Patient-Patient Centered Outcomes Research最新文献

{"title":"Engaging Underserved Populations in Health Preference Research: Challenges and Strategies.","authors":"Paula Sinead Donnelly, Divya Mohan, Hannah Penton, Emily Wilson, Marco Boeri","doi":"10.1007/s40271-025-00745-7","DOIUrl":"10.1007/s40271-025-00745-7","url":null,"abstract":"<p><p>Health preference research plays a critical role in shaping healthcare policy and decision-making; however the underrepresentation of underserved populations challenges the validity and reliability of preference estimates. Despite efforts to diversify recruitment, health preference studies often have limited demographic diversity and non-representative sampling, leading to potentially biased findings that overlook the preferences of underserved populations. We discuss the importance of engaging underserved populations in health preference research from both ethical and research perspectives. We identify key challenges to the inclusion of underserved groups and outline strategies to address them, illustrating these with examples where possible. By prioritising inclusive and flexible methodologies, health preference researchers can generate more representative data, ensuring that estimates reflect the diverse needs and values of all populations. Ultimately, these efforts will support the development of more equitable, evidence-based, and impactful healthcare policies.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"443-459"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144200768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"16th Meeting of the International Academy of Health Preference Research.","authors":"","doi":"10.1007/s40271-025-00758-2","DOIUrl":"10.1007/s40271-025-00758-2","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"563-583"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144876644","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Determining the Content Validity of the EQ-5D-5L, EQ-5D-Y-3L, and CHU9D Instruments for Assessing Generic Child and Adolescent Health-Related Quality of Life: A Qualitative Study.","authors":"Jill Carlton, Philip A Powell, Andrew Kirkcaldy, Donna Rowen","doi":"10.1007/s40271-025-00743-9","DOIUrl":"10.1007/s40271-025-00743-9","url":null,"abstract":"<p><strong>Background: </strong>Health technology assessment agencies typically recommend generic measures of health to generate quality-adjusted life-years. Most agencies provide recommendations on which measure to use for adults, whereas few make recommendations for children. Two widely used preference-weighted measures of child and adolescent health that have evidence of good psychometric performance are the EQ-5D-Y-3L and the Child Health Utility 9D Index (CHU9D). The EQ-5D-5L has also been used to assess adolescent health. However, evidence on their content validity-a core measurement property-is limited. The objective of this study was to explore the content validity of the EQ-5D-5L, EQ-5D-Y-3L, and CHU9D measures, including their relevance, comprehensiveness, and comprehensibility.</p><p><strong>Methods: </strong>We assessed the content validity of the EQ-5D-5L, EQ-5D-Y-3L, and CHU9D using online semi-structured cognitive interviews in the UK. Participants were asked to comment on the relevance, comprehensibility, and comprehensiveness of the measures, including response options, recall period, and completion instructions. Interviews were informed by a topic guide. Purposive sampling allowed for appropriate breadth in the sample, with variation in gender, and presence of health conditions, disease, or disability. Interviews were recorded and transcribed verbatim before thematic content analysis.</p><p><strong>Results: </strong>In total, we conducted 49 interviews between August 2022 and June 2023: 21 children/adolescents aged 8-17 years and 28 parents/guardians of children aged 4-17 years. The mean duration of the interviews was 45 min. Relevance was broadly supported, but issues were identified. Comprehensibility was inconsistent on some items, and participants expressed difficulty with grouped items (e.g., 'anxiety/depression'). Participants had difficulty distinguishing qualitatively between some response options (e.g., 'a little bit/a bit'). Some participants noted that instrument comprehensiveness was insufficient.</p><p><strong>Conclusions: </strong>Although the content of the EQ-5D-5L, EQ-5D-Y-3L, and CHU9D was broadly supported, potential problems were identified in aspects of comprehensibility, relevance, and comprehensiveness. These present opportunities for future research and refinement to ultimately improve the content validity of these measures for assessing child and adolescent health.