Estimating anchor-based minimal important change using longitudinal confirmatory factor analysis.

PURPOSE: The minimal important change (MIC) is defined as the smallest within-individual change in a patient-reported outcome measure (PROM) that patients on average perceive as important. We describe a method to estimate this value based on longitudinal confirmatory factor analysis (LCFA). The method is evaluated and compared with a recently published method based on longitudinal item response theory (LIRT) in simulated and real data. We also examined the effect of sample size on bias and precision of the estimate. METHODS: We simulated 108 samples with various characteristics in which the true MIC was simulated as the mean of individual MICs, and estimated MICs based on LCFA and LIRT. Additionally, both MICs were estimated in existing PROMIS Pain Behavior data from 909 patients. In another set of 3888 simulated samples with sample sizes of 125, 250, 500, and 1000, we estimated LCFA-based MICs. RESULTS: The MIC was equally well recovered with the LCFA-method as using the LIRT-method, but the LCFA analyses were more than 50 times faster. In the Pain Behavior data (with higher scores indicating more pain behavior), an LCFA-based MIC for improvement was estimated to be 2.85 points (on a simple sum scale ranging 14-42), whereas the LIRT-based MIC was estimated to be 2.60. The sample size simulations showed that smaller sample sizes decreased the precision of the LCFA-based MIC and increased the risk of model non-convergence. CONCLUSION: The MIC can accurately be estimated using LCFA, but sample sizes need to be preferably greater than 125.

Extending the PROMIS item bank "ability to participate in social roles and activities": a psychometric evaluation using IRT.

OBJECTIVE: Our objective was to explore whether the extension of the PROMIS item bank Ability to Participate in Social Roles and Activities (APSRA) with new items would result in more effective targeting (i.e., selecting items that are appropriate for each individual's trait level), and more reliable measurements across all latent trait levels. METHODS: A sample of 1,022 Dutch adults completed all 35 items of the original item bank plus 17 new items (in Dutch). The new items presented in this publication have been translated provisionally from Dutch into English for presentation purposes. We evaluated the basic IRT assumptions unidimensionality, local independence, and monotonicity. Furthermore, we examined the item parameters, and assessed differential item functioning (DIF) for sex, education, region, age, and ethnicity. In addition, we compared the test information functions, item parameters, and θ scores, for the original and extended item bank in order to assess whether the measurement range had improved. RESULTS: We found that the extended item bank was compatible with the basic IRT assumptions and showed good reliability. Moreover, the extended item bank improved the measurement in the lower trait range, which is important for reliably assessing functioning in clinical populations (i.e., persons reporting lower levels of participation). CONCLUSION: We extended the PROMIS-APSRA item bank and improved its psychometric quality. Our study contributes to PROMIS measurement innovation, which allows for the addition of new items to existing item banks, without changing the interpretation of the scores and while maintaining the comparability of the scores with other PROMIS instruments.

Methodological quality of 100 recent systematic reviews of health-related outcome measurement instruments: an overview of reviews.

PURPOSE: Systematic reviews evaluating and comparing the measurement properties of outcome measurement instruments (OMIs) play an important role in OMI selection. Earlier overviews of review quality (2007, 2014) evidenced substantial concerns with regards to alignment to scientific standards. This overview aimed to investigate whether the quality of recent systematic reviews of OMIs lives up to the current scientific standards. METHODS: One hundred systematic reviews of OMIs published from June 1, 2021 onwards were randomly selected through a systematic literature search performed on March 17, 2022 in MEDLINE and EMBASE. The quality of systematic reviews was appraised by two independent reviewers. An updated data extraction form was informed by the earlier studies, and results were compared to these earlier studies' findings. RESULTS: A quarter of the reviews had an unclear research question or aim, and in 22% of the reviews the search strategy did not match the aim. Half of the reviews had an incomprehensive search strategy, because relevant search terms were not included. In 63% of the reviews (compared to 41% in 2014 and 30% in 2007) a risk of bias assessment was conducted. In 73% of the reviews (some) measurement properties were evaluated (58% in 2014 and 55% in 2007). In 60% of the reviews the data were (partly) synthesized (42% in 2014 and 7% in 2007); evaluation of measurement properties and data syntheses was not conducted separately for subscales in the majority. Certainty assessments of the quality of the total body of evidence were conducted in only 33% of reviews (not assessed in 2014 and 2007). The majority (58%) did not make any recommendations on which OMI (not) to use. CONCLUSION: Despite clear improvements in risk of bias assessments, measurement property evaluation and data synthesis, specifying the research question, conducting the search strategy and performing a certainty assessment remain poor. To ensure that systematic reviews of OMIs meet current scientific standards, more consistent conduct and reporting of systematic reviews of OMIs is needed.

