Quality of Life Research最新文献

{"title":"Longitudinal impact of sodium bicarbonate therapy on health-related quality of life in pre-dialysis chronic kidney disease with metabolic acidosis: a pragmatic trial in Malaysia.","authors":"Jaime Yoke May Chan, Yi Jing Tan, Farida Islahudin, Clare Hui Hong Tan","doi":"10.1007/s11136-026-04404-8","DOIUrl":"10.1007/s11136-026-04404-8","url":null,"abstract":"<p><strong>Purpose: </strong>Metabolic acidosis (MA) is a complication of chronic kidney disease (CKD) and is associated with adverse clinical outcomes. This study evaluated longitudinal changes in health-related quality of life (HRQoL) among pre-dialysis CKD patients with MA following initiation of sodium bicarbonate therapy.</p><p><strong>Methods: </strong>In this 18-month, single-centre, open-label, pragmatic trial, 74 adults with stage 3-4 CKD and MA (serum bicarbonate < 22 mmol/L) were allocated in a 1:1:1 ratio to receive oral sodium bicarbonate (tablets or solution) or standard care (control). HRQoL was assessed using the KDQOL-36 and EQ-VAS instruments. Longitudinal HRQoL trajectories and the effects of treatment and disease progression were evaluated using multivariable linear mixed-effects models.</p><p><strong>Results: </strong>Baseline physical health was impaired across the cohort, with the Burden subscale scoring the lowest. Sodium bicarbonate increased serum bicarbonate levels during early titration, although only one-third of treated patients achieved the target range of 22-26 mmol/L. Neither sodium bicarbonate formulation significantly improved KDQOL-36 subscale or EQ-VAS scores compared with control. However, worsening CKD stage was significantly associated with lower HRQoL, with clinically meaningful declines observed in the KDQOL-36 Effects and Burden subscales. No significant differences were observed between treatment arms in blood pressure, body weight, or all-cause hospitalisations. Gastrointestinal symptoms and peripheral oedema were common among treated patients but were not significantly associated with HRQoL.</p><p><strong>Conclusion: </strong>Sodium bicarbonate therapy did not improve HRQoL in pre-dialysis CKD patients with MA. Patient-reported well-being appeared to be driven by CKD progression, highlighting the practical challenges and limitations of treat-to-target alkali therapy in routine clinical practice.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149070073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Establishing meaningful score differences for the observer-reported communication ability (ORCA) measure using the scale judgment approach with caregivers of individuals with Angelman syndrome.","authors":"Abigail Rader, Li Lin, Nicole Lucas, Kevin Weinfurt, Jennifer Panagoulias, Allyson Berent, Heather A King, Harrison N Jones, Alexa Namestnik, Kelly L Gordon, Bryce B Reeve","doi":"10.1007/s11136-026-04407-5","DOIUrl":"10.1007/s11136-026-04407-5","url":null,"abstract":"<p><strong>Purpose: </strong>The Observer-Reported Communication Ability (ORCA) measure assesses communication ability in individuals living with Angelman syndrome (AS). As gene therapies progress through clinical trials, we need to identify what within-person changes in communication are meaningful to families. This study used the Scale Judgment approach to estimate meaningful score differences (MSDs) on the ORCA measure for AS.</p><p><strong>Methods: </strong>Caregivers (n = 156) of individuals living with AS completed Scale Judgment tasks showing vignette pairs depicting an individual's communication ability before and after a hypothetical, unspecified treatment. Pairs represented four contexts (lower, higher, expressive, and receptive communication) and score difference magnitudes (2, 4, 6, 8 T-score units). We used an item response theory (IRT) model to estimate a threshold parameter for each vignette pair, then fit a quadratic regression of the parameters on the T-score differences to identify the MSD. ORCA T-scores are calibrated to a mean of 50 and standard deviation of 10 in the AS reference sample.</p><p><strong>Results: </strong>Across all contexts, the estimated MSD was 1.80 T-score units (95% CI, 1.11-2.50). Estimates were larger for the lower communication range than higher (2.21 vs. 1.37) and similar for expressive (1.57) and receptive (1.58) communication. When stratified by age, genotype, and baseline score, MSDs varied from 0.82 to 2.58, though confidence intervals overlapped across all comparisons. In exploratory analysis of hypothetical treatment caregivers imagined during the task, estimates were largest among caregivers who reported imagining gene therapy (3.41).