Finding the best subgroup with differential treatment effect with multiple outcomes.
Stat Med
; 43(13): 2487-2500, 2024 Jun 15.
Article
em En
| MEDLINE
| ID: mdl-38621856
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.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Infecções Urinárias
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Simulação por Computador
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Medicina de Precisão
Limite:
Child
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Humans
Idioma:
En
Revista:
Stat Med
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos