Journal of Patient-Reported Outcomes最新文献

{"title":"A successful model for KSA-based payor-provider collaborative in maternity care.","authors":"Abdulrehman Taher, Serena Sarkis, Hussein H Khachfe, Fares Saade, Jason Greich, Eman ElBadrawy, Dalal AlHamed","doi":"10.1186/s41687-026-01142-w","DOIUrl":"10.1186/s41687-026-01142-w","url":null,"abstract":"<p><strong>Background: </strong>Maternity care in the Kingdom of Saudi Arabia (KSA) faces rising Cesarean sections (C-section) rates, fragmented care pathways, and a lack of standardized outcome measurement. This study evaluates the feasibility and early outcomes of a large-scale payer-provider Value-Based Healthcare (VBHC) pilot integrating Patient-Reported and Clinician-Reported Outcome Measures (PROMs and CROMs) across four tertiary private hospitals with active maternity/obstetrics departments in KSA.</p><p><strong>Methodology: </strong>A multicenter outcomes rollout pilot was conducted across four tertiary hospitals with dedicated maternity/obstetrics departments in KSA between July 2024 and July 2025. The International Consortium for Health Outcomes Measurement (ICHOM) Pregnancy and Childbirth Standard Set PROMs and CROMs were digitally collected at five predefined control points using a centralized interoperable platform from pregnant women aged 18-45 years. Site Rollout Teams were trained in VBHC principles, patient journey mapping, and outcomes utilization. Response rates, longitudinal PROMs trends, minimum important difference (MID) analyses, and PROM-PROM, PROM-CROM, and CROM-CROM correlations were evaluated.</p><p><strong>Results: </strong>A total of 802 women were enrolled, with an overall PROMs response rate of 38.7%. While mean PROMs changes remained below MID thresholds at the population level, approximately one-third of women achieved clinically meaningful individual improvements across key domains, including mental health, continence, breastfeeding confidence, and bonding. Hospitals implementing PROMs-triggered referral pathways demonstrated substantial reductions in abnormal outcome rates. Meaningful correlations were observed across psychosocial, functional, and clinical outcomes, supporting internal data validity and clinical coherence. Two providers achieved ICHOM Level 1 accreditation by project completion.</p><p><strong>Conclusions: </strong>This pilot demonstrates that standardized PROMs and CROMs integration into routine maternity care is feasible and operationally effective in Saudi Arabia. Payer-provider collaboration, digital infrastructure, and clinician engagement enable actionable, patient-centered insights and establish a scalable foundation for VBHC-aligned maternity care and payment reform.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13624220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148382445","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How to prevent missing data in cost-effectiveness analyses alongside cardiovascular disease trials? A review on methods of administration of patient-reported outcomes.","authors":"Manon I Generaal, Emilien C J Wegerif, Çağdaş Ünlü, Gert J de Borst, Miriam P van der Meulen","doi":"10.1186/s41687-026-01108-y","DOIUrl":"10.1186/s41687-026-01108-y","url":null,"abstract":"<p><strong>Aim: </strong>Missing data in patient-reported outcomes (PROs) for cost-effectiveness analyses is a widespread challenge that could bias the outcome. This study aims to determine whether methods of administering PROs are associated with missing data and to identify which factors contribute to missing data.</p><p><strong>Methods: </strong>A systematic literature search was conducted using the terms \"cardiovascular disease\", \"cost\", and \"economic evaluation\". Only cost-effectiveness analyses alongside clinical trials that use PROs to assess quality of life were included. The method of administration of PROs, proportion of missing data, how missing data was handled statistically, and other study characteristics were extracted. Proportions of missing data per administration strategy were calculated.</p><p><strong>Results: </strong>In total, 123 articles were included in this review, median sample size was 441 (range 50 - 29,759). Median rate of missing data was 13% (range 0-44.2%) but was not reported in 35 (28.7%) studies. How PROs were administered was not reported in 36 (29.3%) studies. A combination of methods to administer PROs resulted in the least missing data (10.4%). Postal questionnaires alone had the most missing data (24.0%), while adding telephone reminders to fill in the questionnaire cut this nearly in half (12.6%). Most studies administered PROs during visits, resulting in 18.0% missing data. How missing data were addressed statistically was not reported in 32 (26.0%) studies.