Patient Related Outcome Measures最新文献

{"title":"Development and psychometric evaluation of cardiovascular health beliefs scale.","authors":"Cihan Asaroğlu, Hanife Özlem Sertel Berk","doi":"10.1080/1179271X.2026.2717078","DOIUrl":"10.1080/1179271X.2026.2717078","url":null,"abstract":"<p><strong>Objective: </strong>Cardiovascular disease (CVD) is a leading cause of mortality. This study aimed to develop and psychometrically evaluate the Cardiovascular Health Beliefs Scale (CHBS) to assess CVD-related health beliefs.</p><p><strong>Methods: </strong>A cross-sectional methodological design was utilized in two phases. Phase 1 (<i>n =</i> 150) focused on item generation and exploratory factor analysis. Phase 2 (<i>n =</i> 531) involved confirmatory factor analysis. Data were collected via face-to-face interviews using the newly developed CHBS and the Brief Symptom Inventory (BSI) General Severity Index to test construct validity.</p><p><strong>Results: </strong>The CHBS supported an acceptable six-factor structure: perceived susceptibility, perceived severity, health motivation, perceived benefits, perceived barriers, and self-efficacy. Cronbach's alpha coefficients ranged from .70 to .84, demonstrating adequate internal consistency.</p><p><strong>Conclusion: </strong>The CHBS demonstrated acceptable validity and reliability as a measurement tool. It enables nurses and healthcare professionals to assess risk behaviors and implement culturally congruent, targeted preventive interventions for cardiovascular care within the Turkish population.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"162-171"},"PeriodicalIF":2.4,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13671467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216617","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":"Digital patient-reported outcomes to evaluate treatment response to filgotinib in patients with ulcerative colitis.","authors":"Leona Mazza, Lawrence Matini, Grace Dunbar, Anita Punj Sharda, Lydia White, Andrey Kormilitzin, Hanan Azzu, Sarah Cripps, Jack Sanford, Vincent Cheung, Oliver Brain, Timothy Ambrose, Simon Pl Travis, Alissa Walsh","doi":"10.1080/1179271X.2026.2740952","DOIUrl":"10.1080/1179271X.2026.2740952","url":null,"abstract":"<p><strong>Objective: </strong>Patient-reported outcomes (PROs) are valuable tools for improving long-term patient outcomes and providing patient-centered care. PROs can be used to investigate real-world treatment effectiveness in patients with a chronic inflammatory disease.</p><p><strong>Methods: </strong>This was a real-world cohort study with digital patient-reported data collection by patients with ulcerative colitis (UC) using TrueColours-IBD, a web-based platform for prospective monitoring of symptoms, quality of life, and outcomes in Oxford. Effectiveness of filgotinib was compared between advanced therapy-naïve (ATN, i.e. filgotinib 1st line, <i>n</i> = 44) and advanced therapy-experienced (ATE, i.e. prior biological/small-molecule therapy, <i>n</i> = 56) patients using the following outcomes, evaluated post-induction (8-12 weeks) and at 6 and 12 months: clinical remission (Simple Clinical Colitis Activity Index (SCCAI) ≤2), clinical response (≥2 point SCCAI reduction from baseline), corticosteroid-free remission (defined as clinical remission without any prednisolone or budesonide in the 12 weeks preceding that time point), and treatment discontinuation (defined as stopped prescription of the medication).</p><p><strong>Results: </strong>There were no significant differences between the ATN and ATE groups regarding clinical remission, response, and corticosteroid-free remission at any time point, with <i>P</i> values > 0.1. More than half of the patients in each group achieved clinical remission and response at all time points. Filgotinib discontinuation rates within 12 months were comparable (<i>P</i> = 0.095).</p><p><strong>Conclusion: </strong>This study shows that PROs can serve as an important tool in evaluating real-world treatment outcomes in patients with UC using filgotinib. The results demonstrate good subjective clinical effectiveness, regardless of prior advanced therapy exposure.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"149-161"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13671468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216557","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":"Patients' experiences with using virtual reality for comfort during source isolation: a qualitative feasibility study.","authors":"Tjitske Diederike Groenveld, Suzan Boekelder, Cariline Roosen, Harry van Goor, Getty Huisman-de Waal","doi":"10.1080/1179271X.2026.2739003","DOIUrl":"10.1080/1179271X.2026.2739003","url":null,"abstract":"<p><strong>Background: </strong>Source-isolated patients endure feelings of loneliness, discomfort, and imprisonment with sensory deprivation and loss of autonomy. Supporting self-comforting strategies can increase comfort and autonomy and promote participation in recovery. We aim to explore whether and how source-isolated patients use virtual reality (VR) for self-comfort.