Journal of Patient-Reported Outcomes最新文献

{"title":"Translation, cross-cultural adaptation, and validation of the Norwegian version of the Keratoconus Outcomes Research Questionnaire.","authors":"Eilin Lundanes, Svein Magne Roten, Helle Kristine Falkenberg, Lena Leren, Vibeke Sundling","doi":"10.1186/s41687-025-00896-z","DOIUrl":"10.1186/s41687-025-00896-z","url":null,"abstract":"<p><strong>Purpose: </strong>To translate and adapt the Keratoconus Outcomes Research questionnaire (KORQ) to Norwegian language, culture, and environment, and to validate the translated version in a Norwegian population with keratoconus.</p><p><strong>Methods: </strong>KORQ was translated to Norwegian using a multi-step methodology. Persons with keratoconus submitted responses to KORQ-NO and NEI VFQ-25 through a digital platform, and a retest of KORQ-NO was performed over the telephone. Additional data from a clinical intervention study was included. The psychometric properties of KORQ-NO were assessed by Rasch analysis. Test-retest reliability, construct validity, and responsiveness were explored by Intraclass Correlation Coefficients, Spearman correlations and Wilcoxon Signed-rank test.</p><p><strong>Results: </strong>KORQ-NO and NEI VFQ-25 were completed by 165 participants with keratoconus. With few adjustments, the \"Activity limitations\" (AL) and \"Symptoms\" (S) subscales of KORQ-NO exhibit acceptable psychometric properties with good model fit, high internal reliability, and well-targeted items to the population. Deletion of four items (AL3, AL3b, AL12, AL15) improved dimensionality of the \"Activity limitations\" subscale. Differential item functioning was present in two items (AL4 and AL6). Participants and optometrists confirmed content validity, and KORQ-NO exhibited good test-retest reliability (AL ICC = 0.90 and S ICC = 0.81), construct validity, and responsiveness.</p><p><strong>Conclusions: </strong>Successful translation and adaptation of KORQ to Norwegian language, culture and environment was confirmed by acceptable psychometric properties, with good validity, reliability, and responsiveness. The authors support the use of KORQ-NO in research, clinical practice, and as documentation for national insurance benefit applications.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"57"},"PeriodicalIF":2.4,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12098238/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120319","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 validation of the EORTC QLQ-OES18 in patients with advanced or metastatic esophageal squamous cell carcinoma.","authors":"Lauren Podger, Daniel Serrano, Liyun Li, Lin Zhan, Boxiong Tang, Gisoo Barnes","doi":"10.1186/s41687-025-00891-4","DOIUrl":"10.1186/s41687-025-00891-4","url":null,"abstract":"<p><strong>Background: </strong>The EORTC QLQ-OES18 has previously demonstrated clinical validity; however, there are limited published psychometric data for patients with advanced esophageal squamous cell carcinoma (ESCC). We evaluated the measurement properties of the QLQ-OES18 in a clinical trial population of patients with advanced or metastatic ESCC.</p><p><strong>Methodology: </strong>Analyses used data from RATIONALE 302 (NCT03430843), a randomized phase 3 study of tislelizumab versus investigator-chosen chemotherapy as second-line treatment for patients with advanced or metastatic ESCC. Psychometric validation of the QLQ-OES18 included tests of reliability, construct validity, ability to detect change, and estimation of anchor-based meaningful within-patient change (MWPC) thresholds-the latter two being exploratory given that the trial was not powered to detect efficacy in patient-reported outcome endpoints.</p><p><strong>Results: </strong>In total, 512 patients were randomized to either tislelizumab or chemotherapy; the average age was 61.5 years, and 84.4% were male. Three of the 4 QLQ-OES18 multi-item scales (dysphagia, eating, and pain) and the index scale met the prespecified criterion for acceptable internal consistency as well as acceptable test-retest reliability. Associations between baseline QLQ-OES18 scores and convergent/discriminant validators were generally as expected (i.e., the QLQ-OES18 pain score had a strong positive correlation with the QLQ-C30 pain score). For known-groups validity, 88.6% of analyses demonstrated the hypothesized direction of effect, suggesting that the expected differences in baseline QLQ-OES18 scores between prespecified groups were observed. Ability to detect change analyses indicated that several QLQ-OES18 domain scores demonstrated sensitivity in detecting possible treatment effects, although many patients reported minimal symptoms at baseline, which limited the ability to detect significant improvement.