Targeted learning ensembles for optimal individualized treatment rules with time-to-event outcomes

DOI10.1093/BIOMET/ASY017zbMATH Open1499.62117arXiv1702.04682OpenAlexW2964077653WikidataQ91871272 ScholiaQ91871272MaRDI QIDQ4561024FDOQ4561024

Authors:

Publication date: 10 December 2018

Published in: Biometrika (Search for Journal in Brave)

Abstract: We consider estimation of an optimal individualized treatment rule from observational and randomized studies when a high-dimensional vector of baseline variables is available. Our optimality criterion is with respect to delaying expected time to occurrence of an event of interest (e.g., death or relapse of cancer). We leverage semiparametric efficiency theory to construct estimators with desirable properties such as double robustness. We propose two estimators of the optimal rule, which arise from considering two loss functions aimed at (i) directly estimating the conditional treatment effect (also know as the blip function), and (ii) recasting the problem as a weighted classification problem that uses the 0-1 loss function. Our estimated rules are super learning ensembles that minimize the cross-validated risk of a linear combination in a user-supplied library of candidate estimators. We prove oracle inequalities bounding the finite sample excess risk of the estimator. The bounds depend on the excess risk of the oracle selector and a doubly robust term related to estimation of the nuisance parameters. We discuss some important implications of these oracle inequalities such as the convergence rates of the value of our estimator to that of the oracle selector. We illustrate our methods in the analysis of a phase III randomized study testing the efficacy of a new therapy for the treatment of breast cancer.

Full work available at URL: https://arxiv.org/abs/1702.04682

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zbMATH Keywords

double robustnessoracle inequalitysuper-learningindividualized treatment rule

Mathematics Subject Classification ID

Nonparametric estimation (62G05) Censored data models (62N01) Applications of statistics to biology and medical sciences; meta analysis (62P10)

Cited In (11)

• Targeted sequential design for targeted learning inference of the optimal treatment rule and its mean reward
• Transformation-Invariant Learning of Optimal Individualized Decision Rules with Time-to-Event Outcomes
• Estimation of optimal individualized treatment rules using a covariate-specific treatment effect curve with high-dimensional covariates
• Semiparametric single-index models for optimal treatment regimens with censored outcomes
• On estimating optimal regime for treatment initiation time based on restricted mean residual lifetime
• Doubly robust learning for estimating individualized treatment with censored data
• Towards optimal doubly robust estimation of heterogeneous causal effects
• Minimum Resource Threshold Policy Under Partial Interference
• Modern approaches for evaluating treatment effect heterogeneity from clinical trials and observational data
• Resampling‐based confidence intervals for model‐free robust inference on optimal treatment regimes
• Statistical learning of origin-specific statically optimal individualized treatment rules

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