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时变协变量下交错双重差分处理效应的双重稳健估计AI 解读

With staggered adoption, difference-in-differences (DiD) analyses define the average treatment effect on the treated (ATT) at the group-period level and aggregate it into groupwise, periodwise, dynamic, and overall effects. We study a period-by-period version of conditional parallel trends, with covariates unaffected by treatment while allowing covariates to shift the trends in untreated potential outcomes. We propose an augmented inverse probability weighting (AIPW)…

tteICE：估计两组试验中伴随中间事件的事件时间处理效应的 R 包AI 解读

Evaluating treatment effects in clinical trials with time-to-event outcomes is complicated by intercurrent events (ICEs), e.g., treatment discontinuation or competing events. While the ICH E9 (R1) addendum outlines five strategies to address these challenges, accessible software implementing these methods is limited. This article introduces the \pkg{tteICE} package for \proglang{R}, which implements five strategies to facilitate valid causal inference in the presence of…

存在中间事件时利用多状态数据估计路径处理效应AI 解读

During clinical trials evaluating a drug's effect on a survival endpoint, intermediate events often occur in addition to the primary event. The treatment can exert its effect on the primary endpoint along multiple pathways through intermediate events. Assumptions for identifying mediation effects, such as sequential ignorability in natural effects or the dismissible components condition in separable effects, fail because intermediate events act as treatment-induced…

基于轮廓 M 估计的高维回归去偏推断AI 解读

Debiased inference for high-dimensional regression models has received substantial recent attention to ensure regularized estimators have valid inference. Many existing methods focus on achieving Neyman orthogonality through explicitly constructing projections onto the space of nuisance parameters, which is infeasible when an explicit form of the projection is unavailable. We introduce a general debiasing framework, Debiased Profile $M$-Estimation (DPME), which applies to a…

含中介变量的双重差分AI 解读

Causal mediation analysis is a powerful tool for disentangling the total effect of a treatment into its direct effect on the outcome and its indirect effect mediated through an intermediate variable. However, in observational studies, confounding between treatment and potential outcomes typically renders the total and natural effects non-identifiable. In this work, we advance mediation analysis within the difference-in-differences framework. Under a mediator-adjusted…

带终末事件与不准确诊断的区间删失数据半参数分析AI 解读

Interval-censoring frequently occurs in studies of chronic diseases where disease status is inferred from intermittently collected biomarkers. Although many methods have been developed to analyze such data, they typically assume perfect disease diagnosis, which often does not hold in practice due to the inherent imperfect clinical diagnosis of cognitive functions or measurement errors of biomarkers such as cerebrospinal fluid. In this work, we introduce a semiparametric…

使用估计倾向得分进行逆处理概率加权的调整 Nelson–Aalen 估计AI 解读

Inverse probability of treatment weighting (IPW) has been well applied in causal inference to estimate population-level estimands from observational studies. For time-to-event outcomes, the failure time distribution can be estimated by estimating the cumulative hazard in the presence of random right censoring. IPW can be performed by weighting the event counting process and at-risk process by the inverse treatment probability, resulting in an adjusted Nelson--Aalen estimator…

利用主层分层分析受损随机实验AI 解读

Although randomized controlled trials have long been regarded as the ``gold standard'' for evaluating treatment effects, there is no natural prevention from post-treatment events. For example, non-compliance makes the actual treatment different from the assigned treatment, truncation-by-death renders the outcome undefined or ill-defined, and missingness prevents the outcomes from being measured. In this paper, we develop a statistical analysis framework using principal…

ICH E9（R1）框架下随机试验累积发生率与处理效应的推断AI 解读

In randomized controlled trials (RCTs) that focus on time-to-event outcomes, intercurrent events can arise in two ways: as semi-competing events, which modify the hazard of the primary outcome events, or as competing events, which make the definition of the primary outcome events unclear. Although five strategies have been proposed in the ICH E9 (R1) addendum to address intercurrent events in RCTs, these strategies are not easily applicable to time-to-event outcomes when…

利用多状态模型分解半竞争风险中的可分离路径效应AI 解读

Semi-competing risks refer to the phenomenon where a primary event (such as mortality) can ``censor'' an intermediate event (such as relapse of a disease), but not vice versa. Under the multi-state model, the primary event consists of two specific types: the direct outcome event and an indirect outcome event developed from intermediate events. Within this framework, we show that the total treatment effect on the cumulative incidence of the primary event can be decomposed…

半竞争风险下的直接与间接处理效应AI 解读

Semi-competing risks refer to the phenomenon that the terminal event (such as death) can censor the non-terminal event (such as disease progression) but not vice versa. The treatment effect on the terminal event can be delivered either directly following the treatment or indirectly through the non-terminal event. We consider two strategies to decompose the total effect into a direct effect and an indirect effect under the framework of mediation analysis in completely…

死亡截断与未测混杂下的因果推断AI 解读

Clinical studies sometimes encounter truncation by death, rendering outcomes undefined. Statistical analysis based solely on observed survivors may give biased results because the characteristics of survivors differ between treatment groups. By principal stratification, the survivor average causal effect was proposed as a causal estimand defined in always-survivors. However, this estimand is not identifiable when there is unmeasured confounding between the treatment…