在临床试验中,使用受限平均存活时间作为主要终点的组序列设计
Zhaojin Li1, Xiang Geng1, Yawen Hou2
1Department of Biostatistics, School of Public Health (Guangdong Provincial Key Laboratory of Tropical Disease Research), Southern Medical University, Guangzhou, China.
Statistical methods in medical research
|January 20, 2025
概括
使用受限平均存活时间 (GS-RMST) 的新组序列设计在临床试验中解决不成比例的危险 (NPH). 这些方法提高了解释性,并保持了统计能力,为日志等级测试提供了替代方案.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 生存分析的分析.
背景情况:
- 在临床试验中,比例危险 (PH) 假设经常被违反,使危险比率的解释变得复杂.
- 在试验设计中使用的传统日志级测试可以导致在非比例危险 (NPH) 下的功率损失或通胀.
研究的目的:
- 引入两种基于受限平均存活时间 (GS-RMST) 的新型无模拟组序列设计,以解决NPH.
- 为这些新设计提供计算停车界限和样本大小的方法.
- 为临床试验结果提供可解释的效果指标.
主要方法:
- 开发独立增量GS-RMST (GS-RMSTi) 和非独立增量GS-RMST (GS-RMSTn) 的设计.
- 对两种设计的方差-共方差矩阵的分析表达式的推导.
- 通过模拟研究对设计进行评估,以评估I型错误和功率.
主要成果:
- 两种GS-RMSTi和GS-RMSTn设计都实现了I型名义误差和功率.
- GS-RMSTn Max-Combo测试在NPH场景中证明了检测治疗差异的稳定性.
- GS-RMST设计提供了适合临床医生和患者的易于解释的时间尺度效果测量.
结论:
- 有限制的平均存活时间组顺序设计为具有不成比例风险的临床试验提供了可行的解决方案.
- 与传统方法相比,这些方法提高了治疗效果测量的解释性.
- 在临床试验规划中,GS-RMST设计适用于比例和非比例的危险场景.
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