Patient-Patient Centered Outcomes Research最新文献

{"title":"PAIRED: Quantitative and Qualitative PAtIent-Reported Experiences and perceiveD Benefit of Treatment with Dolutegravir/Lamivudine in the United States.","authors":"Jihad Slim, Andrew P Brogan, Gavin Harper, Katie Mycock, Abigail McMillan, Deanna Merrill, Gustavo Verdier","doi":"10.1007/s40271-025-00779-x","DOIUrl":"https://doi.org/10.1007/s40271-025-00779-x","url":null,"abstract":"<p><strong>Background: </strong>Understanding the treatment experiences and satisfaction of people with HIV using two-drug regimens, including dolutegravir/lamivudine (DTG/3TC), is crucial to assess ongoing needs and meet Ending the HIV Epidemic goals.</p><p><strong>Methods: </strong>PAIRED is a mixed-methods study comprising a quantitative cross-sectional survey (using validated instruments to assess treatment satisfaction, adherence, and health-related quality of life [QOL]) and qualitative interviews. Adults in the USA who switched to DTG/3TC while virologically suppressed and had been using DTG/3TC for ≥ 3 months were eligible. All participants were surveyed, and a sub-set participated in in-depth interviews. Thematic analysis was performed to discern overarching interview themes.</p><p><strong>Results: </strong>Overall, 474 participants completed the survey (assigned female sex at birth, 31%; non-white, 48%; aged ≥ 50 years, 50%); 20 of them participated in qualitative interviews. Treatment satisfaction, adherence, and QOL scores were high. Six themes were extracted from interviews: (1) fewer drugs are important in HIV treatment because of associated reductions in long-term drug exposure and perceived risk of toxicity; (2) high DTG/3TC satisfaction is largely due to achieved expectations regarding efficacy and safety; (3) DTG/3TC simplicity and convenience enable more freedom and autonomy; (4) treatment advancements led to HIV no longer being a \"death sentence\"; (5) managing HIV is no longer the main concern relative to other health conditions; and (6) some HIV unmet needs remain.</p><p><strong>Conclusions: </strong>A large representative population with HIV had primarily positive experiences and few needs after switching to DTG/3TC. These data complement clinical trial data and support DTG/3TC effectiveness and tolerability in real-world settings.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145253683","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: Patients' Needs and Preferences for Cardiac Pacemaker Implantation: A Qualitative Study on Disease and Medical Device Characteristics to Inform a Quantitative Preference Study.","authors":"Alice Vanneste, Peter Sinnaeve, Isabelle Huys, Tom Adriaenssens, Christophe Garweg","doi":"10.1007/s40271-025-00773-3","DOIUrl":"10.1007/s40271-025-00773-3","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145226172","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":"Public Preferences for Emerging Cancer Screening Modalities: A Systematic Review of Discrete Choice Experiments.","authors":"Chee Ern Har, Qi Gao, Wenjia Chen, Yi Wang","doi":"10.1007/s40271-025-00775-1","DOIUrl":"https://doi.org/10.1007/s40271-025-00775-1","url":null,"abstract":"<p><strong>Background: </strong>Early detection of cancer reduces mortality and morbidity, but conventional screening methods often face challenges such as invasiveness, limited accessibility and high resource demands. Emerging cancer screening technologies could overcome these barriers, yet their adoption depends heavily on public acceptance. This systematic review synthesises evidence from discrete choice experiment (DCE) studies examining population preferences for emerging cancer screening technologies.</p><p><strong>Methods: </strong>A systematic review was conducting following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. PubMed, Embase, Scopus, Web of Science and EconLit were systematically searched for studies published up to August 2025. Eligible studies employed DCEs to assess preferences for modalities including liquid biopsy, multi-cancer early detection tests, artificial intelligence reading systems, genetic and genomic testing, mobile Health applications, nanopill-based screening and breath analysis. Studies were screened independently by two reviewers and assessed for reporting quality using the DIRECT checklist. Data were summarised using descriptive statistics and narrative synthesis.</p><p><strong>Results: </strong>In total, 23 studies published between 2014 and 2025 were included, most focusing on genomic testing (n = 8) or liquid biopsy (n = 5). Across studies, 32 attributes were identified and categorised under clinical risk, modality characteristics, screening process, outcomes, or ethical considerations. Respondents generally favoured screening programmes that were clinically effective, accessible and minimally disruptive. Sensitivity was most frequently reported as the most influential attribute. Modality specific attributes influencing public preferences were identified, including data privacy and insurability for genetic and genomic testing, and the role of test providers for mHealth technologies and AI screening tools. Heterogeneity in preferences, attribute importance and predicted uptake were observed across many studies, highlighting differential attitudes towards cancer screening and modality specific attributes within the population.