes of the delivery device. Increased adherence and persistence were associated with better clinical outcomes, leading to lower relapse risk (odds ratio [OR]: 0.71; 95% confidence interval [CI]: 0.59–0.85) and a decrease in health care resource use, such as MS-related hospitalizations (OR: 0.63; 95% CI: 0.47–0.83) and emergency department visits (OR: 0.80; 95% CI: 0.60–1.07). This reduction in resource use led to a patient/year total cost reduction (excluding DMT costs) of up to 22%. Conclusion This review highlights the importance of ensuring adequate adherence in MS patients through treatments and devices better tailored to patients’ needs that could enhance clinical outcomes and reduce MS costs. Understanding the factors underlying satisfaction and compliance with treatment and patients’ preference for certain therapies could help in the development

  18. Survivorship care planning and its influence on long-term patient-reported outcomes among colorectal and lung cancer survivors: The CanCORS disease-free survivor follow-up study

    PubMed Central

    Chrischilles, Elizabeth A.; McDowell, Bradley D.; Rubenstein, Linda; Charlton, Mary; Pendergast, Jane; Juarez, Grelda Yazmin; Arora, Neeraj K.

    2014-01-01

    PURPOSE Evaluate the relationship between survivorship care planning (SCP) and survivorship care and health outcomes reported by long-term lung and colorectal cancer survivors. METHODS Participants (n=832) were diagnosed and enrolled during 2003-2005. In 2012, patient-reported outcomes (survivorship care and health outcomes) and two patient-reported SCP measures (receipt of written summary of cancer treatment and receipt of instructions on who to see for routine cancer follow-up) were collected. Analyses controlled for SCP predictors collected from medical records and an interview 1 year after diagnosis. RESULTS One-in-four survivors reported receiving both SCP elements. Those receiving both were more certain which doctor was in charge (OR 7.0; 95% CI 3.9-12.5), more likely to report follow-up check-ups (OR 5.1; 95% CI 3.3-8.0) and had an MRI/PET/CT scan in the past 2 years (OR 2.8; 95% CI 1.7-4.7) compared to those receiving neither. Physician communication experiences were significantly more positive and having physical exams (OR 2.0; 95% CI 1.2-3.4) and meeting exercise guidelines (OR 1.6; 95% CI 1.004-2.4) more likely. Physical health (p=0.012) and good-to-excellent self-perceived health status (OR 2.2; 95% CI 1.3-3.9) were better for those receiving both elements. CONCLUSION SCP may lead to better cancer follow-up care, long-term physical health, and physician/patient communication experiences. IMPLICATIONS FOR CANCER SURVIVORS The positive association between outcomes and SCP suggest that efforts to implement SCP should be fruitful. PMID:25354481

  19. Incorporating the patient’s voice into electronic health records through patient-reported outcomes as the “review of systems”

    PubMed Central

    Basch, Ethan M

    2015-01-01

    Owing to lack of standardization for eliciting patient symptoms, the limited time available during clinical encounters, and the often-competing priorities of patients and providers, providers may not appreciate the full spectrum of the patient’s symptom experience. Using electronically collected patient-reported outcomes to capture the review of system outside of the clinic visit may not only improve the efficiency, completeness, and accuracy of data collection for the review of system, but also provide the opportunity to operationalize incorporating the patient’s voice into the electronic health record. While the necessary technology is already available, multiple stakeholders, including electronic health record vendors, clinicians, researchers, and professional societies, need to align their interests before this can become a widespread reality. PMID:25614143

  20. Development and preliminary evaluation of a training workshop for the collection of patient-reported outcome (PRO) interview data by research support staff.

    PubMed

    Atkinson, Thomas M; Hurley, Karen; Bylund, Carma L; Berk, Alexandra; Diminni, Kimberly; Ostroff, Jamie S

    2013-03-01

    This paper describes the development and initial evaluation of a didactic curriculum to prepare research support staff with the core knowledge and skills required to collect patient-reported outcomes (PROs) via interviews. Research support staff members (N = 77) were recruited for eight separate workshops, each consisting of a didactic presentation followed by role-play scenarios with trained actors depicting common scenarios they may encounter as part of patient interaction. Trainees were observed and received feedback on their performance from trained facilitators and peers. In comparison to their pre-training assessment, trainees showed significant improvement in their confidence to conduct a research interview, handle a distressed participant, manage a wandering interview, ask participants sensitive questions, and handle irritated patients. Training research support staff in the effective collection of PROs via patient interviews can improve the confidence of these individuals in interacting with patients, which can ultimately lead to increased accuracy of data collection. PMID:23090591

  1. Impact of the Patient-Reported Outcomes Management Information System (PROMIS) upon the Design and Operation of Multi-center Clinical Trials: a Qualitative Research Study

    PubMed Central

    Diener, Lawrence W.; Nahm, Meredith; Weinfurt, Kevin P.

    2013-01-01

    New technologies may be required to integrate the National Institutes of Health’s Patient Reported Outcome Management Information System (PROMIS) into multi-center clinical trials. To better understand this need, we identified likely PROMIS reporting formats, developed a multi-center clinical trial process model, and identified gaps between current capabilities and those necessary for PROMIS. These results were evaluated by key trial constituencies. Issues reported by principal investigators fell into two categories: acceptance by key regulators and the scientific community, and usability for researchers and clinicians. Issues reported by the coordinating center, participating sites, and study subjects were those faced when integrating new technologies into existing clinical trial systems. We then defined elements of a PROMIS Tool Kit required for integrating PROMIS into a multi-center clinical trial environment. The requirements identified in this study serve as a framework for future investigators in the design, development, implementation, and operation of PROMIS Tool Kit technologies. PMID:20703765

  2. Impact of herpes zoster and post-herpetic neuralgia on patients’ quality of life: a patient-reported outcomes survey

    PubMed Central

    Edte, Alexander; Schmitt, Sonja; Lukas, Kati

    2010-01-01

    Background The impact of herpes zoster (HZ) and post-herpetic neuralgia (PHN) on patients’ quality of life (QoL) is currently poorly documented. Subjects and methods Telephone interviews in Germany identified patients ≥50 years old with painful HZ diagnosed during the previous 5 years. Bespoke questions evaluated previous HZ episodes. Results Of 11,009 respondents, 280 met the screening criteria, and 32 (11%) developed PHN. PHN was associated with significantly worse outcomes than HZ (all P < 0.05). Mean pain scores associated with PHN and HZ, respectively, were 7.1 and 6.2 (average) and 8.2 and 7.0 (worst). Many patients with PHN (91%) and HZ (73%) experienced problems with daily activities, including work, studies, housework, family and leisure activities. Mean pain interference scores in patients with PHN versus HZ were highest for sleep (6.5 versus 4.9), normal work (6.1 versus 4.4) and mood (5.9 versus 4.4). Most employed interviewees with PHN (70%) and HZ (64%) stopped work during the disease. Pain and QoL outcomes were not significantly different between all patients versus those diagnosed during the previous 12 months or between patients aged 50–59 years versus ≥60 years. Conclusions HZ causes substantial pain, which seriously interferes with many aspects of daily life, particularly in patients with PHN. PMID:21124645

  3. Harnessing Technology to Improve Clinical Trials: Study of Real-Time Informatics to Collect Data, Toxicities, Image Response Assessments, and Patient-Reported Outcomes in a Phase II Clinical Trial

    PubMed Central

    Pietanza, M. Catherine; Basch, Ethan M.; Lash, Alex; Schwartz, Lawrence H.; Ginsberg, Michelle S.; Zhao, Binsheng; Shouery, Marwan; Shaw, Mary; Rogak, Lauren J.; Wilson, Manda; Gabow, Aaron; Latif, Marcia; Lin, Kai-Hsiung; Wu, Qinfei; Kass, Samantha L.; Miller, Claire P.; Tyson, Leslie; Sumner, Dyana K.; Berkowitz-Hergianto, Alison; Sima, Camelia S.; Kris, Mark G.

    2013-01-01

    Purpose In clinical trials, traditional monitoring methods, paper documentation, and outdated collection systems lead to inaccuracies of study information and inefficiencies in the process. Integrated electronic systems offer an opportunity to collect data in real time. Patients and Methods We created a computer software system to collect 13 patient-reported symptomatic adverse events and patient-reported Karnofsky performance status, semi-automated RECIST measurements, and laboratory data, and we made this information available to investigators in real time at the point of care during a phase II lung cancer trial. We assessed data completeness within 48 hours of each visit. Clinician satisfaction was measured. Results Forty-four patients were enrolled, for 721 total visits. At each visit, patient-reported outcomes (PROs) reflecting toxicity and disease-related symptoms were completed using a dedicated wireless laptop. All PROs were distributed in batch throughout the system within 24 hours of the visit, and abnormal laboratory data were available for review within a median of 6 hours from the time of sample collection. Manual attribution of laboratory toxicities took a median of 1 day from the time they were accessible online. Semi-automated RECIST measurements were available to clinicians online within a median of 2 days from the time of imaging. All clinicians and 88% of data managers felt there was greater accuracy using this system. Conclusion Existing data management systems can be harnessed to enable real-time collection and review of clinical information during trials. This approach facilitates reporting of information closer to the time of events, and improves efficiency, and the ability to make earlier clinical decisions. PMID:23630218

  4. Measuring Outcomes in Children's Services.

    ERIC Educational Resources Information Center

    Christner, Anne Marshall, Ed.

    Outcomes evaluation can provide program managers and clinical directors in child welfare, juvenile justice, child mental health, and child protective services the necessary tools for program quality assurance and accountability. This guide describes the outcomes evaluation process and provides a summary of articles and reports detailing current…

  5. Patient-reported outcomes in type 2 diabetes mellitus: patients’ and primary care physicians’ perspectives in the Spanish health care system

    PubMed Central

    Franch-Nadal, Josep; Labrador Barba, Elena; Gómez-García, M Carmen; Buil-Cosiales, Pilar; Millaruelo, José Manuel; Peña, María Luisa Orera

    2015-01-01

    Objective Understanding patients’ and physicians’ perceptions of type 2 diabetes mellitus (T2DM) management and treatment has important implications for diabetes care, allowing the identification of clinical practice issues that could be improved, leading to patients’ better understanding of the illness and, consequently, healthier self-management behaviors. The objective of this study was to identify differences between physicians’ and T2DM patients’ perceptions related to health status, patient-reported outcomes assessments, and T2DM management and treatment, in routine clinical practice in Spain. Methods This was an observational, cross-sectional study including 1,012 T2DM patients and 974 physicians from 47 and 52 Spanish provinces, respectively. An electronic structured self-administered questionnaire containing 17 questions was designed aiming to address both physicians’ and patient’s perceptions on overall T2DM health status and patient-reported outcomes. Results T2DM patients perceived a worse health status (40% reported having a “good” and 38% a “neither good nor bad” health status) compared with physicians’ perceptions (77% thought patients had a “good” health status). Most patients answered being “satisfied” or “neither satisfied nor unsatisfied” with the given information, while physicians considered that patients were “satisfied” or “very satisfied” with the information for self-monitoring blood glucose and treatment administration. Fifty-seven percent of patients reported that medical recommendations were “important”, while 58% of physicians considered it as “very important”. Fifty-three percent of patients perceived that their current T2DM treatment suited their preferences “quite a lot”, and this was lower than the proportion of physicians (69%) that believed this for their patients. Additionally, a lower percentage of patients (53%) than physicians (79%) believed that their treatment improved

  6. Patient reported outcomes for patients who returned to sport compared with those who did not after hip arthroscopy: minimum 2-year follow-up.

    PubMed

    Domb, Benjamin G; Dunne, Kevin F; Martin, Timothy J; Gui, Chengcheng; Finch, Nathan A; Vemula, S Pavan; Redmond, John M

    2016-07-01

    Previous studies assessed elite athletes' return to sport (RTS) after hip arthroscopy, but few investigated a cohort including athletes from all levels of sport. This study compared athletes who returned to sport to those who did not, based on four patient-reported outcome (PRO) scores, including the Hip Outcome Score-Sports Specific Subscale (HOS-SSS). Between September 2008 and April 2012, hip arthroscopies were performed on 157 patients (168 hips) who reported playing a sport preoperatively and indicated their level of sports activity post-operatively. Two-year follow-up was available for 148 (94%) amateur and professional athletes with a total of 158 hips. Of these 60 cases (65 hips) did not return to sports (NRTS) and were in the NRTS group. The remaining 88 cases (93 hips) constituted the RTS group. The modified Harris Hip Score, Non-Arthric Hip Score, Hip Outcome-Activities of Daily Living (HOS-ADL), and HOS-SSS were used to assess outcomes. The HOS-SSS was used to assess specific sport-related movement. Both groups demonstrated significant improvement at 2 years post-operatively in visual analog score and four PRO scores (P < 0.001). There was no significant preoperative differences in HOS-SSS scores between groups; however, the RTS group had significantly higher HOS-SSS scores at 1 and 2 years post-surgery. Post-operatively, the RTS group had significantly better ability to jump, land from a jump, stop quickly and perform cutting/lateral movements (P < 0.05). In summary, patients who indicated RTSs demonstrated significantly higher PRO scores and abilities to perform several sport-related movements, compared with patients who did not. PMID:27583148

  7. Patient reported outcomes for patients who returned to sport compared with those who did not after hip arthroscopy: minimum 2-year follow-up

    PubMed Central

    Domb, Benjamin G.; Dunne, Kevin F.; Martin, Timothy J.; Gui, Chengcheng; Finch, Nathan A.; Vemula, S. Pavan; Redmond, John M.

    2016-01-01

    Previous studies assessed elite athletes’ return to sport (RTS) after hip arthroscopy, but few investigated a cohort including athletes from all levels of sport. This study compared athletes who returned to sport to those who did not, based on four patient-reported outcome (PRO) scores, including the Hip Outcome Score—Sports Specific Subscale (HOS-SSS). Between September 2008 and April 2012, hip arthroscopies were performed on 157 patients (168 hips) who reported playing a sport preoperatively and indicated their level of sports activity post-operatively. Two-year follow-up was available for 148 (94%) amateur and professional athletes with a total of 158 hips. Of these 60 cases (65 hips) did not return to sports (NRTS) and were in the NRTS group. The remaining 88 cases (93 hips) constituted the RTS group. The modified Harris Hip Score, Non-Arthric Hip Score, Hip Outcome-Activities of Daily Living (HOS-ADL), and HOS-SSS were used to assess outcomes. The HOS-SSS was used to assess specific sport-related movement. Both groups demonstrated significant improvement at 2 years post-operatively in visual analog score and four PRO scores (P < 0.001). There was no significant preoperative differences in HOS-SSS scores between groups; however, the RTS group had significantly higher HOS-SSS scores at 1 and 2 years post-surgery. Post-operatively, the RTS group had significantly better ability to jump, land from a jump, stop quickly and perform cutting/lateral movements (P < 0.05). In summary, patients who indicated RTSs demonstrated significantly higher PRO scores and abilities to perform several sport-related movements, compared with patients who did not. PMID:27583148

  8. Feasibility of Using a Handheld Electronic Device for the Collection of Patient Reported Outcomes Data from Children

    ERIC Educational Resources Information Center

    Vinney, Lisa A.; Grade, John D.; Connor, Nadine P.

    2012-01-01

    The manner in which a communication disorder affects health-related quality of life (QOL) in children is not known. Unfortunately, collection of quality of life data via traditional paper measures is labor intensive and has several other limitations, which hinder the investigation of pediatric quality of life in children. Currently, there is not…

  9. Patient-Reported Outcomes and Therapeutic Affordances of Social Media: Findings From a Global Online Survey of People With Chronic Pain

    PubMed Central

    Gray, Kathleen; Martin-Sanchez, Fernando; Lopez-Campos, Guillermo

    2015-01-01

    Background Patient-reported outcomes (PROs) from social media use in chronic disease management continue to emerge. While many published articles suggest the potential for social media is positive, there is a lack of robust examination into mediating mechanisms that might help explain social media’s therapeutic value. This study presents findings from a global online survey of people with chronic pain (PWCP) to better understand how they use social media as part of self-management. Objective Our aim is to improve understanding of the various health outcomes reported by PWCP by paying close attention to therapeutic affordances of social media. We wish to examine if demographics of participants underpin health outcomes and whether the concept of therapeutic affordances explains links between social media use and PROs. The goal is for this to help tailor future recommendations for use of social media to meet individuals’ health needs and improve clinical practice of social media use. Methods A total of 231 PWCP took part in a global online survey investigating PROs from social media use. Recruited through various chronic disease entities and social networks, participants provided information on demographics, health/pain status, social media use, therapeutic affordances, and PROs from use. Quantitative analysis was performed on the data using descriptive statistics, cross-tabulation, and cluster analysis. Results The total dataset represented 218 completed surveys. The majority of participants were university educated (67.0%, 146/218) and female (83.9%, 183/218). More than half (58.7%, 128/218) were married/partnered and not working for pay (75.9%, 88/116 of these due to ill health). Fibromyalgia (46.6%, 55/118) and arthritis (27.1%, 32/118) were the most commonly reported conditions causing pain. Participants showed a clear affinity for social network site use (90.0%, 189/210), followed by discussion forums and blogs. PROs were consistent, suggesting that social

  10. Patient participation as an integral part of patient-reported outcomes development ensures the representation of the patient voice: a case study from the field of rheumatology

    PubMed Central

    de Wit, M P T; Kvien, T K; Gossec, L

    2015-01-01

    Introduction Patient-reported outcomes (PROs) are important instruments to evaluate healthcare interventions, both in clinical practice and clinical research. Objective To describe how representation of the perspective of people with psoriatic arthritis was obtained through active participation on different levels in the development of PROs. Methods This case study focuses on the methods of involving patients in the elaboration and validation of the Psoriatic Arthritis Impact of Disease (PsAID) score. We used the concept of the participation ladder and the European League Against Rheumatism (EULAR) recommendations for the involvement of patient representatives in scientific projects to analyse the variety of ways patients participated in this process. Results Two patient experts were part of the steering group. 12 patient research partners, coming from 12 different European countries, participated in identifying domains, formulating items for the questionnaire and determining the number of items, the recall period and the questionnaire format. They also helped with the translation of the items into different European languages. Then, 139 patients took part in ranking and prioritising the domains for importance; 65 patients were involved in cognitive debriefing interviews; 499 new patients were recruited for the validation study. Challenges of patient participation in PRO development, such as the representation of patients, are discussed. Conclusions Making patient participation an integral part of the PRO development and validation process is an important requisite for outcome research. The variety of patient contributions at different phases in this case study resulted in an instrument with high face validity. PMID:26509075

  11. Examining the Impact of Patient-Reported Hope for Improvement and Patient Satisfaction with Clinician/Treatment on the Outcome of Major Depressive Disorder Treatment

    PubMed Central

    Vilhauer, Jennice; Kwock, Richard; Wu, Fan; Gohar, Sherif; Collison, Katherine; Thomas, Shannon Nicole; Naghdechi, Lancer; Elashoff, David

    2016-01-01

    Aims This analysis aims at examining if patient-reported variables such as hope for improvement and patient satisfaction with clinician/treatment could influence the outcome major depressive disorder (MDD) treatment, namely depression remission, in the Sequenced Treatment Alternatives to Relieve Depression (STAR*D) trial. Study Design Retrospective cohort study. Place and Duration of Study The STAR*D study was conducted at 18 primary care and 23 psychiatric care settings in the United States from 2001–2007 and was funded by the National Institute of Mental health (NIMH). The analysis contained in this manuscript was conceptualized at the Cedars-Sinai Department of Psychiatry and Behavioral Neurosciences and performed at the UCLA School of Public Health. Methodology Using data from STAR*D, the current study used logistic regression and survival analyses to examine the relationship between depressive symptoms remission and two sets of self-reported factors: Hope for improvement and, Patient satisfaction with treatment/clinician. Results First, more than 90% of STAR*D patients reported having high hope for improvement (agree or strongly agree) and more than 66% endorsed high satisfaction with clinicians and more than 50% expressed high satisfaction with treatments (very or mostly satisfied). Second, hope for improvement was predictive of depression remission (p<0.05). Third, satisfaction with clinician/treatment, did not predict remission. Conclusion This study shows the impact that patients’ subjective hope for improvement can have on predicting depression remission in contrast to satisfaction with clinician/treatment. Future studies should prospectively incorporate patients’ subjective attitudes regarding hope for improvement and satisfaction with clinicians and treatments as mediators and moderators of MDD treatment success. PMID:27294164

  12. Development of a patient reported outcome scale for fatigue in multiple sclerosis: The Neurological Fatigue Index (NFI-MS)

    PubMed Central

    2010-01-01

    Background Fatigue is a common and debilitating symptom in multiple sclerosis (MS). Best-practice guidelines suggest that health services should repeatedly assess fatigue in persons with MS. Several fatigue scales are available but concern has been expressed about their validity. The objective of this study was to examine the reliability and validity of a new scale for MS fatigue, the Neurological Fatigue Index (NFI-MS). Methods Qualitative analysis of 40 MS patient interviews had previously contributed to a coherent definition of fatigue, and a potential 52 item set representing the salient themes. A draft questionnaire was mailed out to 1223 people with MS, and the resulting data subjected to both factor and Rasch analysis. Results Data from 635 (51.9% response) respondents were split randomly into an 'evaluation' and 'validation' sample. Exploratory factor analysis identified four potential subscales: 'physical', 'cognitive', 'relief by diurnal sleep or rest' and 'abnormal nocturnal sleep and sleepiness'. Rasch analysis led to further item reduction and the generation of a Summary scale comprising items from the Physical and Cognitive subscales. The scales were shown to fit Rasch model expectations, across both the evaluation and validation samples. Conclusion A simple 10-item Summary scale, together with scales measuring the physical and cognitive components of fatigue, were validated for MS fatigue. PMID:20152031

  13. Patient-Reported Quality of Life and Satisfaction with Cosmetic Outcomes After Breast Conservation and Mastectomy with and without Reconstruction: Results of a Survey of Breast Cancer Survivors

    PubMed Central

    Jagsi, Reshma; Li, Yun; Morrow, Monica; Janz, Nancy; Alderman, Amy; Graff, John; Hamilton, Ann; Katz, Steven; Hawley, Sarah

    2014-01-01

    Purpose Although breast conservation is therapeutically equivalent to mastectomy for most early-stage breast cancer patients, an increasing number are pursuing mastectomy, which may be followed by breast reconstruction. We sought to evaluate long-term quality of life (QOL) and cosmetic outcomes after different locoregional management approaches, as perceived by patients themselves. Methods We surveyed women diagnosed with non-metastatic breast cancer from 2005-07, as reported to the Los Angeles and Detroit population-based SEER registries. We received responses from 2290 women approximately 9 months after diagnosis (73% response rate) and from 1536 of these 4 years later. We evaluated QOL and patterns and correlates of satisfaction with cosmetic outcomes overall, and more specifically within the subgroup undergoing mastectomy with reconstruction, using multivariable linear regression. Results Of the 1450 patients who responded to both surveys and had not recurred, 963 underwent breast conserving surgery, 263 mastectomy without reconstruction, and 222 mastectomy with reconstruction. Cosmetic satisfaction was similar between those receiving breast conservation and those receiving mastectomy with reconstruction. Among patients receiving mastectomy with reconstruction, reconstruction type and radiation receipt were associated with satisfaction (p<0.001), with an adjusted scaled satisfaction score of 4.7 for patients receiving autologous reconstruction without radiation, 4.4 for patients receiving autologous reconstruction and radiation therapy, 4.1 for patients receiving implant reconstruction without radiation, and 2.8 for patients receiving implant reconstruction and radiation. Discussion Patient-reported cosmetic satisfaction was similar after breast conservation and after mastectomy with reconstruction. In patients undergoing post-mastectomy radiation, use of autologous reconstruction may mitigate radiation's deleterious impact on cosmetic outcomes. PMID:25654742

  14. Patient-reported outcomes at hospital discharge from Heart Centres, a national cross-sectional survey with a register-based follow-up: the DenHeart study protocol

    PubMed Central

    Berg, Selina Kikkenborg; Svanholm, Jette; Lauberg, Astrid; Borregaard, Britt; Herning, Margrethe; Mygind, Anna; Christensen, Anne Vinggaard; Christensen, Anne Illemann; Ekholm, Ola; Juel, Knud; Thrysøe, Lars

    2014-01-01

    Introduction Patient reported health status, which includes symptom burden, functional status and quality of life, is an important measure of health. Differences in health status between diagnostic groups within cardiology have only been sparsely investigated. These outcomes may predict morbidity, mortality, labour market affiliation and healthcare utilisation in various diagnostic groups. A national survey aiming to include all cardiac diagnostic groups from a total Heart Centre population has been designed as the DenHeart survey. Methods and analysis DenHeart is designed as a cross-sectional survey with a register-based follow-up. All diagnostic groups at the five national Heart Centres are included during 1 year (15 April 2013 to 15 April 2014) and asked to fill out a questionnaire at hospital discharge. The total eligible population, both responders and non-responders, will be followed in national registers. The following instruments are used: SF-12, Hospital Anxiety and Depression Scale, EQ-5D, Brief Illness Perception Questionnaire (B-IPQ), HeartQoL and Edmonton Symptom Assessment Scale. The following variables are collected from national registers: action diagnosis, procedures, comorbidity, length of hospital stay, type of hospitalisation, visits to general practitioners and other agents in primary healthcare, dispensed prescription medication, vital status and cause of death. Labour market affiliation, sick leave, early retirement pension, educational degree and income will be collected from registers. Frequency distributions and multiple logistic regression analyses will be used to describe and assess differences in patient reported outcomes at hospital discharge between diagnostic groups and in-hospital predicting factors. Cox proportional hazards regression models with age as the time scale will be used to investigate associations between patient reported outcomes at baseline and morbidity/mortality, labour market affiliation and healthcare utilisation

  15. US Valuation of Health Outcomes Measured Using the PROMIS-29

    PubMed Central

    Craig, Benjamin M.; Reeve, Bryce B.; Brown, Paul M.; Cella, David; Hays, Ron D.; Lipscomb, Joseph; Pickard, A. Simon; Revicki, Dennis A.

