On restricted mean time in favor of treatment

Lu Mao

Biometrics, 2023, vol. 79, issue 1, 61-72

Abstract: The restricted mean time in favor (RMT‐IF) of treatment is a nonparametric effect size for complex life history data. It is defined as the net average time the treated spend in a more favorable state than the untreated over a prespecified time window. It generalizes the familiar restricted mean survival time (RMST) from the two‐state life–death model to account for intermediate stages in disease progression. The overall estimand can be additively decomposed into stage‐wise effects, with the standard RMST as a component. Alternate expressions of the overall and stage‐wise estimands as integrals of the marginal survival functions for a sequence of landmark transitioning events allow them to be easily estimated by plug‐in Kaplan–Meier estimators. The dynamic profile of the estimated treatment effects as a function of follow‐up time can be visualized using a multilayer, cone‐shaped “bouquet plot.” Simulation studies under realistic settings show that the RMT‐IF meaningfully and accurately quantifies the treatment effect and outperforms traditional tests on time to the first event in statistical efficiency thanks to its fuller utilization of patient data. The new methods are illustrated on a colon cancer trial with relapse and death as outcomes and a cardiovascular trial with recurrent hospitalizations and death as outcomes. The R‐package rmt implements the proposed methodology and is publicly available from the Comprehensive R Archive Network (CRAN).

Date: 2023
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