Weighted NPMLE for the Subdistribution of a Competing Risk

J Am Stat Assoc. 2019;114(525):259-270. doi: 10.1080/01621459.2017.1401540. Epub 2018 Jul 9.

Abstract

Direct regression modeling of the subdistribution has become popular for analyzing data with multiple, competing event types. All general approaches so far are based on non-likelihood based procedures and target covariate effects on the subdistribution. We introduce a novel weighted likelihood function that allows for a direct extension of the Fine-Gray model to a broad class of semiparametric regression models. The model accommodates time-dependent covariate effects on the subdistribution hazard. To motivate the proposed likelihood method, we derive standard nonparametric estimators and discuss a new interpretation based on pseudo risk sets. We establish consistency and asymptotic normality of the estimators and propose a sandwich estimator of the variance. In comprehensive simulation studies we demonstrate the solid performance of the weighted NPMLE in the presence of independent right censoring. We provide an application to a very large bone marrow transplant dataset, thereby illustrating its practical utility.

Keywords: Fine-Gray model; cumulative incidence function; nonparametric maximum likelihood estimation; semiparametric transformation models; time-varying covariates.