</p><p><strong>Conclusions: </strong>Reporting on administration of PROs, amount of missing data, and statistical handling of missing data was often lacking. The use of a reminder or a combination of methods to administer PROs seems the most effective approach to substantially decrease the proportion of missing data.</p><p><strong>Clinical trial number: </strong>Not applicable.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13486412/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148362762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and validation of the Patient Involvement in Medication Communication at Hospital discharge Questionnaire (PIMCH-Q).","authors":"Victoria Östman, Emma Bertilsson, Henrik Cam, Kristin Franzon, Sofia Kälvemark Sporrong, Emelie Frödén, Sham Shaat, Ulrika Gillespie","doi":"10.1186/s41687-026-01136-8","DOIUrl":"10.1186/s41687-026-01136-8","url":null,"abstract":"<p><strong>Background: </strong>Medication-related problems are common after hospital discharge, often due to inadequate communication about changes made to medication therapy. Patients' ability to understand and manage medication changes after discharge is essential for safe and effective treatment. However, existing patient-reported outcome measures (PROMs) do not adequately capture how patients perceive their involvement or understanding of peri- and post-discharge medication communication. The aim of this study was to develop the Patient Involvement in Medication Communication at Hospital discharge Questionnaire (PIMCH-Q) and to evaluate its content validity, structural validity, and internal consistency.</p><p><strong>Methods: </strong>The 8-item PIMCH-Q was developed based on literature and expert input. Content validity was assessed through expert panel review and patient interviews. Structural validity was evaluated using exploratory and confirmatory factor analyses (EFA, CFA) based on responses from recently discharged patients (n=57). Internal consistency was assessed using McDonald's omega.</p><p><strong>Results: </strong>Eleven patients participated in interviews. They found the questionnaire relevant and easy to understand. Exploratory factor analysis suggested a three-factor structure representing perceived involvement in medication decisions, knowledge and understanding of medication changes, and confidence in medication self-management. One item did not show a clear primary loading and was excluded, resulting in a seven-item factor structure. Confirmatory factor analysis demonstrated good model fit (χ²(11) = 6.58; CFI = 1.00; TLI = 1.01; RMSEA = 0.00; SRMR = 0.055). Internal consistency was high (ω total = 0.95).</p><p><strong>Conclusions: </strong>This study provides preliminary support for the validity and internal consistency of the PIMCH-Q as a PROM for assessing patient experiences of medication communication at hospital discharge. The findings also suggest that the PIMCH-Q may be useful in both research and clinical practice. Further validation in larger and independent samples is warranted.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13319526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148354025","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Crosswalk and correlation between the Oxford Knee Score (OKS), Knee Society Score (KSS) and Knee Injury and Osteoarthritis Outcome Score (KOOS-12) in knee osteoarthritis.","authors":"Shaun Kai Kiat Chua, Linus Ren Hao Tan, Chien Joo Lim, Michelle Jessica Pereira, Bryan Yijia Tan","doi":"10.1186/s41687-026-01117-x","DOIUrl":"10.1186/s41687-026-01117-x","url":null,"abstract":"<p><strong>Background: </strong>Significant heterogeneity exists in the patient-reported outcome measures (PROMs) used to assess outcomes in knee osteoarthritis (KOA), with limited evidence exploring the correlation and conversion among the various PROMs. Our study aims to explore the correlation and conversion between the Oxford Knee Score (OKS), Knee Injury and Osteoarthritis Outcome Score (KOOS-12) and Knee Society Score (KSS) in patients with non-operatively treated KOA.</p><p><strong>Methodology: </strong>This was a prospective cohort study of 131 patients who received non-operative treatment (standardized physical therapy and analgesia) for KOA between July 2020 and January 2024. Three validated PROMs for KOA (OKS, KOOS-12 and KSS) were measured for patients at baseline, 3-months, 6-months, 1-year and 2-years follow-up. Overall, 498 complete paired outcome scores (KSS/OKS/KOOS-12 at a single time point) were included for analysis. The data were then randomly split into a derivation (75%) and internal validation (25%) cohort. The correlation between the outcome scores were calculated, and linear regression model was used to obtain conversion equations between the measured outcomes. Internal validation of the derived conversion equation was performed subsequently by comparing the mean difference between the equation-derived and actual scores.