患者自我报告结局与临床主观评分的标准化困境

The NIH Pragmatic Trials Collaboratory supports the design and conduct of 27 embedded pragmatic clinical trials, and many of the studies collect patient reported outcome measures as primary or secondary outcomes. Study teams have encountered challenges in the collection of these measures, including challenges related to competing health care system priorities, clinician’s buy-in for adoption of patient-reported outcome measures, low adoption and reach of technology in low resource settings, and lack of consensus and standardization of patient-reported outcome measure selection and administration in the electronic health record. In this article, we share case examples and lessons learned, and suggest that, when using patient-reported outcome measures for embedded pragmatic clinical trials, investigators must make important decisions about whether to use data collected from the participating health system’s electronic health record, integrate externally collected patient-reported outcome data into the electronic health record, or collect these data in separate systems for their studies.

Standardization of treatment outcomes in randomized clinical trials (RCTs) for active, moderate-to-severe Graves’ orbitopathy (GO) is needed to make results of different RCTs comparable and to draw sound conclusions on the efficacy of a given treatment. Both subjective patient-reported outcome (PRO) and objective clinician-reported outcome (CRO) are important in this regard. In this paper, it is proposed that primary PRO should be the evaluation of treatment-related changes in the quality of life by the use of a validated and disease-specific questionnaire (GO-QoL). The proposed primary CRO is a revised composite index, which includes only objective items and provides an overall assessment of the effects of treatment. Secondary outcomes should also be provided in RCTs to show the effects of treatment on individual features of GO, as well on persistence of activity (by the 7-item Clinical Activity Score), safety, relapses of GO, need for subsequent medical and/or surgical treatments, and other indicators (orbital volume, cytokines, TSH receptor antibody levels). Assessment of the overall response to treatment by primary and secondary outcomes should be made 3 months after treatment completion.

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Randomized controlled clinical trials and real-world observational studies provide complementary information but with different validity. Some clinical questions (disease behavior, prognosis, validation of outcome measures, comparative effectiveness, and long-term safety of therapies) are often better addressed using real-world data reflecting larger, more representative populations. Integration of disease history, clinician-reported outcomes, performance tests, and patient-reported outcome measures during patient encounters; imaging and biospecimen analyses; and data from wearable devices increase dataset utility. However, observational studies utilizing these data are susceptible to many potential sources of bias, creating barriers to acceptance by regulatory agencies and the medical community. Therefore, data standardization and validation within datasets, harmonization across datasets, and application of appropriate analysis methods are important considerations. We review approaches to improve the scope, quality, and analyses of real-world data to advance understanding of multiple sclerosis and its treatment, as an example of opportunities to better support patient care and research.

No abstract available

Objectives A new method is presented for both synthesizing treatment effects on multiple outcomes subject to measurement error and estimating coherent mapping coefficients between all outcomes. It can be applied to sets of trials reporting different combinations of patient- or clinician-reported outcomes, including both disease-specific measures and generic health-related quality-of-life measures. It is underpinned by a structural equation model that includes measurement error and latent common treatment effect factor. Treatment effects can be expressed on any of the test instruments that have been used. Methods This is illustrated in a synthesis of eight placebo-controlled trials of TNF-α inhibitors in ankylosing spondylitis, each reporting treatment effects on between two and five of a total six test instruments. Results The method has advantages over other methods for synthesis of multiple outcome data, including standardization and multivariate normal synthesis. Unlike standardization, it allows synthesis of treatment effect information from test instruments sensitive to different underlying constructs. It represents a special case of previously proposed multivariate normal models for evidence synthesis, but unlike the former, it also estimates mappings. Combining synthesis and mapping as a single operation makes more efficient use of available data than do current mapping methods and generates treatment effects that are consistent with the mappings. A limitation, however, is that it can only generate mappings to and from those instruments on which some trial data exist. Conclusions The method should be assessed in a wide range of data sets on different clinical conditions, before it can be used routinely in health technology assessment.

Clinical outcomes are daily used in head and neck oncology clinical practice. Clinical outcome assessments can be classified into categories according to who makes the report: clinician-reported outcome assessment (health professional); observer-reported outcome assessment (caregiver) and patient-reported outcome assessment (patient or proxies). We present our opinion and we share our experience concerning routine assessment of Health Related Quality of Life outcomes and its incorporation in head and neck cancer clinical practice. The critical factors for successful implementation we identified in a decade of experience were: the institutional approval and support, the inclusion of all first line caregivers, the standardization of team objectives and institutional proposes; team formation and training, promotion of defined workflow in daily clinical practice and the continuous monitoring and analysis. Ten years after, our team developed a much broader and inclusive understanding of Health Related Quality of Life importance in the course of the disease. Head and neck cancer patients consider the questionnaires administration as part of the clinical approach they know that someone, somewhere, will use these patient-centered outcomes to improve healthcare quality – this is also our main goal.

Abstract Purpose To compare digitally fabricated complete dentures to conventionally fabricated dentures using patient‐ and clinician‐reported outcome measures. Methods This review was structured according to PRISMA guidelines with the protocol registered in the PROSPERO database (CRD42024526069). An electronic search of the databases with a defined search strategy was completed within PubMed/MEDLINE and Web of Science from January 2000 to March 2024. Grey literature and article references were searched. Articles were screened by title and abstract, and the remaining articles were screened by full‐text review. Articles accepted for inclusion were subjected to a risk‐of‐bias assessment using Cochrane Collaboration tools (RoB 2 and ROBINS‐I). Results From an initial pool of 704 articles, 15 studies met the selection criteria, of which the majority were published within the past 3 years. Within the included studies, there was inconsistency in the assessment methods of patient‐ and clinician‐reported outcomes, making it challenging to draw definitive conclusions. Generally, digital dentures had superior cost‐effectiveness and prosthesis fabrication time. Patient satisfaction and denture quality were not consistently improved with digital technology. Conclusions Studies showed indications of patient satisfaction with digital and conventional dentures. Digital technology may enhance clinical workflows. A trend emerged that milled dentures performed better than printed dentures. Clinicians adopting digital technology into removable prosthodontics may have a learning curve to overcome, and they should consider the patient‐clinician relationship in addition to clinical outcomes to achieve patient satisfaction. Additional studies with standardized tools for assessing patient satisfaction are required to enable meaningful comparisons between digital and conventional workflows.

ABSTRACT Objectives To evaluate the current patient‐reported outcome measures (PROMs) and clinician‐reported outcomes (ClinROs) following sinus floor elevation (SFE) for implant‐based rehabilitation in the fully edentulous maxilla. Methods A systematic search was conducted across PubMed, EMBASE, Scopus, and the Cochrane Central Register of Controlled Trials, covering publications from 2013 to 2024. We included prospective studies assessing PROMs and ClinROs. The COSMIN checklist was used to evaluate PROMs quality. Results Eighteen studies met the inclusion criteria, comprising randomized controlled trials, controlled clinical trials, and case series. Fourteen distinct ClinROs were identified, predominantly implant survival (61.11%) and peri‐implant bone‐level changes (50%). Conversely, ClinROs directly associated with SFE procedures—such as maxillary sinus bone height/volume changes (27.7%) and sinus membrane perforation (10.5%)—were underreported. Prosthetic outcomes were addressed in 11.11% of studies, and PROMs in 50% of the studies. Seven different PROMs were identified, with a focus on masticatory function and overall satisfaction. COSMIN analysis revealed that OHIP‐49 and the denture satisfaction questionnaire (DSQ) were the most robust PROMs, scoring “very good” across multiple domains. Patients' overall satisfaction and complaint‐based PROMs lacked validation. Conclusions Current reporting of ClinROs emphasizes implant‐related metrics, while outcomes directly related to SFE are inconsistently reported. Validated PROMs are needed to ensure consistency and reliability in outcome assessment. OHIP‐49 and DSQ demonstrated strong psychometric properties and are recommended as core PROMs. Future studies should incorporate early and long‐term PROMs and adopt a standardized approach to outcome reporting.

ABSTRACT Objectives To identify patient‐reported outcomes (PROs), clinician‐reported outcomes (ClinROs) and patient‐reported outcome measures (PROMs) in studies evaluating the rehabilitation of the edentulous maxilla with implant‐supported fixed prosthesis using short and/or standard‐length and/or zygomatic implants. Materials and Methods A systematic search was conducted in MEDLINE/PubMed, EMBASE, Scopus, Web of Science, Cochrane Central Register of Controlled Trials, and National Clinical Trial Register for articles published between January 1, 2014, and March 24, 2024. Studies reporting both PROMs and ClinROs related to implant therapy in patients with fully edentulous maxillae were eligible for inclusion. Study characteristics, PROMs, and ClinROs were extracted from each article. Study quality was appraised using Cochrane's Risk of Bias 2 tool, Newcastle‐Ottawa Scale, or the Joanna Briggs Institute Critical Appraisal Tool according to the study design. Results The electronic search identified 1981 titles after removing duplicates. Fourteen publications, reporting both ClinROs and PROMs based on 12 studies (14 publications). These studies evaluated a total of 575 participants and 3367 implants. Eight publications focused exclusively on one type of implant design: seven evaluated standard‐length implants and one examined zygomatic implants. The remaining six publications assessed two implant types: four compared zygomatic and standard‐length implants, while two compared short and standard‐length implants. The most frequently used PROMs among the publications (n = 14) were Oral Health‐Related Quality‐of‐Life using OHIP questionnaires (7/14) and patient satisfaction using unstandardized questionnaires (7/14). The most frequently reported objective ClinROs were complication (14/14), marginal bone loss (10/14) assessed using standardized periapical radiographs, implant mobility/stability/failure (7/14), and implant survival (4/14). Subjective ClinROs related to the clinician's perception were reported in only one study (1/14) and focused on swelling after treatment. Conclusions PROMs and objective ClinROs are often reported together in clinical trials involving short, standard‐length or zygomatic implants for implant‐supported fixed prostheses in edentulous maxillae. However, the frequent use of unstandardized questionnaires for PROMs hinders sound comparisons across studies. Subjective ClinROs based on clinician perception are rarely reported.

ABSTRACT Aim To identify patient‐reported outcomes (PROs) and clinician‐reported outcomes (ClinROs) and their outcome measures (PROMs and CROMs) used in prospective studies comparing immediate, early, and delayed implant loading protocols in edentulous maxilla patients receiving implant‐supported prostheses. Materials and Methods Protocol‐driven electronic searches were conducted across MEDLINE, Embase, Scopus, Web of Science, Cochrane databases—January 2014 to May 2024—to identify prospective interventional and observational studies comparing immediate, early, delayed implant loading in maxillary edentulous patients. Zygomatic implants were excluded. Risk of bias was performed using Cochrane RoB 2 and ROBINS‐I, and results were reported descriptively. Results Five studies were included, of which four compared immediate versus delayed loading and one study compared immediate versus early loading. PROMs like visual analog scale (VAS) tool (reported in 80%) to evaluate 7 PROs: pain, overall satisfaction, comfort, speech, masticatory function, esthetics, and self‐esteem. Three studies reported Oral Health Impact Profile (OHIP‐19, OHIP‐20), McGill Pain Questionnaire, and condition‐specific instruments assessing two outcomes: chewing ability and muscular activity during oral functions. ClinROs varied considerably between studies, demonstrating significant methodological diversity. ClinROs included implant stability, accuracy, occlusal parameters, periodontal health metrics, surgical outcomes, and esthetic evaluation via VAS. Conclusion PROs were predominantly assessed using VAS to evaluate patient satisfaction, pain, function, and esthetics, followed by the OHIP Questionnaire for quality‐of‐life assessment in immediate versus early/delayed loading protocols. ClinROs showed no standardized approach, creating significant heterogeneity in the reported outcomes. Standardization of assessment methods and reporting of PROMs/CROMs is needed to optimize analyzing patient‐centered outcomes.

ABSTRACT Aim To identify patient‐ and clinician‐reported outcomes (PROs and ClinROs) and methods of assessment in clinical trials involving short and/or standard‐length and/or zygomatic implants supporting maxillary implant overdentures (IODs). Materials and Methods A systematic search was conducted in PubMed, EMBASE, Scopus, Web of Science CENTRAL, and National clinical trial register for articles published between January 1, 2014, and March 23, 2024. Studies that included PROs and ClinROs for implant therapy in patients with edentulous maxillae were eligible, and study quality was appraised using the Cochrane Risk of Bias 2 tool, Newcastle‐Ottawa Scale, or Joanna Briggs Institute Critical tool. Results A total of 2331 articles were screened, resulting in 21 studies representing 14 patient cohorts with 462 participants and 2140 implants. Two studies used short implants (6 mm) with standard‐length implants, while the remaining 19 studies focused exclusively on standard‐length implants (7–14 mm); none included zygomatic implants. Patient‐reported outcome measures (PROMs) varied widely: Oral Health Impact Profile questionnaires and visual analogue scales were each used in eight studies, and eleven studies relied on unstandardized questionnaires. Implant survival rate was the most frequently used ClinRO, included in all but one study. Subjective ClinROs based on clinician perception were absent in all studies. Conclusions Although PROMs were frequently employed in clinical trials, the lack of standardization hinders meaningful comparisons across studies. While objective ClinROs were commonly reported, subjective ClinROs reflecting clinicians' perceptions were notably absent.

BACKGROUND Patient-reported outcome measures (PROMs) and clinician-reported outcome measures (CROMs) are designed to capture different dimensions of treatment response: the patient's experience and the clinician's assessment, respectively. Because of that, some degree of discordance between them is expected. However, the nature and magnitude of PROM-CROM correlations across orthopaedic subspecialties have not been systematically characterized. Understanding where they converge and where they diverge may help clinicians and researchers select instrument pairings that capture complementary dimensions of treatment response for different musculoskeletal conditions. QUESTIONS/PURPOSES (1) Do CROMs correlate differently with condition-specific PROMs compared with general health PROMs? (2) Do PROMs show distinct correlations with hard CROMs (objective, performance-based measures without patient-reported outcomes) versus soft CROMs (objective, performance-based measures including patient-reported domains)? (3) How do PROMs and CROMs compare in their ability to detect change over time as measured by responsiveness indices such as effect size, standardized response mean, and other related metrics? METHODS We systematically searched Medline via PubMed, Embase, and Web of Science Core Collection in July 2025. Two reviewers independently performed title, abstract, and full-text screening. Studies were included when they directly compared PROMs and CROMs for orthopaedic-related topics. Extracted variables included study design, sample size, Spearman and Pearson correlation coefficients, and responsiveness. CROMs were classified as "hard" or "soft" based on the extent to which the instrument relied on patient input. Hard CROMs represent objective, performance-based or clinician-measured outcomes that do not require patient-reported information, whereas soft CROMs include clinician-administered instruments that incorporate patient-reported symptoms or functional assessments. PROMs were categorized as either generic or condition specific (joint-, region-, disease-specific) according to established definitions. All reported correlation coefficients were extracted and included regardless of statistical significance to comprehensively characterize the magnitude and variability of associations between PROMs and CROMs. Responsiveness was defined as the comparative sensitivity of PROMs and CROMs to clinical change, assessed using effect sizes, standardized response means, and related metrics. Thirty studies met inclusion criteria; 27 reported PROM-CROM correlations and nine evaluated responsiveness. The included studies encompassed a variety of musculoskeletal conditions, including musculoskeletal tumors (n = 4), distal radius fractures (n = 3), total joint arthroplasty and osteoarthritis (n = 11), pediatric limb deformities (n = 3), foot and ankle disorders (n = 7), spine (n = 1), and trauma (n = 1), with sample sizes ranging from 20 to 717 (mean = 143). Assessment of study quality, using an adapted Consensus-based Standards for the Selection of Health Measurement Instruments risk of bias framework, revealed that most studies were of "low risk" or "some concerns" because of incomplete paired PROM-CROM outcome data rather than flaws in design or analysis. RESULTS In evaluating relationships between PROMs and CROMs across orthopaedic subspecialties, condition-specific PROMs generally showed stronger correlations with corresponding CROMs, including the Toronto Extremity Salvage Score (r = 0.75 to 0.81) and the patient-reported American Orthopaedic Foot & Ankle Society (AOFAS) score (r = 0.70). In contrast, general health measures such as the EQ-5D time trade-off (r = -0.29 to 0.13) and certain region-specific instruments, including the Oswestry Disability Index (r = 0.27) and several upper extremity PROMs (r = -0.046 to 0.41), often demonstrated weaker correlations with CROMs. When evaluating differences in correlation by CROM subtype, PROMs tended to correlate more strongly with soft CROMs, such as the AOFAS score, Musculoskeletal Tumor Society score, and Harris hip score, than with hard CROMs. In comparing responsiveness between PROMs and CROMs, condition-specific PROMs, including the Manchester-Oxford Foot Questionnaire, the WOMAC, and Knee Society Score, were more often sensitive to clinical change than CROMs, except in rehabilitation-focused settings, where hard CROM responsiveness was often greater than for PROMs and soft CROMs. CONCLUSION Based on currently available studies related to musculoskeletal conditions, PROMs and CROMs show variable associations with one another. The stronger correlations between soft CROMs and condition-specific PROMs suggest that when the goal is to establish benchmarks, a condition-specific PROM should be paired with a soft CROM. In contrast, hard CROMs may be particularly useful in short-term rehabilitation settings, where objective performance changes are a primary focus of evaluation. We recommend that orthopaedic studies and registries include, at a minimum, one condition-specific PROM (or soft CROM) and one hard CROM to ensure a multidimensional assessment of patient outcomes and improve interpretability across studies. LEVEL OF EVIDENCE Level III, therapeutic study.

