精准干预重度和/或易恶化型哮喘（PrecISE）适应性平台试验：统计考虑。
The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations.
机构信息
University of North Carolina, Chapel Hill, NC.
Brigham and Women's Hospital and Harvard Medical School, Boston, MA.
出版信息
J Biopharm Stat. 2020 Nov 1;30(6):1026-1037. doi: 10.1080/10543406.2020.1821705. Epub 2020 Sep 17.
Abstract
The Precision Interventions for Severe and/or Exacerbation-prone Asthma (PrecISE) study is an adaptive platform trial designed to investigate novel interventions to severe asthma. The study is conducted under a master protocol and utilizes a crossover design with each participant receiving up to five interventions and at least one placebo. Treatment assignments are based on the patients' biomarker profiles and precision health methods are incorporated into the interim and final analyses. We describe key elements of the PrecISE study including the multistage adaptive enrichment strategy, early stopping of an intervention for futility, power calculations, and the primary analysis strategy.
摘要
精准干预严重和/或易恶化型哮喘（PrecISE）研究是一项适应性平台试验，旨在研究新型干预严重哮喘的方法。该研究采用主协议进行，并采用交叉设计，每个参与者接受多达五种干预措施，至少一种安慰剂。治疗分配基于患者的生物标志物谱，并且在中期和最终分析中纳入了精准健康方法。我们描述了 PrecISE 研究的关键要素，包括多阶段自适应富集策略、干预措施无效的早期停止、功效计算和主要分析策略。
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