Design of sequentially randomized trials for testing adaptive treatment strategies.
Design of sequentially randomized trials for testing adaptive treatment strategies.
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设计用于测试适应性治疗策略的序贯随机试验。
DOI：
10.1002/sim.6747
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发表时间：
2016
影响因子：
2
通讯作者：
Wahed,AbdusS
中科院分区：
文献类型：
--
作者：
Ogbagaber,SemharB;Karp,Jordan;Wahed,AbdusS
自适应治疗策略（ATS）是一种以结果为导向的算法，允许根据患者的疾病状态和治疗史对复杂疾病进行个性化治疗。由于疾病进展和治疗的慢性、多因素性质，艾滋病、抑郁症和癌症等疾病通常需要几个阶段的治疗。序贯多重分配随机 (SMAR) 设计允许同时推断多个 ATS，其中根据反应状态将患者顺序随机分配到不同阶段的治疗。本文的目的是开发一个样本量公式，以确保有足够的功效来比较两个或多个 ATS。基于用于比较多个 ATS 与连续终点的 Wald 型统计，我们开发了一个样本量公式并通过模拟研究对其进行测试。我们通过模拟表明，所提出的样本量公式保持了标称功效。所提出的样本量公式不适用于具有事件发生时间终点的设计，但该公式对于从业者在设计 SMAR 试验以比较适应性治疗策略时很有用。版权所有 © 2015 约翰·威利父子有限公司
An adaptive treatment strategy (ATS) is an outcome‐guided algorithm that allows personalized treatment of complex diseases based on patients' disease status and treatment history. Conditions such as AIDS, depression, and cancer usually require several stages of treatment because of the chronic, multifactorial nature of illness progression and management. Sequential multiple assignment randomized (SMAR) designs permit simultaneous inference about multiple ATSs, where patients are sequentially randomized to treatments at different stages depending upon response status. The purpose of the article is to develop a sample size formula to ensure adequate power for comparing two or more ATSs. Based on a Wald‐type statistic for comparing multiple ATSs with a continuous endpoint, we develop a sample size formula and test it through simulation studies. We show via simulation that the proposed sample size formula maintains the nominal power. The proposed sample size formula is not applicable to designs with time‐to‐event endpoints but the formula will be useful for practitioners while designing SMAR trials to compare adaptive treatment strategies. Copyright © 2015 John Wiley & Sons, Ltd.