Abstract Background Atopic dermatitis (AD) is a chronically relapsing skin disease. Although a definitive cure is not available, appropriate treatment can control the disease. The advent of biologic drugs has led to the need for a clear definition of the disease severity and treatment response. A standardized list of outcomes that defines clinician-reported disease severity and patients’ reported severity are therefore essential. Solid criteria to define the response to treatment and treatment failure are lacking to date. Objective This systematic review defines treatment goals in terms of clinician-reported disease severity and patient-reported outcomes, referring to the published moderate-to-severe AD clinical trials. The application of these goals in daily clinical practice will ensure a better selection of available treatment options, thus increasing patient quality of care. Materials and methods A systematic literature search was performed to identify the treatments goals of randomized controlled clinical trials (RCTs) on moderate-to-severe adult AD published between January 2000 and October 2020. Results In total, 14 studies met the eligibility criteria. The most widely used tools in terms of clinician-reported disease severity were the Scoring of Atopic Dermatitis (SCORAD) followed by the Eczema Area Severity Score (EASI) and Investigator Global Assessment (IGA). For disease severity scales as efficacy outcome in RCTs, the greatest standardization and reproducibility was for improvement of at least 50% in EASI score and IGA score reduction of ≥2 grades from baseline. The most widely used tools from the patients’ perspective were the Dermatology Life Quality Index (DLQI), Numeric Rate Scale (NRS)–itch and Patient Oriented Eczema Measure Score (POEM). In terms of patients’ reported efficacy outcomes in RCTs, a numerical DLQI, NRS–itch and POEM score improvement of at least 4 points from baseline was reported. Conclusions This systematic review highlights the need for collaboration between experts in order to define and optimize treatment outcomes. Despite considerable progress in harmonizing outcome measures, promoted by the foundation of the Harmonizing Outcome Measures for Eczema (HOME) initiative in 2008, our results demonstrate that this endpoint is still an unmet need. Based on the literature data we propose a minimum treatment goal algorithm for use in daily clinical practice aimed at stimulating a discussion on how the care of AD patients could be further improved.

No abstract available

Although progress has been made in developing outcome measures for AA, the use of these measures remains unstandardized. A scoping review was conducted to identify the clinician-reported outcome measures (ClinROMs) and patient-reported outcome measures (PROMs) used in assessing and treating AA, the results of which revealed heterogeneity in AA outcome measures. Of 23 research studies ultimately included, only 2 ClinROMs were used by >15% of studies; likewise, of 110 clinical trials evaluated, numerous outcome instruments were used, but only one ClinROM was used by >5% of trials (Severity of Alopecia Tool). These results suggest the need for consensus and standardization in both research and trial settings.

Across multiple RCTs, discrepancies between patient and clinician reports of PTSD symptoms are at least a partial contributing factor to large discrepancies between treatment outcome effect sizes from self-report and clinician reports within the same patients. Using secondary data from the NIDA-funded Women and Trauma Study, we demonstrated Common Persons Item Response Theory (IRT) Calibration for calibrating self-reported and clinician-reported PTSD severity scores in a manner similar to the process used to produce equated scores across multiple forms of standardized tests (e.g., SAT, GRE). Under IRT calibration, treatment effect sizes between the CAPS and MPSS-SR did not differ, while with the noncalibrated measures, the CAPS effect size was 85% larger than the MPSS-SR. Further, across the range of a combined CAPS/MPSS-SR gold standard, IRT-calibrated CAPS and MPSS-SR individual scores did not differ; for uncalibrated individual scores, MPSS scores were higher than CAPS scores at higher levels of PTSD severity while the reverse was true at lower levels of severity. The use of IRT calibration approaches for calibrating self-report and clinical interview measures of PTSD will allow treatment researchers to reflect the treatment effect on PTSD as a construct (regardless of reporter) as opposed to being limited to reporting treatment effects that may be discrepant within patients and specific to the particular assessment measure being employed. (PsycINFO Database Record (c) 2019 APA, all rights reserved).

Cholestatic pruritus is common in primary biliary cholangitis (PBC), often leading to sleep disturbances and substantially impairing health-related quality of life (HRQoL). Fatigue, also frequent in PBC, can be exacerbated by sleep interference due to nighttime pruritus. Quantitative numerical rating scales (NRS) are appropriate for assessing unidimensional patient-reported outcome (PRO) concepts and are easily interpreted. The PBC-40 is a disease-specific, patient-derived 40-item tool that assesses HRQoL in patients with PBC. To assess pruritus severity in clinical trials, rigorous validation of PROs in the target patient population is required. This study aimed to validate the NRS items for worst itch (WI-NRS), pruritus-related sleep interference (Sleep Interference NRS), and fatigue (Fatigue NRS), plus the PBC-40 (modified with a 7-day recall), among patients with PBC and pruritus. Data were analyzed from the Phase 2b GLIMMER trial (NCT02966834) of linerixibat and a separate observational study in patients with PBC and pruritus. Pruritus severity was assessed using the 0–10 WI-NRS. Psychometric properties of the WI-NRS, Sleep Interference NRS, Fatigue NRS, and PBC-40 (7-day recall) were evaluated. Additional PRO data were used to support validation analyses. Multiple psychometric methods were used to validate the NRS items and PBC-40 (7-day recall). Data for 288 patients (n=147 GLIMMER; n=141 observational study) with PBC experiencing pruritus were included. The internal consistency of PBC-40 (7-day recall) was acceptable to excellent and confirmatory factor analysis confirmed its domain structure. All PBC-40 domains showed acceptable test–retest reliability (intraclass correlation coefficients [ICCs]: 0.72–0.90). The WI-NRS showed acceptable test–retest reliability (ICCs: 0.73–0.81 [GLIMMER]; ICC: 0.78 [observational study]). Test–retest reliability was also acceptable for the Sleep Interference (ICC: 0.85 [GLIMMER]; ICC: 0.77 [observational study]) and Fatigue (ICC: 0.88 [GLIMMER]; ICC: 0.78 [observational study]) NRS items. Convergent, discriminant, and known-groups validity were confirmed for all three NRS items and the PBC-40. Additionally, the NRS and PBC-40 Itch domain demonstrated responsiveness by reflecting change in other PROs over time. The data support the psychometric reliability, validity, and responsiveness of WI-NRS, pruritus-related Sleep Interference NRS, and Fatigue NRS, and PBC-40 (7-day recall) in patients with PBC experiencing pruritus.

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.

Pain is a common symptom in individuals with neurofibromatosis type 1 (NF1) that often is associated with plexiform neurofibroma (pNF) tumors. To date, no patient-reported outcome measures have been validated specifically to assess pNF-related pain intensity or pain interference in this population. Such measures are sorely needed since pain is being considered as an outcome in clinical trials targeting reduction of pNF. The study aims were to: (1) obtain qualitative information from individuals with NF1 and pNFs about their pain and its measurement and (2) modify existing scales to assess pNF-related pain intensity and pain interference for NF1 clinical trials. For this multi-site, qualitative study, 56 individuals (26 children, 6–16 years; 30 adults, 18–68 years) with NF1 and pNF participated in a focus group and/or individual interview about pain intensity and pain interference (concept elicitation) and also provided feedback about existing pain measures (Numeric Rating Scale-11 and Pain Interference Index) assessing these domains (cognitive debriefing). Four additional waves of cognitive debriefing interviews further refined the measures. Qualitative concept elicitation data from transcripts were coded, analyzed using NVivo software, and thematic analysis was conducted using both deductive and inductive techniques. Additional themes and systematic problems and suggestions regarding the measures were gleaned from reviewing the field notes and interview transcriptions generated by the cognitive debriefing sessions. Concept elicitation themes included descriptions of two types of pNF-related pain (chronic and episodic), variability of pain over time, varying ability to recall pain, lack of knowledge of pNFs, and the ways pain interferes with daily activities. Cognitive debriefing themes included information on how to rate pNF-related pain intensity apart from other pain; problems and suggestions regarding the measures included difficulty comprehending some items and preferences for alternative wording and formatting. Based on these qualitative results, the measures’ instructions, items, and formatting were modified to create the PAin INtensity Scale for plexiform neurofibromas (PAINS-pNF) and the Pain Interference Index for plexiform neurofibromas (PII-pNF) for administration on a mobile app or web-based platform. The PAINS-pNF and PII-pNF are promising self-report measures developed using patient engagement to evaluate tumor pain intensity and pain interference in NF1 clinical trials. The second phase of the study to provide reliability, validity, and normative data for individuals with NF1 and pNFs ages 8 years and older is underway.

Patient-reported outcome measures (PROMs) are important for assessing premenstrual syndrome (PMS) and premenstrual dysphoric disorder (PMDD) to effectively capture subjective symptom burden and evaluate treatment effectiveness in clinical and research settings. This systematic review evaluated the psychometric properties of PROMs used to assess PMS/PMDD in Japan. A systematic literature search was conducted in the MEDLINE, CINAHL, Cochrane Library, and Ichushi-Web databases. The COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) methodology was used to assess the methodological quality and measurement properties of the included PROMs. A total of 13 studies that evaluated 12 versions of 11 unique PROMs were included. PROMs were categorized as recall-based (n = 9, 69%) or daily recording scales (n = 4, 31%). The structural validity and internal consistency were relatively well evaluated for most scales. However, evidence was limited for other measurement properties such as reliability, criterion validity, and construct validity. None of the scales reported all psychometric properties outlined by COSMIN. The New Short-Form of the Premenstrual Symptoms Questionnaire and the Japanese version of the Daily Record of Severity of Problems demonstrated sufficient structural validity and internal consistency, although the quality of evidence for other properties was indeterminate. Although some PROMs demonstrated promising psychometric properties, further validation studies are required for most scales. The development of innovative scales with robust measurement properties is essential for advancing the assessment of PMS/PMDD in Japanese clinical and research settings. Careful consideration of the characteristics of each PROM is necessary when selecting instruments for specific purposes.

No abstract available

The commonly used (‘legacy’) PROMs evaluating outcomes of total hip arthroplasty (THA), have several limitations regarding their measurement properties and interpretation of scores. One innovation in PROMs is the use of Computerized Adaptive Testing (CAT). The Patient-Reported Outcomes Measurement Information System (PROMIS®) is a validated system of CATs. The aim of this study was to assess the measurement properties of PROMIS and legacy instruments in patients undergoing THA. Patients in this multicenter study filled out a questionnaire twice, including Dutch-Flemish PROMIS v1.2 Physical Function (PROMIS-PF) and v1.1 Pain Interference (PROMIS-PI) CATs and short forms, PROMIS v1.0 Pain Intensity, and legacy PROMs (Hip disability and Osteoarthritis Outcome Score (HOOS), HOOS-Physical function Shortform (HOOS-PS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Oxford Hip Score (OHS), and two numeric rating scales measuring pain). The reliability, measurement precision (Standard Error of Measurement (SEM)), smallest detectable change (SDC), and burden of PROMIS instruments were presented head-to-head to legacy PROMs. Furthermore, construct validity was assessed. 208 patients were included. All instruments had a sufficient test-retest reliability (range ICC: 0.83–0.96). The SEM of PROMIS CATs and short forms ranged from 1.8 to 2.2 T-score points, the SEM of legacy instruments 2.6–11.1. The SDC of PROMIS instruments ranged from 2.1 to 7.3 T-score points, the SDC of legacy instruments 7.2–30.9. The construct validity of PROMIS CAT and short forms were found sufficient, except for the PROMIS-PI short form. The burden of PROMIS CATs was smaller than PROMIS short forms (range 4.8–5.2 versus 8–20 items, respectively). The burden of legacy instruments measuring physical functioning ranged from 5 to 40 items. The PROMIS-PF is less burdensome, with high measurement precision, and almost no minimal or maximal scores, and an equal reliability compared to legacy instruments measuring physical functioning in patients undergoing THA. The PROMIS Pain Intensity 1a is comparable to the legacy pain instruments in terms of burden, reliability and SDC. Measuring the construct Pain Interference may not have additional value in this population because of its high correlation with instruments measuring physical functioning. The SDC values presented in this study can be used for individual patient monitoring.

ABSTRACT Background: A rating scale is a critical component of patient-reported outcome instrument design, but the optimal rating scale format for pediatric use has not been investigated. We compared rating scale performance when administering potential questionnaire items to children with eye disorders and their parents. Methods: Three commonly used rating scales were evaluated: frequency (never, sometimes, often, always), severity (not at all, a little, some, a lot), and difficulty (not difficult, a little difficult, difficult, very difficult). Ten patient-derived items were formatted for each rating scale, and rating scale testing order was randomized. Both child and parent were asked to comment on any problems with, or a preference for, a particular scale. Any confusion about options or inability to answer was recorded. Results: Twenty-one children, aged 5-17 years, with strabismus, amblyopia, or refractive error were recruited, each with one of their parents. Of the first 10 children, 4 (40%) had problems using the difficulty scale, compared with 1 (10%) using frequency, and none using severity. The difficulty scale was modified, replacing the word “difficult” with “hard.” Eleven additional children (plus parents) then completed all 3 questionnaires. No children had problems using any scale. Four (36%) parents had problems using the difficulty (“hard”) scale and 1 (9%) with frequency. Regarding preference, 6 (55%) of 11 children and 5 (50%) of 10 parents preferred using the frequency scale. Conclusions: Children and parents found the frequency scale and question format to be the most easily understood. Children and parents also expressed preference for the frequency scale, compared with the difficulty and severity scales. We recommend frequency rating scales for patient-reported outcome measures in pediatric populations.

To explore patients’ experience of symptoms and impacts of Crohn’s disease (CD) and evaluate content validity of patient-reported outcome (PRO) measures in moderately-to-severely active CD. Qualitative, web-based interviews were conducted to elicit participants’ experiences with CD, and to obtain their feedback on: Patient Global Rating of Severity (PGRS:1 ‘none’ to 6 ‘very severe’), Urgency Numeric Rating Scale (Urgency NRS:0 ‘no urgency’ to 10 ‘worst possible urgency’), patient-reported symptoms from Crohn’s Disease Activity Index (CDAI; abdominal pain [AP] [severe, moderate, mild, none], well-being [WB] [terrible, very poor, poor, slightly under par, generally well], bowel movement [BM] count and stool frequency), and Bristol Stool Chart (type 1 ‘hard’ to type 7 ‘entirely liquid’), including understanding of response options, recall period and examination of meaningful improvement in CD symptoms. Seventeen adults and 3 adolescents (mean age:37.7 years; 70% female) were interviewed. Overall, 41.2% of adults had a college degree or higher. Most participants reported experiencing fatigue (n = 19), bowel urgency (n = 18), diarrhea (n = 18), AP (n = 17), and/or stool frequency (n = 15). CD impacted participants’ daily activities (n = 20), mood/emotions (n = 19), social activities/relationships (n = 19), productivity/performance at work/school (n = 18), and/or sleep (n = 15). Participants found PRO items and response options easy to understand and use and reported that it was easy to recall their symptoms over 24 h. Participants considered a 1- or 2-point reduction on PGRS as meaningful improvement. Seventeen participants considered 3-point improvement on Urgency NRS as meaningful. Participants with mild/moderate AP (n = 11) and severe AP (n = 13) considered a 1- and 2-point reduction in CDAI-AP score as meaningful improvement. Ten participants reported a 2-point reduction in CDAI-WB score as meaningful improvement if they had ‘terrible’ WB. Generally, participants considered going from 5 to 2 BMs/day as meaningful improvement. Nineteen participants considered improvement to stool type 3 to 5 on Bristol Stool Chart as meaningful. Bowel urgency, diarrhea, and AP were the most common CD symptoms. Cognitive debriefing findings support content validity of PRO measures in patients with CD and provide insights on levels of change in PRO measures considered meaningful by patients.

BackgroundContent validity is the most important measurement property of a patient-reported outcome measure (PROM) and the most challenging to assess. Our aims were to: (1) develop standards for evaluating the quality of PROM development; (2) update the original COSMIN standards for assessing the quality of content validity studies of PROMs; (3) develop criteria for what constitutes good content validity of PROMs, and (4) develop a rating system for summarizing the evidence on a PROM’s content validity and grading the quality of the evidence in systematic reviews of PROMs.MethodsAn online 4-round Delphi study was performed among 159 experts from 21 countries. Panelists rated the degree to which they (dis)agreed to proposed standards, criteria, and rating issues on 5-point rating scales (‘strongly disagree’ to ‘strongly agree’), and provided arguments for their ratings.ResultsDiscussion focused on sample size requirements, recording and field notes, transcribing cognitive interviews, and data coding. After four rounds, the required 67% consensus was reached on all standards, criteria, and rating issues. After pilot-testing, the steering committee made some final changes. Ten criteria for good content validity were defined regarding item relevance, appropriateness of response options and recall period, comprehensiveness, and comprehensibility of the PROM.DiscussionThe consensus-based COSMIN methodology for content validity is more detailed, standardized, and transparent than earlier published guidelines, including the previous COSMIN standards. This methodology can contribute to the selection and use of high-quality PROMs in research and clinical practice.

Patient-reported outcomes measures (PROMs) are increasingly prevalent in healthcare and used for shared decision-making and healthcare quality evaluation. However, the extent to which patients with varying health literacy levels can complete PROMs is often overlooked. This may lead to biased aggregated data and patients being excluded from studies or other PROM collection initiatives. This cross-sectional study evaluates the comprehensibility of 157 well-known and widely used PROM scales using a comprehensibility checklist. Pairs of two independent raters scored 157 PROM scales designed for adults included in the 35 sets of outcome information developed as part of the Dutch Outcome-Based Healthcare Program. The PROM scales were scored on the eight comprehensibility domains of the Pharos Checklist for Questionnaires in Healthcare (PCQH). Interrater agreement of domain ratings was assessed using Intraclass Correlation Coefficients or Cohen’s kappa. Subsequently, final ratings were established through discussion and used to evaluate the domain-specific comprehensibility rating for each PROM scale. Comprehensibility of a large number of PROM scales (n = 157), which cover a wide range of diseases and conditions across Dutch medical specialist care, was assessed. While most PROM scales were written at an accessible language level, with minimal use of medical terms, instruction clarity, number of questions, and response options emerged as significant issues, affecting a substantial proportion of PROM scales. Interrater agreement was high for most domains of the PCQH. This study highlights the need for greater attention to the comprehensibility of PROMs to ensure their accessibility to all patients, including those with low health literacy. The PCQH can be a valuable tool in PROM development in addition to qualitative methods and in selection processes enabling comparison of comprehensibility between PROMs. However, the PCQH needs further development and validation for these purposes. Enhancing the comprehensibility of PROMs is essential for their effective incorporation in healthcare evaluation and decision-making processes.