</p><p><strong>Methods: </strong>A qualitative interview study, including a cohort of source-isolated patients. Patients received an VR intervention encompassing applications for relaxation and distraction and physical exercise during a maximum of 5 days. Patients were instructed using VR at least once a day for 20-30 minutes. Semi-structured face-to-face interviews were conducted after the intervention period or before discharge. Predefined topics were self-comforting strategies and patients overall experiences. Coding was guided by predefined topics, complemented with inductive coding of patients' overall experiences.</p><p><strong>Results: </strong>Twenty patients were eligible, of whom two were excluded and eight declined participation. Ten patients, median (range) age of 56 (40-88) years, used VR for two to five days during a median isolation time of seven days. Beside the predefined self-comforting strategies two additional main topics emerged: \"influencing factors on VR use\" and \"added value of VR\". VR was perceived to enhance positive thinking, mental distraction, active involvement, adaptation to limitations, and physical exercise. Soft- and hardware characteristics, access to the VR headset, patient characteristics, and ward environment may influence the perceived usability of VR. Perceived benefits of VR included promoting physical activity and providing an 'escape' from the hospital environment.</p><p><strong>Conclusion: </strong>VR has the potential to support patients' comfort during hospitalization and may offer opportunities for greater autonomy by enabling patients to actively manage own physical and psychological comfort.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"123-135"},"PeriodicalIF":2.4,"publicationDate":"2026-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13644613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137179","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":"Patient-reported outcomes in venous leg ulcers: results from a prospective multicenter single-arm cohort study of silicone superabsorbent polymer dressings.","authors":"Sebastian Probst, Vladica Veličković, Jacek Mikosinski, Konrad Panczak, Adam Wegrzynowski, Dorota Piechota, Marek Kotala, Przemyslaw Lipinski, Katarzyna Rybolowicz, Agnieszka Lipinska, Jacek Bil, Teresa Szenk, Patrycja Buczak, Cornelia Santoso, Anitha Pitchika, David G Armstrong","doi":"10.1080/1179271X.2026.2734965","DOIUrl":"10.1080/1179271X.2026.2734965","url":null,"abstract":"<p><strong>Background: </strong>Venous leg ulcers (VLUs) are chronic wounds associated with substantial symptom burden, impaired health-related quality of life, and high healthcare costs. Current guidance recommends assessing patient-centered outcomes alongside healing. This study reports the VLU subgroup of a prospective multicenter cohort, focusing on wound-related quality of life and patient-perceived treatment benefit.</p><p><strong>Methods: </strong>This prospective, single-arm cohort study was conducted in specialist wound centers in Poland. Adults with chronic, noninfected, exuding VLUs received RespoSorb Silicone Border or RespoSorb Silicone dressings together with standardized compression therapy for up to 6 weeks. Patient-reported outcomes (PRO) were the Wound Quality of Life questionnaire (Wound-QoL-17) and the Patient Benefit Index for wounds (PBI-W). Changes in Wound-QoL from baseline to final visit, responder analyses using the published 0.50-point minimal important difference and attained PBI-W scores at follow-up were analyzed.</p><p><strong>Results: </strong>Fifty-two patients with VLU were included (44.2% women; mean age 69.1 years). Final PRO data were available for 48 patients. Mean global Wound-QoL improved from 2.03 at baseline to 1.18 at final visit (mean change 0.81; 95% CI 0.49-1.12; <i>p</i> < 0.001; standardized response mean 0.74). Clinically meaningful improvement (≥0.50 points) occurred in 56.3% of patients. Final attained PBI-W benefit was high (mean 2.92 ± 0.97), with 93.8% achieving PBI-W ≥ 1 and 58.3% achieving PBI-W ≥ 3. No dressing-related serious adverse events were reported.</p><p><strong>Conclusions: </strong>Silicone superabsorbent polymer dressings used with compression were associated with improved quality of life and high patient-perceived benefit. Findings support incorporating validated patient-reported outcomes in VLU research and warrant larger comparative studies.