Application of targeted maximum likelihood estimation in public health and epidemiological studies: a systematic review
目标最大似然估计在公共卫生与流行病学中的应用:系统综述AI 解读 ↓
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目标最大似然估计在公共卫生与流行病学中的应用:系统综述AI 解读 ↓
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The Targeted Maximum Likelihood Estimation (TMLE) statistical data analysis framework integrates machine learning, statistical theory, and statistical inference to provide a least biased, efficient and robust strategy for estimation and inference of a variety of statistical and causal parameters. We describe and evaluate the epidemiological applications that have benefited from recent methodological developments. We conducted a systematic literature review in PubMed for articles that applied any form of TMLE in observational studies. We summarised the epidemiological discipline, geographical location, expertise of the authors, and TMLE methods over time. We used the Roadmap of Targeted Learning and Causal Inference to extract key methodological aspects of the publications. We showcase the contributions to the literature of these TMLE results. Of the 81 publications included, 25% originated from the University of California at Berkeley, where the framework was first developed by Professor Mark van der Laan. By the first half of 2022, 70% of the publications originated from outside the United States and explored up to 7 different epidemiological disciplines in 2021-22. Double-robustness, bias reduction and model misspecification were the main motivations that drew researchers towards the TMLE framework. Through time, a wide variety of methodological, tutorial and software-specific articles were cited, owing to the constant growth of methodological developments around TMLE. There is a clear dissemination trend of the TMLE framework to various epidemiological disciplines and to increasing numbers of geographical areas. The availability of R packages, publication of tutorial papers, and involvement of methodological experts in applied publications have contributed to an exponential increase in the number of studies that understood the benefits, and adoption, of TMLE.
靶向深度生存对比:神经网络下处理特定生存收益的有效推断
Neural survival models are increasingly asked to support counterfactual claims---how much a treatment would change survival in a population---rather than only prognostic risk scores. Answering such questions from observational data requires valid inference for treatment-specific survival contrasts under confounding and covariate-dependent censoring, targets for which standard deep survival estimators are biased and provide no honest uncertainty. We propose Targeted Deep…
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…
通向同一答案的三条路径:协调 AIPW、TMLE 与双重机器学习
Augmented inverse-probability weighting (AIPW), targeted maximum likelihood estimation (TMLE), and double/debiased machine learning (DML) are three routes to the same efficient influence function for the average treatment effect --- settled theory we treat as background. This tutorial's contribution is its worked, shared-nuisance reconciliation on real data: what a practitioner must actually match for the routes, and the software packages, to agree. Working the effect of…
具有设计型方差的交叉拟合调查加权 TMLE
Cross-fitting is not a refinement of survey-weighted causal machine learning but, once the nuisances are flexible, what restores valid inference. We study the population average treatment effect under a stratified multistage design, estimated by a survey-aware targeted maximum likelihood estimator (TMLE) whose variance is obtained by Taylor-series linearization of the influence function, treating the primary sampling unit as the replication unit. Our central result is that…
作为观察数据效应估计工具的残差对残差回归
Epidemiologists increasingly use machine learning to adjust for high-dimensional confounding. Augmented inverse probability weighting (AIPW) and targeted maximum likelihood estimation (TMLE) are most widely used but may yield different results and both can become unstable under weak positivity violations. Residual-on-residual regression is a stable alternative that estimates an exposure effect encoded in a partially linear model by fitting confounder adjusted models for the…
利用大规模在线核学习估计双向因果效应
In this study, a scalable online kernel learning framework is proposed for estimating bidirectional causal effects in systems characterized by mutual dependence and heteroskedasticity. Traditional causal inference often focuses on unidirectional effects, overlooking the common bidirectional relationships in real-world phenomena. Building on heteroskedasticity-based identification, the proposed method integrates a quasi-maximum likelihood estimator for simultaneous equation…