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arXiv · 2407.18389

Doubly Robust Targeted Estimation of Subgroup Average Treatment Effects for Time-to-event Outcomes with Competing Risks

Runjia Li·Victor B. Talisa·Chung-Chou H. Chang
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Abstract

In recent years, precision treatment strategies have gained significant attention in medical research, particularly for patient care. We propose a novel framework for estimating subgroup average treatment effects (ATE) in time-to-event data with competing risks, using ICU patients with sepsis as an illustrative example. Our approach, based on cumulative incidence functions and targeted maximum likelihood estimation (TMLE), achieves both asymptotic efficiency and double robustness. The primary contribution of this work lies in our derivation of the efficient influence function for the targeted causal parameter, subgroup ATE. We established the theoretical proofs for these properties, and subsequently confirmed them through simulations. Our TMLE framework is flexible, accommodating various regression and machine learning models, making it applicable in diverse scenarios. In order to identify variables contributing to treatment effect heterogeneity and to facilitate accurate estimation of subgroup ATE, we developed two distinct variable importance measures (VIMs). This work provides a powerful tool for optimizing personalized treatment strategies, furthering the pursuit of precision medicine.

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Runjia Li, Victor B. Talisa, Chung-Chou H. Chang. 2024-07-25. Doubly Robust Targeted Estimation of Subgroup Average Treatment Effects for Time-to-event Outcomes with Competing Risks. https://arxiv.org/abs/2407.18389

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