    2015-01-01

    Objectives Health valuation studies enhance economic evaluations of treatments by estimating the value of health-related quality of life (HRQoL). The Patient-Reported Outcomes Measurement Information System® (PROMIS) includes a 29-item short-form HRQOL measure, the PROMIS-29. Methods To value PROMIS-29 responses on a quality-adjusted life year (QALY) scale, we conducted a national survey (N=7557) using quota sampling based on the US 2010 Census. Based on 541 paired comparisons with over 350 responses each, pair-specific probabilities were incorporated into a weighted least-squared estimator. Results All losses in HRQoL influenced choice; however, respondents valued losses in physical function, anxiety, depression, sleep, and pain more than those in fatigue and social functioning. Conclusions This paper introduces a novel approach to valuing HRQoL for economic evaluations using paired comparisons and provides a tool to translate PROMIS-29 responses into QALYs. PMID:25498780

  16. The Challenge and Opportunity of Capturing Patient Reported Measures of Rheumatoid Arthritis Disease Activity in Vulnerable Populations with Limited Health Literacy and Limited English Proficiency.

    PubMed

    Hirsh, Joel M

    2016-05-01

    Limited health literacy and limited English proficiency are widely prevalent and contribute to rheumatoid arthritis (RA) health care disparities. The RA Patient Global Assessment of Disease Activity often introduces complexity to the health care encounters of patients and research subjects with limited health literacy and limited English proficiency. Important work is being done to ensure that patient-reported outcomes are validated and appropriate for diverse and vulnerable populations. PMID:27133494

  17. Measuring Inclusive Education Outcomes in Alberta, Canada

    ERIC Educational Resources Information Center

    Loreman, Tim

    2014-01-01

    This study details the results of a review of the academic and public sector literature on measuring inclusive education in large systems. It highlights some outcomes drawn from the international literature on inclusion that might be indicative of the presence and quality of inclusive education in an effort to develop a set of outcomes for…

  18. Recommended patient-reported core set of symptoms and quality-of-life domains to measure in ovarian cancer treatment trials.

    PubMed

    Donovan, Kristine A; Donovan, Heidi S; Cella, David; Gaines, Martha E; Penson, Richard T; Plaxe, Steven C; von Gruenigen, Vivian E; Bruner, Deborah Watkins; Reeve, Bryce B; Wenzel, Lari

    2014-07-01

    There is no consensus as to what symptoms or quality-of-life (QOL) domains should be measured as patient-reported outcomes (PROs) in ovarian cancer clinical trials. A panel of experts convened by the National Cancer Institute reviewed studies published between January 2000 and August 2011. The results were included in and combined with an expert consensus-building process to identify the most salient PROs for ovarian cancer clinical trials. We identified a set of PROs specific to ovarian cancer: abdominal pain, bloating, cramping, fear of recurrence/disease progression, indigestion, sexual dysfunction, vomiting, weight gain, and weight loss. Additional PROs identified in parallel with a group charged with identifying the most important PROs across cancer types were anorexia, cognitive problems, constipation, diarrhea, dyspnea, fatigue, nausea, neuropathy, pain, and insomnia. Physical and emotional domains were considered to be the most salient domains of QOL. Findings of the review and consensus process provide good support for use of these ovarian cancer-specific PROs in ovarian cancer clinical trials. PMID:25006190

  19. PATIENT-REPORTED OUTCOMES OF QUALITY OF LIFE, FUNCTIONING, AND DEPRESSIVE SYMPTOM SEVERITY IN MAJOR DEPRESSIVE DISORDER COMORBID WITH PANIC DISORDER BEFORE AND AFTER SSRI TREATMENT IN THE STAR*D TRIAL

    PubMed Central

    IsHak, Waguih William; Mirocha, James; Christensen, Scott; Wu, Fan; Kwock, Richard; Behjat, Joseph; Pi, Sarah; Akopyan, A.; Peselow, Eric D.; Cohen, Robert M.; Elashoff, David

    2014-01-01

    Background Panic disorder (PD) is highly comorbid with major depressive disorder (MDD) with potential impact on patient-reported outcomes of quality of life (QOL), functioning, and depressive symptom severity Methods Using data from the sequenced treatment alternatives to relieve depression (STAR*D) trial, we compared entry and post-SSRI-treatment QOL, functioning, and depressive symptom severity scores in MDD patients with comorbid PD (MDD+PD) to MDD patients without PD (MDDnoPD). We also compared pre- and posttreatment proportions of patients with severe impairments in quality of life and functioning. Results MDD+PD patients experienced significantly lower QOL and functioning and more severe depressive symptoms than MDDnoPD patients at entry. Following treatment with citalopram, both groups showed significant improvements, however, nearly 30–60% of patients still suffered from severe quality of life and functioning impairments. MDD+PD patients exited with lower QOL and functioning than MDDnoPD patients, a difference that became statistically insignificant after adjusting for baseline measures of depressive symptom severity, functioning, and QOL, comorbid anxiety disorders (PTSD, GAD, social, and specific phobias), age, and college education. Conclusions Functional outcomes using QOL and functioning measures should be utilized in treating and researching MDD so that shortfalls in traditional treatment can be identified and additional interventions can be designed to address severe baseline QOL and functioning deficits in MDD comorbid with PD. PMID:23861180

  20. Clinical outcome assessment in malignant glioma trials: measuring signs, symptoms, and functional limitations.

    PubMed

    Blakeley, Jaishri O; Coons, Stephen Joel; Corboy, John R; Leidy, Nancy Kline; Mendoza, Tito R; Wefel, Jeffrey S

    2016-03-01

    The shared goal of all parties developing therapeutics against malignant gliomas is to positively impact the lives of people affected by these cancers. Clinical outcome assessment (COA) tools, including measures of patient-reported outcome, performance outcome, clinician-reported outcome, and observer-reported outcome, allow patient-focused assessments to complement traditional efficacy measures such as overall survival and radiographic endpoints. This review examines the properties of various COA measures used in malignant glioma clinical trials to date and cross references their content to the priority signs, symptoms, and functional limitations defined through a community survey conducted by the National Brain Tumor Society. The overarching goal of this initiative is to identify COA measures that are feasible and have appropriate psychometric properties for use in this patient population as well as highlight where further development is needed. PMID:26989128

  1. Physician outcome measurement: review and proposed model.

    PubMed

    Siha, S

    1998-01-01

    As health care moves from a free-for-service environment to a capitated arena, outcome measurements must change. ABC Children's Medical Center is challenged with developing comprehensive outcome measures for an employed physician group. An extensive literature review validates that physician outcomes must move beyond revenue production and measure all aspects of care delivery. The proposed measurement model for this physician group is a trilogy model. It includes measures of cost, quality, and service. While these measures can be examined separately, it is imperative to understand their integration in determining an organization's competitive advantage. The recommended measurements for the physician group must be consistent with the overall organizational goals. The long-term impact will be better utilization of resources. This will result in the most cost effective, quality care for the health care consumer. PMID:10339092

  2. Challenges in Measuring Outcomes Following Digital Replantation

    PubMed Central

    Sebastin, Sandeep J.; Chung, Kevin C.

    2013-01-01

    In the early period of replantation surgery, the emphasis was on digit survival. Subsequently, with better microsurgical techniques and instrumentation, the focus has shifted to function and in recent years to consideration of cost-effectiveness. Despite over 40 years of effort in refining digital replantation surgery, a rigorous evaluation of the outcomes of digital replantation has not been performed. This is because of the many confounding variables that influence outcome comparisons. These variables include the mechanism of injury (guillotine, crush, avulsion), the injury itself (total, near total, subtotal, partial amputation), and the surgical procedure (replantation, revascularization). In addition, the traditional outcome measures (two-point discrimination, range of motion, grip strength, or the ability to return to work) are reported inconsistently and vary widely among publications. All these factors make meaningful comparison of outcomes difficult. The recent emphasis on outcome research and cost-effectiveness necessitates a rethinking in the way we report outcomes of digital replantation. In this article, the authors summarize the challenges in assessing outcomes of digital replantation and explain the need to measure outcomes using rigorous clinical research designs that incorporate cost-effectiveness studies in the research protocol. PMID:24872766

  3. A Multicenter, Single-Blind Randomized, Controlled Study of a Volumizing Hyaluronic Acid Filler for Midface Volume Deficit: Patient-Reported Outcomes at 2 Years

    PubMed Central

    Few, Julius; Cox, Sue Ellen; Paradkar-Mitragotri, Deepali; Murphy, Diane K.

    2015-01-01

    Background Juvéderm Voluma XC is a volumizing hyaluronic acid filler used for correction of age-related midface volume deficit (MVD). Objectives The effectiveness of Juvéderm Voluma XC was examined from the patient perspective. Methods Patients with moderate to severe age-related MVD (N = 235) received Juvéderm Voluma XC. At quarterly follow-up visits for 2 years, patients rated treatment outcomes on the Global Aesthetic Improvement Scale (GAIS), overall satisfaction with facial appearance, satisfaction with midfacial regions, achievement of treatment goal, Look and Feel of the Midface (LAFM), and Self-Perception of Age (SPA). Patients recorded treatment-site responses in 30-day diaries. Results At 6 months and 2 years after treatment, 92.8% and 79.0% of patients, respectively, rated their cheek volume as improved/much improved on the GAIS. Improvement in satisfaction with facial appearance was noted by 89.8% of patients at 6 months and 75.8% at 2 years. Increased satisfaction with outer and lower cheek areas and cheek-bone projection and clinically significant improvements in LAFM were noted through month 24. Treatment goals were achieved by 67.8% of patients at 6 months and 49.0% at 2 years. Patients reported looking, on average, 5 years younger at 6 months and 3 years younger at 2 years. The most common treatment site responses were tenderness, swelling, firmness, and lumps/bumps; most were mild to moderate in severity and lasted ≤2 weeks. Conclusions Juvéderm Voluma XC for age-related MVD is effective and well-tolerated from the patient perspective, with results lasting up to 2 years. Level of Evidence 4 Therapeutic PMID:25964628

  4. Patient-Reported Outcome Coordinator Did Not Improve Quality of Life Assessment Response Rates: A Report from the Children's Oncology Group

    PubMed Central

    Johnston, Donna; Gerbing, Robert; Alonzo, Todd; Aplenc, Richard; Nagarajan, Rajaram; Schulte, Fiona; Cullen, Patricia; Sung, Lillian

    2015-01-01

    Purpose Health related quality of life (HRQL) assessments during therapy for pediatric cancer provide valuable information to better understand the patient experience. Our objective was to determine the impact of a patient-reported outcome (PRO) coordinator on HRQL questionnaire completion rates during a pediatric acute myeloid leukemia (AML) trial. Methods AAML1031 is a multicenter Children’s Oncology Group therapeutic trial for de novo AML with a secondary aim to assess HRQL of children and adolescents treated with chemotherapy and hematopoietic stem cell transplantation (HSCT). Parents/guardians are the primary respondents and four questionnaires are administered at eight time points. The questionnaires are the PedsQL 4.0 Generic Core Scales, PedsQL 3.0 Acute Cancer Module, PedsQL Multidimensional Fatigue Scale, and the Pediatric Inventory for Parents. To improve response rates, a central PRO coordinator was instituted and reminded sites about upcoming and delinquent questionnaires. The proportion of HRQL questionnaires completed were compared prior to, and following institution of the PRO coordinator. This analysis evaluated the first five assessment time points. Results There were231 families who consented to participate in the HRQL aim. Overall response rates for all questionnaires were 73–83%. At time point 1, within 14 days of chemotherapy initiation, post-PRO coordinator completion rates were significantly higher for three of four questionnaires. However, the effect was not sustained and at time point 4, one month following last chemotherapy or HSCT, completion rates were significantly lower post-PRO coordinator for all four questionnaires. Conclusion Addition of a central PRO coordinator did not result in sustained improvement in HRQL questionnaire completion rates. Efforts to improve response rates must consider other strategies. PMID:25915772

  5. Design, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic review

    PubMed Central

    Mercieca-Bebber, Rebecca; Palmer, Michael J; Brundage, Michael; Stockler, Martin R; King, Madeleine T

    2016-01-01

    Objectives Patient-reported outcomes (PROs) provide important information about the impact of treatment from the patients' perspective. However, missing PRO data may compromise the interpretability and value of the findings. We aimed to report: (1) a non-technical summary of problems caused by missing PRO data; and (2) a systematic review by collating strategies to: (A) minimise rates of missing PRO data, and (B) facilitate transparent interpretation and reporting of missing PRO data in clinical research. Our systematic review does not address statistical handling of missing PRO data. Data sources MEDLINE and Cumulative Index to Nursing and Allied Health Literature (CINAHL) databases (inception to 31 March 2015), and citing articles and reference lists from relevant sources. Eligibility criteria English articles providing recommendations for reducing missing PRO data rates, or strategies to facilitate transparent interpretation and reporting of missing PRO data were included. Methods 2 reviewers independently screened articles against eligibility criteria. Discrepancies were resolved with the research team. Recommendations were extracted and coded according to framework synthesis. Results 117 sources (55% discussion papers, 26% original research) met the eligibility criteria. Design and methodological strategies for reducing rates of missing PRO data included: incorporating PRO-specific information into the protocol; carefully designing PRO assessment schedules and defining termination rules; minimising patient burden; appointing a PRO coordinator; PRO-specific training for staff; ensuring PRO studies are adequately resourced; and continuous quality assurance. Strategies for transparent interpretation and reporting of missing PRO data include utilising auxiliary data to inform analysis; transparently reporting baseline PRO scores, rates and reasons for missing data; and methods for handling missing PRO data. Conclusions The instance of missing PRO data and its

  6. ‘Trial Exegesis’: Methods for Synthesizing Clinical and Patient Reported Outcome (PRO) Data in Trials to Inform Clinical Practice. A Systematic Review

    PubMed Central

    Macefield, Rhiannon C.; Blencowe, Natalie S.; Brookes, Sara T.; Blazeby, Jane M.

    2016-01-01

    Purpose The CONSORT extension for patient reported outcomes (PROs) aims to improve reporting, but guidance on the optimal integration with clinical data is lacking. This study examines in detail the reporting of PROs and clinical data from randomized controlled trials (RCTs) in gastro-intestinal cancer to inform design and reporting of combined PRO and clinical data from trials to improve the ‘take home’ message for clinicians to use in practice. Materials and Methods The case study was undertaken in gastro-intestinal cancer trials. Well-conducted RCTs reporting PROs with validated instruments were identified and categorized into those combining PRO and clinical data in a single paper, or those separating data into linked primary and supplemental papers. Qualitative methods were developed to examine reporting of the critical interpretation of the trial results (trial exegesis) in the papers in relation of the PRO and clinical outcomes and applied to each publication category. Results were used to inform recommendations for practice. Results From 1917 screened abstracts, 49 high quality RCTs were identified reported in 36 combined and 15 linked primary and supplemental papers. In-depth analysis of manuscript text identified three categories for understanding trial exegesis: where authors reported a “detailed”, “general”, or absent PRO rationale and integrated interpretation of clinical and PRO results. A total of 11 (30%) and 6 (16%) combined papers reported “detailed” PRO rationale and integrated interpretation of results although only 2 (14%) and 1 (7%) primary papers achieved the same standard respectively. Supplemental papers provide better information with 11 (73%) and 3 (20%) achieving “detailed” rationale and integrated interpretation of results. Supplemental papers, however, were published a median of 20 months after the primary RCT data in lower impact factor journals (median 16.8 versus 5.2). Conclusion It is recommended that single

  7. Self-Reported Health Experiences of Children Living with Congenital Heart Defects: Including Patient-Reported Outcomes in a National Cohort Study

    PubMed Central

    Tadic, Valerija; Hogan, Ailbhe; Bull, Catherine; Rahi, Jugnoo Sangeeta; Dezateux, Carol

    2016-01-01

    optimising participation. We highlight the potential for informing and evaluating clinical practice and service provision through seeking patient-reported outcomes in paediatric care. PMID:27487183

  8. Patient Reported Outcomes in a Practice Changing Randomized Trial of Bevacizumab in the Treatment of Advanced Cervical Cancer: An NRG Oncology/Gynecologic Oncology Group Study

    PubMed Central

    Penson, Richard T.; Huang, Helen Q.; Wenzel, Lari B.; Monk, Bradley J.; Stockman, Sharon; Long, Harry J.; Ramondetta, Lois M.; Landrum, Lisa M.; Oaknin, Ana; Reid, Thomas J.A.; Leitao, Mario M.; Method, Michael; Michael, Helen; Tewari, Krishnansu S.

    2015-01-01

    Background To analyze patient reported outcomes (PROs) in GOG 240, the practice-changing, randomized phase 3 trial that concluded that chemotherapy (cisplatin-paclitaxel or topotecan-paclitaxel) plus bevacizumab significantly improves overall survival (OS), progression-free survival (PFS), and response rates compared to chemotherapy alone in advanced cervical cancer. Trial registration number: NCT00803062. Methods Patients were assessed pre-cycle 1, 2, and 5 and at 6 and 9 months post-cycle 1 with the Functional Assessment of Cancer Therapy-Cervix Trial Outcome Index (FACT-Cx TOI), and items from the FACT/GOG-Neurotoxicity (Ntx) subscale, and a worst pain item from the Brief Pain Inventory (BPI). Differences in FACT-Cx TOI scores were assessed using a linear mixed model adjusting for baseline score and age. A mixed effects mixed distributions model was fitted to evaluate treatment differences of likelihood to report neurotoxicity and pain, and severity of these symptoms, once reported. The association between baseline health-related quality of life and survival was analyzed using Cox proportional hazards models. Findings Among 390 evaluable patients, PRO completion rates declined from 96% (baseline) to 63% (9 months post-cycle 1). Completion rates were not statistically different among treatment regimens (p=0.67). Patients receiving chemotherapy plus bevacizumab reported 1.2 points lower on average (98.75% CI: −4.1, 1.7; p=0.30) in the FACT-Cx TOI scores than those with chemotherapy alone. Patients treated with chemotherapy plus bevacizumab were less likely to report neurotoxicty (overall odds ratio: 0.58; 98.75% CI: 0.17, 0.98; p=0.01). Severity of neurotoxic symptoms did not differ between the two groups (p=0.69). Both groups had similar odds of complaining of pain (odds ratio=0.96; 95% CI: 0.39, 1.52; p=0.78) and reported similar severity of pain (p=0.1). For the entire population, the baseline FACT-Cx TOI score was significantly associated with OS (HR 0

  9. Development of core outcome sets in hidradenitis suppurativa: systematic review of outcome measure instruments to inform the process.

    PubMed

    Ingram, J R; Hadjieconomou, S; Piguet, V

    2016-08-01

    The recent hidradenitis suppurativa (HS) Cochrane review identified outcome measure heterogeneity as an important issue to address when designing future HS trials. Our objective was to follow the Harmonising Outcome Measures for Eczema (HOME) roadmap, by performing a systematic review of HS outcome measure instruments to inform the development of an HS core outcome set. We performed a systematic review to identify validation evidence for outcome measure instruments used in HS randomized controlled trials (RCTs), and assessed the methodological quality of all HS outcome measure validity studies using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist. The 12 RCTs included in the Cochrane review utilized 30 outcome measure instruments, including 16 physician-reported instruments, 11 patient-reported instruments and three composite measures containing elements of both. Twenty-seven (90%) of the instruments lacked any validation data. Two further instruments have been developed and partially validated. Of the seven studies meeting our inclusion criteria, six were of 'fair' or 'poor' methodological quality, in part because most of the studies were not primarily designed for instrument validation. The HiSCR instrument is supported by good-quality validation data, but there are gaps, including assessment of internal consistency, inter-rater reliability and minimal clinically important difference, and convergent validity fell below the acceptable range for some comparisons. Multiple, usually unvalidated, outcome measure instruments have been used in HS RCTs. Where validation evidence is available there are issues of low methodological quality or incomplete validity assessment and so, currently, no instruments can be fully recommended. PMID:26873867

  10. Challenges in measuring outcomes for caregivers of people with mental health problems

    PubMed Central

    Zendjidjian, Xavier Y.; Boyer, Laurent

    2014-01-01

    Patient-reported outcomes (PROs) are increasingly important in health care and mental health research. Furthermore, caregivers become partners in care for patients with mental disorders, and health workers are more attentive to the expectations and needs of caregivers. A number of outcomes for caregivers are measured and used in daily practice in order to promote actions to improve health care systems and progress in research on the impact of mental disorders on their caregivers. This paper proposes an inventory of the different outcomes and different measurement tools used to assess the impact of disorders, raising a number of methodological and conceptual issues that limit the relevance of measurement tools and complicate their use. Finally, we propose some recommendations promoting the development of relevant outcome measures for caregivers and their integration into current systems of care. PMID:25152655

  11. Association Between Patient-Reported Measures of Psychological Distress and Patient Satisfaction Scores in a Spine Surgery Patient Population

    PubMed Central

    Abtahi, A.M.; Brodke, D.S.; Lawrence, B.D.; Zhang, C.; Spiker, W.R.

    2015-01-01

    Update This article was updated on June 10, 2015, because of previous errors. The title had previously read “Association Between Patient-Reported Measures of Psychological Distress and Patient Satisfaction Scores After Spine Surgery.” It has been changed to “Association Between Patient-Reported Measures of Psychological Distress and Patient Satisfaction Scores in a Spine Surgery Patient Population” to reflect the fact that not all patients had undergone surgery when they completed their questionnaires. The last sentence in the Background paragraph of the Abstract had previously read “The goal of this study was to determine whether psychological distress influences outpatient satisfaction scores following spine surgery.” It now reads “The goal of this study was to determine whether psychological distress influences outpatient satisfaction scores in a spine surgery patient population.” The last sentence before the Materials and Methods section, which previously read “Our aim in conducting this study was to determine whether psychological distress, as measured with the Distress and Risk Assessment Method (DRAM) questionnaire, influences outpatient satisfaction scores following spine surgery,” now reads: “Our aim in conducting this study was to determine whether psychological distress, as measured with the Distress and Risk Assessment Method (DRAM) questionnaire, influences outpatient satisfaction scores in a spine surgery patient population.” Finally, the second sentence in the Materials and Methods section, “Every patient who completed both a patient satisfaction survey and a DRAM questionnaire for the same encounter at any point during the study period was included in this study,” has been changed to “Every patient who completed both a patient satisfaction survey and a DRAM questionnaire for the same encounter, before or after the surgery, at any point during the study period was included in this study.” An erratum has been published

  12. Effect of complications within 90 days on patient-reported outcomes 3 months and 12 months following elective surgery for lumbar degenerative disease.