BACKGROUND To determine the association between socioeconomic status and patient-reported outcome measures in a Dutch cohort who have undergone total hip arthroplasty (THA) or total knee arthroplasty (TKA). METHODS A retrospective national registry study of all patients who underwent primary THA or TKA between 2014-2020 in the Netherlands was performed. Linear mixed-effects regression models were used to assess the association between SES and PROMs for THA and TKA patients separately. The following measures were collected: Numeric pain rating scale, Oxford Hip/Knee Score, Hip/Knee disability and Osteoarthritis Outcome Score and the EuroQol 5-Dimensions-questionnaire. Sex, age, body mass index, American Society of Anesthesiologists-classification, Charnley-classification and smoking status were considered as covariates in the models. RESULTS THA patients (n=97,443) were on average 70 years old with a BMI of 27.4 kg/m2, and TKA patients (n=78,811) were on average 69 years old with a BMI of 29.7 kg/m2. Preoperatively, patients with a lower socioeconomic status undergoing THA or TKA reported more severe symptoms and lower health-related quality of life. At 1-year follow-up, they also reported lower scores and less improvement over time compared to patients with a higher socioeconomic status. CONCLUSION Patients with lower socioeconomic status report worse symptoms when admitted for surgery, and less improvement after surgery. Future research must address potentially mediating factors of the association between socioeconomic status and symptom reporting such as access to surgery and rehabilitation, subjectivity in reporting, and patient expectation for THA and TKA outcomes.

Background: The classical psychedelics psilocybin, peyote, ayahuasca/ N, N-dimethyltryptamine, and lysergic acid diethylamide can temporarily produce altered states of consciousness, characterized by changes in sensory perception, thought, mood, and the sense of self-reality and meaning. It is important to have reliable instruments for quantifying these altered states in trials, due to a plausible link between the acute subjective experience and treatment outcome. Methods: We conducted a review of outcome measures applied in research on classical psychedelics to assess one or more dimensions of the acute subjective psychedelic experience. Three relevant databases were searched electronically. Two reviewers independently conducted article selection and data extraction regarding the instruments, dimensions, geography, population, and psychedelic substance investigated in the included studies. We identified the five most utilized instruments for the most recent 6 years, as well as the five most utilized instruments for each psychedelic. Results: We included 93 papers, which reported on 93 unique trials and utilized 17 different rating scales. Of these, the most utilized were the Five-Dimensional Altered States of Consciousness Questionnaire, visual analog or Likert scales specially developed for the trials, the Hallucinogen Rating Scale, the States of Consciousness Questionnaire, and the Abnormer Psychischer Zustand. Discussion: Considerable variability was found in the instruments utilized in clinical trials on classical psychedelics. We advise and encourage the development of a core outcome set for psychedelic research to enable altered state comparisons across compounds, participants, and settings. We further advise that instruments be designed to assess the “setting” of a psychedelic experience.

BACKGROUND Dysmenorrhea, or menstrual pain, is a subjective experience, and can only be assessed by patient-reported outcomes. These instruments should be reliable, valid and responsive. AIM To identify and critically appraise the available evidence for the measurement properties of specific patient-reported outcome measures used for dysmenorrhea. METHODS The PRISMA statement was used to report this systematic review. Databases searched were PubMed, SCOPUS, CINAHL, Web of Science, ScienceDirect and Google Scholar (April 2021; updated on February 2023). Original studies with primary data collection, with no restriction on language and publication date that reported psychometric properties of one or more dysmenorrhea-related patient-reported outcome measure. The literature searches, selection of studies, data extraction and assessment of the risk of bias were performed independently by two reviewers and followed the COSMIN guidelines. RESULTS Thirty studies were analysed in this review, and 19 patient-reported outcome measures were evaluated. The instruments varied in relation to the measured construct and measurement properties (validity, reliability and responsiveness). The methodological quality of the studies and the quality of evidence of the patient-reported outcome measures were variable. Among the 13 studies that reported the development of patient-reported outcome measures, most had inadequate methodological quality, and the overall rating was insufficient or inconsistent. CONCLUSIONS The Dysmenorrhea Symptom Interference (DSI) scale was the only identified patient-reported outcome measure that has the potential to be recommended because of its sufficient rating combined with moderate quality of evidence for content validity. Future studies should further evaluate the measurement properties of the existing patient-reported outcome measures, or develop new patient-reported outcome measures following the COSMIN methodology. PATIENT OR PUBLIC CONTRIBUTION Not applicable as this is a systematic review. TRIAL REGISTRATION PROSPERO protocol: CRD42021244410. Registration on April 22, 2021.

Bowel urgency is an impactful core symptom of ulcerative colitis (UC). Patient-reported outcome (PRO) questionnaires have been developed and used to assess the patient experience of this important symptom. The objective of this paper is to present evidence from qualitative research conducted to support the use and interpretation of select PRO questionnaires to assess bowel urgency related to the UC patient experience. Qualitative interviews were conducted with ten adults with a clinician-confirmed diagnosis of moderately to severely active UC. Interviews aimed to document patient interpretation of modified recall periods for the Urgency Numeric Rating Scale (Urgency NRS), two global assessments (i.e., the Patient Global Impression of Severity [PGIS] and Patient Global Impression of Change [PGIC]), and four items (Items 11, 16, 23, and 26) of the Inflammatory Bowel Disease Questionnaire (IBDQ), and explore the patient perspective of meaningful change on these questionnaires. Both modified Urgency NRS versions (with 7-day or 3-day recall period) were interpreted as intended by most patients (≥ 88.9%), and slightly more than half of patients (60.0%) reported that the 7-day recall period was more relevant to their bowel urgency experience. Patients reported thinking of bowel urgency (≥ 80.0%) or bowel urgency-related accidents (70.0% of patients) when interpreting the global assessments and IBDQ items. Most patients reported a 1- to 3-point change as the smallest meaningful improvement that would be meaningful on the Urgency NRS (similar to findings on other questionnaires). Adults with UC can understand and respond to the Urgency NRS with modified recall periods (i.e., 7-day or 3-day), interpret the conceptual content of the PGIS, PGIC, and select IBDQ items to be inclusive of bowel urgency and bowel urgency-related accidents, and select answers representing meaningful improvements on the Urgency NRS, PGIS, PGIC, and IBDQ item response scales. These results further contribute patient-centered data to existing UC and bowel urgency research.

BACKGROUND CONTEXT The 6-minute walking test (6WT) has been previously shown to be a reliable and valid outcome measure. It is unclear if the 6WT may further help to detect differences in well performing patients that reach a ceiling effect in PROMs after surgery. PURPOSE To evaluate changes and timing of change in objective functional impairment (OFI) as measured with the smartphone-based 6WT in relation to patient-reported outcome measures (PROMs) after surgery for degenerative lumbar disorders (DLD) STUDY DESIGN: Prospective observational cohort study PATIENT SAMPLE: 50 consecutive patients undergoing surgery for degenerative lumbar disorders OUTCOME MEASURES: Patients self-determined their objective functional impairment using the 6-minute Walking Test application (6WT-app) and completed a set of paper-based patient-reported outcome measures (PROMs) before, six weeks and 3 months after surgery. METHODS 50 patients undergoing surgery for DLD were assessed preoperatively (baseline), 6 weeks (6W) and 3 months (3M) postoperatively. Paired sample t-tests were used to establish significant changes in raw 6-minute walking distance (6WD) and standardised z-scores, as well as PROMs. Pearson correlation coefficient was used to define the relationship between 6WT and PROMs. Floor and ceiling effects were assessed for each PROM (Visual Analogue Scale - VAS, Core Outcome Measure Index - COMI, Zurich Claudication Questionnaire - ZCQ). RESULTS Mean 6WT results improved from 377m (standard deviation - SD 137; z-score: 1.8, SD 1.8) to 490m (SD 126; -0.7, SD 1.5) and 518m (SD 112; -0.4, SD 1.41; all p < 0.05) at 6W and 3M follow-up. No significant improvement was observed between 6W and 3M for the ZCQ, VAS back and leg pain. While correlation between 6WT and all PROMs were weak at baseline, correlation coefficient increased to moderate at 3M. A considerable ceiling effect (best possible score) was observed, most notably for the ZCQ Physical Performance, VAS back and leg pain in 24%, 20% and 16% of patient at 6W and in 30%, 24% and 28% at 3M. CONCLUSION Objective functional tests can describe the continued change in the physical recovery of a patient and may help to detect differences in well performing groups as well as in cases where patients' PROM results cannot further improve because of a ceiling effect.

No abstract available

Patient-reported outcome measures (PROMs) are self-rated scales and indices developed to improve the detection of the patients’ subjective experience. Given that a considerable number of PROMs are available, it is important to evaluate their validity and usefulness in a specific research or clinical setting. Published guidelines, based on psychometric criteria, do not fit in with the complexity of clinical challenges, because of their quest for homogeneity of components and inadequate attention to sensitivity. Psychometric theory has stifled the field and led to the routine use of scales widely accepted yet with a history of poor performance. Clinimetrics, the science of clinical measurements, may provide a more suitable conceptual and methodological framework. The aims of this paper are to outline the major limitations of the psychometric model and to provide criteria for clinimetric patient-reported outcome measures (CLIPROMs). The characteristics related to reliability, sensitivity, validity, and clinical utility of instruments are critically reviewed, with particular reference to the differences between clinimetric and psychometric approaches. Of note is the fact that PROMs, rating scales, and indices developed according to psychometric criteria may display relevant clinimetric properties. The present paper underpins the importance of the clinimetric methodology in choosing the appropriate PROMs. CLIPROM criteria may also guide the development of new indices and the validation of existing PROMs to be employed in clinical settings.

Background: Hidradenitis suppurativa (HS) is a chronic, recurrent inflammatory condition and is associated with significant psychosocial impacts on patient quality of life. Objective: This study aimed to characterize the utilization of patient-reported outcomes (PROs) in HS clinical trials and their concordance with trial primary endpoints. Methods: A systematic review of clinical trials was performed using the publicly available U.S. National Library of Medicine (ClinicalTrials.gov) in June 2023 utilizing the search terms hidradenitis, hidradenitis suppurativa, and suppurativa hidradenitis. Study title, start year, trial status, intervention, design, location, and primary and secondary outcomes were collected. To assess for concordance of patient and provider-reported outcomes, we identified published placebo-controlled trials that included Dermatology Life Quality Index (DLQI) and/or a numeric rating scale (NRS) for pain, the 2 most utilized PROs. Results: One hundred sixty-four HS clinical trials were identified, of which 115 were interventional studies. A total of 65.2% (n = 107) of HS trials included at least one PRO. Pain NRS (42.7%, n = 70) and DLQI (42.1%, n = 69) were the most frequently used PRO instruments. The use of PROs in clinical trials increased over time, with 50% of trials between 2020 and 2023 utilizing PROs. Of 11 published HS trials, 82% (n = 9) trials showed concordance of provider-assessed and PROs in at least one treatment arm. Limitations: Significance and standard deviations of PROs were rarely reported, preventing the calculation of significance and is a limitation of this review. Conclusion: While lesion counts provide single snapshots of disease, PROs can capture perceptions of lesions not actively present. PROs are frequently concordant with both achievement and nonachievement of primary endpoints in clinical trials.

Abstract Background Manual chart abstraction from electronic health records is a critical step in clinical outcomes research but is time-intensive and prone to human error. Advances in artificial intelligence (AI), particularly large language models, offer the potential to automate the extraction of structured data from unstructured clinical documentation with improved efficiency and consistency. Objective This study aimed to evaluate the accuracy and efficiency of an AI-assisted approach for extracting patient-reported outcomes from clinical notes compared with traditional human abstraction. Methods We conducted a retrospective study of 26 patients treated with low-dose radiation therapy for osteoarthritis. Human reviewers abstracted numeric rating scale (NRS; 0‐10) pain scores at baseline, the end of treatment, and the first follow-up, and von Pannewitz score (VPS; 0‐4) improvement scores at posttreatment time points. A HIPAA (Health Insurance Portability and Accountability Act)–compliant generative pretrained transformer–based AI system was prompted to extract the same end points from clinical notes. Concordance was assessed using exact match rates, the intraclass correlation coefficient for the NRS, and weighted Cohen κ for the VPS. The time required for AI vs manual abstraction was recorded. The AI system was not trained or fine-tuned on study data, and performance was evaluated directly against human abstraction to reflect real-world deployment. Results The AI system demonstrated high concordance with human abstraction, achieving an exact match rate of 92% for the NRS (95% CI 84‐96; intraclass correlation coefficient=0.96) and 94% for the VPS (95% CI 84‐98; κ=0.91). All discrepancies were minor, and no spurious values were generated. The AI system identified 1 clinically relevant data point missed during manual review. Average abstraction time per patient decreased from approximately 30 minutes to 2 minutes, representing time savings of >90%. The system also captured trends in analgesic use, but these results were not statistically significant, including reductions without escalation. Conclusions AI-assisted data abstraction demonstrated high concordance with human review in this single-institution cohort while substantially reducing the time requirements. These findings support the feasibility of AI-assisted abstraction workflows, although further validation across larger and more diverse datasets is needed.

The psychometric properties of the Suicide Ideation and Behavior Assessment Tool (SIBAT) were evaluated in 130 participants with varying levels of suicidality. Inter- and intra-rater reliability were assessed for clinician-rated outcomes, including the revised Clinical Global Impressions (CGI) of severity of suicidality (CGI-SS-r). Concurrent validity of patient-reported modules with Patient-reported Outcomes Measurement Information System (PROMIS) depression scale and Sheehan-Suicidality Tracking Scale Clinically Meaningful Change Measure (S-STS CMCM), and concordance between Columbia Classification Algorithm of Suicide Assessment (C-CASA) mappings for SIBAT, S-STS CMCM and Columbia-Suicide Severity Rating Scale (C-SSRS) were assessed. 52/130 participants (mean [SD] age: 38.3 [17.77] years) consented for multiple interviews (C-CASA mappings: n=52; rater-reliability: n=25/52). SIBAT demonstrated good intra-rater reliability (weighted-kappa range:0.64-0.76; CGI-SS-r, 0.75) and adequate inter-rater reliability (ICC range:0.68-0.82; CGI-SS-r, 0.81). There were strong correlations between PROMIS depression scores and SIBAT Module 5 ratings (Spearman correlations, r=0.64-0.74) and moderate correlations (r=0.29-0.72) between S-STS CMCM and SIBAT Modules 2, 3 and 5 ratings. Moderate agreement was noted between SIBAT C-CASA mappings and corresponding mappings from S-STS CMCM (weighted kappa: 0.54) and C-SSRS (weighted kappa: 0.56). Thus, the SIBAT provided valid assessment of suicidal ideation and behavior that could be reliably rated and adequately mapped to the C-CASA.

LGI1‐antibody encephalitis (LGI1‐Ab‐E) is a common form of autoimmune encephalitis where most patients demonstrate ‘good’ clinician‐rated outcomes. However, more targeted questionnaires reveal numerous debilitating symptoms for many years. To better quantify these persistent features, we designed the LGI1‐Antibody Encephalitis Rating (LANTERN) scale, a quantified, disease‐specific patient‐reported outcome measure (PROM), adhering to FDA guidelines.

Chronic graft-versus-host disease (cGVHD) is a leading driver of long-term morbidity after allogeneic transplantation. This review synthesizes evidence on patient-reported outcomes (PROs) across organ systems and survivorship domains, appraises psychometrics and minimal clinically important differences (MCIDs), and translates measures into routine care. Validated tools—including the Lee Symptom Scale (LSS), FACT-BMT, SF-36, PROMIS, and pediatric instruments—capture symptom burden and function beyond clinician ratings, and studies have demonstrated associations of some measures with survival, failure-free survival, and non-relapse mortality. Concordance between provider-rated and PRO-defined responses is limited, while PRO changes often track clinically meaningful improvement. We propose an implementation framework and two practical tables mapping measures to organ involvement and use-cases. Key barriers include workflow integration, patient burden, and cross-cultural adaptation-gaps that are pronounced in Chinese cohorts. Embedding PROs in trials and survivorship models can sharpen risk stratification, inform shared decisions, and improve patient-centered outcomes in cGVHD.

BACKGROUND Functional movement disorder (FMD), characterized by abnormal movements and motor weakness, represents a common manifestation within the spectrum of functional neurological disorders (FND). Integrative therapy, comprising specialised physiotherapy, psychotherapy, and patient education, has demonstrated efficacy in improving motor function and quality of life. To assess symptom severity and treatment response, both patient-reported outcome measures (PROMs) and clinician-rated outcome measures (CROMs) can be employed. METHODS In a preliminary analysis of an ongoing transdisciplinary treatment study, we examined the association between the 'Simplified FMD Rating Scale' (S-FMDRS, CROM) and the 'FMD Questionnaire' (FMDQ, PROM) during integrative therapy and explored their suitability for monitoring treatment-related changes over time. RESULTS Twenty-six patients contributed 60 paired longitudinal assessments. Linear mixed-effects modelling demonstrated significant associations between clinician-rated motor severity (S-FMDRS) and patient-reported total FMDQ scores as well as all functional domains. In the subgroup with complete pre-post data (n = 15), both S-FMDRS and FMDQ scores improved significantly during treatment (S-FMDRS: p = .003, r = 0.76; FMDQ: p = .012, r = 0.65). Concordance in MCID-based responder classification was partial, with 53% agreement between instruments. CONCLUSION Clinician- and patient-reported measures in FMD are significantly associated but not interchangeable at the individual level, supporting their complementary use in treatment evaluation.

The use of Janus Kinase Inhibitors (JAK-Is) in rheumatoid arthritis (RA) has entered in daily practice. In consideration of ORAL-Surveillance trial and the new EULAR recommendations, real-world data are needed to assess Jak-Is safety and effectiveness. The multicenter study presented here aimed to evaluate effectiveness and safety of tofacitinib in a real-life cohort. A retrospective analysis was performed from September 2021 to December 2022. Data were collected when tofacitinib was started (T0) and after 3 (T3), 6 (T6) and 12 (T12) months of treatment. The primary objective was to analyze the efficacy and safety of tofacitinib. Safety was assessed by recording adverse events (AEs) with and without discontinuation. The secondary objective was to assess the difference between Patient-Reported Outcomes (PROs) and Physician's Global Assessment of disease activity (PhGA). 122 patients were included in the study from the following rheumatology Centers: Pisa, Ancona, Florence (two Centers), Siena, and Sardinia. A statistically significant improvement in DAS-28-CRP, CDAI and SDAI score was observed at T3, T6, compared to baseline (p < 0.001). Improvement was confirmed in patients who reach T12. Patients naïve to bDMARDs showed a shorter remission time and higher remission rates. There was also a statistically significant improvement in PROs compared to baseline (p < 0.001). The improvement was rapid and was consistent with PhGA. The 12-month retention rate for tofacitinib was 89.35%. Reasons to stop tofacitinib were: insufficient response (7), gastrointestinal symptoms (2), infection (1), malignancy (1), Zoster (1), pruritus sine materia (1). Tofacitinib is safe and effective in our RA cohort. It induces higher remission rates in patients naive to bDMARDs, suggesting that there may be a benefit using it as first-line therapy. Additionally, improvement in PROs was consistent with PhGA scores, demonstrating how tofacitinib affects both the objective and subjective components of disease activity. Key Points 1. JAK inhibitors are considered at a similar level as biologic agents in terms of effectiveness. 2. After ORAL-Surveillance results, real-world data are needed to assess the benefit/risk profile of Jaki. 3. Disagreement between patients and physicians has been previously reported with biologic therapy among patients with rheumatoid arthritis, with patients rating disease activity higher than physicians. 4. Jak inhibitors could reduce this discrepancy, due to their mechanism of action. Key Points 1. JAK inhibitors are considered at a similar level as biologic agents in terms of effectiveness. 2. After ORAL-Surveillance results, real-world data are needed to assess the benefit/risk profile of Jaki. 3. Disagreement between patients and physicians has been previously reported with biologic therapy among patients with rheumatoid arthritis, with patients rating disease activity higher than physicians. 4. Jak inhibitors could reduce this discrepancy, due to their mechanism of action.

