Journal of Patient-Reported Outcomes最新文献

{"title":"Patient-reported outcome assessment of adults and adolescents with atopic dermatitis: a cross-sectional qualitative interview study.","authors":"Parima Ghafoori, Dharm S Patel, Kimberly Raymond, Elizabeth Brennan, April Mitchell Foster, Kristi Jackson, Helen J Birch, Wen-Hung Chen","doi":"10.1186/s41687-025-00871-8","DOIUrl":"https://doi.org/10.1186/s41687-025-00871-8","url":null,"abstract":"<p><strong>Background: </strong>Atopic dermatitis (AD) is a chronic inflammatory skin disease that impacts patient health and quality of life. Understanding patient experience of relevant symptoms and impacts of AD is crucial for improving outcomes. This study aimed to characterise adult (≥ 18 years) and adolescent (12-17 years) patients' experiences of AD and assess the content validity of selected patient-reported outcomes (PROs).</p><p><strong>Methodology: </strong>This non-interventional, cross-sectional, qualitative study recruited US-based, English-speaking adults and adolescents with moderate-to-severe AD, either naïve or experienced with biologics. A 90-minute interview was conducted via teleconferencing software, consisting of concept elicitation (CE) of AD experiences and cognitive debriefing (CD), where participants provided feedback on PROs assessing skin pain, sleep disturbance, and fatigue. Interview data were coded and analysed using qualitative data software to determine the AD experience and content validity of selected PROs. A conceptual disease model was developed from the CE portion of the interview. Results from the CD portion were mapped to this model to evaluate the conceptual coverage of the PROs.</p><p><strong>Results: </strong>In total, 16 adults (mean age 48 years, 56% White, 63% female, 50% biologic naïve) and 20 adolescents (mean age 16 years, 60% White, 75% female, 50% biologic naïve) were included in the analysis. During CE, 13 symptoms and impacts in 7 domains were identified. The most reported symptom was itchiness (adults, 100%; adolescents, 100%) and the most reported impact was emotional functioning (adults, 94%; adolescents 95%). Participants also commonly reported experiencing pain/discomfort (adults, 69%; adolescents, 80%) and sleep disturbance (adults, 88%; adolescents, 75%). Fatigue was reported by 94% of adults across CE and CD segments. When probed during CE, 65% of adolescents identified fatigue as an impact of AD. During CD, 70-100% of participants confirmed the selected PROs were comprehensible and relevant.</p><p><strong>Conclusions: </strong>This study provides evidence that the experience of AD is similar between adults and adolescents as well as biologic-naïve and biologic-experienced participants. Relevant disease concepts in patients with AD were identified, and content validity was established for the selected PROs, supporting their use in future clinical studies.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"41"},"PeriodicalIF":2.4,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015432","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":"Expert consensus on implementing patient-reported outcomes in telehealth: findings from an international Delphi study.","authors":"Elizabeth J Unni, Liv Marit Valen Schougaard, Olalekan Lee Aiyegbusi, Kedar K V Mate, Elizabeth J Austin, Klara Greffin, Natasha Roberts, Birgith Engelst Grove, Holger Muehlan","doi":"10.1186/s41687-025-00872-7","DOIUrl":"https://doi.org/10.1186/s41687-025-00872-7","url":null,"abstract":"<p><strong>Background: </strong>Using Patient Reported Outcomes (PROs) in clinical care can reduce healthcare service utilization by improving the quality of care. Telehealth, defined by WHO, as the use of \"telecommunications and virtual technology to deliver healthcare outside of traditional healthcare facilities\", can facilitate a dynamic dialogue between patients and healthcare providers for timely interventions. With the increased use of telehealth facilitated by the infrastructure development during the COVID-19 pandemic, there is an opportunity to utilize telehealth for PRO implementation and a need for guidelines for using PROs via telehealth. This study aimed to generate expert consensus on the utilization of PROs in telehealth.</p><p><strong>Methods: </strong>Delphi methodology was used to achieve consensus among international experts with a predetermined consensus threshold of 70%. Experts were mainly identified through the ISOQOL Clinical Practice SIG. Surveys asked a combination of structured and open-ended questions about the conceptualization of PROs in telehealth, its applicability, target population, implementation challenges and successful strategies, evaluation approaches, and the essential stakeholders. Data from each round were iteratively analyzed using descriptive statistics (quantitative data) and content analysis (qualitative data).