Properties Of Patient-reported Outcome Measures Research Articles

Patient-reported outcome measures for life participation in patients with chronic kidney disease: a systematic review

Abstract Background The symptoms, comorbidities and treatment burden associated with chronic kidney disease (CKD) can be debilitating and limit life participation in patients with CKD not requiring kidney replacement therapy (KRT). The aim of this study was to identify the characteristics, content, and psychometric properties of patient-reported outcome measures (PROMs) used to assess life participation in patients with CKD. Methods We searched MEDLINE, Embase, PsycINFO, and CINAHL from database inception to February 2023 for all studies that reported life participation in patients with CKD (Stage 1–5 not requiring kidney replacement therapy). We analyzed the characteristics, dimensions of life participation and psychometric properties of the measures. Results From the 114 studies included, 20 (18%) were randomized trials, 3 (3%) were non-randomized trials, and 91 (80%) were observational studies. Forty-one different measures were used to assess life participation, of which six (15%) were author-developed measures. Twelve (29%) measures assessed life participation specifically, while 29 (71%) measures assessed broader constructs such as quality of life, which included questions relevant to life participation. The 36-Item Short Form Health Survey (SF-36) and Kidney Disease Quality of Life—Short Form (KDQOL-SF) were the most frequently used, in 39 (34%) and 24 (21%) studies, respectively. Many content domains for life participation were assessed including physical activities (walking, running, and sports), social activities, leisure activities, work or study and self-care. None of the measures for life participation were developed specifically for CKD. Four measures (EQ-5D-3 L, FACT-An, SF-6D, and SF-36) had validation data collected in patients with CKD. Conclusion The measures for life participation used in patients with CKD vary in content with few validated in the CKD population. There is a need for a validated measure to assess life participation in a meaningful and consistent way in all patients with CKD worldwide.

Psychometric properties of patient-reported outcome measures assessing recovery from hand fractures: a systematic review.

To extensively review Patient Reported Outcome Measures (PROMs) used to assess outcomes in persons with hand fractures in terms of their psychometric properties. A COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodological review was conducted. Six electronic databases were searched (Medline, Embase, Scopus, Web of Science, PsycINFO, CINAHL) for studies evaluating the psychometric properties of PROMs assessing recovery from hand fracture. Titles and abstracts, full text review, quality assessment and data extraction were performed by two reviewers. Any disagreements were resolved after review by a third, expert reviewer. Quality assessment of included studies was performed using the COSMIN checklist. This COSMIN review found that there were only 4 studies that fulfilled the inclusion criteria in terms of assessing measurement properties of PROMs in hand fractures. Only the construct validity of the Disabilities of the Arm, Shoulder, and Hand (DASH), the Quick DASH (QDASH) and the Duruoz Hand Index (DHI), and the responsiveness of the DASH, the Patient Specific Functional Scale (PSFS), the Patient-Rated Wrist and Hand Evaluation (PRWHE) and the Michigan Hand Outcomes Questionnaire (MHQ) were assessed in these studies. The overall rating of the studies was assessed as insufficient or indeterminate and quality of evidence was assessed as moderate, low, or very low by our team. This study identified that there is a lack of evidence in the medical literature with regards to the measurement properties of PROMs in patients with hand fractures. The 4 included studies do not provide good quality data to support the use of these PROMS in patients with hand fractures. There is a need for more studies for more PROMs. This has important consequences for how outcomes will be measured in clinical studies in hand research and in clinical practice.

Patient-Reported Outcome Measures Used on Patients With Anterior Cruciate Ligament Injury.

Patient-reported knee-related rating scores and scales are widely used in reporting the clinical outcomes of anterior cruciate ligament (ACL) surgery. Understanding the psychometric properties of such measures is vital to recognizing the limitations that such measures may confer. The aim of this study was to review the available evidence as to the psychometric properties of patient-reported outcome measures (PROMs) used in ACL surgery. Eleven studies were identified, the majority being prospective cohort studies. Eight English, ACL-specific patient-reported outcome measures were identified and evaluated: Lysholm score, Tegner Activity Scale (TAS), Cincinnati score, ACL-Quality of Life (QOL) score, International Knee Documentation Committee (IKDC) Subjective Knee Form (SKF), Knee Injury and Osteoarthritis Outcome Score (KOOS)-ACL score, and ACL-Return to Sport Injury (RSI) scale. Only the Lysholm score, ACL-QOL, IKDC SKF, and ACL-RSI were evaluated for internal consistency, having an acceptable Cronbach's α (α>0.70). Most of the scoring systems were assessed for test-retest reliability, with four of them (Lysholm score, TAS, Cincinnati score, and IKDC SKF) having acceptable intraclass correlation coefficient (ICC) values (ICC > 0.70). Criterion validity was assessed for most measures with a good correlation with the IKDC. Effect sizes and standardized response means were large for three instruments that measured responsiveness (Lysholm score, TAS, and Cincinnati score) and moderate for one (ACL-QOL). Evidence is stronger and more robust for the Lysholm score, TAS, ACL-QOL, and IKDC SKF. However, there is variation in their psychometric properties as well as the aspect of knee-related health they are assessing. Hence, none can be universally applicable to all patients with ACL injuries. Recognizing these parameters is vital when choosing which instrument to use in reporting the outcomes of ACL injury or ACL surgery studies.