    PubMed

    Chotai, Silky; Parker, Scott L; Sivaganesan, Ahilan; Sielatycki, J Alex; Asher, Anthony L; McGirt, Matthew J; Devin, Clinton J

    2015-12-01

    OBJECT There is a paradigm shift toward rewarding providers for quality rather than volume. Complications appear to occur at a fairly consistent frequency in large aggregate data sets. Understanding how complications affect long-term patient-reported outcomes (PROs) following degenerative lumbar surgery is vital. The authors hypothesized that 90-day complications would adversely affect long-term PROs. METHODS Nine hundred six consecutive patients undergoing elective surgery for degenerative lumbar disease over a period of 4 years were enrolled into a prospective longitudinal registry. The following PROs were recorded at baseline and 12-month follow-up: Oswestry Disability Index (ODI) score, numeric rating scales for back and leg pain, quality of life (EQ-5D scores), general physical and mental health (SF-12 Physical Component Summary [PCS] and Mental Component Summary [MCS] scores) and responses to the North American Spine Society (NASS) satisfaction questionnaire. Previously published minimum clinically important difference (MCID) threshold were used to define meaningful improvement. Complications were divided into major (surgicalsite infection, hardware failure, new neurological deficit, pulmonary embolism, hematoma and myocardial infarction) and minor (urinary tract infection, pneumonia, and deep venous thrombosis). RESULTS Complications developed within 90 days of surgery in 13% (118) of the patients (major in 12% [108] and minor in 8% [68]). The mean improvement in ODI scores, EQ-5D scores, SF-12 PCS scores, and satisfaction at 3 months after surgery was significantly less in the patients with complications than in those who did not have major complications (ODI: 13.5 ± 21.2 vs 21.7 ± 19, < 0.0001; EQ-5D: 0.17 ± 0.25 vs 0.23 ± 0.23, p = 0.04; SF-12 PCS: 8.6 ± 13.3 vs 13.0 ± 11.9, 0.001; and satisfaction: 76% vs 90%, p = 0.002). At 12 months after surgery, the patients with major complications had higher ODI scores than those without complications (29.1

  13. Multi-institutional Prospective Evaluation of Bowel Quality of Life After Prostate External Beam Radiation Therapy Identifies Patient and Treatment Factors Associated With Patient-Reported Outcomes: The PROSTQA Experience

    SciTech Connect

    Hamstra, Daniel A.; Conlon, Anna S.C.; Daignault, Stephanie; Dunn, Rodney L.; Sandler, Howard M.; Hembroff, A. Larry; Zietman, Anthony L.; Kaplan, Irving; Ciezki, Jay; Kuban, Deborah A.; Wei, John T.; Sanda, Martin G.; Michalski, Jeff M.

    2013-07-01

    Purpose: To evaluate patients treated with external beam radiation therapy as part of the multicenter Prostate Cancer Outcomes and Satisfaction with Treatment Quality Assessment (PROSTQA), to identify factors associated with posttreatment patient-reported bowel health-related quality of life (HRQOL). Methods and Materials: Pretreatment characteristics and treatment details among 292 men were evaluated using a general linear mixed model for their association with measured HRQOL by the Expanded Prostate Cancer Index Composite instrument through 2 years after enrollment. Results: Bowel HRQOL had a median score of 100 (interquartile range 91.7-100) pretreatment and 95.8 (interquartile range 83.3-100) at 2 years, representing new moderate/big problems in 11% for urgency, 7% for frequency, 4% for bloody stools, and 8% for an overall bowel problems. Baseline bowel score was the strongest predictor for all 2-year endpoints. In multivariable models, a volume of rectum ≥25% treated to 70 Gy (V70) yielded a clinically significant 9.3-point lower bowel score (95% confidence interval [CI] 16.8-1.7, P=.015) and predicted increased risks for moderate to big fecal incontinence (P=.0008). No other radiation therapy treatment-related variables influenced moderate to big changes in rectal HRQOL. However, on multivariate analyses V70 ≥25% was associated with increases in small, moderate, or big problems with the following: incontinence (3.9-fold; 95% CI 1.1-13.4, P=.03), rectal bleeding (3.6-fold; 95% CI 1.3-10.2, P=.018), and bowel urgency (2.9-fold; 95% CI 1.1-7.6, P=.026). Aspirin use correlated with a clinically significant 4.7-point lower bowel summary score (95% CI 9.0-0.4, P=.03) and an increase in small, moderate, or big problems with bloody stools (2.8-fold; 95% CI 1.2-6.4, P=.018). Intensity modulated radiation therapy was associated with higher radiation therapy doses to the prostate and lower doses to the rectum but did not independently correlate with bowel HRQOL

  14. Patient-reported outcomes in virologically suppressed, HIV-1-Infected subjects after switching to a simplified, single-tablet regimen of efavirenz, emtricitabine, and tenofovir DF.

    PubMed

    Hodder, Sally L; Mounzer, Karam; Dejesus, Edwin; Ebrahimi, Ramin; Grimm, Kristy; Esker, Stephen; Ecker, Janet; Farajallah, Awny; Flaherty, John F

    2010-02-01

    A randomized, open-label, multicenter study was conducted to evaluate the therapeutic switch to a single-tablet formulation of efavirenz/emtricitabine/tenofovir DF (EFV/FTC/TDF) among virologically suppressed, HIV-1-infected subjects. Eligible subjects on stable antiretroviral therapy (ART) with HIV-1 RNA less than 200 copies per milliliter for 3 months or more were stratified by prior protease inhibitor (PI)- or non-nucleoside reverse transcriptase inhibitor (NNRTI)-based therapy and randomized (2:1) to EFV/FTC/TDF or to stay on their baseline regimen (SBR). Patient-reported measures were quality of life (QOL; SF-36 [version 2]), treatment adherence (visual analogue scale), preference of medication (POM), perceived ease of the regimen for condition (PERC), and a 20-item HIV symptom index. Overall, 203 subjects were randomized to EFV/FTC/TDF and 97 to SBR. Fifty-three percent of subjects had previously received a PI-based regimen; 47% an NNRTI-based therapy. Throughout the study, SF-36 summary scores did not differ significantly from baseline, regardless of previous ART or treatment allocation. Adherence was 96% or more in both groups at baseline and all subsequent study visits. At study conclusion, the EFV/FTC/TDF regimen was considered easier to follow than prior regimens by 97% and 96% of subjects previously receiving PI-based and NNRTI-based therapies, respectively. Overall, 91% of subjects switched to EFV/FTC/TDF indicated a preference over their prior therapy. Switching to EFV/FTC/TDF was associated with transient worsening/emergence of dizziness and sustained improvements in several other HIV-related symptoms. In conclusion, switching virologically suppressed, HIV-1-infected subjects from PI-based or NNRTI-based regimens to EFV/FTC/TDF was associated with maintained QOL and treatment adherence, and improved ease of use and treatment satisfaction. PMID:20156091

  15. Testing Multiple Outcomes in Repeated Measures Designs

    ERIC Educational Resources Information Center

    Lix, Lisa M.; Sajobi, Tolulope

    2010-01-01

    This study investigates procedures for controlling the familywise error rate (FWR) when testing hypotheses about multiple, correlated outcome variables in repeated measures (RM) designs. A content analysis of RM research articles published in 4 psychology journals revealed that 3 quarters of studies tested hypotheses about 2 or more outcome…

  16. General Outcome Measures for Verbal Operants

    ERIC Educational Resources Information Center

    Kubina, Richard M., Jr.; Wolfe, Pamela; Kostewicz, Douglas E.

    2009-01-01

    A general outcome measure (GOM) can be used to show progress towards a long-term goal. GOMs should sample domains of behavior across ages, be sensitive to change over time, be inexpensive and easy to use, and facilitate decision making. Skinner's (1957) analysis of verbal behavior may benefit from the development of GOM. To develop GOM, we…

  17. Toxicities affecting Quality of Life After Chemo-IMRT of Oropharyngeal Cancer: Prospective Study of Patient-Reported, Observer-Rated, and Objective Outcomes

    PubMed Central

    Hunter, Klaudia U; Schipper, Mathew; Feng, Felix Y; Lyden, Teresa; Haxer, Mark; Murdoch-Kinch, Carol-Anne; Cornwall, Benjamin; Lee, Connie SY; Chepeha, Douglas B; Eisbruch, Avraham

    2012-01-01

    Purpose After conventional radiotherapy for head and neck cancer, xerostomia has traditionally been the major effector of patient-reported quality of life (QOL), and recent publications suggest that dysphagia has an even stronger effect. We hypothesized that IMRT aiming to spare the salivary glands and swallowing structures reduced, or eliminated, the effects of these toxicities on QOL. Methods and Materials Prospective longitudinal study: 72 patients with Stage III-IV oropharyngeal cancer treated uniformly with definitive chemo-IMRT sparing the salivary glands and swallowing structures. Overall QOL was assessed by summary scores of the Head Neck QOL (HNQOL) and University of Washington QOL (UWQOL) questionnaires, as well as HNQOL “Overall Bother” question. QOL, observer-rated toxicities (CTCAE v2), and objective evaluations (videofluoroscopy assessing dysphagia and saliva flow rates assessing xerostomia) were recorded pre-therapy through 2 years post-therapy. Correlations between toxicities/objective evaluations and overall QOL were assessed using longitudinal repeated measures of analysis and Pearson correlations. Results All observer-rated toxicities and QOL scores worsened 1-3 months after therapy and improved through 12 months, with minor further improvements through 24 months. At 12 months, dysphagia grades 0-1, 2, and 3, were observed in 95%, 4%, and 1% of patients, respectively. Using all post-therapy observations, observer-rated dysphagia was highly correlated with all overall QOL measures (p<0.0001), while xerostomia, mucosal, and voice toxicities were significantly correlated with some, but not all, overall QOL measures, with lower correlation coefficients than dysphagia. Late overall QOL (≥6 or ≥12 months post-therapy) was primarily associated with observer-rated dysphagia, and to a lesser extent with xerostomia. Videofluoroscopy scores, but not salivary flows, were significantly correlated with some of the overall QOL measures. Conclusion After

  18. An International Standard Set of Patient-Centered Outcome Measures After Stroke

    PubMed Central

    Salinas, Joel; Sprinkhuizen, Sara M.; Ackerson, Teri; Bernhardt, Julie; Davie, Charlie; George, Mary G.; Gething, Stephanie; Kelly, Adam G.; Lindsay, Patrice; Liu, Liping; Martins, Sheila C.O.; Morgan, Louise; Norrving, Bo; Ribbers, Gerard M.; Silver, Frank L.; Smith, Eric E.; Williams, Linda S.

    2016-01-01

    Background and Purpose— Value-based health care aims to bring together patients and health systems to maximize the ratio of quality over cost. To enable assessment of healthcare value in stroke management, an international standard set of patient-centered stroke outcome measures was defined for use in a variety of healthcare settings. Methods— A modified Delphi process was implemented with an international expert panel representing patients, advocates, and clinical specialists in stroke outcomes, stroke registers, global health, epidemiology, and rehabilitation to reach consensus on the preferred outcome measures, included populations, and baseline risk adjustment variables. Results— Patients presenting to a hospital with ischemic stroke or intracerebral hemorrhage were selected as the target population for these recommendations, with the inclusion of transient ischemic attacks optional. Outcome categories recommended for assessment were survival and disease control, acute complications, and patient-reported outcomes. Patient-reported outcomes proposed for assessment at 90 days were pain, mood, feeding, selfcare, mobility, communication, cognitive functioning, social participation, ability to return to usual activities, and health-related quality of life, with mobility, feeding, selfcare, and communication also collected at discharge. One instrument was able to collect most patient-reported subdomains (9/16, 56%). Minimum data collection for risk adjustment included patient demographics, premorbid functioning, stroke type and severity, vascular and systemic risk factors, and specific treatment/care-related factors. Conclusions— A consensus stroke measure Standard Set was developed as a simple, pragmatic method to increase the value of stroke care. The set should be validated in practice when used for monitoring and comparisons across different care settings. PMID:26604251

  19. Recommended Patient-Reported Core Set of Symptoms to Measure in Head and Neck Cancer Treatment Trials

    PubMed Central

    Eisbruch, Avraham; Murphy, Barbara A.; Ridge, John A.; Gavin, Patrick; Reeve, Bryce B.; Bruner, Deborah Watkins; Movsas, Benjamin

    2014-01-01

    We identified a standard core set of patient-reported symptoms and health-related quality-of-life (HRQOL) domains to be assessed in head and neck (H&N) cancer clinical trials. The core symptom and HRQOL domain scores were used to guide recommendations by a working group of experts as part of a National Cancer Institute Symptom Management and HRQOL Clinical Trials Planning Meeting. A PubMed search was conducted using the search terms of “health-related quality of life” and “head & neck cancer,” limited to publications from January 1, 2000, to December 31, 2010. Fifty-four articles were used to guide the choice of recommendations. Twenty-nine symptoms and nine domains were identified, from which 12 H&N-specific core symptoms and HRQOL domains were recommended: swallowing, oral pain, skin changes, dry mouth, dental health, opening mouth/trismus, taste, excess/thick mucous/saliva, shoulder disability/motion, voice/hoarseness, social domain, and functional domain. This core set of 12 H&N-specific, patient-reported symptoms and HRQOL domains should be assessed in future H&N cancer clinical trials. PMID:25006189

  20. Recommended patient-reported core set of symptoms to measure in head and neck cancer treatment trials.

    PubMed

    Chera, Bhishamjit S; Eisbruch, Avraham; Murphy, Barbara A; Ridge, John A; Gavin, Patrick; Reeve, Bryce B; Bruner, Deborah Watkins; Movsas, Benjamin

    2014-07-01

    We identified a standard core set of patient-reported symptoms and health-related quality-of-life (HRQOL) domains to be assessed in head and neck (H&N) cancer clinical trials. The core symptom and HRQOL domain scores were used to guide recommendations by a working group of experts as part of a National Cancer Institute Symptom Management and HRQOL Clinical Trials Planning Meeting. A PubMed search was conducted using the search terms of "health-related quality of life" and "head & neck cancer," limited to publications from January 1, 2000, to December 31, 2010. Fifty-four articles were used to guide the choice of recommendations. Twenty-nine symptoms and nine domains were identified, from which 12 H&N-specific core symptoms and HRQOL domains were recommended: swallowing, oral pain, skin changes, dry mouth, dental health, opening mouth/trismus, taste, excess/thick mucous/saliva, shoulder disability/motion, voice/hoarseness, social domain, and functional domain. This core set of 12 H&N-specific, patient-reported symptoms and HRQOL domains should be assessed in future H&N cancer clinical trials. PMID:25006189

  1. Psychological factors predict adherence to methotrexate in rheumatoid arthritis; findings from a systematic review of rates, predictors and associations with patient-reported and clinical outcomes

    PubMed Central

    Bluett, James; Barton, Anne; Hyrich, Kimme L; Cordingley, Lis; Verstappen, Suzanne M M

    2016-01-01

    Treatment response to methotrexate (MTX) for rheumatoid arthritis (RA) is not universal and non-adherence may partially explain this. The aims of this systematic review were to: (1) summarise existing rates of adherence to MTX, (2) identify predictors of adherence to MTX, and (3) assess the association between non-adherence and patient outcomes. The authors conducted a systematic search of papers published from January 1980 to February 2015 in PubMed, PsycINFO, EMBASE and CINAHL databases. Studies were eligible for inclusion if: (1) MTX was used as monotherapy or in combination with other therapies, (2) MTX was used in an RA or inflammatory polyarthritis population, (3) adherence was defined and measured as the extent to which patients followed their MTX regimen during the period of prescription, and (4) it was an original piece of research. In total, 10 studies met the inclusion criteria and 8 were evaluated as high quality. Rates of adherence ranged from 59% to 107%, and exposed differences in definitions of adherence, study methodologies and sample heterogeneity. A number of potential predictors of MTX adherence were identified; the strongest being related to beliefs in the necessity and efficacy of MTX, absence of low mood, mild disease and MTX monotherapy. Furthermore, 3 studies tested the association of adherence with disease activity as an outcome measure; all 3 found non-adherence associated with poor treatment response. This systematic review shows the importance of adherence to MTX treatment and summarises the associated modifiable factors. PMID:26848403

  2. A Short-Term, Multicenter, Placebo-Controlled, Randomized Withdrawal Study of a Metabotropic Glutamate 2/3 Receptor Agonist Using an Electronic Patient-Reported Outcome Device in Patients With Schizophrenia

    PubMed Central

    Stauffer, Virginia L.; Baygani, Simin K.; Kinon, Bruce J.; Krikke-Workel, Judith O.

    2014-01-01

    Abstract This 6-week, multicenter, randomized withdrawal, placebo-controlled trial sought to determine whether symptoms of physical dependence occur after abrupt cessation of pomaglumetad methionil (LY2140023 monohydrate), a metabotropic glutamate 2/3 receptor agonist, in patients with schizophrenia. Eligible outpatients, 18 to 65 years old who required a modification or initiation of antipsychotic medication received 4 weeks of pomaglumetad methionil during open-label treatment and then were randomized, double-blind, to continue pomaglumetad methionil or receive placebo for 2 weeks. The primary outcome compared results of the 3-day moving mean of the total score on the Discontinuation Symptom Checklist-Modified Rickels for pomaglumetad methionil-treated patients with those on placebo during the randomized withdrawal phase. An electronic patient-reported outcome (ePRO) device was used daily to record these results. During the withdrawal phase, 103 patients were randomized, and 98 patients completed the trial. There was no statistically significant evidence of withdrawal symptoms associated with placebo compared with pomaglumetad methionil continuation as measured by Discontinuation Symptom Checklist-Modified Rickels (P = 0.170). The results are supported by secondary analyses with the clinician-rated, Clinical Institute Withdrawal Assessment of Alcohol Scale Revised, which showed no statistically significant differences between treatment groups. Using the ePRO device, 82.5% of the patients achieved 75% to 100% of compliance. No discontinuations due to worsening of schizophrenia, serious adverse events, deaths, or seizures were reported during either phase of the study. These findings suggest that there is no evidence of withdrawal symptoms associated with the abrupt discontinuation of pomaglumetad methionil and that an ePRO device can be successfully used in a multicenter schizophrenia trial. PMID:25006819

  3. A short-term, multicenter, placebo-controlled, randomized withdrawal study of a metabotropic glutamate 2/3 receptor agonist using an electronic patient-reported outcome device in patients with schizophrenia.

    PubMed

    Stauffer, Virginia L; Baygani, Simin K; Kinon, Bruce J; Krikke-Workel, Judith O

    2014-10-01

    This 6-week, multicenter, randomized withdrawal, placebo-controlled trial sought to determine whether symptoms of physical dependence occur after abrupt cessation of pomaglumetad methionil (LY2140023 monohydrate), a metabotropic glutamate 2/3 receptor agonist, in patients with schizophrenia. Eligible outpatients, 18 to 65 years old who required a modification or initiation of antipsychotic medication received 4 weeks of pomaglumetad methionil during open-label treatment and then were randomized, double-blind, to continue pomaglumetad methionil or receive placebo for 2 weeks. The primary outcome compared results of the 3-day moving mean of the total score on the Discontinuation Symptom Checklist-Modified Rickels for pomaglumetad methionil-treated patients with those on placebo during the randomized withdrawal phase. An electronic patient-reported outcome (ePRO) device was used daily to record these results. During the withdrawal phase, 103 patients were randomized, and 98 patients completed the trial. There was no statistically significant evidence of withdrawal symptoms associated with placebo compared with pomaglumetad methionil continuation as measured by Discontinuation Symptom Checklist-Modified Rickels (P = 0.170). The results are supported by secondary analyses with the clinician-rated, Clinical Institute Withdrawal Assessment of Alcohol Scale Revised, which showed no statistically significant differences between treatment groups. Using the ePRO device, 82.5% of the patients achieved 75% to 100% of compliance. No discontinuations due to worsening of schizophrenia, serious adverse events, deaths, or seizures were reported during either phase of the study. These findings suggest that there is no evidence of withdrawal symptoms associated with the abrupt discontinuation of pomaglumetad methionil and that an ePRO device can be successfully used in a multicenter schizophrenia trial. PMID:25006819

  4. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) and how to select an outcome measurement instrument

    PubMed Central

    Mokkink, Lidwine B.; Prinsen, Cecilia A. C.; Bouter, Lex M.; de Vet, Henrica C. W.; Terwee, Caroline B.

    2016-01-01

    Background: COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) is an initiative of an international multidisciplinary team of researchers who aim to improve the selection of outcome measurement instruments both in research and in clinical practice by developing tools for selecting the most appropriate available instrument. Method: In this paper these tools are described, i.e. the COSMIN taxonomy and definition of measurement properties; the COSMIN checklist to evaluate the methodological quality of studies on measurement properties; a search filter for finding studies on measurement properties; a protocol for systematic reviews of outcome measurement instruments; a database of systematic reviews of outcome measurement instruments; and a guideline for selecting outcome measurement instruments for Core Outcome Sets in clinical trials. Currently, we are updating the COSMIN checklist, particularly the standards for content validity studies. Also new standards for studies using Item Response Theory methods will be developed. Additionally, in the future we want to develop standards for studies on the quality of non-patient reported outcome measures, such as clinician-reported outcomes and performance-based outcomes. Conclusions: In summary, we plea for more standardization in the use of outcome measurement instruments, for conducting high quality systematic reviews on measurement instruments in which the best available outcome measurement instrument is recommended, and for stopping the use of poor outcome measurement instruments. PMID:26786084

  5. Patient-Reported Outcomes in Implant-Based Breast Reconstruction Alone or in Combination with a Titanium-Coated Polypropylene Mesh – A Detailed Analysis of the BREAST-Q and Overview of the Literature

    PubMed Central

    Dieterich, M.; Angres, J.; Stubert, J.; Stachs, A.; Reimer, T.; Gerber, B.

    2015-01-01

    Background: Complication rates and surgical outcomes are well reported for implant-based breast reconstruction (IBBR) using supportive materials for the inferior pole of the breast. Patient-reported outcomes (PRO) are underrepresented. The aim of this study was to compare PRO in IBBR using implants alone or in combination with a synthetic mesh. Methods and Methods: PRO was measured in patients undergoing IBBR alone or in combination with a titanium-covered polypropylene mesh (TiLOOP® Bra). In this non-randomized observational trial PRO was retrospectively assessed using the validated self-reporting BREAST-Q. The raw responses of all questions applied in each domain and transformed BREAST-Q data using the Q-Score are presented. Results: Of 90 eligible women, 42 received IBBR alone and 48 received IBBR in combination with mesh. No differences in complication rates were observed. The return rate was 67.7 % and was comparable between the groups (p = 0.117). PRO revealed no differences regarding satisfaction with breast shape (p = 0.079), outcome (p = 0.604), nipple sensitivity (p = 0.502), preoperative information (p = 0.195), office staff (p = 0.462), psychosocial well-being (p = 0.370), sexual well-being (p = 0.508) and physical well-being (p = 0.654). Significant differences were noted regarding satisfaction with the surgeon (p = 0.013) and medical staff (p = 0.035) as well as the response behavior of certain questions of the sub-domains, thus helping to further stratify PRO with regards to aesthetic outcome. However, no differences were observed in the main BREAST-Q results. Conclusion: Use of the TiLOOP® Bra in IBBR results in comparable BREAST-Q scores compared with IBBR alone. Evaluating the BREAST-Q sub-domains helps to stratify PRO more profoundly and assists in interpreting the overall results and specific research questions. PMID:26257406

  6. Revisiting the Surveillance Epidemiology and End Results Cancer Registry and Medicare Health Outcomes Survey (SEER-MHOS) Linked Data Resource for Patient-Reported Outcomes Research in Older Adults with Cancer.

    PubMed

    Kent, Erin E; Malinoff, Rochelle; Rozjabek, Heather M; Ambs, Anita; Clauser, Steven B; Topor, Marie A; Yuan, Gigi; Burroughs, James; Rodgers, Anne B; DeMichele, Kimberly

    2016-01-01

    Researchers and clinicians are increasingly recognizing the value of patient-reported outcome (PRO) data to better characterize people's health and experiences with illness and care. Considering the rising prevalence of cancer in adults aged 65 and older, PRO data are particularly relevant for older adults with cancer, who often require complex cancer care and have additional comorbid conditions. A data linkage between the Surveillance Epidemiology and End Results (SEER) cancer registry and the Medicare Health Outcomes Survey (MHOS) was created through a partnership between the National Cancer Institute and the Centers for Medicare and Medicaid Services that created the opportunity to examine PROs in Medicare Advantage enrollees with and without cancer. The December 2013 linkage of SEER-MHOS data included the linked data for 12 cohorts, bringing the number of individuals in the linked data set to 95,723 with cancer and 1,510,127 without. This article reviews the features of the resource and provides information on some descriptive characteristics of the individuals in the data set (health-related quality of life, body mass index, fall risk management, number of unhealthy days in the past month). Individuals without (n=258,108) and with (n=3,440) cancer (1,311 men with prostate cancer, 982 women with breast cancer, 689 with colorectal cancer, 458 with lung cancer) were included in the current descriptive analysis. Given increasing longevity, advances in effective therapies and earlier detection, and population growth, the number of individuals aged 65 and older with cancer is expected to reach more than 12 million by 2020. SEER-MHOS provides population-level, self-reported, cancer registry-linked data for person-centered surveillance research on this growing population. PMID:26782871

  7. Biobehavioral Measures as Outcomes: A Cautionary Tale

    PubMed Central

    Kovach, Christine R.; Woods, Diana Lynn; Devine, Elizabeth C.; Logan, Brent R.; Raff, Hershel

    2013-01-01

    This article discusses the use of biobehavioral measures as outcomes for healthcare intervention studies. Effect size (ES) values for salivary cortisol, and observation-based measures of pain and agitation are examined. Effects pre to post treatment were assessed separately for nursing home (NH) residents with and without acute psychotic symptoms. This study revealed large positive effects on both pain and agitation measures in the group with acute psychotic symptoms and small-to-medium positive effects on these same measures in the group without acute psychotic symptoms. In both of these groups the ES values were not consistently positive on the cortisol measures. Prior to determining if a measure can be used to estimate minimum clinically important differences, it is essential to consider if the biomarker will be responsive to therapy in the populations and contexts being studied. PMID:24158972

  8. Clinical outcome measures for Cutaneous Lupus Erythematosus

    PubMed Central

    Albrecht, Joerg; Werth, Victoria P.