The minimal important change is not a universal fixed value across diagnoses when using the FAOS and FAAM in patients undergoing elective foot and ankle surgery.

PURPOSE: This study aimed to calculate region and diagnosis-specific minimal important changes (MICs) of the Foot and Ankle Outcome Score (FAOS) and the Foot and Ankle Ability Measure (FAAM) in patients requiring foot and ankle surgery and to assess their variability across different foot and ankle diagnoses. METHODS: The study used routinely collected data from patients undergoing elective foot and ankle surgery. Patients had been invited to complete the FAOS and FAAM preoperatively and at 3-6 months after surgery, along with two anchor questions encompassing change in pain and daily function. Patients were categorised according to region of pathology and subsequent diagnoses. MICs were calculated using predictive modelling (MICPRED) and receiver operating characteristic curve (MICROC) method and evaluated according to strict credibility criteria. RESULTS: Substantial variability of the MICs between forefoot and ankle/hindfoot region was observed, as well as among specific foot and ankle diagnoses, with MICPRED and MICROC values ranging from 7.8 to 25.5 points and 9.4 to 27.8, respectively. Despite differences between MICROC and MICPRED estimates, both calculation methods exhibited largely consistent patterns of variation across subgroups, with forefoot conditions systematically showing smaller MICs than ankle/hindfoot conditions. Most MICs demonstrated high credibility; however, the majority of the MICs for the FAOS symptoms subscale and forefoot conditions exhibited insufficient or low credibility. CONCLUSION: The MICs of the FAOS and FAAM vary across foot and ankle diagnoses in patients undergoing elective foot and ankle surgery and should not be used as a universal fixed value, but recognised as contextual parameters. This can help clinicians and researchers in more accurate interpretation of the FAOS and FAAM change scores. LEVEL OF EVIDENCE: Level IV.

Patient-reported outcomes for people with diabetes: what and how to measure? A narrative review.

Patient-reported outcomes (PROs) are valuable for shared decision making and research. Patient-reported outcome measures (PROMs) are questionnaires used to measure PROs, such as health-related quality of life (HRQL). Although core outcome sets for trials and clinical practice have been developed separately, they, as well as other initiatives, recommend different PROs and PROMs. In research and clinical practice, different PROMs are used (some generic, some disease-specific), which measure many different things. This is a threat to the validity of research and clinical findings in the field of diabetes. In this narrative review, we aim to provide recommendations for the selection of relevant PROs and psychometrically sound PROMs for people with diabetes for use in clinical practice and research. Based on a general conceptual framework of PROs, we suggest that relevant PROs to measure in people with diabetes are: disease-specific symptoms (e.g. worries about hypoglycaemia and diabetes distress), general symptoms (e.g. fatigue and depression), functional status, general health perceptions and overall quality of life. Generic PROMs such as the 36-Item Short Form Health Survey (SF-36), WHO Disability Assessment Schedule (WHODAS 2.0), or Patient-Reported Outcomes Measurement Information System (PROMIS) measures could be considered to measure commonly relevant PROs, supplemented with disease-specific PROMs where needed. However, none of the existing diabetes-specific PROM scales has been sufficiently validated, although the Diabetes Symptom Self-Care Inventory (DSSCI) for measuring diabetes-specific symptoms and the Diabetes Distress Scale (DDS) and Problem Areas in Diabetes (PAID) for measuring distress showed sufficient content validity. Standardisation and use of relevant PROs and psychometrically sound PROMs can help inform people with diabetes about the expected course of disease and treatment, for shared decision making, to monitor outcomes and to improve healthcare. We recommend further validation studies of diabetes-specific PROMs that have sufficient content validity for measuring disease-specific symptoms and consider generic item banks developed based on item response theory for measuring commonly relevant PROs.

Validity and reliability of the Patient-Reported Outcomes Measurement Information System (PROMIS®) using computerized adaptive testing in patients with advanced chronic kidney disease.

