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高维聚类数据中的估计目标偏移与合成近似AI 解读

In high-dimensional clustered data, covariates whose distributions differ across clusters can act as proxies for unobserved cluster effects. We show that the marginal-model LASSO implicitly uses sparse combinations of such covariates to shift the estimation target from structural coefficients to a contaminated vector, which inflates false selections. We therefore propose the Synthetic Heterogeneous-Effects LASSO (SHEL), which augments the regression design with separately…

设计信息辅助的回归AI 解读

We consider regression problems in which the marginal distribution of the covariates is informative for estimation and variable selection, rather than merely auxiliary. Motivated by random-design, high-dimensional, and latent-effect settings, we propose a general design-assisted regression framework in which the estimating criterion depends on both the conditional model for $Y \mid \bfX$ and structured features of the covariate distribution. The framework identifies two…

超越可交换性：随机试验中考虑分布偏移的外部对照整合AI 解读

Randomized controlled trials (RCTs) are the gold standard for evaluating causal effects but are often costly and difficult to scale; consequently, they are frequently augmented with auxiliary external controls in many applications. Prior approaches for borrowing such data typically rely on exchangeability, under which the external controls are readily usable for inference in the trial population. In practice, however, differences in eligibility criteria, standard of care,…

通过均值或效应尺度可迁移性构造外部对照组AI 解读

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data from an external comparator, which may facilitate head-to-head comparisons of treatments initially studied in different populations. We delineate external comparator analyses under two distinct, but related, identification strategies. The…

随机试验中外部对照数据的稳健整合AI 解读

One approach for increasing the efficiency of randomized trials is the use of "external controls" -- individuals who received the control treatment studied in the trial during routine practice or in prior experimental studies. Existing external control methods, however, can be biased if the populations underlying the trial and the external control data are not exchangeable. Here, we characterize a randomization-aware class of treatment effect estimators in the population…

纵向因果推断中治疗模型的自适应稀疏化与平滑AI 解读

Causal variable selection in time-varying treatment settings is challenging due to evolving confounding effects. Existing methods mainly focus on time-fixed exposures and are not directly applicable to time-varying scenarios. We propose a novel two-step procedure for variable selection when modeling the treatment probability at each time point. We first introduce a novel approach to longitudinal confounder selection using a Longitudinal Outcome Adaptive LASSO (LOAL) that…

重复结果结构嵌套均值模型中效应修饰变量选择的惩罚 G 估计AI 解读

Effect modification occurs when the impact of the treatment on an outcome varies based on the levels of other covariates known as effect modifiers. Modeling these effect differences is important for etiological goals and for purposes of optimizing treatment. Structural nested mean models (SNMMs) are useful causal models for estimating the potentially heterogeneous effect of a time-varying exposure on the mean of an outcome in the presence of time-varying confounding. A…

条件相对效应可迁移假设下的因果推断AI 解读

When extending inferences from a randomized trial to a new target population, the transportability condition for conditional difference effect measures is invoked to identify the marginal causal mean difference in the target population. However, many clinical investigators believe that conditional relative effect measures are more likely to be "transportable" between populations. Here, we examine the identification and estimation of the marginal counterfactual mean…

CausalMetaR：用于具有因果解释的荟萃分析的 R 包AI 解读

Researchers would often like to leverage data from a collection of sources (e.g., primary studies in a meta-analysis) to estimate causal effects in a target population of interest. However, traditional meta-analytic methods do not produce causally interpretable estimates for a well-defined target population. In this paper, we present the CausalMetaR R package, which implements efficient and robust methods to estimate causal effects in a given internal or external target…

利用多来源数据高效估计亚组处理效应AI 解读

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target population. Such a target population can correspond to one of the data sources of the multi-source data or an external population in which the treatment and outcome information may not be available. We develop and evaluate methods for using…

随机对照试验中风险比的不可折叠性与内生选择偏差AI 解读

Background: The hazard ratio of the Cox proportional hazards model is widely used in randomized controlled trials to assess treatment effects. However, two properties of the hazard ratio including the non-collapsibility and built-in selection bias need to be further investigated. Methods: We conduct simulations to differentiate the non-collapsibility effect and built-in selection bias from the difference between the marginal and the conditional hazard ratio. Meanwhile, we…

早期肿瘤试验混合对照方法评估：基于MORPHEUS-UC的模拟研究AI 解读

Phase Ib/II oncology trials, despite their small sample sizes, aim to provide information for optimal internal company decision-making concerning novel drug development. Hybrid controls (a combination of the current control arm and controls from one or more sources of historical trial data [HTD]) can be used to increase the statistical precision. Here we assess combining two sources of Roche HTD to construct a hybrid control in targeted therapy for decision-making via an…

耐多药结核病个体患者数据元分析中的治疗效应修饰建模AI 解读

Effect modification occurs while the effect of the treatment is not homogeneous across the different strata of patient characteristics. When the effect of treatment may vary from individual to individual, precision medicine can be improved by identifying patient covariates to estimate the size and direction of the effect at the individual level. However, this task is statistically challenging and typically requires large amounts of data. Investigators may be interested in…