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Design and analysis considerations for comparing dynamic treatment regimens with binary outcomes from sequential multiple assignment randomized trials

机译:设计和分析注意事项,用于比较动态治疗方案与顺序多次分配随机试验的二元结果

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摘要

In behavioral, educational and medical practice, interventions are often personalized over time using strategies that are based on individual behaviors and characteristics and changes in symptoms, severity, or adherence that are a result of one's treatment. Such strategies that more closely mimic real practice, are known as dynamic treatment regimens (DTRs). A sequential multiple assignment randomized trial (SMART) is a multi-stage trial design that can be used to construct effective DTRs. This article reviews a simple to use weighted and replicated' estimation technique for comparing DTRs embedded in a SMART design using logistic regression for a binary, end-of-study outcome variable. Based on a Wald test that compares two embedded DTRs of interest from the weighted and replicated' regression model, a sample size calculation is presented with a corresponding user-friendly applet to aid in the process of designing a SMART. The analytic models and sample size calculations are presented for three of the more commonly used two-stage SMART designs. Simulations for the sample size calculation show the empirical power reaches expected levels. A data analysis example with corresponding code is presented in the appendix using data from a SMART developing an effective DTR in autism.
机译:在行为,教育和医疗实践中,通常会根据策略使用个性化的干预措施,这些策略基于个人的行为和特征以及因治疗而导致的症状,严重性或依从性的变化。这种更贴近实际实践的策略称为动态治疗方案(DTR)。序贯多任务随机试验(SMART)是可用于构建有效DTR的多阶段试验设计。本文回顾了一种简单易用的加权和复制估算技术,该技术可通过对数研究终点变量的逻辑回归对SMART设计中嵌入的DTR进行比较。基于Wald测试,该测试从加权和复制的回归模型中比较了两个感兴趣的嵌入式DTR,并通过相应的用户友好小程序对样本大小进行了计算,以帮助设计SMART。给出了三种较常用的两阶段SMART设计的分析模型和样本量计算。样本量计算的仿真表明,经验能力达到了预期水平。附录中提供了一个具有相应代码的数据分析示例,该示例使用来自SMART的数据开发了自闭症的有效DTR。

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