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超越单调性的异质幸存者平均因果效应：机械通气临床试验应用AI 解读

Clinical trials in critical care often evaluate outcomes that are truncated by death, such as time to discharge alive, for which treatment effects are not well defined among patients who would not survive under one or both treatment strategies. Principal stratification provides a natural framework for defining survivor causal effects, but existing approaches often rely on monotonicity assumptions that may be implausible in settings where treatment can affect survival in…

簇规模饱和与 g 计算：信息性簇规模下的稳健因果推断AI 解读

Cluster randomized trials (CRTs) can exhibit informative cluster sizes (ICS) where cluster size is associated with outcomes and/or treatment effects. Under ICS, the individual and cluster-average treatment effects (iATE, cATE) can diverge, and the conventional linear mixed-effects model (LMM) and generalized estimating equation (GEE) with an exchangeable working correlation can produce data-dependent weighted contrasts that are not consistent for either estimand. In these…

存在不依从时阶梯楔形设计的随机化推断：姑息治疗试验应用AI 解读

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a stepped-wedge cluster-randomized design to assess the effect of palliative care on a patient-centered outcome. The randomized intervention was a nudge to administer palliative care but did not guarantee receipt of palliative care, resulting in noncompliance. A subsequent analysis using…

超越主层可忽略性：主层分层的非参数敏感性界AI 解读

Principal stratification is an effective framework addressing intermediate variables in causal inference. However, point identification of the principal causal effects (PCEs) often requires the untestable principal ignorability (PI) assumption. This article develops a nonparametric sensitivity analysis framework for evaluating PI violations. We introduce a margin-free bounding factor parameterized by the selection and outcome relative risks of an unmeasured confounder. Using…

用线性混合模型检测整群随机试验处理效应异质性：样本量与功效教程AI 解读

Cluster-randomized trials (CRTs) are a well-established class of designs for evaluating community-based interventions. An essential task in planning these trials is determining the number of clusters and cluster sizes needed to achieve sufficient statistical power for detecting a clinically relevant effect size. While methods for evaluating the average treatment effect (ATE) for the entire study population are well-established, sample size methods for testing heterogeneity…

基于设计的嵌套工具变量分析AI 解读

Two binary instrumental variables (IVs) are nested if individuals who comply under one binary IV also comply under the other. This situation often arises when the two IVs represent different intensities of encouragement or discouragement to take the treatment, with one stronger than the other. In a nested IV structure, treatment effects can be identified for two latent subgroups: always-compliers and switchers. Always-compliers are individuals who comply even under 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…

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…

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…

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…

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…

阶梯楔形整群随机试验中的预期效应AI 解读

In stepped wedge cluster randomized trials (SW-CRTs), the intervention is rolled out to clusters over multiple periods. A standard approach for analyzing SW-CRTs utilizes the linear mixed model, where the treatment effect is only present after the treatment adoption, under the assumption of no anticipation. This assumption, however, may not always hold in practice because stakeholders, providers, or individuals who are aware of the treatment adoption timing (especially when…

超越单调性假设的半参数主分层分析AI 解读

Intercurrent events, common in clinical trials and observational studies, affect the existence or interpretation of final outcomes. Principal stratification addresses this challenge by defining local average treatment effect estimands within subpopulations, but often relies on restrictive assumptions such as monotonicity and counterfactual intermediate independence. To overcome these limitations, we propose a semiparametric framework for principal stratification analysis…

多组临床试验中死亡截断结局的双重稳健估计与敏感性分析AI 解读

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable tools for addressing the average causal effect among always-survivors, i.e., the average treatment effect among a subpopulation defined as those who would survive regardless of treatment assignment. Although robust methods for the…

同时存在死亡截断与缺失的多元结局整群随机试验贝叶斯推断AI 解读

Cluster-randomized trials (CRTs) on fragile populations frequently encounter complex attrition problems where the reasons for missing outcomes can be heterogeneous, with participants who are known alive, known to have died, or with unknown survival status, and with complex and distinct missing data mechanisms for each group. Although existing methods have been developed to address death truncation in CRTs, no existing methods can jointly accommodate participants who drop out…

信息性规模下的整群随机交叉试验究竟估计什么：目标量与常用估计量综述AI 解读

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and cluster-level hypotheses. In multi-period CRT designs, such as the cluster randomized crossover (CRXO) trial, additional weighted average treatment effect estimands help fully reflect the longitudinal nature of these trial designs, namely the…

多干预阶梯楔形整群随机试验中的时变处理效应模型AI 解读

The traditional model specification of stepped-wedge cluster-randomized trials assumes a homogeneous treatment effect across time while adjusting for fixed-time effects. However, when treatment effects vary over time, the constant effect estimator may be biased. In the general setting of stepped-wedge cluster-randomized trials with multiple interventions, we derive the expected value of the constant effect estimator when the true treatment effects depend on exposure time…

整群随机试验中效应修饰变量缺失时的治疗效应异质性评估AI 解读

Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect modifiers has become a common goal in modern randomized trials. However, when one or more potential effect modifiers are missing, complete-case analysis may lead to bias and under-coverage. While statistical methods for handling missing…

整群随机试验中的混合模型协方差分析AI 解读

In the analyses of cluster-randomized trials, mixed-model analysis of covariance (ANCOVA) is a standard approach for covariate adjustment and handling within-cluster correlations. However, when the normality, linearity, or the random-intercept assumption is violated, the validity and efficiency of the mixed-model ANCOVA estimators for estimating the average treatment effect remain unclear. Under the potential outcomes framework, we prove that the mixed-model ANCOVA…

利用基线协变量分析罕见二元结局的小型整群随机试验AI 解读

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account for chance imbalances between treatment arms and increase statistical efficiency in individually-randomized trials, analytical methods for individual-level covariate adjustment in small CRTs have received little attention to date. In this…