Validation of Patient-Reported Outcomes in Patients With Nonmetastatic Breast Cancer Receiving Comprehensive Nodal Irradiation in the RadComp Trial

Our purpose was to evaluate the measurement properties of patient-reported outcome (PRO) measures used in the ongoing RadComp pragmatic randomized clinical trial (PRCT). The deidentified and blinded data set included 774 English-speaking female participants who completed their 6-month posttreatment assessment. Eleven PRO measures were evaluated, including the Trial Outcome Index from the Functional Assessment of Cancer Therapy-Breast (FACT-B), Satisfaction with Breast Cosmetic Outcomes, the BREAST-Q, and selected Patient-Reported Outcomes Measurement Information System (PROMIS) measures. PROs were measured at 3 timepoints: baseline, completion of radiation therapy (RT), and 6 months post-RT. Ten variables were used as validity anchors. Pearson or Spearman correlations were calculated between PROs and convergent validity indicators. Mean PRO differences between clinically distinct categories were compared with analysis of variance methods (known-groups validity). PRO change scores were mapped to change in other variables (sensitivity to change). Most correlations between PROs and validity indicators were large (≥0.5). Mean score for Satisfaction with Breast Cosmetic Outcomes was higher (better) for those with a lumpectomy compared with those with a mastectomy (P < .001). Mean scores for the FACT-B Trial Outcome Index and for PROMIS Fatigue and Ability to Participate in Social Roles and Activities were better for those with good baseline performance status compared with those with poorer baseline performance status (P < .05). At completion of RT and post-RT, mean scores for Satisfaction with Breast Cosmetic Outcomes and BREAST-Q Radiation were significantly different (P < .001) across categories for all Functional Assessment of Chronic Illness Therapy -Treatment Satisfaction - General items. There were medium-sized correlations between change scores for FACT-B Trial Outcome Index, Fatigue, Anxiety, and Ability to Participate in Social Roles and change scores in the Visual Analog Scale. For patients with nonmetastatic breast cancer receiving radiation in the RadComp PRCT, our findings demonstrate high reliability and validity for important PRO measures, supporting their psychometric strength and usefulness to reflect the effect of RT on health-related quality of life.

Questionnaires assessing knowledge and beliefs about musculoskeletal conditions are potentially suitable for use, but further research is needed: a systematic review

ObjectivesTo evaluate the measurement properties of Patient-reported outcome measures (PROMs) for knowledge and/or beliefs about musculoskeletal conditions. Study Design and SettingA systematic review was performed according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines. This review was prospectively registered on PROSPERO – ID: CRD42022303111. Electronic databases, reference lists, forward citation tracking, and contact with experts were used to identify studies. Eligible studies were reports developing or assessing a measurement property of a PROM measuring musculoskeletal condition specific-knowledge and/or beliefs. We assessed the methodological quality and measurement properties of included studies. A modified Grading of Recommendations Assessment Development and Evaluation approach was used to rate the quality of evidence for each PROM. ResultsThe literature search was performed from inception to 11th September 2023. Sixty records were included, reporting 290 individual studies, and provided information on 25 PROMs. Five PROMs presented sufficient structural validity, three presented sufficient cross-cultural validity, ten presented sufficient reliability, three presented sufficient criterion validity, six presented sufficient hypothesis-testing, and four presented sufficient responsiveness. No PROM presented sufficient evidence for content validity, internal consistency, and measurement error. Based on the available evidence, no PROM was classified as suitable for use according to the COSMIN recommendations. Twenty-four PROMs are potentially suitable for use, and one PROM is not recommended for use. ConclusionNo PROM designed to assess knowledge and/or beliefs about musculoskeletal conditions meets the COSMIN criteria of suitable for use. Most PROMs identified in this systematic review were considered as potentially suitable for use and need further high-quality research to assess their measurement properties.

