Journal of Patient-Reported Outcomes最新文献

{"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}

{"title":"Internal consistency, construct validity, and responsiveness of the MRC Prion Disease Rating Scale.","authors":"Leah Leidy, Aaron Yarlas, Robert S Pulido, Jessica Ludwig, Kathleen Glisic, Brian S Appleby","doi":"10.1186/s41687-025-00884-3","DOIUrl":"https://doi.org/10.1186/s41687-025-00884-3","url":null,"abstract":"<p><strong>Background: </strong>The Medical Research Council-Prion Disease Rating Scale (MRC-PDRS) is a 20-point clinician-reported outcome scale to assess disease progression in patients with prion disease, an invariably fatal neurodegenerative disease caused by misfolded prion protein. This study aims to evaluate the measurement properties and interpretability of the MRC-PDRS to support the measure's use for effective disease management and research evaluating effectiveness of treatment options for prion diseases.</p><p><strong>Methodology: </strong>Utilizing patient data from the Telemedicine Assessment Program for CJD (TAPCJD), statistical assessment was conducted of internal consistency, construct validity (including convergent, divergent validity, and known-groups discriminant validity), responsiveness, and interpretation guidelines using distribution-based approaches to estimate thresholds indicating minimal important change (MIC) in MRC-PDRS scores. Criterion measures used for evaluating construct validity and responsiveness included the Telephone Interview for Cognitive Status (TICS) and Neuropsychiatric Inventory-Questionnaire (NPI-Q).</p><p><strong>Results/conclusions: </strong>These findings provide strong preliminary evidence that the MRC-PDRS is reliable, valid, and responsive as a tool for measuring disease progression in patients with prion disease, with preliminary MIC estimates ranging from 1 to 3 points. This supports the use of MRC-PDRS in evaluating potential treatment benefits of prion disease clinical trials, and potentially in clinical practice settings.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"49"},"PeriodicalIF":2.4,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12055732/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144033176","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 patient reported outcome measures assessing tumor pain intensity and tumor pain interference for individuals with neurofibromatosis type 1 and plexiform neurofibromas: qualitative findings.","authors":"Nour Al Ghriwati, Kari Struemph, Staci Martin, Paige Little, Melissa Baker, Jason Levine, Cynthia MacKenzie, James Tonsgard, Elizabeth K Schorry, Karin S Walsh, Pamela L Wolters","doi":"10.1186/s41687-025-00877-2","DOIUrl":"10.1186/s41687-025-00877-2","url":null,"abstract":"<p><strong>Background: </strong>Pain is a common symptom in individuals with neurofibromatosis type 1 (NF1) that often is associated with plexiform neurofibroma (pNF) tumors. To date, no patient-reported outcome measures have been validated specifically to assess pNF-related pain intensity or pain interference in this population. Such measures are sorely needed since pain is being considered as an outcome in clinical trials targeting reduction of pNF. The study aims were to: (1) obtain qualitative information from individuals with NF1 and pNFs about their pain and its measurement and (2) modify existing scales to assess pNF-related pain intensity and pain interference for NF1 clinical trials.</p><p><strong>Methods: </strong>For this multi-site, qualitative study, 56 individuals (26 children, 6-16 years; 30 adults, 18-68 years) with NF1 and pNF participated in a focus group and/or individual interview about pain intensity and pain interference (concept elicitation) and also provided feedback about existing pain measures (Numeric Rating Scale-11 and Pain Interference Index) assessing these domains (cognitive debriefing). Four additional waves of cognitive debriefing interviews further refined the measures. Qualitative concept elicitation data from transcripts were coded, analyzed using NVivo software, and thematic analysis was conducted using both deductive and inductive techniques. Additional themes and systematic problems and suggestions regarding the measures were gleaned from reviewing the field notes and interview transcriptions generated by the cognitive debriefing sessions.</p><p><strong>Results: </strong>Concept elicitation themes included descriptions of two types of pNF-related pain (chronic and episodic), variability of pain over time, varying ability to recall pain, lack of knowledge of pNFs, and the ways pain interferes with daily activities. Cognitive debriefing themes included information on how to rate pNF-related pain intensity apart from other pain; problems and suggestions regarding the measures included difficulty comprehending some items and preferences for alternative wording and formatting. Based on these qualitative results, the measures' instructions, items, and formatting were modified to create the PAin INtensity Scale for plexiform neurofibromas (PAINS-pNF) and the Pain Interference Index for plexiform neurofibromas (PII-pNF) for administration on a mobile app or web-based platform.</p><p><strong>Conclusions: </strong>The PAINS-pNF and PII-pNF are promising self-report measures developed using patient engagement to evaluate tumor pain intensity and pain interference in NF1 clinical trials. The second phase of the study to provide reliability, validity, and normative data for individuals with NF1 and pNFs ages 8 years and older is underway.