在集群随机试验中估计干预效应的受限平均存活时间
Floriane Le Vilain-Abraham1, Elsa Tavernier1, Etienne Dantan2
1INSERM, SPHERE, U1246, Tours University, Nantes University, Tours, France.
Statistical methods in medical research
|August 10, 2023
概括
限制的平均存活时间 (RMST) 为测量干预效应在时间到事件数据中的危险比率提供了替代方案. 这项研究将RMST估计扩展到集群随机试验,提供可靠的方法来分析这些设计中的生存数据.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 流行病学 流行病学
背景情况:
- 限制平均存活时间 (RMST) 是对时间到事件分析中的干预效应的一个有价值的衡量标准.
- 现有的RMST估计方法主要设计用于独立数据.
- 集群随机试验 (CRT) 由于集群中的数据依赖性,存在独特的分析挑战.
研究的目的:
- 将现有的独立数据的RMST估计方法扩展到CRT中的集群数据.
- 通过模拟来评估和比较这些扩展的RMST方法的统计性能.
- 为使用RMST在CRT中分析时间到事件结果提供实用指导.
主要方法:
- 扩展的卡普兰-梅尔曲线集成和伪值回归用于集群数据中的RMST估计.
- 模拟研究用于评估各种CRT场景下的性能 (集群数量/大小,集群程度,效果大小,危险假设).
- 在只有少数集群 (<50) 的CRT中执行伪值回归的顺位测试.
主要成果:
- 扩展的RMST方法准确估计了变异和控制的I型错误,在比例和非比例危险下有足够的集群 (≥50).
- 转换试验对具有有限集群的CRT纠正了I型错误,从而产生了足够的置信区间覆盖.
- 伪值回归提供了共变量调整,这与其他方法相比是一种优势.
结论:
- 扩展的RMST估计方法对CRT具有统计学上的可靠性,为危险比率提供了可靠的替代方案.
- 方法的选择可能取决于集群的数量,对于较小的试验建议进行排列试验.
- 这些方法有助于在集群试验环境中对干预效应进行更全面的分析,正如喘教育计划试验所示.
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