</p><p><strong>Conclusion: </strong>Caregivers consistently endorsed small score changes as meaningful, suggesting that small treatment effects carry real significance for families. Accounting for variability across contexts and subgroups, we recommend an MSD of 2.0 to 3.5 T-score points on the ORCA measure for individuals with AS, bounded below by the smallest score difference presented to caregivers during the scale judgment task and above by the largest subgroup estimate.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149070077","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adaptation and initial psychometric validation of the Latvian version of EORTC QLQ-C30 and QLQ-BR45 in women with breast cancer.","authors":"Ingrida Trups-Kalne, Laura Nesaule, Lauma Springe, Lubova Tihomirova, Vita Steina, Viktorija Pacukevica-Stope, Laura Rotberga, Jelena Maksimenko, Arvids Irmejs, Elita Poplavska","doi":"10.1007/s11136-026-04394-7","DOIUrl":"10.1007/s11136-026-04394-7","url":null,"abstract":"<p><strong>Purpose: </strong>To culturally adapt and initially evaluate the psychometric properties of the Latvian versions of the EORTC QLQ-C30 and breast cancer-specific EORTC QLQ-BR45 in women with breast cancer.</p><p><strong>Methods: </strong>The study was conducted within a pilot project implementing the ICHOM Breast Cancer Standard Set in a specialised university hospital in Latvia. Translation and cultural adaptation followed the EORTC procedure and included expert review and cognitive debriefing. Psychometric evaluation used baseline data from 142 women and six-month follow-up data from 47. Analyses included item-level indicators, internal consistency, multitrait scaling, inter-scale correlations, correlations with the Stress Symptom Scale, and paired change analyses. Conditional QLQ-BR45 items were treated as structurally missing when not applicable.</p><p><strong>Results: </strong>Expert review and cognitive debriefing supported linguistic, conceptual, and cultural appropriateness. Reliability and item-scale correspondence were acceptable for several domains, with particularly strong multitrait scaling results for QLQ-BR45 Body Image, Sexual Functioning, and Breast Symptoms. Correlations between EORTC functioning and symptom domains and the Stress Symptom Scale were generally in the hypothesised directions. Six-month findings showed clinically plausible changes in functioning and treatment-related symptoms, but estimates were based on a smaller follow-up sample and reduced valid samples for some conditional scales.</p><p><strong>Conclusion: </strong>The Latvian EORTC QLQ-C30 and QLQ-BR45 showed acceptable preliminary psychometric performance. The findings provide initial support for their use to assess HRQoL and breast cancer-specific symptoms in Latvia, while larger multicentre and longitudinal studies are needed to evaluate additional measurement properties and responsiveness.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13614982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149064257","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Determining what matters most to patients: qualitative interview techniques and methodological considerations for concept saliency analysis and reporting.","authors":"Chloe Carmichael, Stephanie Fairhurst, Harriet Makin, Catherine Bottomley, Natalie Aldhouse","doi":"10.1007/s11136-026-04395-6","DOIUrl":"10.1007/s11136-026-04395-6","url":null,"abstract":"<p><strong>Purpose: </strong>This commentary considers how qualitative interview techniques can be used to determine the relative importance or saliency of concepts reported by patients in clinical outcome assessment (COA) development. The objective of this commentary is to explore how researchers can identify which symptoms and impacts are most important to patients using qualitative methods. The paper aims to promote alignment on best practices and terminology.</p><p><strong>Methods: </strong>This commentary discusses the implementation of structured qualitative interview tasks that are commonly used to assess concept saliency, including the definition and application of thresholds. These methods are discussed in the context of concept elicitation interviews, with considerations for their design, implementation, and analysis.</p><p><strong>Results: </strong>Ranking and rating tasks offer structured approaches to eliciting concept importance, each with distinct advantages and limitations. Ranking tasks are efficient and engaging but may oversimplify concept prioritization. Rating tasks provide nuanced data but can be time-consuming and burdensome. The authors consider current use of saliency thresholds to be inconsistent, with research generally lacking predefined criteria. A mixed methods analysis approach, combining qualitative insights with descriptive statistics, is recommended by the authors to ensure comprehensive understanding of patient experience.