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"46"},"PeriodicalIF":2.4,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12044096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144051963","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":"Reliability and validity of the Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire impact scales.","authors":"Andrew Lovley, Kristen Hsu, Kaitlin LaGasse, Isabelle Lousada, Kristen L McCausland, Michelle K Carty, Sabrina Rebello, Jakob B Bjorner","doi":"10.1186/s41687-025-00880-7","DOIUrl":"https://doi.org/10.1186/s41687-025-00880-7","url":null,"abstract":"<p><strong>Background: </strong>The diversity of disease phenotypes associated with transthyretin (ATTR) amyloidosis poses challenges for measurement of health outcomes. The Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire is a disease-specific patient-reported outcome (PRO) measure of the symptoms and impacts of ATTR amyloidosis. The objective of this study was to evaluate the structural validity, reliability, and construct validity of the ATTR-QOL Impact scales.</p><p><strong>Methodology: </strong>This was a non-interventional, online survey study of adults with self-reported diagnosis of symptomatic ATTR amyloidosis. The survey included the ATTR-QOL and additional criterion measures. A scoring algorithm was proposed and tested. Factor structure, differential item functioning, and psychometric properties were explored.</p><p><strong>Results: </strong>The analytic sample included 233 patients. Satisfactory fit was found for a 4-factor model of disease impacts (including Daily Activities, Physical Functioning, Social/Role Functioning, and Emotional Wellbeing) and a scoring algorithm was developed. Twelve impact items were dropped from the ATTR-QOLv2 as a result of factor and differential item functioning analyses. Each scale showed evidence of satisfactory internal consistency reliability (Cronbach's α range = 0.85-0.97) and test-retest reliability at 1 week (intraclass coefficient range = 0.84-0.97). Convergent validity was supported by correlations ≥ 0.30 between ATTR-QOL Impact scale scores and other PRO measures of related constructs. The ATTR-QOL Impact scales showed greater impact among patients with worse symptom severity, cardiac functioning, or unemployment due to ATTR amyloidosis (all p < 0.05), supporting known-groups validity.</p><p><strong>Conclusion: </strong>The ATTR-QOL is a reliable and valid measure of impacts meaningful to patients with ATTR amyloidosis. This study resulted in updates to the ATTR-QOL for item reduction and the development of a scoring algorithm. Ongoing studies are collecting data to evaluate the symptom scales of the ATTR-QOL.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"44"},"PeriodicalIF":2.4,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12040782/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144064839","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 modular patient-reported outcome and experience measure on patient needs and benefits in CLL (PBI-CLL).","authors":"Beke Hester, Julia Von Tresckow, Minna Voigtländer, Helen Beckmann, Judith Rusch, Christine Blome","doi":"10.1186/s41687-025-00882-5","DOIUrl":"https://doi.org/10.1186/s41687-025-00882-5","url":null,"abstract":"<p><strong>Background: </strong>Chronic lymphocytic leukaemia (CLL) is the most common form of leukaemia in adults in western countries. Asymptomatic patients are under clinical observation; when indication for treatment according to guidelines is met, treatment is initiated. When choosing from the numerous new treatment options, individual patient needs should be considered. To date, no instrument exists to capture these needs.</p><p><strong>Methodology: </strong>The ePROM was developed based on the Patient Benefit Index (PBI) methodology which captures the importance of treatment goals as well as the achievement of these goals. The development considered the COSMIN guidelines and included semi-structured interviews with 28 patients with CLL and free-text questionnaires (n = 15). Data were analysed via qualitative content analysis according to Kuckartz. The PBI-CLL was finalised through an expert consensus and cognitive debriefing interviews with 14 patients.</p><p><strong>Results: </strong>The content elicitation showed that the individual treatment burden and treatment goals in CLL vary considerably between patients, underlining the heterogeneity of this patient group. Patients reported disease burden in their physical constitution as well as mental burden. Many patients' main goal was to live normally and with the lowest impact possible through the CLL and its therapy. The PBI-CLL developed based on these data consists of three modules: therapy outcomes, process quality and relative treatment preferences. The cognitive debriefing interviews showed that patients find the instrument relevant, comprehensive, and comprehensible.</p><p><strong>Conclusions: </strong>The PBI-CLL is the first instrument to assess patients' needs and benefits in CLL. The heterogeneity we found in patient needs and preferences underlines the importance of a modular instrument which measures treatment goals and benefits in a standardized way. The PBI-CLL shall support both patient-centred therapeutic decision making and treatment evaluation in clinical practice, as well as patient-centred benefit assessment in clinical and health care research. It should therefore be tested for its psychometric properties in a subsequent validation study.