Purpose Health care research increasingly relies on assessment of data extracted from electronic medical records (EMRs). Clinical trial adverse event (AE) logs and patient-reported outcomes (PROs) are sources of data often available in the context of specific research projects. The aim of this study was to evaluate the extent of data concordance from these sources. Patients and Methods Patients enrolled in clinical trials or receiving standard treatment for lung cancer (n = 62) completed validated questionnaires on physical and psychological symptoms at up to three assessment points. Temporally matched documentation was extracted from EMR notes and, for clinical trial participants (n = 41), AE logs. Evaluated data included symptom assessment, vital signs, medication logs, and laboratory values. Agreement (positive, negative) and Cohen’s κ coefficients were calculated to assess concordance of symptoms among sources, with PROs considered the gold standard. Results Patient-reported weight loss correlated significantly with clinical measurements (t = 2.90; P = .02), and average number of PROs correlated negatively with albumin concentration, supporting PROs as the gold standard. Comparisons of PROs versus EMR yielded poor concordance across 11 physical symptoms, anxiety, and depressive symptoms (all κ < 0.40). Providers under-reported the presence of each symptom in the EMR compared with PROs. AE logs showed similarly poor concordance with PROs (all κ < 0.40, except shortness of breath). Negative agreement among sources was higher than positive agreement for all symptoms except pain. Conclusion There was poor concordance between EMR notes and AE logs with PROs. Findings suggest that EMR notes and AE logs may not be reliable sources for capturing physical and psychological symptoms experienced by patients with lung cancer, supporting use of PRO assessments in oncology practices.

ABSTRACT Background Aesthetics and patient‐reported experiences (PREs) and outcomes (PROs) influence treatment choices, but have been largely overlooked in periodontology and implant dentistry. This consensus conference evaluated these factors by focusing on gingival recession defects (GRDs), immediate or early/delayed implant placement and peri‐implant buccal soft‐tissue dehiscences. Methods The workshop discussions were informed by five specifically commissioned systematic reviews covering PREs, PROs and clinician‐reported outcomes (CROs), including effectiveness, aesthetics and other concerns. Results Following treatments such as coronally advanced flaps (CAFs) with a connective tissue graft (CTG) for root coverage (RC), a positive correlation was noted between clinician‐reported aesthetic scores and RC measures for GRDs. However, a corresponding correlation for patient‐reported aesthetic perceptions was not evident. In dental implant procedures, the addition of a CTG immediately after implant placement significantly mitigated the apical shift in the mid‐facial mucosal level. However, clinician‐ and patient‐reported aesthetic scores were not reflective of these changes. Neither set of scores captured the impact of grafting on improving peri‐implant soft‐tissue volume. In the treatment of peri‐implant dehiscences, soft‐tissue augmentation (STA), using CTG beneath CAF, resulted in favourable outcomes in clinician‐ and patient‐perceived aesthetics. Conclusions Clinician and patient views provide complementary perspectives for evidence‐based clinical decision making. They need to be integrated into select interventions. Specific trials are needed encompassing PREs, PROs and CROs and reporting benefits and harms.

OBJECTIVES To compare early implant placement (EP) to alveolar ridge preservation and delayed implant placement (ARP/DP) in terms of contour changes, along with clinician- and patient-reported outcome measures (PROMs) until the delivery of the implant-supported restoration. MATERIALS AND METHODS Patients with a failing single tooth in the maxilla or mandible were recruited in two centres. After tooth extraction, patients were randomly assigned (1:1) to either EP or ARP/DP. At abutment connection and crown delivery, the buccal contour changes at 3 mm below the mucosal margin (primary outcome) along with clinician- (ease of treatment) and patient-reported outcomes were assessed using numeric rating scales and OHIP-14. RESULTS A total of 46 patients were analysed. The mean buccal contour at abutment connection decreased by -1.2 ± 0.6 mm in group EP and -1.6 ± 0.8 mm in group ARP/DP (estimated mean difference; 0.45 [95%CI, -0.02; 0.94]; p = .061) with no significant differences between the groups. ARP/DP was consistently easier than EP across all stages of the surgery (estimated mean difference; 2.0 [95%CI, 1.3; 2.7] p < .001): during flap elevation (EP: 4.8 vs. ARP/DP: 1.6), implant placement (EP: 5.7 vs. ARP/DP: 2.2) and wound closure (EP: 3.4 vs. ARP/DP: 1.6). Both interventions improved quality of life but patients who underwent ARP/DP were significantly more satisfied at the time of crown delivery (ARP/DP: 9.6 vs. EP: 9.1, p = .02). CONCLUSIONS EP and ARP/DP show no significant differences in buccal contour changes, aesthetics and patient-reported outcomes. However, ARP/DP is an easier procedure at all stages of the surgery compared to EP and could therefore be the preferred therapy for less experienced clinicians.

Background: Application of different value sets to health-related quality of life (HRQoL) measured with the EQ-5D-3L may lead to different results due to differences in methods, perspectives, and countries used. Focusing on concordance, this study aimed at understanding the implications of applying EQ-5D-3L value sets from Sweden, Germany, Denmark, and the UK to evaluate HRQoL of patients undergoing total hip replacement (THR) in Sweden before and after surgery. Methods: We performed a longitudinal study of patients in the Swedish Hip Arthroplasty Register from preoperative stage to 1-year follow-up (n = 73,523) using data collected from 2008 to 2016. Eight EQ-5D-3L value sets from the four countries were compared based on a valuation method (visual analogue scale (VAS) or time trade-off (TTO)), perspective (experience-based or hypothetical), and country. Concordance among the value sets with patient-reported EQ VAS score was also assessed. Longitudinal changes in EQ-5D-3L index over the 1-year follow-up were compared across value sets by method, perspective, and country. Results: Value sets based on the same method and perspective showed higher concordance in EQ-5D-3L index at both measurement time points than other comparisons. In the comparisons by perspective, VAS value sets showed higher concordance than TTO value sets. The Swedish VAS and the Danish TTO value sets showed the highest levels of concordance with patient-reported EQ VAS scores. Generally, value sets based on the same method and perspective had the smallest mean differences between changes in EQ-5D-3L indices from preoperative to 1-year postoperative follow-up. Conclusion: Among THR patients value sets based on the same method and perspective, a direct transfer of results across countries could be meaningful. In cases of differences in methods and perspectives among value sets, transfer of value sets across settings would have to consider conversion through crosswalk.

Background This proof-of-concept retrospective case study investigated whether patient-reported outcomes (PRO) instruments, designed to capture symptomatic adverse event data, could identity a known exposure–response (ER) relationship for safety characterized in an original FDA analysis of an approved anti-cancer agent. PRO instruments have been designed to uniquely quantify the tolerability aspects of exposure-associated symptomatic adverse events. We explored whether standard ER analyses of clinician-reported safety data for symptomatic adverse events could be complemented by ER analysis using PRO data that capture and quantify the tolerability aspects of these same symptomatic adverse events. Methods Exposure-associated adverse event data for diarrhea were analyzed in parallel in 120 patients enrolled in a clinical trial using physician reported Common Terminology Criteria for Adverse Events (CTCAE) and patient-reported symptomatic adverse event data captured by the National Cancer Institute’s (NCI) PRO Common Terminology Criteria for Adverse Events (PRO-CTCAE) instrument. Comparative ER analyses of diarrhea were conducted using the same dataset. Results from the CTCAE and PRO-CTCAE ER analyses were assessed for consistency with the ER relationship for diarrhea established in the original NDA using a 750-patient dataset. The analysis was limited to the 120-patient subset with parallel CTCAE and PRO-CTCAE assessments. Results Within the same 120-patient dataset, ER analysis using dense, longitudinal PRO-CTCAE-derived data was sensitive to identify the known ER relationship for diarrhea, whereas the standard CTCAE based ER analysis was not. Conclusions ER analysis using PRO assessed symptomatic adverse event data may be a sensitive tool to complement traditional ER analysis. Improved identification of relationships for safety, by including quantification of the tolerability aspect of symptomatic adverse events using PRO instruments, may be useful to improve the sensitivity of exposure response analysis to support early clinical trial dosage optimization strategies, where decision making occurs within limited small patient datasets.

Sleep loss is a key factor contributing to disease burden in people with atopic dermatitis (AD). Mitigating itch to improve sleep is an important outcome of AD treatment. This study explored the content validity and measurement properties of the Sleep-Loss Scale, a single-item rating scale for assessing itch interference with sleep in clinical trials of AD treatments. Concept elicitation and cognitive debriefing interviews were conducted with 21 adults and adolescents (12–17 years of age) with moderate-to-severe AD to develop a conceptual model of patient experience in AD and explore the content validity of the scale. Data collected from adults with moderate-to-severe AD enrolled in a phase 2b study (NCT03443024) were used to assess Sleep-Loss Scale’s psychometric performance, including reliability, construct validity, and ability to detect change. Meaningful within-patient change (MWPC) thresholds were also determined using anchor-based methods. Qualitative findings from concept elicitation highlighted the importance of sleep-loss related to itch in AD. Debriefing analysis of the Sleep-Loss Scale indicated that the scale was relevant, appropriate, and interpreted as intended. Trial data supported good reliability, construct validity and ability to detect improvement. MWPC was defined as a 1-point improvement using trial data, a finding supported by qualitative data. The Sleep-Loss Scale provides a valid and reliable patient-reported measure of the impact of itch on sleep in patients with AD, and can detect change, indicating it is fit-for-purpose to evaluate the efficacy of AD treatments in moderate-to-severe patients. • The Sleep-Loss Scale is a novel rating scale for assessing the impact of itch on sleep in atopic dermatitis (AD). • A mixed-methods approach was used to explore the content validity and psychometric properties of the Sleep-Loss Scale. • Qualitative results supported sleep loss as a common experience in AD and indicated that the Sleep-Loss Scale was relevant, appropriate, and interpreted as intended. • Quantitative results demonstrated the Sleep-Loss Scale is valid, reliable, and able to detect change. • The Sleep-Loss Scale was found to be fit-for-purpose for use in clinical trials of AD treatments among moderate-to-severe patients.

Zirconia dental implants show excellent biocompatibility and tissue integration, low affinity for plaque, and favorable biomechanical properties. However, these objective measures do not adequately replicate the patient’s perception. This systematic review evaluated the evidence on patient-reported outcome (PROs) in zirconia dental implant treatment. A systematic literature review was conducted following the PRISMA guidelines, utilizing six electronic databases, and supplemented by a manual search of relevant journals and websites to ensure a thorough and comprehensive screening process. The identified studies were subjected to preidentified inclusion criteria. Only controlled clinical trials published in English were considered without limitations on the year of publication. Data on the study characteristics (follow-up, survival rate (%), implant system, number of implants, and type of treatment), PROMs, level of evidence, and Methodological Index for Nonrandomized Studies (MINORS) Bias Score were extracted. The initial database and hand search yielded 596 articles; 189 were included in the title and abstract screening after excluding the duplicates. Eighteen articles were selected based on the inclusion criteria, among which six were excluded because they did not match the research question. Thus, the final selection comprised 12 articles. Most PROMs (aesthetics, speaking, comfort, chewing ability, and general satisfaction) at prosthetic delivery revealed significantly improved average scores than those at pretreatment. Despite the respective limitations of the articles included in this systematic review, patients revealed high satisfaction levels with regard to zirconia dental implants. A high level of heterogeneity was observed among the instruments used for measuring the patient-reported outcomes in patients with zirconia implants, thus highlighting the need to develop specific PROMs in the future.

Patient-reported outcome measures (PROMs) provide invaluable information on patients’ health outcomes and can be used to improve patient-related outcomes at the individual, organizational and policy levels. This systematic review aimed to a) identify contemporary applications and synthesize all evidence on the use of PROMs in these contexts and b) to determine characteristics of interventions associated with increased effectiveness. Five databases were searched for studies providing quantitative evidence of the impact of PROM interventions. Any study design was permitted. An overall benefit (worsening) in outcome was defined as a statistically significant improvement (deterioration) in either a PROM, patient-reported experience measure or clinical outcome. Study quality was assessed using the Effective Public Healthcare Panacea Project’s Quality Assessment Tool for Quantitative Studies. A narrative synthesis was conducted. Seventy-six studies of the 11,121 articles identified met the inclusion criteria. At the individual level, 10 (43%) of 23 studies that fed back PROMs to the patient or healthcare provider showed an improvement in outcome. This percentage increased in studies which used PROMs to monitor disease symptoms and linked these to care-pathways: 17 (68%) of 25 studies using this mechanism showed an improvement. Ten (71%) of 14 studies using PROMs to screen for disease found a benefit. The monitoring and screening approach was most effective using PROMs covering cancer-related, depression and gastro-intestinal symptoms. Three studies found that the mere collection of PROMs resulted in improved outcomes. Another three studies used PROMs in decision aids and found improved decision quality. At the organizational/policy level, none of the 4 studies that used PROMs for benchmarking found a benefit. The three studies that used PROMs for in-depth performance analyses and 1 study in a plan-do-study-act (PDCA) cycle found an improvement in outcome. Studies employing disease-specific PROMs tended to observe improved outcomes more often. There are concerns regarding the validity of findings, as studies varied from weak to moderate quality. The use of PROMs at the individual level has matured considerably. Monitoring/screening applications seem promising particularly for diseases for which treatment algorithms rely on the experienced symptom burden by patients. Organizational/policy-level application is in its infancy, and performance evaluation via in-depth analyses and PDCA-cycles may be useful. The findings of this review may aid stakeholders in the development and implementation of PROM-interventions which truly impact patient outcomes.

To identify available patient-reported outcome measures (PROMs) used to evaluate central sensitization (CS) manifestations in chronic pain conditions and evaluate the quality of psychometric properties of those instruments. A comprehensive search across multiple electronic databases was conducted for relevant studies following the specification of eligibility criteria and development of key search terms. After screening and full-text review, the methodological quality of studies and psychometric properties of PROMs were assessed and summarized using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist and scoring manual. The results were statistically pooled in a meta-analysis, specifically test-retest reliability, based on data availability and consistency of findings across studies. A total of fifty-eight studies evaluating eight instruments in adult patients with chronic pain were included. The methodological quality of the included studies was varied. Most identified PROMs have limited evidence regarding their measurement properties. The Central Sensitization Inventory (CSI) received the highest overall ratings for most measurement properties among all the instruments, followed by Pain Sensitivity Questionnaire (PSQ) and Fibromyalgia Survey Questionnaire (FSQ). Based on pooled data from available studies, the test-retest reliability of the CSI was found to be excellent, with an intra-class correlation coefficient (ICC) of 0.93 (95% CI: 0.91–0.95) for overall chronic pain, 0.90 (95% CI: 0.87–0.93) for chronic musculoskeletal pain and 0.93 (95% CI: 0.88–0.99) for chronic neck pain. PSQ also demonstrated excellent test-retest reliability, showing an ICC of 0.86 (95% CI: 0.72–0.99) for chronic pain. Although not all properties have been studied, the CSI, which received the highest overall ratings, could serve as a reliable PROM assessing CS in chronic pain. More studies should be performed to comprehensively evaluate all measurement properties of all included instruments. Central sensitization (CS) is a key process in many chronic pain conditions where the central nervous system becomes overly sensitive, causing persistent pain even in the absence of clear tissue damage. To evaluate the outcome of care for pain associated with CS, healthcare providers use patient-reported outcome measures (PROMs) as outcome measurement instruments that capture the patient’s pain experience. However, the quality of these PROMs is very important to ensure they accurately reflect the patient’s experience and the outcomes they intend to measure. In this study, we evaluated and rated the psychometric properties (also referred to as measurement properties) - reliability, validity and responsiveness of eight different PROMs used to assess CS manifestation in chronic pain. Our findings revealed that most PROMs lack sufficient evidence supporting their quality. Notably, the Central Sensitization Inventory (CSI) showed promising results, but further research is needed to assess all properties. Our findings recommend more studies to fully evaluate all properties of all PROMs, which would be beneficial for both clinicians and researchers to better understand, measure the outcome of care and manage chronic pain associated with CS.

High quality science relies upon psychometrically valid and reliable measurement, yet very few Patient Reported Outcome Measures (PROMs) have been developed or thoroughly validated for use with autistic individuals. The present commentary summarizes the current state of autism PROM science, based on discussion at the Special Interest Group (SIG) at the 2022 International Society for Autism Research (INSAR) Annual Meeting and collective expertise of the authors. First, we identify current issues in autism PROM research including content and construct operationalization, informant‐structure, measure accessibility, and measure validation and generalization. We then enumerate barriers to conducting and disseminating this research, such as a lack of guidance, concerns regarding funding and time, lack of accessible training and professionals with psychometric skills, difficulties collecting large representative samples, and challenges with dissemination. Lastly, we offer future priorities and resources to improve PROMs in autism research including a need to continue to evaluate and develop PROMs for autistic people using robust methods, to prioritize diverse and representative samples, to expand the breadth of psychometric properties and techniques, and to consider developing field specific guidelines. We remain extremely optimistic about the future directions of this area of autism research. This work is well positioned to have an immense, positive impact on our scientific understanding of autism and the everyday lives of autistic people and their families.