    2011-01-01

    Cutaneous lupus erythematosus is a clinically heterogeneous group of rare skin diseases that only rarely have been subjected to controlled clinical trials. This may be have been partly due to a lack of suitable validated outcome instruments. Recently the FDA mandated that organ specific trials for lupus erythematosus need to use a combination of different outcome measures. The patient’s condition needs to be assessed in terms of quality of life, the patient’s global response and organ specific instruments that measure activity of the disease as well as damage due to the disease. For the skin the only formally validated and published instrument is currently the Cutaneous Lupus Erythematosis Disease Area and Severity Index (CLASI). This paper discusses the background of the development of the CLASI as well as issues related to its use and interpretation in the context of clinical research of CLE. PMID:20693208

  9. Understanding the outcomes measures used in Huntington disease pharmacologicaltrials: A systematic review

    PubMed Central

    Carlozzi, Noelle E; Miciura, Angela; Migliore, Nicholas; Dayalu, Praveen

    2014-01-01

    Background The identification of the gene mutation causing Huntington disease has raised hopes for new treatments to ease symptoms and slow functional decline. As such, there has been a push towards designing efficient pharmacological trials (i.e., drug trials), especially with regard to selecting outcomes measures that are both brief and sensitive to changes across the course of the disease, from subtle prodromal changes, to more severe end-stage changes. Objectives Recently, to aid in efficient development of new HD research studies, the National Institute of Neurological Disorders and Stroke (NINDS) published recommendations for measurement selection in HD. While these recommendations are helpful, many of the recommended measures have little published data in HD. As such, we conducted a systematic review of the literature to identify the most common outcomes measures used in HD clinical trials. Methods Major medical databases, including PubMed, Embase, CINAHL, and the Cochrane Central Register of Controlled Trials, were used to identify peer-reviewed journal articles in English from 2001 through April 2013; 151 pharmacological trials were identified. Results The majority of HD clinical trials employed clinician-reported outcomes measures (93%); patient reported outcome measures (11%) and observer reported outcome measures (3%) were used with much less frequency. Conclusions We provide a review of the most commonly used measures across these trials, compare these measures to the clinical recommendations made by the NINDS working groups, and provide recommendations for selecting measures for future clinical trials that meet the Food and Drug Administration standards. PMID:25300328

  10. Outcome measures for primary Sjögren's syndrome.

    PubMed

    Seror, Raphaèle; Bootsma, Hendrika; Bowman, Simon J; Dörner, Thomas; Gottenberg, Jacques-Eric; Mariette, Xavier; Ramos-Casals, Manel; Ravaud, Philippe; Theander, Elke; Tzioufas, Athanasios; Vitali, Claudio

    2012-08-01

    Lymphocytic infiltration of different exocrine and non-exocrine epithelia is the pathological hallmark of primary Sjögren's syndrome, whereas involvement of salivary and lachrymal glands with the clinical counterpart of dry eye and dry mouth are the predominant features of the disease, together with fatigue and musculoskeletal pain. In addition, systemic manifestations, like arthritis, skin vasculitis, peripheral neuropathy, glomerulonephritis, may also be present in a consistent number of patients. As result, clinical features in SS can be divided into two facets: the benign subjective but disabling manifestations such as dryness, pain and fatigue, and the systemic manifestations. In the past decades, a core set of domains, which included sicca symptoms, objective measurements of tear and saliva production, fatigue, quality of life, disease activity and damage was indicated as essential for outcome assessment in this disorder. Afterwards, great efforts have been made to develop valid tools for the assessment of different domains. Specific questionnaires such as the Profile of Fatigue and Discomfort (PROFAD) and Sicca Symptoms Inventory (SSI) have been proposed as dedicated tools for the evaluation of patients symptoms, whereas different composite indexes have been suggested for the assessment of disease activity and damage. Some of these preliminary studies served as bases of an international project supported by EULAR, aimed at developing two consensus disease activity indexes: the EULAR Sjögren's Syndrome Patients Reported Index (ESSPRI), and the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI), a systemic activity index to assess systemic manifestations. A detailed and critical review of all these indexes is provided in this article. Both EULAR indexes showed, in recent studies, to be feasible, valid, and reliable instruments. After their final validation, which is currently in process, they could be used as consensus outcome criteria in therapeutic

  11. Patient-reported outcomes are superior in patients with Type 2 diabetes treated with liraglutide as compared with exenatide, when added to metformin, sulphonylurea or both: results from a randomized, open-label study

    PubMed Central

    Schmidt, W E; Christiansen, J S; Hammer, M; Zychma, M J; Buse, J B

    2011-01-01

    Aims The Liraglutide Effect and Action in Diabetes 6 trial was an open-label trial comparing liraglutide with exenatide as an ‘add-on’ to metformin and/or sulphonylurea. Methods Patients with Type 2 diabetes were randomized to liraglutide 1.8 mg once daily or exenatide 10 μg twice daily for 26 weeks. This was followed by a 14-week extension phase, in which all patients received liraglutide 1.8 mg once daily. Results Patient-reported outcomes were measured in 379 patients using Diabetes Treatment Satisfaction Questionnaire status (DTSQs) and DTSQ change (DTSQc). The change in overall treatment satisfaction (DTSQs score) from baseline at week 26 with liraglutide was 4.71 and with exentaide was 1.66 [difference between groups 3.04 (95% CI 1.73–4.35), P < 0.0001]. Five of the six items on the DTSQs improved significantly more with liraglutide than with exenatide (differences: current treatment 0.37, P = 0.0093; convenience 0.68, P < 0.0001; flexibility 0.57, P = 0.0002; recommend 0.49, P = 0.0003; continue 0.66, P = 0.0001). Patients perceived a greater reduction in hypoglycaemia at week 26 with liraglutide than with exenatide [difference in DTSQc score 0.48 (0.08–0.89), P = 0.0193] and a greater reduction in perceived hyperglycaemia [difference 0.74 (0.31–1.17), P = 0.0007]. During the extension phase, when all patients received liraglutide, DTSQs scores remained stable in patients who continued on liraglutide and increased significantly (P = 0.0026) in those switching from exenatide. Conclusions These results demonstrate significant improvements in patients’ treatment satisfaction with liraglutide compared with exenatide. PMID:21388442

  12. Residual Effects of Sleep Medications Are Commonly Reported and Associated with Impaired Patient-Reported Outcomes among Insomnia Patients in the United States.

    PubMed

    Fitzgerald, Timothy; Vietri, Jeffrey

    2015-01-01

    Study Objective. To measure the association of symptoms attributed to residual effects of sleep medication (e.g., drowsiness, difficulty concentrating, and impaired memory) on self-reported functioning and satisfaction with these medications. Methods. Individuals using prescription medications for insomnia were invited to complete an Internet-based survey. Respondents were compared according to the presence of self-reported residual effects; relationships between severity of these effects and outcomes were modeled using regression. Measures included the Brief Insomnia Questionnaire, Work Productivity and Activity Impairment Questionnaire, and SATMED-Q. Subgroup analyses were conducted with patients aged ≥65 years. Approximately 80% reported experiencing ≥1 residual effect. The severity of residual effects was associated with increased residual effect-related work impairment, including absenteeism (RR = 1.46, p < 0.001), presenteeism (RR = 1.12, p < 0.001), overall work impairment (RR = 1.13, p < 0.001), and nonwork activity impairment (RR = 1.11, p < 0.001). More severe residual symptoms were also associated with increased difficulty in home management (Beta = .31, p < 0.001), ability to work (Beta = .31, p < 0.001), social relationships, (Beta = .32, p < 0.001), close personal relationships (Beta = .30, p < 0.001), and lower medication satisfaction (Beta = -.37, p < 0.001). Conclusions. Individuals using medications for insomnia commonly experience symptoms considered as residual effects, and these symptoms are associated with greater interference of sleep-related problems at work, at home, and with social relationships. PMID:26783470

  13. Residual Effects of Sleep Medications Are Commonly Reported and Associated with Impaired Patient-Reported Outcomes among Insomnia Patients in the United States

    PubMed Central

    Fitzgerald, Timothy; Vietri, Jeffrey

    2015-01-01

    Study Objective. To measure the association of symptoms attributed to residual effects of sleep medication (e.g., drowsiness, difficulty concentrating, and impaired memory) on self-reported functioning and satisfaction with these medications. Methods. Individuals using prescription medications for insomnia were invited to complete an Internet-based survey. Respondents were compared according to the presence of self-reported residual effects; relationships between severity of these effects and outcomes were modeled using regression. Measures included the Brief Insomnia Questionnaire, Work Productivity and Activity Impairment Questionnaire, and SATMED-Q. Subgroup analyses were conducted with patients aged ≥65 years. Approximately 80% reported experiencing ≥1 residual effect. The severity of residual effects was associated with increased residual effect-related work impairment, including absenteeism (RR = 1.46, p < 0.001), presenteeism (RR = 1.12, p < 0.001), overall work impairment (RR = 1.13, p < 0.001), and nonwork activity impairment (RR = 1.11, p < 0.001). More severe residual symptoms were also associated with increased difficulty in home management (Beta = .31, p < 0.001), ability to work (Beta = .31, p < 0.001), social relationships, (Beta = .32, p < 0.001), close personal relationships (Beta = .30, p < 0.001), and lower medication satisfaction (Beta = −.37, p < 0.001). Conclusions. Individuals using medications for insomnia commonly experience symptoms considered as residual effects, and these symptoms are associated with greater interference of sleep-related problems at work, at home, and with social relationships. PMID:26783470

  14. Patient-Reported Voice and Speech Outcomes After Whole-Neck Intensity Modulated Radiation Therapy and Chemotherapy for Oropharyngeal Cancer: Prospective Longitudinal Study

    SciTech Connect

    Vainshtein, Jeffrey M.; Griffith, Kent A.; Feng, Felix Y.; Vineberg, Karen A.; Chepeha, Douglas B.; Eisbruch, Avraham

    2014-08-01

    Purpose: To describe voice and speech quality changes and their predictors in patients with locally advanced oropharyngeal cancer treated on prospective clinical studies of organ-preserving chemotherapy–intensity modulated radiation therapy (chemo-IMRT). Methods and Materials: Ninety-one patients with stage III/IV oropharyngeal cancer were treated on 2 consecutive prospective studies of definitive chemoradiation using whole-field IMRT from 2003 to 2011. Patient-reported voice and speech quality were longitudinally assessed from before treatment through 24 months using the Communication Domain of the Head and Neck Quality of Life (HNQOL-C) instrument and the Speech question of the University of Washington Quality of Life (UWQOL-S) instrument, respectively. Factors associated with patient-reported voice quality worsening from baseline and speech impairment were assessed. Results: Voice quality decreased maximally at 1 month, with 68% and 41% of patients reporting worse HNQOL-C and UWQOL-S scores compared with before treatment, and improved thereafter, recovering to baseline by 12-18 months on average. In contrast, observer-rated larynx toxicity was rare (7% at 3 months; 5% at 6 months). Among patients with mean glottic larynx (GL) dose ≤20 Gy, >20-30 Gy, >30-40 Gy, >40-50 Gy, and >50 Gy, 10%, 32%, 25%, 30%, and 63%, respectively, reported worse voice quality at 12 months compared with before treatment (P=.011). Results for speech impairment were similar. Glottic larynx dose, N stage, neck dissection, oral cavity dose, and time since chemo-IMRT were univariately associated with either voice worsening or speech impairment. On multivariate analysis, mean GL dose remained independently predictive for both voice quality worsening (8.1%/Gy) and speech impairment (4.3%/Gy). Conclusions: Voice quality worsening and speech impairment after chemo-IMRT for locally advanced oropharyngeal cancer were frequently reported by patients, underrecognized by clinicians, and

  15. The Electronic Patient Reported Outcome Tool: Testing Usability and Feasibility of a Mobile App and Portal to Support Care for Patients With Complex Chronic Disease and Disability in Primary Care Settings

    PubMed Central

    Gill, Ashlinder; Khan, Anum Irfan; Hans, Parminder Kaur; Kuluski, Kerry; Cott, Cheryl

    2016-01-01

    Background People experiencing complex chronic disease and disability (CCDD) face some of the greatest challenges of any patient population. Primary care providers find it difficult to manage multiple discordant conditions and symptoms and often complex social challenges experienced by these patients. The electronic Patient Reported Outcome (ePRO) tool is designed to overcome some of these challenges by supporting goal-oriented primary care delivery. Using the tool, patients and providers collaboratively develop health care goals on a portal linked to a mobile device to help patients and providers track progress between visits. Objectives This study tested the usability and feasibility of adopting the ePRO tool into a single interdisciplinary primary health care practice in Toronto, Canada. The Fit between Individuals, Fask, and Technology (FITT) framework was used to guide our assessment and explore whether the ePRO tool is: (1) feasible for adoption in interdisciplinary primary health care practices and (2) usable from both the patient and provider perspectives. This usability pilot is part of a broader user-centered design development strategy. Methods A 4-week pilot study was conducted in which patients and providers used the ePRO tool to develop health-related goals, which patients then monitored using a mobile device. Patients and providers collaboratively set goals using the system during an initial visit and had at least 1 follow-up visit at the end of the pilot to discuss progress. Focus groups and interviews were conducted with patients and providers to capture usability and feasibility measures. Data from the ePRO system were extracted to provide information regarding tool usage. Results Six providers and 11 patients participated in the study; 3 patients dropped out mainly owing to health issues. The remaining 8 patients completed 210 monitoring protocols, equal to over 1300 questions, with patients often answering questions daily. Providers and patients

  16. The International Dermatology Outcome Measures Group: formation of patient-centered outcome measures in dermatology.

    PubMed

    Gottlieb, Alice B; Levin, Adriane A; Armstrong, April W; Abernethy, April; Duffin, Kristina Callis; Bhushan, Reva; Garg, Amit; Merola, Joseph F; Maccarone, Mara; Christensen, Robin

    2015-02-01

    As quality standards are increasingly in demand throughout medicine, dermatology needs to establish outcome measures to quantify the effectiveness of treatments and providers. The International Dermatology Outcome Measures Group was established to address this need. Beginning with psoriasis, the group aims to create a tool considerate of patients and providers using the input of all relevant stakeholders in assessment of disease severity and response to treatment. Herein, we delineate the procedures through which consensus is being reached and the future directions of the project. PMID:25486914

  17. Impact of efalizumab on patient-reported outcomes in high-need psoriasis patients: results of the international, randomized, placebo-controlled Phase III Clinical Experience Acquired with Raptiva (CLEAR) trial [NCT00256139

    PubMed Central

    Ortonne, Jean-Paul; Shear, Neil; Shumack, Stephen; Henninger, Eric

    2005-01-01

    Background Chronic psoriasis can negatively affect patients' lives. Assessing the impact of treatment on different aspects of a patient's health-related quality of life (HRQOL) is therefore important and relevant in trials of anti-psoriasis agents. The recombinant humanized IgG1 monoclonal antibody efalizumab targets multiple T-cell-dependent steps in the immunopathogenesis of psoriasis. Efalizumab has demonstrated safety and efficacy in several clinical trials, and improves patients' quality of life. Objective: To evaluate the impact of efalizumab on HRQOL and other patient-reported outcomes in patients with moderate to severe plaque psoriasis, including a large cohort of High-Need patients for whom at least 2 other systemic therapies were unsuitable because of lack of efficacy, intolerance, or contraindication. Methods A total of 793 patients were randomized in a 2:1 ratio to receive efalizumab 1 mg/kg/wk (n = 529) or placebo (n = 264) for 12 weeks. The study population included 526 High-Need patients (342 efalizumab, 184 placebo). The treatment was evaluated by patients using the HRQOL assessment tools Short Form-36 (SF-36) and Dermatology Life Quality Index (DLQI). Other patient-reported assessments included the Psoriasis Symptom Assessment (PSA), a visual analog scale (VAS) for itching, and the Patient's Global Psoriasis Assessment (PGPA). Results Efalizumab was associated with improvements at Week 12 from baseline in patient-reported outcomes, both in the total study population and in the High-Need cohort. Among all efalizumab-treated patients, the DLQI improved by 5.7 points from baseline to Week 12, relative to an improvement of 2.3 points for placebo patients (P < .001). Corresponding improvements in DLQI in the High-Need cohort were 5.4 points for efalizumab compared to 2.3 for placebo (P < .001). Improvements from baseline on the SF-36, PSA, PGPA, and itching VAS at Week 12 were also significantly greater in efalizumab-treated patients than for placebo

  18. A promising method for identifying cross-cultural differences in patient perspective: the use of Internet-based focus groups for content validation of new Patient Reported Outcome assessments

    PubMed Central

    Atkinson, Mark J; Lohs, Jan; Kuhagen, Ilka; Kaufman, Julie; Bhaidani, Shamsu

    2006-01-01

    Objectives This proof of concept (POC) study was designed to evaluate the use of an Internet-based bulletin board technology to aid parallel cross-cultural development of thematic content for a new set of patient-reported outcome measures (PROs). Methods The POC study, conducted in Germany and the United States, utilized Internet Focus Groups (IFGs) to assure the validity of new PRO items across the two cultures – all items were designed to assess the impact of excess facial oil on individuals' lives. The on-line IFG activities were modeled after traditional face-to-face focus groups and organized by a common 'Topic' Guide designed with input from thought leaders in dermatology and health outcomes research. The two sets of IFGs were professionally moderated in the native language of each country. IFG moderators coded the thematic content of transcripts, and a frequency analysis of code endorsement was used to identify areas of content similarity and difference between the two countries. Based on this information, draft PRO items were designed and a majority (80%) of the original participants returned to rate the relative importance of the newly designed questions. Findings The use of parallel cross-cultural content analysis of IFG transcripts permitted identification of the major content themes in each country as well as exploration of the possible reasons for any observed differences between the countries. Results from coded frequency counts and transcript reviews informed the design and wording of the test questions for the future PRO instrument(s). Subsequent ratings of item importance also deepened our understanding of potential areas of cross-cultural difference, differences that would be explored over the course of future validation studies involving these PROs. Conclusion The use of IFGs for cross-cultural content development received positive reviews from participants and was found to be both cost and time effective. The novel thematic coding methodology

  19. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2014 CFR

    2014-10-01

    ... 42 Public Health 2 2014-10-01 2014-10-01 false Diabetes outcome measurements. 410.146 Section 410.146 Public Health CENTERS FOR MEDICARE & MEDICAID SERVICES, DEPARTMENT OF HEALTH AND HUMAN SERVICES... Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  20. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2012 CFR

    2012-10-01

    ... 42 Public Health 2 2012-10-01 2012-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  1. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2013 CFR

    2013-10-01

    ... 42 Public Health 2 2013-10-01 2013-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  2. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2010 CFR

    2010-10-01

    ... 42 Public Health 2 2010-10-01 2010-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  3. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2011 CFR

    2011-10-01

    ... 42 Public Health 2 2011-10-01 2011-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  4. Applying Outcome Measurements: A Guide to Educational Outcome Measurements and Their Uses. Seminar No. 5.

    ERIC Educational Resources Information Center

    Glaser, Ezra

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministries, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with the application of outcome…

  5. General Outcome Measures for Verbal Operants

    PubMed Central

    Kubina, Richard M; Wolfe, Pamela; Kostewicz, Douglas E

    2009-01-01

    A general outcome measure (GOM) can be used to show progress towards a long-term goal. GOMs should sample domains of behavior across ages, be sensitive to change over time, be inexpensive and easy to use, and facilitate decision making. Skinner's (1957) analysis of verbal behavior may benefit from the development of GOM. To develop GOM, we conducted a review of the literature on mands, tacts, echoics, and intraverbals. The four areas reviewed included (a) an examination of the participant's response form (i.e., vocal or nonvocal), (b) the type of prompt used, (c) types of materials used, and (d) timing of responses or sessions. Based on the review of the literature, we developed GOM for mands and tacts. This paper attempts to bridge the concept of GOMs with Skinner's analysis of verbal behavior. PMID:22477427

  6. Efficacy and patient-reported outcomes with dose-intense temozolomide in patients with newly diagnosed pure and mixed anaplastic oligodendroglioma: a phase II multicenter study.

    PubMed

    Ahluwalia, Manmeet S; Xie, Hao; Dahiya, Saurabh; Hashemi-Sadraei, Nooshin; Schiff, David; Fisher, Paul G; Chamberlain, Marc C; Pannullo, Susan; Newton, Herbert B; Brewer, Cathy; Wood, Laura; Prayson, Richard; Elson, Paul; Peereboom, David M

    2015-03-01

    Standard initial therapy for patients with pure and mixed anaplastic oligodendrogliomas (AO/MAO) includes chemotherapy and radiation therapy. Anaplastic oligodendrogliomas with 1p/19q co-deletion are more responsive to chemotherapy. There is concern for potential long-term CNS toxicity of radiation. Hence an approach using chemotherapy initially and reserving radiation for progressive disease is attractive. This multicenter phase II trial included patients with newly diagnosed AO/MAO with central pathology review and 1p/19q assay. Temozolomide was given 150 mg/m(2) days 1-7 and 15-21, every 28 days for 8 cycles. The primary endpoint was progression free survival (PFS). Secondary endpoints included response rate, overall survival (OS), treatment toxicity and health-related quality of life (HRQL). Data from 62 patients enrolled between December 2001 and April 2007 at seven centers were analyzed. Among patients with measurable disease, 8 % achieved complete remission, 56 % had stable disease and 36 % had progression. The median PFS and OS were 27.2 months (95 % CI 11.9-36.3) and 105.8 months (95 % CI 51.5-N/A), respectively. Both 1p loss and 1p/19q co-deletion were positive prognostic factors for PFS (p < 0.001) and OS (p < 0.001); and there was some suggestion that 1p/19q co-deletion also predicted better response to chemotherapy (p = 0.007). Grade 3/4 toxicities were mainly hematological. Significantly improved HRQL in the future uncertainty domain of the brain cancer module was seen after cycle 4 (p < 0.001). This trial achieved outcomes similar to those reported previously. Toxicities from dose-intense temozolomide were manageable. Improvement in at least one HRQL domain increased over time. This trial supports the further study of first-line temozolomide monotherapy as an alternative to radiation therapy for patients with newly diagnosed AO/MAO with 1p 19q co-deleted tumors. PMID:25534576

  7. Report from the third international consensus meeting to harmonise core outcome measures for atopic eczema/dermatitis clinical trials (HOME)

    PubMed Central

    Chalmers, JR; Schmitt, J; Apfelbacher, C; Dohil, M; Eichenfield, LF; Simpson, EL; Singh, J; Spuls, P; Thomas, KS; Admani, S; Aoki, V; Ardeleanu, M; Barbarot, S; Berger, T; Bergman, JN; Block, J; Borok, N; Burton, T; Chamlin, SL; Deckert, S; DeKlotz, CC; Graff, LB; Hanifin, JM; Hebert, AA; Humphreys, R; Katoh, N; Kisa, RM; Margolis, DJ; Merhand, S; Minnillo, R; Mizutani, H; Nankervis, H; Ohya, Y; Rodgers, P; Schram, ME; Stalder, JF; Svensson, A; Takaoka, R; Teper, A; Tom, WL; von Kobyletzki, L; Weisshaar, E; Zelt, S; Williams, HC

    2014-01-01

    Summary This report provides a summary of the third meeting of the Harmonising Outcome Measures for Eczema (HOME) initiative held in San Diego, CA, U.S.A., 6–7 April 2013 (HOME III). The meeting addressed the four domains that had previously been agreed should be measured in every eczema clinical trial: clinical signs, patient-reported symptoms, long-term control and quality of life. Formal presentations and nominal group techniques were used at this working meeting, attended by 56 voting participants (31 of whom were dermatologists). Significant progress was made on the domain of clinical signs. Without reference to any named scales, it was agreed that the intensity and extent of erythema, excoriation, oedema/papulation and lichenification should be included in the core outcome measure for the scale to have content validity. The group then discussed a systematic review of all scales measuring the clinical signs of eczema and their measurement properties, followed by a consensus vote on which scale to recommend for inclusion in the core outcome set. Research into the remaining three domains was presented, followed by discussions. The symptoms group and quality of life groups need to systematically identify all available tools and rate the quality of the tools. A definition of long-term control is needed before progress can be made towards recommending a core outcome measure. What's already known about this topic? Many different scales have been used to measure eczema, making it difficult to compare trials in meta-analyses and hampering improvements in clinical practice. HOME core outcome measures must pass the OMERACT (Outcome Measures in Rheumatology) filter of truth (validity), discrimination (sensitivity to change and responsiveness) and feasibility (ease of use, costs, time to perform and interpret). It has been previously agreed as part of the consensus process that four domains should be measured by the core outcomes: clinical signs, patient-reported

  8. Child Outcome Measures in the Study of Child Care Quality

    ERIC Educational Resources Information Center

    Zaslow, Martha; Halle, Tamara; Martin, Laurie; Cabrera, Natasha; Calkins, Julia; Pitzer, Lindsay; Margie, Nancy Geyelin

    2006-01-01

    This article assesses whether there are methodological problems with child outcome measures that may contribute to the small associations between child care quality and child outcomes found in the literature. Outcome measures used in 65 studies of child care quality published between 1979 and December 2005 were examined, taking the previous review…

  9. 42 CFR 486.318 - Condition: Outcome measures.

