多臂多阶段 (MAMS) 随机选择设计:治疗选择规则对操作特征的影响
Babak Choodari-Oskooei1, Alexandra Blenkinsop2, Kelly Handley3
1MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL, 90 High Holborn, WC1V 6LJ, London, United Kingdom. b.choodari-oskooei@ucl.ac.uk.
BMC medical research methodology
|June 3, 2024
概括
多臂多阶段 (MAMS) 选择设计通过减少样本大小来提高试验效率. 预先指定选择规则和停止边界对于控制试验特征和保持统计能力至关重要.
科学领域:
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
- 药学研究 药学研究
背景情况:
- 多臂多阶段 (MAMS) 设计用于在确认性试验中评估多个研究问题.
- 在许多干预试验中的资源限制需要限制非有益武器的方法.
- 治疗选择规则可以在早期阶段实施,以限制在MAMS试验中进展的武器数量.
研究的目的:
- 为在MAMS框架内实施治疗选择提供指导方针.
- 探索治疗选择规则,停止边界和时间对MAMS设计特征的影响.
- 通过治疗选择优化MAMS试验的效率和统计完整性.
主要方法:
- 概述了设计MAMS选择试验的步骤.
- 进行了广泛的模拟研究,以评估设计的操作特性.
- 评估的最大/预期的样本大小,家庭的I型错误率 (FWER),以及在各种停止边界条件下的总功率.
主要成果:
- 预先规定的治疗选择规则在模拟中将最大样本大小减少了约25%.
- 与标准的MAMS设计相比,MAMS选择设计显示了较小的家族类型I错误率.
- 通过严格的选择规则,可以放松最后阶段的意义级别,以避免低于支出的I型错误;维持功率需要选择足够的武器子集.
结论:
- 与标准MAMS相比,MAMS选择设计通过减少总样本大小来提高效率.
- 精心预先规定的选择规则,意义级别和临时停止界限对于控制操作特征至关重要.
- 提供了关于这些设计特征的指导,以确保对试验性能进行可靠的控制.
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