</p><p><strong>Results: </strong>A significant strong positive correlation was found between KOOS-12 and OKS (coefficient:0.79;p < 0.001), and a significant moderate positive correlation was found between the KOOS-12 and KSS (coefficient:0.50;p < 0.001). A significant moderate positive correlation was found between the OKS and KSS (coefficient:0.67;p < 0.001). Six conversion equations derived for the measured PROMs had low mean difference between the equation-derived and actual scores (0.31-1.68). Moderate to strong correlation was found between the actual and equation-derived scores, with weakest strength observed in the conversion between KOOS-12 and KSS (coefficient:0.53) and the strongest strength observed in the conversion between KOOS-12 and OKS (coefficient:0.80).</p><p><strong>Conclusions: </strong>On internal validation, we found good correlation between the mean actual and equation-derived outcome scores despite significant within-individual variability. The conversion equations derived in this study can facilitate pooling and comparison of studies using heterogenous outcomes measures and meta-analysis to increase statistical power and address novel questions in KOA. Nonetheless, the equations should not be utilized to predict individual scores from one to another due to high within-individual variability.</p><p><strong>Clinical trial number: </strong>Not applicable.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13582726/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148346691","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing content validity of the functional assessment of cancer therapy-cutaneous T-cell lymphoma (FACT-CTCL): a qualitative study.","authors":"Caroline Raymundo, Jenny Jung Sun Park, Mengyang Di, Cecilia Larocca, David Cella, Michi M Shinohara","doi":"10.1186/s41687-026-01131-z","DOIUrl":"10.1186/s41687-026-01131-z","url":null,"abstract":"<p><strong>Background: </strong>Cutaneous T-cell lymphomas (CTCLs) are a heterogeneous group of non-Hodgkin lymphomas characterized by neoplastic T cells in the skin. Common subtypes of CTCL are mycosis fungoides (MF) and Sézary syndrome (SS). Patients with MF/SS can experience significant symptoms that affect health-related quality of life (HRQoL). Although the Functional Assessment of Cancer Therapy-Cutaneous T-Cell Lymphoma (FACT-CTCL) was developed as a disease-specific patient-reported outcome measure (PROM) for MF/SS, qualitative evaluation of its content validity in the target population has not been assessed. We therefore evaluated the content validity of the FACT-CTCL in patients with MF/SS and clinicians with expertise in MF/SS.</p><p><strong>Methods: </strong>Semi-structured cognitive interviews of patients with a diagnosis of MF or SS, as well as healthcare providers with expertise in treating MF/SS were conducted in alignment with the Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN). Patients evaluated the relevance, comprehensibility, and comprehensiveness of the FACT-CTCL. Clinicians evaluated relevance and comprehensiveness.</p><p><strong>Results: </strong>Twelve patients (9 MF, 3 SS) and eight clinician experts (3 oncologists, 4 dermatologists, 1 nurse practitioner) participated. The median age of patients was 68 years (range 36-77); six had early-stage (IA - IIA) disease and six had advanced-stage (IIB - IVB) disease. All items were deemed relevant by at least 95% of participants, and 44 of 47 items achieved 100% relevance across patients and clinicians. All items achieved at least 90% comprehensibility among patients, and 39 of 47 scored items were understood by all patients. All patients found the instructions and response options easy to understand, and all participants considered the instrument comprehensive.</p><p><strong>Conclusions: </strong>The FACT-CTCL demonstrated strong content validity in this sample of patients with MF/SS and expert clinicians. These findings support further evaluation and use of the FACT-CTCL as a disease-specific HRQoL instrument in MF/SS, while additional study in larger and more diverse populations remains warranted.