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Interpretable Dynamic Treatment Regimes

可解释的动态治疗方案

Precision medicine is currently a topic of great interest in clinical and intervention science. A key component of precision medicine is that it is evidence-based, i.e., data-driven, and consequently there has been tremendous interest in estimation of precision medicine strategies using observational or randomized study data. One way to formalize precision medicine is through a treatment regime, which is a sequence of decision rules, one per stage of clinical intervention,…

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On Estimation of Optimal Treatment Regimes for Maximizing t -Year Survival Probability

最大化 t 年生存概率的最优治疗策略估计

Summary A treatment regime is a deterministic function that dictates personalized treatment based on patients’ individual prognostic information. There is increasing interest in finding optimal treatment regimes, which determine treatment at one or more treatment decision points to maximize expected long-term clinical outcomes, where larger outcomes are preferred. For chronic diseases such as cancer or human immunodeficiency virus infection, survival time is often the…

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Improving efficiency and robustness of the doubly robust estimator for a population mean with incomplete data

改进不完整数据下总体均值双重稳健估计的效率与稳健性

Abstract Considerable recent interest has focused on doubly robust estimators for a population mean response in the presence of incomplete data, which involve models for both the propensity score and the regression of outcome on covariates. The usual doubly robust estimator may yield severely biased inferences if neither of these models is correctly specified and can exhibit nonnegligible bias if the estimated propensity score is close to zero for some observations. We…

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Latent-model robustness in structural measurement error models

结构测量误差模型中的潜变量模型稳健性

We present methods for diagnosing the effects of model misspecification of the true-predictor distribution in structural measurement error models. We first formulate latent-model robustness theoretically. Then we provide practical techniques for examining the adequacy of an assumed latent predictor model. The methods are illustrated via analytical examples, application to simulated data and with data from a study of coronary heart disease.

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A Two-Step Approach to Measurement Error in Time-Dependent Covariates in Nonlinear Mixed-Effects Models, with Application to IGF-I Pharmacokinetics

非线性混合效应模型中时变协变量测量误差的两步处理:IGF-I药代动力学应用

The usual approach to the analysis of population pharmacokinetic studies is to represent the concentration-time data by a nonlinear mixed-effects model. Primary objectives are to characterize the pattern of drug disposition in the population and to identify individual-specific covariates associated with pharmacokinetic behavior. We consider data from a study of insulin-like growth factor I (IGF-I) administered by intravenous infusion to patients with severe head trauma.…

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Nonlinear Models for Repeated Measurement Data

重复测量数据的非线性模型

Nonlinear mixed effects models for data in the form of continuous, repeated measurements on each of a number of individuals, also known as hierarchical nonlinear models, are a popular platform for analysis when interest focuses on individual-specific characteristics.This framework first enjoyed widespread attention within the statistical research community in the late 1980s, and the 1990s saw vigorous development of new methodological and computational techniques for these…

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A Two-Step Approach to Measurement Error in Time-Dependent Covariates in Nonlinear Mixed-Effects Models, With Application to IGF-I Pharmacokinetics

非线性混合效应模型中时变协变量测量误差的两步法:IGF-I 药代动力学应用

The usual approach to the analysis of population pharmacokinetic studies is to represent the concentration-time data by a nonlinear mixed-effects model. Primary objectives are to characterize the pattern of drug disposition in the population and to identify individual-specific covariates associated with pharmacokinetic behavior. We consider data from a study of insulin-like growth factor I (IGF-I) administered by intravenous infusion to patients with severe head trauma.…

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Bootstrap-Adjusted Calibration Confidence Intervals for Immunoassay

免疫测定中经自助法调整的校准置信区间

In immunoassay, a nonlinear heteroscedastic regression model is used to characterize assay concentration-response, and the model fitted to data from standard samples is used to calibrate unknown test samples. Usual large-sample methods to construct individual confidence intervals for calibrated concentrations have been observed in empirical studies to be seriously inaccurate in terms of achieving the nominal level of coverage. We show theoretically that this inaccuracy is…

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The nonlinear mixed effects model with a smooth random effects density

具有光滑随机效应密度的非线性混合效应模型

The fixed parameters of the nonlinear mixed effects model and the density of the random effects are estimated jointly by maximum likelihood. The density of the random effects is assumed to be smooth but is otherwise unrestricted. The method uses a series expansion that follows from the smoothness assumption to represent the density and quadrature to compute the likelihood. Standard algorithms are used for optimization. Empirical Bayes estimates of random coefficients are…

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