Open Access
2024 A two-sample comparison of mean survival times of uncured subpopulations
Dennis Dobler, Eni Musta
Author Affiliations +
Electron. J. Statist. 18(2): 3107-3169 (2024). DOI: 10.1214/24-EJS2249

Abstract

Comparing the survival times among two groups is a common problem in time-to-event analysis, for example if one would like to understand whether one medical treatment is superior to another. In the standard survival analysis setting, there has been a lot of discussion on how to quantify such difference and what can be an intuitive, easily interpretable, summary estimand. In the presence of subjects that are immune to the event of interest (‘cured’), we illustrate that it is not appropriate to just compare the overall survival functions. Instead, it is more informative to compare the cure fractions and the survival of the uncured subpopulations separately from each other. Our research is mainly driven by the question: if the cure fraction is similar for two available treatments, how else can we determine which is preferable? To this end, we estimate the mean survival times in the uncured fractions of both treatment groups and develop both permutation and asymptotic tests for inference. We first propose a nonparametric approach which is then extended to account for covariates by means of the semi-parametric logistic-Cox mixture cure model. The methods are illustrated through practical applications to breast cancer and leukemia data.

Acknowledgments

The authors would like to thank Sarah Friedrich for useful comments regarding the breast cancer data set, Merle Munko for helpful comments about uniform Hadamard differentiability and Joris Mooij for insightful comments regarding the causal interpretation and the decision theoretic perspective on choosing between two treatments. Furthermore, Dennis Dobler was supported by his new affiliations, i.e., Department of Statistics at TU Dortmund University and Research Center Trustworthy Data Science and Security of University Alliance Ruhr. Also, they would like to thank two anonymous referees and an associate editor whose comments and suggestions helped to improve the manuscript considerably.

Citation

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Dennis Dobler. Eni Musta. "A two-sample comparison of mean survival times of uncured subpopulations." Electron. J. Statist. 18 (2) 3107 - 3169, 2024. https://doi.org/10.1214/24-EJS2249

Information

Received: 1 August 2023; Published: 2024
First available in Project Euclid: 30 July 2024

Digital Object Identifier: 10.1214/24-EJS2249

Subjects:
Primary: 62G09 , 62N02
Secondary: 62F12 , 62G20

Keywords: Asymptotic statistics , cure models , estimand , inference , logistic-Cox mixture model , random permutation , right-censoring

Vol.18 • No. 2 • 2024
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