</p><p><strong>Results: </strong>Out of 24 invitations sent, 17 completed the first round, and 11 completed all three rounds. Respondents were equally distributed between clinicians and researchers and 70% had used PROs via telehealth before the pandemic. Consensus was achieved and some of the relevant aspects are monitoring patients for applicability; individuals with chronic diseases as the target population; resources, staff buy-in, and clinical workflow as the implementation challenges and strategies; utilization metrics for evaluation; and clinicians and patients as essential stakeholders. Though consensus was not reached for the conceptualization of PROs using telehealth, the modified FDA definition of telehealth with the addition of its purpose, and the mode of administration was the most acceptable version. See attached table.</p><p><strong>Conclusion: </strong>The expert consensus achieved provides important insights from an international perspective on how PROs are currently used via telehealth and the needed implementation support to advance their expansion in research and practice. Lack of consensus on the definition of PROs in telehealth signals the continued rapid evolution of their use and the need for additional research.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"40"},"PeriodicalIF":2.4,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11981999/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143999993","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 in patients with unresectable hepatocellular carcinoma treated with SIRT and nivolumab: a sub-analysis of the NASIR-HCC trial.","authors":"Manuel De la Torre-Aláez, Ana Matilla, María Varela, Mercedes Iñarrairaegui, María Reig, José Luis Lledó, Juan Ignacio Arenas, Sara Lorente, Milagros Testillano, Laura Márquez, Gemma Iserte, Josepmaria Argemí, Carlos Gómez-Martin, Macarena Rodríguez-Fraile, José I Bilbao, Richard F Pollock, Johannes Pöhlmann, Ion Agirrezabal, Bruno Sangro","doi":"10.1186/s41687-025-00873-6","DOIUrl":"10.1186/s41687-025-00873-6","url":null,"abstract":"<p><strong>Background: </strong>The health-related quality of life (HRQoL) impact of therapies for hepatocellular carcinoma (HCC) influences decision-making and treatment outcomes. The present study reports HRQoL results from NASIR-HCC, a single-arm study of selective internal radiation therapy (SIRT) with Y90 resin microspheres followed by nivolumab for unresectable HCC.</p><p><strong>Methodology: </strong>Participants completed the EQ-5D-3 L, EQ-VAS, and FACT-Hep at baseline and on the first day of each nivolumab cycle. Linear mixed-effect models were used to calculate changes in outcomes in participants with the baseline and ≥ 1 follow-up measurement. Changes were assessed for clinical meaningfulness versus published minimally important differences.</p><p><strong>Results: </strong>Thirty-two patients from NASIR-HCC were included. Completion rates exceeded 70% at 62% of time points. Across EQ-5D-3 L domains, minimal changes were reported. Most patients had no problems at almost all time points. Mean index values were 0.864 at baseline and 0.763 in cycle 8, but this difference was not clinically meaningful. The small EQ-VAS increase, from 74.8 at baseline to 75.9 in cycle 8, was also not clinically meaningful. The various FACT scales remained stable, although transient but not clinically meaningful declines occurred for some scales. The median time to deterioration was 5.5 months for the FACT-Hep score.</p><p><strong>Conclusions: </strong>Combining SIRT with nivolumab did not compromise HRQoL in patients with unresectable HCC. Study results were limited by the small number of patients but, combined with the previously reported clinical outcomes, suggested that the treatment combination deserves further consideration in this difficult-to-treat population.</p><p><strong>Trial registration number/date of registration: </strong>NCT03380130. First submitted on 2017-10-20; https://clinicaltrials.gov/study/NCT03380130 .