Analysis of clustered competing risks data using subdistribution hazard models with multivariate frailties

Stat Methods Med Res. 2016 Dec;25(6):2488-2505. doi: 10.1177/0962280214526193. Epub 2014 Mar 11.

Abstract

Competing risks data often exist within a center in multi-center randomized clinical trials where the treatment effects or baseline risks may vary among centers. In this paper, we propose a subdistribution hazard regression model with multivariate frailty to investigate heterogeneity in treatment effects among centers from multi-center clinical trials. For inference, we develop a hierarchical likelihood (or h-likelihood) method, which obviates the need for an intractable integration over the frailty terms. We show that the profile likelihood function derived from the h-likelihood is identical to the partial likelihood, and hence it can be extended to the weighted partial likelihood for the subdistribution hazard frailty models. The proposed method is illustrated with a dataset from a multi-center clinical trial on breast cancer as well as with a simulation study. We also demonstrate how to present heterogeneity in treatment effects among centers by using a confidence interval for the frailty for each individual center and how to perform a statistical test for such heterogeneity using a restricted h-likelihood.

Keywords: competing risks; hierarchical likelihood; multivariate frailty; random treatment-by-center interaction; subdistribution hazard.

MeSH terms

  • Breast Neoplasms / drug therapy
  • Computer Simulation
  • Humans
  • Likelihood Functions*
  • Middle Aged
  • Multicenter Studies as Topic
  • Multivariate Analysis*
  • Proportional Hazards Models*
  • Randomized Controlled Trials as Topic
  • Risk
  • Tamoxifen / therapeutic use

Substances

  • Tamoxifen