Science.gov

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. 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

  8. 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

  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. 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

  8. 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

  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. 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

  14. 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

  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. 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

  2. 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

  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. 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

  12. [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

  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. 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.

  5. 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

  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. 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

  1. 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

  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. 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

  12. 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

  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. 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

  5. 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

  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. 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…

  9. 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

  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. 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

  12. 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

  13. 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

  14. 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

  15. 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

  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. 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

  1. 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

  2. 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

  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. 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

  19. 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

  20. 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

  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. 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…

  4. 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

  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