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier NCT07549737 (Date of registration: 24.04.2026). Registration was completed retrospectively due to an administrative oversight during a transition in study leadership. The study protocol and prespecified study procedures were established before enrollment began, and the statistical analysis plan was finalized before database lock. No changes were made to the study objectives, endpoints, eligibility criteria, conduct, statistical analyses, or reporting as a result of the delayed registration.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"136-148"},"PeriodicalIF":2.4,"publicationDate":"2026-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13644622/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137243","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":"Five-year changes in generic and disease-specific patient-reported outcome measures in stable COPD: a real-world cohort study.","authors":"Koichi Nishimura, Masaaki Kusunose, Ayumi Shibayama, Kazuhito Nakayasu","doi":"10.1080/1179271X.2026.2732832","DOIUrl":"10.1080/1179271X.2026.2732832","url":null,"abstract":"<p><strong>Objective: </strong>Evidence comparing the long-term performance of generic and disease-specific patient-reported outcome (PRO) measures in chronic obstructive pulmonary disease (COPD) remains limited. This study compared five-year longitudinal changes and evaluative performance of commonly used generic and disease-specific PRO measures in stable COPD.</p><p><strong>Methods: </strong>This secondary analysis used data from a single-center longitudinal cohort of stable COPD outpatients. Participants completed assessments at baseline and five-years later. Disease- specific instruments included the COPD Assessment Test (CAT) and St. George's Respiratory Questionnaire (SGRQ). Generic instruments included the Medical Outcomes Study 36-Item Short Form (SF-36) and EQ-5D-5L. Distribution-based indices of five-year change were calculated using effect size (ES) and standardized response mean (SRM). Associations between changes in forced expiratory volume in one second (FEV₁) and PRO scores were examined using Spearman correlation coefficients.</p><p><strong>Results: </strong>Thirty-nine participants (mean age 74 years) completed five-year follow up. Disease-specific instruments showed minimal change, with small ES and SRM values (<0.5), although the SGRQ Activity deteriorated significantly. In contrast, several SF-36 subscales, including Physical Functioning, Role Physical, Social Functioning, and Role Emotional, declined significantly. Physical and Role/Social Component Summary scores and the EQ-5D-5L utility index also deteriorated significantly. Moderate distribution-based changes (ES 0.5-0.8) were observed in selected SF-36 subscales and in the EQ-5D-5L index. Mean annual FEV₁ decline was approximately 20 mL/year and was not significantly correlated with changes in PRO scores.</p><p><strong>Conclusion: </strong>In stable COPD, generic PRO measures showed greater long-term deterioration than disease-specific tools, suggesting value for monitoring gradual health decline in routine clinical practice.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"95-106"},"PeriodicalIF":2.4,"publicationDate":"2026-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13596913/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149025848","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 is the \"most bothersome symptom\" construct measured across health conditions? A systematic review of patient-reported outcome measures.","authors":"Alice M Mitchell, Vincent Singh Paramanandam, Dongzhe Lei, Theresa Donhauser, Niamh Waters, Cathy Hayles, Bethany Gower, Danushi Rajapakse, Eleanor Dickens, Ariane Suhood, K Jane Chalmers, Steven J Kamper, Claudia Cheng, Chen X Chen, Richard B Lipton, Martha Hickey, Marian Showell, Sarah Lensen","doi":"10.1080/1179271X.2026.2732841","DOIUrl":"10.1080/1179271X.2026.2732841","url":null,"abstract":"<p><strong>Objective: </strong>Patient-reported outcome measures (PROMs) capture patients' experiences and perspectives of symptoms. One approach involves measuring the \"most bothersome symptom,\" an outcome recommended in some regulatory guidance and core outcome sets. This systematic review aimed to identify and characterize PROMs to measure the \"most bothersome symptom,\" describe available measurement properties, and examine terminology used to describe \"bother.