BACKGROUND: The Patient-Reported Outcomes Measurement Information System (PROMIS®) has been recommended for computerized adaptive testing (CAT) of health-related quality of life. This study compared the content, validity, and reliability of seven PROMIS CATs to the 12-item Short-Form Health Survey (SF-12) in patients with advanced chronic kidney disease. METHODS: Adult patients with chronic kidney disease and an estimated glomerular filtration rate under 30 mL/min/1.73 m2 who were not receiving dialysis treatment completed seven PROMIS CATs (assessing physical function, pain interference, fatigue, sleep disturbance, anxiety, depression, and the ability to participate in social roles and activities), the SF-12, and the PROMIS Pain Intensity single item and Dialysis Symptom Index at inclusion and 2 weeks. A content comparison was performed between PROMIS CATs and the SF-12. Construct validity of PROMIS CATs was assessed using Pearson's correlations. We assessed the test-retest reliability of all patient-reported outcome measures by calculating the intraclass correlation coefficient and minimal detectable change. RESULTS: In total, 207 patients participated in the study. A median of 45 items (10 minutes) were completed for PROMIS CATs. All PROMIS CATs showed evidence of sufficient construct validity. PROMIS CATs, most SF-12 domains and summary scores, and Dialysis Symptom Index showed sufficient test-retest reliability (intraclass correlation coefficient ≥ 0.70). PROMIS CATs had a lower minimal detectable change compared with the SF-12 (range, 5.7-7.4 compared with 11.3-21.7 across domains, respectively). CONCLUSION: PROMIS CATs showed sufficient construct validity and test-retest reliability in patients with advanced chronic kidney disease. PROMIS CATs required more items but showed better reliability than the SF-12. Future research is needed to investigate the feasibility of PROMIS CATs for routine nephrology care.

Psychometric properties and reference values of the Patient-Reported Outcomes Measurement Information System (PROMIS) sleep item banks in the Dutch general population.

Psychometric properties of the v1.0 Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance (27 items) and sleep-related impairment (SRI; 16 items) item banks, short forms derived from the item bank, and simulated computerised adaptive test (CAT), were assessed in a representative sample of 1,006 adults from the Dutch general population. For sleep disturbance all items fitted the item response theory model. Four items showed differential item functioning (i.e., lack of measurement invariance) for age and two for language but the impact on scores (expressed as T-scores) was small. Reliable scores (r > 0.90) were found for 92.2%-96.3% of respondents with the full bank, short forms with six and eight items, and CAT, but for only 25.6% with the four-item short form. For SRI two items did not fit the item response theory model. Four items showed differential item functioning for language but the impact on T-scores was small. Reliable scores were found for 82.1% with the full bank, for 47.8%-69.5% with short forms and CAT. T-scores of 49.7 and 49.3 represent the average score of the Dutch general population for sleep disturbance and SRI, respectively. In conclusion, sufficient structural validity, reliability, and cross-cultural validity was found for the full banks but short forms of four items are not reliable enough for clinical practice. For SRI we recommend the full item bank if this is the primary outcome.

Estimating meaningful thresholds for multi-item questionnaires using item response theory.

PURPOSE: Meaningful thresholds are needed to interpret patient-reported outcome measure (PROM) results. This paper introduces a new method, based on item response theory (IRT), to estimate such thresholds. The performance of the method is examined in simulated datasets and two real datasets, and compared with other methods. METHODS: The IRT method involves fitting an IRT model to the PROM items and an anchor item indicating the criterion state of interest. The difficulty parameter of the anchor item represents the meaningful threshold on the latent trait. The latent threshold is then linked to the corresponding expected PROM score. We simulated 4500 item response datasets to a 10-item PROM, and an anchor item. The datasets varied with respect to the mean and standard deviation of the latent trait, and the reliability of the anchor item. The real datasets consisted of a depression scale with a clinical depression diagnosis as anchor variable and a pain scale with a patient acceptable symptom state (PASS) question as anchor variable. RESULTS: The new IRT method recovered the true thresholds accurately across the simulated datasets. The other methods, except one, produced biased threshold estimates if the state prevalence was smaller or greater than 0.5. The adjusted predictive modeling method matched the new IRT method (also in the real datasets) but showed some residual bias if the prevalence was smaller than 0.3 or greater than 0.7. CONCLUSIONS: The new IRT method perfectly recovers meaningful (interpretational) thresholds for multi-item questionnaires, provided that the data satisfy the assumptions for IRT analysis.

Suitability of the animated activity questionnaire for use as computer adaptive test: establishing the AAQ-CAT.