</p><p><strong>Conclusion: </strong>Overall, a collection of psychometric evidence indicated that the EORTC QLQ-OES18 reliably and validly measured symptom severity in the RATIONALE 302 population. Specifically, the dysphagia domain consistently demonstrated robust psychometric properties. Limitations in data reduced the interpretability of MWPC thresholds and are discussed in detail.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"56"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12095746/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111248","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":"The relationship among patient reported outcome measure scores with health care costs and inpatient admission: results from Utah mEVAL and value driven outcomes.","authors":"Rachel Kroencke, Zoe Gombart, Yue Zhang, Haojia Li, Rachel Hess","doi":"10.1186/s41687-025-00889-y","DOIUrl":"10.1186/s41687-025-00889-y","url":null,"abstract":"<p><strong>Background: </strong>Patient-reported outcomes measures (PROMs) profile patient health status, have been found to be helpful in identifying high health care utilizers, and may be useful in providing targeted interventions to decrease health care costs. In 2013 the University of Utah Health (UU Health) began collecting mental and physical health PROMs using Patient Reported Outcomes Measurement Information System (PROMIS) instruments through a tool called My Evaluation (mEVAL). In 2012 UU Health began cataloguing inpatient and outpatient healthcare-associated costs. The objective of this study was to identify association of poor PROMIS physical function and depression scores with (1) likelihood of inpatient hospitalization and (2) overall inpatient healthcare costs.</p><p><strong>Methodology: </strong>This study was a retrospective observational cohort study including patients seen at UU Health between 1/2013 and 12/2017 who completed PROMIS instruments at an outpatient visit using the mEVAL platform. PROMIS instruments were completed prior to outpatient visits. The primary outcome was time to incident hospitalization modeled by using the Cox proportional hazards approach. For cost analysis, raw inpatient healthcare costs were fitted using a median regression model. Both results were adjusted.</p><p><strong>Results: </strong>Of 92,383 people, the average age was 48 (SD 18.6); 57% were female; and 87% identified as non-Hispanic white. A total of 11,909 patients who completed one or both of the mEVAL PROMIS instruments were admitted. The average PROMIS physical function and depression scores were 44.9 and 51.1, respectively. Those with worse physical function scores and worse depression scores were more likely to be hospitalized [HR = 1.77, 95% confidence interval (CI) (1.678, 1.872); HR (95% CI) = 1.149 (1.059, 1.246), respectively]. A physical function score 1.5 SD below the mean was associated with an increased median hospitalization cost of $2496; there was no statistically significant association between depression score 1.5 SD above mean and hospitalization costs.</p><p><strong>Conclusions: </strong>Poor physical function scores were associated with an increased risk of hospitalization and higher inpatient health costs, while poor depression scores were only associated with increased risk of hospitalization. Future work should examine if improvement in these PROMs alters these metrics.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"55"},"PeriodicalIF":2.4,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089542/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095121","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":"Health-related quality of life, physical and mental capacity at one year follow up of COVID-19 ICU patients: A prospective cohort study.","authors":"Anders Ersson, Henrik Överengen Reuterborg, Anestis Divanoglou, Richard Levi, Lotti Orwelius","doi":"10.1186/s41687-025-00883-4","DOIUrl":"10.1186/s41687-025-00883-4","url":null,"abstract":"<p><strong>Purpose: </strong>In 2020 as COVID-19 rapidly overwhelmed ICU resources, patient care capacity was reduced thus increasing the risk of development of post intensive care syndrome (PICS). Therefore, an increased incidence of survivors with neurocognitive and neuromuscular impairment could be anticipated. This study aimed to describe residual reductions in health-related quality of life (HRQoL) and risk factors for PICS as they pertain to outcomes one year after intensive care.</p><p><strong>Patients and methods: </strong>Between 01-03-2020 and 31-08-2020, all adult COVID-19 ICU patients discharged alive in two Swedish ICU were included. At 2-, 6- and, 12-months post discharge follow up was conducted. Primary outcome parameters were HRQoL up to 12-months after ICU discharge. Secondary outcome parameters were clinimetric results for physical, mental, and cognitive functions at 6 months after intensive care stay.