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目标函数的目标化高度自适应Lasso最小损失估计AI 解读

We propose a Targeted Highly Adaptive Lasso for estimation of non-pathwise differentiable functional parameters such as the dose-response curve (DRC) for continuous exposure. We assume the target function lies in the $k$-th order smoothness class used to define the $k$-th order Highly Adaptive Lasso (HAL), which can be well approximated by linear spans of $k$-th order spline basis functions. We construct a projection of the true target function onto a large finite…

结局子抽样纵向两阶段设计的目标最大似然估计AI 解读

We consider efficient estimation of causal parameters in longitudinal two-stage designs with outcome subsampling, motivated by resampling designs in HIV-related mortality studies. In these studies, many participants become lost to follow-up; resampling designs address this by tracing a subset of lost individuals to ascertain their outcomes. Analyses often use inverse-probability-weighted Kaplan-Meier (wKM) estimators that discard longitudinal covariate information and suffer…

高阶有效估计量：综述与模拟基准研究AI 解读

Higher-order efficient estimators extend standard first-order semiparametric estimators by replacing second-order residuals with third- or higher-order terms, potentially enabling asymptotic efficiency under slower nuisance function convergence rates and improving finite-sample performance. Existing methods achieve higher-order expansions through structurally different approximation strategies, including basis truncation, kernel smoothing, and highly adaptive lasso (HAL)…

通过隐含干预进行因果推断AI 解读

In the context of having an instrumental variable, the standard practice in causal inference begins by targeting an effect of interest and proceeds by formulating assumptions enabling its identification. We turn this around by adhering to the interventions the observational distribution allows to identify, rather than starting with a desired causal estimand and imposing untestable conditions. The randomization of an instrument and its exclusion restriction define a class of…

随机化后伴随用药差异下的生存结局处理效应估计AI 解读

In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the intention-to-treat analysis. We focus on cardiovascular outcomes trials in type-2 diabetes patients of glucose-lowering treatments where patients in the placebo arm are more likely to add other glucose-lowering agents with established…

实际正值性违背下平均处理效应 TMLE 的目标化策略与截断研究AI 解读

Estimating average treatment effects from observational data is challenging under practical violations of the positivity assumption. Targeted Maximum Likelihood Estimators (TMLEs) are widely used because of their double robustness and efficiency, but they can remain sensitive to such violations. We conduct extensive simulation studies to examine how targeting strategies and truncation levels affect TMLE performance under varying degrees of outcome regression misspecification…

未测量混杂下因果直接效应的识别与估计AI 解读

Causal mediation analysis provides techniques for defining and estimating effects that may be endowed with mechanistic interpretations. With many scientific investigations seeking to address mechanistic questions, causal direct and indirect effects have garnered much attention. The natural direct and indirect effects, the most widely used among such causal mediation estimands, are limited in their practical utility due to stringent identification requirements. Accordingly,…

主成分高度自适应经验风险最小化AI 解读

The Highly Adaptive Lasso (HAL) delivers unprecedented guarantees in nonparametric minimum loss estimation under minimal smoothness assumptions, such as dimension-free minimax optimal rates. However, the practical use of HAL has been severely limited by its exponentially growing computationally prohibitive indicator basis expansion in moderate to high dimensions. Existing screening strategies drastically reduce this dimension but lack any theoretical justification. We…

用预后评分调整提升广义线性模型随机试验边际效应分析的功效AI 解读

In randomized clinical trials (RCTs), the accurate estimation of marginal treatment effects is crucial for determining the efficacy of interventions. Enhancing the statistical power of these analyses is a key objective for statisticians. The increasing availability of historical data from registries, prior trials, and health records presents an opportunity to improve trial efficiency. However, many methods for historical borrowing compromise strict type-I error rate control.…