PURPOSE: The animated activity questionnaire (AAQ) is a computer-based measure of activity limitations. To answer a question, patients choose the animation of a person performing an activity that matches their own level of limitation. The AAQ has not yet been tested for suitability to be applied as computer-adaptive test (CAT). Thus, the objective of this study was to develop and evaluate an AAQ-based CAT to facilitate the application of the AAQ in daily clinical care. METHODS: Patients (n = 1408) with hip/knee osteoarthritis from Brazil, Denmark, France, The Netherlands, Norway, Spain, and the UK responded to all 17 AAQ items. Assumptions of item-response theory (IRT) modelling were investigated. To establish item parameters for the CAT, a graded response model was estimated. To evaluate the performance of post-hoc simulated AAQ-based CATs, precision, test length, and construct validity (correlations with well-established measures of activity limitations) were evaluated. RESULTS: Unidimensionality (CFI = 0.95), measurement invariance (R2-change < 2%), and IRT item fit (S-X2 p > .003) of the AAQ were supported. Performing simulated CATs, the mean test length was more than halved (≤ 8 items), while the range of precise measurement (standard error ≤ 0.3) was comparable to the full AAQ. The correlations between original AAQ scores and three AAQ-CAT versions were ≥ 0.95. Correlations of AAQ-CAT scores with patient-reported and performance measures of activity limitations were ≥ 0.60. CONCLUSION: The almost non-verbal AAQ-CAT is an innovative and efficient tool in patients with hip/knee osteoarthritis from various countries, measuring activity limitations with lower respondent burden, but similar precision and construct validity compared to the full AAQ.

Development of a standard set of PROs and generic PROMs for Dutch medical specialist care : Recommendations from the Outcome-Based Healthcare Program Working Group Generic PROMs.

PURPOSE: The added value of measuring patient-reported outcomes (PROs) for delivering patient-centered care and assessment of healthcare quality is increasingly evident. However, healthcare system wide data collection initiatives are hampered by the proliferation of patient-reported outcome measures (PROMs) and conflicting data collection standards. As part of a national initiative of the Dutch Ministry of Health, Welfare and Sport we developed a consensus-based standard set of generic PROs and PROMs to be implemented across Dutch medical specialist care. METHODS: A working group of mandated representatives of umbrella organizations involved in Dutch medical specialist care, together with PROM experts and patient organizations worked through a structured, consensus-driven co-creation process. This included literature reviews, online expert and working group meetings, and feedback from national patient- and umbrella organizations. The 'PROM-cycle' methodology was used to select feasible, valid, and reliable PROMs to obtain domain scores for each of the PROs included in the set. RESULTS: Eight PROs across different domains of health were ultimately endorsed: symptoms (pain & fatigue), functioning (physical, social/participation, mental [anxiety & depression]), and overarching (quality of life & perceived overall health). A limited number of generic PROMs was endorsed. PROMIS short forms were selected as the preferred instruments for all PROs. Several recommendations were formulated to facilitate healthcare system level adoption and implementation of the standard set. CONCLUSIONS: We developed a consensus-based standard set of Generic PROMs and a set of recommendations to facilitate healthcare system wide implementation across Dutch medical specialist care.

Clinician-reported outcome measures for the assessment of vitiligo: A scoping review.

Clinician-reported outcome measures (ClinROMs) are essential for assessment of vitiligo in clinical trials and daily practice. Several instruments have been developed and tested to measure, for example, vitiligo extent, repigmentation and activity. The goal of this review was to identify all introductory publications of ClinROMs for vitiligo that include at least some aspects of validation and to describe the instruments' characteristics, intention for use and practical strengths and limitations. A search strategy was conducted in PubMed, Embase and Cochrane Library (CENTRAL) from inception to July 2022. Based on the literature search (n = 2860), 10 articles were identified, describing 14 different ClinROMs. Six ClinRoms measured disease extent and/or repigmentation, seven evaluated disease activity and one was a composite score. The Vitiligo Area Scoring Index (VASI), and Vitiligo Extent Score (VES and VESplus) measure overall disease extent and/or repigmentation. The VASI relies on hand units (1% body surface area), whereas the VES and VESplus use a picture-based scoring technique. The Vitiligo Extent Score for a Target Area (VESTA) measures repigmentation percentage for target lesions. One global assessment score for extent has been validated. Vitiligo disease activity scores included a static measure of clinical activity signs (Vitiligo Signs of Activity Score [VSAS]) and two measures assessing dynamic evolution (Vitiligo Disease Activity Score [VDAS] and Vitiligo Disease Improvement Score [VDIS]). The Vitiligo European Task Force assessment tool (VETFa) is a composite score. Depending on the practical strengths and limitations as well as the research question and setting (clinical trials vs. daily practice), the choice of an appropriate ClinROM may differ. Fourteen ClinROMs in vitiligo were identified to measure vitiligo extent, repigmentation, and activity. Further research evaluating the validity, reliability, and responsiveness of each instrument and worldwide consensus on which instrument to use for a specific outcome (domain) is greatly needed.

