Journal of Patient-Reported Outcomes最新文献

{"title":"Bowel urgency in ulcerative colitis: effect of baseline urgency and change in urgency in response to mirikizumab.","authors":"David B Clemow, Marla C Dubinsky, Simin K Baygani, Bruce E Sands, Anthony Keohane, Silvio Danese, Stefan Schreiber, Alissa J Walsh, Toshifumi Hibi, Theresa Hunter Gibble, Richard E Moses, Simon P L Travis","doi":"10.1186/s41687-025-00906-0","DOIUrl":"10.1186/s41687-025-00906-0","url":null,"abstract":"<p><strong>Background: </strong>Mirikizumab has demonstrated efficacy in moderately to severely active ulcerative colitis. A 1-2-point change in Urgency Numeric Rating Scale (NRS) score can be meaningful for patients. In these post-hoc analyses, we evaluated the efficacy of mirikizumab compared to placebo by baseline Urgency NRS score groups (0-3, 4-6, and 7-10) and its effect on bowel urgency severity over time.</p><p><strong>Methodology: </strong>Urgency NRS was measured as a secondary outcome at baseline, week 12, and week 52. Bowel urgency improvement was assessed for patients who achieved and did not achieve multiple efficacy endpoints. Data were analyzed using Fisher's exact test with nonresponder imputation.</p><p><strong>Results: </strong>At weeks 12 and 52, a significantly higher percentage of mirikizumab-treated patients achieved clinical response as well as clinical, endoscopic, and symptomatic remission compared to placebo-treated patients, regardless of baseline Urgency NRS score category (higher proportions versus placebo, delta 9%-45%). Improvement in Urgency NRS score category at weeks 12 and 52 for mirikizumab-treated patients was observed when other efficacy outcomes were achieved (13%-90%) and not achieved (12%-75%).</p><p><strong>Conclusions: </strong>A greater proportion of mirikizumab-treated patients with ulcerative colitis achieved symptomatic, clinical, and endoscopic remission endpoints compared to placebo-treated patients, regardless of baseline bowel urgency severity. After one year, bowel urgency was improved to a greater extent with mirikizumab than with placebo, even for patients who did not achieve other clinical outcomes. Small improvements in bowel urgency are associated with significant health-related quality-of-life improvements. Monitoring shifts in urgency severity over time using the Urgency NRS can aid in understanding patients' treatment outcomes.</p><p><strong>Trial registration: </strong>LUCENT-1 (NCT03518086) Registered 04 May 2018 https://clinicaltrials.gov/study/NCT03518086 . LUCENT-2 (NCT03524092) Registered 10 May 2018 https://clinicaltrials.gov/study/NCT03524092 .</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"75"},"PeriodicalIF":2.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144545182","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":"NASH-CHECK patient-reported outcome instrument: evaluation of content and face validity for patients with metabolic dysfunction-associated steatohepatitis and compensated cirrhosis.","authors":"Lynda C Doward, Matthew Breckons, Maria-Magdalena Balp, James Twiss, Luke Vale, Lorraine McSweeney, Clifford A Brass, Quentin M Anstee, Arun J Sanyal","doi":"10.1186/s41687-025-00881-6","DOIUrl":"10.1186/s41687-025-00881-6","url":null,"abstract":"<p><strong>Background: </strong>NASH-CHECK is a patient-reported outcome measure (PROM) that assesses symptoms and broader health-related quality of life (HRQOL) impacts of metabolic dysfunction-associated steatohepatitis (MASH), previously called non-alcoholic steatohepatitis (NASH). The measure was initially developed and validated for patients with non-cirrhotic MASH. This study describes an evaluation of the suitability of NASH-CHECK for patients with compensated cirrhotic MASH.</p><p><strong>Methodology: </strong>Concept elicitation (CE) interviews were conducted with patients with clinically confirmed compensated cirrhotic MASH in the United States (US) and United Kingdom (UK) to determine the symptom burden and broader HRQOL impact of MASH. Symptoms and broader HRQOL impacts identified during analysis of the CE data were compared with the key concepts included in NASH-CHECK; any symptoms reported in the CE interviews but not reflected in NASH-CHECK were reviewed for relevance by clinical experts. The content validity of NASH-CHECK was evaluated further via cognitive debriefing (CD) interviews conducted with patients with compensated cirrhotic MASH in the US and UK.</p><p><strong>Results: </strong>CE interviews were conducted with 33 patients with compensated cirrhotic MASH (US = 9, UK = 24; 60.6% female; mean age, 64.3 years). Key symptoms described were similar to those reported by patients with non-cirrhotic MASH identified during the development of NASH-CHECK; these included abdominal pain, abdominal bloating, itch, fatigue, sleeping difficulties, and cognitive symptoms. Other key HRQOL impacts included activity limitations and emotional, social, relationship, and work impacts. All key symptom and broader HRQOL impacts reported by patients with compensated cirrhotic MASH are currently included in NASH-CHECK, and no additional symptoms or HRQOL impacts reported during the CE interviews were deemed relevant for inclusion. CD interviews were conducted with 17 patients with compensated cirrhotic MASH (US = 8, UK = 9; 47.1% female; mean age, 62.8 years). Patient feedback on NASH-CHECK content confirmed that the concepts captured by the items were considered important, relevant, and comprehensive for addressing the impact of compensated cirrhotic MASH.