量化不同风险层的时间到事件结果的绝对治疗效应异质性:结论的分歧与风险差异和受限制的平均生存时间差异差异
medRxiv : the preprint server for health sciences
|January 7, 2025
概括
限制性平均存活时间 (ΔRMST) 的差异比风险差异 (RD) 建议在时间到事件数据中评估绝对异质治疗效应 (HTE). ΔRMST提供了更准确,更全面的治疗效益的衡量,跨患者风险组.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 医疗决策的制定 医疗决策的制定
背景情况:
- 基于风险的分析对于了解临床试验中异质治疗效应 (HTE) 至关重要.
- 关于高风险患者受益最多的传统假设在时间到事件分析中可能不适用于绝对治疗效应.
- 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.
- 确定 ΔRMST 是否导致比 RD 更有意义的治疗决策.
主要方法:
- 在风险层中使用人工和经验时间到事件数据比较RD和ΔRMST.
- 探索了具有恒定危险比率 (HRs),变化的事件率和风险模型歧视的场景.
- 评估这些指标如何优先考虑不同患者子组的治疗.
主要成果:
- 当事件率和歧视低时,DR和DRMST在高风险患者中都显示出更高的益处.
- 随着事件发生率/歧视的增加,出现了一个"甜点"模式,中等风险患者受益最多.
- 在增加事件率和/或歧视的情况下,RD低估了高风险患者的益处.
结论:
- 通过RD识别的HTE模式可能与ΔRMST显著不同,可能导致治疗向不正确.
- 由于其全面性和准确性,建议在时间到事件数据中评估绝对HTE.
- 与RD相比,ΔRMST更直观,不那么依赖时间地平线,更好地捕捉不同的HR.
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