</p><p><strong>Conclusions: </strong>This systematic review provides the first synthesis of DCE evidence across a wide range of emerging cancer screening technologies. Findings reveal that public preferences were shaped not only by clinical performance, but also by convenience, cost and the perceived credibility of new modalities. Considerable heterogeneity in preferences signals the need for tailored strategies to engage different population subgroups and avoid widening disparities in uptake. As these technologies move towards clinical adoption, robust governance and regulatory safeguards will be essential to ensure their translation into equitable and effective cancer screening programmes.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145214450","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":"Patients as Partners in Sickle Cell Disease Research in Africa: A Framework for Equitable Patient-Engaged Health Research.","authors":"Nchangwi Syntia Munung, Lawrence Osei-Tutu, Arafa Salim Said, Hilda Tutuba, Cynthia Changafu, Solange Mela, Alieu Badara W Sambou, Victoria Nembaware, Emmanuel Chide Okocha, Patience Kuona, Leon Tshilolo, Maya Sabatello, Marsha Treadwell, Julie Makani, Ambroise Wonkam","doi":"10.1007/s40271-025-00772-4","DOIUrl":"https://doi.org/10.1007/s40271-025-00772-4","url":null,"abstract":"<p><strong>Background: </strong>Patient and public involvement (PPI) in health research is gaining global momentum through initiatives such as INVOLVE (UK), the Patient-Centred Outcomes Research Institute (USA), and the Strategy for Patient-Oriented Research (Canada). However, its implementation in Africa remains limited and lacks context-specific guidance. To address this gap, the Sickle Africa Data Coordinating Center supported the development of the Patients as Partners in Sickle Cell Disease Research (SCD-PAPIR) Framework to guide meaningful patient engagement and involvement in SCD research in Africa.</p><p><strong>Methods: </strong>An iterative, participatory approach was adopted. The process involved the creation of a SCD PAPIR working group comprising SCD patient advocates in 14 African countries; 18 virtual working group meetings, one public webinar, and three in-person workshops. The framework was co-created through facilitated discussions reflecting on prior engagement in SCD research as a patient partner, and best practices for effective patient-researcher collaborations. Patient-only sessions and leadership roles were integrated to ensure safe spaces and to minimise power imbalances.</p><p><strong>Results: </strong>The SCD-PAPIR Framework positions SCD patients and their caregivers as citizen researchers. Key to the framework is the principle of subsidiarity, which affirms the agency and experiential knowledge of patients while calling for epistemic humility from researchers. Its core pillars include valuing patient expertise, transparent communication, resource sharing, patient empowerment, collective learning, amplification of patient voice, continuous feedback, and shared benefits. Best practices emphasise two-way communication, addressing power asymmetries, co-learning and patient empowerment, co-ownership of outputs, and formalised PAPIR structures.</p><p><strong>Conclusion: </strong>The SCD-PAPIR Framework provides a contextually grounded model for patient-engaged research in Africa and contributes to efforts to decolonise health research by positioning patients as co-creators of knowledge, and not merely a data source. The effective implementation of the framework will require investment in institutionalising PAPIR in SCD research. Future work should focus on designing implementation toolkits, developing PPI training modules for researchers and patient advocates, and adapting the framework to other health conditions.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145187561","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":"Exploring the Impact of a Context-Adapted Decision Aid and Online Training About Shared Decision Making About Goals of Care with Elderly Patients in the Intensive Care Unit: A Mixed-Methods Study.","authors":"Ariane Plaisance, Julien Turgeon, Lucas Gomes Souza, France Légaré, Stéphane Turcotte, Nathalie Germain, Tommy Jean, Maude Dionne, Félix Antoine Fortier, Patrick Plante, Diane Tapp, Véronique Gélinas, Emmanuelle Bélanger, Mark H Ebell, Christian Chabot, Tom H van de Belt, Alexis F Turgeon, Patrick M Archambault","doi":"10.1007/s40271-025-00761-7","DOIUrl":"https://doi.org/10.1007/s40271-025-00761-7","url":null,"abstract":"<p><strong>Purpose: </strong>The aim of this study was to evaluate the impact of an intervention-comprising a context-adapted decision aid (DA) and online shared decision-making (SDM) training for intensivists-on the quality of goals-of-care discussions (GCDs) and the level of involvement of elderly patients in those discussions within an intensive care unit (ICU).