</p><p><strong>Conclusions: </strong>The results support the content validity of NASH-CHECK for patients with compensated cirrhotic MASH, demonstrating that NASH-CHECK is a suitable PROM for use in clinical trials, studies, and practice for this patient population.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"76"},"PeriodicalIF":2.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144545199","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":"Investigating themes in hearing quality of life with user-nominated goals on the Glasgow Hearing Aid Benefit Profile (GHABP).","authors":"Avivah J Wang, Grace Strong, Kayla W Kilpatrick, Sherri L Smith, Theresa Coles","doi":"10.1186/s41687-025-00886-1","DOIUrl":"10.1186/s41687-025-00886-1","url":null,"abstract":"<p><strong>Background: </strong>The Glasgow Hearing Aid Benefit Profile (GHABP) is a patient-reported outcome measure (PROM) that was developed for the assessment of hearing aid efficacy using standard goals and user-nominated goals. The objective of this study was to describe user-nominated hearing goals to determine themes that are not currently being captured by the standard goals and that could improve comprehensive assessment of hearing quality of life with the GHABP for use in clinical trials.</p><p><strong>Methodology: </strong>We conducted a secondary analysis of a clinical trial at two tertiary care institutions. Adults ≥ 50 years of age with hearing loss completed the GHABP before treatment, including the portion where they provided up to two user-nominated goals for situations where hearing was personally challenging to them. We then categorized these goals into themes.</p><p><strong>Results: </strong>A total of 262 participants completed the standard GHABP and provided a total of 501 user-nominated goals. Common themes were having a conversation with several people in a group (80/501, 16.0%), hearing in background noise (73/501, 14.6%), and listening when unable to see the speaker's mouth (57/501, 11.4%).</p><p><strong>Conclusions: </strong>Themes of listening in background noise and listening when unable to see the speaker's mouth are very important to many individuals with hearing loss. Expanding PROMs to include these goals may improve patient-centeredness of clinical trial and clinical care outcomes tracking.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"73"},"PeriodicalIF":2.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144545198","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":"Decline in quality of life among caregivers of patients undergoing chemotherapy for incurable cancer: implications for early social and medical support.","authors":"Nobumichi Takeuchi, Saiko Kurosawa, Sonomi Yoshida, Kumiko Koike","doi":"10.1186/s41687-025-00912-2","DOIUrl":"10.1186/s41687-025-00912-2","url":null,"abstract":"<p><strong>Background: </strong>Recent advances in cancer treatment have extended patient survival and improved quality of life (QOL), often enabling home-based chemotherapy. However, this shift places a growing burden on informal caregivers, impacting their own well-being. This study aims to explore changes in caregiver QOL over the course of treatment and identify contributing factors.</p><p><strong>Methods: </strong>We conducted a single-institution, prospective observational study involving patients receiving chemotherapy for unresectable or recurrent solid tumors and their primary caregivers. QOL was assessed using the EORTC QLQ-C30 before each treatment line. Scores were stratified by treatment duration and line. Paired t-tests and multiple linear regression analyses were performed.</p><p><strong>Results: </strong>Among 378 patient-caregiver pairs, caregiver emotional and cognitive functioning declined over time, particularly with longer treatment durations and later treatment lines. Fatigue prevalence increased to 100% by the fourth-line treatment. Regression analyses revealed that caregiver QOL was affected by patient symptoms (e.g., insomnia, appetite loss), patient QOL scores, treatment duration, and caregiver age and gender.</p><p><strong>Conclusions: </strong>Caregivers experienced progressive emotional and cognitive declines paralleling the patient's clinical trajectory. These findings highlight the necessity of early and comprehensive support systems for caregivers, including psychological and social support, to maintain their QOL throughout cancer treatment.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"74"},"PeriodicalIF":2.