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Evaluation of Viable Dynamic Treatment Regimes in a Sequentially Randomized Trial of Advanced Prostate Cancer

晚期前列腺癌序贯随机试验中可实施动态治疗策略的评估

We present new statistical analyses of data arising from a clinical trial designed to compare two-stage dynamic treatment regimes (DTRs) for advanced prostate cancer. The trial protocol mandated that patients were to be initially randomized among four chemotherapies, and that those who responded poorly were to be rerandomized to one of the remaining candidate therapies. The primary aim was to compare the DTRs' overall success rates, with success defined by the occurrence of…

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Causal Inference in Hybrid Intervention Trials Involving Treatment Choice

包含治疗选择的混合干预试验中的因果推断

Randomized allocation of treatments is a cornerstone of experimental design but has drawbacks when a limited set of individuals are willing to be randomized, or the act of randomization undermines the success of the treatment. Choice-based experimental designs allow a subset of the participants to choose their treatments. We discuss here causal inferences for hybrid experimental designs in which some participants are randomly allocated to treatments and others receive their…

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Semiparametric Estimation in General Repeated Measures Problems

一般重复测量问题中的半参数估计

Summary The paper considers a wide class of semiparametric problems with a parametric part for some covariate effects and repeated evaluations of a nonparametric function. Special cases in our approach include marginal models for longitudinal or clustered data, conditional logistic regression for matched case–control studies, multivariate measurement error models, generalized linear mixed models with a semiparametric component, and many others. We propose profile kernel and…

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Efficient Semiparametric Marginal Estimation for Longitudinal/Clustered Data

纵向与聚类数据的有效半参数边际估计

We consider marginal generalized semiparametric partially linear models for clustered data. Lin and Carroll derived the semiparametric efficient score function for this problem in the multivariate Gaussian case, but they were unable to construct a semiparametric efficient estimator that actually achieved the semiparametric information bound. Here we propose such an estimator and generalize the work to marginal generalized partially linear models. We investigate asymptotic…

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Marginal Longitudinal Nonparametric Regression

纵向边际模型中的非参数回归

We consider nonparametric regression in a longitudinal marginal model of generalized estimating equation (GEE) type with a time-varying covariate in the situation where the number of observations per subject is finite and the number of subjects is large. In such models, the basic shape of the regression function is affected only by the covariate values and not otherwise by the ordering of the observations. Two methods of estimating the nonparametric function can be…

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Analysis of Multivariate Longitudinal Outcomes With Nonignorable Dropouts and Missing Covariates

含不可忽略退出和缺失协变量的多元纵向结果分析

This article analyzes changes in treatment practices in outpatient methadone treatment units from a national panel study. The analysis of this dataset is challenging due to several difficulties, including multiple longitudinal outcomes, nonignorable nonresponses, and missing covariates. Specifically, the data included several variables that measure the effectiveness of methadone treatment practices for each unit. A substantial percentage of units (33%%) did not respond…

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Nonparametric Function Estimation for Clustered Data When the Predictor is Measured without/with Error

自变量无测量误差或有测量误差时的聚类数据非参数函数估计

We consider local polynomial kernel regression with a single covariate for clustered data using estimating equations. We assume that at most m < ∞ observations are available on each cluster. In the case of random regressors, with no measurement error in the predictor, we show that it is generally the best strategy to ignore entirely the correlation structure within each cluster and instead pretend that all observations are independent. In the further special case of…

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Semiparametric Stochastic Mixed Models for Longitudinal Data

纵向数据的半参数随机混合模型

We consider inference for a semiparametric stochastic mixed model for longitudinal data. This model uses parametric fixed effects to represent the covariate effects and an arbitrary smooth function to model the time effect and accounts for the within-subject correlation using random effects and a stationary or nonstationary stochastic process. We derive maximum penalized likelihood estimators of the regression coefficients and the nonparametric function. The resulting…

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