Validation of the Dutch Version of the BODY-Q Measuring Appearance, Health-Related Quality of Life, and Experience of Healthcare in Patients Undergoing Bariatric and Body Contouring Surgery.

BACKGROUND: The BODY-Q is a patient-reported outcome measure developed for use in bariatric and body contouring surgery. OBJECTIVES: The objective of this study was to examine the validity and reliability of the Dutch version of the BODY-Q. METHODS: The BODY-Q consists of 163 items in 21 independently functioning scales that measure appearance, health-related quality of life, and experience of care. The data used to validate the Dutch BODY-Q were provided by 2 prospective multicenter cohort studies across 3 hospitals in the Netherlands. The BODY-Q was administered before and after surgery at 3 or 4 months and 12 months. Rasch measurement theory (RMT) analysis was used to evaluate the BODY-Q for targeting, category threshold order, Rasch model fit, Person Separation Index, and differential item functioning by language (original English data vs Dutch data). RESULTS: Data were collected between January 2016 and May 2019. The study included 876 participants, who provided 1614 assessments. Validity was supported by 3 RMT findings: most scales showed good targeting, 160 out of 163 items (98.2%) evidenced ordered thresholds, and 142 out of 163 items (87.1%) fitted the RMT model. Reliability was high with Person Separation Index values >0.70 for 19 out of 21 scales. There was negligible influence of differential item functioning by language on person item locations and the scale scoring. CONCLUSIONS: This study provides evidence for the reliability and validity of the Dutch BODY-Q for use in bariatric and body contouring patients in the Netherlands. The Dutch BODY-Q can be used in (inter)national research and clinical practice.

Patient-reported outcome measures for assessing health-related quality of life in people with type 2 diabetes: A systematic review.

Patient-Reported Outcome Measures (PROMs) are important tools to assess outcomes relevant to patients, with Health-Related Quality Of Life (HRQOL) as an important construct to be measured. Many different HRQOL PROMs are used in the type 2 diabetes field, however a complete overview of these PROMs is currently lacking. We therefore aimed to systematically describe and classify the content of all PROMs that have specifically been developed or validated to measure (aspects of) HRQOL in people with type 2 diabetes. A literature search was performed in PubMed and EMBASE until 31 December 2021. Studies on the development or validation of a PROM measuring HRQOL, or aspects of HRQOL, in people with type 2 diabetes were included. Title and abstract and full-text screening were conducted by two independent researchers and data extraction was performed independently by one of the researchers. Data were extracted on language in which the PROM was developed, target population, construct(s) being measured, names of (sub)scales and number of items per (sub)scale. In addition, all PROMs and subscales were classified according to specific aspects of HRQOL based on the Wilson & Cleary model (symptom status, functional status, general health perceptions) to aid researchers in PROM selection. In total 220 studies were identified that developed or validated PROMs that measure (aspects of) HRQOL in people with type 2 diabetes. Of the 116 unique HRQOL PROMs, 91 (of the subscales) measured symptom status, 60 measured functional status and 26 measured general health perceptions. In addition, 16 of the PROMs (subscales) measured global quality of life. 61 of the 116 PROMs (subscales) also include characteristics of the individual (e.g. aspects of personality, coping) or environment (e.g. social or financial support) and patient-reported experience measures (PREMs, e.g. measure of a patient's perception of their personal experience of the healthcare they have received, e.g. treatment satisfaction), which are not part of the HRQOL construct. Only 9 of the 116 PROMs measure all aspects of HRQOL based on the Wilson & Cleary model. Finally, 8 of the 116 PROMs stating to measure HRQOL, measured no HRQOL construct. In conclusion, a large number of PROMs are available for people with type 2 diabetes, which intend to measure (aspects of) HRQOL. These PROMs measure a large variety of (sub)constructs, which are not all HRQOL constructs, with a small amount of PROMs not measuring HRQOL at all. There is a need for consensus on which aspects of HRQOL should be measured in people with type 2 diabetes and which PROMs to use in research and daily practice. PROSPERO: CRD42017071012. COMET database: http://www.comet-initiative.org/studies/details/956 .

Content Validity of Patient-Reported Outcome Measures Developed for Assessing Health-Related Quality of Life in People with Type 2 Diabetes Mellitus: a Systematic Review.