</p><p><strong>Methods: </strong>This was a three-phase before-after mixed-methods implementation study conducted in an ICU in Lévis, Québec, Canada. We followed the StaRI and COREQ reporting guidelines. We recruited patients aged ≥ 65 years and their attending intensivists. We video-recorded GCDs in three phases: Phase I: GCDs without a DA; Phase II: GCDs with a DA about goals of care but no online training; and Phase III: GCDs with both a DA about goals of care and online training about SDM. All GCDs recordings were transcribed verbatim. We measured the level of patient engagement by intensivists in SDM about goals of care through the OPTION scale and evaluated GCDs quality using the Audit of Communication, Care Planning, and Documentation (ACCEPT) indicators. A qualitative thematic analysis of transcriptions of the encounters was also performed.</p><p><strong>Results: </strong>Out of 359 eligible patients, the study included 21 patients (71% men; median age, 77 years; 57% without high school diploma) and five intensivists (80% men; median age, 35 years). Despite completing online training, the DA was never used in recorded encounters. We did not perform any tests of statistical significance to compare results in each study phase because of small sample sizes over each phase. OPTION and ACCEPT scores were low in each phase, but physicians did engage in GCDs. We found that 76% of documented goals of care for life-sustaining therapy were consistent with patient preferences expressed during recorded GCD, a determination made by two independent observers. Several patients expressed confusion about GCDs. Regarding the use of a DA to support SDM in GCDs, intensivists identified several barriers and facilitators. Barriers included physician attitudes, lack of training, and systemic pressures, while a patient-centered approach and positive patient attitudes were seen as key facilitators.</p><p><strong>Conclusion: </strong>Despite implementing a context-adapted DA and online SDM training, this study found no significant improvement in the quality of GCDs or patient engagement, notably as the DA was not utilized. Communication barriers and patient confusion about goals of care highlight the persistent challenges in achieving true SDM in the ICU. Future strategies should focus on overcoming identified barriers for successful integration of such interventions.</p><p><strong>Trial registration number: </strong>NCT04034979.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145151806","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":"Attribute Development and Level Selection for a Discrete Choice Experiment to Elicit Care Preferences of Older Adults and Informal Caregivers Aging in Place in The Netherlands.","authors":"Isabelle Vullings, Luis Pilli, Marie-Julie C H Russchen, Nanon H M Labrie, Joffre Swait, Özgül Uysal-Bozkir, Joost Wammes, Janet L MacNeil Vroomen","doi":"10.1007/s40271-025-00774-2","DOIUrl":"https://doi.org/10.1007/s40271-025-00774-2","url":null,"abstract":"<p><strong>Background: </strong>The Netherlands reformed its long-term care policy to encourage older adults to age in place with the support of informal caregivers. It remains unclear whether the available care and support options align with the needs and preferences of older adults and caregivers. Discrete choice experiments (DCE) are increasingly used to identify individual preferences. This study describes the development of attributes (e.g., emotional support) and attribute levels (e.g., psychologist and case manager) for a DCE on aging-in-place preferences among older adults and informal caregivers in The Netherlands.</p><p><strong>Methods: </strong>Semi-structured interviews were conducted with older adults and informal caregivers to identify key components for successful aging in place. Interviews were transcribed, and reflexive thematic analysis identified patterns that led to a list of attributes. Visuals of these attributes were created and presented to a new sample of informal caregivers and older adults in focus groups to rank attributes and define attribute levels.</p><p><strong>Results: </strong>Attributes identified through the interviews (N = 28) included housing, personal care, household tasks, transportation, social activities, digital skills, and help navigating the healthcare system. Focus groups (N = 35) found that older adults prioritized housing, while informal caregivers prioritized navigating the healthcare system. Transportation and digital skills were ranked as the least important and were excluded from the final list of attributes.</p><p><strong>Conclusions: </strong>Our findings provide a detailed understanding of aging-in-place preferences of older adults and informal caregivers. These insights will inform a DCE to quantify preferences and provide evidence for policymakers. This study increases transparency about the process of attribute development and level selection, contributing to the quality of the final DCE study.