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生存分析中再随机化的渐近理论AI 解读

Rerandomization systematically reduces chance imbalance and can improve the efficiency of the average treatment effect estimator in randomized experiments. While the asymptotic properties of finite-dimensional M-estimators under rerandomization have been established, existing theory does not directly address survival outcomes under censoring, where the target estimand involves infinite-dimensional functional parameters. This article establishes the uniform weak convergence…

时变处理效应阶梯楔形设计应采用哪种小样本修正？AI 解读

Stepped-wedge cluster randomized trials (SW-CRTs) evaluate interventions rolled out across clusters over time. Standard analyses typically use immediate-treatment (IT) models, which assume effects begin at crossover and remain constant thereafter. When effects vary with exposure duration, IT models may misrepresent target effects. Exposure-time indicator (ETI) models address this by allowing treatment effects to differ by time since exposure and by targeting the…

利用机器学习估计整群随机试验中的个体化治疗效应AI 解读

Cluster-randomized trials (CRTs) are widely used to evaluate interventions delivered at the clinic, practice, or community level. Although standard analyses typically target average treatment effects, such summaries mask potentially meaningful variation in treatment response across individuals and clusters. This work addresses the estimation of conditional average treatment effects (CATEs) for continuous outcomes in two-arm parallel CRTs by defining causal estimands that…

部分干扰下网络分位数因果效应的非参数有效推断AI 解读

Interference arises when the treatment assigned to one individual affects the outcomes of other individuals. Commonly, individuals are naturally grouped into clusters, and interference occurs only among individuals within the same cluster, a setting referred to as partial interference. We study network causal effects on outcome quantiles in the presence of partial interference. We develop a general nonparametric efficiency theory for estimating these network quantile causal…

多中介整群随机试验的因果中介分析：溢出分解、识别与有效推断AI 解读

Causal mediation analysis in cluster-randomized trials (CRTs) is complicated by the presence of multiple mediators, intracluster correlation, and within-cluster interference. Existing mediation methods often fall short in accommodating these features simultaneously, and semiparametric efficient estimators that fully address them remain unavailable. We develop a unified framework that defines a class of mediation effect estimands, including exit indirect effects, exit…

纵向整群随机与准实验试验中用于因果推断的固定效应模型AI 解读

This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs) and quasi-experimental (CQTs) designs. We clarify a longstanding misconception in biostatistics, demonstrating that fixed-effects models, traditionally perceived as targeting only finite-sample conditional estimands, can effectively…

局部平均处理效应框架下聚类数据的两阶段最小二乘AI 解读

To estimate the causal effect of an endogenous treatment using clustered data, the canonical two-stage least squares (2sls) estimates a linear regression of the outcome on treatment status using an instrumental variable (IV) and conducts inference with cluster-robust standard errors. When both the treatment and the IV vary within clusters, an alternative two-stage least squares with fixed effects (2sfe) additionally includes cluster indicators in the regression, thereby…

处理后混杂下集群随机试验因果机制的贝叶斯非参数识别与估计AI 解读

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical covariate adjustment. We develop a Bayesian nonparametric framework that simultaneously accommodates interference and a post-treatment confounder that precedes the mediator. Identification is achieved through a multivariate Gaussian copula that…

终末事件截断复发事件的主层分析：嵌套贝叶斯非参数方法AI 解读

Recurrent events often serve as key endpoints in clinical studies but may be prematurely truncated by terminal events such as death, creating selection bias and complicating causal inference. To address this challenge, we develop a Bayesian nonparametric framework to address potential selection bias due to truncation by death within the continuous-time principal stratification framework. We introduce causal estimands for recurrent events in the presence of a terminal event…

未来干预因果效应的预测：混杂与可迁移性问题AI 解读

Recent developments in causal inference allow us to transport a causal effect of a time-fixed treatment from a randomized trial to a target population across space but within the same time frame. In contrast to transportability across space, transporting causal effects across time or forecasting causal effects of future interventions is more challenging due to time-varying confounders and time-varying effect modifiers. In this article, we seek to formally clarify the causal…

含处理与协变量交互项的两阶段最小二乘及处理效应异质性AI 解读

Treatment effect heterogeneity with respect to covariates is common in instrumental variable (IV) analyses. An intuitive approach, which we call the interacted two-stage least squares (2sls), is to postulate a working linear model of the outcome on the treatment, covariates, and treatment-covariate interactions, and instrument it using the IV, covariates, and IV-covariate interactions. We clarify the causal interpretation of the interacted 2sls under the local average…

随机实验中广义因果效应的设计本位推断AI 解读

Generalized causal effect estimands, including the Mann-Whitney parameter and causal net benefit, provide flexible summaries of treatment effects in randomized experiments with non-Gaussian or multivariate outcomes. We develop a unified design-based inference framework for regression adjustment and variance estimation of a broad class of generalized causal effect estimands defined through pairwise contrast functions. Leveraging the theory of U-statistics and…

