Targeted learning of heterogeneous treatment effect curves for right censored or left truncated time-to-event data

AI in healthcare
Published: arXiv: 2603.26502v1
Authors

Matthew Pryce Karla Diaz-Ordaz Ruth H. Keogh Stijn Vansteelandt

Abstract

In recent years, there has been growing interest in causal machine learning estimators for quantifying subject-specific effects of a binary treatment on time-to-event outcomes. Estimation approaches have been proposed which attenuate the inherent regularisation bias in machine learning predictions, with each of these estimators addressing measured confounding, right censoring, and in some cases, left truncation. However, the existing approaches are found to exhibit suboptimal finite-sample performance, with none of the existing estimators fully leveraging the temporal structure of the data, yielding non-smooth treatment effects over time. We address these limitations by introducing surv-iTMLE, a targeted learning procedure for estimating the difference in the conditional survival probabilities under two treatments. Unlike existing estimators, surv-iTMLE accommodates both left truncation and right censoring while enforcing smoothness and boundedness of the estimated treatment effect curve over time. Through extensive simulation studies under both right censoring and left truncation scenarios, we demonstrate that surv-iTMLE outperforms existing methods in terms of bias and smoothness of time-varying effect estimates in finite samples. We then illustrate surv-iTMLE's practical utility by exploring heterogeneity in the effects of immunotherapy on survival among non-small cell lung cancer (NSCLC) patients, revealing clinically meaningful temporal patterns that existing estimators may obscure.

Paper Summary

Problem
The main problem this research paper addresses is the challenge of estimating heterogeneous treatment effects for time-to-event data, which is common in medical research and personalized medicine. The paper focuses on right censored or left truncated data, which can lead to biased and irregular treatment effect estimates. The researchers aim to develop a new method, called surv-iTMLE, that can accurately estimate the difference in conditional survival probabilities under two treatments for a given patient.
Key Innovation
The key innovation of this work is the introduction of surv-iTMLE, a targeted learning procedure that combines machine learning with statistical inference to estimate the difference in conditional survival probabilities under two treatments. Unlike existing estimators, surv-iTMLE accommodates both left truncation and right censoring while enforcing smoothness and boundedness of the estimated treatment effect curve over time. This approach leverages sieve-based targeted learning, known as infinite-dimensional targeted minimum loss-based estimation (iTMLE), within a two-step pseudo-outcome construction.
Practical Impact
The practical impact of this research is significant, as it can be applied to various medical and social science applications where treatment effects are heterogeneous and time-to-event data are involved. The researchers demonstrate the utility of surv-iTMLE by exploring heterogeneity in the effects of immunotherapy on survival among non-small cell lung cancer (NSCLC) patients. The results reveal clinically meaningful temporal patterns that existing estimators may obscure. This can inform personalized treatment decisions, improve health equity, and inform policy decisions.
Analogy / Intuitive Explanation
Imagine trying to estimate how a new medicine affects a patient's survival over time. Traditional methods might struggle with data that is incomplete or biased, leading to irregular estimates. surv-iTMLE is like a new pair of glasses that can help correct these biases and provide a clearer picture of how the medicine affects the patient. By using machine learning and statistical inference together, surv-iTMLE can accurately estimate the difference in conditional survival probabilities under two treatments, helping healthcare professionals make informed decisions about patient care.
Paper Information
Categories:
stat.ME stat.AP stat.ML
Published Date:

arXiv ID:

2603.26502v1

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