Journal of Data Science

Abstract: Observational studies of relatively large data can have potentially hidden heterogeneity with respect to causal effects and propensity scores–patterns of a putative cause being exposed to study subjects. This underlying heterogeneity can be crucial in causal inference for any observational studies because it is systematically generated and structured by covariates which influence the cause and/or its related outcomes. Addressing the causal inference problem in view of data structure, machine learning techniques such as tree analysis can be naturally necessitated. Kang, Su, Hitsman, Liu and Lloyd-Jones (2012) proposed Marginal Tree (MT) procedure to explore both the confounding and interacting effects of the covariates on causal inference. In this paper, we extend the MT method to the case of binary responses along with a clear exposition of its relationship with established causal odds ratio. We assess the causal effect of dieting on emotional distress using both a real data set from the Lalonde’s National Supported Work Demonstration Analysis (NSW) and a simulated data set from the National Longitudinal Study of Adolescent Health (Add Health).

For large observational studies lacking a control group (unlike randomized controlled trials, RCT), propensity scores (PS) are often the method of choice to account for pre-treatment confounding in baseline characteristics, and thereby avoid substantial bias in treatment estimation. A vast majority of PS techniques focus on average treatment effect estimation, without any clear consensus on how to account for confounders, especially in a multiple treatment setting. Furthermore, for time-to event outcomes, the analytical framework is further complicated in presence of high censoring rates (sometimes, due to non-susceptibility of study units to a disease), imbalance between treatment groups, and clustered nature of the data (where, survival outcomes appear in groups). Motivated by a right-censored kidney transplantation dataset derived from the United Network of Organ Sharing (UNOS), we investigate and compare two recent promising PS procedures, (a) the generalized boosted model (GBM), and (b) the covariate-balancing propensity score (CBPS), in an attempt to decouple the causal effects of treatments (here, study subgroups, such as hepatitis C virus (HCV) positive/negative donors, and positive/negative recipients) on time to death of kidney recipients due to kidney failure, post transplantation. For estimation, we employ a 2-step procedure which addresses various complexities observed in the UNOS database within a unified paradigm. First, to adjust for the large number of confounders on the multiple sub-groups, we fit multinomial PS models via procedures (a) and (b). In the next stage, the estimated PS is incorporated into the likelihood of a semi-parametric cure rate Cox proportional hazard frailty model via inverse probability of treatment weighting, adjusted for multi-center clustering and excess censoring, Our data analysis reveals a more informative and superior performance of the full model in terms of treatment effect estimation, over sub-models that relaxes the various features of the event time dataset.