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基于中介相关引导贝叶斯变量选择的高维广义线性模型中介分析AI 解读

High-dimensional mediation analysis aims to identify mediating pathways and to estimate indirect effects linking an exposure to an outcome. In this paper, we propose a Bayesian framework to address key challenges in these analyses, including high dimensionality, complex dependence among omics mediators, and non-continuous outcomes. Furthermore, commonly used approaches assume independent mediators or ignore correlations in the selection stage, which can reduce power when…

外部对照单臂试验与非锚定间接治疗比较的双重稳健增广加权估计AI 解读

Externally controlled single-arm trials are critical to assess treatment efficacy across therapeutic indications for which randomized controlled trials are not feasible. A closely-related research design, the unanchored indirect treatment comparison, is often required for disconnected treatment networks in health technology assessment. We present a unified causal inference framework for both research designs. We develop a novel estimator that augments a popular weighting…

利用现代因果推断提高随机对照试验亚组分析的检验力AI 解读

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power to detect heterogeneous effects. Power can be improved by leveraging predictors of the outcome -- i.e., through covariate adjustment -- as well as by borrowing external data from similar RCTs or observational studies. The benefits of…

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…

分解函数型结局处理效应异质性的双重稳健机器学习方法AI 解读

Causal inference is paramount for understanding the effects of interventions, yet extracting personalized insights from increasingly complex data remains a significant challenge for modern machine learning. This is the case, in particular, when considering functional outcomes observed over a continuous domain (e.g., time, or space). Estimation of heterogeneous treatment effects, known as CATE, has emerged as a crucial tool for personalized decision-making, but existing…

异质处理效应下的工具变量双重差分AI 解读

Many studies exploit variation in the timing of policy adoption across units as an instrument for treatment. This paper formalizes the underlying identification strategy as an instrumented difference-in-differences (DID-IV). In this design, a Wald-DID estimand, which scales the DID estimand of the outcome by the DID estimand of the treatment, captures the local average treatment effect on the treated (LATET). We extend the canonical DID-IV design to multiple period settings…

联立图式动态建模AI 解读

We review theory and methodology of the class of simultaneous graphical dynamic linear models (SGDLMs) that provide flexibility, parsimony and scalability of multivariate time series analysis. Discussion includes core theoretical aspects and summaries of existing Bayesian methodology for forward filtering and forecasting with SGDLMs. The review is complemented by new theory linking dynamic graphical and factor models, and extensions of the Bayesian methodology. This…

分布型工具变量：识别与分位数最小二乘估计AI 解读

We study instrumental-variable designs where policy reforms strongly shift the distribution of an endogenous variable but only weakly move its mean. We formalize this by introducing distributional relevance: instruments may be purely distributional. Within a triangular model, distributional relevance suffices for nonparametric identification of average structural effects via a control function. We then propose Quantile Least Squares (Q-LS), which aggregates conditional…

因果推断：三种框架的比较AI 解读

Causal inference is a central goal across many scientific disciplines. Over the past several decades, three major frameworks have emerged to formalize causal questions and guide their analysis: the potential outcomes framework, structural equation models, and directed acyclic graphs. Although these frameworks differ in language, assumptions, and philosophical orientation, they often lead to compatible or complementary insights. This paper provides a comparative introduction…

通过机器学习与二次推断函数优化阶梯楔形设计的估计精度AI 解读

Stepped-wedge designs are increasingly used in randomized experiments to accommodate logistical and ethical constraints by staggering treatment roll-out over time. Despite their popularity, existing analytical methods largely rely on parametric models with linear covariate adjustment and prespecified correlation structures, which may limit achievable precision in practice. We propose a new class of estimators for the causal average treatment effect in stepped-wedge designs…

因果推断中系统识别与联合分析多重偏差的路线图AI 解读

Observational studies examining causal effects rely on unverifiable assumptions, the violation of which can induce multiple biases. Quantitative bias analysis (QBA) methods examine the sensitivity of findings to such violations, generally, by producing estimates under alternative assumptions, incorporating external information. Although substantial guidance exists for implementing QBA, there is limited guidance on how to systematically determine the assumptions underlying a…

结局优劣排序概率的双重稳健估计及其耐多药菌研究应用AI 解读

In observational studies, adjusting for confounders is required if a treatment comparison is planned. A crude comparison of the primary endpoint without covariate adjustment will suffer from biases, and the addition of regression models could improve precision by incorporating imbalanced covariates and thus help make correct inference. Desirability of outcome ranking (DOOR) is a patient-centric benefit-risk evaluation methodology designed for randomized clinical trials.…

