Garès Valérie, Chauvet Guillaume, Hajage David
Univ Rennes, INSA, CNRS, IRMAR - UMR 6625, F-35000, Rennes, France.
Univ Rennes, ENSAI, CNRS, IRMAR - UMR 6625, F-35000, Rennes, France.
Biom J. 2022 Jan;64(1):33-56. doi: 10.1002/bimj.202000267. Epub 2021 Jul 29.
Propensity score methods are widely used in observational studies for evaluating marginal treatment effects. The generalized propensity score (GPS) is an extension of the propensity score framework, historically developed in the case of binary exposures, for use with quantitative or continuous exposures. In this paper, we proposed variance estimators for treatment effect estimators on continuous outcomes. Dose-response functions (DRFs) were estimated through weighting on the inverse of the GPS, or using stratification. Variance estimators were evaluated using Monte Carlo simulations. Despite the use of stabilized weights, the variability of the weighted estimator of the DRF was particularly high, and none of the variance estimators (a bootstrap-based estimator, a closed-form estimator especially developed to take into account the estimation step of the GPS, and a sandwich estimator) were able to adequately capture this variability, resulting in coverages below the nominal value, particularly when the proportion of the variation in the quantitative exposure explained by the covariates was large. The stratified estimator was more stable, and variance estimators (a bootstrap-based estimator, a pooled linearized estimator, and a pooled model-based estimator) more efficient at capturing the empirical variability of the parameters of the DRF. The pooled variance estimators tended to overestimate the variance, whereas the bootstrap estimator, which intrinsically takes into account the estimation step of the GPS, resulted in correct variance estimations and coverage rates. These methods were applied to a real data set with the aim of assessing the effect of maternal body mass index on newborn birth weight.
倾向得分方法在观察性研究中被广泛用于评估边际治疗效果。广义倾向得分(GPS)是倾向得分框架的扩展,其历史上是在二元暴露的情况下发展起来的,用于定量或连续暴露。在本文中,我们提出了用于连续结果治疗效果估计量的方差估计量。剂量反应函数(DRF)通过对GPS的倒数进行加权估计,或使用分层方法进行估计。通过蒙特卡罗模拟对方差估计量进行评估。尽管使用了稳定权重,但DRF加权估计量的变异性特别高,并且没有一个方差估计量(基于自助法的估计量、专门为考虑GPS的估计步骤而开发的闭式估计量以及三明治估计量)能够充分捕捉这种变异性,导致覆盖率低于名义值,特别是当协变量解释的定量暴露变化比例较大时。分层估计量更稳定,并且方差估计量(基于自助法的估计量、合并线性化估计量和基于合并模型的估计量)在捕捉DRF参数的经验变异性方面更有效。合并方差估计量往往高估方差,而本质上考虑了GPS估计步骤的自助法估计量则能得到正确的方差估计和覆盖率。这些方法被应用于一个真实数据集,旨在评估孕妇体重指数对新生儿出生体重的影响。