    Code of Federal Regulations, 2010 CFR

    2010-10-01

    ... SUPPLIERS Requirements for Certification and Designation and Conditions for Coverage: Organ Procurement Organizations Organ Procurement Organization Outcome Requirements § 486.318 Condition: Outcome measures. (a... of organs transplanted per standard criteria donor, including pancreata used for islet...

  10. 42 CFR 486.318 - Condition: Outcome measures.

    Code of Federal Regulations, 2011 CFR

    2011-10-01

    ... SUPPLIERS Requirements for Certification and Designation and Conditions for Coverage: Organ Procurement Organizations Organ Procurement Organization Outcome Requirements § 486.318 Condition: Outcome measures. (a... of organs transplanted per standard criteria donor, including pancreata used for islet...

  11. 42 CFR 486.318 - Condition: Outcome measures.

    Code of Federal Regulations, 2014 CFR

    2014-10-01

    ... SUPPLIERS Requirements for Certification and Designation and Conditions for Coverage: Organ Procurement Organizations Organ Procurement Organization Outcome Requirements § 486.318 Condition: Outcome measures. (a... transplantation; (ii) The number of organs transplanted per expanded criteria donor, including pancreata used...

  12. 42 CFR 486.318 - Condition: Outcome measures.

    Code of Federal Regulations, 2012 CFR

    2012-10-01

    ... SUPPLIERS Requirements for Certification and Designation and Conditions for Coverage: Organ Procurement Organizations Organ Procurement Organization Outcome Requirements § 486.318 Condition: Outcome measures. (a... transplantation; (ii) The number of organs transplanted per expanded criteria donor, including pancreata used...

  13. Habitual Functional Electrical Stimulation Therapy Improves Gait Kinematics and Walking Performance, but Not Patient-Reported Functional Outcomes, of People with Multiple Sclerosis who Present with Foot-Drop

    PubMed Central

    van der Linden, Marietta L.; Hooper, Julie E.; Cowan, Paula; Weller, Belinda B.; Mercer, Thomas H.

    2014-01-01

    Background People with Multiple Sclerosis (pwMS) often experience a disturbed gait function such as foot-drop. The objective of this pilot study was to investigate the medium term effects of using Functional Electrical Stimulation (FES) to treat foot-drop over a period 12 weeks on gait and patient reported outcomes of pwMS. Methods and Findings Nine pwMS aged 35 to 64 (2 males, 7 females) were assessed on four occasions; four weeks before baseline, at baseline and after six weeks and twelve weeks of FES use. Joint kinematics and performance on the 10 meter and 2 minute walk tests (10WT, 2 minWT) were assessed with and without FES. Participants also completed the MS walking Scale (MSWS), MS impact scale (MSIS29), Fatigue Severity Score (FSS) and wore an activity monitor for seven days after each assessment. Compared to unassisted walking, FES resulted in statistically significant improvements in peak dorsiflexion in swing (p = 0.006), 10MWT (p = 0.006) and 2 minWT (p = 0.002). Effect sizes for the training effect, defined as the change from unassisted walking at baseline to that at 12 weeks, indicated improved ankle angle at initial contact (2.6°, 95% CI −1° to 4°, d = 0.78), and a decrease in perceived exertion over the 2 min walking tests (−1.2 points, 95% CI −5.7 to 3.4, d = −0.86). Five participants exceeded the Minimally Detectable Change (MDC) for a training effect on the 10mWT, but only two did so for the 2 minWT. No effects of the use of FES for 12 weeks were found for MSWS, MSIS29, FSS or step count. Conclusion Although FES to treat foot-drop appears to offer the potential for a medium term training effect on ankle kinematics and walking speed, this was not reflected in the patient reported outcomes. This observed lack of relationship between objective walking performance and patient reported outcomes warrants further investigation. Trial Registration ClinicalTrials.gov NCT01977287 PMID:25133535

  14. Oncology Section EDGE Task Force on Prostate Cancer: A Systematic Review of Outcome Measures for Health-Related Quality of Life

    PubMed Central

    Harrington, Shana; Lee, Jeannette; Colon, Genevieve; Alappattu, Meryl

    2016-01-01

    Background Health-related quality of life (HRQOL) is multidimensional and subjective, encompassing occupational and physical function, psychological state, social interaction, and somatic sensation. Because of the wide scope of HRQOL and its implications for the medical management of prostate cancer survivors, clinicians and researchers need to know the most reliable and valid measures currently available for addressing this clinical construct in men diagnosed with prostate cancer. Purpose To identify and evaluate patient-reported outcome measures used to assess HRQOL in men diagnosed with prostate cancer and make recommendations for the use of these patient-reported outcome measures in both the research and clinic settings. Methods The literature was systematically reviewed for patient-reported outcome measures used in peer-reviewed, published research to assess HRQOL in men diagnosed with prostate cancer. The goals were to examine the psychometric properties of commonly used HRQOL measures in order to determine their clinical utility. Results The following patient-reported outcome measures are highly recommended by the Task Force to assess HRQOL in men diagnosed with prostate cancer: EORTC QLQ-P25 (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire–Prostate 25), EPIC (Expanded Prostate Cancer Index Composite), FACT-P (Functional Assessment of Cancer Therapy–Prostate), UCLA-PCI (UCLA–Prostate Cancer Index), EORTC QLQ-C30 (EORTC Quality of Life Questionnaire–Cancer 30), FACT-G (Functional Assessment of Cancer Therapy–General), and SF (Short-Form) 36, 12, and 8. Conclusions A variety of patient-reported outcome measures have been reported in the literature to assess HRQOL in men diagnosed with prostate cancer. Seven measures were found to have satisfactory psychometric properties, as well as good clinical utility, and are recommended for use by the researchers on this Task Force. PMID:27134804

  15. Quality of life with palbociclib plus fulvestrant in previously treated hormone receptor-positive, HER2-negative metastatic breast cancer: patient-reported outcomes from the PALOMA-3 trial

    PubMed Central

    Harbeck, N.; Iyer, S.; Turner, N.; Cristofanilli, M.; Ro, J.; André, F.; Loi, S.; Verma, S.; Iwata, H.; Bhattacharyya, H.; Puyana Theall, K.; Bartlett, C. H.; Loibl, S.

    2016-01-01

    Background In the PALOMA-3 study, palbociclib plus fulvestrant demonstrated improved progression-free survival compared with fulvestrant plus placebo in hormone receptor-positive, HER2− endocrine-resistant metastatic breast cancer (MBC). This analysis compared patient-reported outcomes (PROs) between the two treatment groups. Patients and methods Patients were randomized 2 : 1 to receive palbociclib 125 mg/day orally for 3 weeks followed by 1 week off (n = 347) plus fulvestrant (500 mg i.m. per standard of care) or placebo plus fulvestrant (n = 174). PROs were assessed on day 1 of cycles 1–4 and of every other subsequent cycle starting with cycle 6 using the EORTC QLQ-C30 and its breast cancer module, QLQ-BR23. High scores (range 0–100) could indicate better functioning/quality of life (QoL) or worse symptom severity. Repeated-measures mixed-effect analyses were carried out to compare on-treatment overall scores and changes from baseline between treatment groups while controlling for baseline. Between-group comparisons of time to deterioration in global QoL and pain were made using an unstratified log-rank test and Cox proportional hazards model. Results Questionnaire completion rates were high at baseline and during treatment (from baseline to cycle 14, ≥95.8% in each group completed ≥1 question on the EORTC QLQ-C30). On treatment, estimated overall global QoL scores significantly favored the palbociclib plus fulvestrant group [66.1, 95% confidence interval (CI) 64.5–67.7 versus 63.0, 95% CI 60.6–65.3; P = 0.0313]. Significantly greater improvement from baseline in pain was also observed in this group (−3.3, 95% CI −5.1 to −1.5 versus 2.0, 95% CI −0.6 to 4.6; P = 0.0011). No significant differences were observed for other QLQ-BR23 functioning domains, breast or arm symptoms. Treatment with palbociclib plus fulvestrant significantly delayed deterioration in global QoL (P < 0.025) and pain (P < 0.001) compared with fulvestrant alone. Conclusion

  16. Supporting Goal-Oriented Primary Health Care for Seniors with Complex Care Needs Using Mobile Technology: Evaluation and Implementation of the Health System Performance Research Network, Bridgepoint Electronic Patient Reported Outcome Tool

    PubMed Central

    Wodchis, Walter P; Upshur, Ross; Cott, Cheryl; McKinstry, Brian; Mercer, Stewart; Palen, Ted E; Ramsay, Tim; Thavorn, Kednapa

    2016-01-01

    Background Older adults experiencing multiple chronic illnesses are at high risk of hospitalization and health decline if they are unable to manage the significant challenges posed by their health conditions. Goal-oriented care approaches can provide better care for these complex patients, but clinicians find the process of ascertaining goals “too complex and too-time consuming,” and goals are often not agreed upon between complex patients and their providers. The electronic patient reported outcomes (ePRO) mobile app and portal offers an innovative approach to creating and monitoring goal-oriented patient-care plans to improve patient self-management and shared decision-making between patients and health care providers. The ePRO tool also supports proactive patient monitoring by the patient, caregiver(s), and health care provider. It was developed with and for older adults with complex care needs as a means to improve their quality of life. Objective Our proposed project will evaluate the use, effectiveness, and value for money of the ePRO tool in a 12-month multicenter, randomized controlled trial in Ontario; targeting individuals 65 or over with two or more chronic conditions that require frequent health care visits to manage their health conditions. Methods Intervention groups using the ePRO tool will be compared with control groups on measures of quality of life, patient experience, and cost-effectiveness. We will also evaluate the implementation of the tool. Results The proposed project presented in this paper will be funded through the Canadian Institute for Health Research (CIHR) eHealth Innovation Partnerships Program (eHIPP) program (CIHR–348362). The expected completion date of the study is November, 2019. Conclusions We anticipate our program of work will support improved quality of life and patient self-management, improved patient-centered primary care delivery, and will encourage the adoption of goal-oriented care approaches across primary

  17. Comparative Effectiveness of Sphincter-Sparing Surgery versus Abdominoperineal Resection in Rectal Cancer: Patient-Reported Outcomes in National Surgical Adjuvant Breast and Bowel Project Randomized Trial R-04

    PubMed Central

    Russell, Marcia M.; Ganz, Patricia A.; Lopa, Samia; Yothers, Greg; Ko, Clifford Y.; Arora, Amit; Atkins, James N.; Bahary, Nathan; Soori, Gamini; Robertson, John M.; Eakle, Janice; Marchello, Benjamin T.; Wozniak, Timothy F.; Beart, Robert W.; Wolmark, Norman

    2015-01-01

    Objective NSABP R-04 was a randomized controlled trial of neoadjuvant chemoradiotherapy in patients with resectable stage II–III rectal cancer. We hypothesized that patients who underwent abdominoperineal resection (APR) would have a poorer quality of life than those who underwent sphincter-sparing surgery (SSS). Methods To obtain patient-reported outcomes (PROs) we administered two symptom scales at baseline and 1 year postoperatively: the Functional Assessment of Cancer Therapy-Colorectal (FACT-C) and the European Organization for the Research and Treatment of Cancer module for patients with Colorectal Cancer Quality of Life Questionnaire (EORTC QLQ-CR38). Scoring was stratified by non-randomly assigned definitive surgery (APR vs SSS). Analyses controlled for baseline scores and stratification factors: age, gender, stage, intended surgery, and randomly assigned chemoradiotherapy. Results Of 1,608 randomly assigned patients, 987 had data for planned analyses; 62% underwent SSS; 38% underwent APR. FACT-C total and subscale scores were not statistically different by surgery at one year. For the EORTC-QLQ-CR38 functional scales, APR patients reported worse body image (70.3 vs 77.0, P=0.0005) at one year than did SSS patients. Males undergoing APR reported worse sexual enjoyment (43.7 vs 54.7, P=0.02) at one year than did those undergoing SSS. For the EORTC-QLQ-CR38 symptom scale scores, APR patients reported worse micturition symptoms than the SSS group at one year (26.9 vs 21.5, P=0.03). SSS patients reported worse GI tract symptoms than did the APR patients (18.9 vs 15.2, P<0.0001), as well as weight loss (10.1 vs 6.0, P=0.002). Conclusions Symptoms and functional problems were detected at one year by EORTC-QLQ-CR38, reflecting different symptom profiles in patients who underwent APR than those who underwent SSS. Information from these PROs may be useful in counseling patients anticipating surgery for rectal cancer. PMID:24670844

  18. Efficacy, patient-reported outcomes and safety profile of ATX-101 (deoxycholic acid), an injectable drug for the reduction of unwanted submental fat: results from a phase III, randomized, placebo-controlled study

    PubMed Central

    Ascher, B; Hoffmann, K; Walker, P; Lippert, S; Wollina, U; Havlickova, B

    2014-01-01

    Background Unwanted submental fat (SMF) may result in an unattractive chin profile and dissatisfaction with appearance. An approved and rigorously tested non-surgical method for SMF reduction is lacking. Objective To evaluate the efficacy and safety of ATX-101 for the pharmacological reduction of unwanted SMF in a phase III randomized, double-blind, placebo-controlled study. Methods Patients (n = 360) with moderate or severe SMF were randomized to receive ATX-101 1 or 2 mg/cm2 or placebo injected into their SMF for up to four treatments ∼28 days apart, with a 12-week follow-up. Coprimary efficacy endpoints were the proportions of treatment responders, defined as a ≥1-point reduction in SMF on the Clinician-Reported Submental Fat Rating Scale (CR-SMFRS), and those satisfied with their appearance in association with their face and chin after treatment on the Subject Self-Rating Scale (SSRS score ≥4). Secondary efficacy endpoints included a ≥1-point improvement in SMF on the Patient-Reported Submental Fat Rating Scale (PR-SMFRS) and changes in the Patient-Reported Submental Fat Impact Scale (PR-SMFIS). Additional patient-reported outcomes and changes in the Skin Laxity Rating Scale were recorded. Adverse events (AEs) and laboratory test results were monitored. Results Compared with placebo, a greater proportion of patients treated with ATX-101 1 and 2 mg/cm2 showed a ≥1-point improvement in CR-SMFRS (58.3% and 62.3%, respectively, vs. 34.5% with placebo; P < 0.001) and patient satisfaction (SSRS score ≥4) with the appearance of their face and chin (68.3% and 64.8%, respectively, vs. 29.3%; P < 0.001). Patient-reported secondary efficacy endpoints showed significant improvements in SMF severity (PR-SMFRS; P = 0.009 for ATX-101 1 mg/cm2, P < 0.001 for ATX-101 2 mg/cm2 vs. placebo) and emotions and perceived self-image (PR-SMFIS; P < 0.001). No overall worsening of skin laxity was observed. AEs were mostly transient, mild to moderate in

  19. Comparison of Patient-Reported Outcome from Neck-Preserving, Short-Stem Arthroplasty and Resurfacing Arthroplasty in Younger Osteoarthritis Patients

    PubMed Central

    Dettmer, Marius; Pourmoghaddam, Amir; Kreuzer, Stefan W.

    2015-01-01

    Hip resurfacing has been considered a good treatment option for younger, active osteoarthritis patients. However, there are several identified issues concerning risk for neck fractures and issues related to current metal-on-metal implant designs. Neck-preserving short-stem implants have been discussed as a potential alternative, but it is yet unclear which method is better suited for younger adults. We compared hip disability and osteoarthritis outcome scores (HOOS) from a young group of patients (n = 52, age 48.9 ± 6.1 years) who had received hip resurfacing (HR) with a cohort of patients (n = 73, age 48.2 ± 6.6 years) who had received neck-preserving, short-stem implant total hip arthroplasty (THA). Additionally, durations for both types of surgery were compared. HOOS improved significantly preoperatively to last followup (>1 year) in both groups (p < 0.0001, η2 = 0.69); there were no group effects or interactions. Surgery duration was significantly longer for resurfacing (104.4 min ± 17.8) than MiniHip surgery (62.5 min ± 14.8), U = 85.0, p < 0.0001, η2 = 0.56. The neck-preserving short-stem approach may be preferable to resurfacing due to the less challenging surgery, similar outcome, and controversy regarding resurfacing implant designs. PMID:26101669

  20. Hypofractionated radiotherapy versus conventionally fractionated radiotherapy for patients with intermediate-risk localised prostate cancer: 2-year patient-reported outcomes of the randomised, non-inferiority, phase 3 CHHiP trial

    PubMed Central

    Wilkins, Anna; Mossop, Helen; Syndikus, Isabel; Khoo, Vincent; Bloomfield, David; Parker, Chris; Logue, John; Scrase, Christopher; Patterson, Helen; Birtle, Alison; Staffurth, John; Malik, Zafar; Panades, Miguel; Eswar, Chinnamani; Graham, John; Russell, Martin; Kirkbride, Peter; O'Sullivan, Joe M; Gao, Annie; Cruickshank, Clare; Griffin, Clare; Dearnaley, David; Hall, Emma

    2015-01-01

    , number ISRCTN97182923. Findings 2100 participants in the CHHiP trial consented to be included in the QoL substudy: 696 assigned to the 74 Gy schedule, 698 assigned to the 60 Gy schedule, and 706 assigned to the 57 Gy schedule. Of these individuals, 1659 (79%) provided data pre-radiotherapy and 1444 (69%) provided data at 24 months after radiotherapy. Median follow-up was 50·0 months (IQR 38·4–64·2) on April 9, 2014, which was the most recent follow-up measurement of all data collected before the QoL data were analysed in September, 2014. Comparison of 74 Gy in 37 fractions, 60 Gy in 20 fractions, and 57 Gy in 19 fractions groups at 2 years showed no overall bowel bother in 269 (66%), 266 (65%), and 282 (65%) men; very small bother in 92 (22%), 91 (22%), and 93 (21%) men; small bother in 26 (6%), 28 (7%), and 38 (9%) men; moderate bother in 19 (5%), 23 (6%), and 21 (5%) men, and severe bother in four (<1%), three (<1%) and three (<1%) men respectively (74 Gy vs 60 Gy, ptrend=0.64, 74 Gy vs 57 Gy, ptrend=0·59). We saw no differences between treatment groups in change of bowel bother score from baseline or pre-radiotherapy to 24 months. Interpretation The incidence of patient-reported bowel symptoms was low and similar between patients in the 74 Gy control group and the hypofractionated groups up to 24 months after radiotherapy. If efficacy outcomes from CHHiP show non-inferiority for hypofractionated treatments, these findings will add to the growing evidence for moderately hypofractionated radiotherapy schedules becoming the standard treatment for localised prostate cancer. Funding Cancer Research UK, Department of Health, and the National Institute for Health Research Cancer Research Network. PMID:26522334

  1. Functional outcome measures for NF1-associated optic pathway glioma clinical trials

    PubMed Central

    Avery, Robert A.; Allen, Jeffrey C.; Ardern-Holmes, Simone L.; Bilaniuk, Larissa T.; Ferner, Rosalie E.; Gutmann, David H.; Listernick, Robert; Martin, Staci; Ullrich, Nicole J.; Liu, Grant T.

    2013-01-01

    Objective: The goal of the Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee is to define the best functional outcome measures for future neurofibromatosis type 1 (NF1)-associated optic pathway glioma (OPG) clinical trials. Methods: The committee considered the components of vision, other ophthalmologic parameters affected by OPG, potential biomarkers of visual function, and quality of life measures to arrive at consensus-based, evidence-driven recommendations for objective and measurable functional endpoints for OPG trials. Results: Visual acuity (VA) assessments using consistent quantitative testing methods are recommended as the main functional outcome measure for NF1-OPG clinical trials. Teller acuity cards are recommended for use as the primary VA endpoint, and HOTV as a secondary endpoint once subjects are old enough to complete it. The optic disc should be assessed for pallor, as this appears to be a contributory variable that may affect the interpretation of VA change over time. Given the importance of capturing patient-reported outcomes in clinical trials, evaluating visual quality of life using the Children's Visual Function Questionnaire as a secondary endpoint is also proposed. Conclusions: The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials. PMID:24249802

  2. Validity and reliability of the Patient-Reported Arthralgia Inventory: validation of a newly-developed survey instrument to measure arthralgia

    PubMed Central

    Castel, Liana D; Wallston, Kenneth A; Saville, Benjamin R; Alvarez, JoAnn R; Shields, Bradley D; Feurer, Irene D; Cella, David

    2015-01-01

    Background There is a need for a survey instrument to measure arthralgia (joint pain) that has been psychometrically validated in the context of existing reference instruments. We developed the 16-item Patient-Reported Arthralgia Inventory (PRAI) to measure arthralgia severity in 16 joints, in the context of a longitudinal cohort study to assess aromatase inhibitor-associated arthralgia in breast cancer survivors and arthralgia in postmenopausal women without breast cancer. We sought to evaluate the reliability and validity of the PRAI instrument in these populations, as well as to examine the relationship of patient-reported morning stiffness and arthralgia. Methods We administered the PRAI on paper in 294 women (94 initiating aromatase inhibitor therapy and 200 postmenopausal women without breast cancer) at weeks 0, 2, 4, 6, 8, 12, 16, and 52, as well as once in 36 women who had taken but were no longer taking aromatase inhibitor therapy. Results Cronbach’s alpha was 0.9 for internal consistency of the PRAI. Intraclass correlation coefficients of test-retest reliability were in the range of 0.87–0.96 over repeated PRAI administrations; arthralgia severity was higher in the non-cancer group at baseline than at subsequent assessments. Women with joint comorbidities tended to have higher PRAI scores than those without (estimated difference in mean scores: −0.3, 95% confidence interval [CI] −0.5, −0.2; P<0.001). The PRAI was highly correlated with the Functional Assessment of Cancer Therapy-Endocrine Subscale item “I have pain in my joints” (reference instrument; Spearman r range: 0.76–0.82). Greater arthralgia severity on the PRAI was also related to decreased physical function (r=−0.47, 95% CI −0.55, −0.37; P<0.001), higher pain interference (r=0.65, 95% CI 0.57–0.72; P<0.001), less active performance status (estimated difference in location (−0.6, 95% CI −0.9, −0.4; P<0.001), and increased morning stiffness duration (r=0.62, 95% CI 0

  3. MEASURING AND IMPROVING RESPIRATORY OUTCOMES IN CYSTIC FIBROSIS LUNG DISEASE: OPPORTUNITIES AND CHALLENGES TO THERAPY

    PubMed Central

    Zemanick, Edith T.; Harris, J. Kirk; Conway, Steven; Konstan, Michael W.; Marshall, Bruce; Quittner, Alexandra L.; Retsch-Bogart, George; Saiman, Lisa; Accurso, Frank J.