Psychometric Properties of Patient-reported Outcome Measures to Assess Resilience in Individuals with Musculoskeletal Pain or Rheumatic Conditions: A COSMIN-based Systematic Review.

The objective of this systematic review was to provide a comprehensive overview of the measurement properties of patient-reported outcome measures (PROMs) used to assess resilience in individuals with musculoskeletal and rheumatic conditions. Four electronic databases (MEDLINE, CINAHL, PsycINFO, Web of Science) were searched. Studies assessing any measurement property in the target populations were included. Two reviewers independently screened all studies and assessed risk of bias using the COSMIN checklist. Thereafter, each measurement property of each PROM was classified as sufficient, insufficient, or inconsistent based on the COSMIN criteria for good measurement properties. Four families of PROMs (Brief Resilient Coping Scale [BRCS]; Resilience Scale [RS-18]; Connor-Davidson Resilience Scale [CD-RISC-10 and CD-RISC-2]; and Pain Resilience Scale [PRS-14 and PRS-12]) were identified from the 9 included studies. Even if no PROM showed sufficient evidence for all measurement properties, the PRS and CD-RISC had the most properties evaluated and showed the best measurement properties; although responsiveness still needs to be assessed for both PROMs. Both PROMs showed good level of reliability (intraclass coefficient correlation 0.61 to 0.8) and good internal consistency (Cronbach's alpha ≥0.70). Minimal detectable change values were 24.5% for PRS and between 4.7% and 29.8% for CD-RISC. Although BRCS, RS-18, CD-RISC or PRS have been used to evaluated resilience in individuals with musculoskeletal and rheumatic conditions, the current evidence only supports the use of PRS or CD-RISC in this population. Further methodological studies are therefore needed and should prioritize the assessment of reliability and responsiveness.

Quality of measurement properties of medication adherence instruments in cardiovascular diseases and type 2 diabetes mellitus: a systematic review and meta-analysis

BackgroundMedication adherence has a major impact on reducing mortality and healthcare costs related to the treatment of cardiovascular diseases and diabetes mellitus. Selecting the best patient-reported outcome measure (PROM) among the many available for this kind of patient is extremely important. This study aims to critically assess, compare and synthesize the quality of the measurement properties of patient-reported outcome measures to assess medication adherence among patients with cardiovascular diseases and/or type 2 diabetes mellitus.MethodsThis review followed the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines and was reported according to the Preferred Reporting Items for Systematic Review and Meta-Analyses (PRISMA). The searches were performed in Web of Science, SCOPUS, PubMed, CINAHL, EMBASE, LILACS, PsycINFO, and ProQuest (gray literature).ResultsA total of 110 records encompassing 27 different PROMs were included in the review. The included records were published between 1986 and 2023, most of which reported studies conducted in the United States and were published in English. None of the PROMs were classified in the category “a”, thus being recommended for use due to the quality of its measurement properties. The PROMs that should not be recommended for use (category “c”) are the MTA, GMAS, DMAS-7, MALMAS, ARMS-D, and 5-item questionnaire. The remaining PROMs, e.g., MMAS-8, SMAQ, MEDS, MNPS, ARMS-12, MGT, MTA-OA, MTA-Insulin, LMAS-14, MARS-5, A-14, ARMS-10, IADMAS, MAQ, MMAS-5, ProMAS, ARMS‐7, 3-item questionnaire, AS, 12-item questionnaire, and Mascard were considered as having the potential to be recommended for use (category “b”).ConclusionNone of the included PROMs met the criteria for being classified as trusted and recommended for use for patients with cardiovascular diseases and/or type 2 diabetes mellitus. However, 21 PROMs have the potential to be recommended for use, but further studies are needed to ensure their quality based on the COSMIN guideline for systematic reviews of PROMs.Systematic review registrationPROSPERO CRD42019129109

Cross-cultural adaptation of the FRAIL scale for critically ill patients in Spain.