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214165/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144545183","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":"Enhancing patient-centric care: the role of PROMs utilizing SRS-30 in pediatric scoliosis management.","authors":"Marina Rosa Filezio, Ramyn Jooma, Paul Fairie, David Parsons, Maria J Santana","doi":"10.1186/s41687-025-00904-2","DOIUrl":"10.1186/s41687-025-00904-2","url":null,"abstract":"<p><strong>Background: </strong>Scoliosis is defined by a curvature of the spine greater than 10 degrees. The most common type of scoliosis is called Adolescent Idiopathic Scoliosis and is found in individuals between 11 to 18 years of age. It corresponds to 90% of the cases of scoliosis in the pediatric population, with an overall prevalence of 0.47-5.2%, affecting girls more than boys (3:1). There are different treatment options for scoliosis, and surgery is reserved for patients with curves greater than 45 degrees while still growing or greater than 50 degrees for skeletally mature patients. There is a growing recognition of the important role of patient-reported outcomes measures (PROMs) for understanding the impact of scoliosis on individuals' lives and its management. This paper explores the importance of PROMs, specifically the Scoliosis Research Society-30 (SRS-30) questionnaire, in assessing and improving the quality of care for pediatric scoliosis patients that were submitted to surgical intervention.</p><p><strong>Methodology: </strong>PROMs data were collected at predefined time points: pre-operatively (baseline), and post-operatively at 3, 6, and 12 months. The evaluation encompassed 23 (pre-operative assessment) to 30 questions (follow-up) and included five key domains: Function/Activity, Pain, Self-Image/Appearance, Mental Health, and Satisfaction with Management, as well as possible changes in the results before and after surgery.</p><p><strong>Results: </strong>115 patients participated in this study, of whom 79% were females (mean age 14.5 years). Function/Activity was the only domain to exhibit a significant score decrease in the post-operative follow-up, with a return to baseline levels at the 12-months mark. All other domains showed statistically significant improvement over time, with the steepest increase observed at 3 months for Self-Image/Appearance and Satisfaction with Management. Age did not significantly influence on the results across any of the five domains.</p><p><strong>Conclusions: </strong>This project highlights the pivotal role of PROMs, with a specific focus on the SRS-30 questionnaire results, in creating a more holistic and patient-centered approach to scoliosis management.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"78"},"PeriodicalIF":2.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214136/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144545184","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":"Internal consistency and structural validity of the parent-report preschool (2-4 years) Strengths and Difficulties Questionnaire in 1-year-old children.","authors":"Sarah L Blower, Kate E Mooney, Nicole Gridley, Fionnuala Larkin, Tracey J Bywater, G J Melendez-Torres","doi":"10.1186/s41687-025-00905-1","DOIUrl":"10.1186/s41687-025-00905-1","url":null,"abstract":"<p><strong>Background: </strong>Prevention and early intervention are key to addressing poor child mental health. Systematic reviews have highlighted a lack of brief, valid and reliable outcome measures that can be implemented in both research and practice to assess social, emotional and behavioural outcomes in the early years. The Preschool Strengths and Difficulties Questionnaire (2-4 years) is a promising candidate to fill this gap, but the measurement properties of this tool are not yet known in very young children.</p><p><strong>Methods: </strong>A secondary data analysis of two clinical trial datasets was conducted to examine the internal consistency reliability and structural validity of the parent-report English preschool version of the Strengths and Difficulties Questionnaire in a sample of 505 infants with mean average age of 18 months (SD .81). The measure was designed for children aged 2-4 years and was not modified prior to use with 1-year-olds in this study. Structural validity was examined in two Confirmatory Factor Analyses (CFA) testing two-factor and five-factor models (representing factor structures proposed by the developers of SDQ), and McDonald's coefficient Omega was estimated for each subscale with values > .70 considered acceptable.</p><p><strong>Results: </strong>The model fit values for the two-factor model demonstrated a poor fit to the data (X² = 626.067(151) = p < .001, CFI = 0.612, RMSEA = 0.079 [90% CI .073 to .085], SRMR = .077) and the omega value was below acceptable at ω = .57 for the internalising subscale and ω = .76 for the externalising subscale. The five-factor model also demonstrated a poor fit to the data (X² = 836.813(242) = p < .001, CFI = 0.676, RMSEA = 0.070 [90% CI .065 to .075], SRMR = .081). Omega values were below acceptable for three out of five subscales.