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边际结构分位数模型的双重稳健估计与敏感性分析AI 解读

The marginal structure quantile model (MSQM) provides a unique lens to understand the causal effect of a time-varying treatment on the full distribution of potential outcomes. Under the semiparametric framework, we derive the efficiency influence function for the MSQM, from which a new doubly robust estimator is proposed for point estimation and inference. We show that the doubly robust estimator is consistent if either of the models associated with treatment assignment or…

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…

多种间歇性治疗的因果效应估计及其新冠应用AI 解读

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying confounding and censoring. Our methods formulate complex longitudinal treatments with multiple start/stop switches as the recurrent events with discontinuous intervals of treatment eligibility. We derive the weights in continuous time to handle…

整群随机实验中模型稳健且高效的协变量调整AI 解读

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of model-based covariate adjustment is unclear when the working models are misspecified, leading to ambiguity of estimands and risk of bias. In this article, we first adapt two conventional model-based methods, generalized estimating equations…

评估处理效应异质性的最大最小最优整群随机设计AI 解读

Cluster randomized trials (CRTs) are studies where treatment is randomized at the cluster level but outcomes are typically collected at the individual level. When CRTs are employed in pragmatic settings, baseline population characteristics may moderate treatment effects, leading to what is known as heterogeneous treatment effects (HTEs). Pre-specified, hypothesis-driven HTE analyses in CRTs can enable an understanding of how interventions may impact subpopulation outcomes.…

协变量和结果缺失时随机临床试验的协变量调整AI 解读

When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate adjustment is the presence of missing data. In this paper, in the light of recent theoretical advancement, we first review several covariate adjustment methods with incomplete covariate data. We investigate the implications of the missing data…

PStrata：用于主分层分析的 R 软件包AI 解读

Post-treatment confounding is a common problem in causal inference, including special cases of noncompliance, truncation by death, surrogate endpoint, etc. Principal stratification (Frangakis and Rubin 2002) is a general framework for defining and estimating causal effects in the presence of post-treatment confounding. A prominent special case is the instrumental variable approach to noncompliance in randomized experiments (Angrist, Imbens, and Rubin 1996). Despite its…

事件时间结局的主分层分析AI 解读

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence of intercurrent events. Despite the extensive existing literature, there lacks generally applicable and accessible methods for principal stratification analysis with time-to-event outcomes. In this paper, we specify two causal estimands…

多元连续结局阶梯楔形设计的功效分析AI 解读

Multivariate outcomes are common in pragmatic cluster randomized trials. While sample size calculation procedures for multivariate outcomes exist under parallel assignment, none have been developed for a stepped wedge design. In this article, we present computationally efficient power and sample size procedures for stepped wedge cluster randomized trials (SW-CRTs) with multivariate outcomes that differentiate the within-period and between-period intracluster correlation…

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…

贝叶斯因果推断：批判性综述AI 解读

This paper provides a critical review of the Bayesian perspective of causal inference based on the potential outcomes framework. We review the causal estimands, identification assumptions, the general structure of Bayesian inference of causal effects, and sensitivity analysis. We highlight issues that are unique to Bayesian causal inference, including the role of the propensity score, definition of identifiability, the choice of priors in both low and high dimensional…

利用重叠权重处理反事实生存函数估计中的极端倾向得分AI 解读

The inverse probability weighting approach is popular for evaluating treatment effects in observational studies, but extreme propensity scores could bias the estimator and induce excessive variance. Recently, the overlap weighting approach has been proposed to alleviate this problem, which smoothly down-weighs the subjects with extreme propensity scores. Although advantages of overlap weighting have been extensively demonstrated in literature with continuous and binary…

具有连续共同主要终点的集群随机试验功效分析AI 解读

Pragmatic trials evaluating health care interventions often adopt cluster randomization due to scientific or logistical considerations. Previous reviews have shown that co-primary endpoints are common in pragmatic trials but infrequently recognized in sample size or power calculations. While methods for power analysis based on $K$ ($K\geq 2$) binary co-primary endpoints are available for CRTs, to our knowledge, methods for continuous co-primary endpoints are not yet…

非嵌套设计中将试验证据推广至目标人群：艾滋病临床试验应用AI 解读

Comparative effectiveness evidence from randomized trials may not be directly generalizable to a target population of substantive interest when, as in most cases, trial participants are not randomly sampled from the target population. Motivated by the need to generalize evidence from two trials conducted in the AIDS Clinical Trials Group (ACTG), we consider weighting, regression and doubly robust estimators to estimate the causal effects of HIV interventions in a specified…

利用伪观测进行生存结局的倾向得分加权分析AI 解读

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes constructs pseudo-observations and allows standard methods such as propensity score weighting to proceed as if the outcomes are completely observed. For a general class of model-free causal estimands with survival outcomes on user-specified…

厘清整群随机试验的选择偏差：目标参数与估计AI 解读

In cluster randomized trials, patients are typically recruited after clusters are randomized, and the recruiters and patients may not be blinded to the assignment. This often leads to differential recruitment and consequently systematic differences in baseline characteristics of the recruited patients between intervention and control arms, inducing post-randomization selection bias. We rigorously define causal estimands in the presence of selection bias. We elucidate the…

通过矩阵补全进行回溯因果推断：欧洲一体化与跨境就业AI 解读

We propose a method of retrospective counterfactual imputation in panel data settings with later-treated and always-treated units, but no never-treated units. We use the observed outcomes to impute the counterfactual outcomes of the later-treated using a matrix completion estimator. We propose a novel propensity-score and elapsed-time weighting of the estimator's objective function to correct for differences in the observed covariate and unobserved fixed effects…

随机临床试验亚组分析中的协变量调整：倾向得分方法AI 解读

Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in expectation, chance imbalance may be amplified in small subgroups and harm the precision. Covariate adjustment in overall analysis of RCT is often conducted, via either ANCOVA or propensity score weighting, but for subgroup analysis has been…