To translate and culturally adapt the FRAIL scale into Spanish and perform a preliminary test of diagnostic accuracy in patients admitted to intensive care units. Cross-sectional diagnostic study. Five intensive care units (ICU) in Spain were participated. Stage 1: Three native Spanish-speaking bilingual translators familiar with the field of critical care translated the scale from English into Spanish. Stage 2: Three native English-speaking bilingual translators familiar with critical care medicine. Stage 3: Authors of the original scale compared the English original and back-translated versions of the scale. Stage 4: Five nurses with more than 5 years of ICU experience and five critical care physicians assessed the comprehension and relevance of each of the items of the Spanish version in 30 patients of 3 different age ranges (<50, 50-65 and >65 years). The FRAIL scale was translated and adapted cross-culturally for patients admitted to intensive care units in Spain. The process consisted of four stages: translation, back translation, comparison and pilot test. There was good correspondence between the original scale and the Spanish version in 100% of the items. The participating patients assessed the relevance (content validity) and comprehensibility (face validity) of each of the items of the first Spanish version. The relevance of some of the items scored low when the scale was used in patients younger than 65 years. We have cross-culturally adapted the FRAIL scale, originally in English, to Spanish for its use in the critical care medical setting in Spanish-speaking countries. Physicians and nurses can apply the new scale to all patients admitted to the intensive care units. Nursing care can be adapted according to frailty, trying to reduce the side effects of admission to these units for the most fragile patients. The manuscript's authors have adhered to the EQUATOR guidelines, using the COSMIN reporting guideline for studies on the measurement properties of patient-reported outcome measures. In a pilot clinical study, we applied the first version of the FRAIL-Spain scale to intensive care unit (ICU) patients. Five nurses with more than 5 years of ICU experience and five critical care physicians assessed the relevance (content validity) and comprehensibility (face validity) of the five items of the first Spanish version. Relevance was assessed using a 4-point Likert scale ranging from 1 (no relevance) to 4 (high relevance), and comprehensibility was assessed as poor, acceptable or good. Each health professional applied the scale to three patients (total number of patients = 30) of three different age ranges (<50, 50-65 and >65 years) and recorded the time of application of the scale to each patient. Although the frailty scales were initially created by geriatricians to be applied to the elders, there is little experience with their application in critically ill patients of any age. Therefore, more information is needed to determine the relevance of using this scale in critical care patients. In this pilot study, we considered that nurses and critical care physicians should evaluate frailty using this adapted scale in adult patients admitted to the Intensive Care Units.

Patient-reported outcome measures of stigma and discrimination for people living with HIV: a systematic review protocol.

The objective of this systematic review is to apply COnsensus-based standards for the selection of health Measurement INstruments (COSMIN) methodology to review psychometric properties of patient-reported outcome measures (PROMs) of HIV-related stigma and/or discrimination. Due to the development of antiretroviral therapy, the life expectancy of people living with HIV is similar to the life expectancy of the rest of the population. However, HIV-related stigma and discrimination are still major barriers to improving the quality of life of persons living with HIV. These barriers are a threat to these individuals and the world's public health care systems. Accurate and reproducible PROMs are prerequisites for robust and reliable results. Therefore, it is important to choose acceptable PROMs with satisfactory psychometric properties to assess HIV-related stigma and/or discrimination. However, there has been no systematic review of HIV-related stigma or discrimination PROMs in the field of HIV care. All studies including adults (age ≥18 years) living with HIV will be eligible for inclusion in this review. The review will consider studies that report 1 or more psychometric properties of HIV-related stigma and/or discrimination PROMs. We will consider studies conducted in any country or setting published in English or Chinese. Nine databases will be searched from January 1, 1996, to the present, including PubMed, Embase, CINAHL, Web of Science, PsycINFO, ProQuest Dissertations and Theses, Cochrane Library, China National Knowledge Infrastructure, and Wanfang. Assessment of methodological quality, data extraction, data synthesis, and assessing certainty in the findings will be conducted using the COSMIN methodology. PROSPERO CRD42022308579.

Properties of Patient-Reported Outcome Measures for Post-Traumatic Stress Disorder in North Korean Defectors: A Scoping Review.

Posttraumatic stress disorder (PTSD) is highly prevalent among North Korean defectors. This scoping review aimed to appraise the measurement properties of patient-reported outcome measures (PROMs) that assess PTSD among North Korean defectors. A systematic search was conducted using Research Information Sharing Service, ScienceON, PubMed, and Embase from their inception up to September 15, 2022. Each measurement properties of the included PROMs were evaluated using COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) methodology. First, the methodological quality of each study on measurement properties was evaluated using the COSMIN Risk of Bias checklist. Second, the result of each single study on a measurement property was rated against the updated criteria for good measurement properties. After screening the articles, nine instruments from 40 studies were included. Among these instruments, Impact of Event ScaleRevised was the most frequently used (13 studies [38.1%]), followed by the Trauma Scale for North Korean Refugees, PTSD Checklist, and Posttraumatic Diagnostic Scale. The theoretical basis of all PROMs was secure in terms of content validity. The most frequently tested characteristics in measurement properties for internal structure were internal consistency, criterion validity, and convergent validity. No study reported sufficient structural validity as confirmatory factor analysis results with a model fit. There is insufficient evidence to support the recommendation of a specific PROM for use among North Korean defectors with PTSD.

