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个体与整群随机试验中存活期间目标量的双重稳健估计AI 解读

Randomized trials in chronic disease settings often measure treatment benefit through recurrent non-fatal events that are truncated by death, where conventional summaries either discard recurrences, conflate the treatment effect with survival, or treat death as censoring and forfeit a causal interpretation. While-alive estimands measure event burden per unit time alive, but doubly robust estimation for the exposure-weighted while-alive rate remains undeveloped, particularly…

整群随机试验中胜负统计量的样本量设计AI 解读

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and win difference (net benefit) have gained traction in individually randomized trials, their methodological development for cluster-randomized trials remains limited. In particular, there is no formal framework for power and sample size…

阶梯楔形集群随机试验中的模型稳健标准化AI 解读

Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, enabling all clusters to receive the intervention through a staggered rollout. Traditional model-based methods, including generalized estimating equations and mixed models, yield estimates that depend on implicit weighting schemes and parametric assumptions, and therefore may target ambiguous estimands under model misspecification. In this article, we propose a…

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…

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…

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…

生存结局聚类随机试验的目标参数与双重稳健估计AI 解读

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…

整群随机试验中的模型稳健标准化AI 解读

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent studies have demonstrated that their treatment effect coefficient estimators may correspond to ambiguous estimands when the models are misspecified or when there exists informative cluster sizes. In this article, we present a unified approach…