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Sample records for patient-reported outcomes measurement

  1. Patient-Reported Outcome Measures for Hand and Wrist Trauma

    PubMed Central

    Dacombe, Peter Jonathan; Amirfeyz, Rouin; Davis, Tim

    2016-01-01

    Background: Patient-reported outcome measures (PROMs) are important tools for assessing outcomes following injuries to the hand and wrist. Many commonly used PROMs have no evidence of reliability, validity, and responsiveness in a hand and wrist trauma population. This systematic review examines the PROMs used in the assessment of hand and wrist trauma patients, and the evidence for reliability, validity, and responsiveness of each measure in this population. Methods: A systematic review of Pubmed, Medline, and CINAHL searching for randomized controlled trials of patients with traumatic injuries to the hand and wrist was carried out to identify the PROMs. For each identified PROM, evidence of reliability, validity, and responsiveness was identified using a further systematic review of the Pubmed, Medline, CINAHL, and reverse citation trail audit procedure. Results: The PROM used most often was the Disabilities of the Arm, Shoulder and Hand (DASH) questionnaire; the Patient-Rated Wrist Evaluation (PRWE), Gartland and Werley score, Michigan Hand Outcomes score, Mayo Wrist Score, and Short Form 36 were also commonly used. Only the DASH and PRWE have evidence of reliability, validity, and responsiveness in patients with traumatic injuries to the hand and wrist; other measures either have incomplete evidence or evidence gathered in a nontraumatic population. Conclusions: The DASH and PRWE both have evidence of reliability, validity, and responsiveness in a hand and wrist trauma population. Other PROMs used to assess hand and wrist trauma patients do not. This should be considered when selecting a PROM for patients with traumatic hand and wrist pathology. PMID:27418884

  2. The Patient-Reported Outcomes Measurement Information System (PROMIS)

    PubMed Central

    Cella, David; Yount, Susan; Rothrock, Nan; Gershon, Richard; Cook, Karon; Reeve, Bryce; Ader, Deborah; Fries, James F.; Bruce, Bonnie; Rose, Mattias

    2010-01-01

    Background The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Roadmap initiative (www.nihpromis.org) is a 5-year cooperative group program of research designed to develop, validate, and standardize item banks to measure patient-reported outcomes (PROs) relevant across common medical conditions. In this article, we will summarize the organization and scientific activity of the PROMIS network during its first 2 years. Design The network consists of 6 primary research sites (PRSs), a statistical coordinating center (SCC), and NIH research scientists. Governed by a steering committee, the network is organized into functional subcommittees and working groups. In the first year, we created an item library and activated 3 interacting protocols: Domain Mapping, Archival Data Analysis, and Qualitative Item Review (QIR). In the second year, we developed and initiated testing of item banks covering 5 broad domains of self-reported health. Results The domain mapping process is built on the World Health Organization (WHO) framework of physical, mental, and social health. From this framework, pain, fatigue, emotional distress, physical functioning, social role participation, and global health perceptions were selected for the first wave of testing. Item response theory (IRT)-based analysis of 11 large datasets supplemented and informed item-level qualitative review of nearly 7000 items from available PRO measures in the item library. Items were selected for rewriting or creation with further detailed review before the first round of testing in the general population and target patient populations. Conclusions The NIH PROMIS network derived a consensus-based framework for self-reported health, systematically reviewed available instruments and datasets that address the initial PROMIS domains. Qualitative item research led to the first wave of network testing which began in the second year. PMID:17443116

  3. Patient-reported outcome measures in arthroplasty registries

    PubMed Central

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

    2016-01-01

    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

  4. Patient-reported outcome measures for asthma: a systematic review

    PubMed Central

    Worth, Allison; Hammersley, Victoria; Knibb, Rebecca; Flokstra-de-Blok, Bertine; DunnGalvin, Audrey; Walker, Samantha; Dubois, Anthony E J; Sheikh, Aziz

    2014-01-01

    Background: Patient-reported outcome measures (PROMs) are measures of the outcome of treatment(s) reported directly by the patient or carer. There is increasing international policy interest in using these to assess the impact of clinical care. Aims: To identify suitably validated PROMs for asthma and examine their potential for use in clinical settings. Methods: We systematically searched MEDLINE, EMBASE and Web of Science databases from 1990 onwards to identify PROMs for asthma. These were critically appraised, then narratively synthesised. We also identified the generic PROMs commonly used alongside asthma-specific PROMs. Results: We identified 68 PROMs for asthma, 13 of which were selected through screening as being adequately developed to warrant full-quality appraisal: 8 for adults, 4 for children and 1 for a child’s caregiver. The PROMs found to be sufficiently well validated to offer promise for use in clinical settings were the Asthma Quality of Life Questionnaire (AQLQ) and mini-AQLQ for adults, and Pediatric Asthma Quality of Life Questionnaire for children. Rhinasthma was considered promising in simultaneously assessing the impact of asthma and rhinitis in those with coexistent disease. We identified 28 generic PROMs commonly used in conjunction with asthma-specific instruments. Conclusions: We identified asthma PROMs that offer the greatest potential for use in clinical settings. Further work is needed to assess whether these are fit-for-purpose for use in clinical practice with individual patients. In particular, there is a need to ensure these are validated for use in clinical settings, acceptable to patients, caregivers and clinicians, and yield meaningful outcomes. PMID:24964767

  5. 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

  6. Assessing patient reported outcome measures: A practical guide for gastroenterologists

    PubMed Central

    Hutchings, Hayley A; Williams, John G

    2014-01-01

    Gastrointestinal illnesses cause physical, emotional and social impact on patients. Patient reported outcome measures (PROMs) are increasingly used in clinical decision-making, clinical research and approval of new therapies. In the last decade, there has been a rapid increase in the number of PROMs in gastroenterology and, therefore, the choice between which of these PROMs to use can be difficult. Not all PROM instruments currently used in research and clinical practice in gastroenterology have gone through a rigorous development methodology. New drugs and therapies will not have access to the market if the PROMs used in their clinical trials are not validated according to the guidelines of the international agencies. Therefore, it is important to know the required properties of PROMs when choosing or evaluating a drug or a clinical intervention. This paper reviews the current literature on how to assess the validity and reliability of PROMs. It summarises the required properties into a practical guide for gastroenterologists to use in assessing an instrument for use in clinical practice or research. PMID:25452841

  7. Measurement properties of patient-reported outcome measures (PROMS) in Patellofemoral Pain Syndrome: a systematic review.

    PubMed

    Green, Andrew; Liles, Clive; Rushton, Alison; Kyte, Derek G

    2014-12-01

    This systematic review investigated the measurement properties of disease-specific patient-reported outcome measures used in Patellofemoral Pain Syndrome. Two independent reviewers conducted a systematic search of key databases (MEDLINE, EMBASE, AMED, CINHAL+ and the Cochrane Library from inception to August 2013) to identify relevant studies. A third reviewer mediated in the event of disagreement. Methodological quality was evaluated using the validated COSMIN (Consensus-based Standards for the Selection of Health Measurement Instruments) tool. Data synthesis across studies determined the level of evidence for each patient-reported outcome measure. The search strategy returned 2177 citations. Following the eligibility review phase, seven studies, evaluating twelve different patient-reported outcome measures, met inclusion criteria. A 'moderate' level of evidence supported the structural validity of several measures: the Flandry Questionnaire, Anterior Knee Pain Scale, Functional Index Questionnaire, Eng and Pierrynowski Questionnaire and Visual Analogue Scales for 'usual' and 'worst' pain. In addition, there was a 'Limited' level of evidence supporting the test-retest reliability and validity (cross-cultural, hypothesis testing) of the Persian version of the Anterior Knee Pain Scale. Other measurement properties were evaluated with poor methodological quality, and many properties were not evaluated in any of the included papers. Current disease-specific outcome measures for Patellofemoral Pain Syndrome require further investigation. Future studies should evaluate all important measurement properties, utilising an appropriate framework such as COSMIN to guide study design, to facilitate optimal methodological quality.

  8. Voice-Related Patient-Reported Outcome Measures: A Systematic Review of Instrument Development and Validation

    ERIC Educational Resources Information Center

    Francis, David O.; Daniero, James J.; Hovis, Kristen L.; Sathe, Nila; Jacobson, Barbara; Penson, David F.; Feurer, Irene D.; McPheeters, Melissa L.

    2017-01-01

    Purpose: The purpose of this study was to perform a comprehensive systematic review of the literature on voice-related patient-reported outcome (PRO) measures in adults and to evaluate each instrument for the presence of important measurement properties. Method: MEDLINE, the Cumulative Index of Nursing and Allied Health Literature, and the Health…

  9. An introduction to patient-reported outcome measures in ophthalmic research

    PubMed Central

    Denniston, A K; Kyte, D; Calvert, M; Burr, J M

    2014-01-01

    Clinical outcomes, such as quantifying the extent of visual field loss by automated perimetry, are valued highly by health professionals, but such measures do not capture the impact of the condition on a patient's life. Patient-reported outcomes describe any report or measure of health reported by the patient, without external interpretation by a clinician or researcher. In this review, we discuss the value of the measures that capture this information (patient-reported outcome measures; PROMs), and why they are important to both the clinician and the researcher. We also consider issues around developing or selecting a PROM for ophthalmic research, the emerging challenges around conducting and reporting PROMs in clinical trials and highlight best practice for their use. Search terms for this review comprised: (1) (patient-reported outcomes OR patient-reported outcome measures) AND (2) randomised controlled trials AND (3) limited to ophthalmic conditions. These terms were expanded as follows: (((‘patients'(MeSH Terms) OR ‘patients'(All Fields) OR ‘patient'(All Fields)) AND (‘research report'(MeSH Terms) OR (‘research'(All Fields) AND ‘report'(All Fields)) OR ‘research report'(All Fields) OR ‘reported'(All Fields)) AND outcomes(All Fields)) OR ((‘patients'(MeSH Terms) OR ‘patients'(All Fields) OR ‘patient'(All Fields)) AND (‘research report'(MeSH Terms) OR (‘research'(All Fields) AND ‘report'(All Fields)) OR ‘research report'(All Fields) OR ‘reported'(All Fields) AND (‘outcome assessment (health care)'(MeSH Terms) OR (‘outcome'(All Fields) AND ‘assessment'(All Fields) AND ‘(health'(All Fields) AND ‘care)'(All Fields)) OR ‘outcome assessment (health care)'(All Fields) OR (‘outcome'(All Fields) AND ‘measures'(All Fields)) OR ‘outcome measures'(All Fields)))) AND (‘randomized controlled trial'(Publication Type) OR ‘randomized controlled trials as topic'(MeSH Terms) OR ‘randomised controlled trials'(All Fields) OR

  10. Patient-reported outcome measures in burning mouth syndrome - a review of the literature.

    PubMed

    Ni Riordain, R; McCreary, C

    2013-04-01

    Oral Diseases (2013) 19, 230-235 This review aims to investigate the patient-reported outcomes currently used in the burning mouth syndrome literature and to explore whether any standardisation of such measures has taken place. Electronic databases were searched for all types of burning mouth syndrome studies using patient-reported outcome measures. Studies were selected by predefined inclusion criteria. Copies of the papers obtained were thoroughly reviewed. A study-specific data extraction form was used, allowing papers to be reviewed in a standardised manner. The initial literature search yielded a total of 173 citations, 43 of which were deemed suitable for inclusion in this study. Symptom severity and symptomatic relief were reported as a patient-reported outcome measure in 40 of the studies and quantified most commonly using a visual analogue scale. Quality of life was reported in 13 studies included in this review. Depression and/or anxiety was reported in 14 of the studies. As is evident from the variety of questionnaires and instruments used in the evaluation of the impact of burning mouth syndrome on patients' lives, no standardisation of patient outcomes has yet been achieved.

  11. 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.

  12. Longitudinal evaluation of Patient Reported Outcomes Measurement Information Systems (PROMIS) measures in pediatric chronic pain

    PubMed Central

    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

    2015-01-01

    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 seven 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. PROMIS measures were administered at the initial visit and two follow-up visits at an outpatient chronic pain clinic (CPC; N=82) and at an intensive amplified pain day-treatment program (AMP; 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 AMP 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 seven 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

  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.

  14. Systematic review of patient-reported outcome measures (PROMs) for assessing disease activity in rheumatoid arthritis

    PubMed Central

    de Jonge, Marieke J; Fransen, Jaap; Kievit, Wietske; van Riel, Piet LCM

    2016-01-01

    Patient assessment of disease activity in rheumatoid arthritis (RA) may be useful in clinical practice, offering a patient-friendly, location independent, and a time-efficient and cost-efficient means of monitoring the disease. The objective of this study was to identify patient-reported outcome measures (PROMs) to assess disease activity in RA and to evaluate the measurement properties of these measures. Systematic literature searches were performed in the PubMed and EMBASE databases to identify articles reporting on clinimetric development or evaluation of PROM-based instruments to monitor disease activity in patients with RA. 2 reviewers independently selected articles for review and assessed their methodological quality based on the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) recommendations. A total of 424 abstracts were retrieved for review. Of these abstracts, 56 were selected for reviewing the full article and 34 articles, presenting 17 different PROMs, were finally included. Identified were: Rheumatoid Arthritis Disease Activity Index (RADAI), RADAI-5, Patient-based Disease Activity Score (PDAS) I & II, Patient-derived Disease Activity Score with 28-joint counts (Pt-DAS28), Patient-derived Simplified Disease Activity Index (Pt-SDAI), Global Arthritis Score (GAS), Patient Activity Score (PAS) I & II, Routine Assessment of Patient Index Data (RAPID) 2–5, Patient Reported Outcome-index (PRO-index) continuous (C) & majority (M), Patient Reported Outcome CLinical ARthritis Activity (PRO-CLARA). The quality of reports varied from poor to good. Typically 5 out of 10 clinimetric domains were covered in the validations of the different instruments. The quality and extent of clinimetric validation varied among PROMs of RA disease activity. The Pt-DAS28, RADAI, RADAI-5 and RAPID 3 had the strongest and most extensive validation. The measurement properties least reported and in need of more evidence were: reliability

  15. 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.

  16. Extending basic principles of measurement models to the design and validation of Patient Reported Outcomes.

    PubMed

    Atkinson, Mark J; Lennox, Richard D

    2006-09-22

    A recently published article by the Scientific Advisory Committee of the Medical Outcomes Trust presents guidelines for selecting and evaluating health status and health-related quality of life measures used in health outcomes research. In their article, they propose a number of validation and performance criteria with which to evaluate such self-report measures. We provide an alternate, yet complementary, perspective by extending the types of measurement models which are available to the instrument designer. During psychometric development or selection of a Patient Reported Outcome measure it is necessary to determine which, of the five types of measurement models, the measure is based on; 1) a Multiple Effect Indicator model, 2) a Multiple Cause Indicator model, 3) a Single Item Effect Indicator model, 4) a Single Item Cause Indicator model, or 5) a Mixed Multiple Indicator model. Specification of the measurement model has a major influence on decisions about item and scale design, the appropriate application of statistical validation methods, and the suitability of the resulting measure for a particular use in clinical and population-based outcomes research activities.

  17. Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science

    PubMed Central

    2011-01-01

    Interest in the patient's views of his or her illness and treatment has increased dramatically. However, our ability to appropriately measure such issues lags far behind the level of interest and need. Too often such measurement is considered to be a simple and trivial activity that merely requires the application of common sense. However, good quality measurement of patient-reported outcomes is a complex activity requiring considerable expertise and experience. This review considers the most important issues related to such measurement in the context of chronic disease and details how instruments should be developed, validated and adapted for use in additional languages. While there is often consensus on how best to undertake these activities, there is generally little evidence to support such accord. The present article questions these orthodox views and suggests alternative approaches that have been shown to be effective. PMID:21756344

  18. Patient reported outcome measures of quality of end-of-life care: A systematic review.

    PubMed

    Kearns, Tara; Cornally, Nicola; Molloy, William

    2017-02-01

    End-of-life (EoL) care(1) is increasingly used as a generic term in preference to palliative care or terminal care, particularly with reference to individuals with chronic disease, who are resident in community and long-term care (LTC) settings. This review evaluates studies based on patient reported outcome measures (PROMS) of quality of EoL care across all health-care settings. From 1041 citations, 12 studies were extracted by searches conducted in EBSCO, Scopus, Web of Science, PubMed, Cochrane, Open Grey and Google Scholar databases. At present, the evidence base for EoL care is founded on cancer care. This review highlights the paucity of studies that evaluate quality of EoL care for patients with chronic disease outside the established cancer-acute care paradigm, particularly in LTC. This review highlights the absence of any PROMs for the estimated 60% of patients in LTC with cognitive impairment. Patient-reported outcomes (PROs) are critical to understanding how EoL care services and practices affect patients' health and EoL experience. PROMs describe the quality of care from the patient's perspective and add balance to existing clinical or proxy-derived knowledge on the quality of care and services provided.

  19. An introduction to patient-reported outcome measures (PROMs) in physiotherapy.

    PubMed

    Kyte, D G; Calvert, M; van der Wees, P J; ten Hove, R; Tolan, S; Hill, J C

    2015-06-01

    The use of patient-reported outcome measures (PROMs) is set to rise in physiotherapy. PROMs provide additional 'patient-centred' data which is unique in capturing the patient's own opinion on the impact of their disease or disorder, and its treatment, on their life. Thus, PROMs are increasingly used by clinicians to guide routine patient care, or for the purposes of audit, and are already firmly embedded in clinical research. This article seeks to summarise the key aspects of PROM use for physiotherapists, both in routine clinical practice and in the research setting, and highlights recent developments in the field. Generic and condition-specific PROMs are defined and examples of commonly used measures are provided. The selection of appropriate PROMs, and their effective use in the clinical and research settings is discussed. Finally, existing barriers to PROM use in practice are identified and recent physiotherapy PROM initiatives, led by the Royal Dutch Society for Physical Therapy are explored.

  20. Implementing patient reported outcome measures (PROMs) in palliative care - users' cry for help

    PubMed Central

    2011-01-01

    Background Patient-reported outcome measurement (PROM) plays an increasingly important role in palliative care. A variety of measures exists and is used in clinical care, audit and research. However, little is known about professionals' views using these measures. The aim of this study is to describe the use and experiences of palliative care professionals with outcome measures. Methods A web-based online survey was conducted in Europe and Africa. Professionals working in clinical care, audit and research in palliative care were invited to the survey via national palliative care associations and various databases. Invitation e-mails were sent with a link to the questionnaire. Results Overall participation rate 42% (663/1592), overall completion rate 59% (392/663). The majority of respondents were female (63.4%), mean age 46 years (SD 9). 68.1% respondents from Europe and 73.3% from Africa had experiences with outcome measures in palliative care. Non-users reported time constraints, burden, lack of training and guidance as main reasons. In clinical care/audit, assessment of patients' situation, monitoring changes and evaluation of services were main reasons for use. Choice of OMs for research was influenced by validity of the instrument in palliative care and comparability with international literature. Main problems were related to patient characteristics, staff, and outcome measures. Participants expressed the need for more guidance and training in the use of PROMs. Conclusions Professionals need more support for the use and implementation of PROMs in clinical practice and research through training and guidance in order to improve patient care. PMID:21507232

  1. Patient-reported outcome measures: an on-line system empowering patient choice

    PubMed Central

    Wilson, J; Arshad, F; Nnamoko, N; Whiteman, A; Ring, J; Roy, B

    2014-01-01

    An innovative web-based system was developed to allow patient-reported outcome measures (PROMs) to be easily administered. Stakeholders guided the design and implementation. The software gives patients access to their current and previous scores. This pilot study focused on patients undergoing arthroscopic subacromial decompression, evaluated using the Oxford shoulder score (OSS). Patients showing good improvement in their OSS were offered the choice to return for routine follow-up clinic appointments, or continue rehabilitation, reassured by their improved score. Thirty-six of 117 patients were eligible. Thirty of these (83%) were opted to avoid further clinics. PROMs 2.0 can be used for any medical intervention with a validated PROM. Evolution and refinement is ongoing. Funding has been granted for 12 primary and secondary healthcare trusts to implement PROMs 2.0. Further work is needed to assess economic impact, patient views and satisfaction with the process. PMID:24013090

  2. Development of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) Gastrointestinal Symptom Scales

    PubMed Central

    Spiegel, Brennan M.R.; Hays, Ron D.; Bolus, Roger; Melmed, Gil Y.; Chang, Lin; Whitman, Cynthia; Khanna, Puja P.; Paz, Sylvia H.; Hays, Tonya; Reise, Steve; Khanna, Dinesh

    2014-01-01

    OBJECTIVES The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS®) is a standardized set of patient-reported outcomes (PROs) that cover physical, mental, and social health. The aim of this study was to develop the NIH PROMIS gastrointestinal (GI) symptom measures. METHODS We first conducted a systematic literature review to develop a broad conceptual model of GI symptoms. We complemented the review with 12 focus groups including 102 GI patients. We developed PROMIS items based on the literature and input from the focus groups followed by cognitive debriefing in 28 patients. We administered the items to diverse GI patients (irritable bowel syndrome (IBS), inflammatory bowel disease (IBD), systemic sclerosis (SSc), and other common GI disorders) and a census-based US general population (GP) control sample. We created scales based on confirmatory factor analyses and item response theory modeling, and evaluated the scales for reliability and validity. RESULTS A total of 102 items were developed and administered to 865 patients with GI conditions and 1,177 GP participants. Factor analyses provided support for eight scales: gastroesophageal reflux (13 items), disrupted swallowing (7 items), diarrhea (5 items), bowel incontinence/soilage (4 items), nausea and vomiting (4 items), constipation (9 items), belly pain (6 items), and gas/bloat/flatulence (12 items). The scales correlated significantly with both generic and disease-targeted legacy instruments, and demonstrate evidence of reliability. CONCLUSIONS Using the NIH PROMIS framework, we developed eight GI symptom scales that can now be used for clinical care and research across the full range of GI disorders. PMID:25199473

  3. 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

  4. The importance of rating scales in measuring patient-reported outcomes

    PubMed Central

    2012-01-01

    Background A critical component that influences the measurement properties of a patient-reported outcome (PRO) instrument is the rating scale. Yet, there is a lack of general consensus regarding optimal rating scale format, including aspects of question structure, the number and the labels of response categories. This study aims to explore the characteristics of rating scales that function well and those that do not, and thereby develop guidelines for formulating rating scales. Methods Seventeen existing PROs designed to measure vision-related quality of life dimensions were mailed for self-administration, in sets of 10, to patients who were on a waiting list for cataract extraction. These PROs included questions with ratings of difficulty, frequency, severity, and global ratings. Using Rasch analysis, performance of rating scales were assessed by examining hierarchical ordering (indicating categories are distinct from each other and follow a logical transition from lower to higher value), evenness (indicating relative utilization of categories), and range (indicating coverage of the attribute by the rating scale). Results The rating scales with complicated question format, a large number of response categories, or unlabelled categories, tended to be dysfunctional. Rating scales with five or fewer response categories tended to be functional. Most of the rating scales measuring difficulty performed well. The rating scales measuring frequency and severity demonstrated hierarchical ordering but the categories lacked even utilization. Conclusion Developers of PRO instruments should use a simple question format, fewer (four to five) and labelled response categories. PMID:22794788

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

    PubMed

    Brédart, Anne; Marrel, Alexia; Abetz-Webb, Linda; Lasch, Kathy; Acquadro, Catherine

    2014-02-05

    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.

  6. 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

  7. Measuring the value of treatment to patients: patient-reported outcomes in drug development.

    PubMed

    Willke, Richard J

    2008-02-01

    Patient-reported outcomes (PROs) can be important measures of the impact and value of new drug treatments to patients. Recently, both multisector stakeholder groups and the U.S. Food and Drug Administration have carefully considered and issued guidance on best practices for the use of PROs in measuring treatment impact. When best practices are followed and PRO data are appropriately included in drug development strategy and clinical trials, these data can be part of the evidence submitted for drug approval and included in drug labeling. One study showed that PRO data were included in 30% of a sample of new drug labels and were more concentrated in certain therapeutic areas, such as anti-inflammatory agents, vaccines, gastrointestinal agents, and respiratory and urologic agents. PRO data included in labeling, or generated in a similar scientific manner, may often then be used in other communication vehicles, such as formulary submission dossiers, journal or direct-to-consumer advertisements, publications, or continuing medical education. Meaningful and reliable PRO results regarding the effects of new treatments on how patients feel and function provide useful information to those who must make decisions about the availability and utilization of such treatments.

  8. 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.

  9. Validation of two generic patient-reported outcome measures in patients with type 2 diabetes

    PubMed Central

    Matza, Louis S; Boye, Kristina S; Yurgin, Nicole

    2007-01-01

    Background Prior to using a generic patient-reported outcome measure (PRO), the measure should be validated within the target population. The purpose of the current study was to validate two generic measures in patients with type 2 diabetes. Methods Patients with type 2 diabetes in Scotland and England completed two generic measures: EQ-5D and Psychological General Well-Being Index (PGWB). Two diabetes-specific measures were administered: ADS and DSC-R. Analyses assessed reliability and validity. Results There were 130 participants (53 Scotland; 77 England; 64% male; mean age = 55.7 years). Responses on the EQ-5D and PGWB reflected moderate impairment consistent with previous diabetes samples: mean EQ-5D Index score, 0.75; EQ-5D VAS, 68.8; PGWB global score, 67.9. All scales of the PGWB demonstrated good internal consistency reliability (Cronbach's alpha = 0.77 to 0.97). The EQ-5D and PGWB demonstrated convergent validity through significant correlations with the ADS (r = 0.48 to 0.61), DSC-R scales (r = 0.33 to 0.81 except ophthalmology subscale), and Body Mass Index (r = 0.15 to 0.38). The EQ-5D and PGWB discriminated between groups of patients known to differ in diabetes-related characteristics (e.g., history of hypoglycemia). Conclusion Results support the use of the EQ-5D and PGWB among patients with type 2 diabetes, possibly in combination with condition-specific measures. PMID:17672906

  10. 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

  11. A Review of HIV-Specific Patient-Reported Outcome Measures.

    PubMed

    Engler, Kim; Lessard, David; Lebouché, Bertrand

    2016-09-16

    The use of patient-reported outcome (PRO) measures to provide added feedback to health providers is receiving interest as a means of improving clinical care and patient outcomes, and contributing to more patient-centered care. In human immunodeficiency virus (HIV), while PROs are used in research, their application in clinical practice has been limited despite their potential utility. PRO selection is an important consideration when contemplating their use. As past reviews of PROs in HIV have focused on particular areas (e.g. disability, satisfaction with care), a more comprehensive review could better inform on the available instruments and their scope. This article reviews HIV-specific PROs to produce an inventory and to identify the central concepts targeted over time. Seven databases were searched (HAPI, MEDLINE, PsychINFO, PubMed, EMBASE, CINAHL, Google Scholar), generating 14,794 records for evaluation. From these records, 117 HIV-specific PROs were identified and categorized based on a content analysis of their targeted concept: Health-Related Quality of Life (23; 20 %), ART and Adherence-Related Views and Experiences (19; 16 %), Healthcare-Related Views and Experiences (15; 13 %), Psychological Challenges (12; 10 %), Symptoms (12; 10 %), Psychological Resources (10; 9 %), HIV Self-Management and Self-Care (8; 7 %), HIV-Related Stigma (8; 7 %), Body and Facial Appearance (4; 3 %), Social Support (3; 3 %), Sexual and Reproductive Health (2; 2 %), and Disability (1; 1 %). This review highlights the variety and evolution of HIV-specific PROs, with the arrival of seven categories of PROs only after the advent of highly-active antiretroviral therapy. Our inventory also offers a useful resource. However, the interest of further HIV-specific PRO development should be explored in sexual health, which received little independent attention.

  12. 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

    2013-12-20

    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

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

    PubMed Central

    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

  14. What is sufficient evidence for the reliability and validity of patient-reported outcome measures?

    PubMed

    Frost, Marlene H; Reeve, Bryce B; Liepa, Astra M; Stauffer, Joseph W; Hays, Ron D

    2007-01-01

    This article focuses on the necessary psychometric properties of a patient-reported outcomes (PROs) measure. Topics include the importance of reliability and validity, psychometric approaches used to provide reliability and validity estimates, the kinds of evidence needed to indicate that a PRO has a sufficient level of reliability and validity, contexts that may affect psychometric properties, methods available to evaluate PRO instruments when the context varies, and types of reliability and validity testing that are appropriate during different phases of clinical trials. Points discussed include the perspective that the psychometric properties of reliability and validity are on a continuum in which the more evidence one has, the greater confidence there is in the value of the PRO data. Construct validity is the type of validity most frequently used with PRO instruments as few "gold standards" exist to allow the use of criterion validity and content validity by itself only provides beginning evidence of validity. Several guidelines are recommended for establishing sufficient evidence of reliability and validity. For clinical trials, a minimum reliability threshold of 0.70 is recommended. Sample sizes for testing should include at least 200 cases and results should be replicated in at least one additional sample. At least one full report on the development of the instrument and one on the use of the instrument are deemed necessary to evaluate the PRO psychometric properties. Psychometric testing ideally occurs before the initiation of Phase III trials. When testing does not occur prior to a Phase III trial, considerable risk is posed in relation to the ability to substantiate the use of the PRO data. Various qualitative (e.g., focus groups, behavioral coding, cognitive interviews) and quantitative approaches (e.g., differential item functioning testing) are useful in evaluating the reliability and validity of PRO instruments.

  15. A systematic review of patient-reported outcome measures of neuropathy in children, adolescents and young adults

    PubMed Central

    Sung, Lillian; Stark, Daniel; Frazier, A. Lindsay; Rosenberg, Abby R.

    2017-01-01

    Purpose Peripheral neuropathy is an important, yet poorly studied, side effect of pediatric cancer treatment. There are many measures of patient-reported peripheral neuropathy in adults but very few in children. We aimed to systematically review and summarize reliable and valid patient-reported peripheral neuropathy scales used in pediatrics. Methods Four major electronic databases (Medline, Embase, EBSCO Host in Cumulative Index to Nursing and Allied Health Literature, and PsycINFO) were reviewed for studies that measured peripheral neuropathy in pediatric patients. Studies eligible for inclusion were those that described use of any patient-reported scale of peripheral neuropathy among children, adolescents, and young adults with any underlying diagnosis (not limited to cancer). Results From a total of 765 articles retrieved, 5 met eligibility criteria and were included. One was a neuropathy symptom score used in patients with diabetes, and the remaining four were in oncology patients and all were based on the total neuropathy score. All involved objective assessments conducted by trained professionals; none relied purely on patient report. Conclusions There are no validated instruments that consist solely of a patient-reported outcome measure of neuropathy in pediatrics and adolescents. Because the clinical evaluation of neuropathy requires specialized training, it is not generalizable in large studies conducted in many diverse institutions. Future studies should validate adult patient-reported neuropathy scales in pediatric and adolescent populations, or develop novel instruments designed for this population. PMID:27037813

  16. 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

  17. 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

  18. 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

  19. 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

  20. Patient-reported outcome measures in a population of medically indigent patients with systemic lupus erythematosus in Puerto Rico

    PubMed Central

    Rodríguez-Rivera, Diana V; Rodríguez-Navedo, Yerania; Nieves-Plaza, Mariely; Vilá, Luis M

    2016-01-01

    Objective: To determine patient-reported outcomes measures in indigent patients with systemic lupus erythematosus receiving their healthcare through the Puerto Rico government managed care system and compare these measures with non-indigent patients treated in a private fee-for-service setting. Methods: A cross-sectional study was conducted in a cohort of 98 Puerto Ricans with systemic lupus erythematosus. Patients from the public group (n = 40) were treated in a university-based specialized systemic lupus erythematosus clinic and the private group (n = 58) in a community-based rheumatology practice. Demographic and clinical features and patient-reported outcomes measures per LupusPRO instrument were determined. LupusPRO captures quality-of-life measures in 12 domains. Differences among study groups were examined using chi-square, Fisher’s exact, t-tests, and the Wilcoxon signed-rank test. Results: The mean (standard deviation) age of the study population was 44.9 (12.0) years; 94 (95.9%) were women. Patients in the public setting were younger and were more likely to have renal disease and elevated anti-double-stranded DNA antibodies, and being treated with azathioprine and cyclophosphamide. Patients from the public sector were more likely to have better quality-of-life measures in the LupusPRO domains of pain/vitality and coping. No significant differences were observed for the domains of lupus symptoms, physical health, emotional health, body image, cognition, procreation, lupus medications, desires/goals, social support, and satisfaction with medical care. Conclusion: Despite having a lower socioeconomic status and worse clinical status, systemic lupus erythematosus patients from the public sector had equal or better patient-reported outcomes measures than those treated in the private setting. This favorable outcome may be associated with the comprehensive healthcare received by these patients in a specialized lupus clinic. PMID:27721978

  1. Patient empowerment: The need to consider it as a measurable patient-reported outcome for chronic conditions

    PubMed Central

    2012-01-01

    Background Health policy in the UK and elsewhere is prioritising patient empowerment and patient evaluations of healthcare. Patient reported outcome measures now take centre-stage in implementing strategies to increase patient empowerment. This article argues for consideration of patient empowerment itself as a directly measurable patient reported outcome for chronic conditions, highlights some issues in adopting this approach, and outlines a research agenda to enable healthcare evaluation on the basis of patient empowerment. Discussion Patient empowerment is not a well-defined construct. A range of condition-specific and generic patient empowerment questionnaires have been developed; each captures a different construct e.g. personal control, self-efficacy/self-mastery, and each is informed by a different implicit or explicit theoretical framework. This makes it currently problematic to conduct comparative evaluations of healthcare services on the basis of patient empowerment. A case study (clinical genetics) is used to (1) illustrate that patient empowerment can be a valued healthcare outcome, even if patients do not obtain health status benefits, (2) provide a rationale for conducting work necessary to tighten up the patient empowerment construct (3) provide an exemplar to inform design of interventions to increase patient empowerment in chronic disease. Such initiatives could be evaluated on the basis of measurable changes in patient empowerment, if the construct were properly operationalised as a patient reported outcome measure. To facilitate this, research is needed to develop an appropriate and widely applicable generic theoretical framework of patient empowerment to inform (re)development of a generic measure. This research should include developing consensus between patients, clinicians and policymakers about the content and boundaries of the construct before operationalisation. This article also considers a number of issues for society and for healthcare

  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. 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.

  4. Development and Validation of Patient-Reported Outcome Measures for Sleep Disturbance and Sleep-Related Impairments

    PubMed Central

    Buysse, Daniel J.; Yu, Lan; Moul, Douglas E.; Germain, Anne; Stover, Angela; Dodds, Nathan E.; Johnston, Kelly L.; Shablesky-Cade, Melissa A.; Pilkonis, Paul A.

    2010-01-01

    Study Objectives: To develop an archive of self-report questions assessing sleep disturbance and sleep-related impairments (SRI), to develop item banks from this archive, and to validate and calibrate the item banks using classic validation techniques and item response theory analyses in a sample of clinical and community participants. Design: Cross-sectional self-report study. Setting: Academic medical center and participant homes. Participants: One thousand nine hundred ninety-three adults recruited from an Internet polling sample and 259 adults recruited from medical, psychiatric, and sleep clinics. Interventions: None. Measurements and Results: This study was part of PROMIS (Patient-Reported Outcomes Information System), a National Institutes of Health Roadmap initiative. Self-report item banks were developed through an iterative process of literature searches, collecting and sorting items, expert content review, qualitative patient research, and pilot testing. Internal consistency, convergent validity, and exploratory and confirmatory factor analysis were examined in the resulting item banks. Factor analyses identified 2 preliminary item banks, sleep disturbance and SRI. Item response theory analyses and expert content review narrowed the item banks to 27 and 16 items, respectively. Validity of the item banks was supported by moderate to high correlations with existing scales and by significant differences in sleep disturbance and SRI scores between participants with and without sleep disorders. Conclusions: The PROMIS sleep disturbance and SRI item banks have excellent measurement properties and may prove to be useful for assessing general aspects of sleep and SRI with various groups of patients and interventions. Citation: Buysse DJ; Yu L; Moul DE; Germain A; Stover A; Dodds NE; Johnston KL; Shablesky-Cade MA; Pilkonis PA. Development and validation of patient-reported outcome measures for sleep disturbance and sleep-related impairments. SLEEP 2010

  5. Hand-related physical function in rheumatic hand conditions: a protocol for developing a patient-reported outcome measurement instrument

    PubMed Central

    Klokker, Louise; Terwee, Caroline B; Wæhrens, Eva Ejlersen; Henriksen, Marius; Nolte, Sandra; Liegl, Gregor; Kloppenburg, Margreet; Westhoven, Rene; Wittoek, Ruth; Kjeken, Ingvild; Haugen, Ida K; Schalet, Ben; Gershon, Richard; Bliddal, Henning

    2016-01-01

    Introduction There is no consensus about what constitutes the most appropriate patient-reported outcome measurement (PROM) instrument for measuring physical function in patients with rheumatic hand conditions. Existing instruments lack psychometric testing and vary in feasibility and their psychometric qualities. We aim to develop a PROM instrument to assess hand-related physical function in rheumatic hand conditions. Methods and analysis We will perform a systematic search to identify existing PROMs to rheumatic hand conditions, and select items relevant for hand-related physical function as well as those items from the Patient Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) item bank that are relevant to patients with rheumatic hand conditions. Selection will be based on consensus among reviewers. Content validity of selected items will be established through the use of focus groups. If patients deem necessary, we will develop new items based on the patients' input. We will examine whether it is valid to score all selected and developed items on the same scale as the original items from the PROMIS PF item bank. Our analyses will follow the methods used for calibrating the original PROMIS PF item bank in US samples, which were largely based on the general PROMIS approach. Ethics and dissemination This study will be carried out in accordance with the Helsinki Declaration. Ethics approvals will be obtained where necessary, and signed informed consent will be obtained from all participants. We aim to disseminate the results of the study through publication in international peer-reviewed journals and at international conferences. PMID:27974367

  6. 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.

  7. An experience with the Patient-Reported Outcomes Measurement Information System: pros and cons and unanswered questions.

    PubMed

    Badger, Terry A; Heitkemper, Margaret; Lee, Kathryn A; Bruner, Deborah Watkins

    2014-01-01

    The goal of the Patient-Reported Outcomes Measurement Information System (PROMIS) is to create efficient, reliable, and valid assessments of adult and child health. The nursing science literature in which PROMIS measures are used is rapidly expanding. Investigators have been encouraged to consider the integration of PROMIS measures into both descriptive studies and clinical trials. Doing this has created opportunities and challenges for investigators. This article highlights three projects to show the perspectives of nurse scientists who incorporated PROMIS measures into their research. The first project describes advantages of PROMIS to allow for comparisons of a study population with a national sample and to compliment legacy measures. The second project examines issues in the translation of tools for region-specific Hispanic populations. The third project provides a perspective on the use of PROMIS measures to capture cancer-related fatigue and to develop new components of a sexual function scale. As indicated by these three examples, nurse scientists can contribute an important role in moving the PROMIS initiative forward. Results from these types of projects also move symptom science forward within a more interdisciplinary approach to common measures of interest.

  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.

  10. 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

  11. 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

  12. 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.

  13. A methodology for successfully producing global translations of patient reported outcome measures for use in multiple countries.

    PubMed

    Two, Rebecca; Verjee-Lorenz, Aneesa; Clayson, Darren; Dalal, Mehul; Grotzinger, Kelly; Younossi, Zobair M

    2010-01-01

    The production of accurate and culturally relevant translations of patient reported outcome (PRO) measures is essential for the success of international clinical trials. Although there are many reports in publication regarding the translation of PRO measures, the techniques used to produce single translations for use in multiple countries (global translations) are not well documented. This article addresses this apparent lack of documentation and presents the methodology used to create global translations of the Chronic Liver Disease Questionnaire-Hepatitis C Virus (CLDQ-HCV). The challenges of creating a translation for use in multiple countries are discussed, and the criteria for a global translation project explained. Based on a thorough translation and linguistic validation methodology including a concept elaboration, multiple forward translations, two back translations, reviews by in-country clinicians and the instrument developer, pilot testing in each target country and multiple sets of proofreading, the key concept of the global translation methodology is consistent international harmonization, achieved through the involvement of linguists from each target country at every stage of the process. This methodology enabled the successful resolution of the translation issues encountered, and resulted in consistent translations of the CLDQ-HCV that were linguistically and culturally appropriate for all target countries.

  14. Content Validity of Patient-Reported Outcomes Measurement Information System (PROMIS) Items in the Context of HIV Clinical Care

    PubMed Central

    Edwards, Todd C.; Fredericksen, Rob J.; Crane, Heidi M.; Crane, Paul K.; Kitahata, Mari M.; Matthews, William C.; Mayer, Kenneth H.; Morales, Leo S.; Mugavero, Michael J.; Solorio, Rosa; Yang, Frances M.; Patrick, Donald L.

    2015-01-01

    Purpose To assess content validity and patient and provider prioritization of Patient Reported Outcomes Measurement Information System (PROMIS) Depression, Anxiety, Fatigue, and Alcohol Use items in the context of clinical care for people living with HIV (PLWH), and to develop and assess new items as needed. Methods We conducted concept elicitation interviews (n=161), item pool matching, prioritization focus groups (n=227 participants), and cognitive interviews (n=48) with English-speaking (~75%) and Spanish-speaking (~25%) PLWH from clinical sites in Seattle, San Diego, Birmingham, and Boston. For each domain we also conducted item review and prioritization with two HIV provider panels of 3 to 8 members each. Results Among items most highly prioritized by PLWH and providers were those that included information regarding personal impacts of the concept being assessed, in addition to severity level. Items that addressed impact were considered most actionable for clinical care. We developed additional items addressing this. For depression we developed items related to suicide and other forms of self-harm, and for all domains we developed items addressing impacts PLWH and/or providers indicated were particularly relevant to clinical care. Across the 4 domains, 16 new items were retained for further psychometric testing. Conclusion PLWH and providers had priorities for what they believed providers should know to provide optimal care for PLWH. Incorporation of these priorities into clinical assessments used in clinical care of PLWH may facilitate patient-centered care. PMID:26245710

  15. Reliability of a patient-reported outcome measure in schizophrenia: Results from back-to-back self-ratings.

    PubMed

    Takeuchi, Hiroyoshi; Fervaha, Gagan; Remington, Gary

    2016-10-30

    This study aimed to assess patient's capacity to perform a patient-reported outcome (PRO) measure (i.e., a self-rating scale) and examine its relationship with clinical characteristics including cognition. Fifty patients with schizophrenia were asked to rate the Subjective Well-being under Neuroleptics scale - Short form (SWNS) twice; the second rating was started immediately after they completed the first to minimize the gap between ratings. At the same time, the Positive and Negative Symptoms Scale (PANSS) and Brief Neurocognitive Assessment (BNA) were administered. The correlations between the two ratings for the SWNS total and each item scores were high (rs=0.94 and rs=0.60-0.84, respectively); however, for 16 (80%) of 20 items, 5 or more patients (i.e., ≥10%) demonstrated a>1 point score difference. There was no significant correlation between the SWNS total score difference and any clinical characteristics including age, education duration, illness duration, antipsychotic dose, psychopathology, and cognition. In contrast, the number of items with a>1 point score difference was significantly correlated with disorganized symptoms and overall severity (rs=0.29 for both), as well as working memory and global cognition (rs=-0.41 and rs=-0.40, respectively). These findings suggest that PROs should be interpreted with caution in patients with schizophrenia with prominent disorganization and cognitive impairment.

  16. 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

  17. Monitoring rheumatoid arthritis using an algorithm based on patient-reported outcome measures: a first step towards personalised healthcare

    PubMed Central

    Hendrikx, Jos; Fransen, Jaap; van Riel, Piet L C M

    2015-01-01

    Objectives The objective of this proof of concept study was to evaluate alerts generated by a patient-reported outcome measure (PROM)-based algorithm for monitoring patients with rheumatoid arthritis (RA). Methods The algorithm was constructed using an example PROM score of an equally weighted mean of visual analogue scale (VAS) general health, VAS disease activity and VAS pain. Based on the PROM score, red flags are generated in 2 instances: the target level of disease activity is not met; change in disease activity surpasses an early alert threshold. To reduce false alarms, 3 consecutive red flags are needed to trigger an alert to the physician. Time series data from patients included consecutively in the practice-based Nijmegen Early RA cohort were analysed to select an appropriate autoregressive integrated moving average (ARIMA) model. This allowed for advanced interpolation of PROM scores and weekly data evaluation. Alerts were evaluated against disease-modifying antirheumatic drug (DMARD)/biologic medication intensification registered in the cohort. Results Data of 165 patients followed in their second year postdiagnosis were analysed. In 89.8% of 716 visits, the algorithm did not generate an alert and medication was not escalated. Positive predictive value, sensitivity and specificity were 24.6%, 55.6% and 69.7%, respectively. Comparable performance was found when analyses were stratified for baseline Disease Activity Score 28-joint count (DAS28) level. Conclusions When using the algorithm to screen scheduled visits, the overall chance of missing patients in need of medication intensification is low. These findings provide evidence that an off-site monitoring system could aid in optimising the number and timing of face-to-face consultations of patients with their rheumatologists. PMID:26629364

  18. 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

  19. Pulmonary symptoms measured by the national institutes of health lung score predict overall survival, nonrelapse mortality, and patient-reported outcomes in chronic graft-versus-host disease.

    PubMed

    Palmer, Jeanne; Williams, Kirsten; Inamoto, Yoshihiro; Chai, Xiaoyu; Martin, Paul J; Tomas, Linus Santo; Cutler, Corey; Weisdorf, Daniel; Kurland, Brenda F; Carpenter, Paul A; Pidala, Joseph; Pavletic, Steven Z; Wood, William; Jacobsohn, David; Arai, Sally; Arora, Mukta; Jagasia, Madan; Vogelsang, Georgia B; Lee, Stephanie J

    2014-03-01

    The 2005 National Institutes of Health (NIH) Consensus Conference recommended assessment of lung function in patients with chronic graft-versus-host disease (GVHD) by both pulmonary function tests (PFTs) and assessment of pulmonary symptoms. We tested whether pulmonary measures were associated with nonrelapse mortality (NRM), overall survival (OS), and patient-reported outcomes (PRO). Clinician and patient-reported data were collected serially in a prospective, multicenter, observational study. Available PFT data were abstracted. Cox regression models were fit for outcomes using a time-varying covariate model for lung function measures and adjusting for patient and transplantation characteristics and nonlung chronic GVHD severity. A total of 1591 visits (496 patients) were used in this analysis. The NIH symptom-based lung score was associated with NRM (P = .02), OS (P = .02), patient-reported symptoms (P < .001) and functional status (P < .001). Worsening of NIH symptom-based lung score over time was associated with higher NRM and lower survival. All other measures were not associated with OS or NRM; although, some were associated with patient-reported lung symptoms. In conclusion, the NIH symptom-based lung symptom score of 0 to 3 is associated with NRM, OS, and PRO measures in patients with chronic GVHD. Worsening of the NIH symptom-based lung score was associated with increased mortality.

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

    PubMed

    2006-10-11

    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

  1. The development of a patient-reported outcome measure for assessing nighttime symptoms of chronic obstructive pulmonary disease

    PubMed Central

    2013-01-01

    Background The assessment of symptoms of chronic obstructive pulmonary disease (COPD) is important for monitoring and managing the disease and for evaluating outcomes of interventions. COPD patients experience symptoms during the day and night, and symptoms experienced at night often disturb sleep. The aim of this paper is to describe methods used to develop a patient-reported outcome (PRO) instrument for evaluating nighttime symptoms of COPD, and to document evidence for the content validity of the instrument. Methods Literature review and clinician interviews were conducted to inform discussion guides to explore patients’ nighttime COPD symptom experience. Data from focus groups with COPD patients was used to develop a conceptual framework and the content of a new PRO instrument. Patient understanding of the new instrument was assessed via cognitive interviews with COPD patients. Results The literature review confirmed that there is no instrument with evidence of content validity currently available to assess nighttime symptoms of COPD. Additionally, the literature review and clinician interviews suggested the need to understand patients’ experience of specific symptoms in order to evaluate nighttime symptoms of COPD. Analyses of patient focus group data (N = 27) supported saturation of concepts and aided in development of a conceptual framework. Items were generated using patients’ terminology to collect data on concepts in the framework including the occurrence and severity of COPD symptoms, use of rescue medication at night, and nocturnal awakening. Response options were chosen to reflect concepts that were salient to patients. Subsequent cognitive interviewing with ten COPD patients demonstrated that the items, response options, recall period, and instructions were understandable, relevant, and interpreted as intended. Conclusions A new PRO instrument, the Nighttime Symptoms of COPD Instrument (NiSCI), was developed with documented evidence of content

  2. Feedback on the FDA's February 2006 draft guidance on Patient Reported Outcome (PRO) measures from a developer of PRO measures.

    PubMed

    Bradley, Clare

    2006-10-09

    I believe that the FDA guidelines have already had an impact in encouraging good practice in the use of PROs. There are, however, important improvements that need to be made to the guidelines, particularly in the use of health status and quality of life terminology. It is essential to distinguish between health status and quality of life and to use both terms. Nothing is to be gained and a great deal will be lost if the term quality of life (which has been misused as an umbrella term in the past) is abandoned and replaced with the term health status. Patients want us to consider their quality of life as well as their health. To abandon the term would be to forget about their quality of life and focus only on their health. Patients are well able to tell us what quality of life means to them and to rate the impact of a condition on their quality of life if we use individualised quality of life measures and individualised condition-specific quality of life measures to allow them to do so. Although my experience with PRO measures would support many of the recommendations in the guidelines there are others that I would not fully agree with or would contradict on the basis of my own research evidence. I have provided references to that research and hope that the FDA will feel able to do the same when they finalize their guidelines.

  3. 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

  4. Measurement properties of patient-reported outcome measures (PROMs) used in adult patients with chronic kidney disease: a systematic review protocol

    PubMed Central

    Aiyegbusi, Olalekan Lee; Kyte, Derek; Cockwell, Paul; Marshall, Tom; Keeley, Thomas; Gheorghe, Adrian

    2016-01-01

    Introduction Chronic kidney disease (CKD) is associated with symptoms that can significantly reduce the quality of life (QoL) of patients. Patient-reported outcome measures (PROMs) may facilitate the assessment of the impact of disease and treatment on the QoL, from a patient perspective. PROMs can be used in research and routine clinical practice. Methods and analysis A systematic review of studies evaluating the measurement properties of PROMs in adults with CKD will be conducted. MEDLINE, EMBASE, PsycINFO and CINAHL Plus will be systematically searched from inception. Hand searching of reference lists and citations of included studies will be carried out. 2 reviewers will independently screen the titles and abstracts of all the studies retrieved during the systematic search to determine their eligibility. The COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) checklist will be used to appraise the methodological quality of the selected studies following the full-text review. Data on the study population, questionnaire characteristics and measurement properties will be extracted from the selected papers. Finally, a narrative synthesis of extracted data will be undertaken. Ethics and dissemination Ethical permissions are not required for this study as data from published research articles will be used. Findings will be disseminated through publication in a peer-reviewed journal and presented at conferences. This systematic review will provide a comprehensive assessment of the measurement properties of PROMs currently available for use in adult patients with CKD and present evidence which may inform the selection of measures for use in research and clinical practice. Trial registration number CRD42016035554. PMID:27733411

  5. Patient-reported outcomes in obsessive-compulsive disorder.

    PubMed

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

    2014-06-01

    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.

  6. 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

  7. 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

  8. 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

  9. Patient Reported Outcomes from Sacroiliac Joint Fusion

    PubMed Central

    McGowan, Shane M.; Audley, Brittany N.; Sokunbi, Gbolabo; Puccio, Steven T.

    2017-01-01

    Study Design Retrospective, case series. Purpose The purpose of this study is to determine morbidity, complications, and patient reported outcomes from minimally invasive sacroiliac joint (SIJ) fusion. Overview of Literature Lumbar back pain emanating from the SIJ can be surgically treated via a percutaneous approach in the appropriately selected patient with minimal morbidity and acceptable functional outcomes. Methods Patients diagnosed by >2 physical examination maneuvers and subjective relief from a computed tomography–guided lidocaine-bupivacaine-steroid injection underwent SIJ fusion after failing conservative management with a combination of oral anti-inflammatory medications, physical therapy, and pelvic belt stabilization. Perioperative data collected include estimated blood loss (EBL) and operative time. Oswestry disability index, 12-item short form health survey (SF-12), visual analogue score, and functional status were analyzed. All complications were noted. Results The study cohort of 45 cases (69% female) achieved postoperative survey follow-up at 9.9 and 32.3 months. SF-12 physical component summary statistically improved while all other scores were equivalent. Mean EBL and operative time were 22 mL and 36 minutes, respectively. Initial survey showed that 64% of patients discontinued narcotics (29/45), 71% do not use an assistive device (32/45), and 15.6% do not work due to pain (7/45). 73% of patients stated they would have the surgery again (33/45). For the second survey, 65% of patients discontinued narcotics (26/40), 70% did not use an assistive device (28/40), and 17.5% did not work due to pain (7/40). A history of thoracolumbar instrumentation (16/45) did not significantly affect outcomes. Three complications described by screw malposition with neurologic deficit (6.7%) were treated with screw repositioning (1 case) and removal of a single superior implant (2 cases) with time to revision of 2.2 months. All three ultimately had resolution of

  10. 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

  11. Patient-reported outcomes in borderline personality disorder.

    PubMed

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

    2014-06-01

    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.

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

    PubMed

    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.

  13. 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

  14. Feasibility of Using the Patient-Reported Outcomes Measurement Information System in Academic Health Centers: Case Series Design on Pain Reduction After Chiropractic Care

    PubMed Central

    Burke, Jeanmarie R.