\"</p><p><strong>Methods: </strong>This review was registered in PROSPERO (CRD42022210384). A systematic search of EMBASE, MEDLINE, PsycINFO, and PROQOLID was conducted. Reviewers independently screened and extracted data on PROMs that directly measured the \"most bothersome symptom\" or indirectly assessed symptom bothersomeness. PROMs using related terminology (e.g. troublesome, distressing) were included. PROMs directly measuring the \"most bothersome symptom\" were evaluated using COSMIN and modified GRADE criteria.</p><p><strong>Results: </strong>In total, 270 articles describing 171 PROMs were included. Most PROMs (n = 135) indirectly captured bothersomeness through symptom scoring. Terminology inconsistencies were common; among 130 PROMs measuring \"bother,\" authors also used \"distress\" (10%), \"severity\" (8%), and \"burden\" (6%). Fourteen PROMs directly measured the \"most bothersome symptom,\" with only three demonstrating adequate content validity.</p><p><strong>Conclusions: </strong>Limited high-quality evidence supporting the measurement properties of PROMs designed to directly assess this construct, particularly content validity, restricts interpretability. More rigorous PROM development is required.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"107-122"},"PeriodicalIF":2.4,"publicationDate":"2026-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13596887/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149025835","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":"Comparison of two self-reported tools for assessment of sleep quality.","authors":"Melissa J Benton, Andrea M Hutchins","doi":"10.1080/1179271X.2026.2724201","DOIUrl":"10.1080/1179271X.2026.2724201","url":null,"abstract":"<p><strong>Objective: </strong>An easy to administer tool is needed for clinical assessment of sleep quality by healthcare providers. This study compared two brief, validated sleep questionnaires in order to identify an appropriate tool for clinical assessment.</p><p><strong>Methods: </strong>Women (N = 121) between the ages of 25-89 years (60.3 ± 15.9 years) completed the Richards-Campbell Sleep Questionnaire, the Restorative Sleep Questionnaire, and associated measures of quality of life, well-being, stress, and physical activity.</p><p><strong>Results: </strong>There were no differences in sleep quality assessments between the two questionnaires for sleep last night (daily), during the past 7 days (weekly), or during the past 4 weeks (monthly). Overall, participants reported good daily, weekly, and monthly sleep quality (mean scores 71.1-75.3 out of 100), although both questionnaires assessed significantly better (<i>p</i> ≤ 0.002) daily sleep quality compared to weekly and monthly. There were no floor effects for either questionnaire, but ceiling effects were noted for the Richards-Campbell Sleep Questionnaire daily and weekly versions. When participants were grouped by sleep quality, very good sleepers reported better quality of life (<i>p</i>˂ 0.05) and well-being (<i>p</i>˂ 0.001), and less stress (<i>p</i>˂ 0.05) compared to poor sleepers for all versions of the Restorative Sleep Questionnaire and for the Richards-Campbell Sleep Questionnaire daily and weekly versions.</p><p><strong>Conclusions: </strong>For overall identification and assessment of good versus poor sleep quality, clinicians can choose either the Richards-Campbell Sleep Questionnaire or the Restorative Sleep Questionnaire. However, caution may be needed for short-term (daily, weekly) assessment for which the Restorative Sleep Questionnaire may be more appropriate due to fewer ceiling effects.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"80-94"},"PeriodicalIF":2.4,"publicationDate":"2026-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13556930/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920571","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":"Aligning leadership incentives with patient outcomes in Somalia's public hospitals.","authors":"Abdirasak Sharif Ali, Mohamed Mustaf Ahmed","doi":"10.1080/1179271X.2026.2727280","DOIUrl":"10.1080/1179271X.2026.2727280","url":null,"abstract":"","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"76-79"},"PeriodicalIF":2.4,"publicationDate":"2026-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13556966/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920620","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":"Trust Building for Optimal Adoption of Digital Healthcare Technologies for Diabetes Management: Qualitative Analysis.","authors":"Jan Liska, Inga Nandzik, Cristina Arias Gonzalez Anleo, Cécile Dessapt-Baradez, Monica Bertolini, Christophe Maillard, Cheryl D Stults, Mikki Nasch, David Kerr","doi":"10.2147/PROM.S498339","DOIUrl":"10.2147/PROM.S498339","url":null,"abstract":"<p><strong>Background: </strong>Trust building is essential for effective healthcare, including care delivered using digital health technologies (DHTs). This analysis aimed to develop an understanding of the mechanisms of trust building in relation to DHTs for diabetes using tracking and measuring tools.