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Federated Adaptive Causal Estimation (FACE) of Target Treatment Effects

目标处理效应的联邦自适应因果估计

Federated learning of causal estimands may greatly improve estimation efficiency by leveraging data from multiple study sites, but robustness to heterogeneity and model misspecifications is vital for ensuring validity. We develop a Federated Adaptive Causal Estimation (FACE) framework to incorporate heterogeneous data from multiple sites to provide treatment effect estimation and inference for a flexibly specified target population of interest. FACE accounts for site-level…

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Robust Inference for Federated Meta-Learning

联邦元学习的稳健推断

Synthesizing information from multiple data sources is critical to ensure knowledge generalizability. Integrative analysis of multi-source data is challenging due to the heterogeneity across sources and data-sharing constraints due to privacy concerns. In this paper, we consider a general robust inference framework for federated meta-learning of data from multiple sites, enabling statistical inference for the prevailing model, defined as the one matching the majority of the…

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Finding the optimal dynamic treatment regimes using smooth Fisher consistent surrogate loss

利用光滑 Fisher 一致替代损失寻找最优动态治疗方案

Large health care data repositories such as electronic health records (EHR) open new opportunities to derive individualized treatment strategies for complicated diseases such as sepsis. In this paper, we consider the problem of estimating sequential treatment rules tailored to a patient’s individual characteristics, often referred to as dynamic treatment regimes (DTRs). Our main objective is to find the optimal DTR that maximizes a discontinuous value function through direct…

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Assessing the Most Vulnerable Subgroup to Type II Diabetes Associated with Statin Usage: Evidence from Electronic Health Record Data

识别他汀使用相关二型糖尿病风险最高的亚组:电子健康记录证据

There have been increased concerns that the use of statins, one of the most commonly prescribed drugs for treating coronary artery disease, is potentially associated with the increased risk of new-onset Type II diabetes (T2D). Nevertheless, to date, there is no robust evidence supporting as to whether and what kind of populations are indeed vulnerable for developing T2D after taking statins. In this case study, leveraging the biobank and electronic health record data in the…

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Optimal Statistical Inference for Individualized Treatment Effects in High-Dimensional Models

高维模型中个体化处理效应的最优统计推断

Abstract The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given individual in the framework of high-dimensional linear models. The methodological novelty lies in the construction of a debiased estimator of the ITE and establishment of its asymptotic normality uniformly for an arbitrary future…

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Model-free approach to quantifying the proportion of treatment effect explained by a surrogate marker

无模型方法量化替代标志物解释的治疗效应比例

Summary In randomized clinical trials, the primary outcome, $Y$, often requires long-term follow-up and/or is costly to measure. For such settings, it is desirable to use a surrogate marker, $S$, to infer the treatment effect on $Y$, $\Delta$. Identifying such an $S$ and quantifying the proportion of treatment effect on $Y$ explained by the effect on $S$ are thus of great importance. Most existing methods for quantifying the proportion of treatment effect are model based and…

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Testing differential networks with applications to the detection of gene-gene interactions

差异网络检验及其在基因交互识别中的应用

Model organisms and human studies have yielded increasing empirical evidence that interactions among genes contribute broadly to genetic variation of complex traits. In the presence of gene-gene interactions, the dimensionality of the feature space becomes extremely high relative to the sample size. This poses a significant methodological challenge in the identification of gene-gene interactions. In this paper, by using a Gaussian graphical model framework, we translate the…

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Landmark Estimation of Survival and Treatment Effect in a Randomized Clinical Trial

随机临床试验中生存率与治疗效应的界标估计

In many studies with a survival outcome, it is often not feasible to fully observe the primary event of interest. This often leads to heavy censoring and thus, difficulty in efficiently estimating survival or comparing survival rates between two groups. In certain diseases, baseline covariates and the event time of nonfatal intermediate events may be associated with overall survival. In these settings, incorporating such additional information may lead to gains in efficiency…

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Effectively Selecting a Target Population for a Future Comparative Study

为未来比较研究有效选择目标人群

When comparing a new treatment with a control in a randomized clinical study, the treatment effect is generally assessed by evaluating a summary measure over a specific study population. The success of the trial heavily depends on the choice of such a population. In this paper, we show a systematic, effective way to identify a promising population, for which the new treatment is expected to have a desired benefit, utilizing the data from a current study involving similar…

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