    2014-01-01

    Objective The purpose of this study was to test the utility of Patient-Reported Outcomes Measurement Information System (PROMIS) as a resource for collecting data on patient-reported outcomes (PRO) within academic health centers at a chiropractic college; and, to describe changes in PRO following pragmatic chiropractic care incorporating instrument-assisted soft tissue mobilization (IASTM) on pain symptoms. Methods This was a pre-post intervention design without a control group (case series) involving 25 patients (14 females and 11 males; 40.5 ± 16.39 years, range 20-70 years) who completed their chiropractic care and their baseline and post-treatment pain assessments. The pragmatic chiropractic care intervention included both spinal manipulation and IASTM to treat pain symptoms. PRO’s were collected using PROMIS to measure pain behavior, pain interference and pain intensity. Results The average pre-post assessment interval was 33 ± 22.5 days (95% CI, 23-42 days). The durations of treatments ranged from one week to 10 weeks. The median number of IASTM treatments was six. Pre-post decreases in T-scores for pain behavior and pain interference were 55.5 to 48.4 and 57.7 to 48.4, respectively (P < .05). Only 12 patients had a baseline T-score for pain intensity greater than 50. The pre-post decrease in pain intensity T-scores for these 12 patients was from 53.4 to 40.9. Conclusion Within the limitations of a case series design, these data provide initial evidence on the utility of PROMIS instruments for clinical and research outcomes in chiropractic patients. PMID:25225465

  15. 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

  16. Patient-reported outcome measures and risk factors in a quality registry: a basis for more patient-centered diabetes care in Sweden.

    PubMed

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

    2014-11-26

    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.

  17. [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.

  18. [Brazilian-Portuguese translation and cultural adaptation of the sleep and wake disturbances domains of the Patient-Reported-Outcomes Measurement Information System (PROMIS)].

    PubMed

    Silva E Costa, Zilma Maria Severino; Pinto, Rogério de Melo Costa; Mendonça, Tânia Maria da Silva; Silva, Carlos Henrique Martins da

    2014-07-01

    Altered sleep and wakefulness affect individuals' mood, memory, and psychomotor performance and thus directly impact their quality of life. Tools to analyze the quality of these factors should be available for clinical evaluation. This study aimed to translate into Brazilian Portuguese and culturally adapt the Patient-Reported Outcomes Measurement Information System (PROMIS), specifically the sleep and wake disturbances domains. The translation and cultural adaptation processes followed the guidelines proposed by the Functional Assessment of Chronic Illness Therapy (FACIT) organization. The methodology included translation, reconciliation, back-translation, review by PROMIS, review by independent reviewers, pretest, and incorporation of the results into the final version. The Portuguese version of the sleep and wake disturbances domains presents semantic, idiomatic, cultural, and conceptual equivalence with the items in the source language.

  19. [Patient reported outcome monitoring using a tablet PC is suitable for measuring quality of life in patients with gliomas].

    PubMed

    Holzner, Bernhard; Schauer-Maurer, Gabriele; Stockhammer, Guenter; Muigg, Armin; Hutterer, Markus; Giesinger, Johannes

    2011-01-01

    A computer-based tool for the monitoring of patient-related outcomes, the "Computer-based Health Evaluation System" (CHES) was developed at the medical university of Innsbruck. The software-generated graphic QOL profiles were found to be an important tool for screening patients for clinically relevant problems and were successfully used in oncology, geriatrics, psychiatry, and psychosomatics. The authors report their experience with CHES in 34 patients with recurrent gliomas. The computer-based QoL monitoring was repeated in median 5.4 times per patients and found feasible and well accepted by patients and personnel.

  20. Socioeconomic deprivation and age are barriers to the online collection of patient reported outcome measures in orthopaedic patients

    PubMed Central

    Jenkins, PJ; Sng, S; Brooksbank, K; Brooksbank, AJ

    2016-01-01

    Introduction Questionnaires are used commonly to assess functional outcome and satisfaction in surgical patients. Although these have in the past been administered through written forms, there is increasing interest in the use of new technology to improve the efficiency of collection. The aim of this study was to assess the availability of internet access for a group of orthopaedic patients and the acceptability of online survey completion. Methods A total of 497 patients attending orthopaedic outpatient clinics were surveyed to assess access to the internet and their preferred means for completing follow-up questionnaires. Results Overall, 358 patients (72%) reported having internet access. Lack of access was associated with socioeconomic deprivation and older age. Multivariable regression confirmed increased age and greater deprivation to be independently associated with lack of internet access. Out of the total group, 198 (40%) indicated a preference for assessment of outcomes via email and the internet. Conclusions Internet access was not universal among the patients in our orthopaedic clinic. Reliance on internet collection of PROMs may introduce bias by not including results from patients in older age groups and those from the more deprived socioeconomic groups. PMID:26688398

  1. Measuring Patient Safety in Primary Care: The Development and Validation of the “Patient Reported Experiences and Outcomes of Safety in Primary Care” (PREOS-PC)

    PubMed Central

    Ricci-Cabello, Ignacio; Avery, Anthony J.; Reeves, David; Kadam, Umesh T.; Valderas, Jose M.

    2016-01-01

    PURPOSE We set out to develop and validate a patient-reported instrument for measuring experiences and outcomes related to patient safety in primary care. METHOD The instrument was developed in a multistage process supported by an international expert panel and informed by a systematic review of instruments, a meta-synthesis of qualitative studies, 4 patient focus groups, 18 cognitive interviews, and a pilot study. The trial version of Patient Reported Experiences and Outcomes of Safety in Primary Care (PREOS-PC) covered 5 domains and 11 scales: practice activation (1 scale); patient activation (1 scale); experiences of patient safety events (1 scale); harm (6 scales); and general perceptions of patient safety (2 scales). The questionnaire was posted to 6,736 patients in 45 practices across England. We used “gold standard” psychometric methods to evaluate its acceptability, reliability, structural and construct validity, and ability to discriminate among practices. RESULTS 1,244 completed questionnaires (18.5%) were returned. Median item-specific response rate was 91.3% (interquartile range 28.0%). No major ceiling or floor effects were observed. All 6 multi-item scales showed high internal consistency (Cronbach’s α 0.75–0.96). Factor analysis, correlation between scales, and known group analyses generally supported structural and construct validity. The scales demonstrated a heterogeneous ability to discriminate between practices. The final version of PREOS-PC consisted of 5 domains, 8 scales, and 58 items. CONCLUSIONS PREOS-PC is a new multi-dimensional patient safety instrument for primary care developed with experts and patients. Initial testing shows its potential for use in primary care, and future developments will further address its use in actual clinical practice. PMID:27184996

  2. Loss to follow-up after total hip replacement: a source of bias in patient reported outcome measures and registry datasets?

    PubMed

    Imam, Mohamed A; Barke, Samuel; Stafford, Giles H; Parkin, David; Field, Richard E

    2014-01-01

    Patient reported outcome measures (PROMs) are used to gauge clinical performance. The PROMs outcome programme at our centre achieves a preoperative data capture rate of 99%. This falls to 90.6%, 89%, 83% and 79% at the six-week, six-month, one-year and two-year time points, respectively. The study aims were to determine factors associated with patients who did not respond to outcome questionnaires following total hip replacement (THR), and the potential implications this may have when assessing patients following THRs. During the first year of the PROMs programme, 1,322 patients underwent unilateral primary THR at our institution. Of these, 1,311 completed preoperative questionnaires. Thirty-eight patients (2.9%) died within two years of surgery and have been excluded. For the remaining 1,273 patients, we identified those who did not return postoperative questionnaires at each of our review time points. Younger age, lower baseline EQ5D and Oxford Hip scores (OHS) were significantly associated with non-response (p<0.001). Patients with lower satisfaction scores, OHS and EQ5D scores, were less likely to respond to subsequent questionnaires. A significant association between non-response and deprivation (p<0.001) was demonstrated. Our findings suggest that the more satisfied patients are over-represented and our reported outcome results are better than they would have been if all patients had responded. This phenomenon may apply to studies where those categorised as "lost to follow-up" represent a subset of patients who have disengaged due to poor outcome or satisfaction.

  3. Patient-reported outcome measure for neuromyelitis optica: pretesting of preliminary instrument and protocol for further development in accordance with international guidelines

    PubMed Central

    Moore, P; Jackson, C; Mutch, K; Methley, A; Pollard, C; Hamid, S; Jacob, A

    2016-01-01

    Objective This study outlines the development of a patient-reported outcome measure (PROM), an instrument to obtain self-reported health status for neuromyeltis optica (NMO), a disabling neurological condition. Design Development was conducted in accordance with international guidance for PROMs including systematic review of existing literature, item generation guided by qualitative interviews, health-related quality of life conceptual framework and clinical expert panel and cognitive interviews with NMO patients. Setting Participants were identified through a national NMO clinic in a tertiary NHS neurosciences service. Participants 15 individuals with NMO participated in cognitive interviews requiring review and ranking of proposed PROM items and qualitative feedback on content, layout and response options. Results Participants endorsed the draft instrument as reflecting their experience of the condition and as being easy to understand. Rating and ranking of item relevance and importance reduced the draft instrument from 106 to 48 items. Participant feedback on overlapping items eliminated a further 2 items and resulted in a preliminary instrument of 46 items. As a direct result of participant feedback ordering of the 10 domains was revised, a 4 option Likert scale was employed and a 4-week recall period for impact of symptoms was selected. Conclusions A 46-item instrument developed in accordance with international PROM development guidelines through literature review, developed by subject matter experts and refined through pretesting examining content validity provides a preliminary measure for assessing patient-report of health status in NMO. Further evaluation is proposed including sensitivity to clinical change, and international contributions to evaluating the measure are encouraged. PMID:27694484

  4. Validity of patient-reported swallowing and speech outcomes in relation to objectively measured oral function among patients treated for oral or oropharyngeal cancer.

    PubMed

    Rinkel, R N P M; Verdonck-de Leeuw, I M; de Bree, R; Aaronson, N K; Leemans, C R

    2015-04-01

    The objective of this study was to test the construct validity of the patient-reported outcomes Swallowing Quality of Life Questionnaire (SWAL-QOL) and Speech Handicap Index (SHI) in relation to objectively measured oral function among patients treated for oral or oropharyngeal cancer. The study sample consisted of patients treated for oral or oropharyngeal cancer. Outcome measures were the SWAL-QOL and the SHI, and the Functional Rehabilitation Outcomes Grade (FROG), a test to measure oral and shoulder function. Spearman's rank correlation coefficient was used to test associations between the SHI and SWAL-QOL scales, and the FROG scales. During a study period of 3 months, 38 patients (21 males, 17 females; mean age 54 years) were included who visited the outpatient clinic for follow-up care 6-155 months after surgical treatment (n = 14) or combined surgery and radiotherapy (n = 24) for oral (n = 21) or oropharyngeal cancer (n = 17). Most SWAL-QOL and SHI scales (except the SWAL-QOL Fatigue scale) correlated significantly with one or more FROG oral function scales. None of the SWAL-QOL and SHI scales correlated significantly with the FROG shoulder function scale. These results support the construct validity of the SWAL-QOL and SHI questionnaires for assessing speech and swallowing problems in daily life that are moderately but significantly related to oral function. A multidimensional assessment protocol is recommended for use in clinical practice and for research purposes for measuring oral function and swallowing- and speech-related problems in daily life among head and neck cancer patients.

  5. Patient-Reported Outcomes for Quality of Life Assessment in Atrial Fibrillation: A Systematic Review of Measurement Properties

    PubMed Central

    Ahmed, Amar; Calvert, Melanie; Lencioni, Mauro; Terwee, Caroline B.; Lane, Deirdre A.

    2016-01-01

    Background Atrial fibrillation is a large and growing burden across all types of healthcare. Both incidence and prevalence are expected to double in the next 20 years, with huge impact on hospital admissions, costs and patient quality of life. Patient wellbeing determines the management strategy for atrial fibrillation, including the use of rhythm control therapy and the clinical success of heart rate control. Hence, evaluation of quality of life is an emerging and important part of the assessment of patients with atrial fibrillation. Although a number of questionnaires to assess quality of life in atrial fibrillation are available, a comprehensive overview of their measurement properties is lacking. Methods and Results We performed a systematic review of the measurement properties of atrial fibrillation-specific health-related quality of life questionnaires. Methodological quality was assessed using the Consensus based Standards for selection of health Measurement Instruments (COSMIN) checklist, with measurement properties rated for quality against optimal criteria and levels of evidence. We screened 2,216 articles, of which eight articles describing five questionnaires were eligible for inclusion: Atrial Fibrillation 6 (AF6), Atrial Fibrillation Effect on QualiTy-of-Life (AFEQT), Atrial Fibrillation Quality of Life Questionnaire (AFQLQ), Atrial Fibrillation Quality of Life (AFQoL), and Quality of Life in Atrial Fibrillation (QLAF). Good reliability (internal consistency and test-retest reliability) was demonstrated for AF6, AFEQT, AFQLQ and AFQoL. Content, construct and criterion validity were positively rated only in AFEQT. Responsiveness was positively rated only in AFEQT, but with limited evidence. Overall, AFEQT showed strong positive evidence for 2 of 9 measurement properties, compared to one for AFQoL and none for the remaining questionnaires. Interpretation Given the low ratings for many measurement properties, no single questionnaire can be recommended

  6. Development of a patient reported outcome measure for fatigue in motor neurone disease: the Neurological Fatigue Index (NFI-MND)

    PubMed Central

    2011-01-01

    Background The objective of this research was to develop a disease-specific measure for fatigue in patients with motor neurone disease (MND) by generating data that would fit the Rasch measurement model. Fatigue was defined as reversible motor weakness and whole-body tiredness that was predominantly brought on by muscular exertion and was partially relieved by rest. Methods Qualitative interviews were undertaken to confirm the suitability of a previously identified set of 52 neurological fatigue items as relevant to patients with MND. Patients were recruited from five U.K. MND clinics. Questionnaires were administered during clinic or by post. A sub-sample of patients completed the questionnaire again after 2-4 weeks to assess test-retest validity. Exploratory factor analyses and Rasch analysis were conducted on the item set. Results Qualitative interviews with ten MND patients confirmed the suitability of 52 previously identified neurological fatigue items as relevant to patients with MND. 298 patients consented to completing the initial questionnaire including this item set, with an additional 78 patients completing the questionnaire a second time after 4-6 weeks. Exploratory Factor Analysis identified five potential subscales that could be conceptualised as representing: 'Energy', 'Reversible muscular weakness' (shortened to 'Weakness'), 'Concentration', 'Effects of heat' and 'Rest'. Of the original five factors, two factors 'Energy' and 'Weakness' met the expectations of the Rasch model. A higher order fatigue summary scale, consisting of items from the 'Energy' and 'Weakness' subscales, was found to fit the Rasch model and have acceptable unidimensionality. The two scales and the higher order summary scale were shown to fulfil model expectations, including assumptions of unidimensionality, local independency and an absence of differential item functioning. Conclusions The Neurological Fatigue Index for MND (NFI-MND) is a simple, easy-to-administer fatigue

  7. 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.

  8. Patient reported outcomes and patient empowerment in clinical genetics services.

    PubMed

    McAllister, M; Dearing, A

    2015-08-01

    Evaluation of clinical genetics services (CGS), including genetic counseling and genetic testing, has been problematic. Patient mortality and morbidity are unlikely to be directly improved by interventions offered in CGS. Patient-reported outcomes (PROs) are not routinely measured in CGS evaluation, but this may change as patient-reported outcome measures (PROMs) become a key part of how healthcare services are managed and funded across the world. However, there is no clear consensus about which PROMs are most useful for CGS evaluation. This review summarizes the published research on how PROs from CGS have been measured and how patients may benefit from using those services, with a focus on patient empowerment. Many patient benefits (PROs) identified repeatedly in the research literature can be re-interpreted within a patient empowerment framework. Other important PROs identified include family functioning, social functioning, altruism, sense of purpose, enabling development of future research and treatment/participating in research. Well-validated measures are available to capture (dimensions of) patient empowerment. Although generic measures of family functioning are available, suitable measures capturing social functioning, development of future treatments, and altruism were not identified in this review. Patient empowerment provides one useful approach to measuring PROs from CGS.

  9. The routine collection of patient-reported outcome measures (PROMs) for long-term conditions in primary care: a cohort survey

    PubMed Central

    Peters, Michele; Crocker, Helen; Jenkinson, Crispin; Doll, Helen; Fitzpatrick, Ray

    2014-01-01

    Objectives To evaluate the feasibility of using patient-reported outcome measures (PROMs) for long-term conditions (LTCs) in primary care. Design A cohort postal survey conducted from September 2010 to April 2012. Setting Primary care practices (n=33) in London and the North-West of England. Participants 4484 patients with a diagnosis of asthma, chronic obstructive pulmonary disease, diabetes, epilepsy, heart failure or stroke were sent a survey at baseline. Main outcome The main outcome was to evaluate the feasibility of and the recruitment strategies for collecting PROMs data in LTCs by assessing the response rates for the baseline and follow-up surveys. Secondary outcomes were the evaluation of change scores of the EQ-5D index and visual analogue scale (VAS) between baseline and follow-up surveys. Results The baseline survey achieved a response rate of 38.4% (n=1721/4485) and at follow-up 71.5% (n=1136/1589). Response rates varied by LTC. Little change was found in health-related quality of life for the total sample (−0.001 for the EQ-5D index score and 0.12 for the EQ-5D VAS) between patients responding to both the baseline and follow-up surveys. Conclusions The response rate to the baseline survey was similar to that of other general practice surveys. Current UK policy aims to assess health service performance in LTCs by means of using PROMs. It thus would be desirable to improve response rates by making the invitation to self-reports of health-related quality of life more engaging for patients. Results on the EQ-5D score raise questions about optimal indicators for LTCs and appropriate timelines for assessment. PMID:24561495

  10. 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

  11. Associations between a patient reported outcome (PRO) measure of sarcopenia and falls, functional status, and physical performance in older patients with cancer

    PubMed Central

    Gewandter, Jennifer S.; Dale, William; Magnuson, Allison; Pandya, Chintan; Heckler, Charles E.; Lemelman, Tatyana; Roussel, Breton; Ifthikhar, Rafa; Dolan, James; Noyes, Katia; Mohile, Supriya G.

    2015-01-01

    Objective In older patients with cancer, we aimed to investigate associations between a patient-reported outcome measure for sarcopenia (SarcoPRO) and the Short Physical Performance Battery (SPPB), self-reported falls, and limitations in instrumental activities of daily living (IADLs). Materials and Methods Assessments were conducted as part of the initial evaluation of older, often frail, patients with cancer seen in the Specialized Oncology Care and Research in the Elderly (SOCARE) clinic. Univariate associations were evaluated using Spearman’s correlation and Wilcoxon sign ranked tests. Logistic regressions were used to identify associations of clinical factors and SarcoPRO scores or SPPB scores with falls and IADL limitations. Results In total, 174 older patients with cancer were evaluated. A moderate correlation was found between the SarcoPRO and the SPPB (ρ = 0.62). After adjusting for multiple clinical factors, neither the SarcoPRO nor the SPPB were associated with falls. In contrast, both higher SarcoPRO (i.e., worse) and lower SPPB (i.e., worse) scores were associated with limitations in IADLs (Odds ratio for one unit change in predictor: SarcoPRO: 1.06, p<0.0001; SPPB: 0.71, p = 0.003, respectively). Models using the SarcoPRO and SPPB explained similar amounts of variability in association with IADL limitations (AUC: 0.88 vs 0.87, respectively). Conclusions The SarcoPRO was moderately associated with the SPPB, an objective measure of physical performance, and was associated with limitations in IADLs. Thus, older patients with cancer who present with IADL limitations should be screened for sarcopenia. The SarcoPRO shows promise as a measure for screening as well as outcome assessment for research on sarcopenia. PMID:26365897

  12. Confirmatory Factor Analysis of the Patient-Reported Outcomes Measurement Information System (PROMIS) Adult Domain Framework Using Item Response Theory Scores

    PubMed Central

    Carle, Adam Christopher; Riley, William; Hays, Ron D.; Cella, David

    2016-01-01

    Background To guide measure development, National Institutes of Health (NIH)-supported Patient Reported Outcomes Measurement Information System (PROMIS®) investigators developed a hierarchical domain framework. The framework specifies health domains at multiple levels. The initial PROMIS domain framework specified that physical function and symptoms such as pain and fatigue indicate Physical Health (PH); depression, anxiety, and anger indicate Mental Health (MH); and social role performance and social satisfaction indicate Social Health (SH). We used confirmatory factor analyses (CFA) to evaluate the fit of the hypothesized framework to data collected from a large sample. Methods We used data (n = 14,098) from PROMIS’s wave 1 field test and estimated domain scores using the PROMIS item response theory parameters. We then used CFA to test whether the domains corresponded to the PROMIS domain framework as expected. Results A model corresponding to the domain framework did not provide ideal fit (RMSEA=0.13; CFI=0.92; TLI=0.88; SRMR=0.09). Based on modification indices and EFA, we allowed Fatigue to load on both PH and MH. This model fit the data acceptably (RMSEA=0.08; CFI=0.97; TLI=0.96; SRMR=0.03). Discussion Our findings generally support the PROMIS domain framework. Allowing fatigue to load on both PH and MH improved fit considerably. PMID:26366521

  13. 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

  14. Tracking patient-reported outcomes in spinal disorders

    PubMed Central

    Nayak, Nikhil R.; Coats, John Mitchell; Abdullah, Kalil G.; Stein, Sherman C.; Malhotra, Neil R.

    2015-01-01

    Background: Patient-reported outcome measures (PROMs) quantify health status from the patient's point of view. While the number of published outcomes studies grows each year, so too has the number of instruments being reported, leading to confusion on which instruments are appropriate to use for various spinal conditions. Methods: A broad search was conducted to identify commonly used PROMs in patients undergoing spinal surgery. We searched PubMed for combinations of terms related to anatomic location and a measure of patient-reported outcome in the title or text. We supplemented the search using the “related articles” feature of PubMed and by manually searching the bibliographies of selected articles. Results: Major categories of PROMs in spine surgery include health-related quality-of-life, pain, and disease-specific disability, for which several different instrument options were identified and detailed. The minimal clinically important difference varies between instruments and differentiates statistical significance from clinical significance. In addition, the accurate estimation of costs has become a challenging but intrinsically linked variable to outcomes as increased attention is paid to the relative value of surgical interventions. Conclusion: While a number of PROMs are available for tracking outcomes in spine surgery, only a handful appear to be widely used. At least one instrument from each category should be measured pre- and post-operatively to quantify treatment effect. In addition, while the primary goal is to select the most appropriate instruments for the patient's condition, one should keep in mind sustainability of efforts with regard to patient and administrative burden. PMID:26605111

  15. Upper-Extremity and Mobility Subdomains From the Patient-Reported Outcomes Measurement Information System (PROMIS) Adult Physical Functioning Item Bank

    PubMed Central

    Hays, Ron D.; Spritzer, Karen L.; Amtmann, Dagmar; Lai, Jin-Shei; DeWitt, Esi Morgan; Rothrock, Nan; DeWalt, Darren A.; Riley, William T.; Fries, James F.; Krishnan, Eswar

    2013-01-01

    Objective To create upper-extremity and mobility subdomain scores from the Patient-Reported Outcomes Measurement Information System (PROMIS) physical functioning adult item bank. Design Expert reviews were used to identify upper-extremity and mobility items from the PROMIS item bank. Psychometric analyses were conducted to assess empirical support for scoring upper-extremity and mobility subdomains. Setting Data were collected from the U.S. general population and multiple disease groups via self-administered surveys. Participants The sample (N=21,773) included 21,133 English-speaking adults who participated in the PROMIS wave 1 data collection and 640 Spanish-speaking Latino adults recruited separately. Interventions Not applicable. Main Outcome Measures We used English- and Spanish-language data and existing PROMIS item parameters for the physical functioning item bank to estimate upper-extremity and mobility scores. In addition, we fit graded response models to calibrate the upper-extremity items and mobility items separately, compare separate to combined calibrations, and produce subdomain scores. Results After eliminating items because of local dependency, 16 items remained to assess upper extremity and 17 items to assess mobility. The estimated correlation between upper extremity and mobility was .59 using existing PROMIS physical functioning item parameters (r=.60 using parameters calibrated separately for upper-extremity and mobility items). Conclusions Upper-extremity and mobility subdomains shared about 35% of the variance in common, and produced comparable scores whether calibrated separately or together. The identification of the subset of items tapping these 2 aspects of physical functioning and scored using the existing PROMIS parameters provides the option of scoring these subdomains in addition to the overall physical functioning score. PMID:23751290

  16. [Portuguese-language translation and cross-cultural adaptation of the Fatigue domain of Patient-Reported-Outcomes Measurement Information System (PROMIS)].

    PubMed

    Alves, Flávio Sérgio Marques; Pinto, Rogério de Melo Costa; Mendonça, Tânia Maria Silva; Silva, Carlos Henrique Martins da

    2014-05-01

    The items bank of the Fatigue domain is part of an American system developed for evaluation of results reported by patients, called Patient-Reported-Outcomes Measurement Information System (PROMIS). This study aimed to translate and cross-culturally adapt this item bank for the Brazilian population, as a promising new tool for evaluating health-related quality of life. The items in this bank were translated using rigorous translation and back-translation protocols. The translated version was pre-tested in twenty Brazilians with a brief cognitive and retrospective interview in order to test the items' conceptual, cultural, and semantic equivalences. In the translation and back-translation process, only three of the 82 items had to be reworded due to the culturally inadequate content. In the pretest, only four items needed to be reworded, but without conceptual and semantic alterations. The results showed that the translated version of this item bank is conceptually, culturally, and semantically equivalent to the original version.

  17. 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.

  18. 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.

  19. Analysis of differential item functioning in the depression item bank from the Patient Reported Outcome Measurement Information System (PROMIS): An item response theory approach

    PubMed Central

    Teresi, Jeanne A.; Ocepek-Welikson, Katja; Kleinman, Marjorie; Eimicke, Joseph P.; Crane, Paul K.; Jones, Richard N.; Lai, Jin-shei; Choi, Seung W.; Hays, Ron D.; Reeve, Bryce B.; Reise, Steven P.; Pilkonis, Paul A.; Cella, David

    2009-01-01

    The aims of this paper are to present findings related to differential item functioning (DIF) in the Patient Reported Outcome Measurement Information System (PROMIS) depression item bank, and to discuss potential threats to the validity of results from studies of DIF. The 32 depression items studied were modified from several widely used instruments. DIF analyses of gender, age and education were performed using a sample of 735 individuals recruited by a survey polling firm. DIF hypotheses were generated by asking content experts to indicate whether or not they expected DIF to be present, and the direction of the DIF with respect to the studied comparison groups. Primary analyses were conducted using the graded item response model (for polytomous, ordered response category data) with likelihood ratio tests of DIF, accompanied by magnitude measures. Sensitivity analyses were performed using other item response models and approaches to DIF detection. Despite some caveats, the items that are recommended for exclusion or for separate calibration were “I felt like crying” and “I had trouble enjoying things that I used to enjoy.” The item, “I felt I had no energy,” was also flagged as evidencing DIF, and recommended for additional review. On the one hand, false DIF detection (Type 1 error) was controlled to the extent possible by ensuring model fit and purification. On the other hand, power for DIF detection might have been compromised by several factors, including sparse data and small sample sizes. Nonetheless, practical and not just statistical significance should be considered. In this case the overall magnitude and impact of DIF was small for the groups studied, although impact was relatively large for some individuals. PMID:20336180

  20. 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.

  1. Longitudinal deteriorations in patient reported outcomes in patients with COPD.

    PubMed

    Oga, Toru; Nishimura, Koichi; Tsukino, Mitsuhiro; Sato, Susumu; Hajiro, Takashi; Mishima, Michiaki

    2007-01-01

    Goals of effective management of patients with chronic obstructive pulmonary disease (COPD) include relieving their symptoms and improving their health status. We examined how such patient reported outcomes would change longitudinally in comparison to physiological outcomes in COPD. One hundred thirty-seven male outpatients with stable COPD were recruited for the study. The subjects health status was evaluated using the St. George's Respiratory Questionnaire (SGRQ) and the Chronic Respiratory Disease Questionnaire (CRQ). Their dyspnoea using the modified Medical Research Council (MRC) scale and their psychological status using the Hospital Anxiety and Depression Scale (HADS) were assessed upon entry and every 6 months thereafter over a 5-year period. Pulmonary function and exercise capacity as evaluated by peak oxygen uptake (VO2) on progressive cycle ergometry were also followed over the same time. Using mixed effects models to estimate the slopes for the changes, scores on the SGRQ, the CRQ, the MRC and the HADS worsened in a statistically significant manner over time. However, changes only weakly correlated with changes in forced expiratory volume in 1s (FEV(1)) and peak (VO2). We demonstrated that although changes in pulmonary function and exercise capacity are well known in patients with COPD, patient reported outcomes such as health status, dyspnoea and psychological status also deteriorated significantly over time. In addition, deteriorations in patient reported outcomes only weakly correlated to changes in physiological indices. To capture the overall deterioration of COPD from the subjective viewpoints of the patients, patient reported outcomes should be followed separately from physiological outcomes.

  2. Patient reported outcomes as endpoints in medical research.

    PubMed

    Fairclough, Diane L

    2004-04-01

    This review covers a number of the many design and analytic issues associated with clinical trials that incorporate patient reported outcomes as primary or secondary endpoints. We use a clinical trial designed to evaluate a new therapy for the prevention of migraines to illustrate how endpoints are defined by the objectives of the study, the methods for handling longitudinal assessments with multiple scales or outcomes, and the methods of analysis in the presence of missing data.

  3. Patient-reported outcomes in idiopathic pulmonary fibrosis research.

    PubMed

    Swigris, Jeffrey J; Fairclough, Diane

    2012-08-01

    Patient-reported outcomes (PROs) include questionnaires or surveys that ask patients for their perceptions about things like symptoms they are experiencing or quality of life. For incurable, morbid, life-shortening conditions like idiopathic pulmonary fibrosis (IPF), PROs are particularly germane: They elucidate for clinicians and researchers what it is like for patients to live with such a disease, and they may detect important treatment effects not captured by other metrics (eg, pulmonary physiology). However, a relative paucity of research on PROs in IPF has left significant knowledge gaps in this area and contributed to the timidity investigators have about using PROs as prominent outcomes in IPF drug trials. Additional research on existing instruments is needed to establish or bolster their basic psychometric properties in IPF. When PROs are used as end points in therapeutic trials, analyzing PRO response data can be challenging, but these challenges can be overcome with a transparent, thoughtful, and sophisticated statistical approach. In this article, we discuss some of the basics of PRO assessment, existing knowledge gaps in IPF-related PRO research, and the potential usefulness of using PROs in IPF trials and conclude by offering specific recommendations for an approach to analyzing repeated-measures PRO data from IPF trials.

  4. 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.

  5. Patient-Reported Outcomes Measurement Information System (PROMIS) Domain Names and Definitions Revisions: Further Evaluation of Content Validity in IRT-derived Item Banks

    PubMed Central

    Riley, William T.; Rothrock, Nan; Bruce, Bonnie; Christodolou, Christopher; Cook, Karon; Hahn, Elizabeth A.; Cella, David

    2013-01-01

    Purpose Content validity of patient-reported outcomes (PROs) is evaluated primarily during item development, but subsequent psychometric analyses, particularly for item-response theory (IRT)-derived scales, often result in considerable item pruning and potential loss of content. After selecting items for the PROMIS banks based on psychometric and content considerations, we invited external content expert reviews of the degree to which the initial domain names and definitions represented the calibrated item bank content. Methods A minimum of four content experts reviewed each item bank and recommended a domain name and definition based on item content. Domain names and definitions then were revealed to the experts who rated how well these names and definitions fit the bank content and provided recommendations for definition revisions. Results These reviews indicated that the PROMIS domain names and definitions remained generally representative of bank content following item pruning, but modifications to two domain names and minor to moderate revisions of all domain definitions were needed to optimize fit with the item bank content. Conclusions This reevaluation of domain names and definitions following psychometric item pruning, although not previously documented in the literature, appears to be an important procedure for refining conceptual frameworks and further supporting content validity. PMID:20593306

  6. 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

  7. Capturing and Incorporating Patient-Reported Outcomes into Clinical Trials: Practical Considerations for Clinicians.

    PubMed

    Botero, Juliana Perez; Thanarajasingam, Gita; Warsame, Rahma

    2016-10-01

    Patient centeredness as the focus of healthcare delivery requires the incorporation of patient-reported outcomes into clinical trials. Clearly defining measurable outcomes as well as selecting the most appropriate validated collection tool to use is imperative for success. Creating and validating one's own instrument is also possible, albeit more cumbersome. Meticulous data collection to avoid missing data is key, as is limiting the number of data collection points to prevent survey fatigue and using electronic systems to facilitate data gathering and analysis. Working in a multidisciplinary team that includes statisticians with expertise in patient reported outcomes is essential to navigate the complexities of statistical analysis of these variables. Use of available and emerging technologies for data collection and analysis as well as data sharing will greatly facilitate the process of incorporating patient-reported outcomes into trials and routine clinical practice.

  8. 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

  9. 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

  10. A randomized controlled trial of patient-reported outcomes with tai chi exercise in Parkinson's disease.

    PubMed

    Li, Fuzhong; Harmer, Peter; Liu, Yu; Eckstrom, Elizabeth; Fitzgerald, Kathleen; Stock, Ronald; Chou, Li-Shan

    2014-04-01

    A previous randomized, controlled trial of tai chi showed improvements in objectively measured balance and other motor-related outcomes in patients with Parkinson's disease. This study evaluated whether patient-reported outcomes could be improved through exercise interventions and whether improvements were associated with clinical outcomes and exercise adherence. In a secondary analysis of the tai chi trial, patient-reported and clinical outcomes and exercise adherence measures were compared between tai chi and resistance training and between tai chi and stretching exercise. Patient-reported outcome measures were perceptions of health-related benefits resulting from participation, assessed by the Parkinson's Disease Questionnaire (PDQ-8) and Vitality Plus Scale (VPS). Clinical outcome measures included motor symptoms, assessed by a modified Unified Parkinson's Disease Rating Scale-Motor Examination (UPDRS-ME) and a 50-foot speed walk. Information on continuing exercise after the structured interventions were terminated was obtained at a 3-month postintervention follow-up. Tai chi participants reported significantly better improvement in the PDQ-8 (-5.77 points, P = 0.014) than did resistance training participants and in PDQ-8 (-9.56 points, P < 0.001) and VPS (2.80 points, P = 0.003) than did stretching participants. For tai chi, patient-reported improvement in the PDQ-8 and VPS was significantly correlated with their clinical outcomes of UPDRS-ME and a 50-foot walk, but these correlations were not statistically different from those shown for resistance training or stretching. However, patient-reported outcomes from tai chi training were associated with greater probability of continued exercise behavior than were either clinical outcomes or patient-reported outcomes from resistance training or stretching. Tai chi improved patient-reported perceptions of health-related benefits, which were found to be associated with a greater probability of exercise

  11. 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

  12. 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

  13. Post-operative radiographic factors and patient-reported outcome after total hip replacement.

    PubMed

    Wylde, Vikki; Maclean, Angus; Blom, Ashley W

    2012-01-01

    Although total hip replacement (THR) is considered a very successful surgical intervention, a proportion of patients experience persistent pain or disability, and/or dissatisfaction with the outcome of surgery. Our aim was to determine whether post-operative radiographic variables were predictive of patient-reported pain, function and satisfaction after primary THR. At 1-3 years after surgery patients completed the WOMAC Pain scale, WOMAC Function scale and a validated measure of satisfaction with the outcome of surgery. Post-operative radiographs taken prior to discharge were graded for the restoration of offset, restoration of leg length, anteroposterior (AP) alignment of the femoral stem and AP acetabular inclination. Binary logistic regression was used to identify whether radiographic variables were significant predictors of patient-reported outcome scores. Radiographic and patient-reported outcomes data were available for 452 THR patients. No radiographic predictors were found to be significant predictors of patient reported pain, function or satisfaction at 1-3 years after THR. This highlights that patients with continuing problems after THR may benefit from a thorough multidisciplinary assessment to diagnose the underlying cause of the problems.

  14. Convergent Validity of the Patient Reported Outcomes Measurement Information System's (PROMIS) Physical Function Computerized Adaptive Test (PF-CAT) for the Knee and Shoulder Injury Sports Medicine Patient Population

    PubMed Central

    Robins, Richard Judd; Zhang, Yingying; Anderson, Mike B.; Presson, Angela P.; Burks, Robert T.; Greis, Patrick E.

    2015-01-01

    Objectives: The Patient Reported Outcomes Measurement Instrument System (PROMIS) Physical Function Computer Adaptive Test (PF-CAT) has been developed by the National Institutes of Health as a means of providing validated outcomes scores. PF-CAT adapts to patient response by applying different validated questions based on prior responses to generate scores using between 4 to 12 questions, and has been validated in a variety of patient populations to assess physical function. The purpose of our study is to compare patient reported outcomes scores in a sports medicine (shoulder and knee injury) patient population by analyzing convergence validity of PF-CAT to IKDC, SST, ASES, and SANE scores. We also wanted to assess and compare time burden placed on patients in completing these outcomes scores. Methods: All patient visits from April through September 2014 presenting with either knee or shoulder complaints were included from a university-based sports medicine clinic, during which both PF-CAT as well as SANE, SST, and ASES legacy outcomes scores for shoulder injury patients, and SANE and IKDC outcomes scores for knee injury patients were obtained via tablet during the initial part of each visit. A total of 415 shoulder and 450 knee clinical evaluations qualified for inclusion in the study. Spearman correlation was used to evaluate pair-wise agreement among outcomes scores. The total scores from each outcome test and the T-scores from the PF-CAT were used in this correlation analysis. For interpretation, results greater than 0.5-0.69 were considered to have fair correlation, while values greater than 0.7 were considered as having good correlation. Significance was assessed at the 0.05 level. Median and interquartile ranges were used to summarize scores. The minimum score (floor effects) and maximum score (ceiling effects) occurrences were tallied and reported as percentages. A Fishers exact test was used to test for a difference in the number of times floor and ceiling

  15. Patient reported outcomes in the assessment of premature ejaculation.

    PubMed

    Althof, Stanley E

    2016-08-01

    The term 'Patient Reported Outcome', abbreviated as PRO, was introduced by the US Food and Drug Administration (FDA) which proposed guidance on the development and validation of PROs. Previously PROs were known as self-report diaries, event-logs, self-administered questionnaires, and clinician administered rating scales. PROs seek to capture the subjective perceptions of patients and/or partner's related to their specific symptoms, degree of bother, efficacy of a medication or psychotherapy intervention, and quality of life issues related to a specific condition. This article reviews the essential psychometric and regulatory agency requirements in the development of PROs. The constructs of reliability, various forms of validity, sensitivity, and specificity as well as concerns with translating a PRO into a different language are reviewed. Three PROs, the Premature Ejaculation Profile (PEP), the Index of Premature Ejaculation (IPE) and the Premature Ejaculation Diagnostic Tool (PEDT) all used in the assessment of premature ejaculation (PE) are discussed. These questionnaires meet or exceed all the psychometric requirements and have been employed in clinical trials and observational studies of men with PE. The article concludes on discussing some of the limitations of PRO use and recommendations for the future.

  16. 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.

  17. 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

  18. Patient-Reported Outcomes After Multiligament Knee Injury

    PubMed Central

    Hanley, Jessica M.; Anthony, Christopher A.; DeMik, David; Glass, Natalie; Amendola, Annunziato; Wolf, Brian R.; Bollier, Matthew

    2017-01-01

    Background: Management of the medial collateral ligament (MCL) in the setting of a multiligamentous knee injury (MLKI) represents an area of great controversy. Purpose: Our study was designed to compare long-term patient-reported outcomes (PROs) after MCL repair versus reconstruction in the setting of a multiligamentous injury of the knee. Study Design: Cohort study; Level of evidence, 3. Methods: At a single institution, 68 patients were identified over a 10-year period as having MCL intervention in the setting of MLKI. Of these patients, 34 (50%) were successfully contacted via telephone to collect Lysholm and International Knee Documentation Committee (IKDC) scores. A retrospective chart review of these subjects was also conducted to identify patient and surgical factors affecting PROs. Results: At a mean 6-year follow-up (range, 2-11 years), the mean Lysholm score was 77.4 ± 23.1 and mean IKDC score was 72.6 ± 23.6. Univariate analyses identified time to surgery (P = .005) and MCL reconstruction (P = .001) as risk factors for Lysholm score ≤75. Univariate analyses identified patient age (P = .049), time to surgery (P = .018), and MCL reconstruction (P = .004) as risk factors for IKDC score ≤75. On subsequent multivariate analysis, MCL reconstruction was found to be a predictor of Lysholm or IKDC score of ≤75. Conclusion: Patients undergoing MCL repair in the setting of MLKI generally had higher PROs than those undergoing reconstructions at a mean 6 years of follow-up. Further work is needed to elucidate patient and surgical factors that may influence subjective outcomes after multiligament knee injuries. PMID:28357408

  19. 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.

  20. Enhancing periodontal health through regenerative approaches: a commentary on the need for patient-reported outcomes.

    PubMed

    Inglehart, Marita R

    2015-02-01

    Starting in the 1970s, social scientists have discussed the importance of assessing subjective indicators of well-being and quality of life. Medical researchers followed this line of reasoning since the 1990s, emphasizing the significance of understanding how disease and its treatment affect patients' quality of life. Since the start of the 21(st) century, oral health-related quality of life (OHRQoL) received increasingly more attention. While research concerning the effects of periodontal disease and its surgical and non-surgical treatment on patients' lives has been considered in numerous studies, research including patient-reported outcomes when assessing how periodontal health can be enhanced through regenerative approaches is largely missing. This commentary proposes to consider 1) OHRQoL and 2) patients' treatment satisfaction as patient-reported outcomes in conjunction with objectively measured patient-centered factors, and discusses the value of such an approach.

  1. Physician-assessed and patient-reported outcome measures in chemotherapy-induced sensory peripheral neurotoxicity: two sides of the same coin

    PubMed Central

    Alberti, P.; Rossi, E.; Cornblath, D. R.; Merkies, I. S. J.; Postma, T. J.; Frigeni, B.; Bruna, J.; Velasco, R.; Argyriou, A. A.; Kalofonos, H. P.; Psimaras, D.; Ricard, D.; Pace, A.; Galiè, E.; Briani, C.; Dalla Torre, C.; Faber, C. G.; Lalisang, R. I.; Boogerd, W.; Brandsma, D.; Koeppen, S.; Hense, J.; Storey, D.; Kerrigan, S.; Schenone, A.; Fabbri, S.; Valsecchi, M. G.; Cavaletti, G.; Cavaletti, G.; Cornblath, D.R.; Merkies, I.S.J.; Postma, T.J.; Valsecchi, M.G; Galimberti, S.; Rossi, E.; Cavaletti, G.; Frigeni, B.; Lanzani, F.; Mattavelli, L.; Piatti, ML.; Alberti, P.; Binda, D.; Bidoli, P..; Cazzaniga, M.; Cortinovis, D.; Bruna, J.; Velasco, R.; Argyriou, AA.; Kalofonos, HP.; Psimaras, D.; Ricard, D.; Pace, A.; Galiè, E.; Briani, C.; Lucchetta, M.; Campagnolo, M.; Dalla Torre, C.; Merkies, ISJ.; Faber, CG.; Merkies, ISJ.; Vanhoutte, EK.; Bakkers, M.; Brouwer, B.; Lalisang, RI.; Boogerd, W.; Brandsma, D.; Koeppen, S.; Hense, J.; Grant, R.; Storey, D.; Kerrigan, S.; Schenone, A.; Reni, L.; Piras, B.; Fabbri, S.; Padua, L.; Granata, G.; Leandri, M.; Ghignotti, I.; Plasmati, R..; Pastorelli, F.; Postma, TJ.; Heimans, JJ.; Eurelings, M.; Meijer, RJ.; Grisold, W.; Lindeck Pozza, E.; Mazzeo, A.; Toscano, A.; Tomasello, C.; Altavilla, G.; Penas Prado, M.; Dominguez Gonzalez, C.; Dorsey, SG.; Brell, JM.

    2014-01-01

    Background The different perception and assessment of chemotherapy-induced peripheral neurotoxicity (CIPN) between healthcare providers and patients has not yet been fully addressed, although these two approaches might eventually lead to inconsistent, possibly conflicting interpretation, especially regarding sensory impairment. Patients and methods A cohort of 281 subjects with stable CIPN was evaluated with the National Cancer Institute—Common Toxicity Criteria (NCI-CTC v. 2.0) sensory scale, the clinical Total Neuropathy Score (TNSc©), the modified Inflammatory Neuropathy Cause and Treatment (INCAT) sensory sumscore (mISS) and the European Organization for Research and Treatment of Cancer CIPN specific self-report questionnaire (EORTC QOL-CIPN20). Results Patients' probability estimates showed that the EORTC QLQ-CIPN20 sensory score was overall more highly related to the NCI-CTC sensory score. However, the vibration perception item of the TNSc had a higher probability to be scored 0 for EORTC QLQ-CIPN20 scores lower than 35, as vibration score 2 for EORTC QLQ-CIPN20 scores between 35 and 50 and as grade 3 or 4 for EORTC QLQ-CIPN20 scores higher than 50. The linear models showed a significant trend between each mISS item and increasing EORTC QLQ-CIPN20 sensory scores. Conclusion None of the clinical items had a perfect relationship with patients' perception, and most of the discrepancies stood in the intermediate levels of CIPN severity. Our data indicate that to achieve a comprehensive knowledge of CIPN including a reliable assessment of both the severity and the quality of CIPN-related sensory impairment, clinical and PRO measures should be always combined. PMID:24256846

  2. Development and Feasibility Testing of PROMPT-Care, an eHealth System for Collection and Use of Patient-Reported Outcome Measures for Personalized Treatment and Care: A Study Protocol

    PubMed Central

    Delaney, Geoff P; Arnold, Anthony; Miller, Alexis Andrew; Levesque, Janelle V; Kaadan, Nasreen; Carolan, Martin G; Cook, Nicole; Masters, Kenneth; Tran, Thomas T; Sandell, Tiffany; Durcinoska, Ivana; Gerges, Martha; Avery, Sandra; Ng, Weng; Della-Fiorentina, Stephen; Dhillon, Haryana M; Maher, Ashley

    2016-01-01

    Background Patient-reported outcome (PRO) measures have been used widely to screen for depression, anxiety, and symptoms in cancer patients. Computer-based applications that collect patients’ responses and transfer them to the treating health professional in real time have the potential to improve patient well-being and cancer outcomes. Objective This study will test the feasibility and acceptability of a newly developed eHealth system which facilitates PRO data capture from cancer patients, data linkage and retrieval to support clinical decisions and patient self-management, and data retrieval to support ongoing evaluation and innovative research. Methods The eHealth system is being developed in consultation with 3 overarching content-specific expert advisory groups convened for this project: the clinical advisory group, technical advisory group, and evaluation advisory group. The following work has already been completed during this phase of the study: the Patient-Reported Outcome Measures for Personalized Treatment and Care (PROMPT-Care) eHealth system was developed, patient-reported outcomes were selected (distress, symptoms, unmet needs), algorithms to inform intervention thresholds for clinical and self-management were determined, clinician PRO feedback summary and longitudinal reports were designed, and patient self-management resources were collated. PROsaiq, a custom information technology system, will transfer PRO data in real time into the hospital-based oncology information system to support clinical decision making. The PROMPT-Care system feasibility and acceptability will be assessed through patients completing PROMPT-Care assessments, participating in face-to-face cognitive interviews, and completing evaluation surveys and telephone interviews and oncology staff participating in telephone interviews. Results Over the course of 3 months, the system will be pilot-tested with up to 50 patients receiving treatment or follow-up care and 6 oncology staff

  3. 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.

  4. 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

  5. Morning stiffness and other patient-reported outcomes of rheumatoid arthritis in clinical practice.

    PubMed

    Sokka, T

    2011-01-01

    Morning stiffness has been recognized in traditional approaches to assessment of disease activity in rheumatoid arthritis (RA). Although morning stiffness is not specific to RA, changes in morning stiffness for an individual patient are helpful when monitoring health status. Health professionals can ask about morning stiffness but the most accurate and consistent approach to assessment from one visit to the next appears to be a patient self-report questionnaire. However, quantitative measures of patient-reported data are not an integral part of clinical monitoring in most clinics. No single measure is adequate for all individual patients, so quantitative measurement of patient-reported data should include many elements such as pain, functional status, fatigue, sleep, morning stiffness, work capacity, and physical and emotional well-being. In daily clinical practice, patient-reported outcomes can be collected easily using a standard questionnaire that patients can complete with pencil and paper or electronically on a touch screen in the waiting room. The results are then immediately available to the rheumatologists, to facilitate doctor-patient communication to improve the quality of patient care, leading to better patient outcomes.

  6. Quality of life as patient-reported outcomes: principles of assessment.

    PubMed

    Bullinger, Monika; Quitmann, Julia

    2014-06-01

    Assessing quality of life (QoL) as a patient-reported outcome in adult psychiatry poses challenges in terms of concepts, methods, and applications in research and practice. This review will outline conceptually the construct of QoL, its dimensionality, and its representation across patient groups. Methodological challenges are examined, along with principles of QoL instrument development and testing, as well as across cultures. Application of instruments in epidemiological, clinical health economics, and health services research is reviewed based on pertinent literature. Validated measures for depression, psychosis, and anxiety disorders are available in adult psychiatry, and are increasingly used in research. Still, targeted measures are lacking for many mental health conditions and only rarely are tools applied in the practice context. Progress has been made in the development of instruments that are now ready for implementation. The information to be gained is valuable for identifying patient-reported needs for and benefits of treatment.

  7. Patient-reported outcomes, patient-reported information: from randomized controlled trials to the social web and beyond.

    PubMed

    Baldwin, Mike; Spong, Andrew; Doward, Lynda; Gnanasakthy, Ari

    2011-01-01

    Internet communication is developing. Social networking sites enable patients to publish and receive communications very easily. Many stakeholders, including patients, are using these media to find new ways to make sense of diseases, to find and discuss treatments, and to give support to patients and their caregivers. We argue for a new definition of patient-reported information (PRI), which differs from the usual patient-reported outcomes (PRO). These new emergent data from the social web have important implications for decision making, at both an individual and a population level. We discuss new emergent technologies that will help aggregate this information and discuss how this will be assessed alongside the use of PROs in randomized controlled trials and how these new emergent data will be one facet of changing the relationship between the various stakeholders in achieving better co-created health.

  8. 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.

  9. Patient-Reported Outcomes after Monitoring, Surgery, or Radiotherapy for Prostate Cancer.

    PubMed

    Donovan, Jenny L; Hamdy, Freddie C; Lane, J Athene; Mason, Malcolm; Metcalfe, Chris; Walsh, Eleanor; Blazeby, Jane M; Peters, Tim J; Holding, Peter; Bonnington, Susan; Lennon, Teresa; Bradshaw, Lynne; Cooper, Deborah; Herbert, Phillipa; Howson, Joanne; Jones, Amanda; Lyons, Norma; Salter, Elizabeth; Thompson, Pauline; Tidball, Sarah; Blaikie, Jan; Gray, Catherine; Bollina, Prasad; Catto, James; Doble, Andrew; Doherty, Alan; Gillatt, David; Kockelbergh, Roger; Kynaston, Howard; Paul, Alan; Powell, Philip; Prescott, Stephen; Rosario, Derek J; Rowe, Edward; Davis, Michael; Turner, Emma L; Martin, Richard M; Neal, David E

    2016-10-13

    Background Robust data on patient-reported outcome measures comparing treatments for clinically localized prostate cancer are lacking. We investigated the effects of active monitoring, radical prostatectomy, and radical radiotherapy with hormones on patient-reported outcomes. Methods We compared patient-reported outcomes among 1643 men in the Prostate Testing for Cancer and Treatment (ProtecT) trial who completed questionnaires before diagnosis, at 6 and 12 months after randomization, and annually thereafter. Patients completed validated measures that assessed urinary, bowel, and sexual function and specific effects on quality of life, anxiety and depression, and general health. Cancer-related quality of life was assessed at 5 years. Complete 6-year data were analyzed according to the intention-to-treat principle. Results The rate of questionnaire completion during follow-up was higher than 85% for most measures. Of the three treatments, prostatectomy had the greatest negative effect on sexual function and urinary continence, and although there was some recovery, these outcomes remained worse in the prostatectomy group than in the other groups throughout the trial. The negative effect of radiotherapy on sexual function was greatest at 6 months, but sexual function then recovered somewhat and was stable thereafter; radiotherapy had little effect on urinary continence. Sexual and urinary function declined gradually in the active-monitoring group. Bowel function was worse in the radiotherapy group at 6 months than in the other groups but then recovered somewhat, except for the increasing frequency of bloody stools; bowel function was unchanged in the other groups. Urinary voiding and nocturia were worse in the radiotherapy group at 6 months but then mostly recovered and were similar to the other groups after 12 months. Effects on quality of life mirrored the reported changes in function. No significant differences were observed among the groups in measures of anxiety

  10. 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.