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"523-537"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408752/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144081756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"What Can Discrete-Choice Experiments Tell Us about Patient Preferences? An Introduction to Quantitative Analysis of Choice Data.","authors":"F Reed Johnson, Wiktor Adamowicz, Catharina Groothuis-Oudshoorn","doi":"10.1007/s40271-024-00705-7","DOIUrl":"10.1007/s40271-024-00705-7","url":null,"abstract":"<p><p>This paper provides an introduction to statistical analysis of choice data using example data from a simple discrete-choice experiment (DCE). It describes the layout of the analysis dataset, types of variables contained in the dataset, and how to identify response patterns in the data indicating data quality. Model-specification options include linear models with continuous attribute levels and non-linear continuous and categorical attribute levels. Advantages and disadvantages of conditional logit, mixed logit, and latent-class analysis are discussed and illustrated using the example DCE data. Readers are provided with links to various software programs for analyzing choice data. References are provided on topics for which there currently is limited consensus and on more advanced techniques to guide readers interested in exploring choice-modeling challenges in greater depth. Supplementary materials include the simulated example data used to illustrate modeling approaches, together with R and Matlab code to reproduce the estimates shown.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"425-440"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141762488","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: What Can Discrete‑Choice Experiments Tell Us about Patient Preferences? An Introduction to Quantitative Analysis of Choice Data.","authors":"F Reed Johnson, Wiktor Adamowicz, Catharina Groothuis-Oudshoorn","doi":"10.1007/s40271-024-00712-8","DOIUrl":"10.1007/s40271-024-00712-8","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"441-442"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"What Constitutes High-Quality Paediatric Palliative Care? A Qualitative Exploration of the Perspectives of Children, Young People, and Parents.","authors":"Debbie Braybrook, Lucy Coombes, Hannah M Scott, Daney Harðardóttir, Anna Roach, Jemimah Bariuan, Clare Ellis-Smith, Julia Downing, Fliss E M Murtagh, Myra Bluebond-Langner, Lorna K Fraser, Richard Harding, Katherine Bristowe","doi":"10.1007/s40271-025-00744-8","DOIUrl":"10.1007/s40271-025-00744-8","url":null,"abstract":"<p><strong>Background: </strong>Globally, over 21 million children need palliative care each year. Although guidelines exist to support paediatric palliative care delivery, they are not informed by the experiences of children themselves.</p><p><strong>Objective: </strong>We aimed to determine what constitutes good quality palliative care from the perspectives of children with life-limiting or life-threatening conditions and their parents.</p><p><strong>Methods: </strong>We analysed semi-structured qualitative interviews using reflexive thematic analysis informed by the European Association for Palliative Care charter of palliative care for children and young people, and Bronfenbrenner's bioecological model. Participants included 26 children aged 5-17 years, and 40 parents of children aged 0-17 years, with a range of cancer and non-cancer diagnoses in nine UK paediatric palliative care services (hospitals and hospices).</p><p><strong>Results: </strong>Quality paediatric palliative care can be both enacted or interrupted across the five domains of the bioecological model. Honest timely communication with the child and family (microsystem), and collaborative relationships between care teams and others in the child's life (mesosystem), are vital. Care experiences are negatively affected by inequities in care provision (exosystems), and society's reluctance to discuss mortality in childhood (macrosystem). Children need to enjoy what matters to them, and maintain social connections, and plan for the future, even if facing a shortened life (chronosystem).</p><p><strong>Conclusions: </strong>Children and parents are experts in their condition and should be actively involved in care discussions, through communication tailored to the child's pace and preferences, and support advocating for and coordinating care services. Fostering strong and collaborative relationships builds trust and helps children and families to feel safe, included and supported.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"539-561"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144144158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Measuring Fatigue in Multiple Sclerosis: A Rapid Review.","authors":"Tergel Namsrai, Anne Parkinson, Richard Burns, Geoffrey Herkes, Mark Elisha, Katrina Chisholm, Janet Drew, Vanessa Fanning, Anne Brüstle, Hanna Suominen, Nicolas Cherbuin, Jane Desborough","doi":"10.1007/s40271-025-00759-1","DOIUrl":"https://doi.org/10.1007/s40271-025-00759-1","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Fatigue is one of the most prevalent and debilitating symptoms of multiple sclerosis (MS), as people with MS describe it. It has a complex pathogenesis and often precedes the clinical symptoms of MS and potentially indicates disease progression. Given its prevalence, impact, and intricate connections to disease pathology, accurate measurement is crucial to manage and study fatigue in people with MS; however, current measurements often lack content validity. A mismatch between key aspects of fatigue and existing fatigue scales will limit these scales' ability to capture the full scope of MS-related fatigue. We aimed to examine the current evidence on MS-related fatigue to define key aspects of fatigue in the literature and compare them with the scales used to measure MS-related fatigue.