4.5 Article

Predicting the restricted mean event time with the subject's baseline covariates in survival analysis

Journal

BIOSTATISTICS
Volume 15, Issue 2, Pages 222-233

Publisher

OXFORD UNIV PRESS
DOI: 10.1093/biostatistics/kxt050

Keywords

Accelerated failure time model; Cox model; Cross-validation; Hold-out sample; Personalized medicine; Perturbation-resampling method

Funding

  1. National Institutes of Health [R01 AI052817, RC4 CA155940, U01 AI068616, UM1 AI068634, R01 AI024643, U54 LM008748, R01 HL089778]

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For designing, monitoring, and analyzing a longitudinal study with an event time as the outcome variable, the restricted mean event time (RMET) is an easily interpretable, clinically meaningful summary of the survival function in the presence of censoring. The RMET is the average of all potential event times measured up to a time point tau and can be estimated consistently by the area under the Kaplan-Meier curve over [0, tau]. In this paper, we study a class of regression models, which directly relates the RMET to its baseline covariates for predicting the future subjects' RMETs. Since the standard Cox and the accelerated failure time models can also be used for estimating such RMETs, we utilize a cross-validation procedure to select the best among all the working models considered in the model building and evaluation process. Lastly, we draw inferences for the predicted RMETs to assess the performance of the final selected model using an independent data set or a hold-out sample from the original data set. All the proposals are illustrated with the data from the an HIV clinical trial conducted by the AIDS Clinical Trials Group and the primary biliary cirrhosis study conducted by the Mayo Clinic.

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