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145088083","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":"Using a Timeline Activity in Virtual Concept Elicitation Interviews with Youth: Methods and Lessons Learned from an Interpretive Descriptive Study to Develop a Patient-Reported Outcome Measure (GENDER-Q Youth).","authors":"Shelby L Kennedy, Susan M Jack, Natasha Johnson, Jennifer Couturier, Charlene Rae, Anne F Klassen","doi":"10.1007/s40271-025-00770-6","DOIUrl":"https://doi.org/10.1007/s40271-025-00770-6","url":null,"abstract":"<p><p>One-to-one interviews and focus groups are used to generate qualitative data about patients' health outcomes and inform the development of patient-reported outcome measures (PROMs). In the development of PROMs for young people, visual elicitation tools can be used with one-to-one interviews and focus groups to enhance the data generation process and data quality. This article aims to (1) provide a detailed description of how visual elicitation tools can be applied in the development of youth-specific PROMs using the GENDER-Q Youth study as an example and (2) share the lessons learned from the GENDER-Q Youth study with PROM developers who are considering using visual elicitation tools in their own virtual qualitative studies. This article discusses processes that took place within the context of a mixed-methods, multi-step study to develop a PROM for youth receiving gender-affirming care called GENDER-Q Youth. Step one was an applied qualitative health research study using an interpretive description approach. Virtual concept elicitation interviews were conducted with transgender and gender diverse youth aged 12-18 years at recruitment using an optional timeline-based visual elicitation tool (i.e., before interviews, youth were invited to create a timeline about their gender-affirming care journeys). The research team navigated ethical and logistical challenges associated with using timeline activities during the data generation process. These challenges occurred during the pre-interview stage (e.g., mailing activity supplies) and during interviews (e.g., incorporating the timeline activity into the interview). Details about the approach used by the research team, challenges faced, and lessons learned are discussed. When conducting one-to-one virtual concept elicitation interviews, visual elicitation tools have the potential to enhance the quality of data generated about participants' outcomes and experiences of healthcare. Visual elicitation tools can also improve the interview experiences of both participants and researchers and are feasible to implement within the context of qualitative PROM development research with young people.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145066192","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":"Which Factors Influence Decisions to Withdraw from Eculizumab: A Qualitative Study of Patients Diagnosed with aHUS.","authors":"Jan Lecouturier, Neil Sheerin","doi":"10.1007/s40271-025-00771-5","DOIUrl":"https://doi.org/10.1007/s40271-025-00771-5","url":null,"abstract":"<p><strong>Background: </strong>Atypical haemolytic uremic syndrome (aHUS) is a rare life-threatening disease. Lifelong treatment with intravenous eculizumab every 2/3 weeks was recommended, but evidence is emerging that many patients can stop complement inhibition and restart should they relapse. However, little is known about the opinions and needs of patients with aHUS on withdrawal.</p><p><strong>Objective: </strong>We aimed to understand the factors that affect decisions to withdraw from treatment.</p><p><strong>Methods: </strong>This was a qualitative study embedded in an eculizumab withdrawal trial. Data were collected through in-depth telephone interviews from January 2019 to June 2022. We conducted a thematic analysis using a constant comparative method. Interviewees included eight adults and the parents of 12 children with aHUS approached to participate in the trial. Follow-up interviews were conducted between January 2021 and June 2024 with those who withdrew from eculizumab.</p><p><strong>Results: </strong>The onset of aHUS had been traumatic for most. Regarding eculizumab, withdrawal group participants talked of the disruptive treatment regimen and side effects, the time off work/school, and impacts on taking holidays. Decisions to withdraw from eculizumab were driven by the wish to lead more normal lives and concerns about long-term treatment. Drivers for declining withdrawal focused on relapse and its perceived impact. After 2 years, the withdrawal group had regained a semblance of normality, though fears about relapse remained, and they were aware of the need for long-term follow-up. Participants had a greater sense of control over the necessary steps should they/their child relapse.</p><p><strong>Conclusion: </strong>An understanding of patient/parent experiences should guide discussions about eculizumab withdrawal. Although the availability of an alternative complement inhibitor reduces the frequency of infusions, most of the factors influencing withdrawal decisions remain unchanged. Support to alleviate fears in the early stages of withdrawal would be beneficial. Evidence from the main trial on successful withdrawal, and recovery time where eculizumab was reinstated, may provide reassurance to those who are uncertain about withdrawal.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145056128","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":"Practical considerations towards the collection of patient reported outcome data among hematopoietic cell transplant recipients.","authors":"Gemma Pugh, Christina Yiallouridou, Dawn Hart, Rachel Miller, Robert Danby","doi":"10.1007/s40271-025-00769-z","DOIUrl":"https://doi.org/10.1007/s40271-025-00769-z","url":null,"abstract":"<p><strong>Background: </strong>There is increasing interest in using patient-reported outcome measures (PROMs) to assess quality of life (QoL) following hematopoietic cell transplant (HCT). However, there is limited consensus on how such data should be collected within HCT services. This survey study investigated health professionals (HCPs) views towards QoL data collection and factors affecting the use of PROMs within HCT centres in the UK.