论文检索

整群随机试验中依从效应与网络因果效应的识别和有效估计AI 解读

Treatment noncompliance is pervasive in infectious disease cluster-randomized trials. Although all individuals within a cluster are assigned the same treatment condition, the treatment uptake status may vary across individuals due to noncompliance. We propose a semiparametric framework to evaluate the individual compliance effect and network assignment effect within principal stratum exhibiting different patterns of noncompliance. The individual compliance effect captures…

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…

有限重叠下平均处理效应的估计：多项式逼近与外推AI 解读

Estimating the average treatment effect (ATE) remains a fundamental challenge in observational studies in the presence of poor or limited covariate overlap. Although the inverse probability weighting (IPW) estimator is a widely used approach for estimating the ATE, its performance can deteriorate substantially when overlap is limited, often resulting in increased finite sample bias and unreliable confidence intervals. One common strategy is to shift attention from the…

基于正交机器学习的非参数异质性因果中介分析AI 解读

Causal mediation analysis decomposes the total effect of an intervention on an outcome into a direct pathway and an indirect pathway transmitted through a mediator, but standard methods typically summarize these pathways using population average effects. In many applications, however, the indirect effect may vary substantially across individual profiles. We propose an orthogonal statistical learning framework for estimating heterogeneous causal mediation effects conditional…

极少集群、多时期的集群随机交叉试验：连续结局应采用哪些分析方法？AI 解读

Cluster randomized crossover (CRXO) trials are often used when individual randomization is impractical and the number of available clusters is limited. However, statistical analysis of CRXO trials is complex because of the need to account for complex correlation structures over time. It becomes especially challenging when very few clusters are used because standard modeling assumptions may lead to unstable variance estimates, poor confidence interval coverage, and inflated…

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…

整群随机试验的校准贝叶斯分析AI 解读

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods have emerged as a strong alternative. Previous work has investigated the use of Bayesian hierarchical models for continuous, binary, and count outcomes in CRTs, but these approaches focus on model-based treatment effect coefficients as the…

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…

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…

贝叶斯变平面回归AI 解读

Change-plane regression represents treatment effect heterogeneity through an interpretable rule that assigns patients to two groups according to whether a linear score of baseline covariates crosses a threshold. Although easy to communicate, likelihood-based inference for the hard threshold is nonregular, invalidating standard large-sample approximations. We develop a Bayesian framework that replaces the sharp indicator with a smooth probit gate at a declared smoothing scale…

因果异质性的分辨率AI 解读

Causal subgroup analyses often report a small number of groups summarizing treatment effect heterogeneity, as if that number were a well-defined estimand. Outside genuinely latent class populations, however, a ``true'' subgroup count is model dependent rather than a population functional. We replace it with a new population estimand, the resolution profile, a functional of the causal feature law giving the fewest groups explaining a prescribed fraction of causal…

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…

主分层下异质性处理效应的双重交叉拟合去偏机器学习AI 解读

Principal stratification provides a foundational framework for causal inference with intermediate outcomes by defining causal effects within subpopulations, yet existing work has largely focused on average effects across strata rather than treatment effect heterogeneity within strata. Such within-stratum heterogeneity informs individualized treatment decisions but the associated methods are sparse. We address this gap by studying the identification and estimation of the…

随机临床试验中有序结果胜出统计量的协变量调整AI 解读

Ordinal outcomes are common in clinical settings where they often represent increasing levels of disease progression or different levels of functional impairment. In this article, we focus on representing the average treatment effect for ordinal outcomes via intrinsic pairwise outcome comparisons captured through win estimands, such as the win ratio and win difference. Recognizing the value of baseline covariate adjustment toward enhanced precision, we first develop…

因果中介敏感性分析：桥接得分、尖锐敏感性界限与校准AI 解读

Causal mediation analysis decomposes the total treatment effect into a portion operating through a hypothesized mediator and a residual direct portion. Identification of natural direct and indirect effects typically rests on the mediator stage of sequential ignorability, which cannot be empirically verified and requires explicit sensitivity analysis. We formulate the \emph{bridge score}, a mediator-stage balancing score, as a low-dimensional vector formed from the two…

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…

观察研究因果推断的样本量与功效计算AI 解读

This paper investigates the theoretical foundation and develops analytical formulas for sample size and power calculations for causal inference with observational data. By analyzing the variance of an inverse probability weighting estimator of the average treatment effect, we decompose the power calculation into three components: propensity score distribution, potential outcome distribution, and their correlation. We show that to determine the minimal sample size of an…

事件时间结局因果推断的样本量与功效计算AI 解读

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as the only predictor. We extend the robust sandwich variance theory and derive the analytical form of the asymptotic variance for the inverse probability weighted partial likelihood estimator. Building on this, we derive a new analytical…

再随机化与分层再随机化下灵活协变量调整的渐近推断AI 解读

Rerandomization is an effective treatment allocation procedure to control for baseline covariate imbalance. For estimating the average treatment effect, rerandomization has been previously shown to improve the precision of the unadjusted and the linearly-adjusted estimators over simple randomization without compromising consistency. However, it remains unclear whether such results apply more generally to the class of M-estimators, including the g-computation formula with…

具有连续效果与实施终点的二型混合整群随机试验设计方法比较AI 解读

Hybrid type 2 studies are gaining popularity for their ability to assess both implementation and health outcomes as co-primary endpoints. Often conducted as cluster-randomized trials (CRTs), five design methods can validly power these studies: p-value adjustment methods, combined outcomes approach, single weighted 1-DF test, disjunctive 2-DF test, and conjunctive test. We compared these methods theoretically and numerically. Theoretical comparisons of power equations allowed…

有序结果的因果推断：基于 Copula 的识别、估计与敏感性分析AI 解读

In causal inference with ordinal outcomes, several interpretable estimands are functions of the probability that the potential outcome under one treatment is larger than that under another treatment for the same unit. This probability depends on the joint distribution of both potential outcomes and is generally not identifiable. Existing work has focused on sharp bounds of this probability based on partial identification, but bounds are often too wide to be informative. We…

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…

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…