The Stool Frequency (SF) and Abdominal Pain (AP) items from the Crohn’s Disease Activity Index are together referred to as the “Patient Reported Outcome” (PRO). The SF item measures the number of very soft/liquid stools and the AP item measures abdominal pain severity, which are common Crohn’s disease (CD) symptoms that patients consider important to treat. This study evaluated the psychometric properties of both PRO items separately and estimated thresholds for clinical remission in moderately to severely active CD. The measurement properties of the PRO items were analyzed using pooled data from VIVID-1 (NCT03926130), a Phase 3, randomized, placebo- and active-controlled study in adults with moderately to severely active CD. Analyses used weekly average scores of the SF and AP items at Weeks 0 (Baseline), 4, 12, and 52. Remission thresholds were estimated using the Patient Global Rating of Severity (PGRS) and Patient Global Impression of Change (PGIC) as primary anchors as well as qualitative evidence from exit interviews. Data from 1065 participants (mean age: 36.2 years [standard deviation: 13 years]) were analyzed. During the trial, scores improved for both PRO items. Both items demonstrated moderate-to-good test-retest reliability for participants defined as stable based on PGRS and PGIC. Most correlations of related assessments were moderate (0.30≤|ρ| <0.70) with SF and moderate-to-large (0.30≤|ρ| ≤0.90) with AP. By contrast, as anticipated, both items had weak correlations (|ρ| <0.30) with endoscopic and laboratory assessments. The PRO items could discriminate between groups of participants known to differ based on other assessments. The PRO items were able to detect change, as score changes in both items between Baseline and Weeks 12 and 52 differed significantly between most PGRS and PGIC categories. Anchor-based analyses combined with responses from the exit interviews suggested that an SF score of ≤ 3 and an AP score of ≤ 1 could together represent clinical remission. These results support the reliability, construct-validity, and responsiveness of both PRO items in moderately to severely active CD and confirm previously suggested scores for both items that could represent clinical remission. Clinicaltrials.gov, NCT03926130. Registered 23 April 2019, https://clinicaltrials.gov/study/NCT03926130. This study focused on two of the questions (or “items”) from a commonly used questionnaires in Crohn’s disease: the Stool Frequency (SF) item, which counts the number of very soft or liquid stools in a 24-hour period, and the Abdominal Pain (AP) item, which rates severity of abdominal pain in the same time period. We are referring to these two items as the “Patient Reported Outcome” (PRO). This study looked at how well each of these two items did in measuring what they are supposed to measure by using data from people with Crohn’s disease who had participated in a clinical trial. Trial participants answered the SF and AP daily during the trial by using an electronic diary device. Some of the participants were also interviewed to find out when they would consider their disease to be in remission (no longer active). The results showed that both PRO items (SF and AP) can be used by people with Crohn’s disease to accurately measure their very soft or liquid stool frequency and abdominal pain. Further, we estimated which scores on SF and AP could tell us whether someone with Crohn’s disease has achieved remission of their very soft or liquid stools and of their abdominal pain.

We thank the authors for their thoughtful and constructive comments on our study and for highlighting important directions for further development of outcome assessment in thoracic myelopathy. We agree that our findings should be regarded as an initial step toward more standardized measurement rather than as establishing a definitive thoracic-specific outcome framework. 1 Our study evaluated temporal test-retest stability, but not agreement between independent examiners. Given the favorable inter-rater reliability of JOA and mJOA reported in degenerative cervical myelopathy, 2 broadly comparable reliability might be expected in isolated thoracic myelopathy, where upper-extremity dysfunction is generally absent and the corresponding JOA/mJOA items therefore typically score at or near the maximum, or are omitted in thoracic-adapted scoring. Nevertheless, direct evaluation using independent paired assessments would be valuable to confirm reproducibility across centers. The authors also appropriately emphasize the potential role of objective performance measures. JOA-based scores demonstrated large internal responsiveness in our study despite relatively weak correlations between their changes and Δ EQ-5D. 1 Rather than identifying a single superior measure, this finding supports a multidimensional approach to recovery. The AO Spine RECODE-DCM initiative identified neurological function, pain, life impact, radiology, economic impact, and adverse events as core outcome domains in degenerative cervical myelopathy. 3 A similarly structured framework should be considered for thoracic myelopathy, with greater emphasis on trunk and lower-extremity function and possible incorporation of objective measures such as gait speed, Timed Up and Go, balance, and wearable-derived mobility. Our experience in conducting this multicenter study also highlighted an important challenge in thoracic myelopathy. Pure thoracic

BACKGROUND 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. OBJECTIVES To identify PROMs that have been developed, validated or cross-culturally adapted to Arabic and evaluate methodological qualities of CCA and measurement properties. METHODS 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. RESULTS 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). CONCLUSION 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.

BACKGROUND Measuring maternity care outcomes based on what women value is critical to promoting woman-centred maternity care. Patient-reported outcome measures (PROMs) are instruments that enable service users to assess healthcare service and system performance. AIM To identify and critically appraise the risk of bias, woman-centricity (content validity) and psychometric properties of maternity PROMs published in the scientific literature. METHODS MEDLINE, CINAHL Plus, PsycINFO and Embase were systematically searched for relevant records between 01/01/2010 and 07/10/2021. Included articles underwent risk of bias, content validity and psychometric properties assessments in line with COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidance. PROM results were summarised according to language subgroups and an overall recommendation for use was determined. FINDINGS Forty-four studies reported on the development and psychometric evaluation of 9 maternity PROMs, grouped into 32 language subgroups. Risk of bias assessments for the PROM development and content validity showed inadequate or doubtful methodological quality. Internal consistency reliability, hypothesis testing (for construct validity), structural validity and test-retest reliability varied markedly in sufficiency and evidence quality. No PROMs received a level 'A' recommendation, required for real-world use. CONCLUSION Maternity PROMs identified in this systematic review had poor quality evidence for their measurement properties and lacked sufficient content validity, indicating a lack of woman-centricity in instrument development. Future research should prioritise women's voices in deciding what is relevant, comprehensive and comprehensible to measure, as this will impact overall validity and reliability and facilitate real-world use.

Purpose Patient-reported outcome measures (PROMs) are used in individuals experiencing long-term effects from COVID-19 infection, or Long COVID, to evaluate the quality of life and functional status of these individuals. However, little is known about which PROMs are being utilised and the psychometric properties of these PROMs. Our purpose was thus to explore which PROMs are used in Long COVID patients and to discuss the psychometric properties of the PROMs. Methods For this rapid review, a systematic literature search was performed in the PubMed, Embase, and CINAHL databases. The found studies were screened using the PRISMA flowchart. We then performed study quality appraisal and assessed the psychometric properties of the found PROMs. Results Per the systematic literature search and after removal of duplicates, 157 publications were identified for individual screening. After screening and eligibility assessment, 74 articles were selected for our review. In total, 74 PROMs were used and primarily comprised quality of life, fatigue, breathlessness, mental health, and smell/taste issues in COVID "long haulers." Five studies used newly developed, COVID-19-specific PROMs. We assessed the psychometric properties of the 10 most-used PROMs. The majority were found to be reliable and valid instruments. EQ-5D-5L was the most popular and highly rated PROM. Conclusions We assessed PROMs used in Long COVID patients and evaluated their psychometric properties. EQ-5D-5L was the most favourably rated PROM. PROMs addressing mental health issues are crucial in managing anxiety and depression in Long COVID patients. New COVID-specific PROMs assess functional status and smell/taste perception and show great utilisation potential in olfactory training at COVID smell clinics. However, many reviewed PROMs currently lack sufficient analysis of their psychometric properties. Therefore, future research needs to examine these measures.

No abstract available

Guidelines for the development and validation of patient-reported outcome measures recommend incorporating patient input to ensure relevance and comprehension, but do not clearly define what patient input entails. As a result, meaningful patient engagement is often conflated with research participation. This study uses the adaptation and validation of the FACE-Q Craniofacial Module patient-reported outcome measure for ophthalmology patients as a case study to showcase how patients can be engaged as partners across the different phases of patient-reported outcome measure adaptation, and illustrate how their involvement influenced the adaptation process. Patient engagement strategies across five phases of the adaptation and validation of the FACE-Q Craniofacial Module (Study development, Item adaptation, Item reduction, Psychometric evaluation, and Dissemination) were retrospectively mapped to the International Association for Public Participation Spectrum. This framework outlines five levels of engagement: Inform, Consult, Involve, Collaborate, and Empower, and was used to evaluate the extent and nature of engagement. Patient partners (n = 8) with lived experience of retinoblastoma, strabismus, corneal anesthesia, and ocular prostheses were engaged across the five phases of the study. Mapping engagement activities to the International Association for Public Participation Spectrum revealed that the “Inform” and “Involve” levels of engagement were present in all five phases. The most extensive engagement, spanning all levels of the International Association for Public Participation Spectrum, occurred during the “Item Reduction” and “Dissemination” phases of the study. The current case study showcases that patient engagement can be incorporated intentionally across all phases of patient-reported outcome measure adaptation and validation. The International Association for Public Participation Spectrum provided a structured approach to map and document the nature and extent of engagement across our study. Our case study may support the planning, execution, and reporting of patient engagement strategies in future patient-reported outcome measure development and adaptation studies.

Treatment burden is the workload of healthcare and self-management and the impact of this workload on patient functioning and wellbeing. Treatment burden can lead to poorer outcomes in people with long-term conditions. The aims of this review are to identify and describe existing treatment burden patient-reported outcome measures (PROMs), explore their psychometric properties, and to compare frequency and predictors of treatment burden and associations with health outcomes. Five databases were searched for studies which used, developed, or validated PROMs of treatment burden in long-term conditions. Studies were synthesised quantitively and narratively. Two-hundred and four studies were included. The largest proportion ( n  = 64, 31.4%) were conducted in the USA and focused on long-term conditions in general or multimorbidity ( n  = 72, 35.3%). We identified 53 validated PROMs and 22 ad hoc measures, but only 10 PROMs explicitly or comprehensively assessed treatment burden. The level of treatment burden varied across studies but was moderate across most long-term conditions, with several studies reporting a high level of treatment burden. Treatment burden was higher in those with more long-term conditions, of younger age, with poor mental health and lower social support. Higher treatment burden was also associated with lower quality of life. This review comprehensively mapped treatment burden PROMs, their psychometric properties, and summarised key considerations for researchers and policy makers who may wish to measure and address treatment burden. Younger adults with multimorbidity and lower levels of social support are particularly at risk of treatment burden and lower quality of life. Interventions could be targeted to those at highest risk.

Artificial intelligence (AI) and machine learning (ML) are increasingly integrated into healthcare, offering potential advancements in patient-reported outcome measures (PROMs) for surgical populations. Improved PROMs can enhance patient-centered care by accurately capturing patient experiences with minimal burden. In the context of surgery, where recovery trajectories vary widely, this study aims to systematically review the use of AI and ML in the development, application, and prediction capabilities of PROMs in surgical populations, with a focus on psychometric properties and the predictive accuracy of post-surgical outcomes. A comprehensive search of the PubMed database was conducted from inception until August 2024. Studies were included if they utilized AI or ML in the development, application, or predicting PROMs for surgical patients. Methodological quality was assessed using COSMIN and PROBAST tools, depending on study design. A qualitative synthesis of findings was performed. Twenty-two studies met the inclusion criteria, with 19 rated as high quality. Six studies focused on developing computer adaptive tests (CAT) PROMs, seven on evaluating psychometric properties, and five on ML for post-surgical outcome prediction. CAT PROMs showed comparable measurement accuracy to traditional PROMs, good to excellent construct validity, and significantly reduced patient burden by reducing the length of questionnaires. ML algorithms, such as logistic regression, random forests, extreme gradient boosting, and neural networks, achieved similar predictive accuracy for post-surgical outcomes, with no single model demonstrating consistent superiority. AI and ML have the potential to improve PROM utilization in surgical care by enhancing efficiency and personalization while maintaining data quality. Clinicians can use AI-driven PROMs to reduce patient burden and integrate ML models for accurate post-surgical outcome prediction, thereby optimizing patient-centered care.

Systematic reviews of patient-reported outcome measures (PROMs) are important tools to select the most suitable PROM for a study or clinical application. Conducting these reviews is challenging, and the quality of these reviews needs to be improved. We updated the COSMIN guideline for systematic reviews of PROMs, including the COSMIN Risk of Bias checklist, and the COSMIN criteria for good measurement properties. Adaptations to the methodology were based on our experience with applying the COSMIN guideline, through discussions among the authors, and results from two related Delphi studies. The updated guideline places more emphasis on key aspects that are often missing or sub optimally conducted in published systematic reviews of PROMs, such as formulating a well-defined research question and developing a comprehensive search strategy, assessing risk of bias, applying criteria for good measurement properties, summarizing results, and grading the quality of the evidence. We also stress the importance of evaluating the measurement properties of each subscale of a PROM separately and evaluating content validity of all included PROMs. The quality of systematic reviews of PROMs can be improved by using this updated version of the COSMIN guideline for systematic reviews of PROMs. Improved quality will lead to better PROM selection and increased standardization of PROM use. Patient-reported outcome measures (PROMs) are questionnaires that measure aspects of health from the patient perspective. To measure a specific health aspect, often dozens of PROMs are available. To choose the best PROM, a systematic review of PROMs can be conducted, in which all information on the quality and feasibility of each available PROM is collected, rated, and compared. Based on such a review a choice for the most suitable PROM for a certain study or clinical application can be made. However, conducting a systematic review of PROMs is very challenging, because nine quality aspects of PROMs need to be taken into account. In this article, we present an updated step-by-step guideline for conducting systematic reviews of PROMs. Each of these steps is described in detail in an accompanying manual. This updated guideline helps researchers to conduct systematic reviews of PROMs in a systematic and transparent way. It also helps readers of systematic reviews to understand how the review was conducted and to check the conclusions about which PROMs are recommended based on their quality.

Patient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses unique challenges because of small patient populations, disease heterogeneity, lack of natural history knowledge, and short-term studies. This research aims to identify key factors to consider when using different types of PROMs in HTA for rare disease treatments (RDTs). A scoping review of scientific and grey literature was conducted, with no date or publication type restrictions. Information on the advantages of and the challenges and potential solutions when using different types of PROMs for RDTs, including psychometric properties, was extracted and synthesized. Of 79 records from PubMed, 32 were included, plus 12 records from the grey literature. PROMs for rare diseases face potential data collection and psychometric challenges resulting from small patient populations and disease heterogeneity. Generic PROMs are comparable across diseases but not sensitive to disease specificities. Disease-specific instruments are sensitive but do not exist for many rare diseases and rarely provide the utility values required by some HTA bodies. Creating new PROMs is time and resource intensive. Potential solutions include pooling data (multi-site/international data collection), using computer-assisted technology, or using generic and disease-specific PROMs in a complementary way. PROMs are relevant in HTA for RDTs but pose a number of difficulties. A deeper understanding of the potential advantages of and the challenges and potential solutions for each can help manage these difficulties.

Cancer worry is a key psychosocial outcome among individuals undergoing genetic testing for hereditary cancer risk, yet validated patient-reported outcome measures (PROMs) in the German-speaking context are limited. We aimed to evaluate the psychometric properties of the German version of the 8-item Cancer Worry Scale (CWS) in a clinical registry-based cohort. In this cross-sectional study, German-speaking adults undergoing genetic testing were recruited from the ATHENA hereditary cancer registry. Structural validity, internal consistency, test–retest reliability, construct validity, and floor and ceiling effects were assessed in accordance with COSMIN recommendations for PROM validation. Predefined hypotheses were used to evaluate construct validity. A total of 202 participants (median age 41 years, 93% female) were included. The CWS demonstrated high internal consistency (Cronbach’s α = 0.87) and a one-factor CFA supported its unidimensional structure (CFI = 0.986, TLI = 0.980, RMSEA = 0.048, SRMR = 0.033). Test–retest ICC was 0.86 (95% CI 0.73–0.93). CWS scores correlated positively with anxiety (τ=0.44) and depression (τ=0.27), and inversely with mental well-being (τ=−0.29), and showed a smaller correlation with physical health (τ=−0.10). Evidence for known-groups validity was inconclusive. The German 8-item CWS demonstrated sufficient structural validity and internal consistency, with a good test-retest reliability point estimate and partial support for the predefined construct validity hypotheses. Evidence for known-groups validity remains inconclusive. Further studies are warranted to establish responsiveness and clinically meaningful thresholds before routine clinical implementation.

BACKGROUND Patient-reported outcomes (PROs) are important measures of treatment effect and can be used to inform the approval of cardiovascular drugs and devices by the U.S. Food and Drug Administration (FDA). PURPOSE To catalogue the health status patient-reported outcome measures (PROMs) validated in cardiovascular diseases (CVDs), describe their psychometric properties, and assess adherence with both FDA recommendations and the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) framework. DATA SOURCES MEDLINE, EMBASE, CINAHL, and Allied and Complementary Medicine Database from inception to August 2022. STUDY SELECTION Studies that developed and/or validated health status PROMs in CVD populations. DATA EXTRACTION Two study authors extracted data on CVD type, PROM psychometric properties, and adherence to FDArecommendations. The risk of bias informing the development or validation of PROMs was assessed using the COSMIN framework. DATA SYNTHESIS Fifty health status PROMs (described in 83 studies) were identified, of which 45 were disease specific and 5 were generic. Eleven (22%) of the 50 PROMs validated in CVDs had minimally important differences (MIDs) established, and 8 (16%) reported on the validation of all psychometric properties recommended by the FDA. By COSMIN standards, only 2 PROMs (4%) had all of their psychometric properties rated as sufficient in quality, and 32 PROMs (64%) had less than 50% of psychometric properties rated as sufficient. LIMITATION The quality of reporting varied across included studies. CONCLUSION Of 50 PROMs validated in CVDs, only a small minority reported on the validation of all FDA-recommended psychometric properties, had psychometric properties rated as sufficient by COSMIN, or had MIDs established. Given the use of PROMs to guide FDA approvals of drugs and devices in CVDs, there is a need for better adherence to quality standards in PROM validation. PRIMARY FUNDING SOURCE None.