</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"39"},"PeriodicalIF":2.4,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11978598/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143812520","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":"Normative values for the hypoparathyroidism patient questionnaire (HPQ28) in the German general population.","authors":"Christian Trummer, Martina Blaschke, Deborah Quint, Maxi Schulz, Christoph Herrmann-Lingen, Matthias Büttner, Heide Siggelkow","doi":"10.1186/s41687-025-00868-3","DOIUrl":"10.1186/s41687-025-00868-3","url":null,"abstract":"<p><strong>Background: </strong>Patients with hypoparathyroidism (HypoPT) suffer from several complaints and reduced quality of life (QoL), even if disease-specific biochemical parameters are within the target range. To be able to quantify symptoms in HypoPT patients, we recently developed a disease-specific questionnaire, the Hypoparathyroidism Patient Questionnaire with 28 items (HPQ28). The aim of this study was to find normative values for the HPQ28 in the German general population.</p><p><strong>Methods: </strong>We tasked an independent market and social research institute to obtain sociodemographic data and HPQ28 results from a representative sample of the German general population. The HPQ28 comprises five scales and three single items. The five scales indicate different areas of complaints: Pain and cramps (PaC) including five items, neurovegetative symptoms (NVS) including five items, loss of vitality (LoV) including six items, depression and anxiety (DaA) including five items, gastro-intestinal symptoms (GiS) including two items and two control items for depression. Three items were not attributable to any of the five scales: numbness and tingling in certain parts of the body (NT), troubled memory (TM), and racing heart (RH).</p><p><strong>Results: </strong>Mean age (± standard deviation) in the representative general population sample (n = 2506) was 49.5 ± 17.8 years, 51% were female. All scales and single items were affected by gender with women presenting significantly more complaints on every scale and single item in comparison to men (p < 0.01, Mann-Whitney U test). In addition, all scales and single items, except for GiS, were affected by age in males and females (p < 0.001, Spearman's correlation). Regression analyses proved a linear trend in the different scores regarding age and gender (p < 0.05 except for age on the GiS scale).</p><p><strong>Conclusions: </strong>We present data from the first application of the HPQ28 in a representative sample of the German general population. Almost all scales and single item of the HPQ28 were dependent on age and gender, with older individuals and females presenting a higher burden of complaints.</p><p><strong>Trial registration: </strong>DRKS, DRKS00027581. Registered 17th of January 2022, https//drks.de/search/de/trial/DRKS00027581.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"38"},"PeriodicalIF":2.4,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11968587/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143774493","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":"Discrepancies in perceptions of well-being: comparing parental and pediatric PROMIS-patient-reported outcomes in Crohn's disease.","authors":"Sara Azevedo, Maria Miguel Oliveira, Paulo Jorge Nogueira, Ana Isabel Lopes","doi":"10.1186/s41687-025-00870-9","DOIUrl":"10.1186/s41687-025-00870-9","url":null,"abstract":"<p><strong>Background: </strong>This study aims to evaluate and compare the perspectives of pediatric Crohn's disease (CD) patients and their parents/caregivers concerning global physical, emotional, and social health as well as health-related quality of life (HRQQL), using both the Patient-Reported Outcomes Measurement Information System (PROMIS) and the IMPACT III questionnaire.</p><p><strong>Methods: </strong>In a cross-sectional study, 31 dyads of pediatric CD patients (aged 8-17 years) and their parents/caregivers were recruited from an outpatient Pediatric Gastroenterology Center. Participants completed PROMIS (Global Health, Depressive Symptoms, Anxiety, Meaning and Purpose Pain Interference Life Satisfaction, Peer Relationships, Physical Activity and Fatigue) and IMPACT III measures. Comparative analyses using t-tests and multivariate analyses assessed the impact of demographic factors on score differences. Cohen's Kappa analysis evaluated the alignment between parent and child perceptions of disease status.