</p><p><strong>Results: </strong>Data from 41 patients were analyzed. Fatigue, anxiety, respiratory impairments, and experienced decline in physical stamina were the dominating findings at 6 months. Criteria for PICS were fulfilled in 93% of the study population and a 60% reduction in overall HRQoL, compared with a normal age adjusted population, was seen at follow up. A slight improvement was seen at 6 months whereafter no further significant improvement in HRQoL was detected. Fatigue was the most dominant complaint, expressed by almost all patients at follow up.</p><p><strong>Conclusion: </strong>Long term outcome reported in this study showed longstanding impairment in HRQoL, mostly related to reduced well-being and perceived limitations in physical ability. Overall, our findings show similarities with previously reported recovery patterns after intensive care. However, the COVID-19 cohort displayed a more profound reduction in HRQoL paralleled with severe fatigue and respiratory limitations. This signals the need for a deeper understanding of pathophysiological mechanisms of COVID-19 induced residual impairments and more precise instruments to tailor an individually designed aftercare.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"52"},"PeriodicalIF":2.4,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12078742/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037809","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":"Quality of Life after Brain Injury in children aged six and seven years (QOLIBRI-KIDDY) - development and scale analysis of the first disease-specific self-report instrument for young children after traumatic brain injury.","authors":"Nicole von Steinbuechel, Marina Zeldovich, Fabian Bockhop, Ugne Krenz, Dagmar Timmermann, Anna Buchheim, Inga K Koerte, Michaela Veronika Bonfert, Steffen Berweck, Matthias Kieslich, Knut Brockmann, Maike Roediger, Sven Greving, Axel Neu, Ulrike Wartemann, Joachim Suss, Christian Auer, Holger Muehlan, Katrin Cunitz","doi":"10.1186/s41687-025-00890-5","DOIUrl":"10.1186/s41687-025-00890-5","url":null,"abstract":"<p><p>To date, there are no age-appropriate instruments for assessing the subjective impact of pediatric traumatic brain injury (TBI) sequelae on multiple domains of health-related quality of life (HRQoL) in young children. The present study therefore aims to develop and examine the psychometric properties of a new disease-specific, self-reported HRQoL instrument, the Quality of Life after Brain Injury for children aged 6-7 years (QOLIBRI-KIDDY). Questionnaire development included focus group interviews, cognitive debriefings, and Delphi expert panels. The pilot version of the instrument was tested in 72 children (6.00-7.92 years of age; 60% boys; 86% after mild TBI). After item reduction based on a confirmatory scale analysis considering the six-factor structure of the questionnaire versions for older children, adolescents, and adults (Cognition, Self, Daily Life & Autonomy, Social Relationships, Emotions, Physical Problems), its reliability and validity were investigated. The final version of the QOLIBRI-KIDDY comprises 23 items. Psychometric analyses indicated internal consistency to be satisfactory (ɑ = 0.49-0.72; ω = 0.57-0.78). Construct validity suggested the expected overlap between generic HRQoL and TBI-specific HRQoL (r = 0.17-0.36). There were small (r > 0.2) to moderate (r > 0.3) correlations between lower TBI-specific HRQoL and participants with lower learning rates, anxiety, depression, and post-concussion symptoms, particularly on the Cognition, Social Relationships, Emotions, and Physical Problems scales. The comparison of known groups revealed significant moderate and significant effects for lower HRQoL in children with depressive symptoms on the Emotions scale (d = - 0.46) and with post-concussion symptoms on the Cognition (d = - 0.42) and Social Relationships scales (d = - 0.56). The QOLIBRI-KIDDY is a comprehensive, yet economical tool, comparable in content and items to the other age-adapted QOLIBRI versions. Its application has the potential to provide longitudinal data on subjects after TBI from childhood to older age, with a subjective perspective that can contribute to improving the therapy, rehabilitation, and daily life of young children.