    2010-01-01

    Cystic fibrosis (CF) is a life-shortening disease with significant morbidity. Despite overall improvements in survival, patients with CF experience frequent pulmonary exacerbations and declining lung function, which often accelerates during adolescence. New treatments target steps in the pathogenesis of lung disease, such as the basic defect in CF (CF Transmembrane Conductance Regulator [CFTR]), pulmonary infections, inflammation, and mucociliary clearance. These treatments offer hope but also present challenges to patients, clinicians, and researchers. Comprehensive assessment of efficacy is critical to identify potentially beneficial treatments. Lung function and pulmonary exacerbation are the most commonly used outcome measures in CF clinical research. Other outcome measures under investigation include measures of CFTR function; biomarkers of infection, inflammation, lung injury and repair; and patient-reported outcomes. Molecular diagnostics may help elucidate the complex CF airway microbiome. As new treatments are developed for patients with CF, efforts should be made to balance treatment burden with quality of life. This review highlights emerging treatments, obstacles to optimizing outcomes, and key future directions for research. PMID:19833563

  4. Clinically Meaningful Differences in Patient-Reported Outcomes With Amifostine in Combination With Chemoradiation for Locally Advanced Non-Small-Cell Lung Cancer: An Analysis of RTOG 9801

    SciTech Connect

    Sarna, Linda Swann, Suzanne; Langer, Corey; Werner-Wasik, Maria; Nicolaou, Nicos; Komaki, Ritsuko; Machtay, Mitchell; Byhardt, Roger; Wasserman, Todd; Movsas, Benjamin

    2008-12-01

    Purpose: The purpose of this study is to analyze changes in quality of life (QOL) and symptoms from pretreatment to 6 weeks posttreatment in a Phase III randomized study (Radiation Therapy Oncology Group 9801) of amifostine (AM) vs. no AM in patients with Stages II-III non-small-cell lung cancer receiving paclitaxel and carboplatin as induction and then concurrently with hyperfractionated radiation therapy (RT). Methods and Materials: One hundred thirty-eight patients with baseline and 6-week posttreatment QOL data were analyzed. There were no significant differences in baseline demographics between those who did and did not have QOL data. The QOL and symptoms were assessed by using the European Organization for Research and Treatment of Cancer (EORTC) Global QOL and Pain subscales and the EORTC-Lung Cancer-13 symptom tool. Clinically relevant changes in QOL were characterized by 10-point differences in individual scores pre/post treatment. A daily diary of patient-rated difficulty swallowing and a weekly physician-rated dysphagia log (using National Cancer Institute Common Toxicity Criteria) were completed during treatment. Weight loss was monitored. Differences in outcomes were examined according to smoking status, alcohol use, and sex. Results: Patients receiving AM reported significantly greater pain reduction after chemoradiation (34% vs. no AM, 21%), less difficulty swallowing during chemoradiation, and less weight loss than patients not receiving AM. However, physician-rated assessments of dysphagia were not significantly different by treatment arm. There were no other significant changes in QOL or symptoms according to treatment arm, smoking status, alcohol use, or sex. Conclusions: Patient evaluations of difficulty swallowing and pain suggest benefits from AM use that are distinct from clinician-rated assessments.

  5. Measuring Program Outcomes: Using Retrospective Pretest Methodology.

    ERIC Educational Resources Information Center

    Pratt, Clara C.; McGuigan, William M.; Katsev, Aphra R.

    2000-01-01

    Used longitudinal data from 307 mothers of firstborn infants participating in a home-visitation, child abuse prevention program in a retrospective pretest methodology. Results shows that when response shift bias was present, the retrospective pretest methodology produced a more legitimate assessment of program outcomes than did the traditional…

  6. Toxicities Affecting Quality of Life After Chemo-IMRT of Oropharyngeal Cancer: Prospective Study of Patient-Reported, Observer-Rated, and Objective Outcomes

    SciTech Connect

    Hunter, Klaudia U.; Schipper, Matthew; Feng, Felix Y.; Lyden, Teresa; Haxer, Mark; Murdoch-Kinch, Carol-Anne; Cornwall, Benjamin; Lee, Connie S.Y.; Chepeha, Douglas B.; Eisbruch, Avraham

    2013-03-15

    Purpose: To test the hypothesis that intensity modulated radiation therapy (IMRT) aiming to spare the salivary glands and swallowing structures would reduce or eliminate the effects of xerostomia and dysphagia on quality of life (QOL). Methods and Materials: In this prospective, longitudinal study, 72 patients with stage III-IV oropharyngeal cancer were treated uniformly with definitive chemo-IMRT sparing the salivary glands and swallowing structures. Overall QOL was assessed by summary scores of the Head Neck QOL (HNQOL) and University of Washington QOL (UWQOL) questionnaires, as well as the HNQOL “Overall Bother” question. Quality of life, observer-rated toxicities (Common Toxicity Criteria Adverse Effects scale, version 2), and objective evaluations (videofluoroscopy assessing dysphagia and saliva flow rates assessing xerostomia) were recorded from before therapy through 2 years after therapy. Correlations between toxicities/objective evaluations and overall QOL were assessed using longitudinal repeated measures of analysis and Pearson correlations. Results: All observer-rated toxicities and QOL scores worsened 1-3 months after therapy and improved through 12 months, with minor further improvements through 24 months. At 12 months, dysphagia grades 0-1, 2, and 3, were observed in 95%, 4%, and 1% of patients, respectively. Using all posttherapy observations, observer-rated dysphagia was highly correlated with all overall QOL measures (P<.0001), whereas xerostomia and mucosal and voice toxicities were significantly correlated with some, but not all, overall QOL measures, with lower correlation coefficients than dysphagia. Late overall QOL (≥6 or ≥12 months after therapy) was primarily associated with observer-rated dysphagia, and to a lesser extent with xerostomia. Videofluoroscopy scores, but not salivary flows, were significantly correlated with some of the overall QOL measures. Conclusion: After chemo-IMRT, although late dysphagia was on average mild

  7. Update on Outcome Measure Development for Large Vessel Vasculitis: Report from OMERACT 12

    PubMed Central

    Aydin, Sibel Zehra; Direskeneli, Haner; Sreih, Antoine; Alibaz-Oner, Fatma; Gul, Ahmet; Kamali, Sevil; Hatemi, Gulen; Kermani, Tanaz; Mackie, Sarah L.; Mahr, Alfred; Meara, Alexa; Milman, Nataliya; Nugent, Heidi; Robson, Joanna; Tomasson, Gunnar; Merkel, Peter A.

    2015-01-01

    Objective The rarity of large vessel vasculitis (LVV) is a major factor limiting randomized controlled trials in LVV, resulting in treatment choices in these diseases that are guided mainly by observational studies and expert opinion. Further complicating trials in LVV is the absence of validated and meaningful outcome measures. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group initiated the Large Vessel Vasculitis task force in 2009 to develop data-driven, validated outcome tools for clinical investigation in LVV. This report summarizes the progress that has been made on a disease activity assessment tool and patient-reported outcomes in LVV as well as the group’s research agenda. Methods The OMERACT LVV task force brought an international group of investigators and patient research partners together to work collaboratively on developing outcome tools. The group initially focused on disease activity assessment tools in LVV. Following a systematic literature review, an international Delphi exercise was conducted to obtain expert opinion on principles and domains for disease assessment. The OMERACT vasculitis working group’s LVV task force is also conducting qualitative research with patients, including interviews, focus groups, and engaging patients as research partners, all to ensure that the approach to disease assessment includes measures of patients’ perspectives and that patients have input into the research agenda and process. Results The preliminary results of both the Delphi exercise and the qualitative interviews were discussed at the OMERACT 12 (2014) meeting and the completion of the analyses will produce an initial set of domains and instruments to form the basis of next steps in the research agenda. Conclusion The research agenda continues to evolve, with the ultimate goal of developing an OMERACT-endorsed core set of outcome measures for use in clinical trials of LVV. PMID:26077399

  8. Learning Outcomes across Disciplines and Professions: Measurement and Interpretation

    ERIC Educational Resources Information Center

    Caspersen, Joakim; Frølich, Nicoline; Karlsen, Hilde; Aamodt, Per Olaf

    2014-01-01

    Learning outcomes of higher education are a quality tool in a changing higher education landscape but cannot be seen as neutral measures across professions and disciplines. Survey results from graduates and recent graduates indicate that prevailing measures of learning outcomes yield the same result within and across disciplinary and professional…

  9. Sensitivity of Outcome Measures for Treatments of Generalized Social Phobia.

    ERIC Educational Resources Information Center

    Taylor, Steven; Woody, Sheila; McLean, Peter D.; Koch, William J.

    1997-01-01

    The sensitivity of five measures of outcomes of treatment for generalized social phobia was studied with 60 people diagnosed with generalized social phobia. Outcome measures were completed before and after treatment and three months later, and effect sizes were computed. Results support the usefulness of the Social Phobia and Anxiety Inventory (S.…

  10. Collagenase clostridium histolyticum in patients with Dupuytren’s contracture: results from POINT X, an open-label study of clinical and patient-reported outcomes

    PubMed Central

    Arner, M.; Pajardi, G.; Reichert, B.; Szabo, Z.; Masmejean, E. H.; Fores, J.; Chapman, D. S.; Gerber, R. A.; Huard, F.; Seghouani, A.; Szczypa, P. P.

    2015-01-01

    In POINT X, a study designed to reflect clinical practice and patient treatment choices, 254 European patients received open-label collagenase for Dupuytren’s contracture. The most severely affected joint was treated first in 74% of patients. In total, 52%, 41%, 7%, and 1% of patients selected the little, ring, middle, and index finger, respectively; 79% had one or two joints treated. Only 9% of patients (n = 24) received 4 or 5 injections. The mean improvement in total passive extension deficit (TPED) was 34° on day 1, improving further by day 7 to 42°. This secondary improvement was maintained by day 90 and month 6. The mean number of injections/joint was 1.2 for the metacarpophalangeal joint and 1.25 for the proximal interphalangeal joint. Median time to recovery was 4 days; the mean improvement in hand function was clinically relevant as measured by the Unité Rhumatologique des Affections de la Main (URAM) score. In total, 87% and 86% of patients and physicians, respectively, were very satisfied or satisfied with treatment at month 6, although correlation between TPED and patient satisfaction was weak (Spearman −0.18, 95% CI −0.32 to −0.06). Collagenase was well tolerated, with 10 (3.9%) patients experiencing severe adverse events. As a real-world study, the POINT X findings can be generalized to the at-large population. PMID:24470559

  11. Treatment for Osteoporosis among Women in Japan: Associations with Patient Characteristics and Patient-Reported Outcomes in the 2008–2011 Japan National Health and Wellness Surveys

    PubMed Central

    Flynn, Jennifer A.; Fujiwara, Saeko

    2014-01-01

    This study was conducted to identify characteristics associated with treatment for osteoporosis among women aged 50 years and older in Japan and to explore differences among patients according to treatment regimen. Data were provided by a large annual survey representative of Japanese aged 18 and older; all measures were by self-report. Women aged 50 and older who reported diagnosed osteoporosis (N = 900) were compared based on current treatment status using bivariate statistics and logistic regression. Approximately 1 in 3 women in this study reporting diagnosed osteoporosis were currently untreated. Factors associated with current treatment for osteoporosis included having ≥1 physician visit in the prior 6 months (OR = 5.4, P < 0.001), self-rated moderate or severe osteoporosis (OR = 2.8, P < 0.001), completion of menopause (OR = 1.6, P < 0.05), and family history of osteoporosis (OR = 1.5, P < 0.05), while longer duration of osteoporosis diagnosis (OR = 0.9, P < 0.05) and arthritis (OR = 0.7, P < 0.05) were associated with lower odds of treatment. These findings suggest that diagnosed patients are not being actively managed in the longer term, and efforts need to be made to ensure that patients stay engaged with their healthcare providers. PMID:25587485

  12. Generating Outcome Measurements: Economic and Societal. A Guide to Educational Outcomes Measurements and Their Uses. Seminar No. 4.

    ERIC Educational Resources Information Center

    Mushkin, Selma J.; Billings, Bradley B.

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministires, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with generating outcome measurements:…

  13. The Harmonizing Outcome Measures for Eczema (HOME) roadmap: a methodological framework to develop core sets of outcome measurements in dermatology.

    PubMed

    Schmitt, Jochen; Apfelbacher, Christian; Spuls, Phyllis I; Thomas, Kim S; Simpson, Eric L; Furue, Masutaka; Chalmers, Joanne; Williams, Hywel C

    2015-01-01

    Core outcome sets (COSs) are consensus-derived minimum sets of outcomes to be assessed in a specific situation. COSs are being increasingly developed to limit outcome-reporting bias, allow comparisons across trials, and strengthen clinical decision making. Despite the increasing interest in outcomes research, methods to develop COSs have not yet been standardized. The aim of this paper is to present the Harmonizing Outcomes Measures for Eczema (HOME) roadmap for the development and implementation of COSs, which was developed on the basis of our experience in the standardization of outcome measurements for atopic eczema. Following the establishment of a panel representing all relevant stakeholders and a research team experienced in outcomes research, the scope and setting of the core set should be defined. The next steps are the definition of a core set of outcome domains such as symptoms or quality of life, followed by the identification or development and validation of appropriate outcome measurement instruments to measure these core domains. Finally, the consented COS needs to be disseminated, implemented, and reviewed. We believe that the HOME roadmap is a useful methodological framework to develop COSs in dermatology, with the ultimate goal of better decision making and promoting patient-centered health care. PMID:25186228

  14. Using Learning Outcome Measures to assess Doctoral Nursing Education

    PubMed Central

    Raup, Glenn H.; King, Jeff; Hughes, Romana J.; Faidley, Natasha

    2010-01-01

    Education programs at all levels must be able to demonstrate successful program outcomes. Grades alone do not represent a comprehensive measurement methodology for assessing student learning outcomes at either the course or program level. The development and application of assessment rubrics provides an unequivocal measurement methodology to ensure a quality learning experience by providing a foundation for improvement based on qualitative and quantitatively measurable, aggregate course and program outcomes. Learning outcomes are the embodiment of the total learning experience and should incorporate assessment of both qualitative and quantitative program outcomes. The assessment of qualitative measures represents a challenge for educators in any level of a learning program. Nursing provides a unique challenge and opportunity as it is the application of science through the art of caring. Quantification of desired student learning outcomes may be enhanced through the development of assessment rubrics designed to measure quantitative and qualitative aspects of the nursing education and learning process. They provide a mechanism for uniform assessment by nursing faculty of concepts and constructs that are otherwise difficult to describe and measure. A protocol is presented and applied to a doctoral nursing education program with recommendations for application and transformation of the assessment rubric to other education programs. Through application of these specially designed rubrics, all aspects of an education program can be adequately assessed to provide information for program assessment that facilitates the closure of the gap between desired and actual student learning outcomes for any desired educational competency. PMID:20567217

  15. Measuring Outcomes for Children Late Placed for Adoption.

    ERIC Educational Resources Information Center

    Rushton, Alan

    1998-01-01

    Describes the selection of outcome measures used by the Maudsley Family Research team to assess outcomes--across a broad range of developmental dimensions--of permanent placement for children and adolescents. Developed a package of instruments to examine child emotional, cognitive, social, and academic development; attachment; and self-esteem, for…

  16. Measuring Assistive Technology Outcomes in Schools Using Functional Assessment.

    ERIC Educational Resources Information Center

    Silverman, Michelle Kaye; Stratman, Kristine Freiberg; Smith, Roger O.

    2000-01-01

    Activities of Project OATS (Outcomes of Assistive Technology in the Schools) are described, including identification and piloting of existing assessment instruments for use as an outcome measure, examining the validity of the School Function Assessment, and field testing the School Function Assessment-Assistive Technology Version, an adaptation of…

  17. Outcome Measures in Quality of Life.

    PubMed

    Tate, D G; Dijkers, M; Johnson-Greene, L

    1996-01-01

    The concept of quality of life (QOL) represents a new paradigm in rehabilitation research and cSinical care. Three measurement approaches have been used to investigate QOL of individuals: (1) evaluative or subjective, (2) objective, and (3) quality adjusted life year (QALY). QALYs represent the utility approach to measurement with emphasis on value of, desirability of, or preference for conditions or status. When the condition in question isdefined as health, health-related quality of life (HQOL) measures are employed. Very few studies are found in the literature addressing QOL of stroke patients. Among studies reviewed, HQOL measures are often used. A summary of these studies and discussion of limitations associated with QOL measures utilized are provided. PMID:27620148

  18. Validation of electronic systems to collect patient-reported outcome (PRO) data-recommendations for clinical trial teams: report of the ISPOR ePRO systems validation good research practices task force.

    PubMed

    Zbrozek, Arthur; Hebert, Joy; Gogates, Gregory; Thorell, Rod; Dell, Christopher; Molsen, Elizabeth; Craig, Gretchen; Grice, Kenneth; Kern, Scottie; Hines, Sheldon

    2013-06-01

    Outcomes research literature has many examples of high-quality, reliable patient-reported outcome (PRO) data entered directly by electronic means, ePRO, compared to data entered from original results on paper. Clinical trial managers are increasingly using ePRO data collection for PRO-based end points. Regulatory review dictates the rules to follow with ePRO data collection for medical label claims. A critical component for regulatory compliance is evidence of the validation of these electronic data collection systems. Validation of electronic systems is a process versus a focused activity that finishes at a single point in time. Eight steps need to be described and undertaken to qualify the validation of the data collection software in its target environment: requirements definition, design, coding, testing, tracing, user acceptance testing, installation and configuration, and decommissioning. These elements are consistent with recent regulatory guidance for systems validation. This report was written to explain how the validation process works for sponsors, trial teams, and other users of electronic data collection devices responsible for verifying the quality of the data entered into relational databases from such devices. It is a guide on the requirements and documentation needed from a data collection systems provider to demonstrate systems validation. It is a practical source of information for study teams to ensure that ePRO providers are using system validation and implementation processes that will ensure the systems and services: operate reliably when in practical use; produce accurate and complete data and data files; support management control and comply with any existing regulations. Furthermore, this short report will increase user understanding of the requirements for a technology review leading to more informed and balanced recommendations or decisions on electronic data collection methods. PMID:23796281

  19. The Development of NOAA Education Common Outcome Performance Measures (Invited)

    NASA Astrophysics Data System (ADS)

    Baek, J.

    2013-12-01

    The National Oceanic and Atmospheric Administration (NOAA) Education Council has embarked on an ambitious Monitoring and Evaluation (M&E) project that will allow it to assess education program outcomes and impacts across the agency, line offices, and programs. The purpose of this internal effort is to link outcome measures to program efforts and to evaluate the success of the agency's education programs in meeting the strategic goals. Using an outcome-based evaluation approach, the NOAA Education Council is developing two sets of common outcome performance measures, environmental stewardship and professional development. This presentation will examine the benefits and tradeoffs of common outcome performance measures that collect program results across a portfolio of education programs focused on common outcomes. Common outcome performance measures have a few benefits to our agency and to the climate education field at large. The primary benefit is shared understanding, which comes from our process for writing common outcome performance measures. Without a shared and agreed upon set of definitions for the measure of an outcome, the reported results may not be measuring the same things and would incorrectly indicate levels of performance. Therefore, our writing process relies on a commitment to developing a shared set of definitions based on consensus. We hope that by taking the time to debate and coming to agreement across a diverse set of programs, the strength of our common measures can indicate real progress towards outcomes we care about. An additional benefit is that these common measures can be adopted and adapted by other agencies and organizations that share similar theories of change. The measures are not without their drawbacks, and we do make tradeoffs as part of our process in order to continue making progress. We know that any measure is necessarily a narrow slice of performance. A slice that may not best represent the unique and remarkable contribution

  20. Accuracy of patient-reported range of elbow motion

    PubMed Central

    Robinson, Paul M; van Rensburg, Lee

    2016-01-01

    Background Patient-reported outcome meaures (PROMs) not only provide valuable insights into subjective indices of joint health, but also may provide limited objective information about range of motion (ROM). We sought to evaluate the accuracy of patient-reported range of elbow motion compared to measured ROM. Methods Sixty clinic patients were recruited, of whom 26 had elbow pathologies and 34 had pathologies other than at the elbow joint. Each patient independently estimated ROM for extension, flexion, pronation and supination before this was measured by a clinician using a universal goniometer, with the mean being the gold standard. Results We found that patients’ ROM estimates were significantly different from measured ROM (p < 0.00001 at 95% confidence interval). There was no statistically significant difference between elbow pathology and non-elbow pathology patients’ estimated ROM. Conclusions There was great disparity between patient-estimated and measured ROM, although estimates of patients with known elbow pathology did not demonstrate any significant difference from their healthy counterparts. These differences may be too great for patient-estimated range of motion to be used as a reliable tool for assessing outcomes.

  1. Outcome measures in neuromuscular disease: is the world still flat?

    PubMed

    Lunn, Michael P; Van den Bergh, Peter Y K

    2015-09-01

    Valid, responsive, and meaningful outcome measures for the measurement of the impairment, activity limitations, and quality of life in patients with neuromuscular disease are crucial to identify the natural history of disease and benefits of therapy in clinical practice and trials. Although understanding of many aspects of neuromuscular diseases has advanced dramatically, the development of outcome measures has received less attention. The scales developed from Rasch theory by the PeriNomS Group represent the biggest significant shift in thought in neuromuscular outcome measures for decades. There remain problems with many of them, and further developments are required. However, incorporating them into our outcome sets for daily use and in clinical trials will lead to the more efficient capture of meaningful change and will result in better assessment of individuals and groups of patients in both clinical trials and neurological practice. PMID:26114965

  2. Palliative care outcome measures in COPD patients: a conceptual review.

    PubMed

    Antoniu, Sabina Antonela; Boiculese, Lucian Vasile

    2016-04-01

    In chronic obstructive pulmonary disease (COPD), palliative care is appropriate in very advanced stages based on recognition of its need when conventional therapy is no longer able to control symptoms, disease morbidity, or to improve/maintain an acceptable quality of life. Palliative care aims to improve quality of life, or, if applied specifically at the end-of-life, to ensure comfortable care. In COPD palliative care effectiveness of interventions should be quantified with outcome measures able to better capture the holistic nature of approaches and not only the specific features of disease. These should include: physical outcomes, psychological outcomes, social outcomes, spiritual outcomes. Such measures are discussed in this review along with arguments supporting their use. PMID:26967768

  3. Report from the fourth international consensus meeting to harmonize core outcome measures for atopic eczema/dermatitis clinical trials (HOME initiative).

    PubMed

    Chalmers, J R; Simpson, E; Apfelbacher, C J; Thomas, K S; von Kobyletzki, L; Schmitt, J; Singh, J A; Svensson, Å; Williams, H C; Abuabara, K; Aoki, V; Ardeleanu, M; Awici-Rasmussen, M; Barbarot, S; Berents, T L; Block, J; Bragg, A; Burton, T; Bjerring Clemmensen, K K; Creswell-Melville, A; Dinesen, M; Drucker, A; Eckert, L; Flohr, C; Garg, M; Gerbens, L A A; Graff, A L B; Hanifin, J; Heinl, D; Humphreys, R; Ishii, H A; Kataoka, Y; Leshem, Y A; Marquort, B; Massuel, M-A; Merhand, S; Mizutani, H; Murota, H; Murrell, D F; Nakahara, T; Nasr, I; Nograles, K; Ohya, Y; Osterloh, I; Pander, J; Prinsen, C; Purkins, L; Ridd, M; Sach, T; Schuttelaar, M-L A; Shindo, S; Smirnova, J; Sulzer, A; Synnøve Gjerde, E; Takaoka, R; Vestby Talmo, H; Tauber, M; Torchet, F; Volke, A; Wahlgren, C-F; Weidinger, S; Weisshaar, E; Wollenberg, A; Yamaga, K; Zhao, C Y; Spuls, P I

    2016-07-01

    This article is a report of the fourth meeting of the Harmonising Outcome Measures for Eczema (HOME) initiative held in Malmö, Sweden on 23-24 April 2015 (HOME IV). The aim of the meeting was to achieve consensus over the preferred outcome instruments for measuring patient-reported symptoms and quality of life for the HOME core outcome set for atopic eczema (AE). Following presentations, which included data from systematic reviews, consensus discussions were held in a mixture of whole group and small group discussions. Small groups were allocated a priori to ensure representation of different stakeholders and countries. Decisions were voted on using electronic keypads. For the patient-reported symptoms, the group agreed by vote that itch, sleep loss, dryness, redness/inflamed skin and irritated skin were all considered essential aspects of AE symptoms. Many instruments for capturing patient-reported symptoms were discussed [including the Patient-Oriented SCOring Atopic Dermatitis index, Patient-Oriented Eczema Measure (POEM), Self-Administered Eczema Area and Severity Index, Itch Severity Scale, Atopic Dermatitis Quickscore and the Nottingham Eczema Severity Score] and, by consensus, POEM was selected as the preferred instrument to measure patient-reported symptoms. Further work is needed to determine the reliability and measurement error of POEM. Further work is also required to establish the importance of pain/soreness and the importance of collecting information regarding the intensity of symptoms in addition to their frequency. Much of the discussion on quality of life concerned the Dermatology Life Quality Index and Quality of Life Index for Atopic Dermatitis; however, consensus on a preferred instrument for measuring this domain could not be reached. In summary, POEM is recommended as the HOME core outcome instrument for measuring AE symptoms. PMID:27436240

  4. Methodological issues regarding power of classical test theory (CTT) and item response theory (IRT)-based approaches for the comparison of patient-reported outcomes in two groups of patients - a simulation study

    PubMed Central

    2010-01-01

    Background Patients-Reported Outcomes (PRO) are increasingly used in clinical and epidemiological research. Two main types of analytical strategies can be found for these data: classical test theory (CTT) based on the observed scores and models coming from Item Response Theory (IRT). However, whether IRT or CTT would be the most appropriate method to analyse PRO data remains unknown. The statistical properties of CTT and IRT, regarding power and corresponding effect sizes, were compared. Methods Two-group cross-sectional studies were simulated for the comparison of PRO data using IRT or CTT-based analysis. For IRT, different scenarios were investigated according to whether items or person parameters were assumed to be known, to a certain extent for item parameters, from good to poor precision, or unknown and therefore had to be estimated. The powers obtained with IRT or CTT were compared and parameters having the strongest impact on them were identified. Results When person parameters were assumed to be unknown and items parameters to be either known or not, the power achieved using IRT or CTT were similar and always lower than the expected power using the well-known sample size formula for normally distributed endpoints. The number of items had a substantial impact on power for both methods. Conclusion Without any missing data, IRT and CTT seem to provide comparable power. The classical sample size formula for CTT seems to be adequate under some conditions but is not appropriate for IRT. In IRT, it seems important to take account of the number of items to obtain an accurate formula. PMID:20338031

  5. Outcome Measures for Artificial Pancreas Clinical Trials: A Consensus Report.

    PubMed

    Maahs, David M; Buckingham, Bruce A; Castle, Jessica R; Cinar, Ali; Damiano, Edward R; Dassau, Eyal; DeVries, J Hans; Doyle, Francis J; Griffen, Steven C; Haidar, Ahmad; Heinemann, Lutz; Hovorka, Roman; Jones, Timothy W; Kollman, Craig; Kovatchev, Boris; Levy, Brian L; Nimri, Revital; O'Neal, David N; Philip, Moshe; Renard, Eric; Russell, Steven J; Weinzimer, Stuart A; Zisser, Howard; Lum, John W

    2016-07-01

    Research on and commercial development of the artificial pancreas (AP) continue to progress rapidly, and the AP promises to become a part of clinical care. In this report, members of the JDRF Artificial Pancreas Project Consortium in collaboration with the wider AP community 1) advocate for the use of continuous glucose monitoring glucose metrics as outcome measures in AP trials, in addition to HbA1c, and 2) identify a short set of basic, easily interpreted outcome measures to be reported in AP studies whenever feasible. Consensus on a broader range of measures remains challenging; therefore, reporting of additional metrics is encouraged as appropriate for individual AP studies or study groups. Greater consistency in reporting of basic outcome measures may facilitate the interpretation of study results by investigators, regulatory bodies, health care providers, payers, and patients themselves, thereby accelerating the widespread adoption of AP technology to improve the lives of people with type 1 diabetes. PMID:27330126

  6. Report from the third international consensus meeting to harmonise core outcome measures for atopic eczema/dermatitis clinical trials (HOME).