混杂变量缺失时的处理效应评估：双重稳健与传统缺失数据方法比较AI 解读

In pharmacoepidemiology, safety and effectiveness are frequently evaluated using readily available administrative and electronic health records data. In these settings, detailed confounder data are often not available in all data sources and therefore missing on a subset of individuals. Multiple imputation (MI) and inverse-probability weighting (IPW) are go-to analytical methods to handle missing data and are dominant in the biomedical literature. Doubly-robust methods,…

因果推断中 Plasmode 模拟研究的注意事项AI 解读

Plasmode simulation has become an important tool for evaluating the operating characteristics of different statistical methods in complex settings, such as pharmacoepidemiological studies of treatment effectiveness using electronic health records (EHR) data. These studies provide insight into how estimator performance is impacted by challenges including rare events, small sample size, etc., that can indicate which among a set of methods performs best in a real-world dataset.…

连续时间靶向极大似然与高度自适应 Lasso 的干预特定复发事件期望次数估计AI 解读

Longitudinal settings involving outcome, competing risks and censoring events occurring and recurring in continuous time are common in medical research, but are often analyzed with methods that do not allow for taking post-baseline information into account. In this work, we define statistical and causal target parameters via the g-computation formula by carrying out interventions directly on the product integral representing the observed data distribution in a…

因果剂量响应函数的非参数推断方法AI 解读

The causal dose response curve is commonly selected as the statistical parameter of interest in studies where the goal is to understand the effect of a continuous exposure on an outcome.Most of the available methodology for statistical inference on the dose-response function in the continuous exposure setting requires strong parametric assumptions on the probability distribution. Such parametric assumptions are typically untenable in practice and lead to invalid inference.…

整群随机试验中的效应定义与估计：方法比较AI 解读

Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs, several challenges remain. First, there are many possible ways to specify the causal effect of interest (e.g., at the individual-level or at the cluster-level). Second, the theoretical and practical performance of common methods for CRT…

具有网络与时间依赖的自适应序贯监测AI 解读

Strategic test allocation plays a major role in the control of both emerging and existing pandemics (e.g., COVID-19, HIV). Widespread testing supports effective epidemic control by (1) reducing transmission via identifying cases, and (2) tracking outbreak dynamics to inform targeted interventions. However, infectious disease surveillance presents unique statistical challenges. For instance, the true outcome of interest - one's positive infectious status, is often a latent…

重审倾向得分的核心作用：连接因果机器学习中的平衡与效率AI 解读

About forty years ago, in a now--seminal contribution, Rosenbaum & Rubin (1983) introduced a critical characterization of the propensity score as a central quantity for drawing causal inferences in observational study settings. In the decades since, much progress has been made across several research fronts in causal inference, notably including the re-weighting and matching paradigms. Focusing on the former and specifically on its intersection with machine learning and…

基于广义倾向得分的修正处理政策效应有效估计AI 解读

Continuous treatments have posed a significant challenge for causal inference, both in the formulation and identification of scientifically meaningful effects and in their robust estimation. Traditionally, focus has been placed on techniques applicable to binary or categorical treatments with few levels, allowing for the application of propensity score-based methodology with relative ease. Efforts to accommodate continuous treatments introduced the generalized propensity…

通过营养干预试验的现实模拟评估目标最大似然估计的稳健性AI 解读

Several recently developed methods have the potential to harness machine learning in the pursuit of target quantities inspired by causal inference, including inverse weighting, doubly robust estimating equations and substitution estimators like targeted maximum likelihood estimation. There are even more recent augmentations of these procedures that can increase robustness, by adding a layer of cross-validation (cross-validated targeted maximum likelihood estimation and…

单条时间序列的自适应序贯试验设计AI 解读

The current work is motivated by the need for robust statistical methods for precision medicine; as such, we address the need for statistical methods that provide actionable inference for a single unit at any point in time. We aim to learn an optimal, unknown choice of the controlled components of the design in order to optimize the expected outcome; with that, we adapt the randomization mechanism for future time-point experiments based on the data collected on the…