</p><p><strong>Conclusion: </strong>Structured interview tasks enhance the rigor and relevance of qualitative research in COA development. By standardizing terminology and analysis approaches, researchers can more accurately identify and prioritize concepts that matter most to patients. This ensures that measurement strategies in clinical trials and real-world studies are truly patient-centered.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13615074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149064233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sample size calculator for reliability and validity evaluation: a Shiny R application.","authors":"Macy Glover, Xi Chen, Joseph C Cappelleri, Andrea Chadwick, Shanshan Qin, Jinxiang Hu","doi":"10.1007/s11136-026-04385-8","DOIUrl":"10.1007/s11136-026-04385-8","url":null,"abstract":"<p><strong>Objectives: </strong>The objective is to provide a user-friendly tool for researchers to compute sample sizes for studies involving psychometric reliability and validity metrics.</p><p><strong>Methods: </strong>The application was developed using R Shiny to create an interactive interface to calculate sample sizes for reliability, validity, and group difference assessments. For metrics already included in existing tools, cross-validation was performed to ensure consistency and accuracy. For metrics unique to this app, validation was based on formulas from the original publications.</p><p><strong>Results: </strong>A comprehensive set of sample size calculation modules was included, covering Pearson correlation, Spearman correlation, tetrachoric correlation, point-biserial correlation, Cohen's kappa, Fleiss' kappa, Cronbach's alpha, intraclass correlation coefficient, Kendall's coefficient of concordance, one-way analysis of variance, and one-way analysis of covariance. Researchers can access detailed outputs, including sample sizes, contextual interpretations, and step-by-step derivations of the results, ensuring transparency and usability.</p><p><strong>Conclusions: </strong>The Sample Size Calculator is a validated and helpful resource for researchers seeking to optimize study design. The calculator can be accessed at the following website: https://biostats-shinyr.kumc.edu/PsychometricN/.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149040344","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment burden, healthcare experiences, and health-related quality of life among youth with chronic kidney disease treated for hypertension.","authors":"Morgan Botdorf, Nicole Marchesani, Rebecca Ruebner, Elana Di Cocco, Amy J Goodwin Davies, India Shepherd Hammond, Jordan Musante, Aneta Jovanovska, Mark Levondosky, Rebecca Levondosky, Victoria H Besser, Donna J Claes, Anne E Dawson, Bradley P Dixon, Caroline Gluck, Susan M Halbach, Lyndsay Harshman, Chryso Katsoufis, Alexander J Kula, Young-Hyun Moon, Larry Patterson, Eloise Salmon, Elizabeth M Talley, Michelle R Denburg, Christopher B Forrest","doi":"10.1007/s11136-026-04401-x","DOIUrl":"10.1007/s11136-026-04401-x","url":null,"abstract":"<p><strong>Background: </strong>Controlling blood pressure (BP) is essential for slowing chronic kidney disease (CKD) progression. This study examined the associations between treatment burden, assessed by antihypertensive medication use, and health-related quality of life (HRQoL) in youth with CKD and hypertension.</p><p><strong>Methods: </strong>Youth (aged 8 to 21 years) were identified through electronic health records from 15 institutions. Youth and caregivers completed an online questionnaire assessing healthcare experiences, medication use, physical symptoms, and BP monitoring. HRQoL and related well-being domains (fatigue, anxiety, sleep disturbance, global health, and life satisfaction) were assessed using PROMIS^® measures. Analyses focused on youth-reported outcomes. Linear regression estimated associations between antihypertensive medication use and HRQoL outcomes, and scores were compared to the general population.</p><p><strong>Results: </strong>The study included 395 youth (M_age = 16.8 years, 54% female) and 238 caregivers. Over 95% of caregivers reported generally positive healthcare experiences. Most youth (92%) reported at least one of the physical symptoms asssessed (median [IQR] = 5 [3, 8] symptoms). Twenty-one percent took no antihypertensive medications, 56% took one, and 23% took multiple. Use of multiple medications was associated with slightly lower global health (B = - 2.6; 95% CI: - 4.9, - 0.3) after adjustment for CKD stage. No associations were observed for other HRQoL domains. Overall, youth reported lower QoL than the general population, with the greatest impairments observed in global health and fatigue.</p><p><strong>Discussion: </strong>Youth with CKD and hypertension reported generally positive healthcare experiences, and antihypertensive medication use, one component of treatment burden, showed minimal association with HRQoL. Despite modest effects, continued attention to HRQoL remains an important aspect of care for youth with CKD and hypertension.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149040430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating patient-reported outcome measurement in fibromyalgia clinical trials: a cross-sectional meta-research analysis of alignment with IMMPACT recommendations.","authors":"Micaylon Moore, Hayden Keeler, Lauren Kocour, Noah Calvert, Makalie Lackey, Tanner Livsey, Ryan Langerman, Amy Nguyen, Arnab Mukherjee, Alicia Ito Ford, Matt Vassar","doi":"10.1007/s11136-026-04393-8","DOIUrl":"10.1007/s11136-026-04393-8","url":null,"abstract":"<p><strong>Purpose: </strong>Fibromyalgia diagnosis and treatment response depend mostly on patient report, making patient-reported outcomes central to trial evidence. The Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials provides recommendations for standardized outcome domains and endorsed patient-reported instruments in chronic pain trials. Whether fibromyalgia trials have converged on a common set of instruments remains unclear. We evaluated instrument standardization and IMMPACT alignment among registered fibromyalgia trials.</p><p><strong>Methods: </strong>We conducted a cross-sectional meta-research analysis of 916 interventional fibromyalgia trials registered on ClinicalTrials.gov. Outcomes were classified against an inductively built dictionary of patient-reported outcome instruments, with alignment defined as registration of at least one consensus-body-endorsed instrument. Completeness and alignment were assessed relative to the consensus body's 2005 publication, and cohort assembly and instrument detection were independently verified.</p><p><strong>Results: </strong>Of 916 eligible trials, 778 (84.9%) named a patient-reported outcome instrument, rising from 47.4% in 2005 to a peak near 98% in 2021. Ninety-five distinct instruments were registered, but only 49.1% of these trials included one the consensus body endorses. The most common measure was the Fibromyalgia Impact Questionnaire, an unendorsed fibromyalgia-specific tool, and the non-endorsed Visual Analog Scale was registered nearly twice as often as the consensus-body-endorsed Numeric Rating Scale (348 vs. 192 trials).</p><p><strong>Conclusion: </strong>Fibromyalgia trials have largely resolved whether to measure patient-reported outcomes, but instrument selection remains fragmented despite two decades of guidance, limiting comparability across trials. Requiring a consensus-body-endorsed instrument alongside a disease-specific measure would close this gap without sacrificing disease-specific detail.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149031309","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Beyond observed changes: how differential item functioning and response shift alter health-related quality of life interpretation in preemptive versus non-preemptive kidney transplant patients.","authors":"Myriam Blanchin, Yseulys Dubuy, Magali Giral, Bénédicte Sautenet, Emmanuel Morelon, Laetitia Albano, Aurélie Meurette, Véronique Sébille","doi":"10.1007/s11136-026-04386-7","DOIUrl":"10.1007/s11136-026-04386-7","url":null,"abstract":"<p><strong>Purpose: </strong>Experiencing dialysis and receiving a kidney transplant may induce differential item functioning (DIF) and response shift (RS), reflecting changes in how patients interpret and evaluate their health-related quality of life (HRQoL). This study investigates the impact of DIF and RS on HRQoL change estimation in preemptive (PKT) and non-preemptive (nPKT) kidney transplant patients before and after transplantation.</p><p><strong>Methods: </strong>In the prospective, multicentre, controlled PreKit-QoL study, HRQoL was assessed using the SF-36 every 6 months before transplantation, at hospital discharge, at 3 and 6 months, and annually until 5 years post-transplantation in PKT (n = 158) and nPKT (n = 226) patients. Changes in HRQoL and DIF and RS occurrence between the last visit before transplantation and 3 months post- transplantation, and between 3 and 6 months post-transplantation were investigated with an item-level DIF and RS detection algorithm, ROSALI. ROSALI estimates DIF and RS effects, and HRQoL changes accounting for these effects using Rasch models.