    PubMed

    Chalmers, J R; Schmitt, J; Apfelbacher, C; Dohil, M; Eichenfield, L F; Simpson, E L; Singh, J; Spuls, P; Thomas, K S; Admani, S; Aoki, V; Ardeleanu, M; Barbarot, S; Berger, T; Bergman, J N; Block, J; Borok, N; Burton, T; Chamlin, S L; Deckert, S; DeKlotz, C C; Graff, L B; Hanifin, J M; Hebert, A A; Humphreys, R; Katoh, N; Kisa, R M; Margolis, D J; Merhand, S; Minnillo, R; Mizutani, H; Nankervis, H; Ohya, Y; Rodgers, P; Schram, M E; Stalder, J F; Svensson, A; Takaoka, R; Teper, A; Tom, W L; von Kobyletzki, L; Weisshaar, E; Zelt, S; Williams, H C

    2014-12-01

    This report provides a summary of the third meeting of the Harmonising Outcome Measures for Eczema (HOME) initiative held in San Diego, CA, U.S.A., 6-7 April 2013 (HOME III). The meeting addressed the four domains that had previously been agreed should be measured in every eczema clinical trial: clinical signs, patient-reported symptoms, long-term control and quality of life. Formal presentations and nominal group techniques were used at this working meeting, attended by 56 voting participants (31 of whom were dermatologists). Significant progress was made on the domain of clinical signs. Without reference to any named scales, it was agreed that the intensity and extent of erythema, excoriation, oedema/papulation and lichenification should be included in the core outcome measure for the scale to have content validity. The group then discussed a systematic review of all scales measuring the clinical signs of eczema and their measurement properties, followed by a consensus vote on which scale to recommend for inclusion in the core outcome set. Research into the remaining three domains was presented, followed by discussions. The symptoms group and quality of life groups need to systematically identify all available tools and rate the quality of the tools. A definition of long-term control is needed before progress can be made towards recommending a core outcome measure. PMID:24980543

  7. Are validated outcome measures used in distal radial fractures truly valid?

    PubMed Central

    Nienhuis, R. W.; Bhandari, M.; Goslings, J. C.; Poolman, R. W.; Scholtes, V. A. B.

    2016-01-01

    Objectives Patient-reported outcome measures (PROMs) are often used to evaluate the outcome of treatment in patients with distal radial fractures. Which PROM to select is often based on assessment of measurement properties, such as validity and reliability. Measurement properties are assessed in clinimetric studies, and results are often reviewed without considering the methodological quality of these studies. Our aim was to systematically review the methodological quality of clinimetric studies that evaluated measurement properties of PROMs used in patients with distal radial fractures, and to make recommendations for the selection of PROMs based on the level of evidence of each individual measurement property. Methods A systematic literature search was performed in PubMed, EMbase, CINAHL and PsycINFO databases to identify relevant clinimetric studies. Two reviewers independently assessed the methodological quality of the studies on measurement properties, using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist. Level of evidence (strong / moderate / limited / lacking) for each measurement property per PROM was determined by combining the methodological quality and the results of the different clinimetric studies. Results In all, 19 out of 1508 identified unique studies were included, in which 12 PROMs were rated. The Patient-rated wrist evaluation (PRWE) and the Disabilities of Arm, Shoulder and Hand questionnaire (DASH) were evaluated on most measurement properties. The evidence for the PRWE is moderate that its reliability, validity (content and hypothesis testing), and responsiveness are good. The evidence is limited that its internal consistency and cross-cultural validity are good, and its measurement error is acceptable. There is no evidence for its structural and criterion validity. The evidence for the DASH is moderate that its responsiveness is good. The evidence is limited that its reliability and the

  8. International Dermatology Outcome Measures Initiative as Applied to Psoriatic Disease Outcomes: An Update.

    PubMed

    Merola, Joseph F; Armstrong, April W; Saraiya, Ami; Latella, John; Garg, Amit; Callis Duffin, Kristina; Gottlieb, Alice B

    2016-05-01

    Previous publications have described the International Dermatology Outcome Measures (IDEOM) group, comprising patients, physicians, health economists, participating pharmaceutical industry partners, payers, and regulatory agencies. The goal of IDEOM is to create patient-centered, validated measures of dermatologic disease progression and treatment efficacy for use in both clinical trials and clinical practice. We provide an update of IDEOM activities as of our 2015 IDEOM meeting in Washington, DC, USA. PMID:27134269

  9. Measuring Student Learning Outcomes Using the SALG Instrument

    ERIC Educational Resources Information Center

    Scholl, Kathleen; Olsen, Heather M.

    2014-01-01

    U.S. higher education institutions are being called to question their central nature, priorities, and functions, with prominent and unprecedented attention being given to accountability and the measurement of student learning outcomes. As higher education evolves in how it assesses student learning and leisure studies and recreation departments…

  10. What Do They Measure? Comparing Three Learning Outcomes Assessments

    ERIC Educational Resources Information Center

    Steedle, Jeffrey; Kugelmass, Heather; Nemeth, Alex

    2010-01-01

    Many postsecondary institutions currently administer standardized tests of general college outcomes; more than a quarter of Association of American Colleges and Universities (AAC&U) member institutions do so. Using standardized tests for accountability purposes has been contentious mainly because these tests do not measure every important outcome…

  11. Conceptualizing Outcome and Impact Measures for Intelligence Services

    ERIC Educational Resources Information Center

    Gainor, Rhiannon; Bouthillier, France

    2014-01-01

    Introduction: The purpose of this qualitative, exploratory study is to clarify ambiguous concepts in intelligence services literature specifically related to measurement of intelligence outcomes and impact. Method: Face to face interviews were held with five subject experts from various intelligence fields and countries regarding their…

  12. Use of Outcome Measurement by Paediatric AHPs in Northern Ireland

    ERIC Educational Resources Information Center

    Harron, Anita; Titterington, Jill

    2016-01-01

    Background: Professional standards advocate routine use of outcome measurement (OM) in the practice of allied health professionals (AHPs). Historically, OM has focused on impairment and its immediate constraints on activity, while current policy encourages the development and addition of impact-based OM. There appears to be an assumption at this…

  13. Measuring Educational Outcomes: Vocational Education and Training. Conference Paper

    ERIC Educational Resources Information Center

    Karmel, Tom

    2009-01-01

    The vocational education and training (VET) sector has a long tradition of measuring and reporting outcomes. The public face of this is the "Annual National Report of the Australian Vocational Education and Training System" published (and tabled in the Commonwealth Parliament) since 1994. The reporting framework has undergone a number of changes…

  14. Psychometric properties of the Spanish version of the Clinical Outcomes in Routine Evaluation – Outcome Measure

    PubMed Central

    Trujillo, Adriana; Feixas, Guillem; Bados, Arturo; García-Grau, Eugeni; Salla, Marta; Medina, Joan Carles; Montesano, Adrián; Soriano, José; Medeiros-Ferreira, Leticia; Cañete, Josep; Corbella, Sergi; Grau, Antoni; Lana, Fernando; Evans, Chris

    2016-01-01

    Objective The objective of this paper is to assess the reliability and validity of the Spanish translation of the Clinical Outcomes in Routine Evaluation – Outcome Measure, a 34-item self-report questionnaire that measures the client’s status in the domains of Subjective well-being, Problems/Symptoms, Life functioning, and Risk. Method Six hundred and forty-four adult participants were included in two samples: the clinical sample (n=192) from different mental health and primary care centers; and the nonclinical sample (n=452), which included a student and a community sample. Results The questionnaire showed good acceptability and internal consistency, appropriate test–retest reliability, and acceptable convergent validity. Strong differentiation between clinical and nonclinical samples was found. As expected, the Risk domain had different characteristics than other domains, but all findings were comparable with the UK referential data. Cutoff scores were calculated for clinical significant change assessment. Conclusion The Spanish version of the Clinical Outcomes in Routine Evaluation – Outcome Measure showed acceptable psychometric properties, providing support for using the questionnaire for monitoring the progress of Spanish-speaking psychotherapy clients. PMID:27382288

  15. Item response theory modeling in health outcomes measurement.

    PubMed

    Reeve, Bryce B

    2003-04-01

    There is a great need in health outcomes research to develop instruments that accurately measure a person's health status with minimal response burden. This need for psychometrically sound and clinically meaningful measures calls for better analytical tools beyond the methods available from traditional measurement theory. Applications of item response theory (IRT) modeling have increased considerably because of its utility for instrument development and evaluation, scale scoring, assessment of cultural equivalence, instrument linking and computerized adaptive testing. IRT models the relationship between a person's response to a survey question and their standing on a health construct, such as fatigue or depression. This review will discuss the theory and basics of IRT models and applications of these models to health outcomes measurement. PMID:19807361

  16. Intelligent outcome measures in liaison psychiatry: essential even if not desirable: Commentary on … a multidimensional Framework for Routine Outcome Measurement in Liaison Psychiatry (FROM-LP).

    PubMed

    Tadros, George

    2016-08-01

    Service development is guided by outcome measures that inform service commissioners and providers. Those in liaison psychiatry should be encouraged to develop a positive approach that integrates the collection of outcome measures into everyday clinical practice. The Framework for Routine Outcome Measurement in Liaison Psychiatry (FROM-LP) is a very useful tool to measure service quality and clinical effectiveness, using a combination of clinician-rated and patient-rated outcome measures and patient-rated experience measures. However, it does not include measures of cost-effectiveness or training activities. The FROM-LP is a significant step towards developing nationally unified outcome measures. PMID:27512588

  17. Treatment Outcome Package: Measuring and facilitating multidimensional change.

    PubMed

    Boswell, James F; Kraus, David R; Castonguay, Louis G; Youn, Soo Jeong

    2015-12-01

    The Treatment Outcome Package (TOP; D. R. Kraus, Seligman, & Jordan, 2005) is a multidimensional routine progress and outcome measure developed for use in diverse naturalistic practice settings. In this article, we (a) provide a brief review and summary of the extant psychometric and research support for the TOP, (b) provide examples of the TOP's use in clinical training and practice, and (c) discuss the implications of the TOP for future psychotherapy training, research, and practice. In particular, we focus on the implications of risk-adjusted progress monitoring for systems of care and mental health care decision making. PMID:26641372

  18. Measurement of Outcomes of Upper Limb Reconstructive Surgery for Tetraplegia.

    PubMed

    Sinnott, K Anne; Dunn, Jennifer A; Wangdell, Johanna; Johanson, M Elise; Hall, Andrew S; Post, Marcel W

    2016-06-01

    Reconstructive arm/hand surgery for tetraplegia is performed to improve arm/hand function and therefore personal well-being for individuals who accept such elective surgeries. However, changes at an impairment level do not always translate into functional or quality of life changes. Therefore, multiple outcome tools should be used that incorporate sufficient responsiveness to detect changes in arm/hand function, activity and participation, and quality of life of the individuals involved. This narrative review aims to assist clinicians to choose the most appropriate tools to assess the need for reconstructive surgery and to evaluate its outcomes. Our specific objectives are (1) to describe aspects to consider when choosing a measure and (2) to describe the measures advised by an international therapist consensus group established in 2007. All advised measures are appraised in terms of the underlying construct, administration, and clinical relevance to arm/hand reconstructions. Essentially there are currently no criterion standard measures to evaluate the consequences of reconstructive arm/hand surgery. However, with judicious use of available measures it is possible to ensure the questions asked or tasks completed are relevant to the surgical reconstruction(s) undertaken. Further work in this field is required. This would be best met by immediate collaboration between 2 outcome's tool developers and by analysis of pre- and postoperative data already held in various international sites, which would allow further evaluation of the measures already in use, or components thereof. PMID:27233592

  19. Medical specialty boards can help measure graduate medical education outcomes.

    PubMed

    Peterson, Lars E; Carek, Peter; Holmboe, Eric S; Puffer, James C; Warm, Eric J; Phillips, Robert L

    2014-06-01

    U.S. graduate medical education (GME) training institutions are under increasing scrutiny to measure program outcomes as a demonstration of accountability for the sizeable funding they receive from the federal government. The Accreditation Council for Graduate Medical Education (ACGME) is a potential agent of measuring GME accountability but has no interaction with physicians after residency training is completed. American Board of Medical Specialty (ABMS) member boards interact with physicians throughout their careers through maintenance of certification (MOC) and are a potential source of valuable data on physician competency and quality of care, both of which could be used to measure GME accountability.The authors propose that ABMS boards and the ACGME deepen their existing relationship to better assess residency training outcomes. ABMS boards have a wealth of data on physicians collected as a by-product of MOC and business operations. Further, many ABMS boards collect practice demographics and scope-of-practice information through MOC enrollment surveys or recertification examination questionnaires. These data are potentially valuable in helping residencies know what their graduates are doing in practice. Part 4 of MOC generally involves assessment of the quality of care delivered in practice, and ABMS boards could share these deidentified data with the ACGME and residency programs to provide direct feedback on the practice outcomes of graduates.ABMS member boards and the ACGME should broaden their long-standing relationship to further develop shared roles and data-sharing mechanisms to better inform residencies and the public about GME training outcomes. PMID:24871232

  20. Comparison of Physician-Predicted to Measured Low Vision Outcomes

    PubMed Central

    Chan, Tiffany L.; Goldstein, Judith E.; Massof, Robert W.

    2013-01-01

    Purpose To compare low vision rehabilitation (LVR) physicians’ predictions of the probability of success of LVR to patients’ self-reported outcomes after provision of usual outpatient LVR services; and to determine if patients’ traits influence physician ratings. Methods The Activity Inventory (AI), a self-report visual function questionnaire, was administered pre and post-LVR to 316 low vision patients served by 28 LVR centers that participated in a collaborative observational study. The physical component of the Short Form-36, Geriatric Depression Scale, and Telephone Interview for Cognitive Status were also administered pre-LVR to measure physical capability, depression and cognitive status. Following patient evaluation, 38 LVR physicians estimated the probability of outcome success (POS), using their own criteria. The POS ratings and change in functional ability were used to assess the effects of patients’ baseline traits on predicted outcomes. Results A regression analysis with a hierarchical random effects model showed no relationship between LVR physician POS estimates and AI-based outcomes. In another analysis, Kappa statistics were calculated to determine the probability of agreement between POS and AI-based outcomes for different outcome criteria. Across all comparisons, none of the kappa values were significantly different from 0, which indicates the rate of agreement is equivalent to chance. In an exploratory analysis, hierarchical mixed effects regression models show that POS ratings are associated with information about the patient’s cognitive functioning and the combination of visual acuity and functional ability, as opposed to visual acuity or functional ability alone. Conclusions Physicians’ predictions of LVR outcomes appear to be influenced by knowledge of patients’ cognitive functioning and the combination of visual acuity and functional ability - information physicians acquire from the patient’s history and examination. However

  1. Generating Outcome Measurements: Achievement and Attitudes. A Guide to Educational Outcome Measurements and Their Uses. Seminar No. 3.

    ERIC Educational Resources Information Center

    Mushkin, Selma J.; Billings, Bradley B.

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministries, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with how to obtain information on…

  2. RASCH ANALYSIS OF CLINICAL OUTCOME MEASURES IN SPINAL MUSCULAR ATROPHY

    PubMed Central

    CANO, STEFAN J.; MAYHEW, ANNA; GLANZMAN, ALLAN M.; KROSSCHELL, KRISTIN J.; SWOBODA, KATHRYN J.; MAIN, MARION; STEFFENSEN, BIRGIT F.; BÉRARD, CAROLE; GIRARDOT, FRANÇOISE; PAYAN, CHRISTINE A.M.; MERCURI, EUGENIO; MAZZONE, ELENA; ELSHEIKH, BAKRI; FLORENCE, JULAINE; HYNAN, LINDA S.; IANNACCONE, SUSAN T.; NELSON, LESLIE L.; PANDYA, SHREE; ROSE, MICHAEL; SCOTT, CHARLES; SADJADI, REZA; YORE, MACKENSIE A.; JOYCE, CYNTHIA; KISSEL, JOHN T.

    2015-01-01

    Introduction Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). Methods Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. Results Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. Conclusions The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges. PMID:23836324

  3. Breast Cancer EDGE Task Force Outcomes: Clinical Measures of Pain

    PubMed Central

    Harrington, Shana; Gilchrist, Laura; Sander, Antoinette

    2014-01-01

    Background Pain is one of the most commonly reported impairments after breast cancer treatment affecting anywhere from 16-73% of breast cancer survivors Despite the high reported incidence of pain from cancer and its treatments, the ability to evaluate cancer pain continues to be difficult due to the complexity of the disease and the subjective experience of pain. The Oncology Section Breast Cancer EDGE Task Force was created to evaluate the evidence behind clinical outcome measures of pain in women diagnosed with breast cancer. Methods The authors systematically reviewed the literature for pain outcome measures published in the research involving women diagnosed with breast cancer. The goal was to examine the reported psychometric properties that are reported in the literature in order to determine clinical utility. Results Visual Analog Scale, Numeric Rating Scale, Pressure Pain Threshold, McGill Pain Questionnaire, McGill Pain Questionnaire – Short Form, Brief Pain Inventory and Brief Pain Inventory – Short Form were highly recommended by the Task Force. The Task Force was unable to recommend two measures for use in the breast cancer population at the present time. Conclusions A variety of outcome measures were used to measure pain in women diagnosed with breast cancer. When assessing pain in women with breast cancer, researchers and clinicians need to determine whether a unidimensional or multidimensional tool is most appropriate as well as whether the tool has strong psychometric properties. PMID:25346950

  4. The Daily Activity Report (DAR) a Novel Measure of Functional Outcome for Serious Mental Illness.

    PubMed

    Velligan, Dawn I; Mintz, Jim; Sierra, Cynthia; Martin, Mona L; Fredrick, Megan; Maglinte, Gregory A; Corey-Lisle, Patricia K

    2016-05-01

    The assessment of real-world functional outcomes in clinical trials for medications targeting negative symptoms and cognitive impairment is extremely important. We tested the psychometric properties of the Daily Activity Report (DAR), a novel assessment of productive daily activity. We administered the DAR and additional assessments of functional outcome, functional capacity, cognition and symptomatology to 50 individuals with schizophrenia at 2 time points, 1 month apart and to 25 healthy controls. The DAR records a person's daily activity for 7 consecutive days based upon phone calls made 3 times a day. A total score and scores in 3 domains; instrumental activities (ie, independent living), social and work or school related activities are generated for the DAR. Inter-item consistency was high 0.89-0.94 for each domain and 0.88 overall. Test-retest reliability across 1 month for the total DAR score was 0.67,P< .0001. The total DAR score as well as scores for social activity and nondomestic work/school differed significantly between control and patient participants (P< .0001). DAR domain scores were associated with negative symptoms and functional outcomes, but the primary score related to these measures was the work/school dimension of the DAR. DAR scores were only weakly and nonsignificantly related to positive symptoms. This study provides preliminary support for the reliability and validity of the DAR using interviewer administration. The development of a patient reported version of the DAR using smart phone technology with automatic scoring is the next step. PMID:26712856

  5. The Daily Activity Report (DAR) a Novel Measure of Functional Outcome for Serious Mental Illness

    PubMed Central

    Velligan, Dawn I.; Mintz, Jim; Sierra, Cynthia; Martin, Mona L.; Fredrick, Megan; Maglinte, Gregory A.; Corey-Lisle, Patricia K.

    2016-01-01

    The assessment of real-world functional outcomes in clinical trials for medications targeting negative symptoms and cognitive impairment is extremely important. We tested the psychometric properties of the Daily Activity Report (DAR), a novel assessment of productive daily activity. We administered the DAR and additional assessments of functional outcome, functional capacity, cognition and symptomatology to 50 individuals with schizophrenia at 2 time points, 1 month apart and to 25 healthy controls. The DAR records a person’s daily activity for 7 consecutive days based upon phone calls made 3 times a day. A total score and scores in 3 domains; instrumental activities (ie, independent living), social and work or school related activities are generated for the DAR. Inter-item consistency was high 0.89–0.94 for each domain and 0.88 overall. Test–retest reliability across 1 month for the total DAR score was 0.67, P < .0001. The total DAR score as well as scores for social activity and nondomestic work/school differed significantly between control and patient participants (P < .0001). DAR domain scores were associated with negative symptoms and functional outcomes, but the primary score related to these measures was the work/school dimension of the DAR. DAR scores were only weakly and nonsignificantly related to positive symptoms. This study provides preliminary support for the reliability and validity of the DAR using interviewer administration. The development of a patient reported version of the DAR using smart phone technology with automatic scoring is the next step. PMID:26712856

  6. Outcome measures for palliative oxygen therapy: relevance and practical utility.

    PubMed

    Antoniu, Sabina; Mihaltan, Florin

    2014-06-01

    Dyspnea is a common symptom in many advanced malignant and non-malignant diseases and often is refractory to the usual therapies. In such circumstances palliative care approaches are necessary and among them palliative care oxygen therapy can be applied although currently its effectiveness is rather uncertain. Palliative oxygen therapy can be given on either continuous basis or on demand. Often the continuous palliative oxygen therapy is seen as long-term oxygen therapy although their aims are rather different. Palliative oxygen therapy was evaluated in populations with mixed underlying diseases, with outcome measures not only the most appropriate for the setting and therefore these limitations might have influenced the overall perceived therapeutic benefit. Therefore an evaluation of this method in subsets defined based on the etiology and pathogenic mechanisms and with appropriate outcome measures would help to better define the criteria for its indication and would increase its acceptability. PMID:24741999

  7. Novel Outcome Measures for Clinical Trials in Cystic Fibrosis

    PubMed Central

    Tiddens, Harm AWM; Puderbach, Michael; Venegas, Jose G; Ratjen, Felix; Donaldson, Scott H; Davis, Stephanie D; Rowe, Steven M; Sagel, Scott D; Higgins, Mark; Waltz, David A

    2015-01-01

    Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc. PMID:25641878

  8. Do clinical outcome measures assess consumer-defined recovery?

    PubMed

    Andresen, Retta; Caputi, Peter; Oades, Lindsay G

    2010-05-30

    There is an international call for mental health services to become recovery-oriented, and also to use evidence-based practices. Addressing this call requires recovery-oriented measurement of outcomes and service evaluation. Mental health consumers view recovery as leading as meaningful life, and have criticised traditional clinical measures for being too disability-oriented. This study compares three measures of consumer-defined recovery from enduring mental illness: the Recovery Assessment Scale, the Mental Health Recovery Measure and the Self-Identified Stage of Recovery, with four conventional clinical measures. Correlational analyses supported the convergent validity of the recovery measures, although certain subscales were unrelated to each other. More importantly, little relationship was found between consumer-defined recovery and the clinical measures. Analyses of variance revealed that scores on the recovery measures increased across self-identified stage of recovery, but scores on most clinical measures did not improve consistently across stage of recovery. The findings demonstrate the qualitative difference between the two types of measures, supporting the claim by consumers that clinical measures do not assess important aspects of recovery. There is a need for further research and refinement of recovery measurement, including assessment of stages of recovery, with the aim of including such measures as an adjunct in routine clinical assessment, service evaluation and research. PMID:20227768

  9. Outcome-Driven Thresholds for Home Blood Pressure Measurement

    PubMed Central

    Niiranen, Teemu J.; Asayama, Kei; Thijs, Lutgarde; Johansson, Jouni K.; Ohkubo, Takayoshi; Kikuya, Masahiro; Boggia, José; Hozawa, Atsushi; Sandoya, Edgardo; Stergiou, George S.; Tsuji, Ichiro; Jula, Antti M.; Imai, Yutaka; Staessen, Jan A.

