Doubly-robust estimators of treatment-specific survival distributions in observational studies with stratified sampling.

Doubly-robust estimators of treatment-specific survival distributions in observational studies with stratified sampling.
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DOI:
10.1111/biom.12076
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发表时间:
2013-12
期刊:
影响因子:
1.9
通讯作者:
O'Brien SM
O'Brien SM
中科院分区:
数学3区
文献类型:
--
作者:
Bai X;Tsiatis AA;O'Brien SM

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经常进行观察性研究以比较两种治疗对生存率的影响。对于此类研究,我们必须关注混杂因素;也就是说,存在影响治疗分配和生存分布的协变量。在存在混杂因素的情况下,通常的治疗特异性Kaplan-Meier估计量可能是基础治疗特异性生存分布的偏倚估计量。本文有两个目的。在第一个目标中,我们使用半参数理论推导出一个双稳健估计的治疗特异性生存分布的情况下,它被认为是所有潜在的混杂因素被捕获。在未捕获所有潜在混杂因素的情况下,可以使用分层抽样方案进行子研究,以捕获可能导致混杂的其他协变量。第二个目的是得到一个双稳健估计的治疗特异性生存分布和方差估计与这样的分层抽样方案。仿真研究表明,一致性和双重鲁棒性。然后将这些估计量应用于ASCERT研究的数据,从而激发了这项研究。
Observational studies are frequently conducted to compare the effects of two treatments on survival. For such studies we must be concerned about confounding; that is, there are covariates that affect both the treatment assignment and the survival distribution. With confounding the usual treatment-specific Kaplan-Meier estimator might be a biased estimator of the underlying treatment-specific survival distribution. This paper has two aims. In the first aim we use semiparametric theory to derive a doubly robust estimator of the treatment-specific survival distribution in cases where it is believed that all the potential confounders are captured. In cases where not all potential confounders have been captured one may conduct a substudy using a stratified sampling scheme to capture additional covariates that may account for confounding. The second aim is to derive a doubly-robust estimator for the treatment-specific survival distributions and its variance estimator with such a stratified sampling scheme. Simulation studies are conducted to show consistency and double robustness. These estimators are then applied to the data from the ASCERT study that motivated this research.
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