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"45"},"PeriodicalIF":2.4,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12040787/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054424","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":"Translation, cultural adaptation and validation of the Danish version of the haematological malignancy patient-reported outcome measure (HM-PRO).","authors":"Sam Salek, Sören Möller, Niels Abildgaard, Tine Rosenberg, Maria Torp Larsen, Peter Asdahl, Kasper Kofod Pedersen, Marie Therese Lassen, Christen Lykkegaard Andersen, Lene Kongsgaard Nielsen","doi":"10.1186/s41687-025-00869-2","DOIUrl":"10.1186/s41687-025-00869-2","url":null,"abstract":"<p><strong>Background: </strong>Assessment of cancer patients´ quality of life (QoL) through patient-reported outcomes (PRO) during and after treatment is gaining ground. The HM-PRO is the first generic Haematological Malignancy specific PRO measure for use in clinical practice and clinical trials. Such generic tools are needed in Denmark. The study aim was to translate and cross-culturally adapt the HM-PRO into Danish and evaluate the psychometric properties.</p><p><strong>Methods: </strong>Translation and cross-cultural adaptation of the original English HM-PRO into Danish followed established guidelines. After cognitive debriefing interviews, it underwent psychometric testing with a variety of hematologic malignancies. Construct validity, internal consistency, dimensionality, item response theory (IRT) and differential item functioning were investigated.</p><p><strong>Results: </strong>295 patients were included for psychometric evaluation; confirmatory factor and bifactor analyses for both HM-PRO parts provided good evidence to support the suggested factor structure (Cronbach's-α Part-A = 0.81, Part-B = 0.84; Part-A CFA CFI = 0.922, TLI = 0.912; bi-factor CFI = 0.989, TLI = 0.978). IRT showed good item-fit and factor loadings and absence of local dependency.</p><p><strong>Conclusion: </strong>The HM-PRO has demonstrated favourable psychometric properties and can be used broadly within the Danish Healthcare system to monitor symptoms as well as QoL impact of patients with haematological cancer and optimize patient engagement during routine cancer care. WHAT IS THE NEW ASPECT OF YOUR WORK?: In response to the intention of the Danish Health Authority to systematically collect PRO data on health-related QoL in Danish cancer patients, this study investigates the translation and cross-cultural adaptation of the original English HM-PRO into Danish. WHAT IS THE CENTRAL FINDING OF YOUR WORK?: Few issues were met with the translation and adaptation of HM-PRO into Danish. WHAT IS (OR COULD BE) THE SPECIFIC CLINICAL RELEVANCE OF YOUR WORK?: The HM-PRO has demonstrated favourable psychometric properties and can be used broadly within the Danish Healthcare system to monitor symptoms as well as QoL impact of patients with haematological cancer and optimize patient engagement during routine cancer care.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"43"},"PeriodicalIF":2.4,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12040771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048419","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":"Content validation of the Angioedema Quality of Life Questionnaire (AE-QoL) in a population of adult and adolescent patients with hereditary angioedema (HAE).","authors":"Lynne Broderick, April Foster, Laura Tesler Waldman, K D Jacobs, Laura Bordone, Aaron Yarlas","doi":"10.1186/s41687-025-00876-3","DOIUrl":"https://doi.org/10.1186/s41687-025-00876-3","url":null,"abstract":"<p><strong>Background: </strong>There is a lack of clear evidence pointing to a fit-for-purpose instrument to measure impacts on health-related quality of life (HRQoL) in adult and adolescent patients with hereditary angioedema (HAE). The purpose of this study was to determine whether the Angioedema Quality of Life Questionnaire (AE-QoL) is content valid and appropriate for capturing the impact of HAE attacks on HRQoL in both adults and adolescents with HAE.</p><p><strong>Methodology: </strong>This study used one-on-one, audio-recorded, cognitive debriefing interviews employing think-aloud and verbal probing approaches to evaluate the relevance, comprehensibility, and comprehensiveness of the AE-QoL in this patient population. All data were quality checked then coded and analyzed using inductive and deductive approaches.</p><p><strong>Results: </strong>This study included 10 adolescents and 12 adults with HAE in the United States. Overall, participants had positive impressions of the AE-QoL, finding the length and recall period appropriate, and the response options clear and easy to understand. Some reported minor concerns with the instructions, but none that prevented them from completing the instrument. Participants found the instrument relevant to their experiences with HAE, noting that items that were not personally relevant were still important to ask. Overall, participants found the AE-QoL comprehensible and comprehensive, although some participants, primarily adolescents, reported being unfamiliar with the word \"leisure,\" making it difficult to answer the item asking about impact of attacks on \"leisure time.\" Adolescents also reported that questions about school-related impacts were missing and would be important to ask about specifically.</p><p><strong>Conclusions: </strong>This study presents evidence that supports the content validity of the AE-QoL in adult and adolescent patients with HAE. While revisions could be considered prior to using the instrument with samples of adolescent patients with HAE, in general, adolescents and adults with HAE found the measure relevant, comprehensive, and comprehensible.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"42"},"PeriodicalIF":2.4,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11993509/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989383","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-reported 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}