  11. Patient reported outcomes in the assessment of premature ejaculation

    PubMed Central

    2016-01-01

    The term ‘Patient Reported Outcome’, abbreviated as PRO, was introduced by the US Food and Drug Administration (FDA) which proposed guidance on the development and validation of PROs. Previously PROs were known as self-report diaries, event-logs, self-administered questionnaires, and clinician administered rating scales. PROs seek to capture the subjective perceptions of patients and/or partner’s related to their specific symptoms, degree of bother, efficacy of a medication or psychotherapy intervention, and quality of life issues related to a specific condition. This article reviews the essential psychometric and regulatory agency requirements in the development of PROs. The constructs of reliability, various forms of validity, sensitivity, and specificity as well as concerns with translating a PRO into a different language are reviewed. Three PROs, the Premature Ejaculation Profile (PEP), the Index of Premature Ejaculation (IPE) and the Premature Ejaculation Diagnostic Tool (PEDT) all used in the assessment of premature ejaculation (PE) are discussed. These questionnaires meet or exceed all the psychometric requirements and have been employed in clinical trials and observational studies of men with PE. The article concludes on discussing some of the limitations of PRO use and recommendations for the future. PMID:27652219

  12. Joint modeling of multiple longitudinal patient-reported outcomes and survival

    PubMed Central

    Hatfield, Laura A.; Boye, Mark E.; Carlin, Bradley P.

    2011-01-01

    Researchers often include patient-reported outcomes (PROs) in Phase III clinical trials to demonstrate the value of treatment from the patient’s perspective. These data are collected as longitudinal repeated measures and are often censored by occurrence of a clinical event that defines a survival time. Hierarchical Bayesian models having latent individual-level trajectories provide a flexible approach to modeling such multiple outcome types simultaneously. We consider the case of many zeros in the longitudinal data motivating a mixture model, and demonstrate several approaches to modeling multiple longitudinal PROs with survival in a cancer clinical trial. These joint models may enhance Phase III analyses and better inform health care decision makers. PMID:21830926

  13. 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.

  14. 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

  15. Life after prostate cancer diagnosis: protocol for a UK-wide patient-reported outcomes study

    PubMed Central

    Downing, Amy; Wright, Penny; Wagland, Richard; Watson, Eila; Kearney, Therese; Mottram, Rebecca; Allen, Majorie; Cairnduff, Victoria; McSorley, Oonagh; Butcher, Hugh; Hounsome, Luke; Donnelly, Conan; Selby, Peter; Kind, Paul; Cross, William; Catto, James W H; Huws, Dyfed; Brewster, David H; McNair, Emma; Matheson, Lauren; Rivas, Carol; Nayoan, Johana; Horton, Mike; Corner, Jessica; Verne, Julia; Gavin, Anna; Glaser, Adam W

    2016-01-01

    Background Prostate cancer and its treatment may impact physically, psychologically and socially; affecting the health-related quality of life of men and their partners/spouses. The Life After Prostate Cancer Diagnosis (LAPCD) study is a UK-wide patient-reported outcomes study which will generate information to improve the health and well-being of men with prostate cancer. Methods and analysis Postal surveys will be sent to prostate cancer survivors (18–42 months postdiagnosis) in all 4 UK countries (n=∼70 000). Eligible men will be identified and/or verified through cancer registration systems. Men will be surveyed twice, 12 months apart, to explore changes in outcomes over time. Second, separate cohorts will be surveyed once and the design will include evaluation of the acceptability of online survey tools. A comprehensive patient-reported outcome measure has been developed using generic and specific instruments with proven psychometric properties and relevance in national and international studies. The outcome data will be linked with administrative health data (eg, treatment information from hospital data). To ensure detailed understanding of issues of importance, qualitative interviews will be undertaken with a sample of men who complete the survey across the UK (n=∼150) along with a small number of partners/spouses (n=∼30). Ethics and dissemination The study has received the following approvals: Newcastle and North Tyneside 1 Research Ethics Committee (15/NE/0036), Health Research Authority Confidentiality Advisory Group (15/CAG/0110), NHS Scotland Public Benefit and Privacy Panel (0516-0364), Office of Research Ethics Northern Ireland (16/NI/0073) and NHS R&D approval from Wales, Scotland and Northern Ireland. Using traditional and innovative methods, the results will be made available to men and their partners/spouses, the funders, the NHS, social care, voluntary sector organisations and other researchers. PMID:27927667

  16. The Effect of Physician Continuing Medical Education on Patient-Reported Outcomes for Identifying and Optimally Managing Obstructive Sleep Apnea

    PubMed Central

    Johnson, Sara S.; Castle, Patricia H.; Van Marter, Deborah; Roc, Anne; Neubauer, David; Auerbach, Sanford; DeAguiar, Emma

    2015-01-01

    Study Objective: To evaluate the effect of continuing medical education (CME) activities on patient reported outcomes with regard to (1) screening for excessive sleepiness (ES) and obstructive sleep apnea (OSA) and (2) appropriate referral and treatment. Methods: A total of 725 patients were recruited from 75 providers who either participated or did not participate in Transtheoretical Model (TTM)-based OSA CME activities. Patient reported outcomes from participating (n = 36) and non-participating providers (n = 39) were compared using generalized estimating equations examining random effects of provider as unit of assignment. Results: Patients' reports demonstrate that participating physicians were 1.7 times more likely to initiate discussion of sleep problems than non-participating physicians (t1,411 = 3.71, p = 0.05) and 2.25–2.86 times more likely to administer validated measures for OSA (Epworth Sleepiness Scale and STOP-BANG). Patient reports also indicated that participating clinicians (79.9%) were significantly more likely to recommend seeing a sleep specialist compared to non-participating clinicians (60.7%; t1,348 = 9.1, p < 0.01, OR = 2.6). Furthermore, while 89.4% of participating clinicians recommended a sleep study, only 73.2% of the non-participating physicians recommended one (t1,363 = 11.46, p < 0.001, OR = 3.1). Conclusions: Participation in TTM-based OSA CME activities was associated with improved patient reported outcomes compared to the non-participating clinicians. Citation: Johnson SS, Castle PH, Van Marter D, Roc A, Neubauer D, Auerbach S, DeAguiar E. The effect of physician continuing medical education on patient-reported outcomes for identifying and optimally managing obstructive sleep apnea. J Clin Sleep Med 2015;11(3):197–204. PMID:25845903

  17. Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID): Frequently asked questions

    PubMed Central

    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 . 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

  18. mHealth and big data will bring meaning and value to patient-reported outcomes

    PubMed Central

    2016-01-01

    The intersection of widespread mobile adoption, cloud computing and healthcare will enable patient-reported outcomes to be used to personalize care, draw insights and shorten the cycle from research to clinical implementation. Today, patient-reported outcomes are largely collected as part of a regulatory shift to value-based or bundled care. When patients are able to record their experiences in real-time and combine them with passive data collection from sensors and mobile devices, this information can inform better care for each patient and contribute to the growing body of health data that can be used to draw insights for all patients. This paper explores the current limitations of patient reported outcomes and how mobile health and big data analysis unlocks their potential as a valuable tool to deliver care. PMID:28293580

  19. Advantages and Psychometric Validation of Proximal Intensive Assessments of Patient Reported Outcomes Collected in Daily Life

    PubMed Central

    Carlson, Eve B.; Field, Nigel P.; Ruzek, Josef I.; Bryant, Richard A.; Dalenberg, Constance J.; Keane, Terence M.; Spain, David A.

    2016-01-01

    Objectives Ambulatory assessment data collection methods are increasingly used to study behavior, experiences, and patient reported outcomes (PROs) such as emotions, cognitions, and symptoms in clinical samples. Data collected close in time at frequent and fixed intervals can assess PROs that are discrete or changing rapidly and provide information about temporal dynamics or mechanisms of change in clinical samples and individuals, but clinical researchers have not yet routinely and systematically investigated the reliability and validity of such measures or their potential added value over conventional measures. The present study provides a comprehensive, systematic evaluation of the psychometrics of several Proximal Intensive Assessment (PIA) measures in a clinical sample and investigates whether PIA appears to assess meaningful differences in phenomena over time. Methods Data was collected on a variety of psychopathology constructs on handheld devices every 4 hours for 7 days from 62 adults recently exposed to traumatic injury of themselves or a family member. Data was also collected on standard self-report measures of the same constructs at the time of enrollment, one week after enrollment, and two months after injury. Results For all measure scores, results showed good internal consistency across items and within persons over time, provided evidence of convergent, divergent, and construct validity, and showed significant between and within-subject variability. Conclusions Results indicate that PIA measures can provide valid measurement of psychopathology in a clinical sample. PIA may be useful to study mechanisms of change in clinical contexts, identify targets for change, and gauge treatment progress. PMID:26567018

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

    PubMed Central

    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

  1. 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.

  2. Comparison of CTT and Rasch-based approaches for the analysis of longitudinal Patient Reported Outcomes.

    PubMed

    Blanchin, Myriam; Hardouin, Jean-Benoit; Le Neel, Tanguy; Kubis, Gildas; Blanchard, Claire; Mirallié, Eric; Sébille, Véronique

    2011-04-15

    Health sciences frequently deal with Patient Reported Outcomes (PRO) data for the evaluation of concepts, in particular health-related quality of life, which cannot be directly measured and are often called latent variables. Two approaches are commonly used for the analysis of such data: Classical Test Theory (CTT) and Item Response Theory (IRT). Longitudinal data are often collected to analyze the evolution of an outcome over time. The most adequate strategy to analyze longitudinal latent variables, which can be either based on CTT or IRT models, remains to be identified. This strategy must take into account the latent characteristic of what PROs are intended to measure as well as the specificity of longitudinal designs. A simple and widely used IRT model is the Rasch model. The purpose of our study was to compare CTT and Rasch-based approaches to analyze longitudinal PRO data regarding type I error, power, and time effect estimation bias. Four methods were compared: the Score and Mixed models (SM) method based on the CTT approach, the Rasch and Mixed models (RM), the Plausible Values (PV), and the Longitudinal Rasch model (LRM) methods all based on the Rasch model. All methods have shown comparable results in terms of type I error, all close to 5 per cent. LRM and SM methods presented comparable power and unbiased time effect estimations, whereas RM and PV methods showed low power and biased time effect estimations. This suggests that RM and PV methods should be avoided to analyze longitudinal latent variables.

  3. 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.

  4. A Systematic Review of Patient-reported Outcomes in Randomized Controlled Trials of Unplanned General Surgery.

    PubMed

    Stevens, Daniel J; Blencowe, Natalie S; McElnay, Philip J; Macefield, Rhiannon C; Savović, Jelena; Avery, Kerry N L; Blazeby, Jane M

    2016-02-01

    Unplanned general surgery represents a major workload and requires comprehensive evaluation with appropriate outcomes. This study aimed to summarize current reporting of patient-reported outcomes (PROs) in randomized clinical trials (RCTs) in unplanned general surgery. A systematic review identified RCTs reporting PROs in the commonest six areas of unplanned general surgery. Details of the PRO measures were examined using the CONSORT extension for PRO reporting in RCTs. Extracted information about each PRO domain included the reporting of baseline PROs, rationale for PRO selection and whether PRO findings were used in conjunction with clinical outcomes to inform treatment recommendations. The internal validity of included studies was assessed using the Cochrane risk of bias tool. 12,519 abstracts were screened and 20 RCTs containing data from 2037 patients included. Included studies used 14 separate PRO measures covering 35 different health domains. A visual analogue assessment of pain was most frequently reported (n = 13). Reporting of baseline PRO data was uncommon (11/35 PRO domains). The rationale for PRO data collection and a PRO-specific hypothesis were provided for 9 (25.7 %) and 5 (14.3 %) domains, respectively. Seventeen RCTs (85.0 %) used the PRO data alongside clinical outcomes to inform treatment recommendations. Of the 116 risk of bias assessments, 77 (66.0 %) were judged as high or unclear. There is a lack of well designed, and conducted RCTs in unplanned general surgery that include PROs. Future work to define relevant PROs and methods for optimal assessment are needed to inform health care decision-making.

  5. Quality of life among breast cancer patients with lymphedema: a systematic review of patient-reported outcome instruments and outcomes

    PubMed Central

    Cemal, Yeliz; Albornoz, Claudia; Klassen, Anne; Cano, Stefan; Sulimanoff, Isabel; Hernandez, Marisol; Massey, Marga; Cordeiro, Peter; Morrow, Monica; Mehrara, Babak

    2013-01-01

    Purpose Lymphedema following breast cancer surgery remains a common and feared treatment complication. Accurate information on health-related quality of life (HRQOL) outcomes among patients with lymphedema is critically needed to inform shared medical decision making and evidence-based practice in oncologic breast surgery. Our systematic review aimed to (1) identify studies describing HRQOL outcomes in breast cancer-related lymphedema (BCRL) patients, (2) assess the quality of these studies, and (3) assess the quality and appropriateness of the patient-reported outcome (PRO) instruments used. Methods Using the PRISMA statement, we performed a systematic review including studies describing HRQOL outcomes among BCRL patients. Studies were classified by levels of evidence and fulfillment of the Efficace criteria. PRO instruments were assessed using the COSMIN criteria. Results Thirty-nine studies met inclusion criteria, including 8 level I and 14 level II studies. Sixteen of 39 studies were compliant with the Efficace criteria. Seventeen HRQOL instruments were used, two specific to lymphedema patients. Exercise and complex decongestive therapy treatment interventions were associated with improved HRQOL. Conclusions High-quality data on HRQOL outcomes is required to inform surgical decisions for breast cancer management and survivors. Of the lymphedema-specific PRO instruments, the Upper Limb Lymphedema 27 (ULL-27) was found to have strong psychometric properties. Future studies should strive to use high-quality condition-specific PRO instruments, follow existing guidelines for HRQOL measurement and to consider economic burdens of BCRL. Implications for Cancer Survivors As lymphedema may develop many years after breast cancer surgery, the ULL-27 may offer greater content validity for use in survivorship research. PMID:23212603

  6. "Not just little adults": qualitative methods to support the development of pediatric patient-reported outcomes.

    PubMed

    Arbuckle, Rob; Abetz-Webb, Linda

    2013-01-01

    The US FDA and the European Medicines Agency (EMA) have issued incentives and laws mandating clinical research in pediatrics. While guidances for the development and validation of patient-reported outcomes (PROs) or health-related quality of life (HRQL) measures have been issued by these agencies, little attention has focused on pediatric PRO development methods. With reference to the literature, this article provides an overview of specific considerations that should be made with regard to the development of pediatric PRO measures, with a focus on performing qualitative research to ensure content validity. Throughout the questionnaire development process it is critical to use developmentally appropriate language and techniques to ensure outcomes have content validity, and will be reliable and valid within narrow age bands (0-2, 3-5, 6-8, 9-11, 12-14, 15-17 years). For qualitative research, sample sizes within those age bands must be adequate to demonstrate saturation while taking into account children's rapid growth and development. Interview methods, interview guides, and length of interview must all take developmental stage into account. Drawings, play-doh, or props can be used to engage the child. Care needs to be taken during cognitive debriefing, where repeated questioning can lead a child to change their answers, due to thinking their answer is incorrect. For the PROs themselves, the greatest challenge is in measuring outcomes in children aged 5-8 years. In this age range, while self-report is generally more valid, parent reports of observable behaviors are generally more reliable. As such, 'team completion' or a parent-administered child report is often the best option for children aged 5-8 years. For infants and very young children (aged 0-4 years), patient rating of observable behaviors is necessary, and, for adolescents and children aged 9 years and older, self-reported outcomes are generally valid and reliable. In conclusion, the development of PRO

  7. 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

  8. Patient Expectations and Patient-Reported Outcomes in Surgery: A Systematic Review

    PubMed Central

    Waljee, Jennifer; McGlinn, Evan P.; Sears, Erika Davis; Chung, Kevin C.

    2014-01-01

    Background Recent events in healthcare reform have brought national attention to integrating patient experiences and expectations into quality metrics. Few studies have comprehensively evaluated the effect of patient expectations on patient-reported outcomes (PROs) following surgery. The purpose of this study is to systematically review the available literature describing the relationship between patient expectations and postoperative PROs. Methods We performed a search of the literature published prior to November 1, 2012. Articles were included in the review if 1) primary data were presented 2) patient expectations regarding a surgical procedure were measured 3) PROs were measured, and 4) the relationship between patient expectations and PROs was specifically examined. PROs were categorized into five subgroups: satisfaction, quality of life (QOL), disability, mood disorder, and pain. We examined each study to determine the relationship between patient expectations and PROs as well as study quality. Results From the initial literature search yielding 1,708 studies, 60 articles were included. Fulfillment of expectations was associated with improved PROs among 24 studies. Positive expectations were correlated with improved PROs for 28 (47%) studies, and poorer PROs for 9 (15%) studies. Eighteen studies reported that fulfillment of expectations was correlated with improved patient satisfaction, and 10 studies identified that positive expectations were correlated with improved postoperative QOL. Finally, patients with positive preoperative expectations reported less pain (8 studies) and disability (15 studies) compared with patients with negative preoperative expectations. Conclusions Patient expectations are inconsistently correlated with PROs following surgery, and there is no accepted method to capture perioperative expectations. Future efforts to rigorously measure expectations and explore their influence on postoperative outcomes can inform clinicians and policy

  9. 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

  10. 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

  11. Rationale for Using Social Media to Collect Patient-Reported Outcomes in Patients with Celiac Disease.

    PubMed

    Park, Kt; Harris, Merissa; Khavari, Nasim; Khosla, Chaitan

    2014-02-01

    Patients with celiac disease (CD) are increasingly interconnected through social media, exchanging patient experiences and health-tracking information between individuals through various web-based platforms. Social media represents potentially unique communication interface between gastroenterologists and active social media users - especially young adults and adolescents with celiac disease-regarding adherence to the strict gluten-free diet, gastrointestinal symptoms, and meaningful discussion about disease management. Yet, various social media platforms may be underutilized for research purposes to collect patient-reported outcomes data. In this commentary, we summarize the scientific rationale and potential for future growth of social media in patient-reported outcomes research, focusing on college freshmen with celiac disease as a case study and provide overview of the methodological approach. Finally, we discuss how social media may impact patient care in the future through increasing mobile technology use.

  12. 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

  13. Two-year patient-reported outcomes following treatment of uveal melanoma.

    PubMed

    Hope-Stone, L; Brown, S L; Heimann, H; Damato, B; Salmon, P

    2016-12-01

    PurposeTreatment of uveal melanoma can impair patients' psychological well-being. We evaluated patient-reported outcome measures (PROMs) of anxiety, depression, and quality of life (QoL) over 2 years following treatment in a consecutive sample of uveal melanoma patients, compared observations to population normative values and examined whether outcomes differed according to patients' age, gender, and whether or not they were treated by enucleation or had a poor prognosis (presence of monosomy 3).DesignProspective longitudinal study.ParticipantsPatients (N=411) with uveal melanoma treated between 2008 and 2011.MethodsSelf-report questionnaire study. We compared mean PROMs scores obtained 6 months, 1 year, and 2 years after treatment to published population normative values using 2-sample t-tests, and tested the association of these scores with gender, age, treatment by enucleation, and monosomy 3 using mixed-model ANOVAs.ResultsOn QoL and depression, patients were similar to or better than normative values at all time points, but there was some evidence that females were more anxious than female normative values (Ps<0.001-<0.05). Younger patients (P<0.01) and female patients (P<0.01) were the most anxious overall. Enucleation was not associated with PROMs. Patients with monosomy 3 showed more depressed mood at all the three time points (P<0.05).ConclusionsPatients treated for uveal melanoma can expect, within 6 months of treatment, to have a QoL that is similar to that of the general population. Younger female patients and patients with monosomy 3 are more likely to be distressed, and clinicians will need to be alert to this.

  14. Using patient reports to measure health care system performance.

    PubMed

    Hargraves, J L; Palmer, R H; Zapka, J; Nerenz, D; Frazier, H; Orav, E J; Warner, C; Ingard, J; Neisuler, R

    1993-01-01

    We developed a self-administered patient questionnaire that asks for data concerning the time to receive services (access to care), communication between providers (coordination of care), and follow up after tests and treatment (continuity of care). From these data, we construct rates of performance about the clinical management systems that support provision of these services. Rates of system performance are calculated for indicators using patients' responses to survey questions. These indicators add the number of patients reporting a problem of those patients who have encountered a particular clinical management system. Information derived from 3000 patient questionnaires is matched with data abstracted from health care medical records. The sensitivity and specificity of patient reports are being evaluated for all indicators classified as gold standards for medical records. Indicators considered gold standard items for patient reports are matched for agreement with any information contained in the medical record. Also, patient characteristics associated with accurate reporting is to be assessed using multivariate logistic regression models.

  15. Upper Airway Stimulation for Obstructive Sleep Apnea: Patient-Reported Outcomes after 48 Months of Follow-up.

    PubMed

    Gillespie, M Boyd; Soose, Ryan J; Woodson, B Tucker; Strohl, Kingman P; Maurer, Joachim T; de Vries, Nico; Steward, David L; Baskin, Jonathan Z; Badr, M Safwan; Lin, Ho-Sheng; Padhya, Tapan A; Mickelson, Sam; Anderson, W McDowell; Vanderveken, Olivier M; Strollo, Patrick J

    2017-04-01

    Objective To assess patient-based outcomes of participants in a large cohort study-the STAR trial (Stimulation Therapy for Apnea Reduction)-48 months after implantation with an upper airway stimulation system for moderate to severe obstructive sleep apnea. Study Design A multicenter prospective cohort study. Setting Industry-supported multicenter academic and clinical setting. Subjects Participants (n = 91) at 48 months from a cohort of 126 implanted participants. Methods A total of 126 participants received an implanted upper airway stimulation system in a prospective phase III trial. Patient-reported outcomes at 48 months, including Epworth Sleepiness Scale (ESS), Functional Outcomes of Sleep Questionnaire (FOSQ), and snoring level, were compared with preimplantation baseline. Results A total of 91 subjects completed the 48-month visit. Daytime sleepiness as measured by ESS was significantly reduced ( P = .01), and sleep-related quality of life as measured by FOSQ significantly improved ( P = .01) when compared with baseline. Soft to no snoring was reported by 85% of bed partners. Two patients required additional surgery without complication for lead malfunction. Conclusion Upper airway stimulation maintained a sustained benefit on patient-reported outcomes (ESS, FOSQ, snoring) at 48 months in select patients with moderate to severe obstructive sleep apnea.

  16. Effectiveness of a multidisciplinary risk assessment and management programme-diabetes mellitus (RAMP-DM) on patient-reported outcomes.

    PubMed

    Wan, Eric Yuk Fai; Fung, Colman Siu Cheung; Wong, Carlos King Ho; Choi, Edmond Pui Hang; Jiao, Fang Fang; Chan, Anca Ka Chun; Chan, Karina Hiu Yen; Lam, Cindy Lo Kuen

    2017-02-01

    Little is known about how the patient-reported outcomes is influenced by multidisciplinary-risk-assessment-and-management-programme for patients with diabetes mellitus (RAMP-DM). This paper aims to evaluate the effectiveness of RAMP-DM on patient-reported outcomes. This was a prospective longitudinal study on 1039 diabetes mellitus patients (714/325 RAMP-DM/non-RAMP-DM) managed in primary care setting. 536 and 402 RAMP-DM participants, and 237 and 187 non-RAMP-DM participants were followed up at 12 and 24 months with completed survey, respectively. Patient-reported outcomes included health-related quality of life, change in global health condition and patient enablement measured by Short Form-12 Health Survey version-2 (SF-12v2), Global Rating Scale, Patient Enablement Instrument respectively. The effects of RAMP-DM on patient-reported outcomes were evaluated by mixed effect models. Subgroup analysis was performed by stratifying haemoglobin A1c (HbA1c) (optimal HbA1c < 7 % and suboptimal HbA1c ≥ 7 %). RAMP-DM with suboptimal HbA1c was associated with greater improvement in SF-12v2 physical component summary score at 12-month (coefficient:3.80; P-value < 0.05) and 24-month (coefficient:3.82;P-value < 0.05), more likely to feel more enabled at 12-month (odds ratio: 2.57; P-value < 0.05), and have improved in GRS at 24-month (odds ratio:4.05; P-value < 0.05) compared to non-RAMP-DM participants. However, there was no significant difference in patient-reported outcomes between RAMP-DM and non-RAMP-DM participants with optimal HbA1c. Participation in RAMP-DM is effective in improving physical component of HRQOL, Global Rating Scale and patient enablement among diabetes mellitus patients with suboptimal HbA1c, but not in those with optimal HbA1c. Patients with sub-optimal diabetes mellitus control should be the priority target population for RAMP-DM. This observational study design may have potential bias in the characteristics between

  17. Influence of response shift on early patient-reported outcomes following autologous chondrocyte implantation

    PubMed Central

    Howard, Jennifer S.; Mattacola, Carl G.; Mullineaux, David R.; English, Robert A.; Lattermann, Christian

    2013-01-01

    Purpose Response shift is the phenomenon by which an individual's standards for evaluation change over time. The purpose of this study was to determine whether patients undergoing autologous chondrocyte implantation (ACI) experience response shift. Methods Forty-eight patients undergoing ACI participated. The “then-test” method was used to evaluate response shift in commonly used patient-reported outcome measures (PROMs)—the SF-36 Physical Component Scale (SF-36 PCS), WOMAC, IKDC, and Lysholm. Each PROM was completed pre- and 6 and 12 months post-surgery. At 6 and 12 months, an additional “then” version of each form was also completed. The “then” version was identical to the original except that patients were instructed to assess how they were prior to ACI. Traditional change, response shift adjusted change, and response shift magnitude were calculated at 6 and 12 months. T tests (p < 0.05) were used to compare traditional change to response-shift-adjusted change, and response shift magnitude values to previously established minimal detectable change. Results There were no differences between traditional change and response-shift-adjusted change for any of the PROMs. The mean response shift magnitude value of the WOMAC at 6 months (15 ± 14, p = 0.047) was greater than the previously established minimal detectable change (10.9). The mean response shift magnitude value for the SF-36 PCS at 12 months (9.4 ± 6.8, p = 0.017) also exceeded the previously established minimal detectable change (6.6). Conclusions There was no evidence of a group-level effect for response shift. These results support the validity of pre-test/post-test research designs in evaluating treatment effects. However, there is evidence that response shifts may occur on a patient-by-patient basis, and scores on the WOMAC and SF-36 in particular may be influenced by response shift. Level of evidence II. PMID:24061717

  18. [The benefits of using patient-reported outcomes in cancer treatment: an overview].

    PubMed

    Wintner, Lisa M; Giesinger, Johannes M; Kemmler, Georg; Sztankay, Monika; Oberguggenberger, Anne; Gamper, Eva-Maria; Sperner-Unterweger, Barbara; Holzner, Bernhard

    2012-05-01

    The patient's perspective evaluated by patient-reported outcomes (PROs) gains more and more importance, since treatment efficacy is no longer solely linked to clinical outcomes like cure and overall survival. Ailments like pain, fatigue and social isolation can only be assessed by patients' direct expression without any interpretation made by medical staff. PROs facilitate the disclosure of quality of life issues and patients feel a stronger support due to improved communication. PROs offer many further advantages like saving of time, cost and staff, targeted intervention and sensitizing of clinicians. Also, internationally validated questionnaires are available and the development of electronic PROs eases data-collection, calculation and storage. PROs collected within clinical routine are versatile concerning their applicability: They can be used for scientific analyses, quality assurance, and health technology assessment.

  19. Recommended Patient-Reported Core Set of Symptoms to Measure in Adult Cancer Treatment Trials

    PubMed Central

    Mitchell, Sandra A.; Dueck, Amylou C.; Basch, Ethan; Cella, David; Reilly, Carolyn Miller; Minasian, Lori M.; Denicoff, Andrea M.; O’Mara, Ann M.; Fisch, Michael J.; Chauhan, Cynthia; Aaronson, Neil K.; Coens, Corneel; Bruner, Deborah Watkins

    2014-01-01

    Background The National Cancer Institute’s Symptom Management and Health-Related Quality of Life Steering Committee held a clinical trials planning meeting (September 2011) to identify a core symptom set to be assessed across oncology trials for the purposes of better understanding treatment efficacy and toxicity and to facilitate cross-study comparisons. We report the results of an evidence-synthesis and consensus-building effort that culminated in recommendations for core symptoms to be measured in adult cancer clinical trials that include a patient-reported outcome (PRO). Methods We used a data-driven, consensus-building process. A panel of experts, including patient representatives, conducted a systematic review of the literature (2001–2011) and analyzed six large datasets. Results were reviewed at a multistakeholder meeting, and a final set was derived emphasizing symptom prevalence across diverse cancer populations, impact on health outcomes and quality of life, and attribution to either disease or anticancer treatment. Results We recommend that a core set of 12 symptoms—specifically fatigue, insomnia, pain, anorexia (appetite loss), dyspnea, cognitive problems, anxiety (includes worry), nausea, depression (includes sadness), sensory neuropathy, constipation, and diarrhea—be considered for inclusion in clinical trials where a PRO is measured. Inclusion of symptoms and other patient-reported endpoints should be well justified, hypothesis driven, and meaningful to patients. Conclusions This core set will promote consistent assessment of common and clinically relevant disease- and treatment-related symptoms across cancer trials. As such, it provides a foundation to support data harmonization and continued efforts to enhance measurement of patient-centered outcomes in cancer clinical trials and observational studies. PMID:25006191

  20. Patient-reported outcomes and associations with pleural effusion in outpatients with heart failure: an observational cohort study

    PubMed Central

    Gundersen, Guri H; Norekvål, Tone M; Graven, Torbjørn; Haug, Hilde H; Skjetne, Kyrre; Kleinau, Jens O; Gustad, Lise T; Dalen, Håvard

    2017-01-01

    Objectives We aimed to study whether patient-reported outcomes, measured by quality of life (QoL) and functional class, are sensitive to pleural effusion (PLE) in patients with heart failure (HF), and to study changes in QoL and functional class during follow-up of PLE. Methods A cohort of 62 patients from an outpatient HF clinic was included. The amount of PLE was quantified using a pocket-sized ultrasound imaging device. Self-reports of QoL and functional class were collected using the Minnesota Living with Heart Failure Questionnaire (MLHFQ) and the New York Heart Association (NYHA) functional classification. Results At baseline, 26 (42%) patients had PLE of which 19 (31%) patients had moderate to severe amounts of PLE. Patients with no to mild PLE had a lower MLHFQ score (mean 42, SD 21) compared with patients with a moderate to severe amount of PLE (mean 55, SD 24), p=0.03. For 28 patients (45%) with follow-up data, we observed a linear improvement of the MLHFQ-score (3.2, 95% CI 1.2 to 5.1) with each centimetre reduction of PLE. Correspondingly, patient-reported NYHA-class followed the same pattern as the MLHFQ-score. Conclusions Our study indicates that patient-reported outcome measures as MLHFQ may be sensitive tools to identify patients with HF at highest risk of symptomatic PLE and that treatment targeting reduction of PLE during follow-up is essential to improvement of QoL and functional capacity of outpatients with HF. Trial registration number NCT01794715; Results PMID:28320791

  1. Sarilumab improves patient-reported outcomes in rheumatoid arthritis patients with inadequate response/intolerance to tumour necrosis factor inhibitors

    PubMed Central

    Strand, Vibeke; Reaney, Matthew; Chen, Chieh-I; Proudfoot, Clare W J; Guillonneau, Sophie; Bauer, Deborah; Mangan, Erin; Graham, Neil M H; van Hoogstraten, Hubert; Lin, Yong; Pacheco-Tena, César; Fleischmann, Roy

    2017-01-01

    Objective To evaluate effects of the anti-interleukin-6 receptor monoclonal antibody sarilumab administered with conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) on patient-reported outcomes (PROs) in the TARGET trial in patients with rheumatoid arthritis (RA) with inadequate response or intolerance to tumour necrosis factor inhibitors (TNF-IR). Methods 546 patients (81.9% female, mean age 52.9 years) were randomised to placebo, sarilumab 150 or 200 mg subcutaneously every 2 weeks + csDMARDs. PROs included patient global assessment (PtGA); pain and morning stiffness visual analogue scales; Health Assessment Questionnaire Disability Index (HAQ-DI); Short Form-36 Health Survey (SF-36); FACIT-Fatigue (FACIT-F); Work Productivity Survey-Rheumatoid Arthritis (WPS-RA) and Rheumatoid Arthritis Impact of Disease (RAID). Changes from baseline at weeks 12 and 24 were analysed using a mixed model for repeated measures; post hoc analyses included percentages of patients reporting improvements ≥ minimum clinically important differences (MCID) and scores ≥ normative values. Results Sarilumab + csDMARDs doses resulted in improvements from baseline at week 12 vs placebo + csDMARDs in PtGA, pain, HAQ-DI, SF-36 and FACIT-F that were maintained at week 24. Sarilumab improved morning stiffness and reduced the impact of RA on work, family, social/leisure activities participation (WPS-RA) and on patients' lives (RAID). Percentages of patients reporting improvements ≥MCID and ≥ normative scores were greater with sarilumab than placebo. Conclusions In patients with TNF-IR RA, 150 and 200 mg sarilumab + csDMARDs resulted in clinically meaningful patient-reported benefits on pain, fatigue, function, participation and health status at 12 and 24 weeks that exceeded placebo + csDMARDs, and were consistent with the clinical profile previously reported. Trial registration number NCT01709578; Results. PMID:28326189

  2. Relationship between patient-reported and objective measurements of hand function in patients with rheumatoid arthritis.

    PubMed

    Günay, S M; Tuna, Z; Oskay, D

    2016-12-31

    Rheumatoid arthritis (RA) often results in impairments in upper extremities, especially in the small joints of hand. Involvement of hand brings limitations in activities of daily living. However, it is commonly observed that patient-reported functional status of hand does not always corresponds to their actual physical performance in the clinical setting. The aim of this pilot study is to investigate the relationship between patient self-reported and objectively measured hand functions in patients with RA. Twenty-six patients (51±13 years) with RA diagnosis participated in the study. Hand grip and pinch (lateral, bipod, tripod) strengths were measured and Jebsen Hand Function Test (JHFT) was performed for objective functional performance. Duruöz Hand Index and Beck Depression Inventory - Turkish version were completed by patients. Grip and all three-pinch strength results significantly correlated with Duruöz Hand Index scores (p<0.05). JHFT results except the sentence writing also correlated with the Duruöz scores (p<0.05). Our results showed that self-reported outcome scales might be used for determining functional level of hand in patients with RA in rheumatology practice. Objective quantitative functional tests are the best methods in evaluating functional level of hand, but require valid and reliable equipment with accurate calibration. Therefore, in case of unavailability of objective assessment tools, patient-reported scales may also reflect the real status of hand functions.

  3. 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.

  4. 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.

  5. 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.

  6. Patient-Reported Outcomes in Oncology Drug Labeling in the United States: A Framework for Navigating Early Challenges

    PubMed Central

    Shields, Alan L.; Hao, Yanni; Krohe, Meaghan; Yaworsky, Andrew; Mazar, Iyar; Foley, Catherine; Mehmed, Faisal; Globe, Denise

    2016-01-01

    Background Despite an increased use of patient-reported outcomes (PROs) in oncology clinical trials, integrating the patient perspective into drug approval decisions and documentation has been challenging. Objectives To review important regulatory and measurement terminology, and to provide oncology outcomes researchers and those involved with building oncology programs with tools to plan PRO data collection, particularly in relation to drug efficacy claims for drug labeling in the United States. Discussion When contemplating a PRO measurement strategy for oncology clinical trials, outcomes researchers are challenged in several ways. First, given multiple stakeholders, researchers must communicate with their scientific, commercial, and regulatory colleagues using often misunderstood terms, such as “label,” “claim,” “end point,” “outcome,” and “concept.” Second, because stakeholders do not always have access to data from early-stage clinical trials and do not contribute to the target drug's profile in early development, researchers are often unable to address the most important question in building a measurement strategy: What do we want to say about our drug? To overcome these challenges, researchers can systematically develop an end point model to facilitate communication among drug development stakeholders using a common language and to link the building blocks of a PRO measurement strategy, including claims, concepts, questionnaires, and end points. We developed a model that characterizes a disease by its proximal signs and/or symptoms and increasingly distal health outcomes to provide researchers potential measurement concepts that can be instrumental in selecting PRO questionnaires for use in studies. Conclusion PRO data collected in clinical trials should be used in drug development to evaluate the drug's efficacy; it is encouraging that US regulators are willing to work with drug sponsors to overcome the challenges associated with the

  7. Comparative Effectiveness Study of Patient-Reported Outcomes following Proton Therapy or IMRT for Prostate Cancer

    PubMed Central

    Hoppe, Bradford S.; Michalski, Jeff M.; Mendenhall, Nancy P.; Morris, Christopher G.; Henderson, Randal H.; Nichols, Romaine C.; Mendenhall, William M.; Williams, Christopher; Regan, Meredith M.; Chipman, Jonathan; Crociani, Catrina; Sandler, Howard M.; Sanda, Martin G.; Hamstra, Daniel A.

    2014-01-01

    Background Data continues to emerge on the relative merits of different treatment modalities for prostate cancer. The purpose of this study is to compare patient-reported quality-of-life outcomes (QOL) after proton therapy (PT) and intensity-modulated radiation therapy (IMRT) for prostate cancer. Methods A comparison was performed of prospectively collected QOL data using the expanded prostate cancer index (EPIC) questionnaire. QOL data was collected during the first 2 years following treatment for men treated with PT and IMRT. PT was delivered to 1,243 men at a single center to 76-82Gy. IMRT was delivered to 204 men included in the Prostate Cancer Quality Assurance Study (PROSTQA) in doses of 75.6-79.4Gy.The Wilcoxon rank sum test was used to compare EPIC outcomes by modality using baseline-adjusted scores at different time points. Individual questions were assessed by converting to binary outcomes and testing with generalized estimating equations. Results No differences in changes in summary scores for bowel, urinary incontinence, urinary irritative/obstructive, and sexual domains were seen between the two cohorts. However, more men treated with IMRT reported moderate/big problems with rectal urgency (p=0.02) and frequent bowel movements (p=0.05) than men treated with PT. Conclusions There were no differences in QOL summary scores between the IMRT and PT cohorts during early follow-up up to 2-years. Response to individual questions suggests possible differences in specific bowel symptoms between the two cohorts. These outcomes highlight the need for further comparative studies of PT and IMRT. PMID:24382757

  8. Development and psychometric evaluation of the FACE-Q satisfaction with appearance scale: a new patient-reported outcome instrument for facial aesthetics patients.

    PubMed

    Pusic, Andrea L; Klassen, Anne F; Scott, Amie M; Cano, Stefan J

    2013-04-01

    Satisfaction with appearance and improved quality of life are key outcomes for patients undergoing facial aesthetic procedures. The FACE-Q is a new patient-reported outcome (PRO) instrument encompassing a suite of independently functioning scales designed to measure a range of important outcomes for facial aesthetics patients. FACE-Q scales were developed with strict adherence to international guidelines for PRO instrument development. This article describes the development and psychometric evaluation of the core FACE-Q scale, the Satisfaction with Facial Appearance scale. Both modern and traditional psychometric methods were used to confirm that this new 10-item scale is a reliable, valid, and responsive measure.

  9. Patient-reported outcome following nonsurgical management of type II odontoid process fractures in adults

    PubMed Central

    Fam, Maged D; Zeineddine, Hussein A; Nassir, Rafiq Muhammed; Bhatt, Pragnesh; Kamel, Mahmoud H

    2017-01-01

    Background: Transverse (type II) odontoid process fracture is among the most commonly encountered cervical spine fractures. Nonsurgical management through external immobilization is occasionally preferred to surgical management but is criticized for its higher rates of failure and lower patient satisfaction. Our aim is to analyze patient-reported outcomes in patients who underwent nonsurgical treatment for type II odontoid fractures. Methods: We identified patients >18-year-old who underwent external immobilization as a treatment for isolated type II odontoid fracture between 2007 and 2012. We collected demographic parameters, clinical presentation, mode of injury, imaging studies and modality and duration of treatment (soft collar, halo-vest, or both). Patients were contacted by telephone to participate in a 15-min survey addressing their recovery including their subjective rate of return to preinjury level of functioning. Results: Fifteen patients met the inclusion/exclusion criteria and participated in our survey. Patients were followed up for an average of 19 months after injury. Overall mean age was 61 years. Injury followed a mechanical fall or a road traffic accident in 11 and 4 cases, respectively. External immobilization was achieved by halo vest only in nine patients, soft collar only in two patients (13%), and through a sequential combination in the remaining 4 (27%). This was deployed for a mean of 7.8 months. Radiological studies at the last follow-up showed bony healing (27%), fibrous nonunion (60%), and persistent instability (13%). Patients reported gradual recovery of function throughout the 1st year after injury with levels above 70% of preinjury functioning achieved by 13% of patients at 6 months, 33% at 9 months, and 47% at 12 months. Overall satisfaction with nonsurgical management was 68%. Conclusion: In selected patients with type II odontoid fractures, external immobilization represents a good option with acceptable course of recovery. PMID

  10. 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.

  11. 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

  12. 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

  13. 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…

  14. Comparing a tablet computer and paper forms for assessing patient-reported outcomes in edentulous patients

    PubMed Central

    Caetano, Thais Angelina; Ribeiro, Adriana Barbosa; Della Vecchia, Maria Paula; Cunha, Tatiana Ramirez; Chaves, Carolina de Andrade Lima

    2016-01-01

    PURPOSE The aim of this study was to determine whether two methods of documentation, print and electronic forms, for the assessment of patient-reported outcomes (PRO) in complete denture wearers provide comparable results. The study also quantified the time needed for filling the forms by each method. MATERIALS AND METHODS Thirty participants enrolled in a university clinic answered two forms (a questionnaire for denture satisfaction and OHIP-EDENT). They provided answers with two application methods in a random order, with a one-month interval between them: (1) electronic forms on a tablet computer; and (2) print forms. The methods were compared in terms of mean results, correlation/agreement, internal consistency, and spent time. RESULTS Mean results for both methods were similar for each denture satisfaction item (100-mm VAS) and OHIP-EDENT summary score. Both questionnaires presented good internal consistency regardless of the application method (Cronbach's α=0.86 or higher). Correlation and agreement between the methods regarding specific items was at least moderate for the majority of cases. Mean time for the electronic and print forms were 9.2 and 8.5 minutes, respectively (paired t test, P=.06, non-significant). CONCLUSION The electronic method is comparable to print forms for the assessment of important PRO of prosthetic treatment for edentulism, considering the results and time needed. Findings suggest the viability of replacing print forms with a tablet for applying the tested inventories in clinical trials. PMID:28018563

  15. Practical and statistical issues in missing data for longitudinal patient-reported outcomes.

    PubMed

    Bell, Melanie L; Fairclough, Diane L

    2014-10-01

    Patient-reported outcomes are increasingly used in health research, including randomized controlled trials and observational studies. However, the validity of results in longitudinal studies can crucially hinge on the handling of missing data. This paper considers the issues of missing data at each stage of research. Practical strategies for minimizing missingness through careful study design and conduct are given. Statistical approaches that are commonly used, but should be avoided, are discussed, including how these methods can yield biased and misleading results. Methods that are valid for data which are missing at random are outlined, including maximum likelihood methods, multiple imputation and extensions to generalized estimating equations: weighted generalized estimating equations, generalized estimating equations with multiple imputation, and doubly robust generalized estimating equations. Finally, we discuss the importance of sensitivity analyses, including the role of missing not at random models, such as pattern mixture, selection, and shared parameter models. We demonstrate many of these concepts with data from a randomized controlled clinical trial on renal cancer patients, and show that the results are dependent on missingness assumptions and the statistical approach.

  16. Impact of genomic testing and patient-reported outcomes on receipt of adjuvant chemotherapy.

    PubMed

    Evans, Chalanda N; Brewer, Noel T; Vadaparampil, Susan T; Boisvert, Marc; Ottaviano, Yvonne; Lee, M Catherine; Isaacs, Claudine; Schwartz, Marc D; O'Neill, Suzanne C

    2016-04-01

    Practice guidelines incorporate genomic tumor profiling, using results such as the Oncotype DX Recurrence Score (RS), to refine recurrence risk estimates for the large proportion of breast cancer patients with early-stage, estrogen receptor-positive disease. We sought to understand the impact of receiving genomic recurrence risk estimates on breast cancer patients' well-being and the impact of these patient-reported outcomes on receipt of adjuvant chemotherapy. Participants were 193 women (mean age 57) newly diagnosed with early-stage breast cancer. Women were interviewed before and 2-3 weeks after receiving the RS result between 2011 and 2015. We assessed subsequent receipt of chemotherapy from chart review. After receiving their RS, perceived pros (t = 4.27, P < .001) and cons (t = 8.54, P < .001) of chemotherapy increased from pre-test to post-test, while perceived risk of breast cancer recurrence decreased (t = 2.90, P = .004). Women with high RS tumors were more likely to receive chemotherapy than women with low RS tumors (88 vs. 5 %, OR 0.01, 0.00-0.02, P < .001). Higher distress (OR 2.19, 95 % CI 1.05-4.57, P < .05) and lower perceived cons of chemotherapy (OR 0.50, 95 % CI 0.26-0.97, P < .05) also predicted receipt of chemotherapy. Distressed patients who saw few downsides of chemotherapy received this treatment. Clinicians should consider these factors when discussing chemotherapy with breast cancer patients.

  17. Patient-Reported Outcomes and Survivorship in Radiation Oncology: Overcoming the Cons

    PubMed Central

    Siddiqui, Farzan; Liu, Arthur K.; Watkins-Bruner, Deborah; Movsas, Benjamin

    2014-01-01

    Purpose Although patient-reported outcomes (PROs) have become a key component of clinical oncology trials, many challenges exist regarding their optimal application. The goal of this article is to methodically review these barriers and suggest strategies to overcome them. This review will primarily focus on radiation oncology examples, will address issues regarding the “why, how, and what” of PROs, and will provide strategies for difficult problems such as methods for reducing missing data. This review will also address cancer survivorship because it closely relates to PROs. Methods Key articles focusing on PROs, quality of life, and survivorship issues in oncology trials are highlighted, with an emphasis on radiation oncology clinical trials. Publications and Web sites of various governmental and regulatory agencies are also reviewed. Results The study of PROs in clinical oncology trials has become well established. There are guidelines provided by organizations such as the US Food and Drug Administration that clearly indicate the importance of and methodology for studying PROs. Clinical trials in oncology have repeatedly demonstrated the value of studying PROs and suggested ways to overcome some of the key challenges. The Radiation Therapy Oncology Group (RTOG) has led some of these efforts, and their contributions are highlighted. The current state of cancer survivorship guidelines is also discussed. Conclusion The study of PROs presents significant benefits in understanding and treating toxicities and enhancing quality of life; however, challenges remain. Strategies are presented to overcome these hurdles, which will ultimately improve cancer survivorship. PMID:25113760

  18. The regulation of patient-reported outcome claims: need for a flexible standard.

    PubMed

    Morris, Louis A; Miller, David W

    2002-01-01

    We review the FDA's policies for the regulation of patient-reported outcome (PRO) claims such as quality of life, productivity, satisfaction and symptom reports and suggest alternative standards for substantiation. We base our review on FDA regulatory activities and public statements in the field of advertising substantiation. We compare these activities to the FDA's label substantiation policies and policies for health-economic (HE) claim substantiation. There is an overt inconsistency between the FDA's policies for substantiation of PRO claims in product labels and substantiation for such claims in advertising materials. This results in a higher standard for PRO claims in promotional vehicles than in product labels. Rather than relying on a "substantial evidence" standard, the FDA should consider a more flexible standard, such as the one currently applied to information included in the Clinical Trials section of product labels, or adopting a "competent and reliable scientific evidence" standard as set forth in Section 114 of the Food and Drug Administration Modernization Act (FDAMA) for HE data. We conclude that there needs to be greater consistency for substantiation in product labels and promotional materials. Furthermore, reconceptualizing most PRO claims as benefit extrapolations as opposed to efficacy information suggests a less rigorous standard is necessary.

  19. The CONSORT Patient-Reported Outcome (PRO) extension: implications for clinical trials and practice

    PubMed Central

    2013-01-01

    To inform clinical guidelines and patient care we need high quality evidence on the relative benefits and harms of intervention. Patient reported outcome (PRO) data from clinical trials can “empower patients to make decisions based on their values” and “level the playing field between physician and patient”. While clinicians have a good understanding of the concept of health-related quality of life and other PROs, evidence suggests that many do not feel comfortable in using the data from trials to inform discussions with patients and clinical practice. This may in part reflect concerns over the integrity of the data and difficulties in interpreting the results arising from poor reporting. The new CONSORT PRO extension aims to improve the reporting of PROs in trials to facilitate the use of results to inform clinical practice and health policy. While the CONSORT PRO extension is an important first step in the process, we need broader engagement with the guidance to facilitate optimal reporting and maximize use of PRO data in a clinical setting. Endorsement by journal editors, authors and peer reviewers are crucial steps. Improved design, implementation and transparent reporting of PROs in clinical trials are necessary to provide high quality evidence to inform evidence synthesis and clinical practice guidelines. PMID:24168680

  20. Dual bronchodilation in COPD: lung function and patient-reported outcomes - a review.

    PubMed

    Price, David; Østrem, Anders; Thomas, Mike; Welte, Tobias

    2017-01-01

    Several fixed-dose combinations (FDCs) of long-acting bronchodilators (a long-acting muscarinic antagonist [LAMA] plus a long-acting β2-agonist [LABA]) are available for the treatment of COPD. Studies of these FDCs have demonstrated substantial improvements in lung function (forced expiratory volume in 1 second) in comparison with their respective constituent monocomponents. Improvements in patient-reported outcomes (PROs), such as symptoms and health status, as well as exacerbation rates, have been reported compared with a LABA or LAMA alone, but results are less consistent. The inconsistencies may in part be owing to differences in study design, methods used to assess study end points, and patient populations. Nevertheless, these observations tend to support an association between improvements in forced expiratory volume in 1 second and improvements in symptom-based outcomes. In order to assess the effects of FDCs on PROs and evaluate relationships between PROs and changes in lung function, we performed a systematic literature search of publications reporting randomized controlled trials of FDCs. Results of this literature search were independently assessed by two reviewers, with a third reviewer resolving any conflicting results. In total, 22 Phase III randomized controlled trials of FDC bronchodilators in COPD were identified, with an additional study including a post-literature search (ten for indacaterol-glycopyrronium once daily, eight for umeclidinium-vilanterol once daily, three for tiotropium-olodaterol once daily, and two for aclidinium-formoterol twice daily). Results from these studies demonstrated that the LAMA-LABA FDCs significantly improved lung function compared with their component monotherapies or other single-agent treatments. Furthermore, LABA-LAMA combinations also generally improved symptoms and health status versus monotherapies, although some discrepancies between lung function and PROs were observed. Overall, the safety profiles of the

  1. Quality of Life and Patient-Reported Outcomes in Breast Cancer Survivors

    PubMed Central

    Macadam, Sheina A.; Zhong, Toni; Weichman, Katie; Papsdorf, Michael; Lennox, Peter A.; Hazen, Alexes; Matros, Evan; Disa, Joseph; Mehrara, Babak; Pusic, Andrea L.; Van Laeken, Nancy; Cordeiro, Peter; Vidal, Dale; Klassen, Anne; Cano, Stefan

    2016-01-01

    Background Approximately 20% of women select autologous tissue for postmastectomy breast reconstruction, and most commonly choose the abdomen as the donor site. An increasing proportion of women are seeking muscle-sparing procedures but the benefit remains controversial. It is therefore important to determine whether better outcomes are associated with these techniques, thereby justifying longer operative times and increased costs. Methods Patients from five North American centers were eligible if they had reconstruction using the deep inferior epigastric artery perforator flap (DIEP), muscle sparing free transverse abdominis myocutaneous flap (msf-TRAM), free transverse abdominis myocutaneous flap (f-TRAM), or the pedicled transverse abdominis myocutaneous flap (p-TRAM) with minimum one-year follow-up. Patients were sent the BREAST-Q©. Demographics and complications were collected by chart review. Results We analyzed 1790 charts representing 670 DIEP, 293 msf-TRAM, 683 p-TRAM, and 144 f-TRAM patients with average follow up of 5.5 years. Flap loss did not differ by flap type. Partial flap loss was higher in p-TRAM compared to DIEP (p=0.002). Fat necrosis was higher in p-TRAM compared to DIEP and msf-TRAM (p<0.001). Hernia/bulge was highest in p-TRAM (p<0.001). Physical Well-Being (Abdomen) scores were higher in DIEP compared to p-TRAM controlling for age, follow-up, BMI, laterality, abdominal surgery, mesh, radiation, income, and education. Conclusions Complications and patient-reported outcomes differ when comparing abdominally-based breast reconstruction techniques. The results of this study show that the DIEP was associated with the highest abdominal well-being and the lowest abdominal morbidity when compared to the p-TRAM, but did not differ from msf-TRAM and f-TRAM. PMID:26910656

  2. Muscle function is associated with future patient-reported outcomes in young adults with ACL injury

    PubMed Central

    Flosadottir, Vala; Roos, Ewa M; Ageberg, Eva

    2016-01-01

    Background/aim Consequences of an anterior cruciate ligament (ACL) injury include worse patient-reported outcomes (PROs) and a decrease in activity level. Muscle function can be improved by targeted exercise. Our aims were to investigate cross-sectional and longitudinal associations among lower extremity muscle function and PROs after ACL injury. Methods Fifty-four participants (15 women, mean 30 years) with ACL injury or reconstruction, from the Knee Anterior Cruciate Ligament, Nonsurgical versus Surgical Treatment (KANON) trial (ISRCTN84752559), were assessed with hop performance, muscle power and postural orientation 3 years (SD 0.85) after ACL injury. PROs at 3 and 5 years after injury included Knee Injury and Osteoarthritis Outcome Score (KOOS) subscales Function in sport and recreation (KOOS Sport/rec) and Knee-related Quality of life (KOOS QoL), KOOS item Q3 (KOOS Q3), Tegner Activity Scale and Activity Rating Scale (ARS). Partial Spearman's rank-order correlation was used to analyse correlations between muscle function and PROs, controlling for gender and treatment. Results Numerous cross-sectional correlations were observed between muscle function and PROs (rsp≈0.3–0.5, p≤0.045). Worse hop performance and worse postural orientation were associated with worse KOOS scores 2 years later (rsp≥0.280, p≤0.045). Worse muscle power was associated with lower future ARS scores (rsp=0.281, p=0.044). Conclusions The moderate associations suggest that improving muscle function during rehabilitation could improve present and future PROs. PMID:27900196

  3. Dual bronchodilation in COPD: lung function and patient-reported outcomes – a review

    PubMed Central

    Price, David; Østrem, Anders; Thomas, Mike; Welte, Tobias

    2017-01-01

    Several fixed-dose combinations (FDCs) of long-acting bronchodilators (a long-acting muscarinic antagonist [LAMA] plus a long-acting β2-agonist [LABA]) are available for the treatment of COPD. Studies of these FDCs have demonstrated substantial improvements in lung function (forced expiratory volume in 1 second) in comparison with their respective constituent monocomponents. Improvements in patient-reported outcomes (PROs), such as symptoms and health status, as well as exacerbation rates, have been reported compared with a LABA or LAMA alone, but results are less consistent. The inconsistencies may in part be owing to differences in study design, methods used to assess study end points, and patient populations. Nevertheless, these observations tend to support an association between improvements in forced expiratory volume in 1 second and improvements in symptom-based outcomes. In order to assess the effects of FDCs on PROs and evaluate relationships between PROs and changes in lung function, we performed a systematic literature search of publications reporting randomized controlled trials of FDCs. Results of this literature search were independently assessed by two reviewers, with a third reviewer resolving any conflicting results. In total, 22 Phase III randomized controlled trials of FDC bronchodilators in COPD were identified, with an additional study including a post-literature search (ten for indacaterol–glycopyrronium once daily, eight for umeclidinium–vilanterol once daily, three for tiotropium–olodaterol once daily, and two for aclidinium–formoterol twice daily). Results from these studies demonstrated that the LAMA–LABA FDCs significantly improved lung function compared with their component monotherapies or other single-agent treatments. Furthermore, LABA–LAMA combinations also generally improved symptoms and health status versus monotherapies, although some discrepancies between lung function and PROs were observed. Overall, the safety

  4. Intraoperative medial pivot affects deep knee flexion angle and patient-reported outcomes after total knee arthroplasty.

