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Calibrated Bayes analysis of cluster-randomized trials

整群随机试验的校准贝叶斯分析

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods have emerged as a strong alternative. Previous work has investigated the use of Bayesian hierarchical models for continuous, binary, and count outcomes in CRTs, but these approaches focus on model-based treatment effect coefficients as the…

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Doubly robust estimators of the restricted mean time in favor estimands in individual- and cluster-randomized trials

个体与整群随机试验中治疗占优限制平均时间的双重稳健估计

Progressive multi-state survival outcomes are common in trials with recurrent or sequential events and require treatment effect estimands that remain interpretable without proportional intensity or Markov assumptions. The restricted mean time in favor of treatment (RMT-IF) extends the restricted mean survival time to ordered multi-state processes and provides such an interpretable estimand. However, existing RMT-IF methods are nonparametric, assume covariate-independent…

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Estimands and doubly robust estimation for cluster-randomized trials with survival outcomes

生存结局聚类随机试验的目标参数与双重稳健估计

Cluster-randomized trials (CRTs) are experimental designs where groups or clusters of participants, rather than the individual participants themselves, are randomized to intervention groups. Analyzing CRT requires distinguishing between treatment effects at the cluster level and the individual level, which requires a clear definition of the estimands under the potential outcomes framework. For analyzing survival outcomes, it is common to assess the treatment effect by…

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Bayesian Sensitivity Analysis for Causal Estimation with Time-varying Unmeasured Confounding

时变未测量混杂下因果估计的贝叶斯敏感性分析

Causal inference relies on the untestable assumption of no unmeasured confounding. Sensitivity analysis can be used to quantify the impact of unmeasured confounding on causal estimates. Among sensitivity analysis methods proposed in the literature for unmeasured confounding, the latent confounder approach is favoured for its intuitive interpretation via the use of bias parameters to specify the relationship between the observed and unobserved variables and the sensitivity…

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A flexible Bayesian g-formula for causal survival analyses with time-dependent confounding

处理时变混杂的灵活贝叶斯生存分析 g 公式

In longitudinal observational studies with time-to-event outcomes, a common objective in causal analysis is to estimate the causal survival curve under hypothetical intervention scenarios. The g-formula is a useful tool for this analysis. To enhance the traditional parametric g-formula, we developed an alternative g-formula estimator, which incorporates the Bayesian Additive Regression Trees (BART) into the modeling of the time-evolving generative components, aiming to…

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Doubly robust estimation and sensitivity analysis for marginal structural quantile models

边际结构分位数模型的双重稳健估计与敏感性分析

The marginal structure quantile model (MSQM) provides a unique lens to understand the causal effect of a time-varying treatment on the full distribution of potential outcomes. Under the semiparametric framework, we derive the efficiency influence function for the MSQM, from which a new doubly robust estimator is proposed for point estimation and inference. We show that the doubly robust estimator is consistent if either of the models associated with treatment assignment or…

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A new method for clustered survival data]{A new method for clustered survival data: Estimation of treatment effect heterogeneity and variable selection

集群生存数据的新方法:处理效应异质性估计与变量选择

We recently developed a new method riAFT-BART to draw causal inferences about population treatment effect on patient survival from clustered and censored survival data while accounting for the multilevel data structure. The practical utility of this method goes beyond the estimation of population average treatment effect. In this work, we exposit how riAFT-BART can be used to solve two important statistical questions with clustered survival data: estimating the treatment…

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Estimating the causal effects of multiple intermittent treatments with application to COVID-19

多种间歇性治疗的因果效应估计及其新冠应用

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying confounding and censoring. Our methods formulate complex longitudinal treatments with multiple start/stop switches as the recurrent events with discontinuous intervals of treatment eligibility. We derive the weights in continuous time to handle…

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CIMTx: An R package for causal inference with multiple treatments using observational data

CIMTx:利用观测数据进行多处理因果推断的 R 包

CIMTx provides efficient and unified functions to implement modern methods for causal inferences with multiple treatments using observational data with a focus on binary outcomes. The methods include regression adjustment, inverse probability of treatment weighting, Bayesian additive regression trees, regression adjustment with multivariate spline of the generalized propensity score, vector matching and targeted maximum likelihood estimation. In addition, CIMTx illustrates…

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A flexible approach for causal inference with multiple treatments and clustered survival outcomes

多重处理与聚类生存结局的灵活因果推断方法

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured confounding for causal analyses. Few off-the-shelf causal inference tools are available to simultaneously tackle these issues. We develop a flexible random-intercept accelerated failure time model, in which we use Bayesian additive…

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Propensity Score Weighting Analysis of Survival Outcomes Using Pseudo-observations

利用伪观测进行生存结局的倾向得分加权分析

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes constructs pseudo-observations and allows standard methods such as propensity score weighting to proceed as if the outcomes are completely observed. For a general class of model-free causal estimands with survival outcomes on user-specified…

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Discussion on "Bayesian Regression Tree Models for Causal Inference: Regularization, Confounding, and Heterogeneous Effects" by Hahn, Murray and Carvalho

对《用于因果推断的贝叶斯回归树模型》的讨论

Hahn et al. (2020) offers an extensive study to explicate and evaluate the performance of the BCF model in different settings and provides a detailed discussion about its utility in causal inference. It is a welcomed addition to the causal machine learning literature. I will emphasize the contribution of the BCF model to the field of causal inference through discussions on two topics: 1) the difference between the PS in the BCF model and the Bayesian PS in a Bayesian…

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