PURPOSE OF REVIEW: We aimed to systematically evaluate the content validity of patient-reported outcome measures (PROMs) specifically developed to measure (aspects of) health-related quality of life (HRQOL) in people with type 2 diabetes. A systematic review was performed in PubMed and Embase of PROMs measuring perceived symptoms, physical function, mental function, social function/participation, and general health perceptions, and that were validated to at least some extent. Content validity (relevance, comprehensiveness, and comprehensibility) was evaluated using COSMIN methodology. RECENT FINDINGS: We identified 54 (different versions of) PROMs, containing 150 subscales. We found evidence for sufficient content validity for only 41/150 (27%) (subscales of) PROMs. The quality of evidence was generally very low. We found 66 out of 150 (44%) (subscales of) PROMs with evidence for either insufficient relevance, insufficient comprehensiveness, or insufficient comprehensibility. For measuring diabetes-specific symptoms, physical function, mental function, social function/participation, and general health perceptions, we identified one to 11 (subscales of) PROMs with sufficient content validity, although quality of the evidence was generally low. For measuring depressive symptoms, no PROM with sufficient content validity was identified. For each aspect of HRQL, we found at least one PROM with sufficient content validity, except for depressive symptoms. The quality of the evidence was mostly very low.

Giving meaning to the scores of the Amsterdam instrumental activities of daily living questionnaire: a qualitative study.

BACKGROUND: Everyday functioning is a clinically relevant concept in dementia, yet little is known about the clinical meaningfulness of scores on functional outcome measures. We aimed to establish clinically meaningful scoring categories for the Amsterdam Instrumental Activities of Daily Living Questionnaire (A-IADL-Q), representing no, mild, moderate and severe problems in daily functioning. METHODS: Informal caregivers (n = 6) of memory-clinic patients and clinicians (n = 13), including neurologists and nurse specialists, working at various memory clinics in The Netherlands. In focus groups, participants individually ranked nine summaries of fictional patients from least to most impairment in daily functioning. Then, they placed bookmarks to demarcate the thresholds for mild, moderate and severe problems. Individual bookmark placements were then discussed to reach consensus. Clinicians completed a survey in which they placed bookmarks, individually. RESULTS: While individual categorizations varied somewhat, caregivers and clinicians generally agreed on the thresholds, particularly about the distinction between 'no' and 'mild' problems. Score categories were no problems (T-score ≥ 60), mild problems (T-score 50-59), moderate problems (T-score 40-49), and severe problems in daily functioning (T-score < 40), on a scale ranging 20-80. CONCLUSIONS: Our findings provide categories for determining the level of functional impairment, which can facilitate interpretation of A-IADL-Q scores. These categories can subsequently be used by clinicians to improve communication with patients and caregivers.

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review.

PURPOSE: Both the International Consortium for Health Outcomes Measurement and the National Institutes of Health recommend the use of Patient-Reported Outcomes Measurement Information System (PROMIS®) measures in clinical care and research for stroke patients. This study aimed to systematically review the literature on the measurement properties and interpretability of PROMIS measures in stroke patients. METHODS: Nine databases were searched from January 1st, 2007 till April 12th, 2021 for studies concerning the measurement properties and interpretability of PROMIS measures in stroke patients. The findings of these studies were analyzed according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline for systematic reviews of Patient-Reported Outcome Measures (PROMs). RESULTS: Ten studies were included. The PROMIS Global Health was studied the most: its two subscales had sufficient structural validity in one study of very good quality, sufficient construct validity with > 75% of hypotheses tested confirmed (high GRADE rating), sufficient internal consistency, i.e. α ≥ 0.70 in two studies (high GRADE rating), sufficient reliability, i.e. ICC ≥ 0.70 in one study of doubtful quality, and indeterminate responsiveness in one study of inadequate quality. For other PROMIS measures, the measurement properties and interpretability were limitedly studied. CONCLUSION: The PROMIS Global Health showed sufficient structural and construct validity and internal consistency in stroke patients. There is a need for further research on content validity, structural validity, and measurement invariance of PROMIS measures in stroke patients. Trial Registration Information: CRD42020203044 (PROSPERO).

Measurement properties of the Dutch PROMIS-29 v2.1 profile in people with and without chronic conditions.