    PubMed

    Nishio, Yusuke; Onodera, Tomohiro; Kasahara, Yasuhiko; Takahashi, Daisuke; Iwasaki, Norimasa; Majima, Tokifumi

    2014-04-01

    The aim of this study was to evaluate the relationship between clinical results including patient-reported outcomes and intraoperative knee kinematic patterns after total knee arthroplasty (TKA). A cross-sectional survey of forty consecutive medial osteoarthritis patients who had a primary TKA using a CT-based navigation system was conducted. Subjects were divided into two groups based on intraoperative kinematic patterns: a medial pivot group (n = 20) and a non-medial pivot group (n = 20). Subjective outcomes with the new Knee Society Score and clinical outcomes were evaluated. The functional activities, patient satisfaction and the knee flexion angle of the medial pivot group were significantly better than those of the non-medial pivot group. An intraoperative medial pivot pattern positively influences deep knee flexion and patient-reported outcomes.

  5. 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

  6. Interpreting patient-reported outcomes from clinical trials in COPD: a discussion.

    PubMed

    Jones, Paul W; Rennard, Stephen; Tabberer, Maggie; Riley, John H; Vahdati-Bolouri, Mitra; Barnes, Neil C

    2016-01-01

    One of the challenges faced by the practising physician is the interpretation of patient-reported outcomes (PROs) in clinical trials and the relevance of such data to their patients. This is especially true when caring for patients with progressive diseases such as COPD. In an attempt to incorporate the patient perspective, many clinical trials now include assessments of PROs. These are formalized methods of capturing patient-centered information. Given the importance of PROs in evaluating the potential utility of an intervention for a patient with COPD, it is important that physicians are able to critically interpret (and critique) the results derived from them. Therefore, in this paper, a series of questions is posed for the practising physician to consider when reviewing the treatment effectiveness as assessed by PROs. The focus is on the St George's Respiratory Questionnaire for worked examples, but the principles apply equally to other symptom-based questionnaires. A number of different ways of presenting PRO data are discussed, including the concept of the minimum clinically important difference, whether there is a ceiling effect to PRO results, and the strengths and weaknesses of responder analyses. Using a worked example, the value of including a placebo arm in a study is illustrated, and the influence of the study on PRO results is considered, in terms of the design, patient withdrawal, and the selection of the study population. For the practising clinician, the most important consideration is the importance of individualization of treatment (and of treatment goals). To inform such treatment, clinicians need to critically review PRO data. The hope is that the questions posed here will help to build a framework for this critical review.

  7. Patient-reported outcomes in ductal carcinoma in situ: A systematic review.

    PubMed

    King, Madeleine T; Winters, Zoë E; Olivotto, Ivo A; Spillane, Andrew J; Chua, Boon H; Saunders, Christobel; Westenberg, A Helen; Mann, G Bruce; Burnett, Petrina; Butow, Phyllis; Rutherford, Claudia

    2017-01-01

    Ductal carcinoma in situ (DCIS) is a pre-invasive breast cancer with excellent prognosis but with potential adverse impacts of diagnosis and treatment on quality of life and other patient-reported outcomes (PROs). We undertook a systematic review to synthesise current evidence about PROs following diagnosis and treatment for DCIS. We searched five electronic databases (from database inception to November 2015), cross-referenced and contacted experts to identify studies that reported PROs after DCIS treatment. Two reviewers independently applied inclusion and quality criteria, and extracted findings. Of 2130 papers screened, 23 were eligible, reporting 17 studies. Short- and long-term PRO evidence about differences between DCIS treatment options was lacking. Evidence pooled across treatments indicated core aspects of quality of life (physical, role, social, emotional function, pain, fatigue) and psychological distress (anxiety, depression) were impacted significantly initially, with most aspects returning to population norms by 6-12 months, and all by 2 years post-operatively. Fears of recurrence and dying from breast cancer were exaggerated, occurred early and persisted for many years. Sexuality and body image impacts were generally low and resolved within 1-3 months after surgery. A minority of women experienced considerable impact, including depression and sexual issues associated with body image problems. Well-powered PRO studies are required to track recovery trajectories and long-term impacts of the range of contemporary and emerging local and systemic treatments for DCIS. PRO data would enable care providers to prepare patients for short-term sequelae and enable patients who have treatment options to exercise preferences in choosing among them.

  8. Systematic Evaluation of the Patient-Reported Outcome (PRO) Content of Clinical Trial Protocols

    PubMed Central

    Kyte, Derek; Duffy, Helen; Fletcher, Benjamin; Gheorghe, Adrian; Mercieca-Bebber, Rebecca; King, Madeleine; Draper, Heather; Ives, Jonathan; Brundage, Michael; Blazeby, Jane; Calvert, Melanie

    2014-01-01

    Background Qualitative evidence suggests patient-reported outcome (PRO) information is frequently absent from clinical trial protocols, potentially leading to inconsistent PRO data collection and risking bias. Direct evidence regarding PRO trial protocol content is lacking. The aim of this study was to systematically evaluate the PRO-specific content of UK National Institute for Health Research (NIHR) Health Technology Assessment (HTA) programme trial protocols. Methods and Findings We conducted an electronic search of the NIHR HTA programme database (inception to August 2013) for protocols describing a randomised controlled trial including a primary/secondary PRO. Two investigators independently reviewed the content of each protocol, using a specially constructed PRO-specific protocol checklist, alongside the ‘Standard Protocol Items: Recommendations for Interventional Trials’ (SPIRIT) checklist. Disagreements were resolved through discussion with a third investigator. 75 trial protocols were included in the analysis. Protocols included a mean of 32/51 (63%) SPIRIT recommendations (range 16–41, SD 5.62) and 11/33 (33%) PRO-specific items (range 4–18, SD 3.56). Over half (61%) of the PRO items were incomplete. Protocols containing a primary PRO included slightly more PRO checklist items (mean 14/33 (43%)). PRO protocol content was not associated with general protocol completeness; thus, protocols judged as relatively ‘complete’ using SPIRIT were still likely to have omitted a large proportion of PRO checklist items. Conclusions The PRO components of HTA clinical trial protocols require improvement. Information on the PRO rationale/hypothesis, data collection methods, training and management was often absent. This low compliance is unsurprising; evidence shows existing PRO guidance for protocol developers remains difficult to access and lacks consistency. Study findings suggest there are a number of PRO protocol checklist items that are not fully

  9. Maintaining primacy of the patient perspective in the development of patient-centered patient reported outcomes

    PubMed Central

    Garver, Amanda; Ljungberg, Inger H.; Schladen, Manon M.; Groah, Suzanne L.

    2017-01-01

    The objectives of this study were to describe and demonstrate a new model of developing patient reported outcomes (PROs) that are patient-centered, and to test the hypothesis that following this model would result in a qualitatively different PRO than if the typical PRO development model were followed. The typical process of developing PROs begins with an initial list of signs or symptoms originating from clinicians or PRO developers; patient validation of this list ensures that the list (i.e., the new PRO) is interpretable by patients, but not that patient perspectives are central or even represented. The new model begins with elicitation from clinicians and patients independently and separately. These perspectives are formally analyzed qualitatively, and the results are iteratively integrated by researchers, supporting clinical relevance and patient centeredness. We describe the application of this new model to the development of a PRO for urinary signs and symptoms in individuals with neuropathic bladder, and test the hypothesis that the two processes generate qualitatively different instruments using a national validation sample of 300 respondents. Of its 29 items, the new instrument included 13 signs/symptoms derived from existing clinical practice guidelines, with 16 others derived from the patient/focus groups. The three most-endorsed items came from the patients, and the three least-endorsed items came from clinical guidelines. Thematic qualitative analysis of the elicitation process, as well as the results from our national sample, support the conclusion that the new model yields an instrument that is clinically interpretable, but more patient-centered, than the typical model would have done in this context. PMID:28257414

  10. Interpreting patient-reported outcomes from clinical trials in COPD: a discussion

    PubMed Central

    Jones, Paul W; Rennard, Stephen; Tabberer, Maggie; Riley, John H; Vahdati-Bolouri, Mitra; Barnes, Neil C

    2016-01-01

    One of the challenges faced by the practising physician is the interpretation of patient-reported outcomes (PROs) in clinical trials and the relevance of such data to their patients. This is especially true when caring for patients with progressive diseases such as COPD. In an attempt to incorporate the patient perspective, many clinical trials now include assessments of PROs. These are formalized methods of capturing patient-centered information. Given the importance of PROs in evaluating the potential utility of an intervention for a patient with COPD, it is important that physicians are able to critically interpret (and critique) the results derived from them. Therefore, in this paper, a series of questions is posed for the practising physician to consider when reviewing the treatment effectiveness as assessed by PROs. The focus is on the St George’s Respiratory Questionnaire for worked examples, but the principles apply equally to other symptom-based questionnaires. A number of different ways of presenting PRO data are discussed, including the concept of the minimum clinically important difference, whether there is a ceiling effect to PRO results, and the strengths and weaknesses of responder analyses. Using a worked example, the value of including a placebo arm in a study is illustrated, and the influence of the study on PRO results is considered, in terms of the design, patient withdrawal, and the selection of the study population. For the practising clinician, the most important consideration is the importance of individualization of treatment (and of treatment goals). To inform such treatment, clinicians need to critically review PRO data. The hope is that the questions posed here will help to build a framework for this critical review. PMID:27994447

  11. Predictive Validity and Responsiveness of Patient-Reported and Performance-Based Measures of Function in the Boston RISE Study

    PubMed Central

    Beauchamp, Marla K.; Jette, Alan M.; Ward, Rachel E.; Kurlinski, Laura A.; Kiely, Dan; Latham, Nancy K.; Bean, Jonathan F.

    2015-01-01

    Background. Patient-reported and performance-based measures (PBMs) are commonly used to measure physical function in studies of older adults. Selection of appropriate measures to address specific research questions is complex and requires knowledge of relevant psychometric properties. The aim of this study was to examine the predictive validity for adverse outcomes and responsiveness of a widely used patient-reported measure, the Late-Life Function and Disability Instrument (LLFDI), compared with PBMs. Methods. We analyzed 2 years of follow-up data from Boston RISE, a cohort study of 430 primary care patients aged ≥65 years. Logistic and linear regression models were used to examine predictive validity for adverse outcomes and effect size and minimal detectable change scores were computed to examine responsiveness. Performance-based functional measures included the Short Physical Performance Battery, 400-m walk, gait speed, and stair-climb power test. Results. The LLFDI and PBMs showed high predictive validity for poor self-rated health, hospitalizations, and disability. The LLFDI function scale was the only measure that predicted falls. Absolute effect size estimates ranged from 0.54 to 0.64 for the LLFDI and from 0.34 to 0.63 for the PBMs. From baseline to 2 years, the percentage of participants with a change ≥ minimal detectable change was greatest for the LLFDI scales (46–59%) followed by the Short Physical Performance Battery (44%), gait speed (35%), 400-m walk (17%), and stair-climb power test (9%). Conclusions. The patient-reported LLFDI showed comparable psychometric properties to PBMs. Our findings support the use of the LLFDI as a primary outcome in gerontological research. PMID:25512569

  12. A Novel Digital Patient-Reported Outcome Platform for Head and Neck Oncology Patients—A Pilot Study

    PubMed Central

    Peltola, Maria K.; Lehikoinen, Joel S.; Sippola, Lauri T.; Saarilahti, Kauko; Mäkitie, Antti A.

    2016-01-01

    INTRODUCTION The patient’s role in toxicity reporting is increasingly acknowledged. There is also a need for developing modern communication methods between the patient and the medical personnel. Furthermore, the increasing number of head and neck cancer (HNC) patients is reflected in the volume of treatment follow-up visits, which remains a challenge for the health care. Electronic patient-reported outcome (ePRO) measures may provide a cost-efficient way to organize follow-up for cancer patients. MATERIALS AND METHODS We tested a novel ePRO application called Kaiku®, which enables real-time, online collection of patient-reported outcomes, such as side effects caused by treatment and quality of life. We conducted a pilot study to assess the suitability of Kaiku® for HNC patients at the Department of Oncology, Helsinki University Hospital, Helsinki, Finland. Patients used Kaiku® during and one month after radiotherapy to report treatment-related side effects and quality of life. Two physicians and a nurse performed the practical electronic communication part of the study. RESULTS Five of the nine patients agreed to participate in the study: three of them had local early-stage larynx cancer (T2N0, T1aN0, and T2N0) and the remaining two patients had early-stage base of tongue cancer (T2N0 and T1N2b). The degree of side effects reported by the patients via Kaiku® ranged from mild to life threatening. The number of outcome data points on patients’ progress was significantly increased, which resulted in a better follow-up and improved communication between the patient and the care team. CONCLUSIONS Kaiku® seems to be a suitable tool to monitor side effects and quality of life during and after radiotherapy among HNC patients. Kaiku® and similar tools could be useful in organizing a cost-effective follow-up process for HNC patients. We recommend conducting a larger study to further assess the impact of an ePRO solution in routine clinical practice. ePRO solutions

  13. 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

  14. Patient-Reported Outcome Instruments for Physical Symptoms Among Patients Receiving Maintenance Dialysis: A Systematic Review

    PubMed Central

    Flythe, Jennifer E.; Powell, Jill D.; Poulton, Caroline J.; Westreich, Katherine D.; Handler, Lara; Reeve, Bryce B.; Carey, Timothy S.

    2015-01-01

    Background Patients with end-stage renal disease (ESRD) receiving dialysis have poor health-related quality of life (HRQoL). Physical symptoms are highly prevalent among dialysis-dependent patients and play important roles in HRQoL. A range of symptom assessment tools have been used in dialysis-dependent patients, but there has been no previous systematic assessment of the existing symptom measures’ content, validity, and reliability. Study Design systematic review of the literature Settings & Population ESRD patients on maintenance dialysis Selection Criteria for Studies instruments with ≥3 physical symptoms previously used in dialysis-dependent patients and evidence of validity or reliability testing Intervention patient-reported physical symptom assessment instrument Outcomes instrument symptom-related content, validity, and reliability Results From 3,148 screened abstracts, 89 full-text articles were eligible for review. After article exclusion and further article identification via reference reviews, 58 articles on 23 symptom assessment instruments with documented reliability or validity testing were identified. Of the assessment instruments, 43.5% were generic and 56.5% were ESRD-specific. Symptoms most frequently assessed were fatigue, shortness of breath, insomnia, nausea and vomiting, and appetite. The instruments varied widely in respondent time burden, recall period, and symptom attributes. Few instruments considered recall periods less than 2 weeks and few assessed a range of symptom attributes. Psychometric testing was completed for congruent validity (70%), known group validity (25%), responsiveness (30%), internal consistency (78%), and test-retest reliability (65%). Content validity was assessed in dialysis populations in 57% of the 23 instruments. Limitations Consideration of physical symptoms only and exclusion of single symptom-focused instruments Conclusions The number of available instruments focused exclusively on physical symptoms in

  15. 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

  16. Patient-Reported Outcomes as a Component of the Primary Endpoint in a Double-Blind, Placebo-Controlled Trial in Advanced Pancreatic Cancer

    PubMed Central

    Eckhardt, S. Gail; De Porre, Peter; Smith, David; Maurel, Joan; Steward, William P.; Bouche, Olivier; van de Velde, Helgi; Michiels, Bart; Bugat, Roland

    2010-01-01

    In this randomized, double-blind, placebo-controlled study comparing gemcitabine + tipifarnib (G + t) or gemcitabine + placebo (G + p) in patients with pancreatic cancer, the primary endpoint of time to deterioration (TTD) was based primarily on patient-reported outcomes. Deterioration was defined as death or worsening of disease-related symptoms, based on patient-reported outcomes of pain intensity and analgesic use in a daily diary, plus investigator-rated weekly performance status. Secondary endpoints included survival and safety. Two hundred and forty-four patients were treated for a total of 4780 weeks, during which the diary was completed daily. Overall, the completion of the diary was found to be feasible: patients completed approximately 95% of scheduled diary entries. Baseline characteristics were well balanced between the two treatment arms. The primary endpoint of TTD was not significantly different between the G + t arm (69 days) and the G + p arm (91 days, P = 0.40). Survival was not significantly different between the G + t arm (202 days) and the G + p arm (221 days, P = 0.66). The combination of G + t had an acceptable toxicity profile, with primarily neutropenia and thrombocytopenia. Methodologically, measurement of patient-reported outcomes is feasible and useful in assessing the effect of anti-cancer therapy in pancreatic cancer if comprehensive initial and ongoing training is provided to all people involved, including not only the patients but also the study personnel. PMID:18723314

  17. Neurocognitive and Patient-Reported Outcomes in Adult Survivors of Childhood Osteosarcoma

    PubMed Central

    Edelmann, Michelle N.; Daryani, Vinay M.; Bishop, Michael W.; Liu, Wei; Brinkman, Tara M.; Stewart, Clinton F.; Mulrooney, Daniel A.; Kimberg, Cara; Ness, Kirsten K.; Cheung, Yin Ting; Srivastava, Deo Kumar; Robison, Leslie L.; Hudson, Melissa M.; Krull, Kevin R.

    2015-01-01

    Importance This study provides the first objective data documenting neurocognitive impairment in long-term survivors of childhood osteosarcoma. Objective To examine neurocognitive, neurobehavioral, emotional and quality of life outcomes in long-term survivors of childhood osteosarcoma treated with intravenous high-dose methotrexate. Design Cross-sectional cohort study, with prospective treatment and chronic health predictors. Outcome data was collected from June 2008 to August 2014. Setting Academic research hospital. Participants Survivors of osteosarcoma (n=80; mean [SD] age = 38.9 [7.6] years; time since diagnosis = 24.7 [6.6] years; 42% female) recruited from the St. Jude Lifetime Cohort Study and compared to community controls (n=39; age = 39.03 [11.71] years; 56.4% female). Main Outcome Measures Objective tests of neurocognitive function. Subjective report of neurobehavioral symptoms, emotional distress and quality of life. Within survivors, outcomes were examined in relation to pharmacokinetic indices of methotrexate exposure and current chronic health conditions, which were assessed through medical examination and coded according to Common Terminology Criteria for Adverse Events v4.03. Results Compared to community controls, survivors demonstrated lower reading (p=0.012), attention (p=0.002), memory (p=0.019), processing speed (p<0.001) and executive function (p=0.006). Survivors also fell below national norms on these outcomes. Results of pharmacokinetic analysis showed high-dose methotrexate maximum plasma concentration, median clearance, and median/cumulative exposure were not associated with neurocognitive outcomes. Any grade 3 or 4 Common Terminology Criteria for Adverse Events cardiac, pulmonary or endocrine condition was associated with poorer memory (p=0.006) and slower processing speed (p=0.002). Survivor-reported poor general health was associated with decreased sustained attention (p=0.05) and processing speed (p=0.006). Conclusions Long

  18. Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier.

    PubMed

    Revicki, Dennis A; Gnanasakthy, Ari; Weinfurt, Kevin

    2007-05-01

    The Food and Drug Administration (FDA) and European Medicines Agency (EMEA) are willing to consider including information on patient reported outcomes (PROs) in product labeling and advertising. Pharmaceutical industry researchers must provide sufficient evidence supporting PRO benefit before an approval may be granted. This report describes the purpose and content of a PRO Evidence Dossier, which consists of important information supporting PRO claims. The dossier should be completed by pharmaceutical industry or other researchers to document the planning of the PRO assessment strategy, psychometric evidence, desired target labeling statements, and the clinical trial evidence of PRO benefits. The systematic reporting and documentation of information on the rationale for including PROs, rationale for the selection of specific PRO instruments, evidence on the psychometric qualities of the PRO measures, and guidelines for interpreting PRO findings will facilitate achieving a PRO labeling or promotional claim. Combining all the relevant information into a single document will facilitate the review and evaluation process for clinical and regulatory reviewers. The PRO Evidence Dossier may also be helpful to industry and academic researchers in identifying further information that will need to be developed to support the clinical development program and the PRO endpoints.

  19. Posterior approach compared to direct lateral approach resulted in better patient-reported outcome after hemiarthroplasty for femoral neck fracture

    PubMed Central

    Kristensen, Torbjørn B; Vinje, Tarjei; Havelin, Leif I; Engesæter, Lars B; Gjertsen, Jan-Erik

    2017-01-01

    Background and purpose — Hemiarthroplasty (HA) is the most common treatment for displaced femoral neck fractures in many countries. In Norway, there has been a tradition of using the direct lateral surgical approach, but worldwide a posterior approach is more often used. Based on data from the Norwegian Hip Fracture Register, we compared the results of HA operated through the posterior and direct lateral approaches regarding patient-reported outcome measures (PROMs) and reoperation rate. Patients and methods — HAs due to femoral neck fracture in patients aged 60 years and older were included from the Norwegian Hip Fracture Register (2005–2014). 18,918 procedures were reported with direct lateral approach and 1,990 with posterior approach. PROM data (satisfaction, pain, quality of life (EQ-5D), and walking ability) were reported 4, 12, and 36 months postoperatively. The Cox regression model was used to calculate relative risk (RR) of reoperation. Results — There were statistically significant differences in PROM data with less pain, better satisfaction, and better quality of life after surgery using the posterior approach than using the direct lateral approach. The risk of reoperation was similar between the approaches. Interpretation — Hemiarthroplasty for hip fracture performed through a posterior approach rather than a direct lateral approach results in less pain, with better patient satisfaction and better quality of life. The risk of reoperation was similar with both approaches. PMID:27805460

  20. What are the appropriate methods for analyzing patient-reported outcomes in randomized trials when data are missing?

    PubMed

    Hamel, J F; Sebille, V; Le Neel, T; Kubis, G; Boyer, F C; Hardouin, J B

    2015-11-06

    Subjective health measurements using Patient Reported Outcomes (PRO) are increasingly used in randomized trials, particularly for patient groups comparisons. Two main types of analytical strategies can be used for such data: Classical Test Theory (CTT) and Item Response Theory models (IRT). These two strategies display very similar characteristics when data are complete, but in the common case when data are missing, whether IRT or CTT would be the most appropriate remains unknown and was investigated using simulations. We simulated PRO data such as quality of life data. Missing responses to items were simulated as being completely random, depending on an observable covariate or on an unobserved latent trait. The considered CTT-based methods allowed comparing scores using complete-case analysis, personal mean imputations or multiple-imputations based on a two-way procedure. The IRT-based method was the Wald test on a Rasch model including a group covariate. The IRT-based method and the multiple-imputations-based method for CTT displayed the highest observed power and were the only unbiased method whatever the kind of missing data. Online software and Stata® modules compatibles with the innate mi impute suite are provided for performing such analyses. Traditional procedures (listwise deletion and personal mean imputations) should be avoided, due to inevitable problems of biases and lack of power.

  1. 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.

  2. 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

  3. [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).

  4. Antimuscarinic Agent Treatment Affecting Patient-Reported Outcomes in Overactive Bladder Syndrome With Depressive Symptoms

    PubMed Central

    2016-01-01

    Purpose We investigated improvements in overactive bladder symptoms and depressive symptoms after solifenacin treatment in overactive bladder patients with or without depressive symptoms. Methods We performed a prospective study of patients who had been diagnosed with overactive bladder from July 2013 to June 2014. Based on the Beck Depression Inventory questionnaire, the test subjects were divided into group 1, without depressive symptoms (0–9 points), and group 2, with depressive symptoms (10 or more points). The patients were administered 5 mg of solifenacin for 3 months. The following outcomes were analyzed at the first visit, 4 weeks, and 12 weeks: the overactive bladder symptom score (OABSS), International Prostate Symptom Score (IPSS), patients’ perceptions of their bladder condition, and the Beck Depression Inventory. Results A total of 72 patients participated, and 52 patients completed the study. Most outcome measures showed improvements in both groups at weeks 4 and 12. Especially in group 2, the questionnaires showed significant improvements from baseline to week 12, indicating that solifenacin was effective at treating overactive bladder symptoms (group 1 vs. group 2: OABSS, −2.67±0.80 vs. −3.00±0.77; P<0.01; IPSS-total, −2.14±2.15 vs. −4.94±1.70; P<0.01). Statistically significant decreases in the Beck Depression Inventory score from baseline to weeks 4 and 12 were observed in group 2 (group 1 vs. group 2: 1.43±0.74 vs. −2.68±4.05 at week 4, P<0.001; 0.10±3.37 vs. −5.52±5.82 at week 12, P<0.001). Conclusions In overactive bladder patients with depressive symptoms, solifenacin can help improve quality of life and depressive symptoms at the same time. PMID:28043114

  5. The impact of the new antiviral regimens on patient reported outcomes and health economics of patients with chronic hepatitis C.

    PubMed

    Younossi, Zobair; Henry, Linda

    2014-12-15

    Hepatitis C is an important cause of chronic liver disease worldwide with an estimated 170 million people infected. Hepatitis C virus (HCV)-infected patients are physically and mentally impacted by fatigue, depression and anxiety causing an impairment of health related quality of life (HRQOL), lower worker productivity and other patient reported outcomes (PROs). Although anti-HCV regimens containing first generation direct acting antiviral agents (DAAs) were associated with significant side effects, the second generation DAAs, sofosbuvir (SOF) and simeprevir (SMV), are associated with fewer side effects, better tolerability and high cure rates. Despite these advantages, key stakeholders are currently trying to find ways to best integrate these new therapeutic regimens into the management of patients with chronic hepatitis C for the benefit of all. The purpose of this article is to offer insight into the other key and equally important outcomes (PRO's, HRQOL and cost) which should be considered when assessing the applicability of these new regimens for the care of patients infected with HCV. Our review provides evidence that the new treatment regimens for HCV not only have high efficacy rates but are also associated with better patient reported outcomes and cost per case of HCV cured. Additionally, compared to other medical interventions, these new regimens are cost-effective from a societal perspective.

  6. Retrospective study of the effect of disease progression on patient reported outcomes in HER-2 negative metastatic breast cancer patients

    PubMed Central

    2011-01-01

    Background This retrospective study evaluated the impact of disease progression and of specific sites of metastasis on patient reported outcomes (PROs) that assess symptom burden and health related quality of life (HRQoL) in women with metastatic breast cancer (mBC). Methods HER-2 negative mBC patients (n = 102) were enrolled from 7 U.S. community oncology practices. Demographic, disease and treatment characteristics were abstracted from electronic medical records and linked to archived Patient Care Monitor (PCM) assessments. The PCM is a self-report measure of symptom burden and HRQoL administered as part of routine care in participating practices. Linear mixed models were used to examine change in PCM scores over time. Results Mean age was 57 years, with 72% of patients Caucasian, and 25% African American. Median time from mBC diagnosis to first disease progression was 8.8 months. Metastasis to bone (60%), lung (28%) and liver (26%) predominated at initial metastatic diagnosis. Results showed that PCM items assessing fatigue, physical pain and trouble sleeping were sensitive to either general effects of disease progression or to effects associated with specific sites of metastasis. Progression of disease was also associated with modest but significant worsening of General Physical Symptoms, Treatment Side Effects, Acute Distress and Impaired Performance index scores. In addition, there were marked detrimental effects of liver metastasis on Treatment Side Effects, and of brain metastasis on Acute Distress. Conclusions Disease progression has a detrimental impact on cancer-related symptoms. Delaying disease progression may have a positive impact on patients' HRQoL. PMID:21689425

  7. Patient reported allergies are a risk factor for poor outcomes in total hip and knee arthroplasty.

    PubMed

    Graves, Christopher M; Otero, Jesse E; Gao, Yubo; Goetz, Devon D; Willenborg, Melissa D; Callaghan, John J

    2014-09-01

    We evaluated 459 patients undergoing THA or TKA who completed preoperative and postoperative WOMAC and/or SF36 surveys. Medical comorbidities and reported allergies were also recorded. Evaluation of surveys was compared for patients with or without 4 or more reported allergies using statistical methods. Patients with 4 or more reported allergies had less improvement on SF36 Physical Component Score (∆PCS=4.2) than those with 0-3 allergies (∆PCS=10.0, P=0.0002). Regression analysis showed that this change was independent of self-reported comorbidities. Patients reporting 4 or more allergies also had less improvement in WOMAC function (∆F=21.4) than those with 0-3 allergies (∆F = 27.2, P=0.036). Similar nonsignificant trends occurred in SF36 mental and WOMAC pain and stiffness scores.

  8. Long-term result and patient reported outcome of wrist splint treatment for carpal tunnel syndrome.

    PubMed

    Povlsen, Bo; Bashir, Muhammad; Wong, Fabian

    2014-06-01

    Carpal tunnel syndrome (CTS) is the commonest peripheral neuropathy presenting to specialist hand and wrist clinics. This study investigated the long-term outcome of carpal tunnel syndrome treated with isolated night wrist splint and the factors determining the likelihood of success of this intervention. Seventy-five patients referred to a specialist hand clinic with CTS were given night wrist splint treatment for 3 months as per a previous study protocol. Fifty-two patients from this cohort did not wish to have surgery after wrist splint treatment and were followed for a further 33-month period. Baseline pain and numbness levels were recorded on a Visual Analogue Scale (VAS) using a questionnaire upon first presentation. A further questionnaire at 36 months reassessed pain and numbness levels, patients' satisfaction with the treatment, and whether they had subsequent surgical decompression. Of the patients who completed the follow-up questionnaire 33 months after their period of conservative management, 43% were successfully treated with splint treatment alone. There was no difference in the VAS for pain or numbness at the baseline and at 36 months between successful and failed treatment groups. Patients successfully treated with wrist splinting alone reported a higher level of satisfaction with their treatment compared to patients who failed wrist splint treatment or had surgical decompression. The results reinforce the previous recommendation on wrist splinting as a first-line treatment in the Primary Care setting. Referral to specialist hand and wrist clinics should be reserved for patients with symptoms refractory to this initial measure.

  9. A CASE CONTROL STUDY OF THE EFFECTIVENESS OF TISSUE PLASMINOGEN ACTIVATOR ON 6 MONTH PATIENT-REPORTED OUTCOMES AND HEALTHCARE UTILIZATION

    PubMed Central

    Lang, Catherine E.; Bland, Marghuretta D.; Cheng, Nuo; Corbetta, Maurizio; Lee, Jin-Moo

    2014-01-01

    We examined the benefit of tissue plasminogen activator (tPA), delivered as part of usual stroke management, on patient-reported outcomes and healthcare utilization. Using a case control design, patients who received tPA as part of usual stroke management were compared with patients who would have received tPA had they arrived to the hospital within the therapeutic time window. Data were collected from surveys 6 months post stroke using standardized patient-reported outcome measures and questions about healthcare utilization. Demographic and medical data were acquired from hospital records. Patients were matched on stroke severity, age, race, and gender. Matching was done with 1:2 ratio of tPA to controls. Results were compared between groups with 1-tailed tests due to directionally-specific hypothesis in favor of the tPA group. The tPA (n = 78) and control (n = 156) groups were matched across the variables, except for stroke severity, which was better in the control group; subsequent analyses controlled for this mismatch. The tPA group reported better physical function, communication, cognitive ability, depressive symptomatology, and quality of life/participation compared to the control group. Fewer people in the tPA group reported skilled nursing facility stays, emergency department visits, and re-hospitalizations after their stroke compared to controls. Reports of other post-acute services were not different between groups. While it is known that tPA reduces disability, this is the first study to demonstrate the effectiveness of tPA in improving meaningful, patient-reported outcomes. Thus, use of tPA provides a large benefit to the daily lives of people with ischemic stroke. PMID:25440368

  10. 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

  11. Treatment and Patient Reported Outcome in Children with Hirschsprung Disease and Concomitant Congenital Heart Disease

    PubMed Central

    Hasserius, Johan; Hedbys, Josefine

    2017-01-01

    Purpose. Congenital heart disease (CHD) is reported to be associated with Hirschsprung disease (HD). The aim was to evaluate any differences between children with HD with and without CHD, respectively, with regard to patient characteristics, medical care, and patient reported bowel function. Method. This is a retrospective chart study and a cross-sectional long-term follow-up of patients older than 4 years old, including all children with HD operated on with transanal endorectal pull-through (TERPT) at a tertiary center of pediatric surgery. Information about patient characteristics, diagnostics, surgery, and medical care was compiled. At long-term follow-up, bowel function was assessed by Bowel Function Score. Results. Included were 53 HD-patients, 13 with CHD and 40 without CHD. Children with CHD more commonly presented with failure to thrive; 4 (23%) compared to those without CHD (0%) (p < 0.01). In the long-term follow-up, including 32 patients (6 with CHD), constipation was more commonly reported by children with CHD 5 (83%) than by children without CHD 4 (27%) (p = 0.01). No differences were shown in the other parameters such as fecal control and incontinence. Conclusion. HD-patients with CHD more commonly presented with failure to thrive and more frequently reported constipation than HD-patients without CHD. The findings indicate that HD-patients with CHD might need special consideration in their initial care and long-term follow-up. PMID:28373976

  12. Recommended patient-reported core set of symptoms to measure in prostate cancer treatment trials.

    PubMed

    Chen, Ronald C; Chang, Peter; Vetter, Richard J; Lukka, Himansu; Stokes, William A; Sanda, Martin G; Watkins-Bruner, Deborah; Reeve, Bryce B; Sandler, Howard M

    2014-07-01

    The National Cancer Institute (NCI) Symptom Management and Health-Related Quality of Life Steering Committee convened four working groups to recommend core sets of patient-reported outcomes to be routinely incorporated in clinical trials. The Prostate Cancer Working Group included physicians, researchers, and a patient advocate. The group's process included 1) a systematic literature review to determine the prevalence and severity of symptoms, 2) a multistakeholder meeting sponsored by the NCI to review the evidence and build consensus, and 3) a postmeeting expert panel synthesis of findings to finalize recommendations. Five domains were recommended for localized prostate cancer: urinary incontinence, urinary obstruction and irritation, bowel-related symptoms, sexual dysfunction, and hormonal symptoms. Four domains were recommended for advanced prostate cancer: pain, fatigue, mental well-being, and physical well-being. Additional domains for consideration include decisional regret, satisfaction with care, and anxiety related to prostate cancer. These recommendations have been endorsed by the NCI for implementation.

  13. Provider perceptions of the value of same-day, electronic patient-reported measures for use in clinical HIV care.

    PubMed

    Fredericksen, R J; Tufano, J; Ralston, J; McReynolds, J; Stewart, M; Lober, W B; Mayer, K H; Mathews, W C; Mugavero, M J; Crane, P K; Crane, H M

    2016-11-01

    Strong evidence suggests that patient-reported outcomes (PROs) aid in managing chronic conditions, reduce omissions in care, and improve patient-provider communication. However, provider acceptability of PROs and their use in clinical HIV care is not well known. We interviewed providers (n = 27) from four geographically diverse HIV and community care clinics in the US that have integrated PROs into routine HIV care, querying perceived value, challenges, and use of PRO data. Perceived benefits included the ability of PROs to identify less-observable behaviors and conditions, particularly suicidal ideation, depression, and substance use; usefulness in agenda setting prior to a visit; and reduction of social desirability bias in patient-provider communication. Challenges included initial flow integration issues and ease of interpretation of PRO feedback. Providers value same-day, electronic patient-reported measures for use in clinical HIV care with the condition that PROs are (1) tailored to be the most clinically relevant to their population; (2) well integrated into clinic flow; and (3) easy to interpret, highlighting chief patient concerns and changes over time.

  14. How to Measure Outcomes of Peripheral Nerve Surgery

    PubMed Central

    Wang, Yirong; Sunitha, Malay; Chung, Kevin C.

    2013-01-01

    Synopsis Evaluation of outcomes after peripheral nerve surgeries include a number of assessment methods that reflect different aspects of recovery, including reinnervation, tactile gnosis, integrated sensory and motor function, pain and discomfort, neurophysiological and patient- reported outcomes. This review makes a list of measurements addressing these aspects as well as advantage and disadvantage of each tool. Because of complexities of neurophysiology, assessment remains a difficult process, which requires researchers focus on measurements best relevant to specific conditions and research questions. PMID:23895715

  15. 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.

  16. Patient-Reported Outcomes Following Living Kidney Donation: A Single Center Experience.

    PubMed

    Rodrigue, James R; Vishnevsky, Tanya; Fleishman, Aaron; Brann, Tracy; Evenson, Amy R; Pavlakis, Martha; Mandelbrot, Didier A

    2015-09-01

    This article describes the development and implementation of an initiative at one transplant center to annually assess psychosocial outcomes of living kidney donors. The current analysis focuses on a cohort of adults (n = 208) who donated a kidney at BIDMC between September 2005 and August 2012, in which two post-donation annual assessments could be examined. One and two year post-donation surveys were returned by 59 % (n = 123) and 47 % (n = 98) of LKDs, respectively. Those who did not complete any survey were more likely to be younger (p = 0.001), minority race/ethnicity (p < 0.001), and uninsured at the time of donation (p = 0.01) compared to those who returned at least one of the two annual surveys. The majority of donors reported no adverse physical or psychosocial consequences of donation, high satisfaction with the donation experience, and no donation decision regret. However, a sizable minority of donors felt more pain intensity than expected and recovery time was much slower than expected, and experienced a clinically significant decline in vitality. We describe how these outcomes are used to inform clinical practice at our transplant center as well as highlight challenges in donor surveillance over time.

  17. 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.

  18. 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

  19. How bad is bile acid diarrhoea: an online survey of patient-reported symptoms and outcomes

    PubMed Central

    Bannaga, Ayman; Kelman, Lawrence; O'Connor, Michelle; Pitchford, Claire; Walters, Julian R F; Arasaradnam, Ramesh P

    2017-01-01

    Objectives Bile acid diarrhoea (BAD) is an underdiagnosed condition producing diarrhoea, urgency and fear of faecal incontinence. How patients experience these symptoms has not previously been studied. Bile Acid Malabsorption (BAM) Support UK was established in 2015 as a national charity with objectives including to provide details regarding how BAD affects patients, to improve earlier recognition and clinical management. Design, setting and main outcome A questionnaire was collected anonymously by BAM Support UK and the Bile Salt Malabsorption Facebook group over 4 weeks at the end of 2015. It comprised 56 questions and aimed to inform patients and clinicians about how BAD affects the respondents. Results The first 100 responses were analysed. 91% of the respondents reported a diagnosis of BAD. 58% of total respondents diagnosed following a Selenium-homocholic acid taurine scan, 69% were diagnosed by a gastroenterologist, with type 2 and 3 BAD comprising 38% and 37%, respectively, of total respondents. Symptoms had been experienced for more than 5 years before diagnosis in 44% of respondents. Following treatment, usually with bile acid sequestrants, 60% of participants reported improvement of diarrhoea and most reported their mental health has been positively impacted. Just over half of the cohort felt as though their symptoms had been dismissed during clinical consultations and 28% felt their GPs were unaware of BAD. Conclusions BAD requires more recognition by clinicians to address the current delays in diagnosis. Treatment improves physical and mental symptoms in the majority of participants. PMID:28123771

  20. All Seasons Vertical Augmentation Mastopexy: A Simple Algorithm, Clinical Experience, and Patient-reported Outcomes

    PubMed Central

    2016-01-01

    Background: The safety of augmentation mastopexy has been questioned. Staging has been recommended for women deemed to be at higher risk, such as women with greater degrees of ptosis. Most existing studies evaluate women treated with multiple methods, including the traditional Wise pattern. This retrospective study specifically evaluates vertical augmentation mastopexy. A simple algorithm is introduced. Methods: From 2002 to 2016, 252 women underwent consecutive vertical augmentation mastopexies performed by the author, with no staged surgery. All patients underwent a vertical mastopexy using a medially based pedicle and intraoperative nipple siting. A subset of women treated from 2012 to 2016 were surveyed to obtain outcome data; 90 patients (inclusion rate, 90%) participated. Results: The complication rate was 32.9%, including persistent ptosis, delayed wound healing, scar deformities, and asymmetry. There were no cases of nipple loss. An increased risk of complications was detected for smokers (P < 0.01), but not for combined procedures, secondary breast augmentations, or secondary mastopexies. The revision rate was 15.5%. Persistent nipple numbness was reported by 13.3% of respondents. Eighty percent of women were self-conscious about their breast appearance before surgery; 22% of respondents were self-conscious about their breasts after surgery. Seventy percent of respondents reported an improved quality of life, 94.4% would repeat the surgery, and 95.6% would recommend it. Conclusions: A simple algorithm may be used to guide treatment in women who desire correction of ptosis and upper pole fullness. An "all seasons" vertical augmentation mastopexy is safe and widely applicable. Staging is unnecessary. PMID:28293517

  1. 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

  2. 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.

  3. Yoga for Risk Reduction of Metabolic Syndrome: Patient-Reported Outcomes from a Randomized Controlled Pilot Study

    PubMed Central

    Sohl, Stephanie J.; Wallston, Kenneth A.; Watkins, Keiana

    2016-01-01

    Lifestyle change is recommended as treatment for adults at risk for metabolic syndrome (MetS), although adoption of new behavioral patterns is limited. In addition, most existing lifestyle interventions do not address psychological stress or quality of life, both of which impact the burden of MetS. Yoga, a form of physical activity that incorporates psychological components (e.g., maintaining attention, relaxation), is a promising intervention for improving the burden of MetS. This randomized controlled trial assessed the feasibility and preliminary efficacy of a 12-week yoga program coupled with an evidence-based health education program (HED) compared to HED alone. A secondary, exploratory aim examined perceived stress, quality of life, and related psychological outcomes (mindfulness, perceived health competence, and mood). Sixty-seven adults at risk for MetS enrolled (mean age [SD]: 58 [10] years; 50% male; 79% non-Hispanic White). Preliminary results revealed significantly larger improvements in two quality of life domains (role-physical and general health perceptions) in the HED plus yoga group versus HED alone (ps < 0.05). This is the first study that implemented lifestyle education along with yoga to evaluate the potential unique effects of yoga on participants at risk for MetS. A larger clinical trial is warranted to further investigate these promising patient-reported outcomes. PMID:27847524

  4. Favorable patient reported outcomes following IMRT for early carcinomas of the tonsillar fossa: Results from a symptom assessment study

    PubMed Central

    Gunn, GB; Hansen, CC; Garden, AS; Fuller, CD; Mohamed, ASRM; Morrison, WH; Frank, SJ; Beadle, BM; Phan, J; Chronowski, GM; Sturgis, EM; Lewis, CM; Lu, C; Hutcheson, KA; Mendoza, TR; Cleeland, CS; Rosenthal, DI

    2016-01-01

    Background A questionnaire-based study was conducted to assess long-term patient reported outcomes (PROs) following definitive IMRT-based treatment for early stage carcinomas of the tonsillar fossa. Methods: Participants had received IMRT with or without systemic therapy for squamous carcinoma of the tonsillar fossa (T1-2 and N0-2b) with a minimum follow-up of 2 years. Patients completed a validated head and neck cancer-specific PRO instrument, the MD Anderson Symptom Inventory-Head and Neck module (MDASI-HN). Symptoms were compared between treatment groups of interest and overall symptom burden was evaluated. Results Of 139 participants analyzed, 51% had received ipsilateral neck IMRT, and 62% single modality IMRT alone (no systemic therapy). There were no differences in mean individual symptom and interference ratings for those treated with bilateral versus ipsilateral neck IMRT alone. However, 40% of those treated with bilateral versus 25% of those treated with ipsilateral neck RT alone reported moderate-to-severe levels of dry mouth (p=0.03). Fatigue, numbness/tingling, and constipation were rated more severe for those who had received systemic therapy (p<0.05 for each), but absolute differences were small. Overall, 51% had no more than mild symptom ratings across all 22 symptoms assessed. Conclusions The long-term patient reported symptom profile in this cohort of tonsil cancer survivors treated with definitive IMRT-based treatment showed a majority of patients with no more than mild symptoms, low symptom interference, and provides an opportunity for future comparison studies with other treatment approaches. PMID:26403258

  5. Is the toxicity of adjuvant aromatase inhibitor therapy underestimated? Complementary information from patient-reported outcomes (PROs).

    PubMed

    Oberguggenberger, Anne; Hubalek, Michael; Sztankay, Monika; Meraner, Verena; Beer, Beate; Oberacher, Herbert; Giesinger, Johannes; Kemmler, Georg; Egle, Daniel; Gamper, Eva-Maria; Sperner-Unterweger, Barbara; Holzner, Bernhard

    2011-07-01

    Adjuvant endocrine treatment-related adverse effects have a strong impact on patients' quality of life and thereby limit therapy's risk benefit ratio resulting in morbidity and treatment discontinuation. Still, many AI adverse effects remain untreated given that they are unrecognized by conservative methods (e.g., proxy ratings). The ability of complementary patient-reported outcomes (PROs) to provide a more comprehensive assessment of side-effects is to be explored. A cross-sectional study sample of 280 postmenopausal, early stage breast cancer patients was subjected to a comprehensive PRO assessment (FACT-B/+ES) at their after-care appointment. Prevalence and severity of patient-reported physical side-effects and psychosocial burden related to adjuvant AI therapy were compared with prevalences derived from pivotal phase IV trials (ATAC 2005, BIG1-98 2005). Across all symptom categories, highest prevalence rates were found for joint pain (59.6%), hot flushes (52%), lost interest in sexual intercourse (51.4%), and lack of energy (40.3%). Overall, PROs resulted in significantly higher prevalence rates as compared to physician ratings for all symptoms published in pivotal clinical trials except vaginal bleeding and nausea. The treatment duration exerted no significant impact on symptom frequency (P > 0.05). Established prevalence rates of endocrine treatment-related toxicity seem to be underestimated. The incorporation of PRO data should be mandatory or at least highly recommended in clinical treatment planning to arrive at a more accurate assessment of a patient's actual symptom burden enabling improved individualized management of side-effects and mediating the preservation of treatment adherence.

  6. Patient-Reported Outcomes After 3-Dimensional Conformal, Intensity-Modulated, or Proton Beam Radiotherapy for Localized Prostate Cancer

    PubMed Central

    Gray, Phillip J.; Paly, Jonathan J.; Yeap, Beow Y.; Sanda, Martin G.; Sandler, Howard. M.; Michalski, Jeff M.; Talcott, James A.; Coen, John J.; Hamstra, Daniel A.; Shipley, William U.; Hahn, Stephen M.; Zietman, Anthony L.; Bekelman, Justin E.; Efstathiou, Jason A.

    2013-01-01

    BACKGROUND Recent studies have suggested differing toxicity patterns for patients with prostate cancer who receive treatment with 3-dimensional conformal radiotherapy (3DCRT), intensity-modulated radiotherapy (IMRT), or proton beam therapy (PBT). METHODS The authors reviewed patient-reported outcomes data collected prospectively using validated instruments that assessed bowel and urinary quality of life (QOL) for patients with localized prostate cancer who received 3DCRT (n = 123), IMRT (n = 153) or PBT (n = 95). Clinically meaningful differences in mean QOL scores were defined as those exceeding half the standard deviation of the baseline mean value. Changes from baseline were compared within groups at the first post-treatment follow-up (2–3 months from the start of treatment) and at 12 months and 24 months. RESULTS At the first post-treatment follow-up, patients who received 3DCRT and IMRT, but not those who received PBT, reported a clinically meaningful decrement in bowel QOL. At 12 months and 24 months, all 3 cohorts reported clinically meaningful decrements in bowel QOL. Patients who received IMRT reported clinically meaningful decrements in the domains of urinary irritation/obstruction and incontinence at the first post-treatment follow-up. At 12 months, patients who received PBT, but not those who received IMRT or 3DCRT, reported a clinically meaningful decrement in the urinary irritation/ obstruction domain. At 24 months, none of the 3 cohorts reported clinically meaningful changes in urinary QOL. CONCLUSIONS Patients who received 3DCRT, IMRT, or PBT reported distinct patterns of treatment-related QOL. Although the timing of toxicity varied between the cohorts, patients reported similar modest QOL decrements in the bowel domain and minimal QOL decrements in the urinary domains at 24 months. Prospective randomized trials are needed to further examine these differences. PMID:23436283

  7. Obesity effect on a multimodal physiotherapy program for low back pain suffers: patient reported outcome

    PubMed Central

    2013-01-01

    Background Several studies have linked obesity to the increased likelihood of lower back pain, but there are no studies focussing on the effectiveness of a multimodal physiotherapy programme (MPP) in obese subjects who suffer from chronic non-specific lower back pain (CNLBP). The aim of this study was to compare the effectiveness of an MPP in obese (G1) (body mass index (BMI):≥30) and non-obese (G2) (BMI:<30) patients with CNLBP. Methods A quasi-experimental study with pre- and post-intervention evaluations of an MPP (lasting 8 weeks) was conducted on obese and non-obese patients with CNLBP. A total of 53 people were included in the study: G1, composed of 19 patients (10 men and 9 women) with a BMI of 33.75 and a mean age of 52.94 years, and G2, composed of 34 patients (18 men and 16 women) with a mean age of 49.19 years and an average BMI of 25.56. All patients were measured to calculate pre-intervention (baseline) and post-intervention (8 weeks) changes in disability (RMQ) and health related quality of life in physical and mental health component state of SF12 and quality of life (EQ-5D and EQ-VAS). Results Post-intervention, non-obese group shown significant high improve than obese group in disability (RMQ: 4.00), physical component state of SF-12: (-7.26) and quality of life (EQ-VAS.: -10.49). Conclusions In patients with CNLBP, a BMI more than or equal to 30 minimises the effects of an MPP lasting 8 weeks. PMID:23663679

  8. Clinical outcome measures in juvenile idiopathic arthritis.

    PubMed

    Consolaro, Alessandro; Giancane, Gabriella; Schiappapietra, Benedetta; Davì, Sergio; Calandra, Serena; Lanni, Stefano; Ravelli, Angelo

    2016-04-18

    Juvenile idiopathic arthritis (JIA), as a chronic condition, is associated with significant disease- and treatment-related morbidity, thus impacting children's quality of life. In order to optimize JIA management, the paediatric rheumatologist has begun to regularly use measurements of disease activity developed, validated and endorsed by international paediatric rheumatology professional societies in an effort to monitor the disease course over time and assess the efficacy of therapeutic interventions in JIA patients.A literature review was performed to describe the main outcome measures currently used in JIA patients to determine disease activity status.The Juvenile Disease Activity Score (JADAS), in its different versions (classic JADAS, JADAS-CRP and cJADAS) and the validated definitions of disease activity and response to treatment represent an important tool for the assessment of clinically relevant changes in disease activity, leading more and more to a treat-to-target strategy, based on a tight and thorough control of the patient condition. Moreover, in recent years, increasing attention on the incorporation of patient-reported or parent-reported outcomes (PRCOs), when measuring the health state of patients with paediatric rheumatic diseases has emerged.We think that the care of JIA patients cannot be possible without taking into account clinical outcome measures and, in this regard, further work is required.