连续纵向中间变量下主层分层的敏感性分析：队列阶梯楔形试验应用AI 解读

Causal inference in the presence of intermediate variables is a challenging problem in many applications. Principal stratification (PS) provides a framework to estimate principal causal effects (PCE) in such settings. However, existing PS methods primarily focus on settings with binary intermediate variables. We propose a novel approach to estimate PCE with continuous intermediate variables in the context of stepped wedge cluster randomized trials (SW-CRTs). Our method…

谁占优势？澄清整群随机试验中基于胜出统计量的估计目标AI 解读

Treatment effect estimands based on win statistics, including the win ratio, win odds, and win difference are increasingly popular targets for summarizing endpoints in clinical trials. Such win estimands offer an intuitive approach for prioritizing outcomes by clinical importance. The implementation and interpretation of win estimands is complicated in cluster randomized trials (CRTs), where researchers can target fundamentally different estimands on the individual-level or…

调查观察研究中倾向得分加权的规范实践AI 解读

Propensity score weighting is a common method for estimating treatment effects with survey data. The method is applied to minimize confounding using measured covariates that are often different between individuals in treatment and control. However, existing literature does not reach a consensus on the optimal use of survey weights for population-level inference in the propensity score weighting analysis. Under the balancing weights framework, we provided a unified solution…

理解双重稳健估计：协变量重叠的作用AI 解读

The doubly-robust (DR) estimator is popular for evaluating causal effects in observational studies and is often perceived as more desirable than inverse probability weighting (IPW) or outcome modeling alone because it provides extra protection against model misspecification. However, double robustness is an asymptotic property that may not hold in finite samples. We investigate how the finite sample performance of the DR estimator depends on the degree of covariate overlap…

多处理或析因处理观察研究中生存结果的倾向得分加权Cox回归AI 解读

In observational studies with survival or time-to-event outcomes, a propensity score weighted marginal Cox proportional hazard model with the treatment variable as the only predictor is commonly used to estimate the causal marginal hazard ratio between two treatments. Observational studies often have more than two treatments, but corresponding analysis methods are limited. In this paper, we combine the propensity score weighting method for multiple treatments and a marginal…

分期推广集群随机实验中动态因果效应的模型辅助推断AI 解读

Staggered rollout cluster randomized experiments (SR-CREs) involve sequential treatment adoption across clusters, requiring analysis methods that address a general class of dynamic causal effects, anticipation, and non-ignorable cluster-period sizes. Without imposing any outcome modeling assumptions, we study regression estimators using individual data, cluster-period averages, and scaled cluster-period totals, with and without covariate adjustment from a design-based…

集群随机交叉试验中存活者平均因果效应的贝叶斯方法AI 解读

In cluster-randomized crossover (CRXO) trials, groups of individuals are randomly assigned to two or more sequences of alternating treatments. Since clusters serve as their own control, the CRXO design is typically more statistically efficient than the usual parallel-arm design. CRXO trials are increasingly popular in many areas of health research where the number of available clusters is limited. Further, in trials among severely ill patients, researchers often want to…

多结局整群随机试验中广义因果效应的最优估计AI 解读

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of treatment effect, yet existing methods largely focus on single-outcome estimands or rely on model-based procedures with unclear causal interpretation or limited robustness. We develop a unified potential outcomes framework for generalized…

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AI 解读

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…

重叠加权是什么、如何发展及何时用于因果推断AI 解读

The growing availability of large health databases has expanded the use of observational studies for comparative effectiveness research. Unlike randomized trials, observational studies must adjust for systematic differences in patient characteristics between treatment groups. Propensity score methods, including matching, weighting, stratification, and regression adjustment, address this issue by creating groups that are comparable with respect to measured covariates. Among…

集群随机试验中间接效应与溢出效应的半参数因果中介分析AI 解读

In cluster-randomized trials (CRTs), there is emerging interest in exploring the causal mechanism in which a cluster-level treatment affects the outcome through an intermediate outcome. The majority of existing causal mediation methods are applicable to independent data and only a few exceptions have considered assessing causal mediation in CRTs, all of which heavily depend on parametric assumptions. In this article, we develop a formal semiparametric efficiency theory to…

揭示实用性老年医学试验中的处理效应异质性AI 解读

Detecting heterogeneity in treatment response enriches the interpretation of gerontologic trials. In aging research, estimating the effect of the intervention on clinically meaningful outcomes faces analytical challenges when it is truncated by death. For example, in the Whole Systems Demonstrator trial, a large cluster-randomized study evaluating telecare among older adults, the overall effect of the intervention on quality of life was found to be null. However, this…