论文标题

贝叶斯自适应集群随机试验的新设计

New designs for Bayesian adaptive cluster-randomized trials

论文作者

Shen, Junwei, Golchi, Shirin, Moodie, Erica E. M., Benrimoh, David

论文摘要

已经提出了自适应方法,允许进行更灵活的试验设计,以节省时间或减少样本量的单独随机试验。但是,用于集群随机试验的自适应设计,其中参与者而非个人被随机分配给治疗臂的设计不太常见。由旨在评估针对治疗抑郁症患者的医师的基于机器学习的临床决策系统的有效性的集群随机试验的动机,提出了两种贝叶斯自适应设计用于聚类随机试验,以允许在预先计划的临时分析中提早停止以提高功效。两种设计之间的差异在于参与者被依次招募的方式。鉴于试验中允许的最大群集以及最大群集大小,一个设计顺序募集具有给定最大群集大小的群集,而另一个设计在试验开始时招募了所有群集,但是依次招募了个体参与者,直到提早停止了效率或最终分析。通过模拟各种场景和两种设计类型的两种设计类型探索了设计操作特性。仿真结果表明,对于不同的结果,设计选择可能不同。我们根据仿真结果对贝叶斯自适应簇伴随试验的设计提出建议。

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants rather than individuals are randomized to treatment arms are less common. Motivated by a cluster-randomized trial designed to assess the effectiveness of a machine-learning based clinical decision support system for physicians treating patients with depression, two Bayesian adaptive designs for cluster-randomized trials are proposed to allow for early stopping for efficacy at pre-planned interim analyses. The difference between the two designs lies in the way that participants are sequentially recruited. Given a maximum number of clusters as well as maximum cluster size allowed in the trial, one design sequentially recruits clusters with the given maximum cluster size, while the other recruits all clusters at the beginning of the trial but sequentially enrolls individual participants until the trial is stopped early for efficacy or the final analysis has been reached. The design operating characteristics are explored via simulations for a variety of scenarios and two outcome types for the two designs. The simulation results show that for different outcomes the design choice may be different. We make recommendations for designs of Bayesian adaptive cluster-randomized trial based on the simulation results.

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