计划阶段子集群随机试验,以检测治疗效果异质性
Fan Li1,2, Xinyuan Chen3, Zizhong Tian4
1Department of Biostatistics, Yale University School of Public Health, New Haven, Connecticut, USA.
Statistics in medicine
|December 20, 2023
概括
这项研究引入了阶段形试验的新方法,以评估平均和小组治疗效果. 这些方法改善了样本大小计算,并优化了集群分配,以进行可靠的干预评估.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 阶梯形设计越来越多地用于干预评估.
- 现有的分析方法主要集中在平均治疗效果上.
- 需要方法来评估在阶段形试验中的治疗效果异质性.
研究的目的:
- 导出差异公式来分析阶段形设计中的治疗效应异质性.
- 提供样本大小和设计配置的方法,以评估平均和异质处理效应.
- 为高效的平均治疗效应分析提供一个框架,使用共变量调整.
主要方法:
- 开发用于治疗效果异质性的确认性分析的新变异公式.
- 该框架应用于横截面和闭合队列阶梯设计.
- 使用模拟研究和现实世界试验数据 (腰椎成像与流行病学试验报告) 进行验证.
主要成果:
- 获得治疗效果异质性的新变异公式.
- 该框架允许通过共变量调整进行高效的平均治疗效果分析.
- 确定最佳的集群分配,以最大限度地提高平均和异质效应的精度.
结论:
- 开发的方法填补了阶段形试验设计和分析中的关键缺口.
- 这些方法支持对患者亚群干预效应的严格评估.
- 这些发现提高了阶段形试验规划和执行的精度和效率.
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