</p><p><strong>Discussion: </strong>We concluded that the measure has poor internal consistency and lacks structural validity in this very young age group. Further research to adapt the SDQ in order to improve content and face validity is recommended prior to any further psychometric analyses with this very young age group. The paucity of robust and practical outcome measures of early social, emotional and behavioural poses significant challenges to the early identification of need and evaluation of interventions.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"72"},"PeriodicalIF":2.4,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12185824/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144477140","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 properties of patient-reported outcome measures to assess premenstrual syndrome/premenstrual dysphoric disorder in japanese: a systematic review using the COSMIN methodology.","authors":"Kaori Tsuyuki, Miho Egawa, Takuma Ohsuga, Yumie Ikeda, Yoshitake Takebayashi, Hideki Sato, Masaki Mandai","doi":"10.1186/s41687-025-00910-4","DOIUrl":"10.1186/s41687-025-00910-4","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methodology: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"71"},"PeriodicalIF":2.4,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12181441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144334059","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 of a unified system for assessing health related quality of life across the cancer care continuum: the EUonQoL Delphi study to identify priorities for quality of life domains.","authors":"Maike G Sweegers, Esther de Jongh, Christopher Bedding, Emma Nicklin, Daniela Doege, Sara Alfieri, Laura Gangeri, Bianca Scacciati, Augusto Caraceni, Cinzia Brunelli, Anne Bredart, Leslye Rojas-Concha, Helle Pappot, Giovanni Apolone, Nanne Bos, Gennaro Ciliberto, Norbert Couespel, Montse Ferrer, Stein Kaasa, Claudio Lombardo, Ricardo Pietrobon, Gabriella Pravettoni, Aude Sirven, Hugo Vachon, Mogens Groenvold, Maria Alice Franzoi, Galina Velikova, Alexandra Gilbert, Lonneke V van de Poll-Franse","doi":"10.1186/s41687-025-00907-z","DOIUrl":"10.1186/s41687-025-00907-z","url":null,"abstract":"<p><strong>Introduction: </strong>Cancer and cancer treatment have a major impact on health related quality of life (HRQoL). To improve the assessment of HRQoL in patients with cancer and evaluate the impact of policy interventions, the European Oncology Quality of Life (EUonQoL) project aims at developing a digital, patient centred system to assess HRQoL based on evaluations and preferences of cancer patients and survivors: the EUonQoL-kit.</p><p><strong>Method: </strong>Patients across the cancer care continuum, healthcare professionals and researchers from six European countries (Denmark, France, Germany, Italy, The Netherlands and United Kingdom) were asked to rate the importance of 44 pre-selected HRQoL subdomains over a maximum of three Delphi survey rounds. We evaluated the importance of HRQoL subdomains for three target populations: patients undergoing active treatment, cancer survivors and patients receiving palliative care. The results were discussed during a consensus meeting.</p><p><strong>Results: </strong>96 patients and 59 healthcare professionals participated in the Delphi study. After three rounds, consensus was reached for 20 subdomains: ability to work, communication with healthcare professionals, diarrhoea, fatigue, fear of recurrence, global health status, impact of treatment side effects, impact on children/family, insomnia, instrumental activities of daily living, maintaining independence, mobility, nausea, overall quality of life, pain, partner relationship, social activity limitations, social isolation, symptom awareness and uncertain prognosis. The subdomains pain and fear of recurrence were rated as important for all three target populations.</p><p><strong>Conclusion: </strong>Subdomains that were considered important for the assessment of HRQoL in patients with cancer can be summarised into: physical symptoms, mobility & activity, future outlook, social roles & activities, family & relationships, social isolation, self-efficacy, overall HRQoL, and healthcare experience. The importance of the subdomains differed for patients in different phases of the cancer care continuum. These findings were used for the creation of the first version of the EUonQoL-Kit, as a base for its further development.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"70"},"PeriodicalIF":2.4,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12179011/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144327118","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":"Validation of the Chinese version of the financial toxicity scale in patients with wet age-related macular degeneration.","authors":"Xiuli Chen, Hong Bian, Zhifeng Wu","doi":"10.1186/s41687-025-00909-x","DOIUrl":"10.1186/s41687-025-00909-x","url":null,"abstract":"<p><strong>Objective: </strong>to confirm the validity of the Comprehensive Score for Financial Toxicity (COST) Scale in Chinese patients with wet age-related macular degeneration.