PURPOSE: To investigate the structural validity, internal consistency, measurement invariance, and construct validity of the Dutch PROMIS-29 v2.1 profile, including seven physical (e.g., pain, physical function), mental (e.g., depression, anxiety), and social (e.g., role functioning) domains of health, in a Dutch general population sample including subsamples with and without chronic diseases. METHODS: The PROMIS-29 was completed by 63,602 participants from the Lifelines cohort study. Structural validity of the PROMIS-29, including unidimensionality of each domain and the physical and mental health summary scores, was evaluated using factor analyses (criteria: CFI ≥ 0.95, TLI ≥ 0.95, RMSEA ≤ 0.06, SRMR ≤ 0.08). Internal consistency, measurement invariance (no differential item functioning (DIF) for age, gender, administration mode, educational level, ethnicity, chronic diseases), and construct validity (hypotheses on known-groups validity and correlations between domains) were assessed per domain. RESULTS: The factor structure of the seven domains was supported (CFI = 0.994, TLI = 0.993, RMSEA = 0.046, SRMR = 0.031) as was unidimensionality of each domain, both in the entire sample and the subsamples. Model fit of the physical and mental health summary scores reached the criteria, and scoring coefficients were obtained. Cronbach's alpha for the seven PROMIS-29 domains ranged from 0.75 to 0.96 in the complete sample. No DIF was detected. Of the predefined hypotheses, 78% could be confirmed. CONCLUSION: Sufficient structural validity, internal consistency and measurement invariance were found, both in the entire sample and in subsamples with and without chronic diseases. Requirements for sufficient evidence for construct validity were (almost) met for most subscales. Future studies should investigate test-retest reliability, measurement error, and responsiveness of the PROMIS-29.

Advances in measuring pediatric overall health: the PROMIS® Pediatric Global Health scale (PGH-7).

In this cross-sectional study, we aimed to assess the reliability, validity, and efficiency of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Global Health scale (PGH-7) to reduce patient burden when assessing overall health in clinical practice. In total, 1082 children (8-18), representative of the Dutch population, completed the PGH-7 and the Pediatric Quality of Life Inventory (PedsQL™ 4.0), a common legacy instrument used in clinical practice to assess overall health. The assumptions for fitting an item response theory model were assessed: unidimensionality, local independence, and monotonicity. Subsequently, a model was fitted to the data to assess item fit and cultural differential item functioning (DIF) between Dutch and US children. A strong correlation (> .70) was expected between the PGH-7 and PedsQL, as both instruments measure physical, mental, and social domains of health. Percentages of participants reliably measured (> 0.90) were assessed using the standard error of measurement (SE(θ) < 0.32). Efficiency was calculated ((1 - SE(θ)2)/nitems) to compare how well both measures performed relative to number of items administered. The PGH-7 met all assumptions and displayed good structural and convergent (r = .69) validity. One item displayed cultural DIF. Both questionnaires measured reliably (%nPGH-7 = 73.8%, %nPedsQL = 76.6%) at the mean and 2SD in clinically relevant direction. PGH-7 items were 2.6 times more efficient in measuring overall health than the PedsQL.   Conclusion: The PGH-7 displays sufficient validity and reliability in the general Dutch pediatric population and measures more efficiently than the PedsQL, the most commonly used legacy instrument. The PGH-7 can be used in research and clinical practice to reduce patient burden when assessing overall health. What is Known: • Generic instruments which validly and reliably assess overall pediatric health are scarce. • Brief instruments are required for implementation of self-report patient-reported outcomes in clinical practice. What is New: • The PROMIS Pediatric Global Health (PGH-7) can be used in research and clinical practice to briefly assess overall pediatric health, while providing valid and reliable measurements. • The PGH-7 provides more efficient assessment of pediatric overall health than the Pediatric Quality of Life Inventory.

Guidelines for Reporting Outcomes in Trial Reports: The CONSORT-Outcomes 2022 Extension.