    2013-01-01

    The lack of outcome-driven operational thresholds limits the clinical application of home blood pressure (BP) measurement. Our objective was to determine an outcome-driven reference frame for home BP measurement. We measured home and clinic BP in 6470 participants (mean age, 59.3 years; 56.9% women; 22.4% on antihypertensive treatment) recruited in Ohasama, Japan (n=2520); Montevideo, Uruguay (n=399); Tsurugaya, Japan (n=811); Didima, Greece (n=665); and nationwide in Finland (n=2075). In multivariable-adjusted analyses of individual subject data, we determined home BP thresholds, which yielded 10-year cardiovascular risks similar to those associated with stages 1 (120/80 mm Hg) and 2 (130/85 mm Hg) prehypertension, and stages 1 (140/90 mm Hg) and 2 (160/100 mm Hg) hypertension on clinic measurement. During 8.3 years of follow-up (median), 716 cardiovascular end points, 294 cardiovascular deaths, 393 strokes, and 336 cardiac events occurred in the whole cohort; in untreated participants these numbers were 414, 158, 225, and 194, respectively. In the whole cohort, outcome-driven systolic/diastolic thresholds for the home BP corresponding with stages 1 and 2 prehypertension and stages 1 and 2 hypertension were 121.4/77.7, 127.4/79.9, 133.4/82.2, and 145.4/86.8 mm Hg; in 5018 untreated participants, these thresholds were 118.5/76.9, 125.2/79.7, 131.9/82.4, and 145.3/87.9 mm Hg, respectively. Rounded thresholds for stages 1 and 2 prehypertension and stages 1 and 2 hypertension amounted to 120/75, 125/80, 130/85, and 145/90 mm Hg, respectively. Population-based outcome-driven thresholds for home BP are slightly lower than those currently proposed in hypertension guidelines. Our current findings could inform guidelines and help clinicians in diagnosing and managing patients. PMID:23129700

  10. Improving Patient Experience and Primary Care Quality for Patients With Complex Chronic Disease Using the Electronic Patient-Reported Outcomes Tool: Adopting Qualitative Methods Into a User-Centered Design Approach

    PubMed Central

    Khan, Anum Irfan; Kuluski, Kerry; McKillop, Ian; Sharpe, Sarah; Bierman, Arlene S; Lyons, Renee F; Cott, Cheryl

    2016-01-01

    Background Many mHealth technologies do not meet the needs of patients with complex chronic disease and disabilities (CCDDs) who are among the highest users of health systems worldwide. Furthermore, many of the development methodologies used in the creation of mHealth and eHealth technologies lack the ability to embrace users with CCDD in the specification process. This paper describes how we adopted and modified development techniques to create the electronic Patient-Reported Outcomes (ePRO) tool, a patient-centered mHealth solution to help improve primary health care for patients experiencing CCDD. Objective This paper describes the design and development approach, specifically the process of incorporating qualitative research methods into user-centered design approaches to create the ePRO tool. Key lessons learned are offered as a guide for other eHealth and mHealth research and technology developers working with complex patient populations and their primary health care providers. Methods Guided by user-centered design principles, interpretive descriptive qualitative research methods were adopted to capture user experiences through interviews and working groups. Consistent with interpretive descriptive methods, an iterative analysis technique was used to generate findings, which were then organized in relation to the tool design and function to help systematically inform modifications to the tool. User feedback captured and analyzed through this method was used to challenge the design and inform the iterative development of the tool. Results Interviews with primary health care providers (n=7) and content experts (n=6), and four focus groups with patients and carers (n=14) along with a PICK analysis—Possible, Implementable, (to be) Challenged, (to be) Killed—guided development of the first prototype. The initial prototype was presented in three design working groups with patients/carers (n=5), providers (n=6), and experts (n=5). Working group findings were

  11. Outcome as a Measure of Quality of Care in Oncology

    PubMed Central

    Burney, Ikram A; Al Moundhri, Mansour S; Rizvi, Azhar J; Ganguly, Shyam S; Al Abri, Rashid; Ashrafi, Rafi A

    2008-01-01

    Objectives: Measurement of outcomes is increasingly employed as an indicator of the quality of clinical care. The most commonly measured outcome in many clinical studies, especially in oncology, still remains the overall survival rate. Sultan Qaboos University Hospital (SQUH), Oman, is striving for excellence through quality management. In seeking continual improvement, quality measurement exercises have been initiated throughout the Hospital. We present the overall survival rate of four of the ten most common cancers diagnosed in Oman. Methods: The cancers included non-Hodgkin’s lymphoma (NHL), Hodgkin’s lymphoma (HL), breast cancer, and stomach cancer. The studies were all retrospective and had been conducted previously. For present purposes, only the overall survival was compared with studies both from the region, and with bench-mark studies. Results: For NHL, with a median follow-up of 8 months, the 2-year overall survival rate was 64%; 90% for low risk, 55% for intermediate risk, and 15% for high risk groups. For HL, the 5-year overall survival rate was 64%; 76% for low risk and 42% for high risk. For breast cancer, the 5-year survival rate was 67%; percentages were 88%, 75% and 59% for Groups I, II, and III respectively. For gastric cancer, the 5-year survival rate was 16.5 %; 24% for the non-metastatic group. Conclusion: The outcome of patients with early stages and fewer adverse prognostic factors is comparable to what has been reported in the international literature; however, the outcome is inferior for patients presenting with advanced stage disease and several adverse prognostic factors. PMID:21654954

  12. Postsecondary Education Outcome Measures and Procedures: A Sourcebook for Administrative Research.

    ERIC Educational Resources Information Center

    Lenning, Oscar T.; And Others

    A guide to acquiring information needed about postsecondary education outcomes is presented for campus and state-level officials. Attention is directed to: over 200 outcome measures or indicators; standard definitions of each outcome measure; data sources and procedures to acquire data for each outcome measure; and suggestions concerning the…

  13. Measuring Outcome in the Treatment of Cocaine Dependence

    PubMed Central

    Crits-Christoph, Paul; Gallop, Robert; Gibbons, Mary Beth Connolly; Sadicario, Jaclyn S.; Woody, George

    2015-01-01

    Background Little in known about the extent to which outcome measures used in studies of the treatment of cocaine dependence are associated with longer-term use and with broader measures of clinical improvement. The current study examined reductions in use, and abstinence-oriented measures, in relation to functioning and longer-term clinical benefits in the treatment of cocaine dependence. Methods Overall drug use, cocaine use, and functioning in a number of addiction-related domains for 487 patients diagnosed with DSM-IV cocaine dependence and treated with one of four psychosocial interventions in the NIDA Cocaine Collaborative Treatment Study were assessed monthly during 6 months of treatment and at 9, 12, 15, and 18 month follow-up. Results Measures of during-treatment reduction in use were moderately correlated with drug and cocaine use measures 12 months, but showed non-significant or small correlations with measures of functioning at 12 months. Highest correlations were evident for abstinence measures (maximum consecutive days abstinence and completely abstinent) during treatment in relation to sustained (3 month) abstinence at 12 months. Latent class analysis of patterns of change over time revealed that most patients initially (months 1 to 4 of treatment) either became abstinent immediately or continued to use every month. Over the couse of follow-up, patients either maintained abstinence or used regularly – intermittent use was less common. Conclusions There were generally small associations between various measures of cocaine use and longer-term clinical benefits, other than abstinence was associated with continued abstinence. No one method of measuring outcome of treatment of cocaine dependence appears superior to others. PMID:26366427

  14. Unconditionally secure bit commitment by transmitting measurement outcomes.

    PubMed

    Kent, Adrian

    2012-09-28

    We propose a new unconditionally secure bit commitment scheme based on Minkowski causality and the properties of quantum information. The receiving party sends a number of randomly chosen Bennett-Brassard 1984 (BB84) qubits to the committer at a given point in space-time. The committer carries out measurements in one of the two BB84 bases, depending on the committed bit value, and transmits the outcomes securely at (or near) light speed in opposite directions to remote agents. These agents unveil the bit by returning the outcomes to adjacent agents of the receiver. The protocol's security relies only on simple properties of quantum information and the impossibility of superluminal signalling. PMID:23030073

  15. Measuring Outcomes for Young Children and Their Families. Outcome Indicators for Everyday Kids, Everyday Lives: A Vision for Pennsylvania's Children

    ERIC Educational Resources Information Center

    Trivette, Carol M.; Dunst, Carl J.

    2011-01-01

    This monograph includes the final report for a project funded by the Pennsylvania Developmental Disabilities Council for "Measuring Outcomes for Children" (2008 RFP). The goal of the project was to "develop and demonstrate the effectiveness of an instrument designed to measure life outcomes of children with disabilities being…

  16. Outcome measures in MMN revisited: further improvement needed.

    PubMed

    Pruppers, Mariëlle H J; Draak, Thomas H P; Vanhoutte, Els K; Van der Pol, W-Ludo; Gorson, Kenneth C; Léger, Jean-Marc; Nobile-Orazio, Eduardo; Lewis, Richard A; van den Berg, Leonard H; Faber, Catharina G; Merkies, Ingemar S J

    2015-09-01

    The objectives of this study were to provide an overview of the outcome measures (OMs) applied in clinical trials in multifocal motor neuropathy (MMN) and to determine the responsiveness of a core set of selected OMs as part of the peripheral neuropathy outcome measures standardization (PeriNomS) study. The following OMs were serially applied in 26 patients with newly diagnosed or relapsing MMN, receiving intravenous immunoglobulin (assessments: T0/T3/T12 months): 14 muscle pairs MRC (Medical Research Council) scale, the Neuropathy Impairment Scale motor-subset, a self-evaluation scale, grip strength, and MMN-RODS© (Rasch-built overall disability scale). All data, except the grip strength, were subjected to Rasch analyses before determining responsiveness. For grip strength, responsiveness was examined using a combined anchor- (SF-36 question-2) and distribution-based (½ × SD) minimum clinically important difference (MCID) techniques, determining the proportion of patients exceeding both the identified cut-offs. For the remaining scales, the magnitude of change for each patient on each scale was determined using the MCID related to the individual SE (responder definition: MCID-SE ≥ 1.96). Overall, a great assortment of measures has been used in MMN trials with different responsiveness definitions. For the selected OMs, responsiveness was poor and only seen in one fourth to one third of the patients, the grip strength being more responsive. Despite the efforts taken to standardize outcome assessment, further clinimetric responsiveness studies are needed in MMN. PMID:26115442

  17. Learning disability and epilepsy. 1, towards common outcome measures.

    PubMed

    Kerr, M P; Espie, C A

    1997-10-01

    A major component of the population of people who have epilepsy are people with a learning disability. As a group, such individuals often have complex epilepsy which is refractory to treatment. Current available measures to assess the outcomes of therapeutic interventions in epilepsy are based on seizure frequency, seizure severity and quality-of-life measures, but have not been validated in people with a learning disability. Thus, we do not know if such measures of outcome serve the needs of this group. This review examines how able we are to assess the efficacy of our interventions to control epilepsy in people with learning disability. It is suggested that a standard data set is necessary as the basis of the assessment of any therapeutic intervention. Central components of this data set would encompass a definition of important characteristics of an individual, a description of their epilepsy and an assessment of the impact of their condition on both their own and their carer's health. The approach to obtaining this information should employ a methodology which can allow for environmental influences. PMID:9663796

  18. Clinical Evaluation of a Mobile Sensor-Based Gait Analysis Method for Outcome Measurement after Knee Arthroplasty

    PubMed Central

    Calliess, Tilman; Bocklage, Raphael; Karkosch, Roman; Marschollek, Michael; Windhagen, Henning; Schulze, Mareike

    2014-01-01

    Clinical scores and motion-capturing gait analysis are today's gold standard for outcome measurement after knee arthroplasty, although they are criticized for bias and their ability to reflect patients' actual quality of life has been questioned. In this context, mobile gait analysis systems have been introduced to overcome some of these limitations. This study used a previously developed mobile gait analysis system comprising three inertial sensor units to evaluate daily activities and sports. The sensors were taped to the lumbosacral junction and the thigh and shank of the affected limb. The annotated raw data was evaluated using our validated proprietary software. Six patients undergoing knee arthroplasty were examined the day before and 12 months after surgery. All patients reported a satisfactory outcome, although four patients still had limitations in their desired activities. In this context, feasible running speed demonstrated a good correlation with reported impairments in sports-related activities. Notably, knee flexion angle while descending stairs and the ability to stop abruptly when running exhibited good correlation with the clinical stability and proprioception of the knee. Moreover, fatigue effects were displayed in some patients. The introduced system appears to be suitable for outcome measurement after knee arthroplasty and has the potential to overcome some of the limitations of stationary gait labs while gathering additional meaningful parameters regarding the force limits of the knee. PMID:25171119

  19. Intelligent outcome measures in liaison psychiatry: essential even if not desirable

    PubMed Central

    Tadros, George

    2016-01-01

    Service development is guided by outcome measures that inform service commissioners and providers. Those in liaison psychiatry should be encouraged to develop a positive approach that integrates the collection of outcome measures into everyday clinical practice. The Framework for Routine Outcome Measurement in Liaison Psychiatry (FROM-LP) is a very useful tool to measure service quality and clinical effectiveness, using a combination of clinician-rated and patient-rated outcome measures and patient-rated experience measures. However, it does not include measures of cost-effectiveness or training activities. The FROM-LP is a significant step towards developing nationally unified outcome measures. PMID:27512588

  20. Connecting Stuttering Management and Measurement: I. Core Speech Measures of Clinical Process and Outcome

    ERIC Educational Resources Information Center

    Shenker, Rosalee C.

    2006-01-01

    Background: There will always be a place for stuttering treatments designed to eliminate or reduce stuttered speech. When those treatments are required, direct speech measures of treatment process and outcome are needed in clinical practice. Aims: Based on the contents of published clinical trials of such treatments, three "core" measures of…

  1. Selecting Rehabilitation Outcome Measures for People with Multiple Sclerosis

    PubMed Central

    Potter, Kirsten; Allen, Diane D.; Bennett, Susan E.; Brandfass, Kathi G.; Widener, Gail L.; Yorke, Amy M.

    2015-01-01

    Despite the well-known benefits of using standardized outcome measures (OMs) in clinical practice, a variety of barriers interfere with their use. In particular, rehabilitation therapists lack sufficient knowledge in selecting appropriate OMs. The challenge is compounded when working with people with multiple sclerosis (MS) owing to heterogeneity of the patient population and symptom variability in individual patients. To help overcome these barriers, the American Physical Therapy Association appointed the Multiple Sclerosis Outcome Measures Task Force to review and make evidence-based recommendations for OM use in clinical practice, education, and research specific to people with MS. Sixty-three OMs were reviewed based on their clinical utility, psychometric properties, and a consensus evaluation of the appropriateness of use for people with MS. We sought to illustrate use of the recommendations for two cases. The first case involves a 43-year-old man with new-onset problems after an exacerbation. The second case pertains to an outpatient clinic interested in assessing the effectiveness of their MS rehabilitation program. For each case, clinicians identified areas that were important to assess and various factors deemed important for OM selection. Criteria were established and used to assist in OM selection. In both cases, the described processes narrowed the selection of OMs and assisted with choosing the most appropriate ones. The recommendations, in addition to the processes described in these two cases, can be used by clinicians in any setting working with patients with MS across the disability spectrum. PMID:26300704

  2. Goal Attainment Scaling. A responsive measure of client outcomes.

    PubMed

    Forbes, D A

    1998-12-01

    Goal Attainment Scaling (GAS) was developed by Kiresuk and Sherman in 1968. Although highly criticized in the early years of its conception, GAS is beginning to be recognized as a reliable, valid, and responsive outcome measurement approach. This article describes the application of GAS, using elderly home health care clients as an example. Reliability and validity issues are examined. Criticism of GAS is often based on traditional psychometric assumptions associated with normative assessment and does not reflect the evaluative nature of the GAS methodology. The advantages and limitations of GAS are discussed. Goal Attainment Scaling has the potential to demonstrate the important contribution home health care programs make to clients by measuring the change that has occurred during their admission to the program. This kind of information is required by policy makers, case managers, and home health care providers to develop policy, allocate limited resources, and offer appropriate and effective services. PMID:10025309

  3. Measuring the Outcome of Biomedical Research: A Systematic Literature Review

    PubMed Central

    Thonon, Frédérique; Boulkedid, Rym; Delory, Tristan; Rousseau, Sophie; Saghatchian, Mahasti; van Harten, Wim; O’Neill, Claire; Alberti, Corinne

    2015-01-01

    Background There is an increasing need to evaluate the production and impact of medical research produced by institutions. Many indicators exist, yet we do not have enough information about their relevance. The objective of this systematic review was (1) to identify all the indicators that could be used to measure the output and outcome of medical research carried out in institutions and (2) enlist their methodology, use, positive and negative points. Methodology We have searched 3 databases (Pubmed, Scopus, Web of Science) using the following keywords: [Research outcome* OR research output* OR bibliometric* OR scientometric* OR scientific production] AND [indicator* OR index* OR evaluation OR metrics]. We included articles presenting, discussing or evaluating indicators measuring the scientific production of an institution. The search was conducted by two independent authors using a standardised data extraction form. For each indicator we extracted its definition, calculation, its rationale and its positive and negative points. In order to reduce bias, data extraction and analysis was performed by two independent authors. Findings We included 76 articles. A total of 57 indicators were identified. We have classified those indicators into 6 categories: 9 indicators of research activity, 24 indicators of scientific production and impact, 5 indicators of collaboration, 7 indicators of industrial production, 4 indicators of dissemination, 8 indicators of health service impact. The most widely discussed and described is the h-index with 31 articles discussing it. Discussion The majority of indicators found are bibliometric indicators of scientific production and impact. Several indicators have been developed to improve the h-index. This indicator has also inspired the creation of two indicators to measure industrial production and collaboration. Several articles propose indicators measuring research impact without detailing a methodology for calculating them. Many

  4. Outcome instruments for prosthetics: clinical applications.

    PubMed

    Heinemann, Allen W; Connelly, Lauri; Ehrlich-Jones, Linda; Fatone, Stefania

    2014-02-01

    Outcome measurement is crucial to assuring high-quality patient services and improving the quality of services provided by prosthetists. This article summarizes recent evidence on the measurement properties of outcome measures, and updates previously published summaries of outcome instruments. The review focuses on measures of mobility, functional status, quality of life, and patient satisfaction, and includes both performance-based and patient-reported outcomes. Amputation-specific and general measures that are suitable for patients served by prosthetists are discussed. It is encouraging that responsiveness of measures is often reported, as this information is needed to improve clinical utility. PMID:24287247

  5. Entanglement-assisted guessing of complementary measurement outcomes

    NASA Astrophysics Data System (ADS)

    Berta, Mario; Coles, Patrick J.; Wehner, Stephanie

    2014-12-01

    Heisenberg's uncertainty principle implies that if one party (Alice) prepares a system and randomly measures one of two incompatible observables, then another party (Bob) cannot perfectly predict the measurement outcomes. This implication assumes that Bob does not possess an additional system that is entangled to the measured one; indeed, the seminal paper of Einstein, Podolsky, and Rosen (EPR) showed that maximal entanglement allows Bob to perfectly win this guessing game. Although not in contradiction, the observations made by EPR and Heisenberg illustrate two extreme cases of the interplay between entanglement and uncertainty. On the one hand, no entanglement means that Bob's predictions must display some uncertainty. Yet on the other hand, maximal entanglement means that there is no more uncertainty at all. Here we follow an operational approach and give an exact relation—an equality—between the amount of uncertainty as measured by the guessing probability and the amount of entanglement as measured by the recoverable entanglement fidelity. From this equality, we deduce a simple criterion for witnessing bipartite entanglement and an entanglement monogamy equality.

  6. Development of the FOCUS (Focus on the Outcomes of Communication under Six), a Communication Outcome Measure for Preschool Children

    ERIC Educational Resources Information Center

    Thomas-Stonell, Nancy L.; Oddson, Bruce; Robertson, Bernadette; Rosenbaum, Peter L.

    2010-01-01

    Aim: Our aim was to develop an outcome measure, called Focus on the Outcomes of Communication Under Six (FOCUS), that captures real-world changes in preschool children's communication. Conceptually grounded in the World Health Organization International Classification of Functioning, Disability and Health framework, the FOCUS items were derived…

  7. The King's Outcome Scale for Childhood Head Injury and Injury Severity and Outcome Measures in Children with Traumatic Brain Injury

    ERIC Educational Resources Information Center

    Calvert, Sophie; Miller, Helen E.; Curran, Andrew; Hameed, Biju; McCarter, Renee; Edwards, Richard J.; Hunt, Linda; Sharples, Peta Mary

    2008-01-01

    The aim of this study was to relate discharge King's Outcome Scale for Childhood Head Injury (KOSCHI) category to injury severity and detailed outcome measures obtained in the first year post-traumatic brain injury (TBI). We used a prospective cohort study. Eighty-one children with TBI were studied: 29 had severe, 15 moderate, and 37 mild TBI. The…

  8. Vision and vision-related outcome measures in multiple sclerosis

    PubMed Central

    Balcer, Laura J.; Miller, David H.; Reingold, Stephen C.

    2015-01-01

    Visual impairment is a key manifestation of multiple sclerosis. Acute optic neuritis is a common, often presenting manifestation, but visual deficits and structural loss of retinal axonal and neuronal integrity can occur even without a history of optic neuritis. Interest in vision in multiple sclerosis is growing, partially in response to the development of sensitive visual function tests, structural markers such as optical coherence tomography and magnetic resonance imaging, and quality of life measures that give clinical meaning to the structure-function correlations that are unique to the afferent visual pathway. Abnormal eye movements also are common in multiple sclerosis, but quantitative assessment methods that can be applied in practice and clinical trials are not readily available. We summarize here a comprehensive literature search and the discussion at a recent international meeting of investigators involved in the development and study of visual outcomes in multiple sclerosis, which had, as its overriding goals, to review the state of the field and identify areas for future research. We review data and principles to help us understand the importance of vision as a model for outcomes assessment in clinical practice and therapeutic trials in multiple sclerosis. PMID:25433914

  9. Vision and vision-related outcome measures in multiple sclerosis.

    PubMed

    Balcer, Laura J; Miller, David H; Reingold, Stephen C; Cohen, Jeffrey A

    2015-01-01

    Visual impairment is a key manifestation of multiple sclerosis. Acute optic neuritis is a common, often presenting manifestation, but visual deficits and structural loss of retinal axonal and neuronal integrity can occur even without a history of optic neuritis. Interest in vision in multiple sclerosis is growing, partially in response to the development of sensitive visual function tests, structural markers such as optical coherence tomography and magnetic resonance imaging, and quality of life measures that give clinical meaning to the structure-function correlations that are unique to the afferent visual pathway. Abnormal eye movements also are common in multiple sclerosis, but quantitative assessment methods that can be applied in practice and clinical trials are not readily available. We summarize here a comprehensive literature search and the discussion at a recent international meeting of investigators involved in the development and study of visual outcomes in multiple sclerosis, which had, as its overriding goals, to review the state of the field and identify areas for future research. We review data and principles to help us understand the importance of vision as a model for outcomes assessment in clinical practice and therapeutic trials in multiple sclerosis. PMID:25433914

  10. Exploring the Implementation and Use of Outcome Measurement in Practice: A Qualitative Study

    ERIC Educational Resources Information Center

    Skeat, J.; Perry, A.

    2008-01-01

    Background: Outcome measurement is important to clinical practice--yet outcome many speech and language therapists find it difficult to apply measures in practice, and not all clinicians and services have been able to implement and/or use outcome measurement successfully. To date there has been little research to explain why implementation is…

  11. Physiotherapy treatment for atraumatic recurrent shoulder instability: early results of a specific exercise protocol using pathology-specific outcome measures

    PubMed Central

    Smith, Benjamin E; Osborne, Sally E; Wilkes, Sally R

    2015-01-01

    Background Recurrent shoulder instability is usually caused by a traumatic event resulting in structural pathology, although a small subgroup of patients experience symptomatic recurrent shoulder i