</p><p><strong>Results: </strong>Four SF-36 dimensions (Bodily Pain, Vitality, Social Functioning, Mental Health) were unaffected by DIF and RS, and showed general improvement shortly after transplantation, with only changes in Bodily Pain differing between groups. Two dimensions (Role Physical, General Health) exhibited DIF and RS shortly after transplantation. Ignoring these phenomena would have masked the deterioration in Role Physical for PKT patients and slightly underestimated the improvement in General Health.</p><p><strong>Conclusion: </strong>Investigating DIF and RS is essential for accurate PROM interpretation, revealing how patients' perception may evolve following transplantation. These insights inform both statistical analysis and understanding of patients' lived experience.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149018023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Making questionnaires easier to reuse: a call for standardized reporting of copyright and permission information.","authors":"Himel Mondal, Shaikat Mondal","doi":"10.1007/s11136-026-04396-5","DOIUrl":"10.1007/s11136-026-04396-5","url":null,"abstract":"<p><p>Published studies reporting newly developed questionnaires often provide detailed information on development and validation but do not clearly report how the questionnaire can be reused. Basic information such as copyright ownership, licensing, permission requirements, fees, and conditions for translation, modification, reproduction, or distribution may be missing or difficult to locate. Existing guidance, including COSMIN, focuses on methodological quality and measurement properties rather than reuse conditions. We propose that every study reporting the development of a questionnaire, scale, or structured measurement instrument include a standardized instrument reuse and licensing information table. This table should clearly report the copyright holder, applicable licence, permission and payment requirements, permitted uses, modification and translation conditions, reproduction and distribution requirements, and contact details. Such a simple reporting practice would reduce uncertainty, save researchers time, and promote transparent, reproducible, and responsible reuse of validated measurement instruments. A questionnaire should not only be validated for use; its conditions for reuse should also be clearly reported.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric evaluation of PROMIS^® fatigue and cognitive function short forms customized for adults living with primary sclerosing cholangitis.","authors":"Bryce B Reeve, Li Lin, Brian Thorsen, Rachel Gomel, Christopher L Bowlus, Donna M Evon","doi":"10.1007/s11136-026-04398-3","DOIUrl":"10.1007/s11136-026-04398-3","url":null,"abstract":"<p><strong>Purpose: </strong>The study goal is to evaluate the psychometric properties of Patient-Reported Outcomes Measurement Information System^® (PROMIS^®) Fatigue and Cognitive Function Short Forms customized for use in adults with primary sclerosing cholangitis (PSC).</p><p><strong>Methods: </strong>Participants completed a survey through the PSC Partners Patient Registry. The survey included the customized PROMIS short forms, the PROMIS Global Health scale, the PROMIS Sleep-related Impairment short form, and self-reported sociodemographic and clinical data. The factor structure was evaluated with confirmatory factor analysis, and reliability indices included coefficient alpha for internal consistency and intra-class correlations for test-retest reliability. Construct validity was evaluated based on the associations of the customized short forms with related patient-reported outcomes and the ability to differentiate between known groups.</p><p><strong>Results: </strong>Participants included 287 adults, including 55% female, 93% White race, with a mean age of 52 years. Both Fatigue and Cognitive Function short forms were confirmed to be unidimensional, with internal consistency reliability of 0.98 and 0.97, and test-retest reliability of 0.92 and 0.89, respectively. The correlation between Fatigue and Cognitive Function scores was - 0.76, and had strong associations with physical health, mental health, sleep-related impairment, work productivity, and daily activities. Both customized short forms found statistically significantly worse fatigue and cognitive functioning in women, those with cirrhosis, and with a hepatic decompensation event in the past 12 months.</p><p><strong>Conclusion: </strong>This study provided strong evidence for the construct validity and reliability of the PROMIS Fatigue and Cognitive Function short forms customized for adults with PSC. These PSC-specific outcome measures will benefit clinical research.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":"35 10","pages":""},"PeriodicalIF":3.4,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149018005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}