Measurement properties of patient-reported outcome measures of mental help-seeking attitude: a systematic review of psychometric properties.

At present, the phenomenon of patients with mental disorders not seeking mental help is very serious, and the mental help-seeking attitude is the central structure of the help process. However, there is no consensus on which patient-reported outcome measure (PROM) should be used to assess mental help-seeking attitudes. The systematic review aims to critically appraise, compare, and summarize the measurement quality of the all-available PROMs about mental help-seeking attitudes to provide evidence-based guidance and reference for clinical researchers. A systematic search was conducted in 9 databases (PubMed, Embase, Web of Science, Medline, APA, CINAHL, Sinomed, CNKI, and WanFang) since the establishment of the database until November 30, 2022 to identify articles on the PROMs of mental help-seeking attitudes. We used the COSMIN guidelines to evaluate the methodological quality and measurement properties of all-available PROMs, and a modified Grading, Recommendation, Assessment, Development, and Evaluation (GRADE) system to evaluate the level of evidence supporting each rating. Finally, the recommendation level is given according to the overall quality of each PROM. We identified 29 studies representing 13 PROMs out of 2,828 screening studies. The overall quality of the included PROMs varied, with 6 rated as class B, 6 as class C, and only the Mental Help Seeking Attitudes Scale (MHSAS) as class A. The measurement characteristics of MHSAS have been the most comprehensive evaluation, and it has good reliability and validity, and high feasibility for clinical application, so it can be temporarily recommended for use, but the above conclusions still need to be supported by more high-quality evidence.

Cross-cultural adaptation and psychometric properties of patient-reported outcome measures in Arabic speaking countries: A scoping review

Patient-reported outcome measures (PROMs) are increasingly recognized as an indicator of healthcare quality and safety. Over the past decades, there has been a growing interest in using PROMs in Arabic-speaking populations. However, there is a paucity of data regarding the quality of their cross-cultural adaptation (CCA) and measurement properties. To identify PROMs that have been developed, validated or cross-culturally adapted to Arabic and evaluate methodological qualities of CCA and measurement properties. MEDLINE, EMBASE, CINAHL, PsycINFO, IPA, and ISI Web of Science were searched using search terms 'PROMs', 'Arabic countries', 'CCA', and 'psychometric properties'. Measurement properties were evaluated using COSMIN quality criteria and CCA quality was assessed using the Oliveria rating method. This review included 260 studies with 317 PROMs, focusing on psychometric testing (83.8%), CCA (75.8%), utilizing PROMs as outcome measures (13.4%), and developing PROMs (2.3%). Out of the 201 cross-culturally adapted PROMs, forward translation was the most frequently reported component of CCA (n=178), followed by back translation (n=174). Out of the 235 PROMs that reported measurement properties, internal consistency was the most frequently reported measurement property (n=214), followed by reliability (n=160) and hypotheses testing (n=143). Other measurement properties were less reported, including responsiveness (n=36), criterion validity (n=22), measurement error (n=12), and cross-cultural validity (n=10). The strongest measurement property was hypotheses testing (n=143), followed by reliability (n=132). There are several caveats regarding the quality of CCA and measurement properties of PROMs included in this review. Only 1 out of 317 Arabic PROMs met CCA and psychometric optimal quality criteria. Therefore, there is a need to improve the methodological quality of CCA and measurement properties of PROMs. This review provides valuable information for researchers and clinicians when choosing PROMs for practice and research. There were only 5 treatment-specific PROMs, indicating the necessity for more research focusing on development and CCA of such measures.

Measurement properties of patient-reported outcome measures used in rehabilitation of adults with chronic musculoskeletal pain: A mapping review.

Choosing measurement tools for diagnostic, prognostic, or evaluative purposes in a chronic musculoskeletal pain (CMP) population is challenging for rehabilitation practice. Implementation of measurement tools for clinical practice is impaired by gaps in knowledge about measurement properties. Identifying evidence about the measurement properties of tools frequently used in Dutch pain rehabilitation practice. A mapping review was conducted of eligible studies that investigated reliability, validity, or responsiveness, and interpretability, as defined by the COSMIN taxonomy, of original versions or Dutch translations of predefined Patient-Reported Outcome Measures (PROMs) in a CMP population. MEDLINE, PsycINFO, EMBASE, and CINAHL were searched in March 2021. Results were visually mapped. Thirty-five studies were included. The results show many knowledge gaps in both original and translated versions. In general, aspects of validity were most frequently reported. The Pain Disability Index, Pain Catastrophizing Scale, and the 12-Item Short Form Health Survey were the most studied measurement tools. No results were found for the Checklist Individual Strength, Illness Perception Questionnaire, and Utrecht Coping List. Little evidence of the measurement properties of PROMs used in rehabilitation of patients with CMP in the Netherlands was found. PROMs need to be used and interpreted with caution in daily practice.

