Journal of Patient-Reported Outcomes最新文献

{"title":"Patient experience with eosinophilic esophagitis symptoms and impacts on daily life based on in-trial qualitative interviews.","authors":"Calvin N Ho, Harald Fjällbrant, Evan S Dellon, Cecilio Santander, Marc E Rothenberg, Julie Bailey, Shehan McFadden, Jason Ritchie","doi":"10.1186/s41687-025-00836-x","DOIUrl":"10.1186/s41687-025-00836-x","url":null,"abstract":"<p><strong>Purpose: </strong>Eosinophilic esophagitis (EoE), a chronic immune-mediated progressive disease, causes dysphagia, food impaction, abdominal pain, vomiting, and heartburn. EoE requires long-term monitoring and can affect quality of life owing to its symptoms and associated emotional and social burden. This study aimed to understand patients' experiences with EoE.</p><p><strong>Methods: </strong>Semi-structured longitudinal interviews were conducted with patients from MESSINA, a phase 3 placebo-controlled trial evaluating benralizumab for EoE. Interviews were held at two different times to assess the impact of EoE on patients' lives before and during trial participation. Data were analyzed qualitatively to develop detailed patient profiles.</p><p><strong>Results: </strong>The MESSINA trial was terminated prematurely. Of the 34 patients recruited for the first interview, 15 (44%) completed the second interview and 11 patient profiles were developed. Patients were a demographically diverse group with varying experiences. The primary reported symptom was difficulty swallowing (n = 11), leading to serious consequences like choking and hospitalization (n = 2). Other symptoms included pain when swallowing (n = 7), reflux (n = 6), and stomachache (n = 6). In the second interview, most (n = 9) patients reported moderate improvements in symptoms, while others experienced symptom recurrence or worsening. EoE had a significant negative impact on social and emotional well-being, and professional lives. Trial participation improved emotional well-being for some; however, concerns about the need for ongoing treatment were noted.</p><p><strong>Conclusion: </strong>This study highlighted emotional and social burdens of EoE. The encouraging feedback on study participation underscores the importance of patient insights in developing holistic management strategies for EoE.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"3"},"PeriodicalIF":2.4,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11711566/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142956251","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":"Clinically meaningful classes of financial toxicity for patients with diabetes.","authors":"Minal R Patel, Jonathan P Troost, Michele Heisler, Noelle E Carlozzi","doi":"10.1186/s41687-024-00834-5","DOIUrl":"https://doi.org/10.1186/s41687-024-00834-5","url":null,"abstract":"<p><strong>Aims: </strong>This study aims to improve the interpretability and clinical utility of the COmprehensive Score for financial Toxicity-Functional Assessment of Chronic Illness Therapy (COST-FACIT) by identifying distinct financial toxicity classes in adults with diabetes.</p><p><strong>Methods: </strong>Data included a sample of 600 adults with Type 1 or Type 2 diabetes and high A1c. Latent Class Analysis was used to identify subgroups of patients based on COST-FACIT score patterns.</p><p><strong>Results: </strong>We identified 3 financial toxicity classes (high, medium and low) with strong indicators of membership classification. Multiple indicators of financial stress, maladaptive cost-coping behaviors, more comorbidities, more prescribed medications, more diabetes distress, more depressive symptoms, closer to the federal poverty level, female, having lower educational attainment and being single were all significant predictors of high financial toxicity class membership. A score of 26 on the COST-FACIT was the strongest threshold for sorting high vs. medium/low financial toxicity, with a positive predictive value (PPV) of 76% and negative predictive value (NPV) of 93%.</p><p><strong>Conclusion: </strong>The COST-FACIT can be used to reliably identify people with diabetes that have high financial toxicity. Integrating this new cut-score into clinical practice may help clinical teams identify people in need of additional support due to financial toxicity.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"2"},"PeriodicalIF":2.4,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11704103/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142956164","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":"Cross-cultural adaptation and validation of Dysphagia Handicap Index in Bangladesh.","authors":"Mehrin Sultana, Md Muid Hossain Reshad, Md Shohidul Islam Mridha","doi":"10.1186/s41687-024-00803-y","DOIUrl":"10.1186/s41687-024-00803-y","url":null,"abstract":"<p><p>The Dysphagia Handicap Index (DHI) is commonly utilized for evaluating how dysphagia impacts the quality of life (QoL) of patients across physical, functional, and emotional dimensions. The primary aim of the research was to linguistically validate and culturally adapt the DHI to the Bangla version. A cross-sectional study design was chosen, with Beaton's protocol