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Learn-As-you-GO (LAGO) Trials: Optimizing Trials for Effectiveness and Power to Prevent Failed Trials

边学边开展的 LAGO 试验:以干预效果与检验功效优化设计

The Learn-As-you-GO (LAGO) design provides a rigorous framework for adapting the intervention package based on accumulating data while the trial is ongoing. This article improves the flexibility of the LAGO design by incorporating statistical power as an optimization criterion (power goal) in LAGO optimizations. We propose the unconditional and conditional power approaches to add a power goal. Both approaches estimate the power at the end of the LAGO trial using data from…

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Estimating treatment effects from observational data under truncation by death using survival-incorporated quantiles

死亡截断下利用纳入生存信息的分位数估计观测数据处理效应

The issue of "truncation by death" commonly arises in clinical research: subjects may die before their follow-up assessment, resulting in undefined clinical outcomes. To address this issue, we focus on survival-incorporated quantiles -- quantiles of a composite outcome combining death and clinical outcomes -- to summarize the effect of treatment. Using inverse probability of treatment weighting (IPTW), we propose an estimator for survival-incorporated quantiles from…

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How estimating nuisance parameters can reduce the variance (with consistent variance estimation)

估计干扰参数如何降低方差并实现一致方差估计

We often estimate a parameter of interest psi when the identifying conditions involve a nuisance parameter theta. Examples from causal inference are Inverse Probability Weighting, Marginal Structural Models and Structural Nested Models. To estimate treatment effects from observational data, these methods posit a (pooled) logistic regression model for the treatment and/or censoring probabilities and estimate these first. These methods are all based on unbiased estimating…

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Causal mediation analysis with mediator values below an assay limit

中介变量低于检测下限时的因果中介分析

Causal indirect and direct effects provide an interpretable method for decomposing the total effect of an exposure on an outcome into the effect through a mediator and the effect through all other pathways. When the mediator is a biomarker, values can be subject to an assay lower limit. The mediator is affected by the treatment and is a putative cause of the outcome, so the assay lower limit presents a compounded problem in mediation analysis. We propose three approaches to…

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Optimal estimation of coarse structural nested mean models with application to initiating ART in HIV infected patients

粗粒度结构嵌套均值模型的最优估计:HIV患者启动抗逆转录病毒治疗的应用

Coarse structural nested mean models are used to estimate treatment effects from longitudinal observational data. Coarse structural nested mean models lead to a large class of estimators. It turns out that estimates and standard errors may differ considerably within this class. We prove that, under additional assumptions, there exists an explicit solution for the optimal estimator within the class of coarse structural nested mean models. Moreover, we show that even if the…

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