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This integrated rapid review (PROSPERO registration: CRD42024505743) synthesised evidence on MS-related fatigue domains and their representation in validated scales. A systematic search was conducted on January 24, 2024, across three electronic databases: PubMed, Scopus, and ProQuest with no restriction on publication date. Eligible studies included those reporting on fatigue domains, signs and symptoms in people with MS and those on validated fatigue scales in MS. The quality of the included studies was assessed using the Mixed Methods Assessment Tool. Data was synthesised with meta-aggregation of the fatigue domains, signs, and symptoms and mapping them against the items from validated fatigue scales.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;We identified 7089 articles and included 85 studies (quantitative: 65; qualitative: 9; mixed methods: 8); 54 investigated fatigue domains, five reported fatigue scale development, and 26 focused on both. The review included 34,984 participants (9814 male; 25,126 female) with a mean age of 47.43 years (range 36-55.4). A total of 791 items related to fatigue domains, signs, symptoms, and experiences were extracted and categorised into three key areas: fatigue triggers, domains, and impacts. We identified eight fatigue triggers (physical, cognitive, psychological, social, medical, lifestyle, temporal, and environmental), five fatigue domains (general, physical, cognitive, psychosocial, and social), and five areas of fatigue impact (global, physical, cognitive, psychological, and social impacts of fatigue). Twenty-nine scales, tests, measures, and indices that measure MS-related fatigue were identified. Nineteen of these were validated by self-reported fatigue scales. The scales fully covered the domains of MS-related fatigue. However, the identified scales did not fully capture medical and lifestyle triggers, as well as psychological and global impacts. Additionally, no single scale fully encompassed all three aspects of fatigue and their corresponding subcategories.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;This review demonstrates the importance of integrating the subjective expe","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144977957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-Reported Side Effect Bother: Understanding the Value of the Baseline Report.","authors":"Jessica Roydhouse, Monique Breslin, Anne Zola, Ethan Basch, Melanie Calvert, David Cella, Mary Lou Smith, Gita Thanarajasingam, John Devin Peipert","doi":"10.1007/s40271-025-00766-2","DOIUrl":"https://doi.org/10.1007/s40271-025-00766-2","url":null,"abstract":"<p><strong>Aim: </strong>Patient-perceived treatment tolerability can affect patient ability and willingness to remain on therapy. We sought to examine completion rates for a single item of overall side effect bother at baseline and at the first on-treatment assessment, the association between this item with other patient-reported outcomes (PROs) and the odds of early discontinuation due to clinician-assessed adverse events or reasons other than disease progression.</p><p><strong>Methods: </strong>Data were from three commercial cancer trials in solid tumours, focusing on the safety population. The GP5 item from the Functional Assessment of Cancer Therapy (FACT) was used for side effect bother. Other PROs included items on specific symptoms, functional impacts and global health status, all drawn from validated measures. Descriptive statistics were used for completion rates, and correlation and logistic regression analyses were used to examine associations. GP5 was dichotomised as 0-1 ('low') versus 2-4 ('high').</p><p><strong>Results: </strong>Completion rates were at or above 90% at baseline for all items. GP5 completion rates were 5% lower than completion rates for other items (89.8% versus 94.9%) at baseline, but this was not seen after baseline. Among patients with non-missing baseline GP5, 11.8-15.7% of cancer treatment-naïve patients reported high bother, compared with 23.9% of treatment-experienced patients. Patients with high bother at baseline had higher odds of early discontinuation compared with those with low bother, but this was not statistically significant after covariate adjustment.</p><p><strong>Conclusions: </strong>Continued collection of the GP5 item and concomitant work aiming to understand reasons for missingness as well as interpretation is important for evaluating tolerability in cancer trials.