</p><p><strong>Method: </strong>Individual survey items were based upon the Consolidated Framework for Implementation Research (CFIR). The CFIR consists of five domains which are known to affect intervention (in this instance PROM) adoption and implementation. National coverage was achieved with survey responses received from all UK adult allograft HCT centres.</p><p><strong>Results: </strong>Findings indicated PROMs use in UK HCT services is ad hoc with PROMs mostly used as screening or diagnostic tools for emotional health or for service improvement activities including audits. HCPs reported that patient-reported outcome (PRO) data collection is limited by a lack of resource, infrastructure and referral pathways if the PROM were to flag a patient issue. A large proportion of HCPs (> 70%) noted that PRO data within their centre would be best enabled through dedicated research staff and data management infrastructure to support data collection and storage. Despite growing evidence of the utility of electronic data capture, most HCPs (> 50%) believed electronic PROMs (ePROMs) may be difficult to implement due to data protection issues and limited access to electronic devices.</p><p><strong>Conclusions: </strong>These findings highlight the opportunities and challenges to PRO data collection in UK HCT services and demonstrate the need for practical solutions. The development of a standardised approach to PROM use among HCT recipients and investment in workforce and data management infrastructure is needed to support an integrated approach to PRO data collection, storage and use.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145034714","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 Systematic Literature Review of Preference Studies in Migraine Treatments.","authors":"Divya Mohan, Harrison Clarke, Natasha Ramachandran, Jaein Seo","doi":"10.1007/s40271-025-00768-0","DOIUrl":"https://doi.org/10.1007/s40271-025-00768-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Migraine care is often suboptimal owing to undertreatment, variation in clinical outcomes and administration methods among existing treatments, and between- and within-individual heterogeneity in the clinical course of migraine. In response to these challenges, preference studies have been increasingly conducted to inform treatment decision-making and development. However, gaps remain in understanding how treatment preferences have been assessed across different migraine studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The aim was to synthesize existing evidence to inform the design and conduct of future preference migraine research. This review examined treatment attributes included in preference studies, focusing on how attributes were developed, framed, and presented; how their values were analyzed and reported; and whether these values differed by respondent characteristics.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A systematic review protocol was registered with the International Prospective Register of Systematic Reviews (PROSPERO) (CRD42025614690). Embase, MEDLINE, and the Cochrane Library were searched for relevant stated preference studies on migraine treatments (October 2024). Two researchers independently screened studies, and data were extracted using a predefined template. Extracted information included study characteristics, methods for attribute and instrument development, choice task design, attribute framing, and analytical approaches. Narrative synthesis and descriptive statistics were used to summarize findings. Attribute importance was assessed by deriving relative rankings of attributes from marginal utilities or importance scores across studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Overall, 18 studies were reviewed from the 186 that were screened. Stated preference methods comprised discrete choice experiment (n = 12), conjoint analysis (n = 1), contingent valuation method (n = 3), thresholding (n = 1), and time trade-off (n = 1). In total, 13 studies reported their attribute development methods, using literature review only (n = 2), expert consultation only (n = 1), and multi-method approaches combining literature reviews with qualitative research and/or expert or payer consultation (n = 10). In addition, 17 studies included at least 1 benefit attribute, resulting in 26 unique attributes grouped into seven overarching concepts. Risk attributes were included in 11 studies, with injection site reactions (n = 5), gastrointestinal effects (n = 4), and cognitive effects (n = 3) as the most common adverse events. Administration-related attributes appeared in ten studies, with mode and/or frequency of administration being the most common (n = 10). Eight studies used visual aids to illustrate attributes. Preference heterogeneity was explored in 14 studies, primarily on the basis of sex (n = 9), monthly migraine days (n = 8), and treatment experience (n = 7).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;This review reveals substa","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145024781","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}