背景:针对自身免疫性疾病的患者动力研究网络(PPRN)在成年人群中广泛用于招募患者并推动以患者为中心的研究,但很少包括儿科患者。我们旨在通过PPRN传播的调查来表征有关研究需求和参与小儿多发性硬化症(POMS)的观点。
方法:这是一个探索性的,横断面研究。研究期为2022年2月1日至2023年2月9日。三份问卷分发给(1)POMS(PwPOMS)患者,(2)PwPOMS(C-PwPOMS)的护理人员,和(3)健康护理提供者/研究人员在POMS(HR-POMS)。
结果:共有88名参与者被纳入分析;44%(n=39)为PwPOMS,42%(n=37)为C-PwPOMS,14%(n=12)为HR-POMS。一些PwPOMS(18%)和C-PwPOMS(9%)表示研究犹豫,但更多,69%的PwPOMS和68%的C-PwPOMS,对参与研究感兴趣。然而,不到一半的PwPOMS(38%)和C-PwPOMS(38%)报告了以前的研究参与。HR-POMS报告了资金(100%)和招募参与者(58%)方面的困难。PwPOMS(67%),C-PwPOMS(62%),HR-POMS(67%)对未来参与PPRN持开放态度。
结论:本研究中患有POMS的参与者表达了对研究参与的强烈兴趣,但也表达了参与的犹豫,这可能会导致研究人员提出的招聘挑战。尽管探索性设计限制了对更大POMS群体的普适性,这项研究表明,PPRN非常适合征求POMS主要利益相关者的态度和意见。利用PPRN进行POMS的未来研究应优先考虑不同的,具有代表性的队列,并专注于理解和缓解阻碍研究参与的问题。
BACKGROUND: Patient-powered research networks (PPRNs) for autoimmune disease are widely used in the adult population to recruit patients and drive patient-centered research, but few have included pediatric patients. We aimed to characterize viewpoints regarding research needs and participation in pediatric-onset multiple sclerosis (POMS) via a PPRN-disseminated survey.
METHODS: This is an exploratory, cross-sectional study. The study period was February 1, 2022, to February 9, 2023. Three questionnaires were disseminated to (1) patients with POMS (PwPOMS), (2) caregivers of PwPOMS (C-PwPOMS), and (3) health care providers/researchers in POMS (HR-POMS).
RESULTS: A total of 88 participants were included for analysis; 44% (n = 39) were PwPOMS, 42% (n = 37) were C-PwPOMS, and 14% (n = 12) were HR-POMS. Some PwPOMS (18%) and C-PwPOMS (9%) expressed research hesitancy, but more, 69% of PwPOMS and 68% of C-PwPOMS, were interested in research participation. Nevertheless, less than half of PwPOMS (38%) and C-PwPOMS (38%) reported previous research involvement. HR-POMS reported difficulties in funding (100%) and recruiting participants (58%). PwPOMS (67%), C-PwPOMS (62%), and HR-POMS (67%) were open to future involvement in PPRNs.
CONCLUSIONS: Participants with POMS in this study expressed strong interest in research involvement but also expressed participation hesitancy, which may contribute to recruiting challenges expressed by researchers. Although the exploratory design limits generalizability to the larger POMS population, this study shows PPRNs are well-suited to soliciting attitudes and opinions of key stakeholders in POMS. Future studies utilizing PPRNs for POMS should prioritize diverse, representative cohorts and focus on understanding and mitigating issues hindering research participation.