  9. Hablamos juntos (together we speak): a brief patient-reported measure of the quality of interpretation

    PubMed Central

    Talamantes, Efrain; Moreno, Gerardo; Guerrero, Lourdes R; Mangione, Carol M; Morales, Leo S

    2014-01-01

    Purpose This study evaluates the psychometric properties of three newly developed items assessing the quality of interpretation from the patient’s perspective among Spanish-speaking limited English proficient Latino patients. Patients and methods The authors examined the psychometric properties of a patient-reported measure of quality of interpretation using a cross-sectional survey study of 1,590 adult Spanish-speaking limited English proficient Latinos in the United States. Quality of interpretation, doctor communication, and satisfaction with care were assessed using a three survey-item, an independent multiple-item measure, and a single-item measure, respectively. Results Sixty-nine percent (1,104) of patients surveyed used interpreters. Cronbach’s alpha for the three items assessing interpreter quality was 0.31, while dropping item three resulted in an alpha of 0.56. Items one and two were moderately correlated with doctor communication (r=0.39) and satisfaction with care scores (r=0.21) supporting construct validity. Conclusion Two out of three survey items can be scaled to measure quality of interpretation from the patient’s perspective. Quality of interpretation reported by patients is moderately associated with doctor communication and satisfaction with care. PMID:25228825

  10. Measuring Wheelchair Intervention Outcomes: Development of the Wheelchair Outcome Measure

    PubMed Central

    Mortenson, William B; Miller, William C; Miller-Pogar, Jan

    2013-01-01

    Purpose Provision of a wheelchair has immediate intuitive benefits; however, it can be difficult to evaluate which wheelchair and seating components best meet an individual’s needs. As well, funding agencies now prefer evidence of outcomes; and therefore measurement upon prescription of a wheelchair or its components is essential to demonstrate the efficacy of intervention. As no existing tool can provide individualized goal-oriented measure of outcome after wheelchair prescription, a research project was undertaken to create the Wheelchair Outcome Measure (WhOM). Method A mixed method research design was employed to develop the instrument, which used in-depth interviews of prescribers, individuals who use wheelchairs and their associates, supplemented by additional questions in which participant preferences in key areas of the measure were quantified. Results The WhOM is a client-specific wheelchair intervention measurement tool that is based on the World Health Organization’s International Classification of Function, Disability, and Health. It identifies desired outcomes at a participation level and also acknowledges concerns about body structure and function. Conclusion The new outcome instrument will allow clients to identify and evaluate the outcomes they wish to achieve with their wheelchairs and seating and provide clinicians a way to quantify outcomes of their interventions in a way that is meaningful to the client and potential funding sources. PMID:19263533

  11. Tofacitinib versus methotrexate in rheumatoid arthritis: patient-reported outcomes from the randomised phase III ORAL Start trial

    PubMed Central

    Strand, Vibeke; Lee, Eun Bong; Fleischmann, Roy; Koncz, Tamas; Zwillich, Samuel H; Gruben, David; Wilkinson, Bethanie; Krishnaswami, Sriram; Wallenstein, Gene

    2016-01-01

    Objectives To compare patient-reported outcomes (PROs) in methotrexate (MTX)-naive patients (defined as no prior treatment or ≤3 doses) receiving tofacitinib versus MTX. Methods In the 24-month, phase III, randomised, controlled, ORAL Start trial (NCT01039688), patients were randomised 2:2:1 to receive tofacitinib 5 mg two times per day (n=373), tofacitinib 10 mg two times per day (n=397) or MTX (n=186). PROs assessed included Patient Global Assessment of disease (PtGA), pain, Health Assessment Questionnaire-Disability Index (HAQ-DI), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and health-related quality of life (Short Form-36 [SF-36]). Results PROs improved following tofacitinib and MTX treatment: benefits were sustained over 24 months. Patients receiving tofacitinib reported earlier responses which were significantly different between each tofacitinib dose and MTX at month 3 through month 24. At month 6 (primary end point), significant improvements versus MTX were observed in PtGA, pain, HAQ-DI, SF-36 Physical Component Summary (PCS), 5/8 domain scores and FACIT-F with tofacitinib 5 mg two times per day; all PROs, except SF-36 Mental Component Summary Score and Medical Outcomes Survey-Sleep, with tofacitinib 10 mg two times per day. At month 6, the proportion of patients reporting improvements ≥minimum clinically important difference were significant versus MTX with tofacitinib 5 mg two times per day in PtGA and 3/8 SF-36 domains; and with tofacitinib 10 mg two times per day in PtGA, pain, HAQ-DI, SF-36 PCS, 4/8 domains and FACIT-F. Conclusions Patients with rheumatoid arthritis receiving tofacitinib 5 and 10 mg two times per day monotherapy versus MTX reported statistically significant and clinically meaningful improvements in multiple PROs over 24 months; onset of benefit with tofacitinib treatment occurred earlier. Trial registration number NCT01039688. PMID:27752357

  12. 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…

  13. 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.

  14. Higher Prescription Opioid Dose is Associated With Worse Patient-Reported Pain Outcomes and More Health Care Utilization.

    PubMed

    Morasco, Benjamin J; Yarborough, Bobbi Jo; Smith, Ning X; Dobscha, Steven K; Deyo, Richard A; Perrin, Nancy A; Green, Carla A

    2017-04-01

    Some previous research has examined pain-related variables on the basis of prescription opioid dose, but data from studies involving patient-reported outcomes have been limited. This study examined the relationships between prescription opioid dose and self-reported pain intensity, function, quality of life, and mental health. Participants were recruited from 2 large integrated health systems, Kaiser Permanente Northwest (n = 331) and VA Portland Health Care System (n = 186). To be included, participants had to have musculoskeletal pain diagnoses and be receiving stable doses of long-term opioid therapy. We divided participants into 3 groups on the basis of current prescription opioid dose in daily morphine equivalent dose (MED): low dose (5-20 mg MED), moderate dose (20.1-50 mg MED), and higher dose (50.1-120 mg MED) groups. A statistically significant trend emerged where higher prescription opioid dose was associated with moderately sized effects including greater pain intensity, more impairments in functioning and quality of life, poorer self-efficacy for managing pain, greater fear avoidance, and more health care utilization. Rates of potential alcohol and substance use disorders also differed among groups. Findings from this evaluation reveal significant differences in pain-related and substance-related factors on the basis of prescription opioid dose.

  15. Efficacy of multimodal exercise-based rehabilitation on physical activity, cardiorespiratory fitness, and patient-reported outcomes in cancer survivors: a randomized, controlled trial

    PubMed Central

    Midtgaard, J.; Christensen, J. F.; Tolver, A.; Jones, L. W.; Uth, J.; Rasmussen, B.; Tang, L.; Adamsen, L.; Rørth, M.

    2013-01-01

    Background Sedentary behavior and impaired cardiovascular reserve capacity are common late effects of cancer therapy emphasizing the need for effective strategies to increase physical activity (PA) in cancer survivors. We examined the efficacy of a 12-month exercise-based rehabilitation program on self-reported PA, cardiorespiratory fitness (VO2peak), strength, and patient-reported outcomes. Patients and methods Two hundred fourteen post-treatment cancer survivors were randomly assigned to a 12-month rehabilitation program consisting of individual (x3) and group-based (x6) counseling in combination with once weekly high-intensity group-based exercise training (the Copenhagen Physical Activity after Cancer Treatment, PACT; n = 108) or to a health evaluation program (HE, n = 106). Study outcomes were assessed at baseline, 6 months, and 12 months. Results After 12 months, the percentage of patients reporting meeting PA goal behavior (≥3 h/week) was significantly increased in the PACT group versus the HE group (70.4% versus 43.4%, P = 0.001). Repeated measures analyses indicated a statistically significant improvement in VO2peak (l min−1) in favour of PACT (treatment effect ratio = 1.04; 95% confidence interval 1.00–1.07; P = 0.032). Significant between group differences were also observed for strength (P < 0.001), depression (P = 0.020) and mental health (P = 0.040). Conclusion A 12-month exercise-based rehabilitation program is an effective strategy to promote PA and improve VO2peak in cancer survivors. PMID:23704198

  16. 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

  17. 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…

  18. 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

  19. 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

  20. Pixel or Paper? Validation of a Mobile Technology for Collecting Patient-Reported Outcomes in Rheumatoid Arthritis

    PubMed Central

    Epis, Oscar Massimiliano; Casu, Cinzia; Belloli, Laura; Schito, Emanuela; Filippini, Davide; Muscarà, Marina; Gentile, Maria Giovanna; Venerelli, Chiara; Sonnati, Massimo; Schiavetti, Irene; Bruschi, Eleonora

    2016-01-01

    Background In the management of chronic disease, new models for telemonitoring of patients combined with the choice of electronic patient-reported outcomes (ePRO) are being encouraged, with a clear improvement of both patients’ and parents’ quality of life. An Italian study demonstrated that ePRO were welcome in patients with rheumatoid arthritis (RA), with excellent matching data. Objective The aim of this study is to evaluate the level of agreement between electronic and paper-and-pencil questionnaire responses. Methods This is an observational prospective study. Patients were randomly assigned to first complete the questionnaire by paper and pencil and then by tablet or in the opposite order. The questionnaire consisted of 3 independent self-assessment visual rating scales (Visual Analog Scale, Global Health score, Patient Global Assessment of Disease Activity) commonly used in different adult patients, including those with rheumatic diseases. Results A total of 185 consecutive RA patients were admitted to hospital and were enrolled and completed the questionnaire both on paper and on electronic versions. For all the evaluated items, the intrarater degree of agreement between 2 approaches was found to be excellent (intraclass correlation coefficient>0.75, P<.001). Conclusions An electronic questionnaire is uploaded in a dedicated Web-based tool that could implement a telemonitoring system aimed at improving the follow-up of RA patients. High intrarater reliability between paper and electronic methods of data collection encourage the use of a new digital app with consequent benefit for the overall health care system. PMID:27852561

  1. Multi-domain patient reported outcomes of irritable bowel syndrome: exploring person centered perspectives to better understand symptom severity scores

    PubMed Central

    Lackner, Jeffrey M.; Jaccard, James; Baum, Charles

    2012-01-01

    Objectives Patient reported outcomes (PRO) assessing multiple gastrointestinal symptoms are central to characterizing the therapeutic benefit of novel agents for irritable bowel syndrome (IBS). Common approaches that sum or average responses across different illness components must be unidimensional and have small unique variances to avoid aggregation bias and misinterpretation of clinical data. This study sought to evaluate the unidimensionality of the IBS Symptom Severity Scale (IBS-SSS) and to explore person centered cluster analytic methods for characterizing multivariate-based patient profiles. Methods Ninety-eight Rome-diagnosed IBS patients completed the IBS-SSS and a single, global item of symptom severity (UCLA Symptom Severity Scale) at pretreatment baseline of an NIH funded clinical trial. A k-means cluster analyses were performed on participants symptom severity scores. Results The IBS-SSS was not unidimensional. Exploratory cluster analyses revealed four common symptom profiles across five items of the IBS-SSS. One cluster of patients (25%) had elevated scores on pain frequency and bowel dissatisfaction, with less elevated but still high scores on life interference and low pain severity ratings. A second cluster (19%) was characterized by intermediate scores on both pain dimensions, but more elevated scores on bowel dissatisfaction. A third cluster (18%) was elevated across all IBS-SSS sub-components. The fourth and most common cluster (37%) had relatively low scores on all dimensions except bowel dissatisfaction and life interference due to IBS symptoms. Conclusions PRO endpoints and research on IBS more generally relying on multicomponent assessments of symptom severity should take into account the multidimensional structure of symptoms to avoid aggregation bias and to optimize the sensitivity of detecting treatment effects. PMID:23337220

  2. Patient-Reported Outcome (PRO) Assessment in Clinical Trials: A Systematic Review of Guidance for Trial Protocol Writers

    PubMed Central

    Calvert, Melanie; Kyte, Derek; Duffy, Helen; Gheorghe, Adrian; Mercieca-Bebber, Rebecca; Ives, Jonathan; Draper, Heather; Brundage, Michael; Blazeby, Jane; King, Madeleine

    2014-01-01

    Background Evidence suggests there are inconsistencies in patient-reported outcome (PRO) assessment and reporting in clinical trials, which may limit the use of these data to inform patient care. For trials with a PRO endpoint, routine inclusion of key PRO information in the protocol may help improve trial conduct and the reporting and appraisal of PRO results; however, it is currently unclear exactly what PRO-specific information should be included. The aim of this review was to summarize the current PRO-specific guidance for clinical trial protocol developers. Methods and Findings We searched the MEDLINE, EMBASE, CINHAL and Cochrane Library databases (inception to February 2013) for PRO-specific guidance regarding trial protocol development. Further guidance documents were identified via Google, Google scholar, requests to members of the UK Clinical Research Collaboration registered clinical trials units and international experts. Two independent investigators undertook title/abstract screening, full text review and data extraction, with a third involved in the event of disagreement. 21,175 citations were screened and 54 met the inclusion criteria. Guidance documents were difficult to access: electronic database searches identified just 8 documents, with the remaining 46 sourced elsewhere (5 from citation tracking, 27 from hand searching, 7 from the grey literature review and 7 from experts). 162 unique PRO-specific protocol recommendations were extracted from included documents. A further 10 PRO recommendations were identified relating to supporting trial documentation. Only 5/162 (3%) recommendations appeared in ≥50% of guidance documents reviewed, indicating a lack of consistency. Conclusions PRO-specific protocol guidelines were difficult to access, lacked consistency and may be challenging to implement in practice. There is a need to develop easily accessible consensus-driven PRO protocol guidance. Guidance should be aimed at ensuring key PRO information

  3. 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

  4. 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

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

    PubMed

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

    2015-06-01

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

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

    PubMed

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

    2016-04-01

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

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

    PubMed Central

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

    2016-01-01

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

  8. The PedsQL™ as a patient-reported outcome in children and adolescents with fibromyalgia: an analysis of OMERACT domains

    PubMed Central

    Varni, James W; Burwinkle, Tasha M; Limbers, Christine A; Szer, Ilona S

    2007-01-01

    Background Fibromyalgia is a chronic health condition characterized by widespread musculoskeletal pain, multiple tender points on physical examination, generalized muscular aching, stiffness, fatigue, nonrestorative sleep pattern, cognitive dysfunction, and mood disturbance. Recently, the Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) Fibromyalgia Syndrome Workshop ranked and prioritized the domains that should be consistently measured in fibromyalgia clinical trials, specifically, pain, generic health-related quality of life, fatigue, sleep quality, and physical function. The focus of these deliberations was exclusively on adult patients, and to our knowledge, these domains have not been previously tested within a multidimensional framework in children and adolescents with fibromyalgia. Methods An analysis to determine the feasibility, reliability, and validity of the PedsQL™ 4.0 (Pediatric Quality of Life Inventory™) Generic Core Scales, PedsQL™ Multidimensional Fatigue Scale, and PedsQL™ Rheumatology Module Pain and Hurt Scale as patient-reported outcome (PRO) measures for pediatric patients with fibromyalgia. The PedsQL™ Scales were completed by 59 families in a pediatric rheumatology clinic in a large children's hospital. Results The PedsQL™ evidenced minimal missing responses (0.53% patient self-report, 0.70% parent proxy-report), achieved excellent reliability for the Generic Core Scales Total Scale Score (α = 0.88 patient self-report, 0.87 parent proxy-report), the Multidimensional Fatigue Scale Total Scale Score (α = 0.94 patient self-report, 0.94 parent proxy-report), and acceptable reliability for the 4-item Rheumatology Module Pain and Hurt Scale (α = 0.68 patient self-report, 0.75 parent proxy-report). The PedsQL™ Generic Core Scales and Multidimensional Fatigue Scale significantly distinguished between pediatric patients with fibromyalgia and healthy children. Pediatric patients with fibromyalgia self

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

    PubMed

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

    2016-10-01

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

  10. Outcome Evaluation in Tendinopathy: Foundations of Assessment and a Summary of Selected Measures.

    PubMed

    Macdermid, Joy C; Silbernagel, Karin Grävare

    2015-11-01

    Synopsis Clinical measurement studies that address outcome evaluation for patients with tendinopathy should consider conceptual, clinical, practical, and measurement issues to guide the selection of valid measures. Clinical outcomes reported in research studies can provide benchmarks that assist with interpretation of scores during clinical decision making. Given the pathophysiology and functional impacts of tendinopathy, there is a need for outcome measures that assess physical impairments, activity performance, and patient-reported symptoms and function. Tendinopathy-specific patient-reported outcome measures have been shown to be superior to more generic tools for some conditions, such as lateral epicondyle tendinopathy (Patient-Rated Tennis Elbow Evaluation) and Achilles tendinopathy (Victorian Institute of Sport Assessment-Achilles), whereas both generic shoulder outcome measures and disease-specific measures perform similarly in individuals with rotator cuff tendinopathy. A patient-reported outcome measure that captures pain and limitation in function should be fundamental to outcome evaluation in patients with tendinopathy. The current measurement literature does not yet provide comprehensive empirical data to define optimal outcome measures for all types of tendinopathy. This article reviews concepts, instruments, and measurement properties that should provide clinicians with a foundation for assessment of condition severity and treatment outcomes in patients with tendinopathy. J Orthop Sports Phys Ther 2015;45(11):950-964. Epub 15 Oct 2015. doi:10.2519/jospt.2015.6054.

  11. Routine outcome measures in Canada.

    PubMed

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

    2015-01-01

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

  12. Clinical outcome measures of psoriasis.

    PubMed

    Bonifati, C; Berardesca, E

    2007-01-01

    Several tools have been introduced in clinical trials to quantify the severity and the response to a given therapeutic regimen of both psoriasis and psoriatic arthritis. Each method present specific advantages and limitations. Here we will discuss some of the most popular clinical outcome measures of both psoriasis (Psoriasis Severity Index, Physician Global Assessment, National Psoriasis Fundation-Psoriasis Score, Dermatology Life Quality Index) and psoriatic arthritis (American College Rheumatology response criteria, Psoriatic Arthritis Response Criteria).

  13. Asthma–COPD overlap syndrome in the US: a prospective population-based analysis of patient-reported outcomes and health care utilization

    PubMed Central

    Vaz Fragoso, Carlos A; Murphy, Terrence E; Agogo, George O; Allore, Heather G; McAvay, Gail J

    2017-01-01

    Background Prior work suggests that asthma–COPD overlap syndrome (ACOS) has a greater health burden than asthma alone or COPD alone. In the current study, we have further evaluated the health burden of ACOS in a nationally representative sample of the US population, focusing on patient-reported outcomes and health care utilization and on comparisons with asthma alone and COPD alone. Patient-reported outcomes are especially meaningful, as these include functional activities that are highly valued by patients and are the basis for patient-centered care. Methods Using data from the Medical Expenditure Panel Survey (MEPS), we evaluated patient-reported outcomes and health care utilization among participants who were aged 40–85 years and had self-reported, physician-diagnosed asthma or COPD. MEPS administered five rounds of interviews, at baseline and approximately every 6 months over 2.5 years. Patient-reported outcomes included activities of daily living (ADLs), mobility, social/recreational activities, disability days in bed, and health status (Short Form 12, Version 2). Health care utilization included outpatient and emergency department (ED) visits, and hospitalization. Results Of 3,486 participants with asthma or COPD, 1,585 (45.4%) had asthma alone, 1,294 (37.1%) had COPD alone, and 607 (17.4%) had ACOS. Relative to asthma alone, ACOS was significantly associated with higher odds of prevalent disability in ADLs and limitations in mobility and social/recreational activities (adjusted odds ratios [adjORs]: 1.91–3.98), as well as with higher odds of incident limitations in mobility and social/recreational activities, disability days in bed, and respiratory-based outpatient and ED visits, and hospitalization (adjORs: 1.86–2.35). In addition, ACOS had significantly worse physical and mental health scores than asthma alone (P-values <0.0001). Relative to COPD alone, ACOS was significantly associated with higher odds of prevalent limitations in mobility and

  14. Patient-reported outcomes from a phase III study of baricitinib in patients with conventional synthetic DMARD-refractory rheumatoid arthritis

    PubMed Central

    Emery, Paul; Blanco, Ricardo; Maldonado Cocco, Jose; Chen, Ying-Chou; Gaich, Carol L; DeLozier, Amy M; de Bono, Stephanie; Liu, Jiajun; Rooney, Terence; Chang, Cecile Hsiao-Chun; Dougados, Maxime

    2017-01-01

    Objectives To evaluate the effect of baricitinib on patient-reported outcomes (PROs) in patients with active rheumatoid arthritis (RA) and an inadequate response or intolerance to conventional synthetic disease-modifying antirheumatic drugs. Methods In this phase III study, patients were randomised 1:1:1 to placebo (N=228), baricitinib 2 mg once daily (QD, N=229) or baricitinib 4 mg QD (N=227). PROs included the Health Assessment Questionnaire-Disability Index (HAQ-DI), Patient's Global Assessment of Disease Activity (PtGA), patient's assessment of pain, measures from patient electronic daily diaries (duration and severity of morning joint stiffness (MJS), Worst Tiredness, Worst Joint Pain), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), SF-36, EuroQol 5-D index scores and visual analogue scales (VAS) and the Work Productivity and Activity Impairment Questionnaire-RA. The primary time point for the study was week 12. Treatment comparisons were assessed with logistic regression for categorical measures and analysis of covariance for continuous variables. Results Statistically significant improvements were observed for both baricitinib groups versus placebo in HAQ-DI, PtGA, pain, daily diary measures, EuroQoL index scores and SF-36 physical component score at week 12 and for those measures when assessed at week 24. Baricitinib 2 mg and baricitinib 4 mg were statistically significantly improved versus placebo for the EuroQoL VAS and FACIT-F, respectively, at week 24. Conclusions Baricitinib 2 or 4 mg provided significant improvement versus placebo in PROs across different domains of RA, including physical function, MJS, fatigue, pain and quality of life. Trial registration number NCT01721057; Results.

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

    PubMed Central

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

    2014-01-01

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

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

    PubMed Central

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

    2014-01-01

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

  17. Preoperative mental health status may not be predictive of improvements in patient-reported outcomes following an anterior cervical discectomy and fusion.

    PubMed

    Mayo, Benjamin C; Massel, Dustin H; Bohl, Daniel D; Narain, Ankur S; Hijji, Fady Y; Long, William W; Modi, Krishna D; Basques, Bryce A; Yacob, Alem; Singh, Kern

    2017-02-01

    OBJECTIVE Prior studies have correlated preoperative depression and poor mental health status with inferior patient-reported outcomes following lumbar spinal procedures. However, literature regarding the effect of mental health on outcomes following cervical spinal surgery is limited. As such, the purpose of this study is to test for the association of preoperative SF-12 Mental Component Summary (MCS) scores with improvements in Neck Disability Index (NDI), SF-12 Physical Component Summary (PCS), and neck and arm pain following anterior cervical discectomy and fusion (ACDF). METHODS A prospectively maintained surgical database of patients who underwent a primary 1- or 2-level ACDF during 2014-2015 was reviewed. Patients were excluded if they did not have complete patient-reported outcome data for the preoperative or 6-week, 12-week, or 6-month postoperative visits. At baseline, preoperative SF-12 MCS score was assessed for association with preoperative NDI, neck visual analog scale (VAS) score, arm VAS score, and SF-12 PCS score. The preoperative MCS score was then tested for association with changes in NDI, neck VAS, arm VAS, and SF-12 PCS scores from the preoperative visit to postoperative visits. These tests were conducted using multivariate regression controlling for baseline characteristics as well as for the preoperative score for the patient-reported outcome being assessed. RESULTS A total of 52 patients were included in the analysis. At baseline, a higher preoperative MCS score was negatively associated with a lower preoperative NDI (coefficient: -0.74, p < 0.001) and preoperative arm VAS score (-0.06, p = 0.026), but not preoperative neck VAS score (-0.03, p = 0.325) or SF-12 PCS score (0.04, p = 0.664). Additionally, there was no association between preoperative MCS score and improvement in NDI, neck VAS, arm VAS, or SF-12 PCS score at any of the postoperative time points (6 weeks, 12 weeks, and 6 months, p > 0.05 for each). The percentage of patients

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

    PubMed Central

    2013-01-01

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

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

    PubMed

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

    2015-12-01

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

  20. Evaluation of patient-reported outcomes data in structured diabetes education intervention: 2-year follow-up data of patient empowerment programme.

    PubMed

    Wong, Carlos K H; Lam, Cindy L K; Wan, Eric Y F; Chan, Anca K C; Pak, C H; Chan, Frank W K; Wong, William C W

    2016-11-01

    To examine the effects of a structured group-based education programme, patient empowerment programme (PEP), compared with usual care on 2-year changes in patient-reported outcomes (PRO) in patients with diabetes mellitus (DM). A prospective observational study of 715 patients (PEP/non-PEP: 390/325) was conducted to complete the baseline PRO survey and followed up for 2 years. Health-related quality of life (HRQOL) was measured using the short-form 12 at baseline and annually at two follow-up assessments, which yielded physical and mental component summary and SF-6D preference-based scores. Perceived control over diabetes and general health status were measured using the patient enablement instrument (PEI) and global rating scale (GRS) at follow-ups. When compared with non-PEP, PEP participants significantly reported improvement in health condition (GRS score > 0; 24.55 % vs 10.16 %; odds ratio = 2.502; P = 0.018) in 2 years and enabled the self-perceived control over diabetes (PEI score > 0; 72.20 % vs 38.40 %; odds ratio = 3.25; P < 0.001) in 1-year follow-up but no sustained effects in year 2 (52.65 % vs 39.04 %; odds ratio = 1.366; P = 0.265). There were no significant differences between PEP and non-PEP groups in the changes in quality of life scores (all P > 0.05) at 1 year. Although HRQOL scores deteriorated over 2-year period in both groups, PEP participants reported similar changes in HRQOL scores to that of non-PEP. PEP for DM patients preserved self-perceived disease control and health condition, whereas PEP participants perceived their HRQOL similar to that of non-PEP participants. Findings of PRO should be considered alongside clinical outcomes when evaluating the overall benefits of PEP.

  1. Quality of Data Entry Using Single Entry, Double Entry and Automated Forms Processing–An Example Based on a Study of Patient-Reported Outcomes

    PubMed Central

    Paulsen, Aksel; Overgaard, Søren; Lauritsen, Jens Martin

    2012-01-01

    Background The clinical and scientific usage of patient-reported outcome measures is increasing in the health services. Often paper forms are used. Manual double entry of data is defined as the definitive gold standard for transferring data to an electronic format, but the process is laborious. Automated forms processing may be an alternative, but further validation is warranted. Methods 200 patients were randomly selected from a cohort of 5777 patients who had previously answered two different questionnaires. The questionnaires were scanned using an automated forms processing technique, as well as processed by single and double manual data entry, using the EpiData Entry data entry program. The main outcome measure was the proportion of correctly entered numbers at question, form and study level. Results Manual double-key data entry (error proportion per 1000 fields = 0.046 (95% CI: 0.001–0.258)) performed better than single-key data entry (error proportion per 1000 fields = 0.370 (95% CI: 0.160–0.729), (p = 0.020)). There was no statistical difference between Optical Mark Recognition (error proportion per 1000 fields = 0.046 (95% CI: 0.001–0.258)) and double-key data entry (p = 1.000). With the Intelligent Character Recognition method, there was no statistical difference compared to single-key data entry (error proportion per 1000 fields = 6.734 (95% CI: 0.817–24.113), (p = 0.656)), as well as double-key data entry (error proportion per 1000 fields = 3.367 (95% CI: 0.085–18.616)), (p = 0.319)). Conclusions Automated forms processing is a valid alternative to double manual data entry for highly structured forms containing only check boxes, numerical codes and no dates. Automated forms processing can be superior to single manual data entry through a data entry program, depending on the method chosen. PMID:22493733

  2. Adjuvant Tamoxifen Plus Ovarian Function Suppression Versus Tamoxifen Alone in Premenopausal Women With Early Breast Cancer: Patient-Reported Outcomes in the Suppression of Ovarian Function Trial

    PubMed Central

    Luo, Weixiu; Bernhard, Jürg; Francis, Prudence A.; Burstein, Harold J.; Ciruelos, Eva; Bellet, Meritxell; Pavesi, Lorenzo; Lluch, Ana; Visini, Marilena; Parmar, Vani; Tondini, Carlo; Kerbrat, Pierre; Perelló, Antonia; Neven, Patrick; Torres, Roberto; Lombardi, Davide; Puglisi, Fabio; Karlsson, Per; Ruhstaller, Thomas; Colleoni, Marco; Coates, Alan S.; Goldhirsch, Aron; Price, Karen N.; Gelber, Richard D.; Regan, Meredith M.; Fleming, Gini F.

    2016-01-01

    Purpose The Suppression of Ovarian Function trial showed improved disease control for tamoxifen plus ovarian function suppression (OFS) compared with tamoxifen alone for the cohort of premenopausal patients who received prior chemotherapy. We present the patient-reported outcomes. Patients and Methods The quality-of-life (QoL) analysis includes 1,722 of 2,045 premenopausal patients with hormone receptor–positive breast cancer randomly assigned to receive adjuvant treatment with 5 years of tamoxifen plus OFS or tamoxifen alone. Chemotherapy use before enrollment was optional. Patients completed a QoL form consisting of global and symptom indicators at baseline, every 6 months for 24 months, and annually during years 3 to 6. Differences in the change of QoL from baseline between the two treatments were tested at 6, 24, and 60 months with mixed models for repeated measures with and without chemotherapy and overall. Results Patients on tamoxifen plus OFS were more affected than patients on tamoxifen alone by hot flushes at 6 and 24 months, by loss of sexual interest and sleep disturbance at 6 months, and by vaginal dryness up to 60 months. Without prior chemotherapy, patients on tamoxifen alone reported more vaginal discharge over the 5 years than patients on tamoxifen plus OFS. Symptom-specific treatment differences at 6 months were less pronounced in patients with prior chemotherapy. Changes in global QoL indicators from baseline were small and similar between treatments over the whole treatment period. Conclusion Overall, OFS added to tamoxifen resulted in worse endocrine symptoms and sexual functioning during the first 2 years of treatment, with variable magnitudes of treatment differences. Short-term differences in symptom-specific QoL, treatment burden, and coping effort between treatment groups were less pronounced for patients with prior chemotherapy, the cohort that benefited most from OFS in terms of disease control. PMID:27022111

  3. Outcome Measures in Clinical Trials for Multiple Sclerosis.

    PubMed

    van Munster, Caspar E P; Uitdehaag, Bernard M J

    2017-02-09

    Due to the heterogeneous nature of the disease, it is a challenge to capture disease activity of multiple sclerosis (MS) in a reliable and valid way. Therefore, it can be difficult to assess the true efficacy of interventions in clinical trials. In phase III trials in MS, the traditionally used primary clinical outcome measures are the Expanded Disability Status Scale and the relapse rate. Secondary outcome measures in these trials are the number or volume of T2 hyperintense lesions and gadolinium-enhancing T1 lesions on magnetic resonance imaging (MRI) of the brain. These secondary outcome measures are often primary outcome measures in phase II trials in MS. Despite several limitations, the traditional clinical measures are still the mainstay for assessing treatment efficacy. Newer and potentially valuable outcome measures increasingly used or explored in MS trials are, clinically, the MS Functional Composite and patient-reported outcome measures, and on MRI, brain atrophy and the formation of persisting black holes. Several limitations of these measures have been addressed and further improvements will probably be proposed. Major improvements are the coverage of additional functional domains such as cognitive functioning and assessment of the ability to carry out activities of daily living. The development of multidimensional measures is promising because these measures have the potential to cover the full extent of MS activity and progression. In this review, we provide an overview of the historical background and recent developments of outcome measures in MS trials. We discuss the advantages and limitations of various measures, including newer assessments such as optical coherence tomography, biomarkers in body fluids and the concept of 'no evidence of disease activity'.

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

    PubMed Central

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

    2015-01-01

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

  5. Measurement outcomes from hip simulators.

    PubMed

    de Villiers, Danielle; Shelton, Julia C

    2016-05-01

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

  6. Outcome measures in inflammatory rheumatic diseases

    PubMed Central

    2009-01-01

    Inflammatory rheumatic diseases are generally multifaceted disorders and, therefore, measurement of multiple outcomes is relevant to most of these diseases. Developments in outcome measures in the rheumatic diseases are promoted by the development of successful treatments. Outcome measurement will increasingly deal with measurement of low levels of disease activity and avoidance of disease consequences. It is an advantage for patient management and knowledge transfer if the same outcomes are used in practice and in trials. Continuous measures of change are generally the most powerful and, therefore, are preferred as primary outcomes in trials. For daily clinical practice, outcome measures should reflect the patients' state and have to be easily derivable. The objective of this review is to describe recent developments in outcome measures for inflammatory rheumatic diseases for trials and clinical practice, with an emphasis on rheumatoid arthritis. PMID:19849821

  7. [Measuring outcome in spasticity rehabilitation].

    PubMed

    Fheodoroff, K; Wissel, J; Entner, T; Freimüller, M

    2001-01-01

    Spasticity is a frequent consequence of upper motor neuron lesion and is associated with a variety of symptoms such as pain, muscle stiffness and reflex patterns that interfere with activities of daily living, dexterity and gait. As therapy strategies in managing spasticity-associated problems have been evolving there is an increasing need for a practicable documentation system which describes spasticity and related symptoms on different levels in order to evaluate especially the level of functioning. In daily routine the single-case-design reflects a useful technique to evaluate the status in terms of technical, functional and individual goals for treatment. However, there is no single tool to measure the different types of changes due to treatment, therefore a variety of selecting tests, based on the functional changes expected from the selected treatment, is recommended. The sensitivity of the selected tests should match the range of expected improvements related to the specific treatment. Technical goals should be evaluated by validated spasticity rating scales. As changes in technical measures of spasticity such as muscle tone, muscle length, range of motion or repetitive voluntary movements may not correlate with clinical improvements, individual functional goals should be defined. Those functional goals should reflect the patients' and care-givers' individual perception of the actual problem. A treatment diary is a useful tool to document subjective perception of changes over time. Some practical issues are adressed below. Reliable outcome measures enable patients and doctors to select further treatment strategies and gives health care providers information on treatment expectations in return for their investments.

  8. Measuring Outcomes in Children's Services.

    ERIC Educational Resources Information Center

    Christner, Anne Marshall, Ed.

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

  9. Comparison of Patient-reported Outcomes after Implant Versus Autologous Tissue Breast Reconstruction Using the BREAST-Q

    PubMed Central

    Pirro, Ortensia; Vindigni, Vincenzo; Sukop, Andrej; Hromadkova, Veronika; Nguyenova, Alena; Vitova, Lenka; Bassetto, Franco

    2017-01-01

    Background: The demand for reconstructive breast procedures of various types has accelerated in recent years. Coupled with increased patient expectations, it has fostered the development of oncoplastic and reconstructive techniques in breast surgery. In the setting of postmastectomy reconstruction, patient satisfaction and quality of life are the most significant outcome variables when evaluating surgical success. The aim of this study was to evaluate the quality of life after implant breast reconstruction compared with autologous breast reconstruction. Materials and Methods: A cross-sectional study design was used. A total of 65 women who had completed postmastectomy implant-based or autologous reconstruction in the participating center were asked to complete the BREAST-Q (Reconstruction Module). Results: Data analysis demonstrated that women with autologous breast reconstruction were significantly more satisfied with their breasts (P = 0.0003) and with the overall outcome (P = 0.0001) compared with women with implant breast reconstruction. All other BREAST-Q parameters that were considered and observed were not significantly different between the 2 patient groups. Conclusions: Through statistical analysis, our results showed that patients who underwent autologous tissue reconstruction had better satisfaction with the reconstructed breast and the outcome, while both techniques appear to equally improve psychosocial well-being, sexual well-being, and chest satisfaction. PMID:28203513

  10. Comparison of objective and patient-reported hot flash measures in men with prostate cancer.

    PubMed

    Hanisch, Laura J; Palmer, Steven C; Marcus, Steven C; Hantsoo, Liisa; Vaughn, David J; Coyne, James C

    2009-01-01

    Hot flashes are one of the bothersome symptoms frequently experienced after endocrine treatments for breast and prostate cancers. Many studies have evaluated interventions for hot flashes, but results are obscured by methodologic limitations. We compared the performance of three techniques to measure hot flashes over 48 hours among 47 patients with prostate cancer undergoing androgen deprivation therapy to determine the feasibility and accuracy of each measure. Sternal skin conductance, electronic event marking, and twice daily diaries identified 478, 410, and 285 hot flashes, respectively. Diaries produced the lowest hourly hot flash rate (M = 0.17), which was significantly different from the rates of the objective profile (M = 0.28) and event marks (M = 0.23).The sensitivity and positive predictive value of the three measures demonstrated that the diaries underperformed, but these values did not exceed moderate levels for any measure (% = 28-59). Study results suggest the combined use of sternal skin conductance and event marking for the measurement of hot flash frequency in pathophysiologic studies and clinical trials with prostate cancer patients. Conversely, the use of retrospective diaries may be adequate for clinical practice to determine clinically significant changes in salient events.

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

    PubMed Central

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

    2014-01-01

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

  12. Response Rates for Patient-Reported Outcomes Using Web-Based Versus Paper Questionnaires: Comparison of Two Invitational Methods in Older Colorectal Cancer Patients

    PubMed Central

    Vissers, Pauline AJ; Mols, Floortje; Thong, Melissa SY; van de Poll-Franse, Lonneke V

    2015-01-01

    Background Improving questionnaire response rates is an everlasting issue for research. Today, the Internet can easily be used to collect data quickly. However, collecting data on the Internet can lead to biased samples because not everyone is able to access or use the Internet. The older population, for example, is much less likely to use the Internet. The Patient-Reported Outcomes Following Initial Treatment and Long-Term Evaluation of Survivorship (PROFILES) registry offers a platform to collect Web-based and paper questionnaires and to try different measures to improve response rates. Objective In this study, our aim was to study the influence of two methods of invitation on the response rate. Our second aim was to examine the preference of questionnaire mode of administration (paper or Web-based) for the older patient in particular. Methods To test these two invitational methods, 3406 colorectal cancer patients between ages 18 and 85 years received an invitation containing an access code for the Web-based questionnaire. They could also request a paper questionnaire with an included reply card (paper-optional group). In contrast, 179 randomly selected colorectal cancer patients received a paper questionnaire with the invitation (paper-included group). They could also choose to fill out the Web-based questionnaire with the included access code. Results Response rates did not differ between the paper-optional and the paper-included groups (73.14%, 2491/3406 and 74.9%, 134/179, P=.57). In the paper-optional group, online response was significantly higher when compared to the paper-included group (41.23%, 1027/2491 vs 12.7%, 17/134, P<.001). The majority of online respondents responded after the first invitation (95.33%, 979/1027), which was significantly higher than the paper respondents (52.19%, 764/1464, P<.001). Respondents aged 70 years and older chose to fill out a paper questionnaire more often (71.0%, 677/954). In the oldest age group (≥80 years), 18

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

    PubMed Central

    Edte, Alexander; Schmitt, Sonja; Lukas, Kati

    2010-01-01

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

  14. Comparative responsiveness of outcome measures for total knee arthroplasty

    PubMed Central

    Giesinger, K.; Hamilton, D.F.; Jost, B.; Holzner, B.; Giesinger, J.M.

    2014-01-01

    Summary Objective The aim of this study was to compare the responsiveness of various patient-reported outcome measures (PROMs) and clinician-reported outcomes following total knee arthroplasty (TKA) over a 2-year period. Methods Data were collected in a prospective cohort study of primary TKA. Patients who had completed Forgotten Joint Score-12 (FJS-12), Western Ontario and McMaster Universities (WOMAC) osteoarthritis (OA) index, EQ-5D, Knee Society Score and range of movement (ROM) assessment were included. Five time points were assessed: pre-operative, 2 months, 6 months, 1 year and 2 years post-operative. Results Data from 98 TKAs were available for analysis. Largest effect sizes (ES) for change from pre-operative to 2-month follow-up were observed for the Knee Society Score (KSS) Knee score (1.70) and WOMAC Total (−1.50). For the period from 6 months to 1 year the largest ES for change were shown by the FJS-12 (0.99) and the KSS Function Score (0.88). The EQ-5D showed the strongest ceiling effect at 1-year follow-up with 84.4% of patients scoring the maximum score. ES for the time from 1- to 2-year follow-up were largest for the FJS-12 (0.50). All other outcome measures showed ES equal or below 0.30. Conclusion Outcome measures differ considerably in responsiveness, especially beyond one year post-operatively. Joint-specific outcome measures are more responsive than clinician-reported or generic health outcome tools. The FJS-12 was the most responsive of the tools assessed; suggesting that joint awareness may be a more discerning measure of patient outcome than traditional PROMs. PMID:24262431

  15. Measuring Assistive Technology Outcomes in Education.

    ERIC Educational Resources Information Center

    Smith, Roger O.

    2000-01-01

    This article reviews relevant theory and the practical implications of measuring assistive technology outcomes of students with disabilities. Rasch Measurement Scaling, Time Series Concurrent and Differential Approach, and the Multi-Attribute Utility Model are proposed for potential use in assistive technology outcome measurement and future…

  16. Patient-reported outcomes instruments: bridging the gap between international copyright laws and common practice for developers and users--a case example.

    PubMed

    Anfray, Caroline

    2009-12-01

    Copyright laws are intended to protect the rights of authors in their literary, scientific, and artistic works. The recent controversy about the standardized version of the Asthma Quality of Life Questionnaire between Elizabeth Juniper and Eirini Grammatopoulou et al. is an example of the difficulties inherent to copyright faced by developers and users of patient-reported outcome (PRO) instruments. This brief communication presents the basics of international copyright laws (i.e., the Berne Convention), the facts behind the controversy, and our analysis of the case based on our experience as a distributing center of PRO instruments. We conclude that better communication between developers and users would prevent most unfortunate complications and misunderstandings.

  17. Impact of the Patient-Reported Outcomes Management Information System (PROMIS) upon the design and operation of multi-center clinical trials: a qualitative research study.

    PubMed

    Eisenstein, Eric L; Diener, Lawrence W; Nahm, Meredith; Weinfurt, Kevin P

    2011-12-01

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

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

    PubMed Central

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

    2013-01-01

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

  19. Patient-Reported Outcomes in Psoriasis Patients with Clear Versus Almost Clear Skin in the Clinical Setting

    PubMed Central

    Takeshita, Junko; Duffin, Kristina Callis; Shin, Daniel B.; Krueger, Gerald G.; Robertson, Andrew D.; Troxel, Andrea B.; Van Voorhees, Abby S.; Gelfand, Joel M.

    2014-01-01

    Background There is little evidence to guide the establishment of treatment goals for moderate-to-severe psoriasis in the clinical setting. Objective To compare Dermatology Life Quality Index scores and prescription topical medication use between patients with clear versus almost clear skin. Methods Multicenter cross-sectional study of 97 patients with clear skin and 441 patients with almost clear skin receiving current systemic therapy or phototherapy for a primary indication of plaque psoriasis evaluated at one of ten US outpatient dermatology sites participating in the Dermatology Clinical Effectiveness Research Network. Results In adjusted analyses, patients with clear versus almost clear skin were more likely to report no impact of psoriasis on quality of life (relative risk 1.60; 95% confidence interval, 1.37–1.86). Patients with clear versus almost clear skin were also more likely to report no prescription topical medication use in the preceding week (relative risk 2.08; 95% confidence interval, 1.73–2.49). Limitations Cross-sectional design prohibits longitudinal assessment of outcomes. Conclusions and Relevance Clinically important differences in quality of life and prescription topical medication use exist between patients with clear versus almost clear skin. Collectively, our results indicate that achievement of clear skin may be an important clinical distinction among patients with moderate-to-severe psoriasis. PMID:24928705

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

    ERIC Educational Resources Information Center

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

    2012-01-01

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

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

    PubMed Central

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

    2016-01-01

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

  2. Body mass index is not a clinically meaningful predictor of patient reported outcomes of primary hip replacement surgery: prospective cohort study

    PubMed Central

    Judge, Andy; Batra, Rajbir N; Thomas, Geraint; Beard, David; Javaid, M Kassim; Murray, David; Dieppe, Paul A; Dreinhoefer, Karsten; Peter-Guenther, Klaus; Field, Richard; Cooper, Cyrus; Arden, Nigel K

    2014-01-01

    Objectives To describe whether body mass index (BMI) is a clinically meaningful predictor of patient reported outcomes following primary total hip replacement (THR) surgery Design Combined data from prospective cohort studies. We obtained information from four cohorts of patients receiving primary THR for osteoarthritis: Exeter Primary Outcomes Study (n=1431); EUROHIP (n=1327); Elective Orthopaedic Centre (n=2832); and St. Helier (n=787). The exposure of interest was pre-operative BMI. Confounding variables included: age, sex, SF-36 mental health, comorbidities, fixed flexion, analgesic use, college education, OA in other joints, expectation of less pain, radiographic K&L grade, ASA grade, years of hip pain. The primary outcome was the Oxford Hip Score (OHS). Regression models describe the association of BMI on outcome adjusting for all confounders. Results For a 5-unit increase in BMI, the attained 12-month OHS decreases by 0.78 points 95%CI (0.27 to 1.28), p-value 0.001. Compared to people of normal BMI (20 to 25), those in the obese class II (BMI 35 to 40) would have a 12-month OHS that is 2.34 points lower. Although statistically significant this effect is small and not clinically meaningful in contrast to the substantial change in OHS seen across all BMI groupings. In obese class II patients achieved a 22.2 point change in OHS following surgery. Conclusions Patients achieved substantial change in OHS after THR across all BMI categories, which greatly outweighs the small difference in attained post-operative score. The findings suggest BMI should not present a barrier to access THR in terms of PROMs. PMID:24418679

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

    PubMed Central

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

    2015-01-01

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

  4. Current status and future perspectives of patient-reported outcome research in clinical trials for patients with breast cancer in Japan.

    PubMed

    Ohsumi, Shozo; Shimozuma, Kojiro

    2013-10-01

    The aim of healthcare providers is to help patients regain their health and/or maintain or increase their happiness. The quality of healthcare can be assessed objectively by using indices such as survival rates, but subjective assessment is sometimes more important than objective evaluation, because whether patients feel healthy or happy or not is their major concern. Quality of life (QOL) research is a typical approach to subjective assessment of health. Self-administered health-related QOL (HRQOL) questionnaires are used to obtain patient-reported outcome (PRO) data. PRO is defined by the US Food and Drug Administration (FDA) as any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else. PRO data are regarded as more accurate than clinician-rated ones in terms of assessing patients' symptoms and HRQOL. The Comprehensive Support Project for Health Outcome Research of the Public Health Research Foundation has conducted much PRO research in collaboration with the Comprehensive Support Project for Oncology Research, especially in randomized controlled trials (RCTs). Here, we review the results of PRO research in RCTs conducted in Japan and examine future perspectives in this field.

  5. Prospective assessment of oral mucositis and its impact on quality of life and patient-reported outcomes during radiotherapy for head and neck cancer.

    PubMed

    Franco, Pierfrancesco; Martini, Stefania; Di Muzio, Jacopo; Cavallin, Chiara; Arcadipane, Francesca; Rampino, Monica; Ostellino, Oliviero; Pecorari, Giancarlo; Garzino Demo, Paolo; Fasolis, Massimo; Airoldi, Mario; Ricardi, Umberto

    2017-05-01

    Oral mucositis (OM) is a common acute side effect during radiotherapy treatments for head and neck cancer (HNC), with a potential impact on patient's compliance to therapy, quality of life (QoL) and clinical outcomes. Its timely and appropriate management is of paramount importance. Several quantitative scoring scales are available to properly assess OM and its influence on patient-reported outcomes (PROs) and QoL. We prospectively assessed OM in a cohort of HNC patients submitted to radiation using the Oral Mucositis Assessment Scale (OMAS), while its impact on PROs and QoL was evaluated employing the Oral Mucositis Weekly Questionnaire-Head and Neck Cancer (OMWQ-HN) and the Functional Assessment of Cancer Therapy-Head and Neck Cancer (FACT-HN). Evaluation of OMAS scores highlighted a progressive increase in OM during treatment and a partial recovery after the end of radiation. These trends were correlated to PROs and QoL as evaluated with OMWQ-HN and FACT-HN questionnaires. In the present study, we provided a quantitative assessment of OM, PROs and QoL in HNC patient undergoing radiotherapy, potentially useful for future comparison.

  6. Comparing the Quality of Ambulatory Surgical Care for Skin Cancer in a Veterans Affairs Clinic and a Fee-For-Service Practice Using Clinical and Patient-Reported Measures

    PubMed Central

    Linos, Eleni; Arron, Sarah T.; Hills, Nancy K.

    2017-01-01

    The Institute of Medicine has identified serious deficiencies in the measurement of cancer care quality, including the effects on quality of life and patient experience. Moreover, comparisons of quality in Veterans Affairs Medical Centers (VA) and other sites are timely now that many Veterans can choose where to seek care. To compare quality of ambulatory surgical care for keratinocyte carcinoma (KC) between a VA and fee-for-service (FFS) practice, we used unique clinical and patient-reported data from a comparative effectiveness study. Patients were enrolled in 1999–2000 and followed for a median of 7.2 years. The practices differed in a few process measures (e.g., median time between biopsy and treatment was 7.5 days longer at VA) but there were no substantial or consistent differences in clinical outcomes or a broad range of patient-reported outcomes. For example, 5-year tumor recurrence rates were equally low (3.6% [2.3–5.5] at VA and 3.4% [2.3–5.1] at FFS), and similar proportions of patients reported overall satisfaction at one year (78% at VA and 80% at FFS, P = 0.69). These results suggest that the quality of care for KC can be compared comprehensively in different health care systems, and suggest that quality of care for KC was similar at a VA and FFS setting. PMID:28141817

  7. Integrative Oncology Physician Consultations at a Comprehensive Cancer Center: Analysis of Demographic, Clinical and Patient Reported Outcomes.

    PubMed

    Lopez, Gabriel; McQuade, Jennifer; Cohen, Lorenzo; Williams, Jane T; Spelman, Amy R; Fellman, Bryan; Li, Yisheng; Bruera, Eduardo; Lee, Richard T

    2017-01-01

    Background: Integrative oncology (IO) is a relatively new field that seeks to bring evidence-based, non-conventional approaches into conventional oncology care in a coordinated and safe manner. Though complementary and alternative medicine (CAM) are highly utilized by cancer patients, little is known about the characteristics of patients seeking IO consultation. Methods: Patients presenting for an outpatient IO consultation completed a CAM use questionnaire, Measure Yourself Concerns and Wellbeing (MYCaW), Edmonton Symptom Assessment Scale (ESAS), Quality of Life Short Form 12 (SF-12), and post-consultation satisfaction item. Results: 2,474 new patient IO consultations were conducted from 9/2009 to 12/2013 and 2367 (96%) completed at least one measure. Most were female (69%); the most frequent cancer type was breast (29%); 38% had distant/advanced disease; 75% had used a CAM approach in prior 12 months. The most common concerns were seeking an integrative/holistic approach (34%), herbs/supplements (34%), and diet/nutrition (21%). Overall symptom burden was low, with baseline symptom scores (ESAS) highest (worst) for sleep (4.2; SD 2.8), fatigue (4.0; SD 2.8), and well-being (3.8; SD 2.6). On the SF-12, the physical health scores (35.3; SD 7.5) were significantly lower than that of a healthy population (50), but mental health scores were not (46.8; SD 10.2). Satisfaction was high (9.4; SD 1.3) with the consultation. Conclusions: Patients presenting for IO consultation tended to have early stage disease, had previously used a CAM approach, had a relatively low symptom burden, and were most interested in developing an integrative approach to their care or discussing herbs/supplement use.

  8. Integrative Oncology Physician Consultations at a Comprehensive Cancer Center: Analysis of Demographic, Clinical and Patient Reported Outcomes

    PubMed Central

    Lopez, Gabriel; McQuade, Jennifer; Cohen, Lorenzo; Williams, Jane T; Spelman, Amy R; Fellman, Bryan; Li, Yisheng; Bruera, Eduardo; Lee, Richard T

    2017-01-01

    Background: Integrative oncology (IO) is a relatively new field that seeks to bring evidence-based, non-conventional approaches into conventional oncology care in a coordinated and safe manner. Though complementary and alternative medicine (CAM) are highly utilized by cancer patients, little is known about the characteristics of patients seeking IO consultation. Methods: Patients presenting for an outpatient IO consultation completed a CAM use questionnaire, Measure Yourself Concerns and Wellbeing (MYCaW), Edmonton Symptom Assessment Scale (ESAS), Quality of Life Short Form 12 (SF-12), and post-consultation satisfaction item. Results: 2,474 new patient IO consultations were conducted from 9/2009 to 12/2013 and 2367 (96%) completed at least one measure. Most were female (69%); the most frequent cancer type was breast (29%); 38% had distant/advanced disease; 75% had used a CAM approach in prior 12 months. The most common concerns were seeking an integrative/holistic approach (34%), herbs/supplements (34%), and diet/nutrition (21%). Overall symptom burden was low, with baseline symptom scores (ESAS) highest (worst) for sleep (4.2; SD 2.8), fatigue (4.0; SD 2.8), and well-being (3.8; SD 2.6). On the SF-12, the physical health scores (35.3; SD 7.5) were significantly lower than that of a healthy population (50), but mental health scores were not (46.8; SD 10.2). Satisfaction was high (9.4; SD 1.3) with the consultation. Conclusions: Patients presenting for IO consultation tended to have early stage disease, had previously used a CAM approach, had a relatively low symptom burden, and were most interested in developing an integrative approach to their care or discussing herbs/supplement use. PMID:28261340

  9. Patient-reported outcomes of azelaic acid foam 15% for patients with papulopustular rosacea: secondary efficacy results from a randomized, controlled, double-blind, phase 3 trial.