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13554038/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148303408","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characterizing and quantifying disease impacts of generalized myasthenia gravis (gMG) in the United States: insights from patient and caregiver interviews and surveys.","authors":"Pushpa Narayanaswami, Jamie Sullivan, Craig Thiele, Christina Ramirez, Susan dosReis, Allison Foss, Karen S Yee, Mayvis Rebeira, Naila Wahid, Alexis C Garduno, Elizabeth Crevier, Christine Rowe, Kelly G Gwathmey","doi":"10.1186/s41687-026-01124-y","DOIUrl":"10.1186/s41687-026-01124-y","url":null,"abstract":"<p><strong>Background: </strong>Treatment value is traditionally assessed through clinical benefits, health-related quality of life, and healthcare utilization. However, rare diseases such as generalized myasthenia gravis (gMG) that heavily affect families and/or caregivers require a more holistic approach that centers around impacted individuals across all areas of daily life. Here, key domains impacted by gMG were identified through individual interviews with both patients and caregivers and these impacts were further assessed through a survey in an independent sample.</p><p><strong>Methods: </strong>Interviews with patients with gMG and caregivers identified 25 patient-centered \"impact elements\" across 8 life domains (occupation, financial, emotional, physical, sleep, social, planning and autonomy, and safety). Independently recruited patients and caregivers who completed a subsequent electronic, quantitative survey rated the relevance of impact elements on a scale of 1 (lowest) to 5 (highest).</p><p><strong>Results: </strong>Respondents included 239 patients with gMG (63%, aged 18-49 y; 69%, female) and 81 caregivers (58%, aged 18-49 y; 42% female). The 5 domains most impacted by gMG were occupational (mean domain score [SD]: 4.0 [1.2]), planning and autonomy (4.0 [1.1]), financial (3.9 [1.2]), physical (3.9 [1.1]), and sleep (3.9 [1.2]) for patients and financial (3.7 [1.2]), sleep (3.7 [1.1]), occupational (3.6 [1.2]), planning and autonomy (3.6 [1.1]), and safety (3.6 [1.1]) for caregivers. Numerically higher impact scores were observed among patients with more severe disease, younger patients, and caregivers who were unemployed, retired, or disabled.</p><p><strong>Conclusions: </strong>These findings provide additional insight into the comprehensive burden of gMG that extends beyond symptoms and medical costs.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13400517/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148309717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric analysis of new lymphoma-specific patient-reported symptom measures derived from the EORTC item library.","authors":"Angély Loubert, Kristin Creel, Naleen Raj Bhandari, Lisa M Hess, Amy S Ruppert, Paolo Abada, Antoine Regnault, Nalin Payakachat","doi":"10.1186/s41687-026-01126-w","DOIUrl":"10.1186/s41687-026-01126-w","url":null,"abstract":"<p><strong>Introduction: </strong>Three patient-reported outcome (PRO) measures specific to lymphoma symptoms were previously defined using mixed-methods research, comprising questions from the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 items (QLQ-C30) and Item Library (IL): measures of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) Symptoms, Mantle Cell Lymphoma (MCL) Symptoms, and an Expanded Fatigue measure. The objective of this study was to assess psychometric properties of the measures and provide guidance for interpretation by identifying thresholds for determining meaningful within-patient change (MWPC).</p><p><strong>Methods: </strong>Psychometric properties of the CLL/SLL-related Symptoms, MCL-related Symptoms, and Expanded Fatigue measures were assessed using classical test theory (CTT) and Rasch measurement theory (RMT) using blinded data collected from two phase 3 trials in participants with CLL/SLL (BRUIN-CLL-321, N = 211) and MCL (BRUIN-MCL-321, N = 523). CTT analyses assessed reliability, construct validity and ability to detect change, while RMT analyses explored adequacy of summing items to generate summary scores. MWPC for each score was estimated using anchor-based and distribution-based methods.