论文标题
将实验治疗臂添加到多臂多级平台试验中
Adding experimental treatment arms to Multi-Arm Multi-Stage platform trials in progress
论文作者
论文摘要
多臂多阶段（MAMS）平台试验是比较多种治疗方法的有效工具。假设我们希望在正在进行的试验中添加一种治疗方法，以获取MAMS设计的好处。这应该如何完成？ MAMS框架需要预先计划的选项，以在每个阶段进行试验以控制家庭错误率。因此，很难同时进行计划和计划外的设计修改。条件误差方法是一种工具，可以在维持总体错误率的同时计划外设计修改。在这项工作中，我们使用条件错误方法来允许在进行的MAMS试验中添加新的武器。 我们证明了将其他假设纳入测试结构的原则。使用此框架，我们展示了如何更新正在进行的MAMS试验的测试程序，以结合其他治疗臂。模拟说明了使用固定规则的方式和何时进行设计修改的固定规则，说明了此类过程的可能操作特性。
Multi-Arm Multi-Stage (MAMS) platform trials are an efficient tool for the comparison of several treatments. Suppose we wish to add a treatment to a trial already in progress, to access the benefits of a MAMS design. How should this be done? The MAMS framework requires pre-planned options for how the trial proceeds at each stage in order to control the family-wise error rate. Thus, it is difficult to make both planned and unplanned design modifications. The conditional error approach is a tool that allows unplanned design modifications while maintaining the overall error rate. In this work, we use the conditional error approach to allow adding new arms to a MAMS trial in progress. We demonstrate the principles of incorporating additional hypotheses into the testing structure. Using this framework, we show how to update the testing procedure for a MAMS trial in progress to incorporate additional treatment arms. Simulations illustrate the possible operating characteristics of such procedures using a fixed rule for how and when the design modification is made.