Psychometric properties of patient-reported outcome measures assessing self-efficacy in patients with inflammatory bowel disease: A systematic review.

The aim of this study is to conduct a comprehensive evaluation of the patient-reported outcome measure assessing self-efficacy in patients with inflammatory bowel disease, and recommend the most robust measurement. A systematic review of psychometric properties. We performed systematic electronic searches in the following databases from inception to 26 May 2022: PubMed, Embase, CINAHL, Web of Science, Cochrane Library, and PsycINFO via OVID. This review evaluated the tools' quality in accordance with the Selection of Consensus-Based Health Measurement Instruments 2018 system Evaluation guidelines. Three patient-reported outcome measures were identified in the five included studies. The "IBD-yourself" questionnaire revealed very low evidence for a sufficient hypothesis test for construct validity, moderate evidence for insufficient internal consistency, and very low evidence for uncertain measurement error. Evidence from Inflammatory Bowel Disease Self-Efficacy Scale validated internal consistency, structural validity, criterion validity, and hypothesis test for construct validity. However, evidence of moderate quality corroborated this reliability. The Adolescents and Young Adults Inflammatory Bowel Disease Self-Efficacy Scale demonstrated very low evidence for sufficient internal consistency, moderate evidence for a sufficient hypothesis test for construct and content validity, and very low evidence for uncertain reliability. Compared with the other two measures, the Inflammatory Bowel Disease Self-Efficacy Scale has higher quality evidence of higher overall ratings for some of its psychometric properties, but there were some methodological problems that must be further studied to determine their quality. It is uncertain whether a scale to assess self-efficacy in patients with inflammatory bowel disease has good measurement performance in clinical applications. This study first presents the methodological quality and psychometric properties of the self-efficacy scale used to assess patients with inflammatory bowel disease, which is based on the Consensus-Based Health Measurement Instruments standard. This study can help researchers and physicians decide which scale is the most suitable and reliable for patients with inflammatory bowel disease. Because this was a systematic review based on synthesizing information from previous studies, no human participants were included.

Psychometric properties of self-reported measures of self-management for chronic heart failure patients: a systematic review.

To identify and assess the psychometric properties of patient-reported outcome measures (PROMs) of self-management for chronic heart failure (CHF) patients. Eleven databases and two websites were searched from the inception to 1 June 2022. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) risk of bias checklist was used to assess the methodological quality. The COSMIN criteria were used to rate and summarize the psychometric properties of each PROM. The modified Grading of Recommendation Assessment, Development, and Evaluation (GRADE) was used to assess the certainty of evidence. In total, 43 studies reported on the psychometric properties of 11 PROMs. Structural validity and internal consistency were the parameters evaluated most frequently. Limited information was identified on hypotheses testing for construct validity, reliability, criterion validity, and responsiveness. No data regarding measurement error and cross-cultural validity\measurement invariance were achieved. High-quality evidence on psychometric properties was provided for Self-care of Heart Failure Index (SCHFI) v6.2, SCHFI v7.2, and European Heart Failure Self-care Behavior Scale 9-item (EHFScBS-9). Based on the findings from the included studies, SCHFI v6.2, SCHFI v7.2, and EHFScBS-9 could be recommended to evaluate self-management for CHF patients. Further studies are necessary to evaluate more psychometric properties, such as measurement error, cross-cultural validity\measurement invariance, responsiveness, and criterion validity, and carefully evaluate the content validity. PROSPERO CRD42022322290.

Mepolizumab improvements in health-related quality of life and disease symptoms in a patient population with very severe chronic rhinosinusitis with nasal polyps: psychometric and efficacy analyses from the SYNAPSE study

