一种用于降低成本、更快捷进行早期阿尔茨海默病临床试验的简单遗传分层方法：tau PET 和认知结果的初步研究。
A simple genetic stratification method for lower cost, more expedient clinical trials in early Alzheimer's disease: A preliminary study of tau PET and cognitive outcomes.
机构信息
University of California, San Diego, California, USA.
East China Normal University, Shanghai, China.
出版信息
Alzheimers Dement. 2023 Jul;19(7):3078-3086. doi: 10.1002/alz.12952. Epub 2023 Jan 26.
INTRODUCTION
Identifying individuals who are most likely to accumulate tau and exhibit cognitive decline is critical for Alzheimer's disease (AD) clinical trials.
METHODS
Participants (N = 235) who were cognitively normal or with mild cognitive impairment from the Alzheimer's Disease Neuroimaging Initiative were stratified by a cutoff on the polygenic hazard score (PHS) at 65th percentile (above as high-risk group and below as low-risk group). We evaluated the associations between the PHS risk groups and tau positron emission tomography and cognitive decline, respectively. Power analyses estimated the sample size needed for clinical trials to detect differences in tau accumulation or cognitive change.
RESULTS
The high-risk group showed faster tau accumulation and cognitive decline. Clinical trials using the high-risk group would require a fraction of the sample size as trials without this inclusion criterion.
DISCUSSION
Incorporating a PHS inclusion criterion represents a low-cost and accessible way to identify potential participants for AD clinical trials.
简介
识别最有可能积累 tau 并出现认知能力下降的个体对于阿尔茨海默病（AD）临床试验至关重要。
方法
根据多基因危险评分（PHS）在 65%分位数（高于高风险组，低于低风险组）对来自阿尔茨海默病神经影像学倡议的认知正常或轻度认知障碍的参与者（N=235）进行分层。我们分别评估了 PHS 风险组与 tau 正电子发射断层扫描和认知下降之间的关联。功效分析估计了临床试验检测 tau 积累或认知变化差异所需的样本量。
结果
高风险组表现出更快的 tau 积累和认知能力下降。与没有此纳入标准的试验相比，使用高风险组的临床试验所需的样本量要小得多。
讨论
纳入 PHS 纳入标准是一种低成本且易于实施的方法，可以识别 AD 临床试验的潜在参与者。
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