在量化绝对治疗效应异质性时,从风险差异和受限平均生存时间的差异中得出不同的结论
Carolien C H M Maas1,2,3, David M Kent3, Avinash G Dinmohamed1,2
1Department of Public Health, Erasmus University Medical Center, Rotterdam, Netherlands.
International journal of epidemiology
|November 29, 2025
概括
限制平均存活时间 (ΔRMST) 的差异比风险差异 (RD) 建议用于评估时间到事件数据中的绝对异质治疗效应,因为RD可能导致治疗的错误向.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 生存分析的分析.
背景情况:
- 基于风险的分析越来越多地用于临床试验中的异质治疗效应 (HTEs).
- 对于整个风险层的绝对治疗效应,时间到事件分析的传统假设可能不成立.
- 绝对治疗效应可以通过风险差异 (RD) 或受限平均存活时间 (ΔRMST) 的差异来衡量.
研究的目的:
- 在时间到事件数据中检查基于风险的HTE分析.
- 通过风险层来识别绝对HTE的模式.
- 为了比较 ΔRMST 与 RD 在指导治疗决策中的实用性.
主要方法:
- 利用人工和实证的时间到事件数据.
- 在风险层中比较RD (Kaplan-Meier估计在某个时间点的差异) 和ΔRMST (Kaplan-Meier曲线之间的面积).
- 探索了具有恒定危险比率 (HRs),变化事件率和风险模型歧视的场景.
主要成果:
- 当事件率和歧视低时,RD和DRMST都显示单调增加,有利于高风险患者.
- 随着事件发生率和/或歧视的增加,出现了一个"甜点"模式,有利于中等风险患者.
- 即使在 ΔRMST 偏好较高风险患者时,RD 也发现了"甜蜜点"模式,这可能会低估益处并导致错误准.
结论:
- 通过 RD 评估的 HTE 模式可能与 ΔRMST 有显著差异.
- 使用RD可能会导致低于最佳的治疗决策和治疗的错误向.
- 推 ΔRMST 用于在时间到事件数据中评估绝对 HTE.
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