On restricted optimal treatment regime estimation for competing risks data

It is well accepted that individualized treatment regimes may improve the clinical outcomes of interest. However, positive treatment effects are often accompanied by certain side effects. Therefore, when choosing the optimal treatment regime for a patient, we need to consider both efficacy and safety issues. In this article, we propose to model time to a primary event of interest and time to severe side effects of treatment by a competing risks model and define a restricted optimal treatment regime based on cumulative incidence functions. The estimation approach is derived using a penalized value search method and investigated through extensive simulations. The proposed method is applied to an HIV dataset obtained from Health Sciences South Carolina, where we minimize the risk of treatment or virologic failures while controlling the risk of serious drug-induced side effects.

Automated summary

This study proposes a restricted optimal treatment regime based on competing risks model and cumulative incidence functions, which is estimated using a penalized value search method and investigated through simulations. The method is applied to an HIV dataset to minimize the risk of treatment or virologic failures while controlling the risk of serious drug-induced side effects.