as the guiding framework for validating and adapting the DHI. It has followed a systematic process of forward translation, participation in expert discussions, and subsequent back translation to obtain a reviewed version. The Bangla version, DHI-Ban, was administered purposefully to 50 dysphagia patients in the Clinical Speech and Language Therapy (SLT) Department of the Centre for the Rehabilitation of the Paralyzed (CRP) and was also administered to 50 healthy individuals for comparison. Of the fifty, eighteen dysphagia subjects were assigned again after two weeks for the retest. The DHI-Ban demonstrated strong internal consistency (Cronbach's α = 0.89) and good test-retest reproducibility (ICC = 0.86). The Spearman test confirmed significant construct validity (p < 0.01), and the Wilcoxon test identified significant differences (p < 0.001) between patients and healthy individuals. Feedback from participants was also taken into account for acceptance and clarity. In conclusion, the adapted DHI-Ban has emerged to be a reliable patient-reported tool for assessing dysphagia in Bangla-speaking individuals. Incorporating the Bangla language framework facilitates its comprehension and effectiveness, further solidifying its reliability.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"1"},"PeriodicalIF":2.4,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11695556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142923699","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":"Comprehension of the adapted Urticaria Activity Score measure and patient guidance document: qualitative interviews with adults and adolescents with chronic spontaneous urticaria.","authors":"Anne M Skalicky, Yan Wang, Olabimpe R Eseyin, Marissa Stefan, Pallavi B Rane, Julie McLaren, Marcus Maurer","doi":"10.1186/s41687-024-00830-9","DOIUrl":"10.1186/s41687-024-00830-9","url":null,"abstract":"<p><strong>Objective: </strong>A key component of determining that a patient-reported outcome (PRO) measure is fit-for-purpose is to ensure that respondents understand its instructions and items. Any modification to a measure should be evaluated for relevance and understandability. The objective of the study was to assess comprehension of the adapted Urticaria Activity Score (UAS) questionnaire among adolescents aged populations with chronic spontaneous urticaria (CSU) and the modification to UAS question 2 to include patient-friendly terminology \"wheals (hives).\" A patient guidance document for completing the adapted UAS was also examined.</p><p><strong>Methods: </strong>A non-interventional, cross-sectional, qualitative study involving hybrid concept elicitation and cognitive interviews was conducted among adults and adolescents with CSU. Eligibility included clinician confirmation of CSU and experience of itch and hives for ≥ 6 weeks. Study participants were recruited from US clinical sites and online CSU patient communities. Telephone interviews were conducted using a semi-structured interview guide. Participants reviewed the UAS and provided their input on the UAS guidance document.</p><p><strong>Results: </strong>Twenty-two interviews were conducted (seven adolescents and 15 adults; mean age 34 ± 18 years, 64% female, 77% White, 77% non-Hispanic, and 59% moderate to severe CSU symptoms). All participants (n = 22/22, 100%) stated that the adapted UAS was clear and \"easy\" to understand. For the adapted UAS \"wheals (hives)\" item, several participants were unfamiliar with the term \"wheals,\" but the term \"hives\" was well understood by US participants. Most participants reported that it was \"easy\" or \"not difficult\" to count and recall the number of hives they had over the past 24 h. Participants found the adapted UAS guidance document \"helpful\" and \"easy to understand\" for determining and counting hives in a 24-hour period. Suggestions for improving the guide included adding a picture to aid in counting hives.</p><p><strong>Conclusions: </strong>Itch and hives are important symptoms of CSU. Results support the content validity of an adapted UAS as a daily measure of severity of itch and hives and provided valuable suggestions for improving its patient guidance, which can be used in future clinical trials involving adults and adolescents ≥ 12 years old with CSU to assess the severity of itch and hives.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"153"},"PeriodicalIF":2.4,"publicationDate":"2024-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11668714/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883125","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":"Identifying meaningful aspects of health and concepts of interest for assessment in systemic lupus erythematosus: implications for digital clinical measure development.","authors":"Paul Kamudoni, Kate Lyden, Oliver Günther, Vikas Jaitely, Thiago Dantas Araujo, Erica Spies, Josephine Park, Erik Thomas, Joy Buie, Jennifer M Blankenship, Laurent Arnaud","doi":"10.1186/s41687-024-00832-7","DOIUrl":"10.1186/s41687-024-00832-7","url":null,"abstract":"<p><strong>Objectives: </strong>Systemic lupus erythematosus (SLE) is a chronic autoimmune disease with heterogeneous clinical manifestations which significantly impacts the daily lives of patients. Herein, we aimed to (i) investigate patients' perspectives on and experience with SLE; (ii) identify meaningful aspects of health (MAHs) and concepts of interest (COIs) in SLE that could be evaluated using digital clinical measures (DCMs); and (iii) identify target DCMs for their assessment.