</p><p><strong>Results: </strong>The three measures demonstrated adequate internal consistency and test-retest reliability, and strong evidence of construct validity. Each showed a good ability to detect worsening and, to a lesser degree, improvement. RMT analyses confirmed adequacy of the response scales but suggested suboptimal targeting for participants with the lowest symptoms severity. For the two symptoms-related measures, items related to fatigue were over-discriminating, suggesting that they are driving the measures. Analyses of MWPC resulted in the following estimates: for the CLL/SLL-related Symptoms score, an improvement of -15.38 points (worsening unable to be estimated); for the MCL-related Symptoms score, an improvement of -10.25 points and a worsening of 5.12 points; and for the Expanded Fatigue score, an improvement -16.66 points in both CLL/SLL and MCL populations and a worsening of 11.11 points in participants with MCL (unable to be estimated in CLL/SLL).</p><p><strong>Conclusion: </strong>The CLL/SLL-related Symptoms, MCL-related Symptoms, and Expanded Fatigue measures showed strong psychometric properties in both CTT and RMT frameworks. Combined with previously published qualitative data, this research showed that the measures are appropriate for use in clinical trials of participants with CLL/SLL and MCL.</p><p><strong>Trial registrations: </strong>BRUIN-CLL-321, NCT04666038. Registered 07 December 2020, https://www.</p><p><strong>Clinicaltrials: </strong>gov/study/NCT04666038 ; BRUIN-MCL-321, NCT04662255, Registered 04 December 2020, https://clinicaltrials.gov/study/NCT04662255 .</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13328598/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148296678","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Translation and initial psychometric evaluation of the Danish version of the treatment expectation questionnaire (TEX-Q).","authors":"Josephine Zachodnik, Louise Linding, Kasper Højgaard Thybo, Anja Geisler, Magnus Sandberg","doi":"10.1186/s41687-026-01121-1","DOIUrl":"10.1186/s41687-026-01121-1","url":null,"abstract":"<p><strong>Background: </strong>Patient expectations are important psychological determinants of health outcomes. The Treatment Expectation Questionnaire (TEX-Q) is a validated, multidimensional instrument for assessing treatment expectations; however, no Danish version has previously been available. This study aimed to translate and conduct an initial psychometric evaluation of a Danish version of the TEX-Q in patients undergoing spinal surgery.</p><p><strong>Methods: </strong>The TEX-Q was translated using a structured forward-backward procedure in accordance with ISPOR guidelines, including expert review by clinicians from different professional backgrounds. Consecutive adult patients scheduled for spinal surgery at a university hospital were invited to participate. The initial psychometric evaluation followed established PROM reporting standards and included assessment of feasibility, internal consistency, test-retest reliability, floor and ceiling effects, and construct validity.</p><p><strong>Results: </strong>The Danish TEX-Q demonstrated high comprehensibility and linguistic clarity, with mean item ratings ranging from 5.4 to 5.8 on a six-point Likert scale. Reliability varied across domains, with good internal consistency and test-retest reliability observed for most domains. Floor effects were negligible, while ceiling effects exceeded 15% in selected domains. Construct validity analyses showed no association between the TEX-Q total score and functional disability, while only limited domain-specific associations were observed, and a small but statistically significant positive association with quality of life.</p><p><strong>Conclusion: </strong>The Danish version of TEX-Q was successfully translated and demonstrated promising initial psychometric properties in a population of patients undergoing spinal surgery. However, variability in reliability across domains warrants cautious interpretation of domain-level results. Further studies are needed to assess structural validity and confirm the psychometric properties of the instrument in larger and more diverse clinical populations.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13287362/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148296197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and pilot testing of the quality of life of parents of children with achondroplasia questionnaire.","authors":"Adekunle Adedeji, Stefanie Witt, Maria Salomão, Tabea Steinhoff, Florian Innig, Inês Alves, Chiara Provasi, Marco Sessa, Klaus Mohnike, Julia Quitmann","doi":"10.1186/s41687-026-01127-9","DOIUrl":"10.1186/s41687-026-01127-9","url":null,"abstract":"<p><strong>Background: </strong>Parents of children with achondroplasia face sustained caregiving demands that may affect multiple dimensions of well-being. Despite growing recognition of these challenges, no validated, condition-specific instrument exists to assess the quality of life (QoL) of parents of children with achondroplasia. This study aimed to develop, and pilot test the Quality of Life of Parents of Children with Achondroplasia (QOLA) questionnaire.