将HIV临床试验的生存率转移至外部目标人群
Transporting survival of an HIV clinical trial to the external target populations
摘要：
由于随机对照试验（RCT）和外部目标人群的异质性，RCT估计的处理效应不直接适用于目标人群。例如，ACTG 175 HIV试验的患者特征与三个关注的外部目标人群（美国早期HIV患者、泰国HIV患者和埃塞俄比亚南部HIV患者）的患者特征显著不同。本文考虑了将ACTG 175 HIV试验的处理效应转移至试验人群以外的目标人群的几种方法。大多数转运方法关注连续和二元结果;相反，我们推导并讨论了几种用于生存结局的转运方法：基于Cox比例风险（PH）模型的结果回归方法，基于治疗分配、抽样评分和删失的模型的逆概率加权方法，以及结合两种方法的双重稳健方法，增强校准加权法（ACW）。然而，由于发现PH假设对于ACTG 175试验是不正确的，因此依赖于PH假设的方法可能导致处理效应的偏倚量化。为了解释违反PH假设，我们扩展了ACW方法与线性样条为基础的风险回归模型，不需要PH假设。应用上述可移植性方法，我们探索了PH假设或违反PH假设对将ACTG 175试验的生存结果转移到各种外部人群的影响。
英文摘要：
Due to the heterogeneity of the randomized controlled trial (RCT) and external target populations, the estimated treatment effect from the RCT is not directly applicable to the target population. For example, the patient characteristics of the ACTG 175 HIV trial are significantly different from that of the three external target populations of interest: US early-stage HIV patients, Thailand HIV patients, and southern Ethiopia HIV patients. This paper considers several methods to transport the treatment effect from the ACTG 175 HIV trial to the target populations beyond the trial population. Most transport methods focus on continuous and binary outcomes; on the contrary, we derive and discuss several transport methods for survival outcomes: an outcome regression method based on a Cox proportional hazard (PH) model, an inverse probability weighting method based on the models for treatment assignment, sampling score, and censoring, and a doubly robust method that combines both methods, called the augmented calibration weighting (ACW) method. However, as the PH assumption was found to be incorrect for the ACTG 175 trial, the methods that depend on the PH assumption may lead to the biased quantification of the treatment effect. To account for the violation of the PH assumption, we extend the ACW method with the linear spline-based hazard regression model that does not require the PH assumption. Applying the aforementioned methods for transportability, we explore the effect of PH assumption, or the violation thereof, on transporting the survival results from the ACTG 175 trial to various external populations.
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