</p><p><strong>Results: </strong>The sample comprised 58% females with a mean age of 15.2 (± 2) years and a mean disease duration of 2.7 (± 2.7) years. Most patients were in disease remission (83.9%) and perceived their disease as better or unchanged in the past 6 months. Concerning PROMIS scores, parents reported significantly lower global health scores (p < 0.001) and higher meaning and purpose scores (p = 0.029) compared to their children. Parental education and professional status significantly influenced PROMIS score differences. Specifically, mothers with specialized professions showed smaller differences in PROMIS depression and pain interference, although greater differences in PROMIS meaning and purpose, as compared to their respective children's scores. Fathers with specialized professions demonstrated greater differences in PROMIS anxiety scores but smaller differences in PROMIS life satisfaction scores. A significant misalignment between parent and child subjective perceptions of disease status was observed (p = 0.004), suggesting that parents may overestimate symptom severity or underestimate improvements compared to their children's experiences.</p><p><strong>Conclusion: </strong>This study highlights the importance of integrating patient and parental perspectives in the clinical management of pediatric CD. The observed discrepancies in disease-related perceptions, influenced by parental educational and professional background, underscore the need for comprehensive assessments to ensure accurate, patient-centered care. For broader generalization, further research should explore these dynamics in newly diagnosed and hospitalized patients.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"37"},"PeriodicalIF":2.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11953492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143744283","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and psychometric evaluation of a patient-reported symptom index for patients with non-muscle invasive bladder cancer: the NMIBC-SI.","authors":"Claudia Rutherford, Margaret-Ann Tait, Daniel S J Costa, Madeleine T King, David P Smith, Shomik Sengupta, Joseph Ischia, Andrew Mitterdorfer, Dickon Hayne, Roger Watson, Paul Anderson, Mark Frydenberg, Peter Gilling, Nicholas Buchan, Euan Green, Noel Clarke, Stephen A Boorjian, Badrinath Konety, Jeffrey M Holzbeierlein, Peter C Black, Venu Chalasani, Jörg Henseler, Manish I Patel","doi":"10.1186/s41687-025-00864-7","DOIUrl":"10.1186/s41687-025-00864-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring frequent follow-up with endoscopic examinations, tumour resections and intravesical treatments. In this clinical context, patient-reported outcomes (PROs) have enormous potential to inform treatment assessment and recommendations for NMIBC. We aimed to develop and evaluate a patient-reported NMIBC Symptom Index (NMIBC-SI) to facilitate clinical research and enhance care.</p><p><strong>Methods: </strong>NMIBC-SI items were developed based on existing literature and qualitative interviews with patients and clinicians, and evaluated in two field tests: item reduction, using NMIBC-SI data from 220 patients on active treatment from nine Australian centres; reliability and validity evaluation of item-reduced version using NMIBC-SI data from 232 patients from five countries.</p><p><strong>Results: </strong>NMIBC-SI assesses disease and treatment-related symptom burden and two treatment-specific side-effects (cystoscopy, intravesical BCG/Chemotherapy). Composite analysis supported a single composite model including core symptom and cystoscopy index items (Intravesical index items were not tested due to small sample). Test-retest reliability was strong (range 0.894-0.91). As expected, the NMIBC-SI was able to discriminate between no treatment and any treatment groups, and no treatment and chemo/BCG groups, providing evidence towards validity.</p><p><strong>Conclusions and clinical implications: </strong>NMIBC-SI assesses patients' self-reported symptom burden and can be used to evaluate NMIBC treatments from the perspective of patients. The NMIBC-SI is acceptable to patients and has evidence for reliability and validity. Future validation work with patients with greater symptom burden is warranted.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"36"},"PeriodicalIF":2.4,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143721709","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' acceptability of a patient-reported outcome measure in cardiac rehabilitation (the PRO-Heart-DK)-a mixed methods study using the Theoretical Framework of Acceptability.","authors":"Emma Dedic, Heidi Sønderby Vistisen, Ann-Dorthe Zwisler, Bente Faurby Pedersen, Karin Lundsby Kappel, Helle Kanstrup, Rikke Elmose Mols, Cecilie Lindström Egholm","doi":"10.1186/s41687-024-00831-8","DOIUrl":"10.1186/s41687-024-00831-8","url":null,"abstract":"<p><strong>Background: </strong>The integration of Patient Reported Outcome Measures (PROM) in cardiac rehabilitation practice has potential to enhance patient involvement and management. User acceptance is crucial for successful implementation of healthcare interventions, but limited literature addresses PROM acceptability among cardiovascular patients. This study explored the acceptability of a new national PROM in cardiac rehabilitation clinical practice for patients with ischemic heart disease (IHD) in Denmark.