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

竞争风险生存结局的亚组平均处理效应双重稳健目标估计

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…

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A Doubly Robust Instrumental Variable Approach for Estimating Average Treatment Effects in Time-to-Event Data with Unmeasured Confounding: Application to Real-World Data on ICU Patients with Septic Shock

未测混杂下生存结局平均处理效应的双重稳健工具变量方法

Motivated by conflicting conclusions regarding hydrocortisone's treatment effect on ICU patients with vasopressor-dependent septic shock, we developed a novel instrumental variable (IV) estimator to assess the average treatment effect (ATE) in time-to-event data. In real-world data, IV methods are widely used for estimating causal treatment effects in the presence of unmeasured confounding, but existing approaches for time-to-event outcomes are often constrained by strong…

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Sensitivity Analysis of Causal Treatment Effect Estimation for Clustered Observational Data with Unmeasured Confounding

聚类观察性数据中未测混杂下因果处理效应估计的敏感性分析

Identifying causal treatment (or exposure) effects in observational studies requires the data to satisfy the unconfoundedness assumption which is not testable using the observed data. With sensitivity analysis, one can determine how the conclusions might change if assumptions are violated to a certain degree. In this paper, we propose a new technique for sensitivity analysis applicable to clusters observational data with a normally distributed or binary outcome. The proposed…

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A Tree-based Model Averaging Approach for Personalized Treatment Effect Estimation from Heterogeneous Data Sources

异质数据来源下个性化处理效应的树模型平均方法

Accurately estimating personalized treatment effects within a study site (e.g., a hospital) has been challenging due to limited sample size. Furthermore, privacy considerations and lack of resources prevent a site from leveraging subject-level data from other sites. We propose a tree-based model averaging approach to improve the estimation accuracy of conditional average treatment effects (CATE) at a target site by leveraging models derived from other potentially…

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