    PubMed

    Tyring, Stephen; Solomon, James A; Staedtler, Gerald; Lott, Jason P; Nkulikiyinka, Richard; Shakery, Kaweh

    2016-10-01

    Patient-reported treatment outcomes are important for evaluating the impact of drug therapies on patient experience. A randomized, double-blind, vehicle-controlled, parallel-group, multicenter, phase 3 study was conducted in 961 participants to assess patient perception of efficacy, utility, and effect on quality of life (QOL) of an azelaic acid (AzA) 15% foam formulation for the treatment of papulopustular rosacea (PPR). Secondary end points included patient-reported global assessment of treatment response, global assessment of tolerability, and opinion on cosmetic acceptability and practicability of product use. Quality of life assessments included the Dermatology Quality of Life Index (DLQI) and Rosacea Quality of Life Index (RosaQOL). Self-reported global assessment of treatment response favored AzA foam over vehicle foam (P<.001), with 57.2% of the AzA foam group reporting excellent or good improvement versus 44.7% in the vehicle foam group. Tolerability was rated excellent or good in 67.8% of the AzA foam group versus 78.2% of the vehicle foam group. Mean overall DLQI scores at end of treatment (EoT) were improved (P=.018) in favor of the AzA foam group compared with the vehicle foam group. Both treatment groups showed improvements in RosaQOL. Treatment with AzA foam was associated with improved QOL and meaningful reductions in the patient-perceived burden of PPR, which correlates with earlier reported primary end points of this study and supports the inclusion of patient perspectives in studies evaluating the effects of topical dermatologic treatments.

  10. Measuring Inclusive Education Outcomes in Alberta, Canada

    ERIC Educational Resources Information Center

    Loreman, Tim

    2014-01-01

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

  11. Time for a new era in the evaluation of targeted therapies for patients with chronic myeloid leukemia: inclusion of quality of life and other patient-reported outcomes.

    PubMed

    Efficace, F; Cocks, K; Breccia, M; Sprangers, M; Meyers, C A; Vignetti, M; Baccarani, M; Mandelli, F

    2012-02-01

    Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) might be crucial in comparing effectiveness of treatments as they could provide invaluable information to better inform clinical decision-making. This is particularly true in the era of targeted therapies (TT). A systematic review was undertaken on all studies with CML patients published from 1980 to 2010 and including a PRO evaluation. Out of 619 articles scrutinized, 15 met eligibility criteria and no study was published before 1995. Six dealt mainly with interferon-based therapies, 7 with bone marrow transplantation and only 2 evaluated PROs in the context of TT. No disease-specific, validated PRO instrument for these patients was found. The main evidence being that Imatinib provides clear advantage in terms of HRQOL over interferon-based treatments. There is lack of data concerning PROs in patients treated with current TT. Documenting HRQOL and side effects of CML treatments, from the patients' perspective is needed to evaluate overall treatment effectiveness and net clinical benefit of newer therapeutic strategies.

  12. Quality of Patient-Reported Outcome (PRO) Reporting Across Cancer Randomized Controlled Trials According to the CONSORT PRO Extension: A Pooled Analysis of 557 Trials

    PubMed Central

    Efficace, Fabio; Fayers, Peter; Pusic, Andrea; Cemal, Yeliz; Yanagawa, Jane; Jacobs, Marc; la Sala, Andrea; Cafaro, Valentina; Whale, Katie; Rees, Jonathan; Blazeby, Jane

    2016-01-01

    Background The main objectives of this study were to identify the number of randomized controlled trials (RCTs) that have included a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate completeness of PRO reporting according to the CONSORT PRO extension. Methods RCTs with a PRO endpoint, conducted across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated based on previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaboration's tool for assessing RCT Risk of Bias. Analyses were also conducted by type of PRO endpoint (primary versus secondary) and by cancer disease site. Results A total of 56,696 potentially eligible records were scrutinized and 557 RCTs with a PRO evaluation, enrolling overall 254,677 patients, were identified. PROs were most frequently used in RCTs of breast (N=123), lung (N=85) and colorectal (N=66) cancer. Overall, PROs were secondary endpoint in 421 (76%) RCTs. Four out of six CONSORT PRO items evaluated were documented in less than 50% of the RCTs. Level of reporting was higher in RCTs with PRO as a primary endpoint. Presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PRO as primary (β=0.19; P=0.001) or secondary endpoint (β=0.30; P<0.001). Conclusion Implementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute in revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting. PMID:26079197

  13. PATIENT-REPORTED OUTCOMES OF QUALITY OF LIFE, FUNCTIONING, AND DEPRESSIVE SYMPTOM SEVERITY IN MAJOR DEPRESSIVE DISORDER COMORBID WITH PANIC DISORDER BEFORE AND AFTER SSRI TREATMENT IN THE STAR*D TRIAL

    PubMed Central

    IsHak, Waguih William; Mirocha, James; Christensen, Scott; Wu, Fan; Kwock, Richard; Behjat, Joseph; Pi, Sarah; Akopyan, A.; Peselow, Eric D.; Cohen, Robert M.; Elashoff, David

    2014-01-01

    Background Panic disorder (PD) is highly comorbid with major depressive disorder (MDD) with potential impact on patient-reported outcomes of quality of life (QOL), functioning, and depressive symptom severity Methods Using data from the sequenced treatment alternatives to relieve depression (STAR*D) trial, we compared entry and post-SSRI-treatment QOL, functioning, and depressive symptom severity scores in MDD patients with comorbid PD (MDD+PD) to MDD patients without PD (MDDnoPD). We also compared pre- and posttreatment proportions of patients with severe impairments in quality of life and functioning. Results MDD+PD patients experienced significantly lower QOL and functioning and more severe depressive symptoms than MDDnoPD patients at entry. Following treatment with citalopram, both groups showed significant improvements, however, nearly 30–60% of patients still suffered from severe quality of life and functioning impairments. MDD+PD patients exited with lower QOL and functioning than MDDnoPD patients, a difference that became statistically insignificant after adjusting for baseline measures of depressive symptom severity, functioning, and QOL, comorbid anxiety disorders (PTSD, GAD, social, and specific phobias), age, and college education. Conclusions Functional outcomes using QOL and functioning measures should be utilized in treating and researching MDD so that shortfalls in traditional treatment can be identified and additional interventions can be designed to address severe baseline QOL and functioning deficits in MDD comorbid with PD. PMID:23861180

  14. International multiphase mixed methods study protocol to develop a cross-cultural patient-reported outcome instrument for children and young adults with cleft lip and/or palate (CLEFT-Q)

    PubMed Central

    Wong Riff, Karen W Y; Tsangaris, Elena; Goodacre, Tim; Forrest, Christopher R; Pusic, Andrea L; Cano, Stefan J; Klassen, Anne F

    2017-01-01

    Introduction Patient-reported outcome (PRO) instruments should be developed according to rigorous guidelines in order to provide clinically meaningful, scientifically sound measurement. Understanding the methodology behind instrument development informs the selection of the most appropriate tool. This mixed methods protocol describes the development of an internationally applicable PRO instrument, the CLEFT-Q, for evaluating outcomes of treatment for cleft lip and/or palate (CL/P). Methods and analysis The study includes three main phases that occur iteratively and interactively. In phase I, we determine what concepts are important to patients regarding their outcome. A conceptual framework for the CLEFT-Q is formed through a systematic review and an extensive international qualitative study. The systematic review ascertains what concepts have previously been measured in patients with CL/P. The qualitative study employs interpretive description and involves in-depth interviews with patients in high-income and lower-middle income countries. Preliminary items are generated from the qualitative data. Preliminary scales are then created for each theme in the framework. Cognitive debriefing interviews and expert clinician input are used to refine the scales in an iterative process. In phase II, the preliminary scales are administered to a large international group of patients with CL/P. The modern psychometric method of Rasch Measurement Theory analysis is employed to define the measurement characteristics. The preliminary scales are shortened based on these results. In phase III, further tests assess reliability, validity and responsiveness of the instrument. Ethics and dissemination The study is approved by Research Ethics Boards for each participating site. Findings from this study will be published in open access peer-reviewed journals and presented at national and international conferences. Integrated knowledge translation is employed to engage stakeholders from

  15. Preliminary Patient Reported Outcomes Analysis of 3DCRT versus IMRT on the High Dose Arm of the Radiation Therapy Oncology Group (RTOG) 0126 Prostate Cancer Trial

    PubMed Central

    Bruner, Deborah Watkins; Hunt, Daniel; Michalski, Jeff; Bosch, Walter; Galvin, James; Amin, Mahul; Xiao, Canhua; Bahary, Jean-Paul; Patel, Malti; Chafe, Susan; Rodrigues, George; Lau, Harold; Duclos, Marie; Baikadi, Madhava; Deshmukh, Snehal; Sandler, Howard

    2015-01-01

    Purpose A preliminary report of patient reported outcomes (PROs) between men receiving high-dose radiation therapy (RT) on RTOG 0126, a phase III dose-escalation trial treated with either 3-dimensional conformal RT (3D-CRT) or intensity modulated RT (IMRT). Methods 3D-CRT patients received 55.8 Gy to the prostate and proximal seminal vesicles (P+PSV) and allowed for an optional field reduction, then 23.4 Gy to prostate only. IMRT patients received 79.2 Gy to the P+PSV. PROs were assessed at 0 (baseline), 3, 6, 12, and 24 months and included bladder and bowel function assessed with the Functional Alterations due to Changes in Elimination (FACE) and erectile function assessed with the International Index of Erectile Function (IIEF). Analyses included those who completed all data at baseline and at least one follow-up and compared to an imputed data set. Results Of 763 patients randomized to the 79.2-Gy arm, 551 and 595 patients who responded to FACE, 505 and 577 who responded to the IIEF were included in the completed and imputed analyses, respectively. There were no significant differences between modalities for any of the FACE or IIEF subscale or total scores at any time point for either the completed or imputed data sets. Conclusions Despite significant reductions in dose and volume to normal structures using IMRT, this robust analysis of 3D-CRT and IMRT showed no difference in PRO bowel, bladder and sexual functions for similar doses delivered to the P+PSV for IMRT compared to 3D-CRT delivered to either the P+PSV or prostate alone. PMID:25847819

  16. Design, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic review

    PubMed Central

    Mercieca-Bebber, Rebecca; Palmer, Michael J; Brundage, Michael; Stockler, Martin R; King, Madeleine T

    2016-01-01

    Objectives Patient-reported outcomes (PROs) provide important information about the impact of treatment from the patients' perspective. However, missing PRO data may compromise the interpretability and value of the findings. We aimed to report: (1) a non-technical summary of problems caused by missing PRO data; and (2) a systematic review by collating strategies to: (A) minimise rates of missing PRO data, and (B) facilitate transparent interpretation and reporting of missing PRO data in clinical research. Our systematic review does not address statistical handling of missing PRO data. Data sources MEDLINE and Cumulative Index to Nursing and Allied Health Literature (CINAHL) databases (inception to 31 March 2015), and citing articles and reference lists from relevant sources. Eligibility criteria English articles providing recommendations for reducing missing PRO data rates, or strategies to facilitate transparent interpretation and reporting of missing PRO data were included. Methods 2 reviewers independently screened articles against eligibility criteria. Discrepancies were resolved with the research team. Recommendations were extracted and coded according to framework synthesis. Results 117 sources (55% discussion papers, 26% original research) met the eligibility criteria. Design and methodological strategies for reducing rates of missing PRO data included: incorporating PRO-specific information into the protocol; carefully designing PRO assessment schedules and defining termination rules; minimising patient burden; appointing a PRO coordinator; PRO-specific training for staff; ensuring PRO studies are adequately resourced; and continuous quality assurance. Strategies for transparent interpretation and reporting of missing PRO data include utilising auxiliary data to inform analysis; transparently reporting baseline PRO scores, rates and reasons for missing data; and methods for handling missing PRO data. Conclusions The instance of missing PRO data and its

  17. Association of obesity with patient-reported outcomes in patients with axial spondyloarthritis: a cross-sectional study in an urban Asian population.

    PubMed

    Lee, Yi Xuan; Kwan, Yu Heng; Png, Wan Yu; Lim, Ka Keat; Tan, Chuen Seng; Lui, Nai Lee; Chew, Eng Hui; Thumboo, Julian; Østbye, Truls; Fong, Warren

    2017-04-04

    To determine if obesity is associated with poorer patient-reported outcomes (PROs) in patients with axial spondyloarthritis (axSpA), we conducted a cross-sectional study using data of the PRESPOND registry from a tertiary referral center in Singapore between 2011 and 2015. Demographics, clinical, and PRO variables were collected. Patients were divided into three categories: normal (BMI < 23 kg/m(2)), overweight (23 kg/m(2) ≤ BMI < 27.5 kg/m(2)) and obese (BMI ≥ 27.5 kg/m(2)), using Asian BMI classification. The dependent variables are Pain score, Bath Ankylosing Spondylitis Patient Global Score (BAS-G), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), Bath Ankylosing Spondylitis Functional Index (BASFI), Health Assessment Questionnaire (HAQ), and Medical Outcomes Study Short Form 36 version 2 (SF-36). Multivariate regression analyses were performed with these dependent variables and obesity categories, adjusting for confounders. Among 194 patients with axSpA, 32% are overweight while 22% are obese. We found that obese patients had significant poorer pain (β: 11.87, 95%CI 2.13, 21.60) and BAS-G scores (β: 10.18, 95%CI 1.59, 18.76) when compared to normal BMI patients. However, obesity was not associated with BASDAI (β 0.50, 95%CI -0.22, 1.22), BASFI (β 0.08, 95%CI -0.66, 0.81), HAQ (β -0.07, 95%CI -0.21, 0.06), physical component summary (β -0.02, 95%CI -4.47, 4.44), and mental component summary (β -2.85, 95%CI -7.57, 1.88) of SF-36. Obesity was associated with pain score and BAS-G but not with BASDAI, BASFI, HAQ, and SF-36. Further study is needed to examine the causal relationship between obesity and poorer PROs.

  18. Self-Reported Health Experiences of Children Living with Congenital Heart Defects: Including Patient-Reported Outcomes in a National Cohort Study

    PubMed Central

    Tadic, Valerija; Hogan, Ailbhe; Bull, Catherine; Rahi, Jugnoo Sangeeta; Dezateux, Carol

    2016-01-01

    optimising participation. We highlight the potential for informing and evaluating clinical practice and service provision through seeking patient-reported outcomes in paediatric care. PMID:27487183

  19. Challenges in measuring outcomes for caregivers of people with mental health problems.

    PubMed

    Zendjidjian, Xavier Y; Boyer, Laurent

    2014-06-01

    Patient-reported outcomes (PROs) are increasingly important in health care and mental health research. Furthermore, caregivers become partners in care for patients with mental disorders, and health workers are more attentive to the expectations and needs of caregivers. A number of outcomes for caregivers are measured and used in daily practice in order to promote actions to improve health care systems and progress in research on the impact of mental disorders on their caregivers. This paper proposes an inventory of the different outcomes and different measurement tools used to assess the impact of disorders, raising a number of methodological and conceptual issues that limit the relevance of measurement tools and complicate their use. Finally, we propose some recommendations promoting the development of relevant outcome measures for caregivers and their integration into current systems of care.

  20. Patient-reported outcomes in clinical trials of CKD-related therapies: report of a symposium sponsored by the national kidney foundation and the U.S. Food and Drug Administration.

    PubMed

    Perrone, Ronald D; Coons, Stephen Joel; Cavanaugh, Kerri; Finkelstein, Fred; Meyer, Klemens B

    2013-12-01

    The National Kidney Foundation and the U.S. Food and Drug Administration (FDA) convened a symposium in September 2010, bringing together more than 70 experts, including representatives from the FDA, the National Institutes of Health, the Critical Path Institute, nephrologists, patients, and the pharmaceutical industry to discuss the feasibility and process of developing patient-reported outcome (PRO) measures to access how patients feel or function to be used in clinical trials for regulatory review of treatment benefit. Three disease areas were evaluated for development of end point models in which PRO measures may be useful: anemia secondary to chronic kidney disease, autosomal dominant polycystic kidney disease (ADPKD), and nephrotic syndrome. The participants thought it valuable to use observational data to generate hypotheses regarding patient baseline characteristics that are likely to predict clinically important changes in PROs in response to anemia treatment and to design adequately powered blinded randomized controlled trials of anemia treatment using PROs as primary rather than secondary end points. Validated PRO instruments that reflect the patient experience in ADPKD and nephrotic syndrome are essential to incorporate into clinical trials of new therapeutic interventions because glomerular filtration rate decline may occur late in the disease course, at which point therapeutic benefit is less likely. Conference attendees addressed how PRO measures could be used to evaluate, monitor, provide care, and facilitate the introduction of treatments for patients with these challenging conditions.

  1. Electromyographic and patient-reported outcomes of a computer-guided occlusal adjustment performed on patients suffering from chronic myofascial pain

    PubMed Central

    Dib, Abraham; Sanchez, José-Miguel; López-Valverde, Antonio

    2015-01-01

    Objectives: Muscular hyperactivity is a potential source of symptoms in patients with temporal-mandibular disorders. An adequate occlusal adjustment may relieve such symptoms. This study aims to measure the effect of shortening the protrusive disclusion time (DT) and balancing the center of occlusal forces (COF) on the EMG recordings and assess the pain reported by chronic patients one month after the computer-guided occlusal adjustment. Study Design: The sample studied comprised 34 patients suffering from chronic facial pain in which the EMG activity of both masseters was recorded by electromyography. By selective grinding we alleviated all the occlusal interferences during the mandibular protrusion from the habitual closure position in order to establish an immediate posterior disclusion and an equilibration of the COF. Results: At follow-up 76.5% of the patients reported no facial pain. Moreover, the EMG activity and protrusive DT were significantly reduced, and occlusal and muscular function were significantly more symmetric than at baseline. Conclusions: According to this EMG study, this computer-guided occlusal adjustment is able to reduce the activity of the masseters and the self-reported muscular pain of patients one-month after treatment. Key words:Myofascial pain, occlusal adjustment, electromyography, T-Scan III, occlusal interferences. PMID:25475783

  2. Testing Multiple Outcomes in Repeated Measures Designs

    ERIC Educational Resources Information Center

    Lix, Lisa M.; Sajobi, Tolulope

    2010-01-01

    This study investigates procedures for controlling the familywise error rate (FWR) when testing hypotheses about multiple, correlated outcome variables in repeated measures (RM) designs. A content analysis of RM research articles published in 4 psychology journals revealed that 3 quarters of studies tested hypotheses about 2 or more outcome…

  3. Systematic evaluation of patient-reported outcome (PRO) protocol content and reporting in UK cancer clinical trials: the EPiC study protocol

    PubMed Central

    Ahmed, Khaled; Kyte, Derek; Keeley, Thomas; Efficace, Fabio; Armes, Jo; Brown, Julia M; Calman, Lynn; Copland, Chris; Gavin, Anna; Glaser, Adam; Greenfield, Diana M; Lanceley, Anne; Taylor, Rachel; Velikova, Galina; Brundage, Michael; Mercieca-Bebber, Rebecca; King, Madeleine T

    2016-01-01

    Introduction Emerging evidence suggests that patient-reported outcome (PRO)-specific information may be omitted in trial protocols and that PRO results are poorly reported, limiting the use of PRO data to inform cancer care. This study aims to evaluate the standards of PRO-specific content in UK cancer trial protocols and their arising publications and to highlight examples of best-practice PRO protocol content and reporting where they occur. The objective of this study is to determine if these early findings are generalisable to UK cancer trials, and if so, how best we can bring about future improvements in clinical trials methodology to enhance the way PROs are assessed, managed and reported. Hypothesis: Trials in which the primary end point is based on a PRO will have more complete PRO protocol and publication components than trials in which PROs are secondary end points. Methods and analysis Completed National Institute for Health Research (NIHR) Portfolio Cancer clinical trials (all cancer specialities/age-groups) will be included if they contain a primary/secondary PRO end point. The NIHR portfolio includes cancer trials, supported by a range of funders, adjudged as high-quality clinical research studies. The sample will be drawn from studies completed between 31 December 2000 and 1 March 2014 (n=1141) to allow sufficient time for completion of the final trial report and publication. Two reviewers will then review the protocols and arising publications of included trials to: (1) determine the completeness of their PRO-specific protocol content; (2) determine the proportion and completeness of PRO reporting in UK Cancer trials and (3) model factors associated with PRO protocol and reporting completeness and with PRO reporting proportion. Ethics and dissemination The study was approved by the ethics committee at University of Birmingham (ERN_15-0311). Trial findings will be disseminated via presentations at local, national and international conferences, peer

  4. Patient Reported Outcome (PRO) assessment in epilepsy: a review of epilepsy-specific PROs according to the Food and Drug Administration (FDA) regulatory requirements

    PubMed Central

    2013-01-01

    Despite collection of patient reported outcome (PRO) data in clinical trials of antiepileptic drugs (AEDs), PRO results are not being routinely reported on European Medicines Agency (EMA) and Food and Drug Administration (FDA) product labels. This review aimed to evaluate epilepsy-specific PRO instruments against FDA regulatory standards for supporting label claims. Structured literature searches were conducted in Embase and Medline databases to identify epilepsy-specific PRO instruments. Only instruments that could potentially be impacted by pharmacological treatment, were completed by adults and had evidence of some validation work were selected for review. A total of 26 PROs were reviewed based on criteria developed from the FDA regulatory standards. The ability to meet these criteria was classified as either full, partial or no evidence, whereby partial reflected some evidence but not enough to comprehensively address the FDA regulatory standards. Most instruments provided partial evidence of content validity. Input from clinicians and literature was common although few involved patients in both item generation and cognitive debriefing. Construct validity was predominantly compromised by no evidence of a-priori hypotheses of expected relationships. Evidence for test-retest reliability and internal consistency was available for most PROs although few included complete results regarding all subscales and some failed to reach recommended thresholds. The ability to detect change and interpretation of change were not investigated in most instruments and no PROs had published evidence of a conceptual framework. The study concludes that none of the 26 have the full evidence required by the FDA to support a label claim, and all require further research to support their use as an endpoint. The Subjective Handicap of Epilepsy (SHE) and the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) have the fewest gaps that would need to be addressed through

  5. Patient Reported Outcome (PRO) assessment in epilepsy: a review of epilepsy-specific PROs according to the Food and Drug Administration (FDA) regulatory requirements.

    PubMed

    Nixon, Annabel; Kerr, Cicely; Breheny, Katie; Wild, Diane

    2013-03-11

    Despite collection of patient reported outcome (PRO) data in clinical trials of antiepileptic drugs (AEDs), PRO results are not being routinely reported on European Medicines Agency (EMA) and Food and Drug Administration (FDA) product labels. This review aimed to evaluate epilepsy-specific PRO instruments against FDA regulatory standards for supporting label claims. Structured literature searches were conducted in Embase and Medline databases to identify epilepsy-specific PRO instruments. Only instruments that could potentially be impacted by pharmacological treatment, were completed by adults and had evidence of some validation work were selected for review. A total of 26 PROs were reviewed based on criteria developed from the FDA regulatory standards. The ability to meet these criteria was classified as either full, partial or no evidence, whereby partial reflected some evidence but not enough to comprehensively address the FDA regulatory standards. Most instruments provided partial evidence of content validity. Input from clinicians and literature was common although few involved patients in both item generation and cognitive debriefing. Construct validity was predominantly compromised by no evidence of a-priori hypotheses of expected relationships. Evidence for test-retest reliability and internal consistency was available for most PROs although few included complete results regarding all subscales and some failed to reach recommended thresholds. The ability to detect change and interpretation of change were not investigated in most instruments and no PROs had published evidence of a conceptual framework. The study concludes that none of the 26 have the full evidence required by the FDA to support a label claim, and all require further research to support their use as an endpoint. The Subjective Handicap of Epilepsy (SHE) and the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) have the fewest gaps that would need to be addressed through

  6. Multi-institutional Prospective Evaluation of Bowel Quality of Life After Prostate External Beam Radiation Therapy Identifies Patient and Treatment Factors Associated With Patient-Reported Outcomes: The PROSTQA Experience

    SciTech Connect

    Hamstra, Daniel A.; Conlon, Anna S.C.; Daignault, Stephanie; Dunn, Rodney L.; Sandler, Howard M.; Hembroff, A. Larry; Zietman, Anthony L.; Kaplan, Irving; Ciezki, Jay; Kuban, Deborah A.; Wei, John T.; Sanda, Martin G.; Michalski, Jeff M.

    2013-07-01

    Purpose: To evaluate patients treated with external beam radiation therapy as part of the multicenter Prostate Cancer Outcomes and Satisfaction with Treatment Quality Assessment (PROSTQA), to identify factors associated with posttreatment patient-reported bowel health-related quality of life (HRQOL). Methods and Materials: Pretreatment characteristics and treatment details among 292 men were evaluated using a general linear mixed model for their association with measured HRQOL by the Expanded Prostate Cancer Index Composite instrument through 2 years after enrollment. Results: Bowel HRQOL had a median score of 100 (interquartile range 91.7-100) pretreatment and 95.8 (interquartile range 83.3-100) at 2 years, representing new moderate/big problems in 11% for urgency, 7% for frequency, 4% for bloody stools, and 8% for an overall bowel problems. Baseline bowel score was the strongest predictor for all 2-year endpoints. In multivariable models, a volume of rectum ≥25% treated to 70 Gy (V70) yielded a clinically significant 9.3-point lower bowel score (95% confidence interval [CI] 16.8-1.7, P=.015) and predicted increased risks for moderate to big fecal incontinence (P=.0008). No other radiation therapy treatment-related variables influenced moderate to big changes in rectal HRQOL. However, on multivariate analyses V70 ≥25% was associated with increases in small, moderate, or big problems with the following: incontinence (3.9-fold; 95% CI 1.1-13.4, P=.03), rectal bleeding (3.6-fold; 95% CI 1.3-10.2, P=.018), and bowel urgency (2.9-fold; 95% CI 1.1-7.6, P=.026). Aspirin use correlated with a clinically significant 4.7-point lower bowel summary score (95% CI 9.0-0.4, P=.03) and an increase in small, moderate, or big problems with bloody stools (2.8-fold; 95% CI 1.2-6.4, P=.018). Intensity modulated radiation therapy was associated with higher radiation therapy doses to the prostate and lower doses to the rectum but did not independently correlate with bowel HRQOL

  7. Definitions and outcome measures of clinical trials regarding opioid-induced constipation: a systematic review.

    PubMed

    Gaertner, Jan; Siemens, Waldemar; Camilleri, Michael; Davies, Andrew; Drossman, Douglas A; Webster, Lynn R; Becker, Gerhild

    2015-01-01

    Opioid-induced constipation (OIC) is a frequent symptom in patients treated with opioids and impacts the patients' quality of life. However, there is no generally accepted definition for OIC. The aims of this study were to identify definitions for OIC in clinical trials and Cochrane Reviews and to compile assessment tools and outcome measures that were used in clinical trials. In a systematic review, 5 databases (MEDLINE, PubMed, The Cochrane Library, Web of Science, and EMBASE) were searched to identify clinical trials assessing OIC in adult patients or healthy volunteers. Studies published between 1993 and August 2013 were included. A total of 1488 studies were retrieved and 47 publications were included in the analysis. A minority of the publications (n=16, 34%) provided a clear definition for OIC. The definitions were highly variable and the present or recent history of opioid therapy was frequently (n=6, 38%) not included in these definitions. Of 46 clinical trials, 17 (37%) relied exclusively on objective measures such as bowel movement frequency, whereas another 17 studies additionally included patient-reported outcome measures such as, "feeling of incomplete bowel evacuation." Few trials (n=7, 15%) assessed the patient-reported global burden of OIC. Standard definitions and outcome measures are necessary (i) for consistency in OIC diagnosis in clinical practice and clinical trials; and (ii) to assure comparability of trial findings (eg, in meta-analyses). An OIC definition and outcome measures are proposed.

  8. Optimal functional outcome measures for assessing treatment for Dupuytren’s disease: a systematic review and recommendations for future practice

    PubMed Central

    2013-01-01

    Background Dupuytren's disease of the hand is a common condition affecting the palmar fascia, resulting in progressive flexion deformities of the digits and hence limitation of hand function. The optimal treatment remains unclear as outcomes studies have used a variety of measures for assessment. Methods A literature search was performed for all publications describing surgical treatment, percutaneous needle aponeurotomy or collagenase injection for primary or recurrent Dupuytren’s disease where outcomes had been monitored using functional measures. Results Ninety-one studies met the inclusion criteria. Twenty-two studies reported outcomes using patient reported outcome measures (PROMs) ranging from validated questionnaires to self-reported measures for return to work and self-rated disability. The Disability of Arm, Shoulder and Hand (DASH) score was the most utilised patient-reported function measure (n=11). Patient satisfaction was reported by eighteen studies but no single method was used consistently. Range of movement was the most frequent physical measure and was reported in all 91 studies. However, the methods of measurement and reporting varied, with seventeen different techniques being used. Other physical measures included grip and pinch strength and sensibility, again with variations in measurement protocols. The mean follow-up time ranged from 2 weeks to 17 years. Conclusions There is little consistency in the reporting of outcomes for interventions in patients with Dupuytren’s disease, making it impossible to compare the efficacy of different treatment modalities. Although there are limitations to the existing generic patient reported outcomes measures, a combination of these together with a disease-specific questionnaire, and physical measures of active and passive individual joint Range of movement (ROM), grip and sensibility using standardised protocols should be used for future outcomes studies. As Dupuytren’s disease tends to recur

  9. Psychological factors predict adherence to methotrexate in rheumatoid arthritis; findings from a systematic review of rates, predictors and associations with patient-reported and clinical outcomes

    PubMed Central

    Bluett, James; Barton, Anne; Hyrich, Kimme L; Cordingley, Lis; Verstappen, Suzanne M M

    2016-01-01

    Treatment response to methotrexate (MTX) for rheumatoid arthritis (RA) is not universal and non-adherence may partially explain this. The aims of this systematic review were to: (1) summarise existing rates of adherence to MTX, (2) identify predictors of adherence to MTX, and (3) assess the association between non-adherence and patient outcomes. The authors conducted a systematic search of papers published from January 1980 to February 2015 in PubMed, PsycINFO, EMBASE and CINAHL databases. Studies were eligible for inclusion if: (1) MTX was used as monotherapy or in combination with other therapies, (2) MTX was used in an RA or inflammatory polyarthritis population, (3) adherence was defined and measured as the extent to which patients followed their MTX regimen during the period of prescription, and (4) it was an original piece of research. In total, 10 studies met the inclusion criteria and 8 were evaluated as high quality. Rates of adherence ranged from 59% to 107%, and exposed differences in definitions of adherence, study methodologies and sample heterogeneity. A number of potential predictors of MTX adherence were identified; the strongest being related to beliefs in the necessity and efficacy of MTX, absence of low mood, mild disease and MTX monotherapy. Furthermore, 3 studies tested the association of adherence with disease activity as an outcome measure; all 3 found non-adherence associated with poor treatment response. This systematic review shows the importance of adherence to MTX treatment and summarises the associated modifiable factors. PMID:26848403

  10. [Patient reported outcome of tyrosine kinase inhibitor related side effects and their impact on daily life in Chinese patients with chronic myeloid leukemia in the chronic phase].

    PubMed

    Yu, L; Wang, H B; Jiang, Q

    2016-11-14

    Objective: To explore the impact of patient reported outcome of tyrosine kinase inhibitor (TKI) related side effects on daily life in Chinese patients with chronic myeloid leukemia (CML) in the chronic phase (CP). Methods: From May to November in 2014, anonymous questionnaires were distributed to adult CML patients who were receiving TKI treatment in China. The impact of TKI-related side effects on daily life were assessed by the score of 1 (no impact) to 5 (high impact) from patient self-report. Results: Data from 731 respondents in the CP who reported the score of the impact of TKI-related side effects on daily life were collected. 407 (56%) were male. The median age was 41 years (range, 18 to 88 years). 560 (77%) started TKI treatment within 1 year after diagnosis. With a median treatment duration of 3 years (range, <1 to 13 years), 549 (75% ) respondents achieved a complete cytogenetic response (CCyR) and 301 (41%) achieved a complete molecular response (CMR). The most common TKI-related adverse effects were edema (n=323, 44% ), fatigue (n=277, 38% ), gastrointestinal disorders (n=235, 32% ), skin color changes (n=142, 19% ), muscle cramps (n=137, 19% ), rash (n=105, 14% ), hepatic function abnormalities (n=91, 12%), weight gain (n=86, 12%), and cytopenia (n=59, 8%). Multivariate analyses showed that TKI treatment duration <4 years was the factor associated with fatigue; Edema was more observed in female, ≥40 years old and use of 1st generation TKI; Gastrointestinal disorders in use of 1st generation TKI; Hepatic function abnormalities and rash in use of 2nd generation TKI; Weight gain in female; Muscle cramps in long-term interval from diagnosis to therapy and use of 1st generation TKI; Low blood counts in use of generic TKI. There was no impact of TKI-related side effects on daily life in 218 (30%)respondents (1 score). 375 (51%)respondents reported their daily life were slightly or moderately decreased (2 or 3 score), while 138 (19%) significantly

  11. Patient-Reported Outcomes in Implant-Based Breast Reconstruction Alone or in Combination with a Titanium-Coated Polypropylene Mesh - A Detailed Analysis of the BREAST-Q and Overview of the Literature.

    PubMed

    Dieterich, M; Angres, J; Stubert, J; Stachs, A; Reimer, T; Gerber, B

    2015-07-01

    Background: Complication rates and surgical outcomes are well reported for implant-based breast reconstruction (IBBR) using supportive materials for the inferior pole of the breast. Patient-reported outcomes (PRO) are underrepresented. The aim of this study was to compare PRO in IBBR using implants alone or in combination with a synthetic mesh. Methods and Methods: PRO was measured in patients undergoing IBBR alone or in combination with a titanium-covered polypropylene mesh (TiLOOP® Bra). In this non-randomized observational trial PRO was retrospectively assessed using the validated self-reporting BREAST-Q. The raw responses of all questions applied in each domain and transformed BREAST-Q data using the Q-Score are presented. Results: Of 90 eligible women, 42 received IBBR alone and 48 received IBBR in combination with mesh. No differences in complication rates were observed. The return rate was 67.7 % and was comparable between the groups (p = 0.117). PRO revealed no differences regarding satisfaction with breast shape (p = 0.079), outcome (p = 0.604), nipple sensitivity (p = 0.502), preoperative information (p = 0.195), office staff (p = 0.462), psychosocial well-being (p = 0.370), sexual well-being (p = 0.508) and physical well-being (p = 0.654). Significant differences were noted regarding satisfaction with the surgeon (p = 0.013) and medical staff (p = 0.035) as well as the response behavior of certain questions of the sub-domains, thus helping to further stratify PRO with regards to aesthetic outcome. However, no differences were observed in the main BREAST-Q results. Conclusion: Use of the TiLOOP® Bra in IBBR results in comparable BREAST-Q scores compared with IBBR alone. Evaluating the BREAST-Q sub-domains helps to stratify PRO more profoundly and assists in interpreting the overall results and specific research questions.

  12. Revisiting the Surveillance Epidemiology and End Results Cancer Registry and Medicare Health Outcomes Survey (SEER-MHOS) Linked Data Resource for Patient-Reported Outcomes Research in Older Adults with Cancer.

    PubMed

    Kent, Erin E; Malinoff, Rochelle; Rozjabek, Heather M; Ambs, Anita; Clauser, Steven B; Topor, Marie A; Yuan, Gigi; Burroughs, James; Rodgers, Anne B; DeMichele, Kimberly

    2016-01-01

    Researchers and clinicians are increasingly recognizing the value of patient-reported outcome (PRO) data to better characterize people's health and experiences with illness and care. Considering the rising prevalence of cancer in adults aged 65 and older, PRO data are particularly relevant for older adults with cancer, who often require complex cancer care and have additional comorbid conditions. A data linkage between the Surveillance Epidemiology and End Results (SEER) cancer registry and the Medicare Health Outcomes Survey (MHOS) was created through a partnership between the National Cancer Institute and the Centers for Medicare and Medicaid Services that created the opportunity to examine PROs in Medicare Advantage enrollees with and without cancer. The December 2013 linkage of SEER-MHOS data included the linked data for 12 cohorts, bringing the number of individuals in the linked data set to 95,723 with cancer and 1,510,127 without. This article reviews the features of the resource and provides information on some descriptive characteristics of the individuals in the data set (health-related quality of life, body mass index, fall risk management, number of unhealthy days in the past month). Individuals without (n=258,108) and with (n=3,440) cancer (1,311 men with prostate cancer, 982 women with breast cancer, 689 with colorectal cancer, 458 with lung cancer) were included in the current descriptive analysis. Given increasing longevity, advances in effective therapies and earlier detection, and population growth, the number of individuals aged 65 and older with cancer is expected to reach more than 12 million by 2020. SEER-MHOS provides population-level, self-reported, cancer registry-linked data for person-centered surveillance research on this growing population.

  13. Patient-reported outcomes in a phase III, randomized study of sunitinib versus interferon-α as first-line systemic therapy for patients with metastatic renal cell carcinoma in a European population

    PubMed Central

    Castellano, D.; del Muro, X. Garcia; Pérez-Gracia, J. L.; González-Larriba, J. L.; Abrio, M. V.; Ruiz, M. A.; Pardo, A.; Guzmán, C.; Cerezo, S. Díaz; Grande, E.

    2009-01-01

    Background: The purpose of this study is to evaluate the impact on the health-related quality of life (HRQoL) of sunitinib versus interferon-alpha (IFN-α) treatment in patients with metastatic renal cell carcinoma (mRCC). Patients and methods: In all, 304 mRCC patients (European cohort) were randomized 1 : 1 to receive sunitinib (50 mg/day for 4 weeks, followed by 2 weeks off) or IFN-α (9 million units s.c. injection three times/week). The following questionnaires were completed (days 1 and 28 per cycle): Functional Assessment of Cancer Therapy-General (FACT-G), the FACT-Kidney Symptom Index and the EuroQol Group's EQ-5D self-report questionnaire (EQ-5D). Results correspond to an ongoing trial with progression-free survival time as primary end point, and patients were still being followed up. Data were analyzed using repeated measures mixed effects models (MEMs) that allow the inclusion of initial differences and uncompleted repeated measures, with the assumption of data missing at random. Six-cycle results were included. Results: Results consistently showed that patients in sunitinib group experienced statistically significantly milder kidney-related symptoms, better cancer-specific HRQoL and general health status (in social utility scores) during the study period as measured by these patient-reported outcome end points. No statistical differences between groups were found on the FACT-G physical well-being subscale or the EQ-5D VAS values. Conclusions: Results from MEM showed the sunitinib's benefit on HRQoL compared with IFN-α. PMID:19549706

  14. Understanding the outcomes measures used in Huntington disease pharmacologicaltrials: A systematic review

    PubMed Central

    Carlozzi, Noelle E; Miciura, Angela; Migliore, Nicholas; Dayalu, Praveen

    2014-01-01

    Background The identification of the gene mutation causing Huntington disease has raised hopes for new treatments to ease symptoms and slow functional decline. As such, there has been a push towards designing efficient pharmacological trials (i.e., drug trials), especially with regard to selecting outcomes measures that are both brief and sensitive to changes across the course of the disease, from subtle prodromal changes, to more severe end-stage changes. Objectives Recently, to aid in efficient development of new HD research studies, the National Institute of Neurological Disorders and Stroke (NINDS) published recommendations for measurement selection in HD. While these recommendations are helpful, many of the recommended measures have little published data in HD. As such, we conducted a systematic review of the literature to identify the most common outcomes measures used in HD clinical trials. Methods Major medical databases, including PubMed, Embase, CINAHL, and the Cochrane Central Register of Controlled Trials, were used to identify peer-reviewed journal articles in English from 2001 through April 2013; 151 pharmacological trials were identified. Results The majority of HD clinical trials employed clinician-reported outcomes measures (93%); patient reported outcome measures (11%) and observer reported outcome measures (3%) were used with much less frequency. Conclusions We provide a review of the most commonly used measures across these trials, compare these measures to the clinical recommendations made by the NINDS working groups, and provide recommendations for selecting measures for future clinical trials that meet the Food and Drug Administration standards. PMID:25300328

  15. The International Dermatology Outcome Measures Group: formation of patient-centered outcome measures in dermatology.

    PubMed

    Gottlieb, Alice B; Levin, Adriane A; Armstrong, April W; Abernethy, April; Duffin, Kristina Callis; Bhushan, Reva; Garg, Amit; Merola, Joseph F; Maccarone, Mara; Christensen, Robin

    2015-02-01

    As quality standards are increasingly in demand throughout medicine, dermatology needs to establish outcome measures to quantify the effectiveness of treatments and providers. The International Dermatology Outcome Measures Group was established to address this need. Beginning with psoriasis, the group aims to create a tool considerate of patients and providers using the input of all relevant stakeholders in assessment of disease severity and response to treatment. Herein, we delineate the procedures through which consensus is being reached and the future directions of the project.

  16. How is physical activity measured in lung cancer?A systematic review of outcome measures and their psychometric properties.

    PubMed

    Edbrooke, Lara; Denehy, Linda; Parry, Selina M; Astin, Ronan; Jack, Sandy; Granger, Catherine L

    2017-02-01

    Physical activity (PA) levels are low in patients with lung cancer. Emerging evidence supports the use of interventions to increase PA in this population. We aimed to (1) identify and synthesize outcome measures which assess PA levels in patients with lung cancer and (2) to evaluate, synthesize and compare the psychometric properties of these measures. A systematic review of articles from searches was conducted of five electronic databases and personal records. Eligible studies were those which assessed PA using either performance-based or patient-reported measures. For aim 2, studies identified in aim 1 reporting on at least one psychometric property (validity, reliability, responsiveness or measurement error) were included. Two independent reviewers assessed eligibility and risk of bias with the COnsensus-based Standards for the selection of health status Measurement INstruments. Thirty-four studies using 21 different measures of PA were identified. Seventeen studies used performance-based measures. The Godin Leisure Time Exercise Questionnaire (GLTEQ) was the most frequently used patient-reported measure. Psychometric properties were reported for 13 of these measures and most frequently for movement sensors. Two studies reported on properties of the GLTEQ. Quality ratings for risk of bias were low. There is significant heterogeneity amongst studies regarding method of PA measurement along the lung cancer continuum. Greater consensus could be achieved by using a consensus approach such as a Delphi process. Future studies should include assessment of psychometric properties of the measurement tool being used. Currently, it is recommended where feasible, both performance-based and patient-reported measurements of PA should be undertaken.

  17. The primary outcome measure and its importance in clinical trials.

    PubMed

    Andrade, Chittaranjan

    2015-10-01

    The primary outcome measure is the outcome that an investigator considers to be the most important among the many outcomes that are to be examined in the study. The primary outcome needs to be defined at the time the study is designed. There are 2 reasons for this: it reduces the risk of false-positive errors resulting from the statistical testing of many outcomes, and it reduces the risk of a false-negative error by providing the basis for the estimation of the sample size necessary for an adequately powered study. This article discusses the setting of the primary outcome measure, the need for it, the increased risk of false-positive and false-negative errors in secondary outcome results, how to regard articles that do not state the primary outcome, how to interpret results when secondary outcomes are statistically significant but not the primary outcome, and limitations of the concept of a primary outcome measure in clinical trial research.

  18. Residual Effects of Sleep Medications Are Commonly Reported and Associated with Impaired Patient-Reported Outcomes among Insomnia Patients in the United States

    PubMed Central

    Fitzgerald, Timothy; Vietri, Jeffrey

    2015-01-01

    Study Objective. To measure the association of symptoms attributed to residual effects of sleep medication (e.g., drowsiness, difficulty concentrating, and impaired memory) on self-reported functioning and satisfaction with these medications. Methods. Individuals using prescription medications for insomnia were invited to complete an Internet-based survey. Respondents were compared according to the presence of self-reported residual effects; relationships between severity of these effects and outcomes were modeled using regression. Measures included the Brief Insomnia Questionnaire, Work Productivity and Activity Impairment Questionnaire, and SATMED-Q. Subgroup analyses were conducted with patients aged ≥65 years. Approximately 80% reported experiencing ≥1 residual effect. The severity of residual effects was associated with increased residual effect-related work impairment, including absenteeism (RR = 1.46, p < 0.001), presenteeism (RR = 1.12, p < 0.001), overall work impairment (RR = 1.13, p < 0.001), and nonwork activity impairment (RR = 1.11, p < 0.001). More severe residual symptoms were also associated with increased difficulty in home management (Beta = .31, p < 0.001), ability to work (Beta = .31, p < 0.001), social relationships, (Beta = .32, p < 0.001), close personal relationships (Beta = .30, p < 0.001), and lower medication satisfaction (Beta = −.37, p < 0.001). Conclusions. Individuals using medications for insomnia commonly experience symptoms considered as residual effects, and these symptoms are associated with greater interference of sleep-related problems at work, at home, and with social relationships. PMID:26783470

  19. Patient-Reported Voice and Speech Outcomes After Whole-Neck Intensity Modulated Radiation Therapy and Chemotherapy for Oropharyngeal Cancer: Prospective Longitudinal Study

    SciTech Connect

    Vainshtein, Jeffrey M.; Griffith, Kent A.; Feng, Felix Y.; Vineberg, Karen A.; Chepeha, Douglas B.; Eisbruch, Avraham

    2014-08-01

    Purpose: To describe voice and speech quality changes and their predictors in patients with locally advanced oropharyngeal cancer treated on prospective clinical studies of organ-preserving chemotherapy–intensity modulated radiation therapy (chemo-IMRT). Methods and Materials: Ninety-one patients with stage III/IV oropharyngeal cancer were treated on 2 consecutive prospective studies of definitive chemoradiation using whole-field IMRT from 2003 to 2011. Patient-reported voice and speech quality were longitudinally assessed from before treatment through 24 months using the Communication Domain of the Head and Neck Quality of Life (HNQOL-C) instrument and the Speech question of the University of Washington Quality of Life (UWQOL-S) instrument, respectively. Factors associated with patient-reported voice quality worsening from baseline and speech impairment were assessed. Results: Voice quality decreased maximally at 1 month, with 68% and 41% of patients reporting worse HNQOL-C and UWQOL-S scores compared with before treatment, and improved thereafter, recovering to baseline by 12-18 months on average. In contrast, observer-rated larynx toxicity was rare (7% at 3 months; 5% at 6 months). Among patients with mean glottic larynx (GL) dose ≤20 Gy, >20-30 Gy, >30-40 Gy, >40-50 Gy, and >50 Gy, 10%, 32%, 25%, 30%, and 63%, respectively, reported worse voice quality at 12 months compared with before treatment (P=.011). Results for speech impairment were similar. Glottic larynx dose, N stage, neck dissection, oral cavity dose, and time since chemo-IMRT were univariately associated with either voice worsening or speech impairment. On multivariate analysis, mean GL dose remained independently predictive for both voice quality worsening (8.1%/Gy) and speech impairment (4.3%/Gy). Conclusions: Voice quality worsening and speech impairment after chemo-IMRT for locally advanced oropharyngeal cancer were frequently reported by patients, underrecognized by clinicians, and

  20. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2011 CFR

    2011-10-01

    ... 42 Public Health 2 2011-10-01 2011-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  1. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2013 CFR

    2013-10-01

    ... 42 Public Health 2 2013-10-01 2013-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  2. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2012 CFR

    2012-10-01

    ... 42 Public Health 2 2012-10-01 2012-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  3. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2014 CFR

    2014-10-01

    ... 42 Public Health 2 2014-10-01 2014-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  4. 42 CFR 410.146 - Diabetes outcome measurements.

    Code of Federal Regulations, 2010 CFR

    2010-10-01

    ... 42 Public Health 2 2010-10-01 2010-10-01 false Diabetes outcome measurements. 410.146 Section 410... MEDICARE PROGRAM SUPPLEMENTARY MEDICAL INSURANCE (SMI) BENEFITS Outpatient Diabetes Self-Management Training and Diabetes Outcome Measurements § 410.146 Diabetes outcome measurements. (a)...

  5. Applying Outcome Measurements: A Guide to Educational Outcome Measurements and Their Uses. Seminar No. 5.

    ERIC Educational Resources Information Center

    Glaser, Ezra

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministries, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with the application of outcome…

  6. The Electronic Patient Reported Outcome Tool: Testing Usability and Feasibility of a Mobile App and Portal to Support Care for Patients With Complex Chronic Disease and Disability in Primary Care Settings

    PubMed Central

    Gill, Ashlinder; Khan, Anum Irfan; Hans, Parminder Kaur; Kuluski, Kerry; Cott, Cheryl

    2016-01-01

    Background People experiencing complex chronic disease and disability (CCDD) face some of the greatest challenges of any patient population. Primary care providers find it difficult to manage multiple discordant conditions and symptoms and often complex social challenges experienced by these patients. The electronic Patient Reported Outcome (ePRO) tool is designed to overcome some of these challenges by supporting goal-oriented primary care delivery. Using the tool, patients and providers collaboratively develop health care goals on a portal linked to a mobile device to help patients and providers track progress between visits. Objectives This study tested the usability and feasibility of adopting the ePRO tool into a single interdisciplinary primary health care practice in Toronto, Canada. The Fit between Individuals, Fask, and Technology (FITT) framework was used to guide our assessment and explore whether the ePRO tool is: (1) feasible for adoption in interdisciplinary primary health care practices and (2) usable from both the patient and provider perspectives. This usability pilot is part of a broader user-centered design development strategy. Methods A 4-week pilot study was conducted in which patients and providers used the ePRO tool to develop health-related goals, which patients then monitored using a mobile device. Patients and providers collaboratively set goals using the system during an initial visit and had at least 1 follow-up visit at the end of the pilot to discuss progress. Focus groups and interviews were conducted with patients and providers to capture usability and feasibility measures. Data from the ePRO system were extracted to provide information regarding tool usage. Results Six providers and 11 patients participated in the study; 3 patients dropped out mainly owing to health issues. The remaining 8 patients completed 210 monitoring protocols, equal to over 1300 questions, with patients often answering questions daily. Providers and patients

  7. Measuring patient outcomes in breast augmentation: introducing the BREAST-Q Augmentation module.

    PubMed

    Pusic, Andrea L; Reavey, Patrick L; Klassen, Anne F; Scott, Amie; McCarthy, Colleen; Cano, Stefan J

    2009-01-01

    The Breast-Q Augmentation module is a new and unique questionnaire for measuring patient-reported outcomes following breast augmentation. It has undergone a rigorous development and validation process and is currently the only questionnaire for breast augmentation that meets international and federal standards for questionnaire development. The Breast-Q Augmentation module covers a comprehensive set of concerns of breast augmentation patients, including satisfaction with breasts and impact on quality of life. With its excellent psychometric properties, the Breast-Q Augmentation module can provide clinicians and researchers with a wealth of essential data to improve the field of breast augmentation from the perspectives of both surgeons and patients.

  8. A promising method for identifying cross-cultural differences in patient perspective: the use of Internet-based focus groups for content validation of new Patient Reported Outcome assessments

    PubMed Central

    Atkinson, Mark J; Lohs, Jan; Kuhagen, Ilka; Kaufman, Julie; Bhaidani, Shamsu

    2006-01-01

    Objectives This proof of concept (POC) study was designed to evaluate the use of an Internet-based bulletin board technology to aid parallel cross-cultural development of thematic content for a new set of patient-reported outcome measures (PROs). Methods The POC study, conducted in Germany and the United States, utilized Internet Focus Groups (IFGs) to assure the validity of new PRO items across the two cultures – all items were designed to assess the impact of excess facial oil on individuals' lives. The on-line IFG activities were modeled after traditional face-to-face focus groups and organized by a common 'Topic' Guide designed with input from thought leaders in dermatology and health outcomes research. The two sets of IFGs were professionally moderated in the native language of each country. IFG moderators coded the thematic content of transcripts, and a frequency analysis of code endorsement was used to identify areas of content similarity and difference between the two countries. Based on this information, draft PRO items were designed and a majority (80%) of the original participants returned to rate the relative importance of the newly designed questions. Findings The use of parallel cross-cultural content analysis of IFG transcripts permitted identification of the major content themes in each country as well as exploration of the possible reasons for any observed differences between the countries. Results from coded frequency counts and transcript reviews informed the design and wording of the test questions for the future PRO instrument(s). Subsequent ratings of item importance also deepened our understanding of potential areas of cross-cultural difference, differences that would be explored over the course of future validation studies involving these PROs. Conclusion The use of IFGs for cross-cultural content development received positive reviews from participants and was found to be both cost and time effective. The novel thematic coding methodology

  9. A multiple-domain framework of clinical, economic, and patient-reported outcomes for evaluating benefits of intervention in atopic dermatitis.

    PubMed

    Boguniewicz, Mark; Abramovits, William; Paller, Amy; Whitaker-Worth, Diane L; Prendergast, Mary; Cheng, J Wang; Wang, Patrick; Tong, Kuo B

    2007-04-01

    Atopic dermatitis (AD) increases health care utilization, affects patient quality of life, places a burden on caregivers, decreases patient/parent productivity, and adds to health care costs. Few studies have examined the effect of specific treatment modalities across a variety of AD-related outcomes. This prospective, multicenter, open-label longitudinal study of adult and pediatric patients with moderate to severe AD was conducted to evaluate the effect of a specific therapeutic intervention on AD-related outcomes over a period of 6 months. Surveys collected physician clinical assessments and patient- and caregiver-reported data across the following domains: clinical outcome, health care utilization/costs, quality of life, physical appearance, productivity/absenteeism, and medication compliance. This study is intended to help guide future research efforts on the net costs and benefits of different interventions across a diverse set of domains and in larger populations.

