Planning for Prevention of Parkinson Disease: Now Is the Time.
Planning for Prevention of Parkinson Disease: Now Is the Time.
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DOI：
10.1212/wnl.0000000000200789
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发表时间：
2022-08-16
期刊：
影响因子：
9.9
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中科院分区：
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帕金森病（PD）是一种慢性进行性神经退行性疾病，在全球范围内患病率不断增加。尽管对明显帕金森病的神经保护疗法进行了许多试验，但尚未建立疾病缓解疗法。在过去的几十年里，一系列的突破已经确定了离散人群患帕金森病的风险大大增加。基于这些知识，现在是设计和实施 PD 预防试验的时候了。这项努力建立在阿尔茨海默病和亨廷顿病早期预防试验的经验基础上。本文首先回顾了这些其他神经退行性疾病的预防试验先例，然后重点讨论 PD 预防试验的关键设计要素，包括谁参加这些试验、测试什么疗法以及如何衡量预防试验的结果。我们的观点反映了进展和仍然存在的挑战，正是这些挑战推动了 2021 年召开“帕金森病预防规划：试验设计研讨会和研讨会”会议。
Parkinson disease (PD) is a chronic progressive neurodegenerative disease with increasing worldwide prevalence. Despite many trials of neuroprotective therapies in manifest PD, no disease-modifying therapy has been established. Over the past several decades, a series of breakthroughs have identified discrete populations at substantially increased risk of developing PD. Based on this knowledge, now is the time to design and implement PD prevention trials. This endeavor builds on experience gained from early prevention trials in Alzheimer disease and Huntington disease. This article first reviews prevention trial precedents in these other neurodegenerative diseases before focusing on the critical design elements for PD prevention trials, including whom to enroll for these trials, what therapeutics to test, and how to measure outcomes in prevention trials. Our perspective reflects progress and remaining challenges that motivated a 2021 conference, “Planning for Prevention of Parkinson: A Trial Design Symposium and Workshop.”