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"53"},"PeriodicalIF":2.4,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12078902/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019461","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":"Research trends among new investigators at ISOQOL: a bibliometric analysis from 2019 to 2023.","authors":"Jae-Yung Kwon, Manraj N Kaur, Ellen B M Elsman, Ava Mehdipour, Lori Suet Hang Lo, Ahmed M Y Osman, Sandrine Herbelet, Carrie-Anne Ng, Lotte van der Weijst","doi":"10.1186/s41687-025-00878-1","DOIUrl":"10.1186/s41687-025-00878-1","url":null,"abstract":"<p><strong>Background: </strong>New investigators (NI), encompassing graduate students, recent doctoral graduates, and early-career faculty, are instrumental in advancing quality of life (QoL) research through innovative methodologies and diverse perspectives. Within the International Society for Quality of Life Research (ISOQOL), the New Investigators Special Interest Group (NI-SIG) fosters collaboration and supports this community. This study utilizes bibliometric analysis to examine the contributions of NI-SIG members, focusing on publication trends, collaboration patterns, and thematic developments in QoL research.</p><p><strong>Methodology: </strong>Data on publications authored by 56 NI-SIG members between 2019 and 2023 were extracted from Web of Science and Scopus. A two-step screening process, guided by the Wilson and Cleary model of QoL, identified 561 unique documents for analysis. Descriptive metrics included publication trends, citations, journal impact factors, and geographic distribution, while network analysis explored co-authorship patterns. Thematic mapping was conducted using clustering algorithms to identify established and emerging research areas.</p><p><strong>Results: </strong>Publication output rose steadily from 2019 to 2022, peaking at 163 publications before declining to 135 in 2023, accompanied by a reduction in average citations per document from 4.8 to 1.3. The majority of publications appeared in leading journals such as Quality of Life Research (n = 128), Journal of Patient-Reported Outcomes (n = 17), and BMJ Open (n = 15). Geographic analysis revealed that most contributors were from high-income countries, with the United States, Canada, and Australia accounting for over 50% of publications. Co-authorship network analysis highlighted a robust, interconnected cluster of authors, though opportunities remain to enhance global partnerships, particularly with low- and middle-income countries. Thematic analysis identified well-established areas, including psychometric validation and cancer, alongside emerging topics such as mixed methods in QoL research.</p><p><strong>Conclusion: </strong>This study highlights robust collaborations among NI-SIG members while identifying opportunities to enhance international collaboration and methodological innovation. Expanding partnerships with underrepresented regions and embracing advanced technologies such as natural language processing could foster inclusivity and drive transformative advancements in QoL measurement and application.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"54"},"PeriodicalIF":2.4,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12078741/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989587","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 validation of the Spanish for Ecuador Family Reported Outcome Measure (FROM-16) and its application to measure impact on family members of patients with skin diseases.","authors":"Andrea Cueva, Faraz M Ali, Jeffrey Johns, Andrew Y Finlay, Sam Salek","doi":"10.1186/s41687-025-00866-5","DOIUrl":"https://doi.org/10.1186/s41687-025-00866-5","url":null,"abstract":"<p><strong>Background: </strong>The Family Reported Outcome Measure (FROM-16) is a generic tool to measure the impact of health conditions on patients' family members and partners (FMs). This study aimed to translate, validate, evaluate, and implement FROM-16 in Ecuadorian Spanish and to assess the quality of life (QoL) of FMs of patients with skin diseases.</p><p><strong>Methods: </strong>A cross-sectional study of patients and their FMs was performed using FROM-16 and a five-point-Likert scale to evaluate patient´s skin health by the FM and physician. Construct validity, confirmatory factor analysis and item response modelling of FROM-16 were assessed.</p><p><strong>Results: </strong>195 FMs completed Ecuadorian FROM-16. Inter-item correlation was 0.40 and factor analysis confirmed the original two-factor model: Cronbach's alpha 0.89, with factor loadings of 0.44-0.76. Mean age of patients = 41.8 ± 31.1 years and of FMs = 47.3 ± 7; diseases were classified as inflammatory (n = 88) or non-inflammatory (n = 107). The mean FROM-16 score was 12.5 ± 7 meaning \"a moderate effect on QoL;\" however, scores of 29.2% (57 of 195) indicated a \"very large\" (n = 47) or \"extremely large\" (n = 10) effect. Populations with the highest burden were adult children main carers, not cohabiting with their sick parents (mean FROM-16 = 17 ± 7.7, n = 8, p = 0.05 versus those cohabiting), and FM of patients with inflammatory conditions (mean = 14 ± 6.9, n = 88, p = 0.006 versus those with non-inflammatory dermatoses).