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144977948","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Subcutaneous Infusion of Benzathine Penicillin G Is Acceptable and Preferred Over Intramuscular Injections for Syphilis in Western Australian Sexual Health Clinic Attendees: A Mixed Methods Sub-study.","authors":"Shakayla L Walley, Jane Oliver, Stephanie L Enkel, Alice Kupara, Simon Swallow, Joseph Cotter, Gerard F Hoyne, Thel K Hla, Laurens Manning","doi":"10.1007/s40271-025-00764-4","DOIUrl":"https://doi.org/10.1007/s40271-025-00764-4","url":null,"abstract":"<p><strong>Background: </strong>Controlling the syphilis epidemic in Australia is a public health priority. Regular intramuscular (IM) injections of benzathine penicillin G (BPG) are the current standard of care for late latent syphilis in Australia; however, repeated IM BPG injections are painful, and treatment completion rates are low. Early-phase clinical trials have demonstrated the tolerability and safety of high-dose subcutaneous infusions of BPG (SCIP), where the total treatment dose can be delivered at a single visit. Here we describe the experiences and preferences of attendees of Western Australian sexual health clinics in the Perth metropolitan region who have syphilis and were treated with SCIP.</p><p><strong>Methods: </strong>This was a mixed methods sub-study of a phase IIa clinical trial (ACTRN12622000349741). Eligible participants attended South Terrace Sexual Health Clinic in Fremantle, Western Australia, after referral from physicians to the South Metropolitan Health Service. Nine participants were followed up with for 24 weeks after receiving SCIP. Semi-structured interviews were conducted at three time points. All interviews were audio recorded, transcribed verbatim and underwent content and thematic analyses.</p><p><strong>Results: </strong>Two themes were identified: (1) tolerance of SCIP and (2) SCIP is preferred over IM BPG. SCIP was well-tolerated by participants, who praised the ease and convenience of the procedure. Participants described instances of mild to moderate pain during and after SCIP, substantiated by self-reported pain scores and adapted Skindex-16 scores. All participants stated that they would prefer SCIP over IM BPG as syphilis treatment, and that they would be willing to receive SCIP again should re-infection occur.</p><p><strong>Conclusion: </strong>All participants described tolerable infusion experiences with a clear preference for SCIP over IM BPG to treat syphilis. SCIP has potential to replace IM BPG in clinical practice. Further research is needed to determine the acceptability and efficacy of SCIP in diverse cohorts.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144978019","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Qualitative Study on Patient Experience with Signs, Symptoms, and Daily Impacts of Immune Thrombocytopenia.","authors":"Waleed Ghanima, Nichola Cooper, Sylvie Bozzi, Ahmed Daak, Imene Gouia, Matias Cordoba, Javier Barrio, Michael Kostikas, Owen Cooper, Howard Liebman","doi":"10.1007/s40271-025-00762-6","DOIUrl":"https://doi.org/10.1007/s40271-025-00762-6","url":null,"abstract":"<p><strong>Background: </strong>Primary immune thrombocytopenia (ITP) adversely impacts a patient's health-related quality of life (HRQoL).</p><p><strong>Objective: </strong>This study explored patients' experiences with signs, symptoms, and impacts of ITP and updated the conceptual disease model for HRQoL in patients with ITP.</p><p><strong>Methods: </strong>Adult patients with ITP were included in the study. Patients with any comorbidity where fatigue was a key symptom (e.g., anemia) were excluded. A concept elicitation interview assessed signs, symptoms, and impacts from the patients' perspective, and cognitive debriefing assessed the validity of the selected patient-reported outcome instruments (ITP-Patient Assessment Questionnaire, Patient Global Impression of Severity of Fatigue, Patient Global Impression on Severity, and Patient Global Impression on Change scales). Symptoms/impacts mentioned by ≥ 50% of patients and a \"bothersomeness\" rating of ≥ 5 were considered salient. The preliminary conceptual model was updated based on the concepts reported by more than one patient.</p><p><strong>Results: </strong>A total of 18 patients (mean age 42.7 ± standard deviation 14.7 years; female 78%) were interviewed. Six salient symptoms included fatigue (94%; n = 17), bruising (83%; n = 15), petechiae (72%; n = 13), difficulty staying awake during the day, difficulty falling asleep, and difficulty staying asleep (all symptoms related to sleep: 50%, n = 9). Six key impacts that emerged included those on work (72%; n = 13), worry and fear (67%; n = 12), limited physical or sporting activities, the perceived need to be cautious to avoid getting hurt, and family, friends, and social life (all 56%; n = 10).</p><p><strong>Conclusion: </strong>This study highlighted six key symptoms of ITP and its impacts on patients with ITP. The concept elicitation findings were used to update the conceptual model, and-during cognitive debriefing-patients found the patient-reported outcome instruments easy to understand and relevant to their experience.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144859908","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}