Journal of Patient-Reported Outcomes最新文献

{"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}

{"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}