Idiographic patient-reported outcome measures (I-PROMs) are a growing set of individualized tools for use in routine outcome monitoring (ROM) in psychological therapies. This paper presents a position statement on their conceptualization, use, and analysis, based on contemporary evidence and clinical practice. Four problem-based, and seven goal-based, I-PROMs, with some evidence of psychometric evaluation and use in psychotherapy, were identified. I-PROMs may be particularly valuable to the evaluation of psychological therapies because of their clinical utility and their alignment with a patient-centered approach. However, there are several challenges for I-PROMs: how to generate items in a robust manner, their measurement model, methods for establishing their reliability and validity, and the meaning of an aggregated I-PROM score. Based on the current state of the literature, we recommend that I-PROMs are used to complement nomothetic measures. Research recommendations are also made regarding the most appropriate methods for analyzing I-PROM data.

Introduction Woman-centred care is the right of every woman receiving maternity care, irrespective of where care is being received and who is providing care. This protocol describes a planned systematic review that will identify, describe and critically appraise the psychometric properties of maternity patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs). The woman-centricity of PROM and PREM development and content validation (ie, the extent to which women were involved in these processes) will also be assessed. This information will be used to develop a maternity PROMs and PREMs database to support service and system performance measurement, and value-based maternity care initiatives. Methods and analysis This study will be guided by the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline for systematic reviews of outcome measurement instruments. Studies identified via MEDLINE, CINAHL Plus, PsycINFO and EMBASE describing the development, content validation and/or psychometric evaluation of PROMs and PREMs specifically designed for maternity populations throughout pregnancy, childbirth and postnatal periods will be considered if published from 2010 onward, in English, and available in full text. The COSMIN risk of bias checklist will be used to evaluate the quality of studies reporting on the development, content validation and/or psychometric evaluation of PROMs and PREMs. COSMIN criteria for good content validity will be used to assess the woman-centricity of PROM and PREM development and content validation studies. COSMIN standards of good psychometric properties will be used to evaluate the validity and reliability of the identified instruments. Ethics and dissemination Ethical permission for this research is not required. The findings of this research will be submitted for publication in an international, peer-reviewed journal. Abstracts for national and international conference presentations will also be submitted. The proposed maternity PROMs and PREMs database will be freely accessible online, and developed with consumer input to ensure its usefulness to a range of maternity care stakeholders. PROSPERO registration number CRD42021288854.

Background Patient-reported outcome measures (PROMs) are key elements of assessing the efficacy of perioperative pain management. Here, we aimed to capture the association of 10 individually reported aspects of patient's specific impression of change since surgery (PSIC) related to four outcome domains of a previously defined core outcome set, relative to the patient's global impression of change (PGIC). We further evaluated the influence of type of surgery, sex, preoperative baseline characteristics, and satisfaction with pain management on PGIC. Methods This exploratory analysis used the PROMPT NIT-1 study data (2661 patients, 18 sites, four surgery types: total knee arthroplasty, sternotomy, breast cancer surgery, or endometriosis surgery). Male and female adults were included. All PROMs were assessed on postoperative day 3. We used ordinal regression models with PGIC as a dependent variable and PSICs as independent variables. Results The overall model achieved a pseudo-R2 of 0.55 (relative domain contributions: pain intensity 55%, self-efficacy 19%, adverse events 15%, and pain-related interference of physical functioning 10%). Pain-related worrying and depression had no association with the PGIC, whereas anxiety, preoperative pain, opioid intake, low satisfaction with and wish for more treatment, low treatment agency, and overall dissatisfaction were associated with less improvement after surgery. Receiving information about treatment was associated with greater improvement on the PGIC. Conclusions Although all four domains contributed to PGIC after surgery, pain intensity was the most important. These findings highlight the importance of both managing postoperative pain and optimising patient experience by addressing self-efficacy, adverse events, and pain-related interference of physical functioning. Clinical trial registration NCT 03834922

Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare, severe, childhood-onset developmental and epileptic encephalopathies characterized by treatment-resistant epilepsy and varying intellectual disability levels. Clinical outcome assessments (COAs) describe how patients feel, function, or survive, thus providing valuable information on a therapy’s efficacy and impact. Individuals with DS or LGS are heterogeneous, and many have limited verbal abilities and intellectual disability. Existing epilepsy-specific COA measures are unsuitable for DS and LGS clinical trials as many items demonstrate floor effects in these populations. As patients often cannot self-report symptoms, caregiver feedback on the measures’ relevance and understandability is critical when developing COAs to ensure their suitability for the intended population, and that caregivers can help clinicians complete the measures when necessary. We aimed to develop a novel clinician-reported outcomes measure, to be completed in consultation with caregivers at clinic visits, to assess non-seizure symptoms in individuals with DS or LGS using a Clinical Global Impression of Improvement (CGI–I) approach: the CGI-I Non-seizure Symptoms measure. A 13-item initial draft measure was reviewed by experts in a three-round Delphi panel to confirm each item’s relevance and refine descriptions, reduce overlap, and limit respondent burden. Following panel review, three items reached consensus (≥70% agreement of no revision required) and were included in the final measure: communication, alertness, and disruptive behaviors. To ensure caregivers can help clinicians complete the measure, and to establish levels of change in each item domain considered meaningful from their perspective, the three-item measure was cognitively debriefed with caregivers of individuals with DS or LGS. Caregivers showed that each item was understandable by describing their child using the descriptions provided in the measure and reported that items were relevant or important to assess in DS or LGS. Most caregivers reported that even a minimal change to their child’s condition in each domain would be meaningful to them and their child. This CGI-I Non-seizure Symptoms measure represents relevant non-seizure outcomes considered important to individuals with DS or LGS and their families. The systematic development and refinement approach presented here supports its use in DS and LGS clinical trials.

Youth with functional neurological symptom disorder (FNSD) often perceive themselves as having limited capabilities, which may not align with clinical evaluations. This study assessed the disparities between clinician evaluations and patient-reported outcome measures (PROMs) regarding pain, motor function, and learning difficulties in youth with FNSD. Sixty-two youths with FNSD participated in this study, all of whom reported experiencing pain, motor problems, and/or learning difficulties. Clinicians also assessed these domains, resulting in a two-by-two categorization matrix: (1) agreement: child and clinician report "problems"; (2) agreement: child and clinician report "no problems"; (3) disagreement: child reports "problems" while the clinician does not; and (4) disagreement: clinician reports "problems" while the child does not. Agreement/disagreement differences were analyzed. No significant differences in prevalence were observed between the evaluators regarding pain (clinician-85%, child-88%), motor (clinician-98%, child-95%), or learning problems (clinician-69%, child-61%). More than 80% of the children and clinicians report pain and motor disorders. Instances in which children and clinicians reported learning problems (40.3%) exceeded cases in which both reported no problems (9.6%) or only the child reported problems (20.9%). Overall, the agreement between pain and motor function assessments was high (>90%), whereas that concerning learning difficulties was moderate (49.9%). Disagreement in pain/motor assessments was minimal (<5%), whereas for learning difficulties, disagreement rates were high (>20%). In conclusion, a significant concordance exists between PROMs and clinician assessments of pain and motor problems. However, the higher frequency of disagreements regarding learning difficulties emphasizes the importance of incorporating patient and clinician evaluations in pediatric FNSD treatment.

Establishing clinically meaningful changes in pain experiences remains important for clinical trials of chronic pain treatments. Regulatory guidance and pain measurement initiatives have recommended including patient-reported global assessment measures (e.g., Patient-Global Impression of Change [PGIC]) to aid interpretation of within-patient differences in domain-specific clinical trial outcomes (e.g., pain intensity). The objectives of this systematic review were to determine the frequency of global assessment measures inclusion, types of measures, domains assessed, number and types of response options, and how measures were analyzed. Of 4,172 abstracts screened across 6 pain specialty journals, we reviewed 96 clinical trials of chronic pain treatments. Fifty-two (54.2%) studies included a global assessment measure. The PGIC was most common (n=28; 53.8%), with relatively infrequent use of other measures. The majority of studies that used a global assessment measure (n=31; 59.6%) assessed change or improvement in an unspecified domain. Others assessed overall condition severity (n=9; 17.3%), satisfaction (n=8; 15.4%), or overall health status/recovery (n=5; 9.6%). The number, range, and type of response options were variable and frequently not reported. Response options and reference periods even differed within the PGIC. Global assessment measures were most commonly analyzed as continuous variables (n=24; 46.2%) or as dichotomous variables with positive categories combined to calculate the proportion of participants with a positive response to treatment (n=18; 34.6%). This review highlights the substantial work necessary to clarify measurement and use of patient global assessment in chronic pain trials, and provides short and longer-term considerations for measure selection, reporting and analysis, and measure development.

We read with great interest the multicenter prospective study by Kato et al, which provides an important first step toward standardizing outcome assessment in surgically treated thoracic myelopathy. 1 The finding that JOA, JOAt, mJOA, and JOACMEQ lower-extremity function (JOACMEQ-LF) show favorable responsiveness and reliability is clinically valuable, particularly in a condition for which outcome assessment remains poorly standardized. One important next step is to establish reproducibility across clinicians. The present reliability analysis evaluates temporal stability, but not inter-rater agreement, which is especially relevant for clinician-scored instruments intended for multicenter use. In degenerative cervical myelopathy, total mJOA reliability can be good while clinically meaningful disagreement between examiners still occurs. 2 Paired assessment by independent clinicians, accompanied by standardized rater training and evaluation of site-and examiner-level variance, could clarify whether JOAt and mJOA are sufficiently reproducible for routine cross-center comparison. The discrepancy between the large internal responsiveness of JOA-based scores and their weak correlations with change in EQ-5D also suggests that these instruments should be considered complementary rather than interchangeable. Adding objective performance measures such as gait speed, Timed Up and Go, tandem gait, balance testing, fall frequency, or wearable-derived mobility may provide a more direct functional anchor for lower-extremity recovery. Recent studies in degenerative cervical myelopathy indicate that targeted gait and balance measures, as well as smartphone-based performance testing, can capture clinically relevant impairment longitudinally. 3,4 Such measures could also help characterize patients in whom neurological improvement and patient-perceived recovery diverge. A further

Use of patient-reported outcomes (PROs) is recommended by regulatory authorities for improving patient care. A multicenter Italian study tests the psychometric properties of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) across cancer types in Italy. Here the analysis of the ovarian cancer (OC) cohort. A PRO-CTCAE list including 122 items (78 symptomatic adverse events [AEs]), reduced to 78 items (43 symptomatic AEs), was administered to patients with OC on anticancer treatment, at baseline (visit 1) and after 2–6 weeks (visit 2). Convergent validity with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - ovarian cancer module (EORTC QLQ-OV28) and Hospital Anxiety and Depression Scale (HADS) was assessed by Pearson correlation. Known-groups validity and responsiveness were tested with Eastern Cooperative Oncology Group performance status (ECOG PS 0 vs. > 0) and Patient Global Impression of Change (PGIC) scale as anchors. From July 2019 to March 2024, 214 patients filled PRO-CTCAE, 210 OV28 and 209 HADS at visit 1; 192 and 182 filled PRO-CTCAE and PGIC at visit 2, respectively. Median age (IQR) was 60.8 (53.0–68.8) yrs and 88.3% had PS 0. Convergent validity analysis showed high correlation ( r  ≥ 0.50) of PRO-CTCAE hot flushes, hair loss, hand/foot neuropathy, dysgeusia, bloating, abdominal pain, muscle pain, vaginal dryness, heartburn, joint pain, sadness, depression and fatigue with selected OV28 anchors. Anxiety, depression and sadness had medium to large correlation with HADS (r ranging from 0.48 to 0.66). Known-group validity showed small to large correlations (Cohen coefficients ≥ 0.20) with PS for several items. Patients classified as worsened according to the PGIC showed greater deterioration in multiple PRO-CTCAE symptom domains. Psychometric properties of PRO-CTCAEs indicate good construct validity and responsiveness, supporting their role in cancer care.

Radiation dermatitis is a common adverse effect of radiotherapy (RT) in breast cancer patients. Although radiation dermatitis is reported by either the clinician or the patient, previous studies have shown disagreement between clinician-reported outcomes (CROs) and patient-reported outcomes (PROs). This review evaluated the extent of discordance between CROs and PROs for radiation dermatitis. Studies reporting both clinician and patient-reported outcomes for external beam RT were eligible. Nine studies met the inclusion criteria for the systematic review, while 8 of these studies were eligible for inclusion in a meta-analysis of acute and late skin toxicities. We found an overall agreement between CROs and PROs of acute skin colour change, fibrosis and/or retraction, and moist desquamation (p > 0.005). Reporting of late breast pain, breast edema, skin colour change, telangiectasia, fibrosis and/or retraction and induration/fibrosis alone (p > 0.005) were also in agreement between clinicians and patients. Our meta-analysis revealed a greater reporting of acute breast pain by patients (RR = 0.89, 95% CI 0.87–0.92, p < 0.001), greater reporting of acute breast edema by physicians (RR = 1.80, 95% CI 1.65–1.97, p < 0.001) and a greater reporting of late breast shrinkage by patients (RR = 0.61, 95% CI 0.44–0.86, p = 0.005). However, our review was limited by the discrepancies between PRO and CRO measurement tools as well as the absence of standard time points for evaluation of radiation dermatitis. Given potential discrepancies between CROs and PROs, both measures should be reported in future studies. Ultimately, we advocate for the development of a single tool to assess symptoms from both perspectives.

Objective: Assess the inter-rater agreement among DBS outcome measures as rated by patients, caregivers, and clinicians. Background: The Clinician Global Impression (CGI) score and the Patient Global Impression (PGI) score are validated scores that are used to assess patient outcomes. We are using these scores to study outcomes in Parkinson9s disease patients with DBS. Methods: CGI score and two PGI scores (one patient-rated and one caregiver-rated) were obtained for 136 idiopathic Parkinson’s patients following DBS surgery at 3, 6, and 12 months post-intervention. The patient pool included all idiopathic PD patients who underwent DBS surgery as a part of the St. Jude DBS study. To assess inter-rater agreement, we assigned equally spaced scores of 0 - 4 to each outcome category in the two scales and calculated the performed Cochran-Mantel-Haenszel procedure with a 95[percnt] CI and subsequently assigned a weighted Kappa score. Results: There were 84 men and 52 women in the study and the mean age of the cohort was 60.05 ± 8.3 years. The percentage of complete agreement between the examiner and patient scores was 61.8[percnt] at 3 months, 67.4[percnt] at 6 months and 65.2[percnt] at 12 months. The percentage of agreement (with one or less category of disagreement) was 94.9[percnt] at 3 months, 97[percnt] at 6 months and 97[percnt] at 12 months. A similar pattern was observed for the examiner-caregiver and caregiver-patient comparison (p values for all <0.0001). Conclusions: Our pilot study shows relatively strong agreement among patient, caregiver, and clinician ratings of global outcomes following DBS for PD, and this agreement is consistent across the time intervals studied. However, these ratings should not be considered interchangeable. Further investigation of these preliminary findings is needed, particularly in light of the growing emphasis on patient-reported outcomes. Disclosure: Dr. Shahgholi Ghahfarokhi has nothing to disclose. Dr. Deeb has nothing to disclose. Dr. Foote has received personal compensation for activities with Medtronic as an advisory board member. Dr. Wagle-Shukla has nothing to disclose. Dr. Akbar has nothing to disclose. Dr. Rossi has nothing to disclose. Dr. De Jesus has nothing to disclose. Dr. Okun has received royalty payments from Demos, Humana, Amazon, Smashwords, Books4Patients, and Cambridge. Dr. Okun has received research support from the Michael J. Fox Foundation, the National Parkinson Foundation, the Parkinson Alliance, the Smallwo

BACKGROUND Although validated patient-reported outcome (PRO) measurements can categorize patients with inflammatory bowel disease (IBD) into clinical remission or active disease, patients may have different definitions of remission. The purpose of this study was to compare patient-defined remission to remission based on PRO measures and physician global assessment (PGA) and to understand the clinical and demographic factors associated with disagreements. METHODS We retrospectively analyzed 3257 de-identified surveys from 2004 IBD patients who consented to participate in the Crohn's and Colitis Foundation's IBD Qorus Learning Health System between September 2019 and February 2021. We used logistic regression models with generalized estimating equations to analyze the clinical and demographic factors (eg, age, disease duration, health confidence) associated with discordance between patient-defined remission (yes/no) and PRO-defined remission for ulcerative colitis (UC; PRO2: stool frequency, rectal bleeding) and Crohn's disease (CD; PRO-3: average number of liquid stools, abdominal pain, well-being). RESULTS Among patients with UC, overall concordance was 79% between patient self-report and PRO2-defined remission and 49% between patient self-report and PGA-defined remission. Among patients with CD, overall concordance was 69% between patient self-report and PRO3-defined remission and 54% between patient self-report and PGA-defined remission. Patients in PRO-defined remission were more likely to report active disease if they had IBD <5 years and low health confidence. Patients with PRO-defined active disease were more likely to report remission if they were not using prednisone and had high health confidence. CONCLUSION Discordance exists between how remission is defined by patients, PRO measures, and PGA.

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OBJECTIVES Demonstrate psychometric sensitivity analyses for testing the stability of study findings to assumptions made about patient-reported outcome measures (PROMs). STUDY DESIGN AND SETTING We performed secondary analyses of Disability of Arm, Shoulder, Hand (DASH) data collected within the PROSPER clinical trial, which compared upper limb function scores in women who had undergone breast cancer surgery, randomized to either an exercise program or usual care. We repeated the principal trial analyses after grouping DASH items into subscales suggested by factorial analyses in this dataset, and applied item response theory (IRT) to account for unequal item weighting. We checked for measurement invariance by participant age and response shift bias using established techniques. RESULTS Our analyses suggested that the DASH measured two constructs: motor function and sensory symptoms. The majority of the 6-month difference in DASH score was driven by motor function. With IRT scoring, we found differences in both constructs at 12 months (p = 0.019 and p = 0.007), but in neither construct at 6 months, contrary to the original trial results. We found no differential item function by age or between baseline and 12-month measurements. CONCLUSIONS Psychometric sensitivity analyses aid in the interpretation of the PROSPER trial's results.

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Cardiovascular research frequently involves comparing patient-reported outcomes across groups. These groups can include individuals from different countries or that have different cardiovascular conditions, and it is frequently assumed that their understanding of the assessed outcome is similar. However, to ascertain that this is indeed the case, measurement invariance needs to be evaluated. This psychometric property helps us understand whether a test measures the same underlying construct in the same way across different groups. In the absence of measurement invariance, conclusions regarding group comparisons of the construct at hand may be inappropriate. This methods corner paper provides an overview of measurement invariance as well as an example of how it can be evaluated.

