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帕金森病预防之路:结论与展望。

Path to Parkinson Disease Prevention: Conclusion and Outlook.

机构信息

From the Department of Neurology (D.B.), Christian-Albrechts-University, Kiel, Germany; Molecular Neurobiology Laboratory (G.F.C., M.A.S.), Mass General Institute for Neurodegenerative Disease, Department of Neurology, Massachusetts General Hospital, Charlestown; Harvard Medical School (G.F.C., M.A.S.), Boston, MA; Parkinson's Foundation Research Advocates Program (J.L.K.), Parkinson's Foundation, Miami, FL/New York, NY; Northwestern University Feinberg School of Medicine (T.S.), Weill Institute for Neuroscience (C.T.), Department of Neurology, University of California - San Francisco; and Parkinson's Disease Research Education and Clinical Center (C.T.), San Francisco Veterans Affairs Medical Center.

出版信息

Neurology. 2022 Aug 16;99(7 Suppl 1):76-83. doi: 10.1212/WNL.0000000000200793.

Abstract

Tremendous progress in our understanding of the pathophysiology and clinical manifestations of the prodromal phase of Parkinson disease (PD) offers a unique opportunity to start therapeutic interventions as early as possible to slow or even stop the progression to clinically manifest motor PD. A Parkinson's Prevention Conference, "Planning for Prevention of Parkinson's: A trial design symposium and workshop" was convened to discuss all issues that need to be addressed before the launch of the first PD prevention study. In this review, we summarize the major opportunities and challenges in designing prevention trials in PD, organized by the following critical trial design questions: ? ? ? and the pivotal question, during the prodromal disease ? We outline the implications of these questions and their meaning for a responsible, sustainable, and fruitful further planning for prevention trials. Despite the great progress that has been made, it needs to be acknowledged that several queries remain to be carefully considered and addressed because prevention trials are being planned and become a reality.

摘要

我们对帕金森病(PD)前驱期的病理生理学和临床表现的理解取得了巨大进展,这为尽早开始治疗干预提供了独特的机会,以减缓甚至阻止向有临床运动表现的 PD 的进展。为了讨论在启动首个 PD 预防研究之前需要解决的所有问题,召开了一次帕金森预防会议,题为“规划帕金森预防:试验设计研讨会和讲习班”。在这篇综述中,我们根据以下关键试验设计问题总结了 PD 预防试验设计的主要机遇和挑战:??? 和关键问题, 在前驱期疾病期间? 我们概述了这些问题的含义及其对预防试验负责任、可持续和富有成效的进一步规划的意义。尽管已经取得了巨大的进展,但需要承认,由于正在计划和实施预防试验,因此仍有几个问题需要仔细考虑和解决。

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