Wang Jixian
Celgene International Sarl, Boudry, Switzerland.
Pharm Stat. 2018 Feb;17(1):38-48. doi: 10.1002/pst.1834. Epub 2017 Nov 1.
Survival functions are often estimated by nonparametric estimators such as the Kaplan-Meier estimator. For valid estimation, proper adjustment for confounding factors is needed when treatment assignment may depend on confounding factors. Inverse probability weighting is a commonly used approach, especially when there is a large number of potential confounders to adjust for. Direct adjustment may also be used if the relationship between the time-to-event and all confounders can be modeled. However, either approach requires a correctly specified model for the relationship between confounders and treatment allocation or between confounders and the time-to-event. We propose a pseudo-observation-based doubly robust estimator, which is valid when either the treatment allocation model or the time-to-event model is correctly specified and is generally more efficient than the inverse probability weighting approach. The approach can be easily implemented using standard software. A simulation study was conducted to evaluate this approach under a number of scenarios, and the results are presented and discussed. The results confirm robustness and efficiency of the proposed approach. A real data example is also provided for illustration.
生存函数通常由非参数估计器(如Kaplan-Meier估计器)进行估计。为了进行有效的估计,当治疗分配可能取决于混杂因素时,需要对混杂因素进行适当调整。逆概率加权是一种常用的方法,特别是当有大量潜在混杂因素需要调整时。如果可以对事件发生时间与所有混杂因素之间的关系进行建模,也可以使用直接调整。然而,这两种方法都需要为混杂因素与治疗分配之间或混杂因素与事件发生时间之间的关系正确指定模型。我们提出了一种基于伪观测的双重稳健估计器,当治疗分配模型或事件发生时间模型正确指定时,该估计器是有效的,并且通常比逆概率加权方法更有效。该方法可以使用标准软件轻松实现。进行了一项模拟研究,以在多种情况下评估该方法,并展示和讨论了结果。结果证实了所提出方法的稳健性和有效性。还提供了一个实际数据示例进行说明。