</p><p><strong>Conclusion: </strong>The FROM-16 is a succinct, well-structured two-domain instrument. It can be used to identify the largely overlooked impact on FMs of dermatology patients. Understanding this impact may contribute to better holistic care, inform physicians' decisions, and encourage further support for families.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"51"},"PeriodicalIF":2.4,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12064521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144051045","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 evaluation of the HFDD, PROMIS SD SF 8b, and MENQOL questionnaire in women experiencing vasomotor symptoms associated with menopause.","authors":"Andrew Trigg, Melissa Barclay, Sophie Whyman, Asha Lehane, Helena Bradley, Christoph Gerlinger, Christian Seitz, Adam Gater, Claudia Haberland","doi":"10.1186/s41687-025-00875-4","DOIUrl":"10.1186/s41687-025-00875-4","url":null,"abstract":"<p><strong>Background: </strong>Vasomotor symptoms (VMS; hot flashes) associated with menopause have significant impacts on health-related quality of life and are a leading cause for women seeking medical attention. Patient-reported outcome (PRO) instruments are commonly used to assess treatment benefit in VMS clinical trials and must demonstrate supportive evidence of measurement properties within the context of use. This study evaluated the measurement properties of scores from the Hot Flash Daily Diary (HFDD), PROMIS Sleep Disturbance Short Form 8b (PROMIS SD SF 8b) and Menopause-Specific Quality of Life (MENQOL) for measuring treatment efficacy in VMS clinical trials.</p><p><strong>Methods: </strong>Measurement properties of the HFDD, PROMIS SD SF 8b, and MENQOL scores were assessed using data (n = 400 participants) from a randomized, placebo-controlled, phase 3 study evaluating the efficacy and safety of elinzanetant for the treatment of VMS in postmenopausal women (OASIS 2). Analyses assessed distributional properties, reliability, validity, responsiveness, and thresholds for meaningful change.</p><p><strong>Results: </strong>Minimal floor and ceiling effects were found across the instruments at baseline. Inter-item correlations, and confirmatory factor analysis or item-response theory supported dimensionality and scoring for the MENQOL and PROMIS SD SF 8b, respectively. Test-retest reliability between Weeks 8 and 12 was good to excellent for HFDD Frequency and Severity of moderate-to-severe hot flashes scores, PROMIS SD SF 8b T-score and MENQOL Total score (intra-class correlation coefficients 0.835-0.971). Convergent and divergent correlations with instruments assessing similar or distinct constructs were consistent with pre-specified hypotheses. Known-groups validity was supported by significant differences (p < 0.0001) between subgroups hypothesized a priori as being clinically distinct. Responsiveness was indicated by consistent and statistically significant differences (p < 0.0001) in mean changes from baseline to Week 4 and 12 between groups of participants classified as 'improved', 'stable' and 'worsened' (effect sizes for improvement 0.81-4.62). Triangulation of estimates from multiple anchor-based analyses derived meaningful within-individual change thresholds for the HFDD, PROMIS SD SF 8b and MENQOL scores that were likely to exceed measurement error.</p><p><strong>Conclusions: </strong>Findings provide evidence that HFDD, PROMIS SD SF 8b, and MENQOL scores are valid, reliable and responsive to change, supporting their use for assessing key efficacy endpoints in VMS clinical trials.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"50"},"PeriodicalIF":2.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12058613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144004901","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":"Engaging patients, family caregivers and healthcare providers to develop metrics tailored to a palliative care population: a content validity process.","authors":"Taylor Shorting, Madeline McCoy, Marianne Weiss, Jessica Schue, Natalie C Ernecoff, Shirley H Bush, Genevieve Lalumière, Jill Rice, Meaghen Hagarty, Daniel Vincent, Kirsten Wentlandt, Daniel David, Krystal Kehoe MacLeod, Vinay Kumar Mysore, Meghan Savigny, Edward Fitzgibbon, Sarina R Isenberg","doi":"10.1186/s41687-025-00885-2","DOIUrl":"https://doi.org/10.1186/s41687-025-00885-2","url":null,"abstract":"<p><strong>Background: </strong>Assessment of patient readiness for hospital discharge has been advocated as an important component of discharge preparation. However, no measures focused on hospital-to-home transitions for patients receiving a palliative approach to care, or the associated difficulties in coping at home after hospital discharge, have been developed to date. Using a co-design approach, the purpose of this study was to (1) adapt two scales to a palliative care population, one of which was developed to assess readiness for the hospital-to-home transition and another developed to assess difficulty in coping post-transition and to (2) test the content validity of both scales from the perspectives of patients, family caregivers, and healthcare providers. The scales chosen for adaptation were the Readiness for Hospital Discharge Scale and Post-Discharge Coping Difficulty Scale.