</p><p><strong>Methods: </strong>A mixed-methods, multistep approach was deployed for (i) exploring patients' experience with SLE through a social media listening study and focused group discussions with patients; (ii) mapping patients' experiences to define MAHs and identify COIs measurable using DCMs; (iii) selecting DCMs for the target COIs; and (iv) identifying types of wearable sensors for measuring COIs in the patients.</p><p><strong>Results: </strong>Six MAHs related to physical behavior and sleep were identified: difficulty in ambulating, lack of energy, inability to perform activities of daily living, difficulty engaging in sustained walking, inability to perform leisure activities and exercise, and lack of restful sleep. Measurable COIs represented walking (fatigue and pain) and sleep (sleep and pain) characteristics. Five and six DCMs related to stepping behavior and sleep quality, respectively, were identified. Several wearable sensors are available to derive DCMs for physical behavior and sleep; however, patients showed a strong preference for a wrist-worn actigraphy sensor.</p><p><strong>Conclusion: </strong>We identified DCMs for physical behavior and sleep that are relevant and meaningful to patients with SLE, measurable in a real-world environment with wearable sensors, and have the potential to aid personalized patient care.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"154"},"PeriodicalIF":2.4,"publicationDate":"2024-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11668707/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883127","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":"Chronic rhinosinusitis with nasal polyps (CRSwNP) symptom verbal response scales: content validity testing for use in adults with CRSwNP.","authors":"Tom Keeley, Nina Gaw, Waseem Ahmed, Rafael Alfonso-Cristancho, Ana R Sousa, Katie Forde, Rosie Sharp, Sophie Whyman, Adam Gater","doi":"10.1186/s41687-024-00827-4","DOIUrl":"10.1186/s41687-024-00827-4","url":null,"abstract":"<p><strong>Background: </strong>PRO measures of symptoms in clinical trials have historically utilized visual and numerical scales but verbal descriptors may make it easier for patients to clearly differentiate between response options. This study assessed content validity and meaningful change in five verbal response scales (VRSs) used to assess chronic rhinosinusitis with nasal polyp (CRSwNP) symptom severity.</p><p><strong>Methodology: </strong>This qualitative, semi-structured interview study recruited adults from the US, Germany, and China with confirmed moderate-to-severe CRSwNP. Interviews included a concept elicitation section, where participants were asked about their experience of living with CRSwNP including symptoms and health-related quality-of-life impacts, and a cognitive debriefing section, where participants were debriefed and participant understanding and real-life relevance of the CRSwNP symptom VRS content were assessed. Interview transcripts were qualitatively analyzed using thematic analysis methods.</p><p><strong>Results: </strong>Among the 24 participants interviewed, the most frequently reported CRSwNP symptoms were nasal obstruction, runny nose, mucus in the throat, loss of smell and facial pain/pressure. Participants demonstrated good understanding of the CRSwNP symptom VRS instructions, items, recall period, and response options. The five CRSwNP symptom VRS items were relevant to the majority of participants' experience of CRSwNP. At the item level, a one-category within-person improvement was the level most frequently reported by participants to be a meaningful change.</p><p><strong>Conclusion: </strong>The CRSwNP symptom VRSs assess relevant and bothersome symptoms experienced by patients with moderate-to-severe CRSwNP, supporting content validity of this measure. The findings of this study provided preliminary insights into meaningful change in the VRS. Further quantitative assessment of meaningful change is needed, and psychometric evaluation of the CRSwNP symptom VRSs will be required to evaluate their appropriateness for assessment of clinical trial endpoints in patients with CRSwNP.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"152"},"PeriodicalIF":2.4,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11662121/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865729","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":"Assessing tolerability with the Functional Assessment of Cancer Therapy item GP5: psychometric evidence from LIBRETTO-531, a phase 3 trial of selpercatinib in medullary thyroid cancer.","authors":"Antoine Regnault, Laurine Bunod, Angely Loubert, Marcia S Brose, Lisa M Hess, Patricia Maeda, Yan Lin, Rebecca M Speck, Adrienne M Gilligan, Nalin Payakachat","doi":"10.1186/s41687-024-00823-8","DOIUrl":"10.1186/s41687-024-00823-8","url":null,"abstract":"<p><strong>Background: </strong>This psychometric analysis generated evidence to support the use of the Functional Assessment of Cancer Therapy item GP5 (GP5) as a measure of tolerability and confirms the appropriateness of categorizing \"high side-effect burden\" using a rating of 3 or 4 (score ranges 0-4) in patients with advanced/metastatic RET-mutant medullary thyroid cancer (MTC).