  10. Universal health outcome measures for older persons with multiple chronic conditions

    PubMed Central

    2012-01-01

    Older adults with multiple chronic conditions (MCC) require considerable health services and complex care. As health status is affected along multiple dimensions by the persistence and progression of diseases and courses of treatments, well-validated universal outcome measures across diseases are needed for research, clinical care and administrative purposes. An expert panel meeting held by the National Institute on Aging (NIA) in September 2011 recommends that older persons with MCC complete a brief initial composite measure that includes general health, pain, fatigue, and physical, mental health and social role function, along with gait speed measurement. Suitable composite measures include the Short-form 8 (SF-8) and 36 (SF-36) and the Patient Reported Outcomes Measurement Information System29-item Health Profile (PROMIS-29). Based on responses to items within the initial measure, short follow-on measures should be selectively targeted to the following areas: symptom burden, depression, anxiety and daily activities. Persons unable to walk a short distance for gait speed should be assessed using a physical function scale. Remaining gaps to be considered for measure development include disease burden, cognitive function and caregiver burden. Routine outcome assessment of MCC patients could facilitate system-based care improvement and clinical effectiveness research. PMID:23194184

  11. Linked Patient-Reported Outcomes Data From Patients With Multiple Sclerosis Recruited on an Open Internet Platform to Health Care Claims Databases Identifies a Representative Population for Real-Life Data Analysis in Multiple Sclerosis

    PubMed Central

    Ghodge, Bhaskar; Bonzani, Ian C; Korn, Jonathan R; Medin, Jennie; Saraykar, Tanmay; Sengupta, Souvik; Saini, Deepanshu; Olson, Melvin

    2016-01-01

    Background An enormous amount of information relevant to public health is being generated directly by online communities. Objective To explore the feasibility of creating a dataset that links patient-reported outcomes data, from a Web-based survey of US patients with multiple sclerosis (MS) recruited on open Internet platforms, to health care utilization information from health care claims databases. The dataset was generated by linkage analysis to a broader MS population in the United States using both pharmacy and medical claims data sources. Methods US Facebook users with an interest in MS were alerted to a patient-reported survey by targeted advertisements. Eligibility criteria were diagnosis of MS by a specialist (primary progressive, relapsing-remitting, or secondary progressive), ≥12-month history of disease, age 18-65 years, and commercial health insurance. Participants completed a questionnaire including data on demographic and disease characteristics, current and earlier therapies, relapses, disability, health-related quality of life, and employment status and productivity. A unique anonymous profile was generated for each survey respondent. Each anonymous profile was linked to a number of medical and pharmacy claims datasets in the United States. Linkage rates were assessed and survey respondents’ representativeness was evaluated based on differences in the distribution of characteristics between the linked survey population and the general MS population in the claims databases. Results The advertisement was placed on 1,063,973 Facebook users’ pages generating 68,674 clicks, 3719 survey attempts, and 651 successfully completed surveys, of which 440 could be linked to any of the claims databases for 2014 or 2015 (67.6% linkage rate). Overall, no significant differences were found between patients who were linked and not linked for educational status, ethnicity, current or prior disease-modifying therapy (DMT) treatment, or presence of a relapse in

  12. A Standard Set of Value-Based Patient-Centered Outcomes for Breast Cancer: The International Consortium for Health Outcomes Measurement (ICHOM) Initiative.

    PubMed

    Ong, Wee Loon; Schouwenburg, Maartje G; van Bommel, Annelotte C M; Stowell, Caleb; Allison, Kim H; Benn, Karen E; Browne, John P; Cooter, Rodney D; Delaney, Geoff P; Duhoux, Francois P; Ganz, Patricia A; Hancock, Patricia; Jagsi, Reshma; Knaul, Felicia M; Knip, Anne M; Koppert, Linetta B; Kuerer, Henry M; McLaughin, Sarah; Mureau, Marc A M; Partridge, Ann H; Reid, Dereesa Purtell; Sheeran, Lisa; Smith, Thomas J; Stoutjesdijk, Mark J; Vrancken Peeters, Marie Jeanne T F D; Wengström, Yvonne; Yip, Cheng-Har; Saunders, Christobel

    2016-12-29

    A major challenge in value-based health care is the lack of standardized health outcomes measurements, hindering optimal monitoring and comparison of the quality of health care across different settings globally. The International Consortium for Health Outcomes Measurement (ICHOM) assembled a multidisciplinary international working group, comprised of 26 health care providers and patient advocates, to develop a standard set of value-based patient-centered outcomes for breast cancer (BC). The working group convened via 8 teleconferences and completed a follow-up survey after each meeting. A modified 2-round Delphi method was used to achieve consensus on the outcomes and case-mix variables to be included. Patient focus group meetings (8 early or metastatic BC patients) and online anonymized surveys of 1225 multinational BC patients and survivors were also conducted to obtain patients' input. The standard set encompasses survival and cancer control, and disutility of care (eg, acute treatment complications) outcomes, to be collected through administrative data and/or clinical records. A combination of multiple patient-reported outcomes measurement (PROM) tools is recommended to capture long-term degree of health outcomes. Selected case-mix factors were recommended to be collected at baseline. The ICHOM will endeavor to achieve wide buy-in of this set and facilitate its implementation in routine clinical practice in various settings and institutions worldwide.

  13. Supporting Goal-Oriented Primary Health Care for Seniors with Complex Care Needs Using Mobile Technology: Evaluation and Implementation of the Health System Performance Research Network, Bridgepoint Electronic Patient Reported Outcome Tool

    PubMed Central

    Wodchis, Walter P; Upshur, Ross; Cott, Cheryl; McKinstry, Brian; Mercer, Stewart; Palen, Ted E; Ramsay, Tim; Thavorn, Kednapa

    2016-01-01

    Background Older adults experiencing multiple chronic illnesses are at high risk of hospitalization and health decline if they are unable to manage the significant challenges posed by their health conditions. Goal-oriented care approaches can provide better care for these complex patients, but clinicians find the process of ascertaining goals “too complex and too-time consuming,” and goals are often not agreed upon between complex patients and their providers. The electronic patient reported outcomes (ePRO) mobile app and portal offers an innovative approach to creating and monitoring goal-oriented patient-care plans to improve patient self-management and shared decision-making between patients and health care providers. The ePRO tool also supports proactive patient monitoring by the patient, caregiver(s), and health care provider. It was developed with and for older adults with complex care needs as a means to improve their quality of life. Objective Our proposed project will evaluate the use, effectiveness, and value for money of the ePRO tool in a 12-month multicenter, randomized controlled trial in Ontario; targeting individuals 65 or over with two or more chronic conditions that require frequent health care visits to manage their health conditions. Methods Intervention groups using the ePRO tool will be compared with control groups on measures of quality of life, patient experience, and cost-effectiveness. We will also evaluate the implementation of the tool. Results The proposed project presented in this paper will be funded through the Canadian Institute for Health Research (CIHR) eHealth Innovation Partnerships Program (eHIPP) program (CIHR–143559). The expected completion date of the study is November, 2019. Conclusions We anticipate our program of work will support improved quality of life and patient self-management, improved patient-centered primary care delivery, and will encourage the adoption of goal-oriented care approaches across primary

  14. A Review of Outcome Measures in Early Childhood Programs

    ERIC Educational Resources Information Center

    Mannan, Hasheem; Summers, Jean Ann; Turnbull, Ann P.; Poston, Denise J.

    2006-01-01

    The authors undertook a review of measures available for assessing outcomes of early childhood services for children with disabilities and their families. With principles of family-centered practice mandating the inclusion of both family and child outcome measures in effective evaluation plans, the review examined measures with established…

  15. Functional outcome measures for NF1-associated optic pathway glioma clinical trials

    PubMed Central

    Avery, Robert A.; Allen, Jeffrey C.; Ardern-Holmes, Simone L.; Bilaniuk, Larissa T.; Ferner, Rosalie E.; Gutmann, David H.; Listernick, Robert; Martin, Staci; Ullrich, Nicole J.; Liu, Grant T.

    2013-01-01

    Objective: The goal of the Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee is to define the best functional outcome measures for future neurofibromatosis type 1 (NF1)-associated optic pathway glioma (OPG) clinical trials. Methods: The committee considered the components of vision, other ophthalmologic parameters affected by OPG, potential biomarkers of visual function, and quality of life measures to arrive at consensus-based, evidence-driven recommendations for objective and measurable functional endpoints for OPG trials. Results: Visual acuity (VA) assessments using consistent quantitative testing methods are recommended as the main functional outcome measure for NF1-OPG clinical trials. Teller acuity cards are recommended for use as the primary VA endpoint, and HOTV as a secondary endpoint once subjects are old enough to complete it. The optic disc should be assessed for pallor, as this appears to be a contributory variable that may affect the interpretation of VA change over time. Given the importance of capturing patient-reported outcomes in clinical trials, evaluating visual quality of life using the Children's Visual Function Questionnaire as a secondary endpoint is also proposed. Conclusions: The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials. PMID:24249802

  16. Health Outcome after Major Trauma: What Are We Measuring?

    PubMed Central

    Hoffman, Karen; Cole, Elaine; Playford, E. Diane; Grill, Eva; Soberg, Helene L.; Brohi, Karim

    2014-01-01

    Importance Trauma is a global disease and is among the leading causes of disability in the world. The importance of outcome beyond trauma survival has been recognised over the last decade. Despite this there is no internationally agreed approach for assessment of health outcome and rehabilitation of trauma patients. Objective To systematically examine to what extent outcomes measures evaluate health outcomes in patients with major trauma. Data Sources MEDLINE, EMBASE, and CINAHL (from 2006–2012) were searched for studies evaluating health outcome after traumatic injuries. Study selection and data extraction Studies of adult patients with injuries involving at least two body areas or organ systems were included. Information on study design, outcome measures used, sample size and outcomes were extracted. The World Health Organisation International Classification of Function, Disability and Health (ICF) were used to evaluate to what extent outcome measures captured health impacts. Results 34 studies from 755 studies were included in the review. 38 outcome measures were identified. 21 outcome measures were used only once and only five were used in three or more studies. Only 6% of all possible health impacts were captured. Concepts related to activity and participation were the most represented but still only captured 12% of all possible concepts in this domain. Measures performed very poorly in capturing concepts related to body function (5%), functional activities (11%) and environmental factors (2%). Conclusion Outcome measures used in major trauma capture only a small proportion of health impacts. There is no inclusive classification for measuring disability or health outcome following trauma. The ICF may provide a useful framework for the development of a comprehensive health outcome measure for trauma care. PMID:25051353

  17. Comparative Effectiveness of Sphincter-Sparing Surgery versus Abdominoperineal Resection in Rectal Cancer: Patient-Reported Outcomes in National Surgical Adjuvant Breast and Bowel Project Randomized Trial R-04

    PubMed Central

    Russell, Marcia M.; Ganz, Patricia A.; Lopa, Samia; Yothers, Greg; Ko, Clifford Y.; Arora, Amit; Atkins, James N.; Bahary, Nathan; Soori, Gamini; Robertson, John M.; Eakle, Janice; Marchello, Benjamin T.; Wozniak, Timothy F.; Beart, Robert W.; Wolmark, Norman

    2015-01-01

    Objective NSABP R-04 was a randomized controlled trial of neoadjuvant chemoradiotherapy in patients with resectable stage II–III rectal cancer. We hypothesized that patients who underwent abdominoperineal resection (APR) would have a poorer quality of life than those who underwent sphincter-sparing surgery (SSS). Methods To obtain patient-reported outcomes (PROs) we administered two symptom scales at baseline and 1 year postoperatively: the Functional Assessment of Cancer Therapy-Colorectal (FACT-C) and the European Organization for the Research and Treatment of Cancer module for patients with Colorectal Cancer Quality of Life Questionnaire (EORTC QLQ-CR38). Scoring was stratified by non-randomly assigned definitive surgery (APR vs SSS). Analyses controlled for baseline scores and stratification factors: age, gender, stage, intended surgery, and randomly assigned chemoradiotherapy. Results Of 1,608 randomly assigned patients, 987 had data for planned analyses; 62% underwent SSS; 38% underwent APR. FACT-C total and subscale scores were not statistically different by surgery at one year. For the EORTC-QLQ-CR38 functional scales, APR patients reported worse body image (70.3 vs 77.0, P=0.0005) at one year than did SSS patients. Males undergoing APR reported worse sexual enjoyment (43.7 vs 54.7, P=0.02) at one year than did those undergoing SSS. For the EORTC-QLQ-CR38 symptom scale scores, APR patients reported worse micturition symptoms than the SSS group at one year (26.9 vs 21.5, P=0.03). SSS patients reported worse GI tract symptoms than did the APR patients (18.9 vs 15.2, P<0.0001), as well as weight loss (10.1 vs 6.0, P=0.002). Conclusions Symptoms and functional problems were detected at one year by EORTC-QLQ-CR38, reflecting different symptom profiles in patients who underwent APR than those who underwent SSS. Information from these PROs may be useful in counseling patients anticipating surgery for rectal cancer. PMID:24670844

  18. Stakeholders' views on measuring outcomes for people with learning disabilities.

    PubMed

    Young, Anita F; Chesson, Rosemary A

    2006-01-01

    What works and how do we know? These are recurring questions for health and social care professionals, although mediated through differing philosophies and historical perspectives. The aims of the study reported here were to discover views of managers and commissioners of services for people with learning disabilities in Scotland regarding (a) current approaches to service evaluation (as an indication of what is to be measured) and (b) healthcare outcome measurement (as an indication of preferences regarding how this should be measured). A postal questionnaire was used to survey 94 stakeholders from the NHS, Local Authorities, and non-statutory organisations across Scotland. Respondents' views were sought on current approaches to service evaluation within learning disabilities; outcome measurement; appropriateness of specified methods of measuring health outcomes; desired future methods of outcome measurement within learning disabilities; and service user involvement in care. A 77% (73/94) response rate to the questionnaire was achieved. Different methods of service evaluation were used by different stakeholders. Staff appraisal was the most frequently identified method (used by 85% of respondents). Specific outcome measures were used by 32% of respondents although there were differences of opinion as to what constitutes specific outcome measures. Overall there was strong support for goal-setting and reviewing (83%) and individualised outcome measures (75%) as appropriate methods for use with people with learning disabilities. The hypothetical question asking what outcome measures should be introduced for this client group had by far the lowest response rate (51/73). The overwhelming majority of all respondents, 68 (92%), reported user involvement in their service. Staff ambivalence to outcome measurement was evident in the research and respondents highlighted the complexity and multidimensional nature of outcomes for this service user group. Managers recognised

  19. [Outcomes count: the importance of measuring the outcomes of hospital care].

    PubMed

    Sangha, Oliver; Schneeweiss, Sebastian

    2002-01-01

    A century ago the Boston surgeon Earnest A. Codman described in detail the requirements for monitoring quality of care and emphasized the importance of outcomes in evaluating care. At the time the medical societies were disconcerted by his ideas, which were perceived as revolutionary. After several decade of focusing on the structures and processes of care we are now witnessing a renaissance of measuring outcomes. This paper emphasizes the need for outcomes measurement monitor quality of care. The introduction of diagnosis-related groups in Germany is the most recent development that underlines the importance of outcomes measurement and benchmarking.

  20. Psychometric evaluation of self-report outcome measures for prosthetic applications

    PubMed Central

    Hafner, Brian J.; Morgan, Sara J.; Askew, Robert L.; Salem, Rana

    2016-01-01

    Documentation of clinical outcomes is increasingly expected in delivery of prosthetic services and devices. However, many outcome measures suitable for use in clinical care and research have not been psychometrically tested with prosthesis users. The aim of this study was to determine test-retest reliability, mode-of-administration (MoA) equivalence, standard error of measurement (SEM), and minimal detectable change (MDC) of standardized, self-report instruments that assess constructs of importance to people with lower limb loss. Prosthesis users (n=201) were randomly assigned to groups based on MoA (i.e., paper, electronic, or mixed-mode). Participants completed two surveys 2-3 days apart. Instruments included the Prosthetic Limb Users Survey of Mobility, Prosthesis Evaluation Questionnaire–Mobility Subscale, Activities-Specific Balance Confidence Scale, Quality of Life in Neurological Conditions–Applied Cognition/General Concerns, Patient Reported Outcomes Measurement Information System Profile, and Socket Comfort Score. Intraclass correlation coefficients indicated all instruments are appropriate for group-level comparisons and select instruments are suitable for individual-level applications. Several instruments showed evidence of possible floor and ceiling effects. All were equivalent across MoAs. SEM and MDC were quantified to facilitate interpretation of outcomes and change scores. These results can enhance clinicians' and researchers' ability to select, apply, and interpret scores from instruments administered to prosthesis users. PMID:28273329

  1. MEASURING AND IMPROVING RESPIRATORY OUTCOMES IN CYSTIC FIBROSIS LUNG DISEASE: OPPORTUNITIES AND CHALLENGES TO THERAPY

    PubMed Central

    Zemanick, Edith T.; Harris, J. Kirk; Conway, Steven; Konstan, Michael W.; Marshall, Bruce; Quittner, Alexandra L.; Retsch-Bogart, George; Saiman, Lisa; Accurso, Frank J.

    2010-01-01

    Cystic fibrosis (CF) is a life-shortening disease with significant morbidity. Despite overall improvements in survival, patients with CF experience frequent pulmonary exacerbations and declining lung function, which often accelerates during adolescence. New treatments target steps in the pathogenesis of lung disease, such as the basic defect in CF (CF Transmembrane Conductance Regulator [CFTR]), pulmonary infections, inflammation, and mucociliary clearance. These treatments offer hope but also present challenges to patients, clinicians, and researchers. Comprehensive assessment of efficacy is critical to identify potentially beneficial treatments. Lung function and pulmonary exacerbation are the most commonly used outcome measures in CF clinical research. Other outcome measures under investigation include measures of CFTR function; biomarkers of infection, inflammation, lung injury and repair; and patient-reported outcomes. Molecular diagnostics may help elucidate the complex CF airway microbiome. As new treatments are developed for patients with CF, efforts should be made to balance treatment burden with quality of life. This review highlights emerging treatments, obstacles to optimizing outcomes, and key future directions for research. PMID:19833563

  2. Arthrodesis Rate and Patient Reported Outcomes After Anterior Lumbar Interbody Fusion Utilizing a Plasma-Sprayed Titanium Coated PEEK Interbody Implant: A Retrospective, Observational Analysis

    PubMed Central

    Bergen, Sophea R.; Staples, Miranda; Liang, Kevin; Raiszadeh, Ramin

    2017-01-01

    to a zero-profile PEEK lumbar interbody spacer facilitates rapid and stable fixation at the bone-implant interface. This facilitated osteogenesis is associated with significantly improved pain outcomes, low implant subsidence and a high definitive rate of arthrodesis. Future studies should include a prospective, randomized, controlled, multi-center approach to directly compare arthrodesis rates and clinical outcomes longitudinally between standard PEEK and biomaterial-coated PEEK interbody spacer systems. PMID:28377862

  3. Recommendations for a first Core Outcome Measurement set for complex regional PAin syndrome Clinical sTudies (COMPACT).

    PubMed

    Sharon, Grieve; Roberto Sgm, Perez; Frank, Birklein; Florian, Brunner; Stephen, Bruehl; Norman, Harden R; Tara, Packham; Francois, Gobeil; Richard, Haigh; Janet, Holly; Astrid, Terkelsen; Lindsay, Davies; Jennifer, Lewis; Ilona, Thomassen; Robyn, Connett; Tina, Worth; Jean-Jacques, Vatine; S, McCabe Candida

    2017-02-04

    Complex Regional Pain Syndrome (CRPS) is a persistent pain condition that remains incompletely understood and challenging to treat. Historically, a wide range of different outcome measures have been used to capture the multidimensional nature of CRPS. This has been a significant limiting factor in the advancement of our understanding of the mechanisms and management of CRPS.In 2013, an international consortium of patients, clinicians, researchers and industry representatives was established, to develop and agree on a minimum core set of standardised outcome measures for use in future CRPS clinical research, including but not limited to clinical trials within adult populationsThe development of a core measurement set was informed through workshops and supplementary work, using an iterative consensus process. 'What is the clinical presentation and course of CRPS, and what factors influence it?' was agreed as the most pertinent research question that our standardised set of patient-reported outcome measures should be selected to answer. The domains encompassing the key concepts necessary to answer the research question were agreed as: pain, disease severity, participation and physical function, emotional and psychological function, self efficacy, catastrophizing and patient's global impression of change. The final core measurement set included the optimum generic or condition-specific patient-reported questionnaire outcome measures, which captured the essence of each domain, and one clinician reported outcome measure to capture the degree of severity of CRPS. The next step is to test the feasibility and acceptability of collecting outcome measure data using the core measurement set in the CRPS population internationally.

  4. Learning Outcomes across Disciplines and Professions: Measurement and Interpretation

    ERIC Educational Resources Information Center

    Caspersen, Joakim; Frølich, Nicoline; Karlsen, Hilde; Aamodt, Per Olaf

    2014-01-01

    Learning outcomes of higher education are a quality tool in a changing higher education landscape but cannot be seen as neutral measures across professions and disciplines. Survey results from graduates and recent graduates indicate that prevailing measures of learning outcomes yield the same result within and across disciplinary and professional…

  5. Clinically Meaningful Differences in Patient-Reported Outcomes With Amifostine in Combination With Chemoradiation for Locally Advanced Non-Small-Cell Lung Cancer: An Analysis of RTOG 9801

    SciTech Connect

    Sarna, Linda Swann, Suzanne; Langer, Corey; Werner-Wasik, Maria; Nicolaou, Nicos; Komaki, Ritsuko; Machtay, Mitchell; Byhardt, Roger; Wasserman, Todd; Movsas, Benjamin

    2008-12-01

    Purpose: The purpose of this study is to analyze changes in quality of life (QOL) and symptoms from pretreatment to 6 weeks posttreatment in a Phase III randomized study (Radiation Therapy Oncology Group 9801) of amifostine (AM) vs. no AM in patients with Stages II-III non-small-cell lung cancer receiving paclitaxel and carboplatin as induction and then concurrently with hyperfractionated radiation therapy (RT). Methods and Materials: One hundred thirty-eight patients with baseline and 6-week posttreatment QOL data were analyzed. There were no significant differences in baseline demographics between those who did and did not have QOL data. The QOL and symptoms were assessed by using the European Organization for Research and Treatment of Cancer (EORTC) Global QOL and Pain subscales and the EORTC-Lung Cancer-13 symptom tool. Clinically relevant changes in QOL were characterized by 10-point differences in individual scores pre/post treatment. A daily diary of patient-rated difficulty swallowing and a weekly physician-rated dysphagia log (using National Cancer Institute Common Toxicity Criteria) were completed during treatment. Weight loss was monitored. Differences in outcomes were examined according to smoking status, alcohol use, and sex. Results: Patients receiving AM reported significantly greater pain reduction after chemoradiation (34% vs. no AM, 21%), less difficulty swallowing during chemoradiation, and less weight loss than patients not receiving AM. However, physician-rated assessments of dysphagia were not significantly different by treatment arm. There were no other significant changes in QOL or symptoms according to treatment arm, smoking status, alcohol use, or sex. Conclusions: Patient evaluations of difficulty swallowing and pain suggest benefits from AM use that are distinct from clinician-rated assessments.

  6. Information perception, wishes, and satisfaction in ambulatory cancer patients under active treatment: patient-reported outcomes with QLQ-INFO25

    PubMed Central

    Pinto, Ana Catarina; Ferreira-Santos, Fernando; Lago, Lissandra Dal; de Azambuja, Evandro; Pimentel, Francisco Luís; Piccart-Gebhart, Martine; Razavi, Darius

    2014-01-01

    Background Information is vital to cancer patients. Physician–patient communication in oncology presents specific challenges. The aim of this study was to evaluate self-reported information of cancer patients in ambulatory care at a comprehensive cancer centre and examine its possible association with patients’ demographic and clinical characteristics. Patients and methods This study included adult patients with solid tumours undergoing chemotherapy at the Institute Jules Bordet’s Day Hospital over a ten-day period. EORTC QLQ-C30 and QLQ-INFO25 questionnaires were administered. Demographic and clinical data were collected. Descriptive and inferential statistics were used. Results 101 (99%) fully completed the questionnaires. They were mostly Belgian (74.3%), female (78.2%), with a mean age of 56.9 ± 12.8 years. The most frequent tumour was breast cancer (58.4%). Patients were well-informed about the disease and treatments, but presented unmet information domains. The Jules Bordet patients desired more information on treatment side effects, long-term outcome, nutrition, and recurrence symptoms. Patients on clinical trials reported having received less information about their disease and less written information than patients outside clinical trials. Higher information levels were associated with higher quality of life (QoL) scores and higher patient satisfaction. Conclusion Patients were satisfied with the information they received and this correlated with higher QoL, but they still expressed unmet information wishes. Additional studies are required to investigate the quality of the information received by patients enrolled in clinical trials. PMID:24834120

  7. Toxicities Affecting Quality of Life After Chemo-IMRT of Oropharyngeal Cancer: Prospective Study of Patient-Reported, Observer-Rated, and Objective Outcomes

    SciTech Connect

    Hunter, Klaudia U.; Schipper, Matthew; Feng, Felix Y.; Lyden, Teresa; Haxer, Mark; Murdoch-Kinch, Carol-Anne; Cornwall, Benjamin; Lee, Connie S.Y.; Chepeha, Douglas B.; Eisbruch, Avraham

    2013-03-15

    Purpose: To test the hypothesis that intensity modulated radiation therapy (IMRT) aiming to spare the salivary glands and swallowing structures would reduce or eliminate the effects of xerostomia and dysphagia on quality of life (QOL). Methods and Materials: In this prospective, longitudinal study, 72 patients with stage III-IV oropharyngeal cancer were treated uniformly with definitive chemo-IMRT sparing the salivary glands and swallowing structures. Overall QOL was assessed by summary scores of the Head Neck QOL (HNQOL) and University of Washington QOL (UWQOL) questionnaires, as well as the HNQOL “Overall Bother” question. Quality of life, observer-rated toxicities (Common Toxicity Criteria Adverse Effects scale, version 2), and objective evaluations (videofluoroscopy assessing dysphagia and saliva flow rates assessing xerostomia) were recorded from before therapy through 2 years after therapy. Correlations between toxicities/objective evaluations and overall QOL were assessed using longitudinal repeated measures of analysis and Pearson correlations. Results: All observer-rated toxicities and QOL scores worsened 1-3 months after therapy and improved through 12 months, with minor further improvements through 24 months. At 12 months, dysphagia grades 0-1, 2, and 3, were observed in 95%, 4%, and 1% of patients, respectively. Using all posttherapy observations, observer-rated dysphagia was highly correlated with all overall QOL measures (P<.0001), whereas xerostomia and mucosal and voice toxicities were significantly correlated with some, but not all, overall QOL measures, with lower correlation coefficients than dysphagia. Late overall QOL (≥6 or ≥12 months after therapy) was primarily associated with observer-rated dysphagia, and to a lesser extent with xerostomia. Videofluoroscopy scores, but not salivary flows, were significantly correlated with some of the overall QOL measures. Conclusion: After chemo-IMRT, although late dysphagia was on average mild

  8. Gastrointestinal (GI)-Specific patient reported outcomes instruments differentiate between renal transplant patients with or without GI symptoms: results from a South American cohort

    PubMed Central

    Machnicki, Gerardo; Pefaur, Jacqueline; Gaite, Luis; Linchenco, Ana M; Raimondi, Clemente; Schiavelli, Ruben; Otero, Alcira; Margolis, Mary Kay

    2008-01-01

    Background Immunosuppressive therapies have burdensome side effects which may lead to sub-therapeutic dosing and non-compliance. Patients on different immunosuppressant regimens may feel less bothered by Gastrointestinal (GI) side effects or report better health-related quality of life (HRQL). We evaluated the reliability and validity of two GI-specific outcome instruments (Gastrointestinal Symptom Rating Scale (GSRS; higher scores = increased severity) and Gastrointestinal Quality of Life Index (GIQLI; higher scores = better GI-specific HRQL)) in renal transplant patients in South America. Methods Data from 5 South American centers participating in an international, longitudinal, observational study were analyzed. Patients were ≥ 1 month post transplant and on mycophenolate mofetil (MMF) and a calcineurin inhibitor. Patients completed the GSRS, GIQLI, and Psychological General Well-Being (PGWB; higher scores = better HRQL) Index at baseline and at 4–6 weeks. Internal consistency, test-retest reliability and construct and discriminant validity were assessed. Results Sixty-two participants were enrolled. Mean age was 42 years; mean time since transplant was 3.3 years; 57% were male; 65% received a deceased organ transplant and 68%had GI events. The GSRS and GIQLI demonstrated high internal consistency (Cronbach's alphas 0.72–0.96). Test-retest reliability was adequate (intraclass correlation coefficient > 0.6) for all GIQLI subscales and all GSRS subscales except Diarrhea and Reflux syndrome. Correlations between the GSRS and PGWB were moderate (range: -0.21 to -0.53, all p < 0.001 except 6 correlations with p < 0.05); correlations between the GIQLI and PGWB were higher (range: 0.36 to 0.71 p < 0.001), indicating good construct validity. The GSRS and GIQLI demonstrated good discriminant validity, as they clinically and statistically distinguished between patients with and without GI complaints and among patients with varying GI complication severity. Patients

  9. Generating Outcome Measurements: Economic and Societal. A Guide to Educational Outcomes Measurements and Their Uses. Seminar No. 4.

    ERIC Educational Resources Information Center

    Mushkin, Selma J.; Billings, Bradley B.

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministires, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with generating outcome measurements:…

  10. Progress towards a core set of outcome measures in small-vessel vasculitis. Report from OMERACT 9.

    PubMed

    Merkel, Peter A; Herlyn, Karen; Mahr, Alfred D; Neogi, Tuhina; Seo, Philip; Walsh, Michael; Boers, Maarten; Luqmani, Raashid

    2009-10-01

    The past decade has seen a substantial increase in the number and quality of clinical trials of new therapies for vasculitis, including randomized, controlled, multicenter trials that have successfully incorporated measures of disease activity and toxicity. However, because current treatment regimens for severe disease effectively induce initial remission and reduce mortality, future trials will focus on any of several goals including: (a) treatment of mild-moderate disease; (b) prevention of chronic damage; (c) reduction in treatment toxicity; or (d) more subtle differences in remission induction or maintenance. Thus, new trials will require outcome measure instruments that are more precise and are better able to detect effective treatments for different disease states and measure chronic manifestations of disease. The OMERACT Vasculitis Working Group comprises international clinical investigators with expertise in vasculitis who, since 2002, have worked collaboratively to advance the refinement of outcome measures in vasculitis, create new measures to address domains of illness not covered by current research approaches, and harmonize outcome assessment in vasculitis. The focus of the OMERACT group to date has been on outcome measures in small-vessel vasculitis with an overall goal of creating a core set of outcome measures for vasculitis, each of which fulfills the OMERACT filter of truth, discrimination, feasibility, and identifying additional domains requiring further research. This process has been informed by several ongoing projects providing data on outcomes of disease activity, disease-related damage, multidimensional health-related quality of life, and patient-reported ratings of the burden of vasculitis.

  11. Patient-reported outcomes among patients with type 2 diabetes mellitus treated with dapagliflozin in a triple-therapy regimen for 52 weeks.

    PubMed

    Grandy, S; Sternhufvud, C; Ryden, A; Sugg, J; Rohwedder, K

    2016-03-01

    Patients with type 2 diabetes (T2DM) and inadequate glycaemic control on combination metformin (MET) and sulphonylurea (SU) were enrolled in a 24-week, double-blind, randomized, placebo-controlled study with a 28-week extension. The five-dimension EuroQol questionnaire (EQ-5D), SHIELD Weight Questionnaire-9 (WQ-9), Impact of Weight on Quality of Life-Lite (IWQOL-Lite) questionnaire and the Diabetes Treatment Satisfaction Questionnaire (DTSQ) were used to evaluate health status and health-related quality of life (HRQoL) at baseline and week 52. Patients with dapagliflozin 10 mg + MET + SU (n = 108) were compared with patients treated with placebo + MET + SU (n = 108), using a repeated-measures mixed model. EQ-5D visual analogue scale scores, IWQOL-Lite and DTSQ scores improved in the dapagliflozin and placebo groups from baseline to week 52; however, there was no significant difference between groups (p > 0.20). EQ-5D index scores remained the same from baseline to week 52 for dapagliflozin and placebo (p = 0.54). A numerically greater proportion of the dapagliflozin group reported improvement in all nine SHIELD WQ-9 items compared with placebo, and the difference was statistically significant for physical health (p = 0.017). Over 52 weeks of therapy, patients maintained their health status and HRQoL when dapagliflozin was added to the treatment.

  12. Treatment for Osteoporosis among Women in Japan: Associations with Patient Characteristics and Patient-Reported Outcomes in the 2008–2011 Japan National Health and Wellness Surveys

    PubMed Central

    Flynn, Jennifer A.; Fujiwara, Saeko

    2014-01-01

    This study was conducted to identify characteristics associated with treatment for osteoporosis among women aged 50 years and older in Japan and to explore differences among patients according to treatment regimen. Data were provided by a large annual survey representative of Japanese aged 18 and older; all measures were by self-report. Women aged 50 and older who reported diagnosed osteoporosis (N = 900) were compared based on current treatment status using bivariate statistics and logistic regression. Approximately 1 in 3 women in this study reporting diagnosed osteoporosis were currently untreated. Factors associated with current treatment for osteoporosis included having ≥1 physician visit in the prior 6 months (OR = 5.4, P < 0.001), self-rated moderate or severe osteoporosis (OR = 2.8, P < 0.001), completion of menopause (OR = 1.6, P < 0.05), and family history of osteoporosis (OR = 1.5, P < 0.05), while longer duration of osteoporosis diagnosis (OR = 0.9, P < 0.05) and arthritis (OR = 0.7, P < 0.05) were associated with lower odds of treatment. These findings suggest that diagnosed patients are not being actively managed in the longer term, and efforts need to be made to ensure that patients stay engaged with their healthcare providers. PMID:25587485

  13. Effects of betahistine on patient-reported outcomes in routine practice in patients with vestibular vertigo and appraisal of tolerability: experience in the OSVaLD study.

    PubMed

    Benecke, Heike; Pérez-Garrigues, Herminio; Bin Sidek, Dinsuhaimi; Uloziene, Ingrida; D, Kuessner; Sondag, Eric; Theeuwes, Ad

    2010-01-01

    This was a 3-month multicentre, open-label post-marketing surveillance study of betahistine (24 mg b.i.d. or 16 mg t.i.d.) in patients with vertigo of peripheral vestibular origin. Study endpoints comprised on-treatment changes in the Dizziness Handicap Index (DHI), Hospital Anxiety and Depression Score (HADS) and the Short-Form (SF)-36v2. Total DHI score improved 37.2 points (of a 100-point scale) following betahistine treatment. Corresponding improvements occurred in all three DHI scale domains (all p < 0.001 vs baseline). Betahistine therapy was also accompanied by progressive, significant improvements in both HADS-A and HADS-D scores (p < 0.001), and improvements in the distribution profiles of anxiety and depression scores. Significant improvements in the Physical Component Summary and Mental Component Summary scores of the SF-36v2 were recorded during betahistine treatment. Betahistine was generally well tolerated. A total of 76 adverse drug reactions (ADRs) were recorded in 49 patients (2.4%), of which 75 were classified as mild or moderate and 54 were possibly related to betahistine. ADRs led to study drug discontinuation in 17 patients. These data illustrate that treatment with betahistine 48 mg/day in patients with recurrent peripheral vestibular vertigo is associated with improvements in objective measures of health-related quality of life and satisfactory tolerability.

  14. The Harmonizing Outcome Measures for Eczema (HOME) roadmap: a methodological framework to develop core sets of outcome measurements in dermatology.

    PubMed

    Schmitt, Jochen; Apfelbacher, Christian; Spuls, Phyllis I; Thomas, Kim S; Simpson, Eric L; Furue, Masutaka; Chalmers, Joanne; Williams, Hywel C

    2015-01-01

    Core outcome sets (COSs) are consensus-derived minimum sets of outcomes to be assessed in a specific situation. COSs are being increasingly developed to limit outcome-reporting bias, allow comparisons across trials, and strengthen clinical decision making. Despite the increasing interest in outcomes research, methods to develop COSs have not yet been standardized. The aim of this paper is to present the Harmonizing Outcomes Measures for Eczema (HOME) roadmap for the development and implementation of COSs, which was developed on the basis of our experience in the standardization of outcome measurements for atopic eczema. Following the establishment of a panel representing all relevant stakeholders and a research team experienced in outcomes research, the scope and setting of the core set should be defined. The next steps are the definition of a core set of outcome domains such as symptoms or quality of life, followed by the identification or development and validation of appropriate outcome measurement instruments to measure these core domains. Finally, the consented COS needs to be disseminated, implemented, and reviewed. We believe that the HOME roadmap is a useful methodological framework to develop COSs in dermatology, with the ultimate goal of better decision making and promoting patient-centered health care.

  15. A Binomial Test of Group Differences with Correlated Outcome Measures

    ERIC Educational Resources Information Center

    Onwuegbuzie, Anthony J.; Levin, Joel R.; Ferron, John M.

    2011-01-01

    Building on previous arguments for why educational researchers should not provide effect-size estimates in the face of statistically nonsignificant outcomes (Robinson & Levin, 1997), Onwuegbuzie and Levin (2005) proposed a 3-step statistical approach for assessing group differences when multiple outcome measures are individually analyzed…

  16. Measuring Assistive Technology Outcomes in Schools Using Functional Assessment.

    ERIC Educational Resources Information Center

    Silverman, Michelle Kaye; Stratman, Kristine Freiberg; Smith, Roger O.

    2000-01-01

    Activities of Project OATS (Outcomes of Assistive Technology in the Schools) are described, including identification and piloting of existing assessment instruments for use as an outcome measure, examining the validity of the School Function Assessment, and field testing the School Function Assessment-Assistive Technology Version, an adaptation of…

  17. The Development of NOAA Education Common Outcome Performance Measures (Invited)

    NASA Astrophysics Data System (ADS)

    Baek, J.

    2013-12-01

    The National Oceanic and Atmospheric Administration (NOAA) Education Council has embarked on an ambitious Monitoring and Evaluation (M&E) project that will allow it to assess education program outcomes and impacts across the agency, line offices, and programs. The purpose of this internal effort is to link outcome measures to program efforts and to evaluate the success of the agency's education programs in meeting the strategic goals. Using an outcome-based evaluation approach, the NOAA Education Council is developing two sets of common outcome performance measures, environmental stewardship and professional development. This presentation will examine the benefits and tradeoffs of common outcome performance measures that collect program results across a portfolio of education programs focused on common outcomes. Common outcome performance measures have a few benefits to our agency and to the climate education field at large. The primary benefit is shared understanding, which comes from our process for writing common outcome performance measures. Without a shared and agreed upon set of definitions for the measure of an outcome, the reported results may not be measuring the same things and would incorrectly indicate levels of performance. Therefore, our writing process relies on a commitment to developing a shared set of definitions based on consensus. We hope that by taking the time to debate and coming to agreement across a diverse set of programs, the strength of our common measures can indicate real progress towards outcomes we care about. An additional benefit is that these common measures can be adopted and adapted by other agencies and organizations that share similar theories of change. The measures are not without their drawbacks, and we do make tradeoffs as part of our process in order to continue making progress. We know that any measure is necessarily a narrow slice of performance. A slice that may not best represent the unique and remarkable contribution

  18. Identifying meaningful outcome measures for the intensive care unit.

    PubMed

    Martinez, Elizabeth A; Donelan, Karen; Henneman, Justin P; Berenholtz, Sean M; Miralles, Paola D; Krug, Allison E; Iezzoni, Lisa I; Charnin, Jonathan E; Pronovost, Peter J

    2014-01-01

    Despite important progress in measuring the safety of health care delivery in a variety of health care settings, a comprehensive set of metrics for benchmarking is still lacking, especially for patient outcomes. Even in high-risk settings where similar procedures are performed daily, such as hospital intensive care units (ICUs), these measures largely do not exist. Yet we cannot compare safety or quality across institutions or regions, nor can we track whether safety is improving over time. To a large extent, ICU outcome measures deemed valid, important, and preventable by clinicians are unavailable, and abstracting clinical data from the medical record is excessively burdensome. Even if a set of outcomes garnered consensus, ensuring adequate risk adjustment to facilitate fair comparisons across institutions presents another challenge. This study reports on a consensus process to build 5 outcome measures for broad use to evaluate the quality of ICU care and inform quality improvement efforts.

  19. Measuring family planning sustainability at the outcome and programme levels.

    PubMed

    Stephenson, Rob; Tsui, Amy Ong; Knight, Rodney

    2004-03-01

    The paper examines the validity of two indices of sustainability: family planning programme sustainability (PSI) and outcome sustainability (OSI) developed by Tsui and Knight (1997) by applying their original method to recent data. The indices succeed in identifying the directional path of programme and outcome sustainability. Close correlations are found between PSI and OSI predicted values and actual programme and outcome values. The indices provide a repeatable method for measuring sustainability, although they are sensitive to data measurement errors. The indices provide a policy tool for funding decisions but should be used with other data sources to judge sustainability.

  20. Validation of electronic systems to collect patient-reported outcome (PRO) data-recommendations for clinical trial teams: report of the ISPOR ePRO systems validation good research practices task force.

    PubMed

    Zbrozek, Arthur; Hebert, Joy; Gogates, Gregory; Thorell, Rod; Dell, Christopher; Molsen, Elizabeth; Craig, Gretchen; Grice, Kenneth; Kern, Scottie; Hines, Sheldon

    2013-06-01

    Outcomes research literature has many examples of high-quality, reliable patient-reported outcome (PRO) data entered directly by electronic means, ePRO, compared to data entered from original results on paper. Clinical trial managers are increasingly using ePRO data collection for PRO-based end points. Regulatory review dictates the rules to follow with ePRO data collection for medical label claims. A critical component for regulatory compliance is evidence of the validation of these electronic data collection systems. Validation of electronic systems is a process versus a focused activity that finishes at a single point in time. Eight steps need to be described and undertaken to qualify the validation of the data collection software in its target environment: requirements definition, design, coding, testing, tracing, user acceptance testing, installation and configuration, and decommissioning. These elements are consistent with recent regulatory guidance for systems validation. This report was written to explain how the validation process works for sponsors, trial teams, and other users of electronic data collection devices responsible for verifying the quality of the data entered into relational databases from such devices. It is a guide on the requirements and documentation needed from a data collection systems provider to demonstrate systems validation. It is a practical source of information for study teams to ensure that ePRO providers are using system validation and implementation processes that will ensure the systems and services: operate reliably when in practical use; produce accurate and complete data and data files; support management control and comply with any existing regulations. Furthermore, this short report will increase user understanding of the requirements for a technology review leading to more informed and balanced recommendations or decisions on electronic data collection methods.

  1. Measuring and communicating meaningful outcomes in neonatology: A family perspective.

    PubMed

    Janvier, Annie; Farlow, Barbara; Baardsnes, Jason; Pearce, Rebecca; Barrington, Keith J

    2016-12-01

    Medium- and long-term outcomes have been collected and described among survivors of neonatal intensive care units for decades, for a number of purposes: (1) quality control within units, (2) comparisons of outcomes between NICUs, (3) clinical trials (whether an intervention improves outcomes), (4) end-of-life decision-making, (5) to better understand the effects of neonatal conditions and/or interventions on organs and/or long-term health, and finally (6) to better prepare parents for the future. However, the outcomes evaluated have been selected by investigators, based on feasibility, availability, cost, stability, and on what investigators consider to be important. Many of the routinely measured outcomes have major limitations: they may not correlate well with long-term difficulties, they may artificially divide continuous outcomes into dichotomous ones, and may have no clear relationship with quality of life and functioning of children and their families. Several investigations, such as routine term cerebral resonance imaging for preterm infants, have also not yet been shown to improve the outcome of children nor their families. In this article, the most common variables used in neonatology as well as some variables which are rarely measured but may be of equal importance for families are presented. The manner in which these outcomes are communicated to families will be examined, as well as recommendations to optimize communication with parents.

  2. Routine outcome measures in Norway: Only partly implemented.

    PubMed

    Ruud, Torleif

    2015-01-01

    Norway has not had any strategy exclusively for the implementation of routine outcome measurement in the mental health services, but some efforts have been made as part of strategies for a national patient register and quality indicators. Fifteen years after the decision to make the rating of the Global Assessment of Functioning scale (GAF) mandatory at admission and discharge of each treatment episode in adult mental health services, this is still not fully implemented. An unknown and probably very low proportion of mental health services use GAF as a routine outcome measure in everyday clinical practice. Well-established electronic patient records in the mental health services and established procedures for reporting routine data to the National Patient Register should make it possible to collect and use routine outcome data. Implementation of routine outcome measurement in mental health services must be done with due emphasis on the critical steps in the various phases of the implementation process. The regional health authorities have a key role in establishing electronic systems that make relevant outcome measurements available in a seamless way for clinicians as well as for patients, and by contributing to a culture where quality and outcome are valued and given priority.

  3. Outcome Measures for Artificial Pancreas Clinical Trials: A Consensus Report.

    PubMed

    Maahs, David M; Buckingham, Bruce A; Castle, Jessica R; Cinar, Ali; Damiano, Edward R; Dassau, Eyal; DeVries, J Hans; Doyle, Francis J; Griffen, Steven C; Haidar, Ahmad; Heinemann, Lutz; Hovorka, Roman; Jones, Timothy W; Kollman, Craig; Kovatchev, Boris; Levy, Brian L; Nimri, Revital; O'Neal, David N; Philip, Moshe; Renard, Eric; Russell, Steven J; Weinzimer, Stuart A; Zisser, Howard; Lum, John W

    2016-07-01

    Research on and commercial development of the artificial pancreas (AP) continue to progress rapidly, and the AP promises to become a part of clinical care. In this report, members of the JDRF Artificial Pancreas Project Consortium in collaboration with the wider AP community 1) advocate for the use of continuous glucose monitoring glucose metrics as outcome measures in AP trials, in addition to HbA1c, and 2) identify a short set of basic, easily interpreted outcome measures to be reported in AP studies whenever feasible. Consensus on a broader range of measures remains challenging; therefore, reporting of additional metrics is encouraged as appropriate for individual AP studies or study groups. Greater consistency in reporting of basic outcome measures may facilitate the interpretation of study results by investigators, regulatory bodies, health care providers, payers, and patients themselves, thereby accelerating the widespread adoption of AP technology to improve the lives of people with type 1 diabetes.

  4. Measuring preferences for schizophrenia outcomes with the time tradeoff method.

    PubMed

    Shumway, Martha; Chouljian, Tandy L; Battle, Cynthia L

    2005-01-01

    Measuring preferences for schizophrenia outcomes facilitates meaningful integration of multiple outcome measures and multiple perspectives on treatment outcomes. The Time Tradeoff (TTO) technique, specifically developed for measuring health state preferences, is used widely in health research, but some evidence suggests that the TTO may work less well with schizophrenia than with other health conditions. This study tested the hypotheses that tailoring the time frame of the standard TTO to the course of schizophrenia and simplifying its presentation format would improve its feasibility and efficiency. Forty clinicians provided TTO ratings using 1 of 4 combinations of time frame and presentation format. Numeric ratings and quantitative and qualitative measures of feasibility showed that while participants preferred the simpler format, none of the alterations improved feasibility. Participants' ratings were prone to logical inconsistencies and participants found all 4 versions of the TTO confusing and poorly suited to the context of schizophrenia treatment.

  5. OMERACT 10 Patient Perspective Virtual Campus: valuing health; measuring outcomes in rheumatoid arthritis fatigue, RA sleep, arthroplasty, and systemic sclerosis; and clinical significance of changes in health.

    PubMed

    Kirwan, John R; Boonen, Annelies; Harrison, Mark J; Hewlett, Sarah E; Wells, George A; Singh, Jasvinder A; Furst, Daniel E; Dworkin, Robert H

    2011-08-01

    This workshop reviewed progress in a number of areas related to patient perspective outcomes that were not specifically included within other areas of the program. A substantial review of the work of the valuing health outcomes group (the "QALY" working group) with participation and feedback from the plenary audience resulted in guidance to refocus on the use of patient preferences in the elaboration of more robust outcome measures for patient-reported outcomes and life impact measures. Progress and developments in the areas of fatigue and sleep in rheumatoid arthritis, outcome measures in hip and knee arthroplasty clinical trials, and scleroderma were outlined, and the challenge of truly understanding the nature of clinically important improvement was reviewed.

  6. Can a future choice affect a past measurement's outcome?

    NASA Astrophysics Data System (ADS)

    Aharonov, Yakir; Cohen, Eliahu; Elitzur, Avshalom C.

    2015-04-01

    An EPR experiment is studied where each particle within the entangled pair undergoes a few weak measurements (WMs) along some pre-set spin orientations, with the outcomes individually recorded. Then the particle undergoes one strong measurement along an orientation chosen at the last moment. Bell-inequality violation is expected between the two final measurements within each EPR pair. At the same time, statistical agreement is expected between these strong measurements and the earlier weak ones performed on that pair. A contradiction seemingly ensues: (i) Bell's theorem forbids spin values to exist prior to the choice of the orientation measured; (ii) A weak measurement is not supposed to determine the outcome of a successive strong one; and indeed (iii) Almost no disentanglement is inflicted by the WMs; and yet (iv) The outcomes of weak measurements statistically agree with those of the strong ones, suggesting the existence of pre-determined values, in contradiction with (i). Although the conflict can be solved by mere mitigation of the above restrictions, the most reasonable resolution seems to be that of the Two-State-Vector Formalism (TSVF), namely, that the choice of the experimenter has been encrypted within the weak measurement's outcomes, even before the experimenters themselves know what their choice will be.

  7. Measures of Outcome for Stimulant Trials: ACTTION Recommendations and Research Agenda

    PubMed Central

    Kiluk, Brian D.; Carroll, Kathleen M.; Duhig, Amy; Falk, Daniel E.; Kampman, Kyle; Lai, Shengan; Litten, Raye Z.; McCann, David J.; Montoya, Ivan D.; Preston, Kenzie L.; Skolnick, Phil; Weisner, Constance; Woody, George; Chandler, Redonna; Detke, Michael J.; Dunn, Kelly; Dworkin, Robert H.; Fertig, Joanne; Gewandter, Jennifer; Moeller, F. Gerard; Ramey, Tatiana; Ryan, Megan; Silverman, Kenneth; Strain, Eric C.

    2015-01-01

    Background The development and approval of an efficacious pharmacotherapy for stimulant use disorders has been limited by the lack of a meaningful indicator of treatment success, other than sustained abstinence. Methods In March, 2015, a meeting sponsored by Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks (ACTTION) was convened to discuss the current state of the evidence regarding meaningful outcome measures in clinical trials for stimulant use disorders. Attendees included members of academia, funding and regulatory agencies, pharmaceutical companies, and healthcare organizations. The goal was to establish a research agenda for the development of a meaningful outcome measure that may be used as an endpoint in clinical trials for stimulant use disorders. Results and Conclusions Based on guidelines for the selection of clinical trial endpoints, the lessons learned from prior addiction clinical trials, and the process that led to identification of a meaningful indicator of treatment success for alcohol use disorders, several recommendations for future research were generated. These include a focus on the validation of patient reported outcome measures of functioning, the exploration of patterns of stimulant abstinence that may be associated with physical and/or psychosocial benefits, the role of urine testing for validating self-reported measures of stimulant abstinence, and the operational definitions for reduction-based measures in terms of frequency rather than quantity of stimulant use. These recommendations may be useful for secondary analyses of clinical trial data, and in the design of future clinical trials that may help establish a meaningful indicator of treatment success. PMID:26652899

  8. Network meta-analysis of multiple outcome measures accounting for borrowing of information across outcomes

    PubMed Central

    2014-01-01

    Background Network meta-analysis (NMA) enables simultaneous comparison of multiple treatments while preserving randomisation. When summarising evidence to inform an economic evaluation, it is important that the analysis accurately reflects the dependency structure within the data, as correlations between outcomes may have implication for estimating the net benefit associated with treatment. A multivariate NMA offers a framework for evaluating multiple treatments across multiple outcome measures while accounting for the correlation structure between outcomes. Methods The standard NMA model is extended to multiple outcome settings in two stages. In the first stage, information is borrowed across outcomes as well across studies through modelling the within-study and between-study correlation structure. In the second stage, we make use of the additional assumption that intervention effects are exchangeable between outcomes to predict effect estimates for all outcomes, including effect estimates on outcomes where evidence is either sparse or the treatment had not been considered by any one of the studies included in the analysis. We apply the methods to binary outcome data from a systematic review evaluating the effectiveness of nine home safety interventions on uptake of three poisoning prevention practices (safe storage of medicines, safe storage of other household products, and possession of poison centre control telephone number) in households with children. Analyses are conducted in WinBUGS using Markov Chain Monte Carlo (MCMC) simulations. Results Univariate and the first stage multivariate models produced broadly similar point estimates of intervention effects but the uncertainty around the multivariate estimates varied depending on the prior distribution specified for the between-study covariance structure. The second stage multivariate analyses produced more precise effect estimates while enabling intervention effects to be predicted for all outcomes, including

  9. Dietary changes and dietary supplement use, and underlying motives for these habits reported by colorectal cancer survivors of the Patient Reported Outcomes Following Initial Treatment and Long-Term Evaluation of Survivorship (PROFILES) registry.