Importance: Clinicians, patients, and policy makers rely on published results from clinical trials to help make evidence-informed decisions. To critically evaluate and use trial results, readers require complete and transparent information regarding what was planned, done, and found. Specific and harmonized guidance as to what outcome-specific information should be reported in publications of clinical trials is needed to reduce deficient reporting practices that obscure issues with outcome selection, assessment, and analysis. Objective: To develop harmonized, evidence- and consensus-based standards for reporting outcomes in clinical trial reports through integration with the Consolidated Standards of Reporting Trials (CONSORT) 2010 statement. Evidence Review: Using the Enhancing the Quality and Transparency of Health Research (EQUATOR) methodological framework, the CONSORT-Outcomes 2022 extension of the CONSORT 2010 statement was developed by (1) generation and evaluation of candidate outcome reporting items via consultation with experts and a scoping review of existing guidance for reporting trial outcomes (published within the 10 years prior to March 19, 2018) identified through expert solicitation, electronic database searches of MEDLINE and the Cochrane Methodology Register, gray literature searches, and reference list searches; (2) a 3-round international Delphi voting process (November 2018-February 2019) completed by 124 panelists from 22 countries to rate and identify additional items; and (3) an in-person consensus meeting (April 9-10, 2019) attended by 25 panelists to identify essential items for the reporting of outcomes in clinical trial reports. Findings: The scoping review and consultation with experts identified 128 recommendations relevant to reporting outcomes in trial reports, the majority (83%) of which were not included in the CONSORT 2010 statement. All recommendations were consolidated into 64 items for Delphi voting; after the Delphi survey process, 30 items met criteria for further evaluation at the consensus meeting and possible inclusion in the CONSORT-Outcomes 2022 extension. The discussions during and after the consensus meeting yielded 17 items that elaborate on the CONSORT 2010 statement checklist items and are related to completely defining and justifying the trial outcomes, including how and when they were assessed (CONSORT 2010 statement checklist item 6a), defining and justifying the target difference between treatment groups during sample size calculations (CONSORT 2010 statement checklist item 7a), describing the statistical methods used to compare groups for the primary and secondary outcomes (CONSORT 2010 statement checklist item 12a), and describing the prespecified analyses and any outcome analyses not prespecified (CONSORT 2010 statement checklist item 18). Conclusions and Relevance: This CONSORT-Outcomes 2022 extension of the CONSORT 2010 statement provides 17 outcome-specific items that should be addressed in all published clinical trial reports and may help increase trial utility, replicability, and transparency and may minimize the risk of selective nonreporting of trial results.

Guidelines for Reporting Outcomes in Trial Protocols: The SPIRIT-Outcomes 2022 Extension.

Importance: Complete information in a trial protocol regarding study outcomes is crucial for obtaining regulatory approvals, ensuring standardized trial conduct, reducing research waste, and providing transparency of methods to facilitate trial replication, critical appraisal, accurate reporting and interpretation of trial results, and knowledge synthesis. However, recommendations on what outcome-specific information should be included are diverse and inconsistent. To improve reporting practices promoting transparent and reproducible outcome selection, assessment, and analysis, a need for specific and harmonized guidance as to what outcome-specific information should be addressed in clinical trial protocols exists. Objective: To develop harmonized, evidence- and consensus-based standards for describing outcomes in clinical trial protocols through integration with the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) 2013 statement. Evidence Review: Using the Enhancing the Quality and Transparency of Health Research (EQUATOR) methodological framework, the SPIRIT-Outcomes 2022 extension of the SPIRIT 2013 statement was developed by (1) generation and evaluation of candidate outcome reporting items via consultation with experts and a scoping review of existing guidance for reporting trial outcomes (published within the 10 years prior to March 19, 2018) identified through expert solicitation, electronic database searches of MEDLINE and the Cochrane Methodology Register, gray literature searches, and reference list searches; (2) a 3-round international Delphi voting process (November 2018-February 2019) completed by 124 panelists from 22 countries to rate and identify additional items; and (3) an in-person consensus meeting (April 9-10, 2019) attended by 25 panelists to identify essential items for outcome-specific reporting to be addressed in clinical trial protocols. Findings: The scoping review and consultation with experts identified 108 recommendations relevant to outcome-specific reporting to be addressed in trial protocols, the majority (72%) of which were not included in the SPIRIT 2013 statement. All recommendations were consolidated into 56 items for Delphi voting; after the Delphi survey process, 19 items met criteria for further evaluation at the consensus meeting and possible inclusion in the SPIRIT-Outcomes 2022 extension. The discussions during and after the consensus meeting yielded 9 items that elaborate on the SPIRIT 2013 statement checklist items and are related to completely defining and justifying the choice of primary, secondary, and other outcomes (SPIRIT 2013 statement checklist item 12) prospectively in the trial protocol, defining and justifying the target difference between treatment groups for the primary outcome used in the sample size calculations (SPIRIT 2013 statement checklist item 14), describing the responsiveness of the study instruments used to assess the outcome and providing details on the outcome assessors (SPIRIT 2013 statement checklist item 18a), and describing any planned methods to account for multiplicity relating to the analyses or interpretation of the results (SPIRIT 2013 statement checklist item 20a). Conclusions and Relevance: This SPIRIT-Outcomes 2022 extension of the SPIRIT 2013 statement provides 9 outcome-specific items that should be addressed in all trial protocols and may help increase trial utility, replicability, and transparency and may minimize the risk of selective nonreporting of trial results.