</p><p><strong>Methodology: </strong>Blinded, pooled interim data from the safety population (n=290) enrolled in the phase 3 LIBRETTO-531 trial (NCT04211337) were used. Intraclass correlation coefficients (ICC) were calculated for test-retest reliability using data from cycles 1-2 post-baseline. Construct validity was evaluated by examining the correlations of GP5 ratings with (a) symptomatic adverse events (AEs; measured by the PRO-CTCAE), and (b) functioning scores of EORTC QLQ-C30. The ability to detect change over time was examined by Cochrane-Mantel-Haenszel tests for GP5 ratings and PRO-CTCAE. The relationship of \"high side-effect burden\" categories with QLQ-C30 functioning scores was examined.</p><p><strong>Results: </strong>ICCs for the GP5 ratings after cycle 1 ranged between 0.80 and 0.85, indicating good reliability. Correlations between GP5 and PRO-CTCAE items ranged from 0.18 to 0.62 and ranged from -0.37 to -0.50 for QLQ-C30 functioning scores, consistent with study assumptions. Post-baseline GP5 ratings showed significant associations with PRO-CTCAE scores (p<0.001). Participants with GP5 ratings of 3 or 4 had worse physical function than those with GP5 ratings of 0 to 2 (p<0.0001).</p><p><strong>Conclusions: </strong>This analysis generated evidence supportive of the psychometric properties of the GP5 as a fit-for-purpose measure to assess treatment tolerability in patients with advanced/metastatic MTC. The definition of \"high side-effect burden\" was associated with the clinical feature of tolerability.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"149"},"PeriodicalIF":2.4,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11655800/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855821","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 systematic review of condition-specific preference-based measures used in young people and their valuation methods.","authors":"William King, Lauren Hockley, Tomos Robinson, Angela Bate, Laura Ternent","doi":"10.1186/s41687-024-00826-5","DOIUrl":"10.1186/s41687-024-00826-5","url":null,"abstract":"<p><strong>Background: </strong>Condition-specific health-related quality-of-life (HRQoL) instruments can be more responsive and sensitive to specific conditions and diseases than generic HRQoL instruments. This systematic review aims to identify the condition-specific preference-based instruments that have been used with young people and identify how preference values have been obtained for these instruments. This review will inform future researchers about the methods used to elicit utilities for condition-specific HRQoL instruments.</p><p><strong>Methods: </strong>A comprehensive search strategy was used to identify condition-specific HRQoL instruments used in young people and the methods used to value these instruments. Published medical and health economic databases were searched from January 1990-March 2022. Articles were deemed eligible for inclusion if a condition-specific preference-based instrument was used in young people (age < 18). Screening, data extraction and quality assessment were conducted independently by at least two reviewers.</p><p><strong>Results: </strong>After deduplication, a total of 4273 articles were eligible for title and abstract screening. Of these, 98 articles were eligible for full-text screening. After full-text screening, 18 articles were included in the review. Valuation studies were the most prevalent study design in the review (44%), followed by mapping studies (38%) and then other designs (18%). Among the valuation studies, the choice of HRQoL instrument, preference elicitation method, anchoring method and perspective varied considerably.</p><p><strong>Conclusion: </strong>To our knowledge, this review is the first to explore what condition-specific HRQoL instruments have been used in young people. Findings from this review could inform researchers in their choice of methods for measuring and valuing HRQoL. This review illustrates that to date there does not appear to be clear consensus of how to measure and value HRQoL in young people when using condition-specific instruments. The lack of consensus could be influenced by challenges identified in prior research such as limited guidance, ethical issues, and uncertain normative decisions regarding the choice of preference elicitation method. Ordinal methods such as discrete choice experiment and best-worst scaling appear to be preferable for use in this population.