将生物标志物结果纳入唐氏综合征阿尔茨海默病临床试验中。
Integrating Biomarker Outcomes into Clinical Trials for Alzheimer's Disease in Down Syndrome.
机构信息
Michael S. Rafii, Alzheimer's Therapeutic Research Institute (ATRI), Keck School of Medicine, University of Southern California, USA,
出版信息
J Prev Alzheimers Dis. 2021;8(1):48-51. doi: 10.14283/jpad.2020.35.
Abstract
The NIH-funded Alzheimer's Biomarker Consortium Down Syndrome (ABC-DS) and the European Horizon 21 Consortium are collecting critical new information on the natural history of Alzheimer's Disease (AD) biomarkers in adults with Down syndrome (DS), a population genetically predisposed to developing AD. These studies are also providing key insights into which biomarkers best represent clinically meaningful outcomes that are most feasible in clinical trials. This paper considers how these data can be integrated in clinical trials for individuals with DS. The Alzheimer's Clinical Trial Consortium - Down syndrome (ACTC-DS) is a platform that brings expert researchers from both networks together to conduct clinical trials for AD in DS across international sites while building on their expertise and experience.
摘要
美国国立卫生研究院（NIH）资助的阿尔茨海默病生物标志物联盟唐氏综合征（ABC-DS）和欧洲地平线 21 联盟正在收集唐氏综合征（DS）成人中阿尔茨海默病（AD）生物标志物自然史的关键新信息，该人群具有发生 AD 的遗传倾向。这些研究还提供了关键的见解，即哪些生物标志物最能代表在临床试验中最可行的具有临床意义的结果。本文考虑了如何将这些数据整合到 DS 个体的临床试验中。阿尔茨海默病临床试验联盟 - 唐氏综合征（ACTC-DS）是一个平台，将来自两个网络的专家研究人员聚集在一起，在国际站点开展 DS 中的 AD 临床试验，同时利用他们的专业知识和经验。
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