集群随机试验中的受限平均存活时间:改进从伪值回归中对干预效应的差异估计
Floriane Le Vilain-Abraham1,2, Solène Desmée1, Jennifer A Thompson3
1Univ Tours, Nantes Université, INSERM, MethodS in Patients-centered outcomes and HEalth Research, SPHERE, Tours, France.
Statistical methods in medical research
|January 12, 2026
概括
本研究引入了改进的统计方法,用于分析集群随机试验 (CRT) 中的受限制平均生存时间 (RMST),集群少于50个. 建议的方法确保准确的干预效应估计和适当的I型错误控制在生存数据分析.
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) 量化了时间到事件试验中的干预效应.
- 集群随机试验 (CRT) 需要考虑集群内相关性.
- 在CRT中估计ΔRMST的先前方法已在≥50个集群中得到验证.
研究的目的:
- 开发和评估用于在CRT中估计ΔRMST的统计方法,其集群数量很少 (<50).
- 评估一般化估计方程 (GEE) 偏差校正方法的差异估计器.
- 为了比较GEE Wald测试的正常与学生t分布的性能.
主要方法:
- 模拟研究是根据比例和非比例危险假设进行的.
- 评估了GEE三明治差异估计器的四种偏差校正方法.
- 对比了Wald测试统计数据的正常和学生t分布的表现.
主要成果:
- 与正常分布相比,Student t-分布显示出对I型错误率的优越控制.
- 费伊和格劳巴德偏差校正的差异估计器在所有集群号中保持了适当的I型错误率.
- 建议使用GEE进行伪值回归,并结合Fay和Graubard校正和Student t分布.
结论:
- 提出的方法提供了可靠的估计干预效应在CRT的少数集群.
- 在CRT中推使用Fay和Graubard差异估计器和Student t分布进行伪值回归.
- 这些方法在具有挑战性的试验设计中增强了对生存数据的分析,正如DEMETER试验所示.
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