</p><p><strong>Methods: </strong>This was a multimethod qualitative analysis conducted in Germany, Spain, and the United States, using a behavioral science approach. Interviews were conducted with different groups of participants from June 2022 to August 2022. The interviews consisted of one-on-one interviews (90 min) with experts in the diabetes support system, online dyad interviews (120 min), and online homework (120 min) with adults with type 2 diabetes (T2D) with different mind states (struggling, juggling, and controlling) and their caregivers. Focus group discussions were conducted with healthcare professionals (HCPs; each 150 min), including general physicians, specialists, and nurses, representing a conservative or innovator persona.</p><p><strong>Results: </strong>Nine interviews were conducted with experts (n=9), 28 dyad interviews were conducted with people with T2D (n=38) and caregivers (n=18), and 15 focus group discussions were conducted with 59 HCPs. Building trust in DHTs appeared to be created differently from building trust in medications, and requires activating, motivating, and sustaining desired behavior change. DHT trust builds systematically in three levels: trust in individual's own role in diabetes management (self-efficacy), trust in data as a tool to create empowerment, and trust in viability of sustained behavior change.</p><p><strong>Conclusion: </strong>People who had built trust on all three levels felt in control of their diabetes and were motivated and engaged in DHTs. Focus on trust building is important for digital healthcare launch strategies, particularly in user conversion from consideration phase to actual initiation of use of DHTs.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"498339"},"PeriodicalIF":2.4,"publicationDate":"2026-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13523089/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148888174","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 the effectiveness of the TURALIO (pexidartinib) risk evaluation and mitigation strategy program: patient knowledge, attitudes, and behavior survey (2020-2024).","authors":"Charles Dharmani, Oluwatosin Fofah, Margaret Wooddell, Janet L Ramsey, Michele Riggen","doi":"10.1080/1179271X.2026.2688849","DOIUrl":"10.1080/1179271X.2026.2688849","url":null,"abstract":"<p><strong>Background: </strong>Treatment of tenosynovial giant cell tumor with TURALIO (pexidartinib) is associated with serious hepatotoxicity risk. To ensure its safe use, a Risk Evaluation and Mitigation Strategy (REMS) program was implemented, incorporating structured education, mandatory enrollment, and liver function monitoring. The assessment of patient and caregiver knowledge, attitudes, and behaviors (KAB) is a central component of evaluating REMS effectiveness.</p><p><strong>Methods: </strong>Four waves of TURALIO Patient KAB Survey were conducted between 2020 and 2024 among REMS-enrollees. Surveys assessed understanding of two Key Risk Messages (KRMs): (KRM 1) hepatotoxicity risk and symptom recognition, and (KRM 2) liver monitoring requirements before and during treatment. Response rates and demonstrated understanding of KRMs were summarized descriptively.</p><p><strong>Results: </strong>Across four waves of this voluntary survey, 239 participants completed KAB surveys. Survey response rates ranged from approximately one-third to one-half of invited participants across waves. Correct response rates for individual knowledge items were consistently high. Demonstrated understanding of hepatotoxicity risk remained at ≥80% (the predefined threshold) across waves (87.5% in Wave 1; 80.0% in Wave 4). Understanding liver monitoring requirements improved over time, reaching 100% in Wave 3. Combined demonstrated understanding of KRM1 and KRM2 was <80% in three survey waves, particularly in Wave 2 (73.4%). Demonstrated understanding of KRMs remained generally stable despite evolving patient demographics and treatment duration over time.</p><p><strong>Conclusion: </strong>Across KAB Survey waves, patients receiving TURALIO demonstrated sustained knowledge of hepatotoxicity risk and monitoring requirements supporting the effectiveness of the tREMS program in ensuring the safe real-world use of pexidartinib.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"17 1","pages":"62-75"},"PeriodicalIF":2.4,"publicationDate":"2026-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13536961/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148888122","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}