</p><p><strong>Methods: </strong>QOLA was developed using a multi-phase mixed-methods design in accordance with established standards for developing self-reported outcome measures for caregivers and parents. Phase 1 comprised semi-structured qualitative interviews with 17 parents of children with achondroplasia to identify relevant QoL domains and language. Interview data were analysed using qualitative content analysis and informed systematic item generation (Phase 2). Conceptual structure was examined through researcher-led card sorting (Phase 3) and two rounds of international card sorting following translation (Phase 4). The resulting 63-item questionnaire across eight domains was pilot-tested in a cross-sectional, multi-country study with embedded cognitive debriefing in Germany, Italy, and Portugal (total N = 50).</p><p><strong>Results: </strong>The final pilot version of QOLA comprised 63 items across eight domains covering healthcare experiences, challenges and support, physical health, mental health, social life and relationships, coping, family and daily life, and worries and future concerns. Item-level missing data were minimal, and no pronounced floor or ceiling effects were observed. Internal consistency was acceptable to good for domains (α = 0.624-0.821) and good for the total scale (α = 0.798). Inter-domain correlations were generally moderate to strong. Cognitive debriefing was highly acceptable and relevant across countries, with some suggestions for further refinement.</p><p><strong>Conclusions: </strong>QOLA shows strong preliminary evidence of acceptability and internal consistency and addresses a key measurement gap in achondroplasia research. Further large-scale psychometric validation is warranted.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13282410/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148279154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Measurement properties of the Activities-specific Balance Confidence (ABC) scale in community-dwelling adults aged 50 years and older: a COSMIN systematic review.","authors":"Hadi Kooshiar, Dan Song, Elissa Dabkowski, Karen Missen","doi":"10.1186/s41687-026-01119-9","DOIUrl":"10.1186/s41687-026-01119-9","url":null,"abstract":"<p><strong>Aim: </strong>To systematically review and grade the measurement properties of the Activities-specific Balance Confidence (ABC) scale in community-dwelling older adults using the COSMIN framework.</p><p><strong>Methods: </strong>Five electronic databases were searched through July 2025 from inception. Eligible studies included adults aged 50 years or older and examined the ABC scale in any version or translation. Methodological quality was evaluated using the COSMIN Risk of Bias checklist, and the certainty of the evidence was graded with the COSMIN-adapted GRADE approach. The protocol was registered with PROSPERO (ID: CRD420251178952).</p><p><strong>Results: </strong>A total of 22 studies were included, covering the ABC-16 (n = 16), ABC-6 (n = 5), and ABC-S (n = 1) in 11 languages. Internal consistency (α = 0.86-0.99) and test-retest reliability (ICC = 0.82-0.99) were consistently sufficient with moderate certainty. Structural and construct validity were generally supported. In contrast, content validity was primarily assessed during cross-cultural adaptations, measurement error was seldom assessed, and cross-cultural validity and responsiveness were rarely tested. The Arabic ABC-16 showed the strongest overall evidence and was categorized as ready for use (Category A). Versions in Chinese-Canadian, Cantonese, Mandarin ABC-6, Brazilian Portuguese, Persian, German, Urdu, Sepedi, Chinese, English (Canadian), and Hindi demonstrated promising properties but require local validation (Category B). Dutch and Mandarin ABC-16 versions as well as the earlier Portuguese version did not meet current COSMIN standards and require re-validation (Category C).</p><p><strong>Conclusion: </strong>The ABC scale generally demonstrates strong reliability and validity of balance confidence in older adults. The ABC-16 remains the most comprehensive version, while the ABC-6 provides an efficient screening tool. Category A versions can be considered for use in clinical and research settings. Future work should focus on responsiveness, cross-cultural invariance, and interpretability to strengthen clinical utility.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13534378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148279176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}