BackgroundAlthough the psychometric properties of patient-reported outcome measures (e.g. the 22-item Sino-nasal Outcomes Test [SNOT-22]) in chronic rhinosinusitis with nasal polyps (CRSwNP) have been defined, these definitions have not been extensively studied in patients with very severe CRSwNP, as defined by recurrent disease despite ≥ 1 previous surgery and a current need for further surgery. Therefore, the psychometric properties of the symptoms visual analogue scales (VAS) were evaluated, and meaningful within-patient change thresholds were calculated for VAS and SNOT-22.MethodsSYNAPSE (NCT03085797), a randomized, double-blind, placebo-controlled, 52-week trial, assessed the efficacy and safety of 4-weekly mepolizumab 100 mg subcutaneously added to standard of care in very severe CRSwNP. Enrolled patients (n = 407) completed symptom VAS (six items) daily and SNOT-22 every 4 weeks from baseline until Week 52. Blinded psychometric assessment of individual and composite VAS was performed post hoc, including anchor-based thresholds for meaningful within-patient changes for VAS and SNOT-22, supported by cumulative distribution function and probability density function plots. The effect of mepolizumab versus placebo for 52 weeks on VAS and SNOT-22 scores was then determined using these thresholds using unblinded data.ResultsInternal consistency was acceptable for VAS and SNOT-22 scores (Cronbach’s α-coefficients ≥ 0.70). Test–retest reliability was demonstrated for all symptom VAS (Intra-Class Correlation coefficients > 0.75). Construct validity was acceptable between individual and composite VAS and SNOT-22 total score (r = 0.461–0.598) and between individual symptom VAS and corresponding SNOT-22 items (r = 0.560–0.780), based upon pre-specified ranges. Known-groups validity assessment demonstrated generally acceptable validity based on factors associated with respiratory health, with all VAS responsive to change. Mepolizumab treatment was associated with significantly increased odds of meeting or exceeding meaningful within-patient change thresholds, derived for this very severe cohort using six anchor groups for individual VAS (odds ratio [OR] 2.19–2.68) at Weeks 49–52, and SNOT-22 (OR 1.61–2.96) throughout the study.ConclusionsSymptoms VAS and SNOT-22 had acceptable psychometric properties for use in very severe CRSwNP. Mepolizumab provided meaningful within-patient improvements in symptom severity and health-related quality of life versus placebo, indicating mepolizumab provides substantial clinical benefits in very severe CRSwNP.

Properties of Patient-Reported Outcome Measures in Recessive Stargardt Disease

Introduction: The study aimed to explore the psychometric properties of the National Eye Institute Visual Function Questionnaire (NEI VFQ) and Impact of Vision Impairment (IVI) profile in recessive Stargardt disease (STGD1). Methods: The NEI VFQ-25 and IVI-28 were administered to individuals with STGD1. Responses were analyzed following psychometrically established dimension structures of the NEI VFQ (visual function [VF] subscale; socioemotional [SE] subscale) and of the IVI (functional [F] subscale; emotional [E] subscale). We analyzed internal consistency, dimensionality, item fit, and differential item functioning (DIF), using latent trait models. Criterion validity was assessed using Pearson correlation coefficients. Results: Seventy-one participants (42 females, 29 males; mean age, 44 ± 19 years) were included. Self-reported difficulty levels were lower than the mean difficulty of items in both instruments. Person reliability and person separation index of the instruments were 0.85 and 2.40 (NEI VFQ-VF), 0.69 and 1.49 (NEI-VFQ-SE), 0.88 and 2.77 (IVI-F), and 0.72 and 1.62 (IVI-E). No items showed misfit at a level distorting the measurement system. One IVI item showed DIF by gender but was retained as person measures were largely unaffected by its removal. NEI VFQ-VF and IVI-F as well as NEI VFQ-SE and IVI-E were positively correlated (r = 0.79 and 0.64, respectively). Conclusion: The NEI VFQ and IVI have acceptable psychometric properties in STGD1 with the IVI allowing more sensitive person stratification. Targeting of questionnaires to individuals with STGD1 might be improved by including additional content domains specific to the disease.

Psychometric Properties of the Patient-Reported Outcome Measures for People With Ulnar Nerve Entrapment at the Elbow: A Systematic Review.

The objective of this study was to systematically review, critically appraise, and synthesize evidence on the measurement properties of patient-reported outcome measures (PROMs) for people with ulnar nerve entrapment at the elbow (UNE). CINAHL, MEDLINE, Embase, and Scopus were searched for studies reporting on the psychometric properties of the PROMs used to assess pain, disability, and quality of life of people with UNE. Relevant data were extracted based on the Consensus-Based Standards for the Selection of Health Measurement Instruments 2018 checklist. Two authors independently rated the quality and risk of bias of the studies. A qualitative synthesis was performed according to the Consensus-Based Standards for the Selection of Health Measurement Instruments guidelines. The results were categorized based on the identified PROMs, and all the psychometric properties were summarized and presented. Eight included studies had a quality score of very good or higher. Among all the included PROMs, the Patient-Rated Ulnar Nerve Evaluation (PRUNE) was comprehensively evaluated in 4 studies and had high reliability, appropriate correlation to the physical component summary score of the 36-Item Short-Form Health Survey (r = -0.68), excellent content (cognitive interviews), structural (factor analysis) and discriminative validities, and high responsiveness. The Disabilities of the Arm, Shoulder and Hand Questionnaire was assessed in 5 studies and had appropriate properties, showing a high correlation with the 8-Item Short-Form Health Survey (r = 0.77) and PRUNE (r = 0.75) and moderate responsiveness. The PRUNE was the most specific to UNE, had the most supporting studies and evidence of excellent reliability, validity, and responsiveness and therefore was the preferred PROM. The Disabilities of the Arm, Shoulder and Hand Questionnaire demonstrated more limited validation across 5 studies and had moderate responsiveness. Information on other included PROMs was limited in terms of psychometric properties. This study provides insight into which PROM is potentially more suitable for measuring outcomes related to UNE.