</p><p><strong>Methods: </strong>Patients who responded to the PROM were invited to complete two brief surveys evaluating perceived relevance, usefulness, and satisfaction. A purposefully selected subsample participated in semi structured interviews to gather in-depth experiences. A parallel convergent mixed-methods design was used with the Theoretical Framework of Acceptability applied to structure and interpret findings.</p><p><strong>Results: </strong>105 and 119 patients respectively responded to the two evaluation surveys (response-rates 56.5% and 53.4% respectively) and 25 patients were interviewed. The study showed a strong willingness to engage with PROMs, indicating a high overall acceptability. Most patients perceived the PROM helpful for preparation and enhancing communication during consultations. A minority of patients reported emotional reactions and experienced issues with questionnaire comprehensiveness, structure, and relevance.</p><p><strong>Conclusion: </strong>The findings indicate that most IHD patients find PROM relevant and useful in cardiac rehabilitation. To enhance acceptability and, hence, future implementation, improvements are needed in clinical settings by providing adequate patient information, effectively using PRO results, and addressing patients' emotional reactions. Additionally, PROM development should focus on ensuring the questionnaire's relevance, comprehensiveness, and structure.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"35"},"PeriodicalIF":2.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11937467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143711558","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":"A new generation of patient-reported outcome measures with large language models.","authors":"Jan Henrik Terheyden, Maren Pielka, Tobias Schneider, Frank G Holz, Rafet Sifa","doi":"10.1186/s41687-025-00867-4","DOIUrl":"10.1186/s41687-025-00867-4","url":null,"abstract":"<p><strong>Background: </strong>Patient-reported outcome measures (PROMs) are cornerstones of patient-centered clinical medicine and reflect patients' abilities, difficulties, perceptions and behaviors. The highly structured questionnaire format of PROMs currently limits their real-world validity and acceptability to patients, which becomes increasingly relevant with the high clinical interest in PROM data. In this short commentary, we aim to demonstrate the potential use of large language models (LLMs) in the context of PROM data collection and interpretation.</p><p><strong>Main body: </strong>The popularization of LLMs enables the development of a new generation of PROMs generated and administered through digital technology that interact with patients and score their responses in real time based on artificial intelligence. LLM-PROMs will need to be developed with multi-stakeholder input and careful validation against established PROMs. LLM-PROMs could complement traditional PROMs particularly in real-world clinical applications.</p><p><strong>Conclusion: </strong>LLM-PROMs could allow quantifying patient-relevant dimensions based on less structured contents and foster the use of patient-reported data in digital, clinical applications of PROMs.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"34"},"PeriodicalIF":2.4,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11933620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143701730","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":"Interpretation of clinically meaningful change in cancer palliative care patients' quality of life: minimally important difference for EORTC QLQ-C15-PAL.","authors":"Kikuko Miyazaki, Yoshimi Suzukamo, Masayuki Ikenaga, Shozo Ohsumi, Mari Saito, Eriko Satomi, Kojiro Shimozuma, Takeo Nakayama","doi":"10.1186/s41687-025-00858-5","DOIUrl":"10.1186/s41687-025-00858-5","url":null,"abstract":"<p><strong>Background: </strong>Palliative care for cancer helps improve and maintain patients' quality of life (QOL). Clinically meaningful changes in QOL measures are helpful when considering how a patient would want to spend the final days of their life. This study aimed to estimate the minimally important differences (MIDs) for within-person change for the European Organisation for Research and Treatment of Cancer QOL Questionnaire Core 15 Palliative Care (EORTC QLQ-C15-PAL) domains in advanced cancer patients in palliative care.