Cui Yifan, Zhu Ruoqing, Kosorok Michael
Department of Statistics and Operations Research, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Department of Statistics, University of Illinois at Urbana-Champaign, Champaign, IL 61820, USA.
Electron J Stat. 2017;11(2):3927-3953. doi: 10.1214/17-EJS1305. Epub 2017 Oct 18.
Estimating individualized treatment rules is a central task for personalized medicine. [23] and [22] proposed outcome weighted learning to estimate individualized treatment rules directly through maximizing the expected outcome without modeling the response directly. In this paper, we extend the outcome weighted learning to right censored survival data without requiring either inverse probability of censoring weighting or semiparametric modeling of the censoring and failure times as done in [26]. To accomplish this, we take advantage of the tree based approach proposed in [28] to nonparametrically impute the survival time in two different ways. The first approach replaces the reward of each individual by the expected survival time, while in the second approach only the censored observations are imputed by their conditional expected failure times. We establish consistency and convergence rates for both estimators. In simulation studies, our estimators demonstrate improved performance compared to existing methods. We also illustrate the proposed method on a phase III clinical trial of non-small cell lung cancer.
估计个体化治疗规则是个性化医疗的核心任务。[23]和[22]提出了结果加权学习法,通过最大化预期结果直接估计个体化治疗规则,而无需直接对反应进行建模。在本文中,我们将结果加权学习法扩展到右删失生存数据,无需像[26]那样进行删失加权的逆概率计算或对删失时间和失败时间进行半参数建模。为实现这一点,我们利用[28]中提出的基于树的方法,以两种不同方式对生存时间进行非参数估计。第一种方法用预期生存时间替代每个个体的奖励,而在第二种方法中,仅对删失观测值用其条件预期失败时间进行估计。我们建立了两种估计器的一致性和收敛速度。在模拟研究中,与现有方法相比,我们的估计器表现出更好的性能。我们还在一项非小细胞肺癌的III期临床试验中展示了所提出 的方法。