Kianian Behzad, Kim Jung In, Fine Jason P, Peng Limin
Department of Biostatistics and Bioinformatics, Emory University.
Departments of Biostatistics, University of North Carolina at Chapel Hill.
Stat Sin. 2021 Apr;31(2):673-699. doi: 10.5705/ss.202019.0096.
Instrumental variables (IV) are a useful tool for estimating causal effects in the presence of unmeasured confounding. IV methods are well developed for uncensored outcomes, particularly for structural linear equation models, where simple two-stage estimation schemes are available. The extension of these methods to survival settings is challenging, partly because of the nonlinearity of the popular survival regression models and partly because of the complications associated with right censoring or other survival features. Motivated by the Prostate, Lung, Colorectal and Ovarian (PLCO) Cancer screening trial, we develop a simple causal hazard ratio estimator in a proportional hazards model with right censored data. The method exploits a special characterization of IV which enables the use of an intuitive inverse weighting scheme that is generally applicable to more complex survival settings with left truncation, competing risks, or recurrent events. We rigorously establish the asymptotic properties of the estimators, and provide plug-in variance estimators. The proposed method can be implemented in standard software, and is evaluated through extensive simulation studies. We apply the proposed IV method to a data set from the Prostate, Lung, Colorectal and Ovarian cancer screening trial to delineate the causal effect of flexible sigmoidoscopy screening on colorectal cancer survival which may be confounded by informative noncompliance with the assigned screening regimen.
工具变量(IV)是在存在未测量混杂因素的情况下估计因果效应的有用工具。IV方法在无删失结局方面已经得到了很好的发展,特别是对于结构线性方程模型,其中有简单的两阶段估计方案。将这些方法扩展到生存分析场景具有挑战性,部分原因是流行的生存回归模型具有非线性,部分原因是与右删失或其他生存特征相关的复杂性。受前列腺、肺、结肠和卵巢(PLCO)癌症筛查试验的启发,我们在具有右删失数据的比例风险模型中开发了一种简单的因果风险比估计器。该方法利用了IV的一种特殊特征,使得能够使用一种直观的逆加权方案,该方案通常适用于具有左截断、竞争风险或复发事件的更复杂的生存分析场景。我们严格建立了估计器的渐近性质,并提供了插件方差估计器。所提出的方法可以在标准软件中实现,并通过广泛的模拟研究进行评估。我们将所提出的IV方法应用于前列腺、肺、结肠和卵巢癌筛查试验的数据集,以描述灵活的乙状结肠镜检查筛查对结直肠癌生存的因果效应,这可能会因对指定筛查方案的信息性不依从而产生混杂。