</p><p><strong>Methods: </strong>A tertiary hospital in Wuxi's ophthalmology outpatient clinic treated 217 patients with wet age-related macular degeneration (wAMD) were chosen using the convenience sample approach for a questionnaire survey between October 2023 and February 2024. The Chinese version of the COST and general patient information were included in the survey. Critical ratio analysis and correlation analysis were used to examine the items on the scale. The structural validity of the scale was evaluated using factor analysis, the reliability of the scale was evaluated using Cronbach's α coefficient and retest reliability, and the content validity of the scale was evaluated using the Content Validity Index (CVI).</p><p><strong>Results: </strong>The item analysis results demonstrated that high and low subgroups could be identified using The COST scale's Chinese translation (P < 0.01).; A linear positive correlation was observed between the scores of each item and the scale's overall scores (r values of 0.243 ~ 0.878, P < 0.01), and the scores of factor 1, factor 2, and factor 3 showed a linear positive correlation with the total scores of the scale (accordingly, r values were 0.974, 0.505, and 0.300; P < 0.01). and the scores of each item were linearly and positively correlated with the scores of the common factors to which they belonged (r values of 0.642 to 1.000, P < 0.01). Content validity showed that the I-CVI of each item was 0.857 ~ 1.00, and the S-CVI was 0.974. Three metrics in all, with a cumulative variance contribution rate of 67.739%, were obtained through exploratory factor analysis. Each item's loading on the corresponding dimensions varied from 0.638 to 0.954. The Cronbach's α coefficient for the entire scale was 0.876(95% CI: 0.85-0.899). The reliability of the retest was 0.970 (95% CI: 0.936-0.990).</p><p><strong>Conclusion: </strong>The Chinese version of the COST scale shows potential applicability pending further validation.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"69"},"PeriodicalIF":2.4,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170966/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144303118","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":"Assessment of current patient reported outcome measures for three core outcome domains for single-sided deafness device intervention trials.","authors":"Roulla Katiri, Deborah A Hall, Derek J Hoare, Sandra Smith, Bethany Adams, Kathryn Fackrell, Adele Horobin, Nicholas Hogan, Nóra Buggy, Pádraig T Kitterick","doi":"10.1186/s41687-025-00902-4","DOIUrl":"10.1186/s41687-025-00902-4","url":null,"abstract":"<p><strong>Background: </strong>Outcome reporting in clinical trials of auditory interventions for adults with Single-Sided Deafness (SSD) is inconsistent. The Core Rehabilitation Outcome Set for Single-Sided Deafness (CROSSSD) initiative has recommended three outcome domains as a minimum standard in the design of SSD intervention clinical trials. These are, Spatial orientation, Group conversations in noisy social situations, and Impact on social situations. The study objectives were to (i) understand exactly what the outcome domains mean to SSD experts, and (ii) identify and assess candidate PROMs in terms of how well they measure the experts' conceptualisation of those SSD outcome domains.</p><p><strong>Methodology: </strong>Stakeholder representatives participated in two semi-structured online focus groups. Participants were four adults diagnosed with SSD with experience of auditory interventions, two healthcare professionals working in the field, and one clinical researcher with experience in evaluating interventions. Thematic analysis was used to determine conceptual elements of each domain. COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) initiative recommendations were adopted to assess the relevance and comprehensiveness (content validity) of available candidate instruments.</p><p><strong>Results: </strong>Multiple key concepts were identified for each outcome domain, and presented as a taxonomy. To be acceptable, any measurement instrument would need to achieve good coverage of all concepts in this taxonomy. From the 76 candidate instruments reviewed, none met accepted standards for content validity for SSD. The best performing candidates were (i) Spatial orientation: the Spatial Hearing Questionnaire and two variants of the Speech, Spatial and Qualities scale (SSQ-12, SSQ-18-C), (ii) Group conversations in noisy situations: the Communication Profile for Hearing Impaired (CPHI) questionnaire, SSQ-12, SSQ-18-C, and a multi-item questionnaire developed by Schafer and colleagues, and (iii) Impact on social situations: the CPHI questionnaire.</p><p><strong>Conclusions: </strong>Multi-dimensional outcome domains introduce specific considerations for how they should be measured. Although some candidates instruments had reasonable comprehensiveness, modification is needed to ensure that there is overall greater relevance to the key concepts.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"68"},"PeriodicalIF":2.4,"publicationDate":"2025-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167737/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144303117","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}