</p><p><strong>Methodology: </strong>The research team made initial adaptations to scale language prior to developing three parallel versions of each scale to be patient-, family caregiver-, and healthcare provider-facing. We conducted content validity testing of the items on both scales by asking each participant group to rate scale items on their usefulness, and to provide suggestions on ways items could be improved. We calculated the Item Content Validity Index and a modified Kappa statistic for each scale item, and calculated the Scale Content Validity Index for each of the three versions of the scales. Refinements were informed by qualitative feedback provided by participants during the content validity process. Final refinements were informed by members of a Patient and Family Advisory Council, and healthcare provider research team members.</p><p><strong>Results: </strong>Moderate modifications were required to the three versions of both scales. Modifications included adding items, modifying item language, and adding examples in parentheses to enhance item context. Patients, family caregivers, and healthcare providers deemed the research team's initial modifications to the scales useful, as evidenced by each scale yielding a Scale Content Validity Index of higher than 0.5.</p><p><strong>Conclusion: </strong>The methodology provided can be used as an example of ways to engage and leverage the experiences of healthcare system users and healthcare providers throughout the outcome measures development process. The next steps will be to utilize the adapted scales as intervention outcome measures in a subsequent implementation study.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"47"},"PeriodicalIF":2.4,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12055745/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001503","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":"Improved quality of life in cystic fibrosis patients observed up to 36 months after starting Elexacaftor/Tezacaftor/Ivacaftor treatment.","authors":"Buniotto Francesca, Tridello Gloria, De Scolari Antonella, Meneghelli Ilaria, Pintani Emily, Perobelli Sandra, Cipolli Marco","doi":"10.1186/s41687-025-00879-0","DOIUrl":"https://doi.org/10.1186/s41687-025-00879-0","url":null,"abstract":"<p><strong>Background: </strong>Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a therapy approved for cystic fibrosis (CF) that has given improved clinical outcomes in patients carrying the F508del mutation. There are few published data regarding ETI's effects on patients' quality of life (QoL). This study aims to (fill the data gap in current literature by assessing) evaluate the long-term effects of ETI on QoL.</p><p><strong>Methodology: </strong>A prospective observational study was conducted with thirty-seven severe patients that received ETI for compassionate use (group A), 184 received it for on-label use (group B). All carried one F508del mutation. Patients were assessed using the CFQ-R (Cystic Fibrosis Questionnaire-Revised). The evaluation time-points were pre-treatment (T0), and after 12 (T1) and 24 months (T2); group A was also assessed after 36 months (T3). Twenty-five patients completed 3 years of treatment and 65 patients completed 2 years of treatment, in groups A and B respectively.</p><p><strong>Results: </strong>At T1, median values for almost all areas of CFQ-R statistically significant increased in group A, particularly Physical Functioning (+ 25.0), Respiratory (+ 22.2) and Health Perception (+ 22.2).The Social Functioning area statistically significant increased at T2 (+ 5.6). At T3, these improvements remained stable. At T1, all areas of CFQ-R statistically significant increased in group B, particularly the Health Perception (+ 22,2) heading. At T2, these improvements remained stable. For both groups, the changes identified at the last follow-up showed no major differences by gender, age or genetic status.</p><p><strong>Conclusions: </strong>Treatment with ETI significantly improved patients' QoL in both groups at 12-24 months, these improvements remaining stable in patients tested at 36 months.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"48"},"PeriodicalIF":2.4,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12055669/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144020036","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}