患者和家属对儿科多发性硬化症研究重点以及临床试验和研究设计的看法

IF 4.8 2区 医学 Q1 CLINICAL NEUROLOGY Multiple Sclerosis Journal Pub Date : 2024-09-17 DOI:10.1177/13524585241274580
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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引用次数: 0

摘要

背景与目标:本调查研究旨在(1)确定儿科发病型多发性硬化症(POMS)患者/家庭的研究重点;(2)确定研究/临床试验设计、参与和实施的优化方法:(方法:参与者包括:(1)在国家登记处登记的POMS患儿(18岁)的父母;(2)在登记处登记的POMS青少年(13-17岁);(3)在登记处附属诊所接受治疗的POMS成人(18-40岁)。在293名符合条件的参与者中,有192人完成了调查。结果显示:人们对医疗保健和药物治疗的体验总体上是积极的,但仍有需要重点改进的地方。参加临床试验的激励措施包括:之前测试过的药片形式的药物、每 3 个月⩾ 次的血液检查/研究访问、⩽ 1 小时的认知测试、差旅和时间补偿、继续服用当前多发性硬化症(MS)药物的能力、服用安慰剂时可选择服用研究药物以及个性化的研究反馈。临床研究的重点是:(1)社会心理影响;(2)认知/学业影响;(3)环境风险;以及(4)营养。结论:研究结果凸显了采用综合方法进行研究设计以及关注疾病对日常生活的影响对患者和家属参与POMS临床试验和研究的重要性。
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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.
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来源期刊
Multiple Sclerosis Journal
Multiple Sclerosis Journal 医学-临床神经学
CiteScore
10.90
自引率
6.90%
发文量
186
审稿时长
3-8 weeks
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
期刊最新文献
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