Sofer Tamar, Cornelis Marilyn C, Kraft Peter, Tchetgen Tchetgen Eric J
University of Washington and Harvard T.H. Chan School of Public Health.
Stat Sin. 2017 Apr;27(2):785-804. doi: 10.5705/ss.202015.0116.
Case-control studies are designed towards studying associations between risk factors and a single, primary outcome. Information about additional, secondary outcomes is also collected, but association studies targeting such secondary outcomes should account for the case-control sampling scheme, or otherwise results may be biased. Often, one uses inverse probability weighted (IPW) estimators to estimate population effects in such studies. IPW estimators are robust, as they only require correct specification of the mean regression model of the secondary outcome on covariates, and knowledge of the disease prevalence. However, IPW estimators are inefficient relative to estimators that make additional assumptions about the data generating mechanism. We propose a class of estimators for the effect of risk factors on a secondary outcome in case-control studies that combine IPW with an additional modeling assumption: specification of the disease outcome probability model. We incorporate this model via a mean zero control function. We derive the class of all regular and asymptotically linear estimators corresponding to our modeling assumption, when the secondary outcome mean is modeled using either the identity or the log link. We find the efficient estimator in our class of estimators and show that it reduces to standard IPW when the model for the primary disease outcome is unrestricted, and is more efficient than standard IPW when the model is either parametric or semiparametric.
病例对照研究旨在研究风险因素与单一主要结局之间的关联。关于其他次要结局的信息也会被收集,但针对此类次要结局的关联研究应考虑病例对照抽样方案,否则结果可能会有偏差。通常,人们在这类研究中使用逆概率加权(IPW)估计量来估计总体效应。IPW估计量具有稳健性,因为它们只要求正确设定次要结局关于协变量的均值回归模型,以及疾病患病率的知识。然而,相对于对数据生成机制做出额外假设的估计量,IPW估计量效率较低。我们提出了一类用于病例对照研究中风险因素对次要结局影响的估计量,该估计量将IPW与一个额外的建模假设相结合:疾病结局概率模型的设定。我们通过一个均值为零的控制函数纳入这个模型。当使用恒等或对数连接对次要结局均值进行建模时,我们推导了与我们的建模假设相对应的所有正则且渐近线性估计量的类别。我们在我们的估计量类别中找到了有效估计量,并表明当主要疾病结局的模型不受限制时,它会简化为标准IPW,而当模型为参数模型或半参数模型时,它比标准IPW更有效。