Su Chien-Lin, Platt Robert W, Plante Jean-François
Department of Epidemiology, Biostatistics and Occupational Health, McGill University and Centre for Clinical Epidemiology, Lady Davis Institute, Jewish General Hospital, Montréal, Québec, Canada.
Department of Decision Sciences, HEC Montréal, Montréal, Québec, Canada.
Biostatistics. 2022 Jan 13;23(1):189-206. doi: 10.1093/biostatistics/kxaa020.
Recurrent event data are commonly encountered in observational studies where each subject may experience a particular event repeatedly over time. In this article, we aim to compare cumulative rate functions (CRFs) of two groups when treatment assignment may depend on the unbalanced distribution of confounders. Several estimators based on pseudo-observations are proposed to adjust for the confounding effects, namely inverse probability of treatment weighting estimator, regression model-based estimators, and doubly robust estimators. The proposed marginal regression estimator and doubly robust estimators based on pseudo-observations are shown to be consistent and asymptotically normal. A bootstrap approach is proposed for the variance estimation of the proposed estimators. Model diagnostic plots of residuals are presented to assess the goodness-of-fit for the proposed regression models. A family of adjusted two-sample pseudo-score tests is proposed to compare two CRFs. Simulation studies are conducted to assess finite sample performance of the proposed method. The proposed technique is demonstrated through an application to a hospital readmission data set.
复发事件数据在观察性研究中经常遇到,其中每个受试者可能会随着时间的推移反复经历特定事件。在本文中,当治疗分配可能取决于混杂因素的不平衡分布时,我们旨在比较两组的累积率函数(CRF)。提出了几种基于伪观测值的估计器来调整混杂效应,即治疗权重逆概率估计器、基于回归模型的估计器和双重稳健估计器。基于伪观测值提出的边际回归估计器和双重稳健估计器被证明是一致的且渐近正态的。提出了一种自举方法用于所提出估计器的方差估计。给出了残差的模型诊断图以评估所提出回归模型的拟合优度。提出了一族调整后的两样本伪得分检验来比较两个CRF。进行了模拟研究以评估所提出方法的有限样本性能。通过应用于医院再入院数据集展示了所提出的技术。