Ellen O’Donnell, Allison Schuette, Michael Waltz, Gregory Aaen, Leslie Benson, Mark Gorman, Timothy Lotze, Soe Mar, Jayne Ness, Moses Rodriguez, Jan-Mendelt Tillema, Teri Schreiner, Yolanda Wheeler, T Charles Casper, Tanuja Chitnis
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Of 293 eligible participants, 192 completed surveys.Results:Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition.Conclusions:Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":4.8000,"publicationDate":"2024-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Patient and family views on research priorities and design of clinical trials and research studies in pediatric multiple sclerosis\",\"authors\":\"Ellen O’Donnell, Allison Schuette, Michael Waltz, Gregory Aaen, Leslie Benson, Mark Gorman, Timothy Lotze, Soe Mar, Jayne Ness, Moses Rodriguez, Jan-Mendelt Tillema, Teri Schreiner, Yolanda Wheeler, T Charles Casper, Tanuja Chitnis\",\"doi\":\"10.1177/13524585241274580\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Background and Objectives:This survey study aimed to (1) identify patient/family research priorities in pediatric-onset multiple sclerosis (POMS), and (2) delineate optimized methods for research study/clinical trials design, engagement, and implementation.Methods:Participants were as follows: (1) parents of a child (<18 years) with POMS enrolled in a national registry, (2) adolescents (13–17 years) with POMS in the registry, and (3) adults (18–40 years) with POMS receiving care at a registry affiliated clinic. Of 293 eligible participants, 192 completed surveys.Results:Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition.Conclusions:Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.\",\"PeriodicalId\":18874,\"journal\":{\"name\":\"Multiple Sclerosis Journal\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":4.8000,\"publicationDate\":\"2024-09-17\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Multiple Sclerosis Journal\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/13524585241274580\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Multiple Sclerosis Journal","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/13524585241274580","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Patient and family views on research priorities and design of clinical trials and research studies in pediatric multiple sclerosis
Background and Objectives:This survey study aimed to (1) identify patient/family research priorities in pediatric-onset multiple sclerosis (POMS), and (2) delineate optimized methods for research study/clinical trials design, engagement, and implementation.Methods:Participants were as follows: (1) parents of a child (<18 years) with POMS enrolled in a national registry, (2) adolescents (13–17 years) with POMS in the registry, and (3) adults (18–40 years) with POMS receiving care at a registry affiliated clinic. Of 293 eligible participants, 192 completed surveys.Results:Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition.Conclusions:Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.
期刊介绍:
Multiple Sclerosis Journal is a peer-reviewed international journal that focuses on all aspects of multiple sclerosis, neuromyelitis optica and other related autoimmune diseases of the central nervous system.
The journal for your research in the following areas:
* __Biologic basis:__ pathology, myelin biology, pathophysiology of the blood/brain barrier, axo-glial pathobiology, remyelination, virology and microbiome, immunology, proteomics
* __Epidemology and genetics:__ genetics epigenetics, epidemiology
* __Clinical and Neuroimaging:__ clinical neurology, biomarkers, neuroimaging and clinical outcome measures
* __Therapeutics and rehabilitation:__ therapeutics, rehabilitation, psychology, neuroplasticity, neuroprotection, and systematic management
Print ISSN: 1352-4585