</p><p><strong>Method: </strong>Participants in this multicenter observational study comprised patients with advanced cancer receiving palliative care in the last year before death. The EORTC QLQ-C15-PAL was administered at two-week intervals. During the second assessment, patients also completed the Global Rating of Change (GRC) scale to collect their subjective assessments of changes in their condition since the first assessment. MID for QOL score with a correlation of 0.3 or more with GRC score changes were estimated using anchor- and distribution-based methods.</p><p><strong>Results: </strong>Among the 257 screened patients at 13 facilities, we analyzed 181 (92 male; mean age: 67). The mean survival time was 131 days. Notably, the number of patients who responded \"no change\" for the GRC items was large (63-128). Anchor-based MIDs differed depending on the change direction (improvement vs. deterioration). The MIDs for meaningful within-person change may be used in clinical practice.</p><p><strong>Conclusion: </strong>We estimated the MIDs in EORTC QLQ-C15-PAL in patients with advanced cancer with a life expectancy of less than one year, both anchor- and distribution-based.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"33"},"PeriodicalIF":2.4,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11923346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664049","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 Chronic Ocular Pain Questionnaire (COP-Q).","authors":"Amy Findley, Brigitte J Sloesen, Nicola Hodson, Agkreta Leventi, Ben Pascoe, Rob Arbuckle, Paul O'Brien, Christel Naujoks, Michela Montecchi-Palmer, Diana Plaza, Paul M Karpecki, Pedram Hamrah","doi":"10.1186/s41687-025-00862-9","DOIUrl":"10.1186/s41687-025-00862-9","url":null,"abstract":"<p><strong>Background: </strong>The Chronic Ocular Pain Questionnaire (COP-Q) is a newly developed patient-reported outcome (PRO) measure intended to assess symptoms and impacts associated with Chronic Ocular Surface Pain (COSP). This study assessed the psychometric properties of the COP-Q to determine the adequacy of the COP-Q as a 'fit-for-purpose' instrument to derive trial endpoints for future clinical studies in COSP.</p><p><strong>Methods: </strong>Patients with COSP completed the COP-Q daily for four weeks on an electronic, touch-screen, tablet device as part of a longitudinal, observational study in the United States (N = 124). Analyses were conducted to assess item properties, dimensionality and scoring, reliability and validity, and interpretation of scores. In addition, 4-hour and 24-hour recall period versions of the COP-Q Symptom Module were compared.</p><p><strong>Results: </strong>Item responses were distributed across the full response scale for most COP-Q items. Inter-item correlations did not identify any redundant items (r > 0.90) and all items correlated at > 0.40 in their respective module. Confirmatory factor analysis (CFA) provided acceptable support for the unidimensional structure of the multi-item scales in the COP-Q and calculation of a total score for each module. However, CFA and Rasch analysis outlined potential redundant items for the COP-Q Visual Tasking Module (VTM), which were removed, resulting in a six-item VTM. The multi-item COP-Q modules had excellent internal consistency (α range = 0.91-0.96) and suggested fair to excellent test-retest reliability (ICC/Kappa range = 0.651-0.940) for all COP-Q modules. Construct validity for all COP-Q modules was supported by a logical pattern of correlations with concurrent measures and evidence of ability to distinguish between known-groups, with statistically significant differences between COSP severity groups. Paired t-tests, coefficient of determination (CoD) and concordance correlation coefficients (CCC) showed statistically significant differences between the two recall period versions of the Symptom Module, although the magnitude of the difference was small, and each version shares a high level of reproducibility in scores.</p><p><strong>Conclusions: </strong>Findings provide evidence that the COP-Q is a valid and reliable measure of patient-reported COSP symptoms and impacts for use in future clinical trials in COSP. Both 4-hour and 24-hour Symptom Module recall period versions are likely to yield consistent results and are equally robust.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"32"},"PeriodicalIF":2.4,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11903982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143617459","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}