Two-sample inference procedures under nonproportional hazards.
Two-sample inference procedures under nonproportional hazards.
复制标题
非比例风险下的二样本推理程序。
DOI：
10.1002/pst.2324
复制
发表时间：
2023
影响因子：
1.5
通讯作者：
Wells,MartinT
中科院分区：
文献类型：
--
作者：
Tai,Yi-Cheng;Wang,Weijing;Wells,MartinT
我们引入了一个新的双样本推理程序，以评估两组随时间的相对表现。我们的无模型方法不假设比例风险，因此适用于可能存在非比例风险的情况。我们的程序包括一个诊断tau图，以确定危险的时间和正式的推理程序的变化。我们开发的基于tau的指标具有临床意义，并提供了可解释的被估量，以总结随时间推移的治疗效果。我们提出的统计量是一个U统计量，并表现出鞅结构，使我们能够构建置信区间并进行假设检验。我们的方法是强大的截尾分布。我们还展示了我们的方法如何应用于由于随访不足而缺失尾部信息的情况下的敏感性分析。在没有删失的情况下，我们提出的Kendall的tau估计量减少到Wilcoxon-Mann-Whitney统计量。我们使用模拟来评估我们的方法，以将其性能与受限平均生存时间和对数秩统计量进行比较。我们还将我们的方法应用于可能存在非比例风险的几项已发表的肿瘤学临床试验的数据。
We introduce a new two‐sample inference procedure to assess the relative performance of two groups over time. Our model‐free method does not assume proportional hazards, making it suitable for scenarios where nonproportional hazards may exist. Our procedure includes a diagnostic tau plot to identify changes in hazard timing and a formal inference procedure. The tau‐based measures we develop are clinically meaningful and provide interpretable estimands to summarize the treatment effect over time. Our proposed statistic is a U‐statistic and exhibits a martingale structure, allowing us to construct confidence intervals and perform hypothesis testing. Our approach is robust with respect to the censoring distribution. We also demonstrate how our method can be applied for sensitivity analysis in scenarios with missing tail information due to insufficient follow‐up. Without censoring, Kendall's tau estimator we propose reduces to the Wilcoxon‐Mann–Whitney statistic. We evaluate our method using simulations to compare its performance with the restricted mean survival time and log‐rank statistics. We also apply our approach to data from several published oncology clinical trials where nonproportional hazards may exist.