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Sample size calculation based on precision for pilot sequential multiple assignment randomized trial (SMART)
Biometrical Journal ( IF 1.7 ) Pub Date : 2020-06-11 , DOI: 10.1002/bimj.201900364
Xiaoxi Yan 1 , Palash Ghosh 1, 2 , Bibhas Chakraborty 1, 3, 4
Affiliation  

The sequential multiple assignment randomized trial (SMART) is a design used to develop dynamic treatment regimes (DTRs). Given that DTRs are generally less well researched, pilot SMART studies are often necessary. One challenge in pilot SMART is to determine the sample size such that it is small yet meaningfully informative for future full-fledged SMART. Here, we develop a precision-based approach, where the calculated sample size confines the marginal mean outcome of a DTR within a prespecified margin of error. The sample size calculations will be presented for two-stage SMARTs, and for various common outcome types.

中文翻译:

基于试点顺序多重分配随机试验 (SMART) 精度的样本量计算

序贯多重分配随机试验 (SMART) 是一种用于开发动态治疗方案 (DTR) 的设计。鉴于 DTR 的研究通常较少,因此通常需要进行试点 SMART 研究。试点 SMART 的一项挑战是确定样本量,使其规模小但对未来成熟的 SMART 具有有意义的信息。在这里,我们开发了一种基于精度的方法,其中计算出的样本量将 DTR 的边际平均结果限制在预先指定的误差范围内。将针对两阶段 SMART 和各种常见结果类型提供样本量计算。
更新日期:2020-06-11
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