Purpose The response shift effect occurs in Patient-reported outcome measures (PROMs) as a consequence of internal changes in perception of the measured concept. The purpose of this study was to assess the potential presence of the response shift effect within the French version of the Brief Resilience Scale (BRS) in a population of university students in the Lorraine region of France. Methods To measure resilience, the French version of the BRS was used. To examine the potential presence of the response shift effect within this questionnaire, the Oort’s procedure and Rasch Measurement Theory based ROSALI-RMT algorithm was used. Results The BRS demonstrated longitudinal measurement invariance across Time 0 and Time 1, as assessed using the ROSALI algorithm. A slight but significant increase in resilience was observed over time (time effect = 0.22, p < 0.0001), with no evidence of response shift effects, suggesting that the observed changes in resilience were genuine and not influenced by recalibration. Conclusion These findings support the validity of the BRS in measuring resilience over time and highlight its utility in capturing genuine changes in resilience.

Introduction Measuring patient reported outcomes can improve the quality and effectiveness of healthcare interventions. The aim of this study was to identify the final set of items that can be included in a patient-reported outcome measure to assess recovery of patients following percutaneous coronary interventions. Methods A consecutive sample of 200 patients registered in the Victorian Cardiac Outcomes Registry participated in a telephone survey 30 days following their percutaneous cardiac procedure. Rasch analysis was used to select the best set of items to form a concise and psychometrically sound patient-reported outcome measure. Key measurement properties assessed included overall fit to the Rasch measurement model, unidimensionality, response formats (thresholds), targeting, internal consistency and measurement invariance. Results Five items were identified as being reliable and valid measures of patient-reported outcomes: pain or discomfort, shortness of breath, confidence in performing usual activities, feeling unhappy and having trouble sleeping. Data showed overall fit to a Rasch model of expected item functioning (χ2 16.99; p = 0.07) and all items demonstrated unidimensionality (t-test less than 0.05 threshold value). Internal consistency was acceptable (equivalent Cronbach’s α 0.65) given there are only five items, but there was a ceiling effect (mean logit score -1.24) with compromised score precision for patients with better recovery. Conclusions We identified a succinct set of items that can be used in a patient-reported outcome measure following percutaneous coronary interventions. This patient-report outcome measure has good structural validity and acceptable internal consistency. While further psychometric evaluations are recommended, the items identified capture the patient’s perspective of their recovery following a percutaneous coronary intervention.

Abstract Study Objectives To develop a patient-reported outcome measure to assess sleep amongst people experiencing problems with alcohol or other drugs. Methods Item development included secondary analyses of qualitative interviews with drug or alcohol users in residential treatment, a review of validated sleep measures, focus groups with drug or alcohol users in residential treatment, and feedback from drug or alcohol users recruited from community and residential settings. An initial version of the measure was completed by 549 current and former drug or alcohol users (442 in person and 107 online). Analyses comprised classical test theory methods, exploratory and confirmatory factor analysis, measurement invariance assessment, and item response theory (IRT). Results The initial measure (30 items) had good content and face validity and was named the Substance Use Sleep Scale (SUSS) by addiction service users. After seven items were removed due to low item-factor loadings, two factors were retained and labeled: “Mind and Body Sleep Problems” (14 items) and “Substance-Related Sleep Problems” (nine items). Measurement invariance was confirmed with respect to gender, age, and administration format. IRT (information) and classical test theory (internal consistency and stability) indicated measure reliability. Standard parametric and nonparametric techniques supported convergent and discriminant validity. Conclusions SUSS is an easy-to-complete patient–reported outcome measure of sleep for people with drug or alcohol problems. It can be used by those concerned about their own sleep, and by treatment providers and researchers seeking to better understand, assess, and potentially treat sleep difficulties amongst this population. Further validity testing with larger and more diverse samples is now required.

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AIMS To identify and assess the psychometric properties of patient-reported outcome measures (PROMs) of self-management for chronic heart failure (CHF) patients. METHODS AND RESULTS 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). CONCLUSION 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. REGISTRATION PROSPERO CRD42022322290.

Objective Validated patient-reported outcome measures for patients with hip-abductor tendon pathology are lacking. Recent studies indicate that the Copenhagen Hip and Groin Outcome Score (HAGOS), in the original or the revised scoring format, and the Oxford Hip Score (OHS) may be relevant for use in patients with hip-abductor tendon pathology. The objective of this study was to assess the validity of the Danish version of the HAGOS, the revised HAGOS, and the OHS psychometrically in patients with hip-abductor tendon pathology. This involved the six subscales in the original and the revised version of HAGOS, the standard one-factor scoring of the OHS and the two-factor scoring of the OHS. Methods We included individual responses to HAGOS and OHS from 210 patients with hip-abductor tendon pathology (17 men; 193 women; median age 56 years, range 18–91, IQR 48–65). Overall fit, model fit, individual item fit and measurement invariance were examined using confirmatory factor analysis (CFA) and item response theory. Results CFA rejected psychometric validity of three original HAGOS subscales (symptoms, pain, physical function in sport and recreation) (p<0.0001) and the OHS (p<0.0001), whereas the revised scoring format for HAGOS subscales fit adequately. Results indicate a notable discrepancy between observed and expected scores on OHS item 10 in the patient group. Conclusion The original HAGOS and OHS are not psychometrically valid, but the revised HAGOS scoring format is an appropriate PROM for patients with hip-abductor tendon pathology. The OHS would need slight adjustment before being used in patients with hip-abductor tendon pathology.

No abstract available

BackgroundItem response theory (IRT) has been increasingly applied to patient-reported outcome (PRO) measures. The purpose of this study is to apply IRT to examine item properties (discrimination and severity of depressive symptoms), measurement precision and score comparability across five depression measures, which is the first study of its kind in the Chinese context.MethodsA clinical sample of 207 Hong Kong Chinese outpatients was recruited. Data analyses were performed including classical item analysis, IRT concurrent calibration and IRT true score equating. The IRT assumptions of unidimensionality and local independence were tested respectively using confirmatory factor analysis and chi-square statistics. The IRT linking assumptions of construct similarity, equity and subgroup invariance were also tested. The graded response model was applied to concurrently calibrate all five depression measures in a single IRT run, resulting in the item parameter estimates of these measures being placed onto a single common metric. IRT true score equating was implemented to perform the outcome score linking and construct score concordances so as to link scores from one measure to corresponding scores on another measure for direct comparability.ResultsFindings suggested that (a) symptoms on depressed mood, suicidality and feeling of worthlessness served as the strongest discriminating indicators, and symptoms concerning suicidality, changes in appetite, depressed mood, feeling of worthlessness and psychomotor agitation or retardation reflected high levels of severity in the clinical sample. (b) The five depression measures contributed to various degrees of measurement precision at varied levels of depression. (c) After outcome score linking was performed across the five measures, the cut-off scores led to either consistent or discrepant diagnoses for depression.ConclusionsThe study provides additional evidence regarding the psychometric properties and clinical utility of the five depression measures, offers methodological contributions to the appropriate use of IRT in PRO measures, and helps elucidate cultural variation in depressive symptomatology. The approach of concurrently calibrating and linking multiple PRO measures can be applied to the assessment of PROs other than the depression context.

BACKGROUND The Disabilities of the Arm, Shoulder, and Hand (DASH) questionnaire is one of the most widely used patient-reported outcome measures (PROMs) in upper extremity rehabilitation. Previous reviews have focused on specific diagnoses or shortened versions, with methodological limitations. PURPOSE To comprehensively evaluate the measurement properties of the DASH questionnaire across upper extremity conditions using COSMIN guidelines. STUDY DESIGN Systematic review of measurement properties. METHODS Following COSMIN guidelines, we systematically evaluated content validity, structural validity, internal consistency, cross-cultural validity, measurement invariance, reliability, measurement error, criterion validity, construct validity, and responsiveness. Risk of bias was assessed, and the quality of evidence was graded. The review protocol was prospectively registered in PROSPERO (CRD42024599669). RESULTS A total of 146 studies were included. Five diagnostic categories with sufficient evidence were analyzed separately: upper extremity disorders, shoulder disorders, carpal tunnel syndrome, rheumatoid arthritis, and distal radius fractures. Content validity demonstrated moderate-to-high evidence across most populations. Structural validity findings were heterogeneous, with many studies lacking adequate factor analysis or sample size justification. Internal consistency and reliability showed strong evidence across disorders. Cross-cultural validity and measurement invariance were insufficiently evaluated, particularly outside the original language context. Evidence for construct validity and responsiveness was generally moderate but limited in some populations. CONCLUSION The DASH demonstrates overall strong reliability and acceptable validity across upper extremity conditions, though structural and cross-cultural validity require further investigation. These findings provide guidance for hand therapy clinicians and researchers regarding appropriate interpretation and application of the DASH in clinical practice and research.

OBJECTIVE To examine the clinical validity of the Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Sleep Disturbance (SD) and Sleep-Related Impairment (SRI) short forms. METHODS Youth (8-17 years) from clinical populations with known SDs (sleep clinic n = 126, autism n = 276, asthma n = 82, asthma + eczema n = 68) and the general population (n = 902) completed the PROMIS Pediatric SD and SRI 8-item short forms, along with established measures of sleep (Children's Report of Sleep Patterns, Sleep Habits Survey), PROMIS Pediatric Fatigue, and parent-reported clinical indicators (does child have sleep problem, use melatonin, use prescription sleep medication). RESULTS Confirmatory factor analyses demonstrated factorial invariance for all clinical groups. Significant differences between the general population and clinical groups were found for SD and SRI (medium to large effect sizes). Convergent validity was demonstrated through separate hierarchical regression models that showed significant associations between parent-reported clinical indicators and SD and SRI, above and beyond clinical group, as well as moderate to strong correlations between the PROMIS sleep measures and both established measures of sleep and fatigue. CONCLUSIONS The PROMIS Pediatric SD and SRI short forms provide clinicians and researchers a brief, accurate, and valid way to measure patient-reported sleep outcomes in pediatric populations.

Background and purpose Patient-reported outcome measures need to reflect true differences rather than measurement artifacts resulting from translations when scores are compared across countries. We evaluated the measurement invariance of the English, Dutch, Danish, and French Oxford Hip Score (OHS) and Oxford Knee Score (OKS) in patients undergoing hip and knee arthroplasty. Methods OHS and OKS responses from patients undergoing primary hip or knee arthroplasty for osteoarthritis between 2019 and 2022 were included from national and single-center registries in the United Kingdom, the Netherlands, Denmark, and Switzerland. We evaluated unidimensionality, monotonicity (Hi > 0.3) and local independence (Yen Q3 > 0.20 above the average indicating independence) in each language version separately. Model fit was considered satisfactory when Comparative Fit Index (CFI) and Tucker–Lewis Index (TLI) were ≥ 0.95, root mean square error of approximation (RMSEA) ≤ 0.06, and standardized root mean square residual (SRMR) ≤ 0.08. Measurement invariance across languages was analyzed with multiple group—confirmatory factor analysis on full, random, and matched sample datasets. Results Across the hip and knee cohorts, unidimensionality was acceptable in the English (n = 21,108 and 28,230), Dutch (n = 36,792 and 29,651), Danish (n = 815 and 1,015), and French (n = 590 and 459) versions. RMSEA ranged from 0.072 to 0.092 for OHS, and from 0.057 to 0.061 for OKS and other fit indices were acceptable. The multiple group-confirmatory factor analysis showed acceptable measurement invariance across languages for both scores, with changes in RMSEA < 0.15 and CFI < 0.1 across gradually more constrained models. Conclusion Measurement invariance of the OHS and OKS was supported across the English, Dutch, Danish, and French versions, with multiple group-confirmatory factor analyses model fit within predefined acceptable limits, supporting cross-comparison of the different language versions of these instruments.

BACKGROUND Validated patient-reported outcome measures are needed to assess personal recovery alongside symptom-based outcomes in mental health care. The Questionnaire about the Process of Recovery (QPR) assesses subjective recovery aligned with CHIME processes, but no version of the QPR has previously been validated in Danish, including the widely used QPR-15 and the more recently developed QPR-7. This study assessed psychometric properties, sensitivity to change, and meaningful difference thresholds for the Danish QPR in community mental health samples. METHODS Pooled analyses were conducted using baseline (n = 353) and follow-up (n = 234) data from adults with mental illness in Danish community mental health services. Confirmatory factor analysis, measurement invariance testing across subgroups, content validity, internal consistency, convergent/discriminant validity, responsiveness analyses were applied, as well as anchor-based methods (CGI-S, MANSA) to establish meaningful difference thresholds. RESULTS QPR-15 showed poor unidimensional fit. QPR-7 demonstrated satisfactory fit (CFI = 0.96, TLI = 0.95, SRMR = 0.039, RMSEA = 0.061), internal consistency, invariance across sex, age, education, mental disorder type, and over time. The data were normally distributed without floor/ceiling effects, and correlations supported convergent/discriminant validity. QPR-7 change was strongly associated with changes in quality of life. Meaningful difference values: 2.5 (small)/3.5 (moderate) points within-person; 1.5(small)/2.5 (moderate) points between-group. CONCLUSION The Danish QPR-7 is a valid and reliable brief measure for patient-centered recovery assessment in mental health care. Its invariance properties, interpretability thresholds, and low respondent burden support its usefulness in routine monitoring of individual recovery gains alongside clinical outcomes.

OBJECTIVES In economic evaluations, quality of life is measured using patient-reported outcome measures (PROMs), such as the EQ-5D-5L. A key assumption for the validity of PROMs data is measurement invariance, which requires that PROM items and response options are interpreted the same across respondents. If measurement invariance is violated, PROMs exhibit differential item functioning (DIF), whereby individuals from different groups with the same underlying health respond differently, potentially biasing scores. One important group of healthcare consumers who have been shown to have different views or priorities over health is older adults. This study investigates age-related DIF in the EQ-5D-5L using item response theory (IRT) and ordinal logistic regression approaches. METHODS Multiple-group IRT models were used to investigate DIF, by assessing whether older adults aged 65+ years and younger adults aged 18 to 64 years with the same underlying health had different IRT parameter estimates and expected item and EQ-5D-5L level sum scores. Ordinal logistic regression was also used to examine whether DIF resulted in meaningful differences in expected EQ level sum scores. Effect sizes examined whether DIF indicated meaningful score differences. RESULTS The anxiety/depression item exhibited meaningful DIF in both approaches, with older adults less likely to report problems. Pain/discomfort and mobility exhibited DIF to a lesser extent. CONCLUSIONS When using the EQ-5D-5L to evaluate interventions and make resource allocation decisions, scoring bias due to DIF should be controlled for to prevent inefficient service provision, where the most cost-effective services are not provided, which could be detrimental to patients and the efficiency of health budgets.

PurposeTo develop a practical, efficient, and reliable pediatric global health (PGH) measure that would be useful for clinical, quality improvement, and research applications.MethodsUsing the Patient Reported Outcome Measurement Information System (PROMIS) mixed-methods approach for item bank development, we identified an item pool that was well understood by children as young as age 8 years and tested its psychometric properties in an internet panel sample of 3,635 children 8–17 years old and 1,807 parents of children 5–17 years old.ResultsThe final version of the PGH measure included 7 items assessing general, physical, mental, and social health. Four of these items had the same wording as the PROMIS adult global health measure. Internal consistency was 0.88 for the child-report form and 0.84 for the parent form; both had excellent test–retest reliability. The measures showed factor invariance across age categories. There was no differential item functioning by age, gender, race, or ethnicity. Because the measure includes the general health rating question, it is possible to estimate the PGH scale using this widely used single item.ConclusionsThe PROMIS PGH-7 measure is a brief and reliable 7-item measure of a child’s global health.

In clinical trials, the single-item-per-screen format is commonly used for electronic patient-reported outcomes (ePROs). However, participant preferences for this format over multiple items per screen have not been investigated. This study evaluated participant preferences for single-item-per-screen vs. multiple-items-per-screen ePRO formats, the effect on completion times, and the comparability of scores between formats. Participation in this randomized, crossover, observational study involved ePRO completion in both single-item-per-screen and multiple-items-per-screen formats on an electronic tablet device during two study visits. A paper-based preference questionnaire was completed at each visit. Thirty-seven adults (mean [SD] age = 49.6 [15.4] years; 51.4% female; 54.1% White) enrolled and 36 participants completed both visits. Twelve participants (33.3%) preferred the multiple-item format, 12 (33.3%) preferred the single-item format, 10 (27.8%) had no preference, and 2 (5.6%) did not notice a difference. Seventeen participants (47.2%) preferred the single-item format when participating in a clinical trial, and most (n = 20; 55.6%) believed that others would prefer this format in a clinical trial. The ePRO completion time (minutes:seconds) was longer for the single-item format than the multiple-item format (mean [SD], 6:42 [2:24] vs. 6:21 [2:22]; p = 0.1540). The ePRO scores were similar across both formats. This study provided evidence that both single-item and multiple-items-per-screen presented on an electronic tablet device are acceptable to users and that format preference may be specific to each individual. Thus, clinical programs need to consider the targeted study population, the purpose of use, and the overall trial design when designing ePRO solutions.

Improved cancer treatment outcomes have increased the demand for medical care that considers the quality of life of patients with cancer. Patient-reported outcomes (PROs) help assess the quality of life because they involve direct evaluation of the patients. Recently, electronic PROs (ePROs) have been used in clinical cancer care settings in Europe and the United States. Electronic PROs positively affected communication between patients with cancer and healthcare providers, enhanced education, optimized self-management, contributed to healthcare economics, assisted in monitoring adverse events, and improved prognosis. However, challenges such as adherence, burden on healthcare providers, lack of personalized formats, low digital literacy, and implementation costs remain. Therefore, carefully selecting the items to be recorded by ePROs in alignment with specific objectives is essential. Additionally, developing systems using lifelogs—digital records of daily activities—and creating mechanisms that automatically encourage patient behavioral changes based on the reported data are crucial. This review delineates the advantages and challenges of ePROs according to their history and proposes the prospects of ePRO.