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"151"},"PeriodicalIF":2.4,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11659529/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855818","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":"Impact of eptinezumab on work productivity beyond reductions in monthly migraine days: post hoc analysis of the DELIVER trial.","authors":"Piero Barbanti, Susanne F Awad, Heather Rae-Espinoza, Stephane A Regnier, Xin Ying Lee, Peter J Goadsby","doi":"10.1186/s41687-024-00813-w","DOIUrl":"10.1186/s41687-024-00813-w","url":null,"abstract":"<p><strong>Background: </strong>Eptinezumab's impact on self-reported work productivity in adults with migraine and 2‒4 prior preventive migraine treatment failures is not fully understood.</p><p><strong>Methodology: </strong>Electronic diaries captured monthly migraine days (MMDs) reported by patients enrolled in the randomized, double-blind, placebo-controlled DELIVER trial (NCT04418765). The migraine-specific Work Productivity and Activity Impairment questionnaire, administered at baseline and each monthly visit, was a secondary outcome of DELIVER and used to model changes from baseline in self-reported monthly hours of absenteeism (decreased work attendance) and presenteeism (reduced work efficiency while at work with a migraine) in Canada, as the base case. Path analysis illustrated eptinezumab's impact on work productivity beyond MMDs.</p><p><strong>Results: </strong>As MMDs increased, monthly hours of absenteeism increased linearly while those of presenteeism increased quadratically. Best-fit models were improved after including an eptinezumab treatment effect, showing benefit over placebo after controlling for MMD frequency. Compared to placebo, patients treated with eptinezumab (pooled) had a modeled reduction from baseline of 7.2 h/month (absenteeism) (95% CI: -9.9, -4.5; P < 0.001) and 21.4 h/month (presenteeism) (95% CI: -26.3, -16.5; P < 0.001) over weeks 1‒24. Beyond MMD reductions, improvements in patient-identified most bothersome symptom (PI-MBS) and reductions in percent of severe migraine attacks contributed to eptinezumab's effect.</p><p><strong>Conclusions: </strong>Eptinezumab decreased absenteeism and presenteeism based on patient reports, with data highlighting the importance of considering the PI-MBS. The greater change from baseline than placebo in self-reported absenteeism and presenteeism was only partly explained by changes in MMDs, presenting a potential opportunity to decrease the cost of migraine in the workplace.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov (Identifier: NCT04418765); EudraCT (Identifier: 2019-004497-25).</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"146"},"PeriodicalIF":2.4,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11655748/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142847947","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":"Time to deterioration of patient-reported outcome endpoints in cancer clinical trials: targeted literature review and best practice recommendations.","authors":"Kim Cocks, Bellinda L King-Kallimanis, Joel Sims, Gill Worthy, Julia Stein, Lara Ayala-Nunes, Monika Achra, Zhanglin Lin Cui, Nalin Payakachat","doi":"10.1186/s41687-024-00824-7","DOIUrl":"10.1186/s41687-024-00824-7","url":null,"abstract":"<p><strong>Background: </strong>Time to deterioration (TTD) endpoints are often utilized in the analysis of patient-reported outcome (PRO) data in oncology clinical trials but different endpoint definitions and analysis frameworks exist that can impact result interpretation. This review examined the analysis, reporting and heterogeneity of TTD endpoints in the literature, the impact of analysis methods on results, and provides recommendations for future trials.</p><p><strong>Methods: </strong>A targeted literature review of articles published between 2017 and 2022 was performed to collate TTD endpoints reported in oncology randomized controlled trials (RCTs). Details of endpoints and results were extracted including; deterioration definition, PRO assessment schedule, methods for handling intercurrent events, statistical analysis methods, main trial results (overall survival and/or progression-free survival) and TTD endpoint results.</p><p><strong>Results: </strong>Seventy RCTs were included covering 849 individual TTD endpoints. There were 17 primary cancer types, with lung (26%), breast (11%), and prostate (7%) cancers the most common. Most trials (71%) were for people with advanced cancer. Full definitions of TTD endpoints were often missing. There were no clear trends for a specific TTD definition within cancer types or stages. However, statistical analysis methods were consistent among trials.</p><p><strong>Conclusion: </strong>The TTD definition can vary and is ultimately driven by the research question. Points to consider for successfully implementing PRO TTD endpoints in oncology include consideration of the trial setting (e.g., early vs. advanced cancer), expected treatment effect (e.g., improvement vs. worsening), likely adverse event profile (including early vs. delayed) and PRO data collection frequency in order to improve utility of these endpoints.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"8 1","pages":"150"},"PeriodicalIF":2.4,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11655910/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855834","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}