학술논문

Finding the best subgroup with differential treatment effect with multiple outcomes.
Document Type
Article
Source
Statistics in Medicine. 6/15/2024, Vol. 43 Issue 13, p2487-2500. 14p.
Subject
*TREATMENT effectiveness
*URINARY tract infections
*TREATMENT effect heterogeneity
*INDIVIDUALIZED medicine
Language
ISSN
0277-6715
Abstract
Precision medicine aims to identify specific patient subgroups that may benefit the most from a particular treatment than the whole population. Existing definitions for the best subgroup in subgroup analysis are based on a single outcome and do not consider multiple outcomes; specifically, outcomes of different types. In this article, we introduce a definition for the best subgroup under a multiple‐outcome setting with continuous, binary, and censored time‐to‐event outcomes. Our definition provides a trade‐off between the subgroup size and the conditional average treatment effects (CATE) in the subgroup with respect to each of the outcomes while taking the relative contribution of the outcomes into account. We conduct simulations to illustrate the proposed definition. By examining the outcomes of urinary tract infection and renal scarring in the RIVUR clinical trial, we identify a subgroup of children that would benefit the most from long‐term antimicrobial prophylaxis. [ABSTRACT FROM AUTHOR]