    PubMed

    Bours, Martijn J; Beijer, Sandra; Winkels, Renate M; van Duijnhoven, Fränzel J; Mols, Floortje; Breedveld-Peters, José J; Kampman, Ellen; Weijenberg, Matty P; van de Poll-Franse, Lonneke V

    2015-07-01

    In the present study, we aimed to describe dietary changes made post-diagnosis and current dietary supplement use by survivors of colorectal cancer (CRC), and explore the underlying motives for these lifestyle habits. Cross-sectional analyses were performed for 1458 stage I-IV CRC survivors of the Patient Reported Outcomes Following Initial Treatment and Long-Term Evaluation of Survivorship (PROFILES) registry, diagnosed between 2000 and 2009. Lifestyle, sociodemographic and clinical information was collected. Prevalence of and motivations for dietary changes and supplement use were assessed. Associations between lifestyle, sociodemographic and clinical variables were analysed by multivariable logistic regression. CRC survivors (57% male) were on average 70 (SD 9) years of age and diagnosed 7 (SD 3) years ago. Dietary changes post-diagnosis were reported by 36% of the survivors and current supplement use by 32%. Motivations for dietary changes were mostly cancer-related (44% reported 'prevention of cancer recurrence' as the main reason), while motivations for supplement use were less frequently related to the cancer experience (38% reported 'to improve health and prevent disease in general' as the main reason). Dietary changes were significantly associated with dietary supplement use (OR 1.5, 95% CI 1.1, 2.1). Survivors who had received dietary advice, were non-smokers, under 65 years of age, and had no stoma were more likely to have changed their diet. Survivors who were female, had multiple co-morbidities, and no overweight or obesity were more likely to use supplements. In conclusion, many CRC survivors alter their diet post-diagnosis and use dietary supplements, in part for different reasons. Insights into motivations behind these lifestyle habits and characteristics of CRC survivors adopting these habits can improve the tailoring of lifestyle counselling strategies.

  10. Methodological issues regarding power of classical test theory (CTT) and item response theory (IRT)-based approaches for the comparison of patient-reported outcomes in two groups of patients - a simulation study

    PubMed Central

    2010-01-01

    Background Patients-Reported Outcomes (PRO) are increasingly used in clinical and epidemiological research. Two main types of analytical strategies can be found for these data: classical test theory (CTT) based on the observed scores and models coming from Item Response Theory (IRT). However, whether IRT or CTT would be the most appropriate method to analyse PRO data remains unknown. The statistical properties of CTT and IRT, regarding power and corresponding effect sizes, were compared. Methods Two-group cross-sectional studies were simulated for the comparison of PRO data using IRT or CTT-based analysis. For IRT, different scenarios were investigated according to whether items or person parameters were assumed to be known, to a certain extent for item parameters, from good to poor precision, or unknown and therefore had to be estimated. The powers obtained with IRT or CTT were compared and parameters having the strongest impact on them were identified. Results When person parameters were assumed to be unknown and items parameters to be either known or not, the power achieved using IRT or CTT were similar and always lower than the expected power using the well-known sample size formula for normally distributed endpoints. The number of items had a substantial impact on power for both methods. Conclusion Without any missing data, IRT and CTT seem to provide comparable power. The classical sample size formula for CTT seems to be adequate under some conditions but is not appropriate for IRT. In IRT, it seems important to take account of the number of items to obtain an accurate formula. PMID:20338031

  11. Eliciting the child’s voice in adverse event reporting in oncology trials: Cognitive interview findings from the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events initiative

    PubMed Central

    Reeve, Bryce B.; McFatrich, Molly; Pinheiro, Laura C.; Weaver, Meaghann S.; Sung, Lillian; Withycombe, Janice S.; Baker, Justin N.; Mack, Jennifer W.; Waldron, Mia K.; Gibson, Deborah; Tomlinson, Deborah; Freyer, David R.; Mowbray, Catriona; Jacobs, Shana; Palma, Diana; Martens, Christa E.; Gold, Stuart H.; Jackson, Kathryn D.; Hinds, Pamela S.

    2017-01-01

    Background Adverse event (AE) reporting in oncology trials is required, but current practice does not directly integrate the child’s voice. The Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is being developed to assess symptomatic AEs via child/adolescent self-report or proxy-report. This qualitative study evaluates the child’s/adolescent’s understanding and ability to provide valid responses to the PRO-CTCAE to inform questionnaire refinements and confirm content validity. Procedure From seven pediatric research hospitals, children/adolescents ages 7–15 years who were diagnosed with cancer and receiving treatment were eligible, along with their parent-proxies. The Pediatric PRO-CTCAE includes 130 questions that assess 62 symptomatic AEs capturing symptom frequency, severity, interference, or presence. Cognitive interviews with retrospective probing were completed with children in the age groups of 7–8, 9–12, and 13–15 years. The children/adolescents and proxies were interviewed independently. Results Two rounds of interviews involved 81 children and adolescents and 74 parent-proxies. Fifteen of the 62 AE terms were revised after Round 1, including refinements to the questions assessing symptom severity. Most participants rated the PRO-CTCAE AE items as “very easy” or “somewhat easy” and were able to read, understand, and provide valid responses to questions. A few AE items assessing rare events were challenging to understand. Conclusions The Pediatric and Proxy PRO-CTCAE performed well among children and adolescents and their proxies, supporting its content validity. Data from PRO-CTCAE may improve symptomatic AE reporting in clinical trials and enhance the quality of care that children receive. PMID:27650708

  12. Measuring outcomes and efficiency in medicare value-based purchasing.

    PubMed

    Tompkins, Christopher P; Higgins, Aparna R; Ritter, Grant A

    2009-01-01

    The Medicare program may soon adopt value-based purchasing (VBP), in which hospitals could receive incentives that are conditional on meeting specified performance objectives. The authors advocate for a market-oriented framework and direct measures of system-level value that are focused on better outcomes and lower total cost of care. They present a multidimensional framework for measuring outcomes of care and a method to adjust incentive payments based on efficiency. Incremental reforms based on VBP could provoke transformational changes in total patient care by linking payments to value related to the whole patient experience, recognizing shared accountability among providers.

  13. Are validated outcome measures used in distal radial fractures truly valid?

    PubMed Central

    Nienhuis, R. W.; Bhandari, M.; Goslings, J. C.; Poolman, R. W.; Scholtes, V. A. B.

    2016-01-01

    Objectives Patient-reported outcome measures (PROMs) are often used to evaluate the outcome of treatment in patients with distal radial fractures. Which PROM to select is often based on assessment of measurement properties, such as validity and reliability. Measurement properties are assessed in clinimetric studies, and results are often reviewed without considering the methodological quality of these studies. Our aim was to systematically review the methodological quality of clinimetric studies that evaluated measurement properties of PROMs used in patients with distal radial fractures, and to make recommendations for the selection of PROMs based on the level of evidence of each individual measurement property. Methods A systematic literature search was performed in PubMed, EMbase, CINAHL and PsycINFO databases to identify relevant clinimetric studies. Two reviewers independently assessed the methodological quality of the studies on measurement properties, using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist. Level of evidence (strong / moderate / limited / lacking) for each measurement property per PROM was determined by combining the methodological quality and the results of the different clinimetric studies. Results In all, 19 out of 1508 identified unique studies were included, in which 12 PROMs were rated. The Patient-rated wrist evaluation (PRWE) and the Disabilities of Arm, Shoulder and Hand questionnaire (DASH) were evaluated on most measurement properties. The evidence for the PRWE is moderate that its reliability, validity (content and hypothesis testing), and responsiveness are good. The evidence is limited that its internal consistency and cross-cultural validity are good, and its measurement error is acceptable. There is no evidence for its structural and criterion validity. The evidence for the DASH is moderate that its responsiveness is good. The evidence is limited that its reliability and the

  14. Deconstructing race and ethnicity: implications for measurement of health outcomes.

    PubMed

    Manly, Jennifer J

    2006-11-01

    A crucial issue for health researchers is how to measure health and health-related behaviors across racial/ethnic groups. This commentary outlines an approach that involves the deconstruction of race/ethnicity, which clarifies the independent influences of acculturation, quality of education, socioeconomic class, and racial socialization on outcomes of interest. Research on the influence of these variables on health outcomes in general, and cognitive test performance specifically, is presented. This research indicates that when variables such as quality of education, wealth, and perceived racism are taken into account, the effect of race/ethnicity on health outcomes is greatly reduced. In other words, race/ethnicity serves as a proxy for these more meaningful variables, and explicit measurement of these constructs will improve research of health within majority and minority ethnic groups.

  15. Implementing routine outcome measurement in psychiatric rehabilitation services in Israel.

    PubMed

    Roe, David; Gelkopf, Marc; Gornemann, Miriam Isolde; Baloush-Kleinman, Vered; Shadmi, Efrat

    2015-01-01

    In this article we present the design, development and implementation of the Psychiatric Rehabilitation Routine Outcome Measurement (PR-ROM) project, the first systematic effort to implement mental health routine outcome measures in Israel. The goal of the PR-ROM is to provide updated information about the process and impact of psychiatric rehabilitation services in Israel and to establish a sustainable infrastructure and foundation for routine outcome monitoring of rehabilitation services to improve care, inform policy, generate incentives for service improvement, increase informed decision-making and provide data for research purposes. The rehabilitation services evaluated and the characteristics of the population being served are described and the methods and nature of the collected data as well as some preliminary findings are presented. We discuss the major barriers encountered, our efforts to deal with them and lessons learned during the process. We conclude with a description of the current state of the initiative and plans for the future.

  16. A clinically meaningful theory of outcome measures in rehabilitation medicine.

    PubMed

    Massof, Robert W

    2010-01-01

    Comparative effectiveness research in rehabilitation medicine requires the development and validation of clinically meaningful and scientifically rigorous measurements of patient states and theories that explain and predict outcomes of intervention. Patient traits are latent (unobservable) variables that can be measured only by inference from observations of surrogate manifest (observable) variables. In the behavioral sciences, latent variables are analogous to intensive physical variables such as temperature and manifest variables are analogous to extensive physical variables such as distance. Although only one variable at a time can be measured, the variable can have a multidimensional structure that must be understood in order to explain disagreements among different measures of the same variable. The use of Rasch theory to measure latent trait variables can be illustrated with a balance scale metaphor that has randomly added variability in the weights of the objects being measured. Knowledge of the distribution of the randomly added variability provides the theoretical structure for estimating measures from ordinal observation scores (e.g., performance measures or rating scales) using statistical inference. In rehabilitation medicine, the latent variable of primary interest is the patient's functional ability. Functional ability can be estimated from observations of surrogate performance measures (e.g., speed and accuracy) or self-report of the difficulty the patient experiences performing specific activities. A theoretical framework borrowed from project management, called the Activity Breakdown Structure (ABS), guides the choice of activities for assessment, based on the patient's value judgments, to make the observations clinically meaningful. In the case of low vision, the functional ability measure estimated from Rasch analysis of activity difficulty ratings was discovered to be a two-dimensional variable. The two visual function dimensions are independent

  17. Assessment and Outcomes Measurement: Statewide and Systemwide Initiatives.

    ERIC Educational Resources Information Center

    Talbott, Mary J.; Church, Kathleen

    As part of the final report by the Arizona Board of Regents' Task Force on Excellence, Efficiency and Competitiveness, information on statewide activities on assessment and outcomes measurement is presented. The information comes from a 1986 survey by the Educational Commission of the States, American Association for Higher Education, and State…

  18. Conceptualizing Outcome and Impact Measures for Intelligence Services

    ERIC Educational Resources Information Center

    Gainor, Rhiannon; Bouthillier, France

    2014-01-01

    Introduction: The purpose of this qualitative, exploratory study is to clarify ambiguous concepts in intelligence services literature specifically related to measurement of intelligence outcomes and impact. Method: Face to face interviews were held with five subject experts from various intelligence fields and countries regarding their…

  19. What Do They Measure? Comparing Three Learning Outcomes Assessments

    ERIC Educational Resources Information Center

    Steedle, Jeffrey; Kugelmass, Heather; Nemeth, Alex

    2010-01-01

    Many postsecondary institutions currently administer standardized tests of general college outcomes; more than a quarter of Association of American Colleges and Universities (AAC&U) member institutions do so. Using standardized tests for accountability purposes has been contentious mainly because these tests do not measure every important…

  20. Measuring Educational Outcomes: Vocational Education and Training. Conference Paper

    ERIC Educational Resources Information Center

    Karmel, Tom

    2009-01-01

    The vocational education and training (VET) sector has a long tradition of measuring and reporting outcomes. The public face of this is the "Annual National Report of the Australian Vocational Education and Training System" published (and tabled in the Commonwealth Parliament) since 1994. The reporting framework has undergone a number of…

  1. Measuring Student Learning Outcomes Using the SALG Instrument

    ERIC Educational Resources Information Center

    Scholl, Kathleen; Olsen, Heather M.

    2014-01-01

    U.S. higher education institutions are being called to question their central nature, priorities, and functions, with prominent and unprecedented attention being given to accountability and the measurement of student learning outcomes. As higher education evolves in how it assesses student learning and leisure studies and recreation departments…

  2. Use of Outcome Measurement by Paediatric AHPs in Northern Ireland

    ERIC Educational Resources Information Center

    Harron, Anita; Titterington, Jill

    2016-01-01

    Background: Professional standards advocate routine use of outcome measurement (OM) in the practice of allied health professionals (AHPs). Historically, OM has focused on impairment and its immediate constraints on activity, while current policy encourages the development and addition of impact-based OM. There appears to be an assumption at this…

  3. Psychometric properties of the Spanish version of the Clinical Outcomes in Routine Evaluation – Outcome Measure

    PubMed Central

    Trujillo, Adriana; Feixas, Guillem; Bados, Arturo; García-Grau, Eugeni; Salla, Marta; Medina, Joan Carles; Montesano, Adrián; Soriano, José; Medeiros-Ferreira, Leticia; Cañete, Josep; Corbella, Sergi; Grau, Antoni; Lana, Fernando; Evans, Chris

    2016-01-01

    Objective The objective of this paper is to assess the reliability and validity of the Spanish translation of the Clinical Outcomes in Routine Evaluation – Outcome Measure, a 34-item self-report questionnaire that measures the client’s status in the domains of Subjective well-being, Problems/Symptoms, Life functioning, and Risk. Method Six hundred and forty-four adult participants were included in two samples: the clinical sample (n=192) from different mental health and primary care centers; and the nonclinical sample (n=452), which included a student and a community sample. Results The questionnaire showed good acceptability and internal consistency, appropriate test–retest reliability, and acceptable convergent validity. Strong differentiation between clinical and nonclinical samples was found. As expected, the Risk domain had different characteristics than other domains, but all findings were comparable with the UK referential data. Cutoff scores were calculated for clinical significant change assessment. Conclusion The Spanish version of the Clinical Outcomes in Routine Evaluation – Outcome Measure showed acceptable psychometric properties, providing support for using the questionnaire for monitoring the progress of Spanish-speaking psychotherapy clients. PMID:27382288

  4. Unconditionally secure bit commitment by transmitting measurement outcomes.

    PubMed

    Kent, Adrian

    2012-09-28

    We propose a new unconditionally secure bit commitment scheme based on Minkowski causality and the properties of quantum information. The receiving party sends a number of randomly chosen Bennett-Brassard 1984 (BB84) qubits to the committer at a given point in space-time. The committer carries out measurements in one of the two BB84 bases, depending on the committed bit value, and transmits the outcomes securely at (or near) light speed in opposite directions to remote agents. These agents unveil the bit by returning the outcomes to adjacent agents of the receiver. The protocol's security relies only on simple properties of quantum information and the impossibility of superluminal signalling.

  5. Generating Outcome Measurements: Achievement and Attitudes. A Guide to Educational Outcome Measurements and Their Uses. Seminar No. 3.

    ERIC Educational Resources Information Center

    Mushkin, Selma J.; Billings, Bradley B.

    This guide is essentially designed as a teaching aid for those who would inform planners, officials of educational ministries, school administrators, principals, and teachers about educational outcome measurements. In outline and graphic form, the guide presents topics for discussion in a seminar dealing with how to obtain information on…

  6. Ultrasound cervical length measurement in prediction of labor induction outcome.

    PubMed

    Kehila, M; Abouda, H S; Sahbi, K; Cheour, H; Chanoufi, M Badis

    2016-05-17

    Induction of labor is one of the most common procedures in modern obstetrics, with an incidence of approximately 20% of all deliveries. Not all of these inductions result in vaginal delivery; some lead to cesarean sections, either for emergency reasons or for failed induction. That's why, It seems necessary to outline strategies for the improvement of the success rate of induced deliveries. Traditionally, the identification of women in whom labor induction is more likely to be successful is based on the Bishop score. However, several studies have shown it to be subjective, with high variation and a poor predictor of the outcome of labor induction. Transvaginal sonography for cervical measurement can be a more objective criterion in assessing the success of labor induction. Many studies have been done recently to compare cervical measurement and Bishop Score in labor induction.This paper reviewed the literature that evaluated sonographic cervical length measurement to predict induction of labor outcome.

  7. RASCH ANALYSIS OF CLINICAL OUTCOME MEASURES IN SPINAL MUSCULAR ATROPHY

    PubMed Central

    CANO, STEFAN J.; MAYHEW, ANNA; GLANZMAN, ALLAN M.; KROSSCHELL, KRISTIN J.; SWOBODA, KATHRYN J.; MAIN, MARION; STEFFENSEN, BIRGIT F.; BÉRARD, CAROLE; GIRARDOT, FRANÇOISE; PAYAN, CHRISTINE A.M.; MERCURI, EUGENIO; MAZZONE, ELENA; ELSHEIKH, BAKRI; FLORENCE, JULAINE; HYNAN, LINDA S.; IANNACCONE, SUSAN T.; NELSON, LESLIE L.; PANDYA, SHREE; ROSE, MICHAEL; SCOTT, CHARLES; SADJADI, REZA; YORE, MACKENSIE A.; JOYCE, CYNTHIA; KISSEL, JOHN T.

    2015-01-01

    Introduction Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). Methods Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. Results Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. Conclusions The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges. PMID:23836324

  8. Comparison of Physician-Predicted to Measured Low Vision Outcomes

    PubMed Central

    Chan, Tiffany L.; Goldstein, Judith E.; Massof, Robert W.

    2013-01-01

    Purpose To compare low vision rehabilitation (LVR) physicians’ predictions of the probability of success of LVR to patients’ self-reported outcomes after provision of usual outpatient LVR services; and to determine if patients’ traits influence physician ratings. Methods The Activity Inventory (AI), a self-report visual function questionnaire, was administered pre and post-LVR to 316 low vision patients served by 28 LVR centers that participated in a collaborative observational study. The physical component of the Short Form-36, Geriatric Depression Scale, and Telephone Interview for Cognitive Status were also administered pre-LVR to measure physical capability, depression and cognitive status. Following patient evaluation, 38 LVR physicians estimated the probability of outcome success (POS), using their own criteria. The POS ratings and change in functional ability were used to assess the effects of patients’ baseline traits on predicted outcomes. Results A regression analysis with a hierarchical random effects model showed no relationship between LVR physician POS estimates and AI-based outcomes. In another analysis, Kappa statistics were calculated to determine the probability of agreement between POS and AI-based outcomes for different outcome criteria. Across all comparisons, none of the kappa values were significantly different from 0, which indicates the rate of agreement is equivalent to chance. In an exploratory analysis, hierarchical mixed effects regression models show that POS ratings are associated with information about the patient’s cognitive functioning and the combination of visual acuity and functional ability, as opposed to visual acuity or functional ability alone. Conclusions Physicians’ predictions of LVR outcomes appear to be influenced by knowledge of patients’ cognitive functioning and the combination of visual acuity and functional ability - information physicians acquire from the patient’s history and examination. However

  9. Cross-Sectional Comparisons of Patient-Reported Disease Control, Disease Severity, and Symptom Frequency in Children with Atopic Dermatitis.

    PubMed

    Chang, J; Bilker, W B; Hoffstad, O; Margolis, D J

    2017-02-24

    Atopic dermatitis (AD) is a chronic inflammatory disease of the skin that commonly affects children. Research in AD has utilized an increasing variety of scoring measures to monitor disease, and this lack of standardization has been cited as an obstacle to evidence-based decision making. The Harmonizing Outcome Measures for Eczema (HOME) initiative aims to establish consensus on a core set of outcome measures for AD and currently recommends the Patient-Oriented Eczema Measure (POEM) for recording patient-reported outcomes. This article is protected by copyright. All rights reserved.

  10. Opioid-induced constipation in patients with chronic noncancer pain in the USA, Canada, Germany, and the UK: descriptive analysis of baseline patient-reported outcomes and retrospective chart review

    PubMed Central

    Coyne, Karin S; LoCasale, Robert J; Datto, Catherine J; Sexton, Chris C; Yeomans, Karen; Tack, Jan

    2014-01-01

    Background The characteristics of patients who suffer from noncancer pain and opioid-induced constipation are not well understood. Methods Cross-sectional patient survey and chart review data from the baseline assessment of an ongoing longitudinal study in the USA, Canada, Germany, and the UK were evaluated via descriptive statistics. Participants had confirmation of daily opioid therapy ≥30 mg for ≥4 weeks and self-reported opioid-induced constipation. Response to laxatives was defined by classifying participants into categories of laxative use and evaluating the prevalence of inadequate response to one laxative agent and two or more agents from at least two different laxative classes. Outcomes included the Patient Assessment of Constipation-Symptoms, Work Productivity and Activity Impairment Questionnaire-Specific Health Problem, EuroQOL 5 Dimensions, and Global Assessment of Treatment Benefit, Satisfaction, and Willingness to Continue. Results Patients reported a mean of 1.4 bowel movements not preceded by laxatives and 3.7 bowel movements with laxative use per week; 83% wanted at least one bowel movement per day. Most commonly reported on Patient Assessment of Constipation-Symptoms were straining/squeezing to pass bowel movements (83%), bowel movements too hard (75%), flatulence (69%), and bloating (69%). Eighty-four percent were taking natural or behavioral therapies; 60% were taking at least one over-the-counter laxative; and 19% were taking at least one prescription laxative. Prevalence of inadequate response to one laxative agent was 94%; inadequate response to two or more agents from at least two different laxative classes was 27%. Mean Work Productivity and Activity Impairment Questionnaire-Specific Health Problem values for percent work time missed, percent impairment while working, and percent activity impairment were 9%, 32% (equivalent of 14 hours of lost productivity per week), and 38%. Mean EuroQOL 5 Dimensions index and visual analog scale

  11. Novel Outcome Measures for Clinical Trials in Cystic Fibrosis

    PubMed Central

    Tiddens, Harm AWM; Puderbach, Michael; Venegas, Jose G; Ratjen, Felix; Donaldson, Scott H; Davis, Stephanie D; Rowe, Steven M; Sagel, Scott D; Higgins, Mark; Waltz, David A

    2015-01-01

    Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc. PMID:25641878

  12. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy.

    PubMed

    McDonald, Craig M; McDonald, Dawn A; Bagley, Anita; Sienko Thomas, Susan; Buckon, Cathleen E; Henricson, Eric; Nicorici, Alina; Sussman, Michael D

    2010-09-01

    In Duchenne muscular dystrophy, data directly linking changes in clinical outcome measures to patient-perceived well-being are lacking. This study evaluated the relationship between clinical outcome measures used in clinical trials of ambulatory Duchenne muscular dystrophy (Vignos functional grade, quantitative knee extension strength, timed functional performance measures, and gait velocity) and 2 health-related quality of life measures--the Pediatric Outcomes Data Collection Instrument and Pediatric Quality of Life Inventory-in 52 ambulatory Duchenne muscular dystrophy subjects and 36 controls. Those with the disease showed significant decrements in parent proxy-reported health-related quality of life measures versus controls across all domains. The Pediatric Outcomes Data Collection Instrument transfers/basic mobility and sports/ physical function and the Pediatric Quality of Life Inventory physical functioning domains had significant associations with age (and hence disease progression) and traditional clinical outcome measures employed in clinical trials of ambulatory boys with Duchenne muscular dystrophy. Selected domains of the Pediatric Outcomes Data Collection Instrument and generic Pediatric Quality of Life Inventory are potential patient-reported outcome measures for clinical trials in ambulatory individuals with the disease.

  13. Outcome-Driven Thresholds for Home Blood Pressure Measurement

    PubMed Central

    Niiranen, Teemu J.; Asayama, Kei; Thijs, Lutgarde; Johansson, Jouni K.; Ohkubo, Takayoshi; Kikuya, Masahiro; Boggia, José; Hozawa, Atsushi; Sandoya, Edgardo; Stergiou, George S.; Tsuji, Ichiro; Jula, Antti M.; Imai, Yutaka; Staessen, Jan A.

    2013-01-01

    The lack of outcome-driven operational thresholds limits the clinical application of home blood pressure (BP) measurement. Our objective was to determine an outcome-driven reference frame for home BP measurement. We measured home and clinic BP in 6470 participants (mean age, 59.3 years; 56.9% women; 22.4% on antihypertensive treatment) recruited in Ohasama, Japan (n=2520); Montevideo, Uruguay (n=399); Tsurugaya, Japan (n=811); Didima, Greece (n=665); and nationwide in Finland (n=2075). In multivariable-adjusted analyses of individual subject data, we determined home BP thresholds, which yielded 10-year cardiovascular risks similar to those associated with stages 1 (120/80 mm Hg) and 2 (130/85 mm Hg) prehypertension, and stages 1 (140/90 mm Hg) and 2 (160/100 mm Hg) hypertension on clinic measurement. During 8.3 years of follow-up (median), 716 cardiovascular end points, 294 cardiovascular deaths, 393 strokes, and 336 cardiac events occurred in the whole cohort; in untreated participants these numbers were 414, 158, 225, and 194, respectively. In the whole cohort, outcome-driven systolic/diastolic thresholds for the home BP corresponding with stages 1 and 2 prehypertension and stages 1 and 2 hypertension were 121.4/77.7, 127.4/79.9, 133.4/82.2, and 145.4/86.8 mm Hg; in 5018 untreated participants, these thresholds were 118.5/76.9, 125.2/79.7, 131.9/82.4, and 145.3/87.9 mm Hg, respectively. Rounded thresholds for stages 1 and 2 prehypertension and stages 1 and 2 hypertension amounted to 120/75, 125/80, 130/85, and 145/90 mm Hg, respectively. Population-based outcome-driven thresholds for home BP are slightly lower than those currently proposed in hypertension guidelines. Our current findings could inform guidelines and help clinicians in diagnosing and managing patients. PMID:23129700

  14. Outcomes of Nipple-Sparing Mastectomy: Role of Anatomic Measurements.

    PubMed

    Pairawan, Seyed S; Cody, Derek; Kim, Hahns; Hughes, M Katherine; Solomon, Naveenraj; Senthil, Maheswari; Garberoglio, Carlos; Lum, Sharon

    2016-10-01

    Nipple-sparing mastectomy (NSM) offers improved cosmesis for women undergoing mastectomy; however, there is increased risk for complications with this technique. We sought to determine if standard anatomic measurements could be used to predict complications of NSM. We performed a retrospective review of NSM for which anthropometric measurements of sternal notch to nipple distance, base width, and inframammary fold to nipple distance were available, and compared outcomes by anatomic measurements. We identified 102 cases of NSM with measurements available for study performed in 55 patients. Areola necrosis was associated with base width of greater than 15 cm (42.9% vs 10.9%, P = 0.02), infections were more likely with inframammary fold to nipple distance of more than 10 cm (29.2% vs 10.3%, P = 0.02), hematomas were more likely with sternal notch to nipple distance more than 30 cm (22.2% vs 4.3%, P = 0.03), and delayed wound healing was more likely with sternal notch to nipple distance of more than 25 cm (10.3% vs 1.6%, P = 0.03). There were no significant differences in nipple necrosis, skin flap necrosis, wound care requirements, or operative intervention based on anatomic measurement. Standard anatomic measurements are inconsistent predictors of outcome from NSM and should not be used alone to exclude attempts at NSM.

  15. The Daily Activity Report (DAR) a Novel Measure of Functional Outcome for Serious Mental Illness

    PubMed Central

    Velligan, Dawn I.; Mintz, Jim; Sierra, Cynthia; Martin, Mona L.; Fredrick, Megan; Maglinte, Gregory A.; Corey-Lisle, Patricia K.

    2016-01-01

    The assessment of real-world functional outcomes in clinical trials for medications targeting negative symptoms and cognitive impairment is extremely important. We tested the psychometric properties of the Daily Activity Report (DAR), a novel assessment of productive daily activity. We administered the DAR and additional assessments of functional outcome, functional capacity, cognition and symptomatology to 50 individuals with schizophrenia at 2 time points, 1 month apart and to 25 healthy controls. The DAR records a person’s daily activity for 7 consecutive days based upon phone calls made 3 times a day. A total score and scores in 3 domains; instrumental activities (ie, independent living), social and work or school related activities are generated for the DAR. Inter-item consistency was high 0.89–0.94 for each domain and 0.88 overall. Test–retest reliability across 1 month for the total DAR score was 0.67, P < .0001. The total DAR score as well as scores for social activity and nondomestic work/school differed significantly between control and patient participants (P < .0001). DAR domain scores were associated with negative symptoms and functional outcomes, but the primary score related to these measures was the work/school dimension of the DAR. DAR scores were only weakly and nonsignificantly related to positive symptoms. This study provides preliminary support for the reliability and validity of the DAR using interviewer administration. The development of a patient reported version of the DAR using smart phone technology with automatic scoring is the next step. PMID:26712856

  16. Development of indicators for measuring outcomes of water safety plans

    PubMed Central

    Lockhart, Gabriella; Oswald, William E.; Hubbard, Brian; Medlin, Elizabeth; Gelting, Richard J.

    2015-01-01

    Water safety plans (WSPs) are endorsed by the World Health Organization as the most effective method of protecting a water supply. With the increase in WSPs worldwide, several valuable resources have been developed to assist practitioners in the implementation of WSPs, yet there is still a need for a practical and standardized method of evaluating WSP effectiveness. In 2012, the Centers for Disease Control and Prevention (CDC) published a conceptual framework for the evaluation of WSPs, presenting four key outcomes of the WSP process: institutional, operational, financial and policy change. In this paper, we seek to operationalize this conceptual framework by providing a set of simple and practical indicators for assessing WSP outcomes. Using CDC’s WSP framework as a foundation and incorporating various existing performance monitoring indicators for water utilities, we developed a set of approximately 25 indicators of institutional, operational, financial and policy change within the WSP context. These outcome indicators hold great potential for the continued implementation and expansion of WSPs worldwide. Having a defined framework for evaluating a WSP’s effectiveness, along with a set of measurable indicators by which to carry out that evaluation, will help implementers assess key WSP outcomes internally, as well as benchmark their progress against other WSPs in their region and globally. PMID:26361540

  17. Outcomes 'out of africa': the selection and implementation of outcome measures for palliative care in Africa

    PubMed Central

    2012-01-01

    Background End-of-life care research across Africa is under-resourced and under-developed. A central issue in research in end-of-life care is the measurement of effects and outcomes of care on patients and families. Little is known about the experiences of health professionals' selection and implementation of outcome measures (OM) in clinical care, research, audit, or teaching in Africa. Methods An online survey was undertaken of those using outcome measures across the region, as part of the PRISMA project. A questionnaire addressing the use of OMs was developed for a similar survey in Europe and adapted for Africa. Participants were sampled through the contacts database of APCA. Invitation emails were sent out in January 2010 and reminders in February 2010. Results 168/301 invited contacts (56%) from 24 countries responded, with 78 respondents having previously used OM (65% in clinical practice, 12% in research and 23% for both). Main reasons for not using OM were a lack of guidance/training on using and analysing OM, with 49% saying that they would use the tools if this was provided. 40% of those using OM in clinical practice used POS, and 80% used them to assess, evaluate and monitor change. The POS was also the main tool used in research, with the principle criteria for use being validation in Africa, access to the tool and time needed to complete it. Challenges to the use of tools were shortage of time and resources, lack of guidance and training for the professionals, poor health status of patients and complexity of OM. Researchers also have problems analysing OM data. The APCA African POS was the most common version of the POS used, and was reported as a valuable tool for measuring outcomes. Respondents indicated the ideal outcome tool should be short, multi-dimensional and easy to use. Conclusion This was the first survey on professionals' views on OM in Africa. It showed that the APCA African POS was the most frequently OM used. Training and support are

  18. The International Dermatology Outcome Measures initiative as applied to psoriatic disease outcomes: a report from the GRAPPA 2013 meeting.

    PubMed

    Gottlieb, Alice B; Armstrong, April W; Christensen, Robin; Garg, Amit; Duffin, Kristina Callis; Boehncke, Wolf-Henning; Merola, Joseph F; Gladman, Dafna D; Mease, Philip J; Swerlick, Robert A; Rosen, Cheryl F; Abernethy, April

    2014-06-01

    In the United States, access to care is the number one issue facing our patients with dermatological conditions. In part, this is because we do not have outcome measures that are useful in clinical practice and available in databases where payers and governmental agencies can compare the performance of physicians and treatments. There is a growing recognition that insufficient attention has been paid to the outcomes measured in clinical trials and subsequently in clinical practice. The International Dermatology Outcome Measures group includes all willing stakeholders: patients, physicians, payers, and pharmaceutical scientists. As reported herein, the group's goal is to develop outcome measures in dermatology that address the needs of all involved.

  19. Measuring alternative learning outcomes: dispositions to study in higher education.

    PubMed

    Pampaka, Maria; Williams, Julian; Hutcheson, Graeme; Black, Laura; Davis, Pauline; Hernandez-Martinez, Paul; Wake, Geoff

    2013-01-01

    In this paper we describe the validation of two scales constructed to measure pre-university students' changing disposition (i) to enter Higher Education (HE) and (ii) to further study mathematically-demanding subjects. Items were selected drawing on interview data, and on a model of disposition as socially- as well as self- attributed. Rasch analyses showed that the two scales each produce robust one-dimensional measures on what we call a 'strength of commitment to enter HE' and 'disposition to study mathematically-demanding subjects further' respectively. However, the former scale was initially found to suffer psychometrically from a ceiling effect, which we 'corrected' by adding some harder items at a later data point, and revised the scale according to our interpretation of subsequent results. We finally discuss the potential significance of the constructed measures of learning outcomes, as variables in monitoring or even explaining students' progress into different subjects in HE.

  20. Measuring Outcomes for Young Children and Their Families. Outcome Indicators for Everyday Kids, Everyday Lives: A Vision for Pennsylvania's Children

    ERIC Educational Resources Information Center

    Trivette, Carol M.; Dunst, Carl J.

    2011-01-01

    This monograph includes the final report for a project funded by the Pennsylvania Developmental Disabilities Council for "Measuring Outcomes for Children" (2008 RFP). The goal of the project was to "develop and demonstrate the effectiveness of an instrument designed to measure life outcomes of children with disabilities being…

  1. Connecting Stuttering Management and Measurement: I. Core Speech Measures of Clinical Process and Outcome

    ERIC Educational Resources Information Center

    Shenker, Rosalee C.

    2006-01-01

    Background: There will always be a place for stuttering treatments designed to eliminate or reduce stuttered speech. When those treatments are required, direct speech measures of treatment process and outcome are needed in clinical practice. Aims: Based on the contents of published clinical trials of such treatments, three "core" measures of…

  2. Outcome revealed by preference in schizophrenia (OPS): development of a new class of outcome measurements.

    PubMed

    Falissard, Bruno; Bazin, Nadine; Hardy-Bayle, Marie-Christine

    2006-01-01

    The objective of this paper is to describe the development of a new type of outcome measurement, based on revealed preference, which can be used in serious chronic illnesses. Fifteen texts of about 200 words each were written by one of the authors on the day-to-day life of 15 schizophrenic patients. These 15 'slices of life' thus described were then ranked in terms of acceptability by a second group of 10 schizophrenic patients and by a group of 12 relatives of schizophrenic patients. From these rankings, six situations were selected so as to obtain evenly distributed positioning on an axis of acceptability. These six situations comprised the final instrument. In administration, the patients were first asked if the 'slices of life' that were described were acceptable or not, then if the 'slices of life' described were more or less acceptable than their own lives. Two scores were derived, one for an absolute level and the other for a relative level of the patient's satisfaction with his or her existence. Validation results were presented to a new sample of 229 schizophrenic patients. Internal consistency appeared good and the initial ranking of the six situations in terms of acceptability was confirmed. This study encourages the development of global outcome measures based on revealed preference in chronic serious illnesses.

  3. Selecting Rehabilitation Outcome Measures for People with Multiple Sclerosis

    PubMed Central

    Potter, Kirsten; Allen, Diane D.; Bennett, Susan E.; Brandfass, Kathi G.; Widener, Gail L.; Yorke, Amy M.

    2015-01-01

    Despite the well-known benefits of using standardized outcome measures (OMs) in clinical practice, a variety of barriers interfere with their use. In particular, rehabilitation therapists lack sufficient knowledge in selecting appropriate OMs. The challenge is compounded when working with people with multiple sclerosis (MS) owing to heterogeneity of the patient population and symptom variability in individual patients. To help overcome these barriers, the American Physical Therapy Association appointed the Multiple Sclerosis Outcome Measures Task Force to review and make evidence-based recommendations for OM use in clinical practice, education, and research specific to people with MS. Sixty-three OMs were reviewed based on their clinical utility, psychometric properties, and a consensus evaluation of the appropriateness of use for people with MS. We sought to illustrate use of the recommendations for two cases. The first case involves a 43-year-old man with new-onset problems after an exacerbation. The second case pertains to an outpatient clinic interested in assessing the effectiveness of their MS rehabilitation program. For each case, clinicians identified areas that were important to assess and various factors deemed important for OM selection. Criteria were established and used to assist in OM selection. In both cases, the described processes narrowed the selection of OMs and assisted with choosing the most appropriate ones. The recommendations, in addition to the processes described in these two cases, can be used by clinicians in any setting working with patients with MS across the disability spectrum. PMID:26300704

  4. Methods for assessing relative importance in preference based outcome measures.

    PubMed

    Kaplan, R M; Feeny, D; Revicki, D A

    1993-12-01

    This paper reviews issues relevant to preference assessment for utility based measures of health-related quality of life. Cost/utility studies require a common measurement of health outcome, such as the quality adjusted life year (QALY). A key element in the QALY methodology is the measure of preference that estimates subjective health quality. Economists and psychologists differ on their preferred approach to preference measurement. Economists rely on utility assessment methods that formally consider economic trades. These methods include the standard gamble, time-trade off and person trade-off. However, some evidence suggests that many of the assumptions that underlie economic measurements of choice are open to challenge because human information processors do poorly at integrating complex probability information when making decisions that involve risk. Further, economic analysis assumes that choices accurately correspond to the way rational humans use information. Psychology experiments suggest that methods commonly used for economic analysis do not represent the underlying true preference continuum and some evidence supports the use of simple rating scales. More recent research by economists attempts integrated cognitive models, while contemporary research by psychologists considers economic models of choice. The review also suggests that difference in preference between different social groups tends to be small.

  5. Clinical evaluation of a mobile sensor-based gait analysis method for outcome measurement after knee arthroplasty.

    PubMed

    Calliess, Tilman; Bocklage, Raphael; Karkosch, Roman; Marschollek, Michael; Windhagen, Henning; Schulze, Mareike

    2014-08-28

    Clinical scores and motion-capturing gait analysis are today's gold standard for outcome measurement after knee arthroplasty, although they are criticized for bias and their ability to reflect patients' actual quality of life has been questioned. In this context, mobile gait analysis systems have been introduced to overcome some of these limitations. This study used a previously developed mobile gait analysis system comprising three inertial sensor units to evaluate daily activities and sports. The sensors were taped to the lumbosacral junction and the thigh and shank of the affected limb. The annotated raw data was evaluated using our validated proprietary software. Six patients undergoing knee arthroplasty were examined the day before and 12 months after surgery. All patients reported a satisfactory outcome, although four patients still had limitations in their desired activities. In this context, feasible running speed demonstrated a good correlation with reported impairments in sports-related activities. Notably, knee flexion angle while descending stairs and the ability to stop abruptly when running exhibited good correlation with the clinical stability and proprioception of the knee. Moreover, fatigue effects were displayed in some patients. The introduced system appears to be suitable for outcome measurement after knee arthroplasty and has the potential to overcome some of the limitations of stationary gait labs while gathering additional meaningful parameters regarding the force limits of the knee.

  6. Measuring cognitive outcomes in a pre-clinical bioethics course.

    PubMed

    Fernandes, Ashley K; Borges, Nicole; Rodabaugh, Heather

    2012-05-01

    Medical schools universally accept the idea that bioethics courses are essential components of education, but few studies which measure outcomes (i.e., knowledge or retention) have demonstrated their educational value in the literature. The goal of this study was to examine whether core concepts of a pre-clinical bioethics course were learned and retained. Over the course of 2 years, a pre-test comprising 25 multiple-choice questions was administered to two classes (2008-2010) of first-year medical students prior to the start of a 15-week ethics course, and an identical post-test was administered at the end of the course. A total of 189 students participated. Paired t tests showed a significant difference between pre-test scores and post-test scores. The pre-test average score was 69.8 %, and the post-test average was 82.6 %, an increase of 12.9 % after the ethics course. The pre- and post-test results also suggested a shift in difficulty level of the questions, with students finding identical questions easier after the intervention. Given the increase in post-test scores after the 15-week intervention, the study suggests that core concepts in medical ethics were learned and retained. These results demonstrate that an introductory bioethics course can improve short-term outcomes in knowledge and comprehension, and should provide impetus to educators to demonstrate improved educational outcomes in ethics at higher levels of B.S. Bloom's Taxonomy of Learning.

  7. Vision and vision-related outcome measures in multiple sclerosis.

    PubMed

    Balcer, Laura J; Miller, David H; Reingold, Stephen C; Cohen, Jeffrey A

    2015-01-01

    Visual impairment is a key manifestation of multiple sclerosis. Acute optic neuritis is a common, often presenting manifestation, but visual deficits and structural loss of retinal axonal and neuronal integrity can occur even without a history of optic neuritis. Interest in vision in multiple sclerosis is growing, partially in response to the development of sensitive visual function tests, structural markers such as optical coherence tomography and magnetic resonance imaging, and quality of life measures that give clinical meaning to the structure-function correlations that are unique to the afferent visual pathway. Abnormal eye movements also are common in multiple sclerosis, but quantitative assessment methods that can be applied in practice and clinical trials are not readily available. We summarize here a comprehensive literature search and the discussion at a recent international meeting of investigators involved in the development and study of visual outcomes in multiple sclerosis, which had, as its overriding goals, to review the state of the field and identify areas for future research. We review data and principles to help us understand the importance of vision as a model for outcomes assessment in clinical practice and therapeutic trials in multiple sclerosis.

  8. Vision and vision-related outcome measures in multiple sclerosis

    PubMed Central

    Balcer, Laura J.; Miller, David H.; Reingold, Stephen C.

    2015-01-01

    Visual impairment is a key manifestation of multiple sclerosis. Acute optic neuritis is a common, often presenting manifestation, but visual deficits and structural loss of retinal axonal and neuronal integrity can occur even without a history of optic neuritis. Interest in vision in multiple sclerosis is growing, partially in response to the development of sensitive visual function tests, structural markers such as optical coherence tomography and magnetic resonance imaging, and quality of life measures that give clinical meaning to the structure-function correlations that are unique to the afferent visual pathway. Abnormal eye movements also are common in multiple sclerosis, but quantitative assessment methods that can be applied in practice and clinical trials are not readily available. We summarize here a comprehensive literature search and the discussion at a recent international meeting of investigators involved in the development and study of visual outcomes in multiple sclerosis, which had, as its overriding goals, to review the state of the field and identify areas for future research. We review data and principles to help us understand the importance of vision as a model for outcomes assessment in clinical practice and therapeutic trials in multiple sclerosis. PMID:25433914

  9. The King's Outcome Scale for Childhood Head Injury and Injury Severity and Outcome Measures in Children with Traumatic Brain Injury

    ERIC Educational Resources Information Center

    Calvert, Sophie; Miller, Helen E.; Curran, Andrew; Hameed, Biju; McCarter, Renee; Edwards, Richard J.; Hunt, Linda; Sharples, Peta Mary

    2008-01-01

    The aim of this study was to relate discharge King's Outcome Scale for Childhood Head Injury (KOSCHI) category to injury severity and detailed outcome measures obtained in the first year post-traumatic brain injury (TBI). We used a prospective cohort study. Eighty-one children with TBI were studied: 29 had severe, 15 moderate, and 37 mild TBI. The…

  10. Development of the FOCUS (Focus on the Outcomes of Communication under Six), a Communication Outcome Measure for Preschool Children

    ERIC Educational Resources Information Center

    Thomas-Stonell, Nancy L.; Oddson, Bruce; Robertson, Bernadette; Rosenbaum, Peter L.

    2010-01-01

    Aim: Our aim was to develop an outcome measure, called Focus on the Outcomes of Communication Under Six (FOCUS), that captures real-world changes in preschool children's communication. Conceptually grounded in the World Health Organization International Classification of Functioning, Disability and Health framework, the FOCUS items were derived…

  11. Outcome Measures for Clinical Drug Trials in Autism

    PubMed Central

    Aman, Michael G.; Novotny, Sherie; Samango-Sprouse, Carole; Lecavalier, Luc; Leonard, Elizabeth; Gadow, Kenneth D.; King, Bryan H.; Pearson, Deborah A.; Gernsbacher, Morton Ann; Chez, Michael

    2015-01-01

    This paper identifies instruments and measures that may be appropriate for randomized clinical trials in participants with autism spectrum disorders (ASDs). The Clinical Global Impressions scale was recommended for all randomized clinical trials. At this point, however, there is no “perfect” choice of outcome measure for core features of autism, although we will discuss five measures of potential utility. Several communication instruments are recommended, based in part on suitability across the age range. In trials where the intention is to alter core features of ASDs, adaptive behavior scales are also worthy of consideration. Several “behavior complexes” common to ASDs are identified, and instruments are recommended for assessment of these. Given the prevalence of cognitive impairment in ASDs, it is important to assess any cognitive effects, although cognitive data from ASD randomized clinical trials, thus far, are minimal. Guidance from trials in related pharmacologic areas and behavioral pharmacology may be helpful. We recommend routine elicitation of side effects, height and weight, vital signs, and (in the case of antipsychotics) extrapyramidal side-effects assessment. It is often appropriate to include laboratory tests and assessments for continence and sleep pattern. PMID:14999174

  12. A psychometric analysis of outcome measures in peripheral spondyloarthritis

    PubMed Central

    Turina, Maureen C; Baeten, Dominique L; Mease, Philip; Paramarta, Jacqueline E; Song, In-Ho; Pangan, Aileen L; Landewé, Robert

    2016-01-01

    Objectives To assess the discriminatory capacity of various outcome measures and response criteria in patients with peripheral spondyloarthritis (pSpA). Methods Data originated from two randomised controlled trials, ABILITY-2 and Tnf Inhibition in PEripheral SpondyloArthritis (TIPES). Continuous outcome measures included patient's global assessment (PGA)/physician's global assessment of disease (PhGA), C-reactive protein (CRP), tender joint counts (TJC)/swollen joint counts (SJC), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), and the Ankylosing Spondylitis Disease Activity Score (ASDAS). Dichotomous response criteria included Peripheral SpondyloArthritis Response Criteria (PSpARC), American College of Rheumatology (ACR), ASDAS and BASDAI response criteria. The capacity to discriminate between adalimumab and placebo groups was assessed by standardised mean differences (SMD) for continuous variables, and Pearson's χ2 for dichotomous response criteria. Results Within each trial, the composite indices for axial SpA assessment, ASDAS-CRP (SMD: −0.63 and −0.89 in ABILITY-2 and the TIPES trial, respectively) and BASDAI (SMD: −0.50 and −0.73), and the single-item measures PGA (SMD: −0.47 and −1.12) and PhGA (SMD: −0.64 and −0.87) performed better than other single-item measures, such as CRP (SMD: −0.18 and −0.53), SJC or TJC. In general, the PSpARC and ACR response criteria discriminated better than ASDAS and BASDAI response criteria. Conclusions The axial SpA-specific ASDAS-CRP and BASDAI, but also PGA and PhGA, demonstrated good discriminatory ability in patients with pSpA. The pSpA-specific pSpARC response criteria and the rheumatoid arthritis-specific ACR response criteria also discriminated well. To fully capture typical pSpA manifestations, it may be worth developing new pSpA-specific indices with better performance and face validity. Trial registration numbers ABILITY-2: NCT01064856; TIPES: EUDRACT 2008-006885-27. PMID:26245756

  13. Outcome instruments for prosthetics: clinical applications.

    PubMed

    Heinemann, Allen W; Connelly, Lauri; Ehrlich-Jones, Linda; Fatone, Stefania

    2014-02-01

    Outcome measurement is crucial to assuring high-quality patient services and improving the quality of services provided by prosthetists. This article summarizes recent evidence on the measurement properties of outcome measures, and updates previously published summaries of outcome instruments. The review focuses on measures of mobility, functional status, quality of life, and patient satisfaction, and includes both performance-based and patient-reported outcomes. Amputation-specific and general measures that are suitable for patients served by prosthetists are discussed. It is encouraging that responsiveness of measures is often reported, as this information is needed to improve clinical utility.

  14. Results of a Two-Year Pilot Study of Clinical Outcome Measures in Collagen VI-related Myopathy and LAMA2-related Muscular Dystrophy

    PubMed Central

    Meilleur, Katherine G.; Jain, Minal S.; Hynan, Linda S.; Shieh, Ching-Yi; Kim, Eunice; Waite, Melissa; McGuire, Michelle; Fiorini, Courtney; Glanzman, Allan M.; Main, Marion; Rose, Kristy; Duong, Tina; Bendixen, Roxanna; Linton, Melody M.; Arveson, Irene C.; Nichols, Carmel; Yang, Kelly; Fischbeck, Kenneth H.; Wagner, Kathryn R.; North, Kathryn; Mankodi, Ami; Grunseich, Christopher; Hartnett, Elizabeth J.; Smith, Michaele; Donkervoort, Sandra; Schindler, Alice; Kokkinis, Angela; Leach, Meganne; Foley, A. Reghan; Collins, James; Muntoni, Francesco; Rutkowski, Anne; Bönnemann, Carsten G

    2016-01-01

    Potential therapies are currently under development for two congenital muscular dystrophy (CMD) subtypes: Collagen VI Related Myopathy (COL6-RM) and Laminin α2-related dystrophy (LAMA2-RD). However, appropriate clinical outcome measures to be used in clinical trials have not been validated in CMDs. We conducted a two-year pilot study to evaluate feasibility, reliability, and validity of various outcome measures, particularly the Motor Function Measure 32, in 33 subjects with COL6-RM and LAMA2-RD. In the first year, outcome measures tested included: Motor Function Measure 32 (MFM32), forced vital capacity (FVC) percent predicted sitting, myometry, goniometry, 10-meter walk, Egen Klassification 2, and PedsQL™ Generic and Neuromuscular Cores. In the second year, we added the North Star Ambulatory Assessment (NSAA), Hammersmith Functional Motor Scale (HFMS), timed functional tests, Measure of Activity Limitations (ACTIVLIM), Quality of Upper Extremity Skills Test (QUEST), and Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue subscale. The MFM32 showed strong inter-rater (0.92) and internal consistency (0.96) reliabilities. Concurrent validity for the MFM32 was supported by large correlations (range 0.623–0.936) with the following: FVC, NSAA, HFMS, timed functional tests, ACTIVLIM, and QUEST. Significant correlations of the MFM32 were also found with select myometry measurements, mainly of the proximal extremities and domains of the PedsQL™ scales focusing on physical health and neuromuscular disease. Goniometry measurements were less reliable. The Motor Function Measure is reliable and valid in the two specific subtypes of CMD evaluated, COL6-RM and LAMA2-RD. The NSAA is useful as a complementary outcome measure in ambulatory individuals. Preliminary concurrent validity of several other clinical outcome measures was also demonstrated for these subtypes. PMID:25307854

  15. Novel mechanisms, treatments, and outcome measures in childhood sleep

    PubMed Central

    Colonna, Annalisa; Smith, Anna B.; Pal, Deb K.; Gringras, Paul

    2015-01-01

    Sleep disorders and sleep of insufficient duration and quality are on the increase due to changes in our lifestyle, particularly in children and adolescents. Sleep disruption is also more common in children with medical conditions, compounding their difficulties. Recent studies have focused on new mechanisms that explain how learning and cognitive performance depend on a good night’s sleep. Growing alongside this latest understanding is an innovative new field of non-drug interventions that improve sleep architecture, with resulting cognitive improvements. However, we need to rigorously evaluate such potentially popular and self-administered sleep interventions with equally state-of-the-art outcome measurement tools. Animated hand-held games, that incorporate embedded sleep-dependent learning tasks, promise to offer new robust methods of measuring changes in overnight learning. Portable computing technology has the potential to offer practical, inexpensive and reliable tools to indirectly assess the quality of sleep. They may be adopted in both clinical and educational settings, providing a unique way of monitoring the effect of sleep disruption on learning, leading also to a radical rethink of how we manage chronic diseases. PMID:26029140

  16. Quantifying prosthetic gait deviation using simple outcome measures

    PubMed Central

    Kark, Lauren; Odell, Ross; McIntosh, Andrew S; Simmons, Anne

    2016-01-01

    AIM: To develop a subset of simple outcome measures to quantify prosthetic gait deviation without needing three-dimensional gait analysis (3DGA). METHODS: Eight unilateral, transfemoral amputees and 12 unilateral, transtibial amputees were recruited. Twenty-eight able-bodied controls were recruited. All participants underwent 3DGA, the timed-up-and-go test and the six-minute walk test (6MWT). The lower-limb amputees also completed the Prosthesis Evaluation Questionnaire. Results from 3DGA were summarised using the gait deviation index (GDI), which was subsequently reg