Evaluation of psychometric properties of patient-reported outcome measures frequently used in narcolepsy randomized controlled trials: a systematic review.

Study ObjectivesTo systematically determine subjective and objective outcome measures used to measure the efficacy of narcolepsy interventions in randomized controlled trials (RCTs) in adults and children and assess psychometric properties of patient-reported outcome measures (PROMs) used.MethodsWe searched bibliographical databases and clinical trial registries for narcolepsy RCTs and extracted objective and subjective outcome measures. If PROMs were used, we searched for psychometric studies conducted in a narcolepsy population using bibliographical databases and appraised using Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) guidelines.ResultsIn total, 80 different outcome measures were used across 100 RCTs. Epworth Sleepiness Scale (ESS) (n = 49) and Maintenance of Wakefulness Test (n = 47) were the most frequently used outcome measures. We found 19 validation studies of 10 PROMs in narcolepsy populations. There was limited evidence for validity or responsiveness of the ESS; yet sufficient reliability (pooled ICC: 0.81–0.87). Narcolepsy Severity Scale (NSS) had sufficient reliability (pooled ICC: 0.71–0.92) and both adult and pediatric versions had sufficient discriminant validity (treated/untreated). Content validity was only evaluated in pediatric populations for ESS-CHAD and NSS-P and rated inconclusive. Quality of evidence of the psychometric studies for all scales ranged from very low to low.ConclusionsAlthough recognized by regulatory bodies and widely used as primary outcome measures in trials, there is surprisingly little evidence for the validity, reliability, and responsiveness of PROMs frequently used to assess treatment efficacy in narcolepsy. The field needs to establish patient-centered minimal clinically important differences for the PROMs used in these trials.

Psychometric properties of patient-reported outcome measures (PROMs) in wrist osteoarthritis: test–retest reliability and construct validity

BackgroundPatient-reported outcome measures (PROMs) are frequently used to assess the effects of treatments in patients with wrist osteoarthritis (OA), but their psychometric properties have not been evaluated in this group of patients. Our aim was to evaluate the psychometric properties of the Numeric Rating Scale (NRS pain at rest, pain on motion without load, and pain on load), the Disabilities of the Arm, Shoulder and Hand (DASH) and the Patient Rated Wrist Evaluation (PRWE) questionnaires in patients with wrist OA regarding test–retest reliability and construct validity.MethodsThe NRS, DASH and PRWE were self-administered by 50 patients (40 men and 10 women, mean age 66 years) in a postal survey on two occasions, two weeks apart. Test–retest reliability was evaluated by Kappa statistics and the Spearman rank correlation coefficients (rho) were calculated to evaluate construct validity.ResultsThe Kappa coefficients for DASH, PRWE and NRS pain on motion without load and NRS pain on load were > 0.90, 95% CI ranging from 0.84 to 0.98, while NRS pain at rest was 0.83, 95% CI 0.73–0.92. The construct validity of the PROMs was confirmed by three formulated hypotheses: a higher correlation between PRWE and NRS (rho 0.80–0.91, p < 0.001) was found, compared to DASH and NRS (rho 0.68–0.80, p < 0.001); the NRS pain on motion without load and NRS pain on load correlated more strongly to PRWE and DASH (rho 0.71–0.91, p < 0.001) compared to NRS pain at rest (rho 0.68–0.80) and a high correlation between PRWE and DASH was found (rho 0.86, p < 0.001).ConclusionsThe NRS, DASH and PRWE demonstrate excellent test–retest reliability and moderate to high construct validity in patients with wrist OA. These PROMs are highly related, but they also differ. Therefore, they complement each other in ensuring a comprehensive evaluation of perceived disability in wrist OA. As PRWE showed the highest test–retest reliability and the highest relation to the other PROMs, the sole use of the PRWE can be recommended in clinical practice.