ABSTRACT Background The associations between self-reported chronic kidney disease–associated pruritus (CKD-aP) and patient-reported outcomes (PROs) have been reported using various instruments to assess itch. Data collection via multiple CKD-aP instruments allows the evaluation of different domains and measurements of CKD-aP burden and may help tailor data capture for future research or clinical care. Methods An electronic PRO (ePRO) survey was distributed to European hemodialysis (HD) patients enrolled in the Dialysis Outcomes and Practice Patterns Study (DOPPS) in 2021–23. The DOPPS is an international cohort study that aims to investigate practice patterns and outcomes in HD patients. The ePRO survey included multiple CKD-aP instruments: Average Itch and Worst Itching Intensity Numerical Rating Scales (AI-NRS, WI-NRS) and a Kidney Disease Quality of Life (KDQOL)-36 single question. Linear and logistic regression were used to estimate adjusted associations between CKD-aP instruments and various PROs. Results This analysis included 769 patients who completed the WI-NRS from HD facilities in France, Germany, Italy, Spain, Sweden and the UK. The correlation between WI-NRS and the KDQOL-36 itch question was 0.88 overall and 0.46 among patients at least somewhat bothered by itch. Mean WI-NRS scores stratified by response to the KDQOL-36 itch question were 8.1, 6.4, 4.1 and 3.1 for extremely, very much, moderately and somewhat bothered, respectively. Patients with worse WI-NRS scores reported worse sleep quality, greater fatigue, more depressive symptoms, and lower mental and physical quality of life; these associations were similar to those observed for the KDQOL-36 itch question. Conclusion Correlation between CKD-aP instruments was high overall, but moderate among the subgroup of patients bothered by itch; differences can be partially attributed to the recall period for the KDQOL-36 (4 weeks) vs the AI- and WI-NRS (24 h). The consistent associations of these instruments with poor outcomes underscores the importance to identify and effectively treat HD patients suffering from pruritus.

With sophisticated mobile and wearable technologies available, there has been interest in leveraging these devices to help gather and analyze patient-generated health data (PGHD). This information could be used to better address health concerns, aid in treatment decision-making, and guide interventional strategies to improve outcomes. Among PGHD, electronic patient-reported outcomes, direct reports of patient experience usually collected via validated scales and questionnaires, are increasingly integrated into routine clinical practice to monitor patient status. Electronic patient-reported outcomes have been shown to improve outcomes, including symptom control, quality of life, and overall survival, in several clinical trials. Electronic patient-reported outcome collection is now being implemented across broader clinical practice settings but with limited evaluation of impact thus far. Wearable devices and mobile apps provide opportunities to collect additional PGHD, including continuous physiologic measures, and to generate algorithms with which to monitor patients with cancer and guide interventions. In this article, we discuss several topics related to PGHD and technology, including electronic patient-reported outcomes, mobile apps, and wearable devices and how their introduction into oncology care has the potential to improve the collection and use of PGHD in the future. We also highlight the challenges and future directions needed for mobile and wearable technologies to provide meaningful information that can be acted upon and thus can improve oncologic care.

Background Researchers and clinicians studying symptoms experienced by people with cancer must choose from various scales. It would be useful to know how the scores on one measure translate to another. Methods Using item response theory (IRT) with the single-group design, in which the same sample answers all measures, we produced crosswalk tables linking five 0–10 numeric rating scale (NRS) and 15 items from Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE, scored on a 1–5 scale) to the T-Score metric of six different scales from the NIH Patient reported Outcomes Measurement Information System (PROMIS®). The constructs, for which we conducted linking, include emotional distress-anxiety, emotional distress-depression, fatigue, sleep disturbance, pain intensity, and pain interference. We tested the IRT linking assumption of construct similarity between measures by comparing item content and testing unidimensionality of item sets comprising each construct. We also investigated the correlation of the measures to be linked and, by inspecting standardized mean differences, whether the linkage is invariant across age and gender subgroups. For measures that satisfied the assumptions, we conducted linking. Results In general, an NRS score of 0 corresponded to about 38.2 on the PROMIS T-Score scale (mean = 50; SD = 10); whereas an NRS score of 10 corresponded to a PROMIS T-Score of approximately 72.7. Similarly, the lowest/best score of 1 on PRO-CTCAE corresponded to 39.8 on T-score scale and the highest/worst score of 5 corresponded to 72.0. Conclusion We produced robust linking between single item symptom measures and PROMIS short forms.

Depression is a leading mental health concern across the U.S. and worldwide. There are myriad assessments to evaluate depressive symptoms, including the Edinburgh Postnatal Depression Scale (EPDS), which is widely used to evaluate women’s pre- and postnatal depression but not as prevalent at other timepoints in adulthood, limiting its utility for longitudinal research. As part of the National Institutes of Health’s (NIH) Environmental influences on Child Health Outcomes (ECHO) Research Program, the current study sought to develop a common metric so that scores on the EPDS can be converted to the standardized Patient-Reported Measurement Information System (PROMIS®) T-score metric. Drawing on data from the ECHO—Prenatal Alcohol in SIDS and Stillbirth cohort, this study used a single-group linking design, where 1,263 mothers completed the EPDS and PROMIS-Depression measures at the same time. Score linking was conducted using equipercentile and item response theory (IRT) methods. Results showed both linking methods provide robust, congruent results, and subgroup invariance held across age, race, ethnicity, education, and geographic location. The IRT-based unidimensional fixed-parameter calibration was selected due to its model simplicity, and a crosswalk table was established to convert scores from the EPDS to PROMIS T-scores. Overall, this study provides a way to aggregate data across various depression measures and timepoints, such that researchers and clinicians now have the ability to directly compare and combine EPDS data with PROMIS and other depression measures already score-linked to PROMIS.

"Legacy" patient-reported outcome measures (PROs) have been used for decades; however, they have many limitations. The National Institutes of Health-funded PRO Measurement Information System (PROMIS) was developed to be a generic, flexible, precise, and reliable tool to measure core and additional domains of physical and emotional health and social well-being. Unlike Legacy PROs, PROMIS measures can be implemented across diseases, and use a common T-score metric-based scoring system derived using item response theory. PROMIS measure scores have potential to predict scores of Legacy PROs and could be the only set of measures needed to assess PROs.

Interest in measuring patient-reported outcomes has increased dramatically in recent decades. This has simultaneously produced numerous assessment options and confusion. In the case of depressive symptoms, there are many commonly used options for measuring the same or a very similar concept. Public and professional reporting of scores can be confused by multiple scale ranges, normative levels, and clinical thresholds. A common reporting metric would have great value and can be achieved when similar instruments are administered to a single sample and then linked to each other to produce cross-walk score tables (e.g., Dorans, 2007; Kolen & Brennan, 2004). Using multiple procedures based on item response theory and equipercentile methods, we produced cross-walk tables linking 3 popular “legacy” depression instruments—the Center for Epidemiologic Studies Depression Scale (Radloff, 1977; N = 747), the Beck Depression Inventory–II (Beck, Steer, & Brown, 1996; N = 748), and the 9-item Patient Health Questionnaire (Kroenke, Spitzer, & Williams, 2001; N = 1,120)—to the depression metric of the National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS; Cella et al., 2010). The PROMIS Depression metric is centered on the U.S. general population, matching the marginal distributions of gender, age, race, and education in the 2000 U.S. census (Liu et al., 2010). The linking relationships were evaluated by resampling small subsets and estimating confidence intervals for the differences between the observed and linked PROMIS scores; in addition, PROMIS cutoff scores for depression severity were estimated to correspond with those commonly used with the legacy measures. Our results allow clinicians and researchers to retrofit existing data of 3 popular depression measures to the PROMIS Depression metric and vice versa.

PurposeDepression is a significant mental health concern. There are numerous depression questionnaires, several of which can be scored onto the Patient Reported Outcomes Measurement Information System (PROMIS®) Depression metric. This study expands the unified metric by linking depression subscales from the Adult Self-Report (ASR) and the Brief Symptom Inventory (BSI) to it.MethodsAn online sample of 2009 men who have sex with men (MSM) was recruited. Item factor analysis was used to evaluate the dimensionality of the aggregated measures and confirm the statistical assumptions for linking. Then, linking was conducted using equipercentile and item response theory (IRT) methods. Equipercentile linking considered varying degrees of post-smoothing. IRT-based linking used fixed-anchor calibration and separate calibration with Stocking-Lord linking constants.ResultsAll three scales were broadly unidimensional. This MSM sample had slightly higher average depression scores than the general population (mean = 54.4, SD = 9.6). Both linking methods provided robust, largely comparable results. Subgroup invariance held for age, race, and HIV status. Given the broad comparability across methods, the crosswalk between raw sum scores and the unified T-score metric used fixed-anchor IRT-based methods.ConclusionsPROMIS provides a unified, interpretable metric for depression reporting. The results of this study allow the depression subscales from the ASR and BSI to be rescored onto the unified metric with reasonable caution. This will benefit epidemiological projects aggregating data across various measures or time points.

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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 calibration using nationally representative samples of adults and children within countries is essential to demonstrate that all items possess expected strong measurement properties. Finally, it is important to demonstrate that the PROMIS measures are valid, reliable and responsive to change when used in an international context.IRT item banking will allow for tailoring within countries and facilitate growth and evolution of PROs through contributions from the international measurement community. A number of opportunities and challenges of international development of PROs item banks are discussed.

Researchers and clinicians wishing to assess anxiety must choose from among numerous assessment options, many of which purport to measure the same or a similar construct. A common reporting metric would have great value, and can be achieved when similar instruments are administered to a single sample and then linked to each other to produce cross-walk score tables. Using item response theory (IRT), we produced cross-walk tables linking three popular “legacy” anxiety instruments – MASQ (N = 743), GAD-7 (N = 748), and PANAS (N = 1120) – to the anxiety metric of the NIH Patient Reported Outcomes Measurement Information System (PROMIS®). The linking relationships were evaluated by resampling small subsets and estimating confidence intervals for the differences between the observed and linked PROMIS scores. Our results allow clinical researchers to retrofit existing data of three commonly-used anxiety measures to the PROMIS Anxiety metric and to compare clinical cut-off scores.

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Objectives To investigate whether a multi-item performance outcome measure, the physical performance test (PPT), can be calibrated to a common scale with patient-reported outcome measures, using the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function (PF) metric. Study Design and Setting We analyzed baseline data (N = 1,113) from the CONVINCE study, an international trial in end-stage kidney disease patients comparing high-dose hemodiafiltration with high-flux hemodialysis. Assumptions of item response theory (IRT) modelling were investigated for the combined set of the nine-item PPT and a four-item PROMIS PF short form (PROMIS-PF4a). We applied unidimensional IRT linking for calibrating the PPT to the PROMIS PF metric. Results Although some evidence for multidimensionality was found, classical test statistics (Cronbach's Alpha = 0.93), Mokken (Loevinger's H = 0.50), and bifactor analysis (explained common variance = 0.65) indicated that PPT and PROMIS-PF4a items can be used to assess a common PF construct. On the group level, the agreement between PROMIS-PF4a and linked PPT scores was stable across several subsamples. On the individual level, scores differed considerably. Conclusion We found preliminary evidence that the PPT can be linked to the PROMIS PF metric in hemodialysis patients, enabling group comparisons across patient-reported outcome and performance outcome measures. Alternative linking methods should be applied in future studies using a more comprehensive PROMIS PF item set.

There is a clear need to harmonize outcome measurement. Some authors propose to express scores as T scores to facilitate interpretation of PROM results in clinical practice. While this is a step in the right direction, there are important limitations to the acceptance of the T score metric as a common metric when T scores are based on raw sum scores of ordinal items: Such T scores of different instruments are not exactly comparable because they are not interval scaled; T scores of different measures are only on the same scale if exactly the same reference group is used; and the T sore metric cannot be maintained because it is reference population-dependent and needs to be updated regularly. These limitations can be overcome by using an item response theory (IRT)-based metric. Items from different measures can be placed on the same IRT metric to make scores comparable on an interval scale. The PROMIS initiative used IRT to develop item banks for measuring various health outcomes. Other PROMs have been linked to the PROMIS metric. Although PROMIS uses a T-score metric for practical reasons, the underlying PROMIS metric is actually an IRT metric. An IRT approach also enables further development of an item bank while preserving the underlying metric. Therefore, IRT-based metrics should be considered as common metrics for the future.

Physical function (PF) is a central patient-reported outcome (PRO) in many clinical conditions. However, the variety of existing PRO measures (PROMs) yield scores on different scales, limiting the score comparability and interpretability. To overcome this gap, the Patient-Reported Outcomes Measurement Information System (PROMIS®) established a standardized T-score metric using item response theory (IRT). As such, different PROMs measuring PF can be linked to this common metric, allowing for efficient harmonization of scores. Linking algorithms allow conversion of SF-36 PF-10 scores to the PROMIS-PF metric, but these methods have not been validated in independent clinical samples. To validate and compare two established linking methods for the translation of SF-36 PF-10 scores to the PROMIS-PF metric in clinical populations. Two previously proposed linking approaches were applied to estimate PROMIS-PF T-scores based on the SF-36 PF-10: Item-level linking, Cross-walk tables. Item-level linking, Cross-walk tables. The directly observed T-scores from the 20-item PROMIS-PF short form (PROMIS-PF20a) served as a benchmark against which the linked T-scores from the SF-36 PF-10 were compared. Results were compared to a newly estimated IRT-model based on the study’s dataset. Patients from cardiology (n = 185), rheumatology (n = 172), and psychosomatic medicine (n = 262), who completed both the PROMIS-PF20a and the SF-36 PF-10. PROMIS-PF20a, SF-36 PF-10. All linking approaches demonstrated high association with observed PROMIS-PF20a T-scores (Pearson correlation ≥ 0.84) and indicated negligible practical differences at the group level (standardized mean difference < 0.2). Two currently available linking approaches can reliably translate SF-36 PF-10 scores to standardized PROMIS-PF T-scores across different clinical samples, eliminating the need for re-estimating models in new datasets. As all linking algorithms ultimately presented highly comparable results, cross-walk tables may be preferred as the most practicable approach, allowing for score conversion without complex statistical modeling.

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No abstract available

Purpose In many research and clinical settings where patient reported outcomes (PRO) measures are used, it is often desirable to link scores across disparate measures, or use scores from one measure to describe scores on a separate measure. However, PRO measures are scored using a variety of metrics, making such comparisons difficult. In this paper, we provide an example of how to transform scores across disparate measures (the Marks Asthma Quality of Life Questionnaire (AQLQ-Marks) and the newly developed RAND-Negative Impact of Asthma on Quality of Life item bank (RAND-IAQL-Bank)) using an IRT-based linking method Methods Our sample of adults with asthma (N=2032) completed two measures of asthma-specific quality of life: the AQLQ-Marks and the RAND-IAQL-Bank. We use item response theory- (IRT) based co-calibration of the two measures to provide a linkage, or a common metric between the two. Co-calibration refers to the process of using IRT to estimate item parameters that describe the responses to the scales’ items according to a common metric, in this case a normal distribution transformed to a T-scale with a mean of 50 and a standard deviation of 10. Findings Results indicated that the scales measured highly similar constructs and were sufficiently unidimensional for IRT co-calibration. Once linked, scores from the two measures were invariant across subgroups. A crosswalk is provided that allows researchers and clinicians using AQLQ-Marks to crosswalk to the RAND-IAQL toolkit. Implications The ability to translate scores from the RAND-IAQL toolkit to other ‘legacy’ measures increases the value of the new toolkit, aids in interpretation, and hopefully will facilitate adoption by asthma researchers and clinicians. More generally, the techniques we illustrate can be applied to other newly developed or existing measures in the PRO research field to obtain crosswalks with widely used traditional, legacy instruments.

Objective: To link the Activity Measure for Post-Acute Care (AM-PAC) Applied Cognition to the Patient-Reported Outcomes Measurement Information System (PROMIS) Cognitive Function, allowing for a common metric across scales. Design: Cross-sectional survey study. Setting: Outpatient rehabilitation clinics. Participants: Consecutive sample of 500 participants (N=500) aged ≥18 years presenting for outpatient therapy (physical, occupation, speech). Interventions: Not applicable. Main Outcome Measures: AM-PAC Medicare and Generic Cognition short forms and PROMIS Cognitive Function items representing the PROMIS Cognitive Function item bank. Results: The calibration of 25 AM-PAC cognition items with 11 fixed PROMIS cognitive function item parameters using item-response theory indicated that items were measuring the same underlying construct (cognition). Both scales measured a wide range of functioning. The AM-PAC Generic Cognitive assessment showed more reliability with lower levels of cognition, whereas the PROMIS Cognitive Function full-item bank was more reliable across a larger distribution of scores. Data were appropriate for a fixed-anchor item response theory-based crosswalk and AM-PAC Cognition raw scores were mapped onto the PROMIS metric. Conclusions: The crosswalk developed in this study allows for converting scores from the AM-PAC Applied Cognition to the PROMIS Cognitive Function scale.

We scaled a measure of physical functioning to a population-based normative sample by extending self-reported basic and instrumental activities of daily living with items from the Medical Outcomes Study 12-Item Short Form Survey. We used item response theory to place items administered to a sample of older elective surgery patients on a common metric linked to the Patient Reported Outcomes Measurement Information System (PROMIS) normative sample using published data. The summary measure for physical functioning was internally consistent (Cronbach’s α = .83), reliable across a broad range of functioning, and was moderately correlated with walking speed (r = .52) and energy expenditure (r = .40). Demonstrating predictive criterion validity, less impaired scores were associated with lower risk of discharge to a rehabilitation facility (odds ratio = .38, 95% confidence interval [CI] [0.22, 0.66]) and shorter hospital stays (incidence rate ratio [IRR] = 0.87, 95% CI [0.79, 0.97]). Our approach may facilitate direct comparison of physical functioning measures across existing and future studies using a common, population-based metric, when overlapping items with the National Institutes of Health (NIH) PROMIS item bank are present.

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This study introduces a Bayesian multidimensional hierarchical item response theory (MHIRT) model to improve patient-reported outcome (PRO) assessments in total knee arthroplasty (TKA). Traditional unidimensional scoring fails to capture the multifaceted nature of recovery. Our model uncovers latent traits and inter-item relationships directly from PROMs such as the OKS and the EQ-5D-3L, without relying on predefined subscales. MHIRT flexibly decomposes PROMs into clinically meaningful traits like pain, mobility, self-care, and confidence. These traits captured more domain-specific variation, showed stronger sensitivity to temporal changes, and better reflected demographic factors than traditional total scores. The model was trained on a large NHS dataset and externally validated on PROMs from the moveUP digital platform. In predictive modeling of postoperative outcomes, MHIRT-derived features consistently outperformed unidimensional scores and conventional multidimensional IRT models. These findings suggest that MHIRT offers a potentially interpretable framework for tracking recovery and predicting health outcomes.