通过元学习估计因果图不确定时的干预分布AI 解读

In scientific domains -- from biology to the social sciences -- many questions boil down to \textit{What effect will we observe if we intervene on a particular variable?} If the causal relationships (e.g.~a causal graph) are known, it is possible to estimate the intervention distributions. In the absence of this domain knowledge, the causal structure must be discovered from the available observational data. However, observational data are often compatible with multiple…

死亡截断下生活质量与生存的联合框架及去偏估计AI 解读

Evaluating quality-of-life (QoL) outcomes in populations with high mortality risk is complicated by truncation by death, since QoL is undefined for individuals who do not survive to the planned measurement time. We propose a framework that jointly models the distribution of QoL and survival without extrapolating QoL beyond death. Inspired by multistate formulations, we extend the joint characterization of binary health states and mortality to continuous QoL outcomes. Because…

三重稳健面板估计量AI 解读

This paper studies estimation of causal effects in a panel data setting. We introduce a new estimator, the Triply RObust Panel (TROP) estimator, that combines (i) a flexible model for the potential outcomes based on a low-rank factor structure on top of a two-way-fixed effect specification, with (ii) unit weights intended to upweight units similar to the treated units and (iii) time weights intended to upweight time periods close to the treated time periods. We study the…

通过修正函数型处理策略估计函数型处理的因果效应AI 解读

Functional data are increasingly prevalent in biomedical research. While functional data analysis has been established for decades, causal inference with functional treatments remains largely unexplored. Existing methods typically focus on estimating the causal average dose response functional (ADRF), which requires strong positivity assumptions and offers limited interpretability. In this work, we target a new causal estimand, the modified functional treatment policy…

面向稳定因果中介分析的偏差针对性非参数平衡AI 解读

Influence function (IF)-based estimators are widely used in mediation analysis due to their modeling flexibility, but standard implementations require direct estimation of the distribution functions of the mediator and treatment variables. Since these functions appear in the denominator of IF-based estimators, they can induce significant instability, particularly with continuous mediators. In this work, we propose an alternative implementation of IF-based estimators for both…

评估协变量无法解释的因果效应异质性AI 解读

There has been considerable interest in estimating heterogeneous causal effects across individuals or subpopulations. Researchers often assess causal effect heterogeneity based on the subjects' covariates using the conditional average causal effect (CACE). However, substantial heterogeneity may persist even after accounting for the covariates. Existing work on causal effect heterogeneity unexplained by covariates mainly focused on binary treatment and outcome. In this paper,…

临床试验治疗个性化的因果机器学习框架：溃疡性结肠炎应用AI 解读

Randomized controlled trials estimate average treatment effects, but treatment response heterogeneity motivates personalized approaches. A critical question is whether statistically detectable heterogeneity translates into improved treatment decisions -- these are distinct questions that can yield contradictory answers. We present a modular causal machine learning framework that evaluates each question separately: permutation importance identifies which features predict…

多元因果效应：贝叶斯因果回归因子模型AI 解读

The impact of wildfire smoke on air quality is a growing concern, contributing to air pollution through a complex mixture of chemical species with important implications for public health. While previous studies have primarily focused on its association with total particulate matter (PM2.5), the causal relationship between wildfire smoke and the chemical composition of PM2.5 remains largely unexplored. Exposure to these chemical mixtures plays a critical role in shaping…

圆形散光生物标志物治疗效应的双曲几何统计推断AI 解读

Circular biomarkers arise naturally in many biomedical applications, particularly in ophthalmology, where angular measurements such as astigmatism are routinely recorded. Similar directional variables also occur in the study of human body rotations, including movements of the hand, waist, neck, and lower limbs. Motivated by a clinical dataset comprising angular measurements of astigmatism induced by two cataract surgery procedures, we propose a novel two-sample testing…

通过考虑推断的政策优化缓解赢家诅咒AI 解读

There has been a surge of recent interest in automatically learning policies to target treatment decisions based on rich individual covariates. In addition, practitioners want confidence that the learned policy has better performance than the incumbent policy according to downstream policy evaluation. However, due to the winner's curse -- an issue where the policy optimization procedure exploits prediction errors rather than finding actual improvements -- predicted…

一般工具变量下因果效应的识别与去偏学习AI 解读

Instrumental variable methods are fundamental to causal inference when treatment assignment is confounded by unobserved variables. In this article, we develop a general nonparametric causal framework for identification and learning with multi-categorical or continuous instrumental variables. Specifically, the mean potential outcomes and the average treatment effect can be identified via a regular weighting function derived from the proposed framework. Leveraging…

复发事件临床试验的事件驱动型设计AI 解读

It is a common practice in randomized clinical trials with the standard survival outcome to follow patients until a prespecified number of events have been observed, a type of trial known as the event-driven trial. The event-driven design ensures that the target power for a specified type 1 error rate is achieved to detect the target hazard ratio, regardless of the specification of other quantities. To understand the treatment effect for chronic conditions, the analysis of…