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Effectively Selecting a Target Population for a Future Comparative Study

为未来比较研究有效选择目标人群

When comparing a new treatment with a control in a randomized clinical study, the treatment effect is generally assessed by evaluating a summary measure over a specific study population. The success of the trial heavily depends on the choice of such a population. In this paper, we show a systematic, effective way to identify a promising population, for which the new treatment is expected to have a desired benefit, utilizing the data from a current study involving similar…

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Landmark Prediction of Long-Term Survival Incorporating Short-Term Event Time Information

纳入短期事件时间信息的长期生存 Landmark 预测

In recent years, a wide range of markers have become available as potential tools to predict risk or progression of disease. In addition to such biological and genetic markers, short term outcome information may be useful in predicting long term disease outcomes. When such information is available, it would be desirable to combine this along with predictive markers to improve the prediction of long term survival. Most existing methods for incorporating censored short term…

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Nonconcave penalized composite conditional likelihood estimation of sparse Ising models

稀疏 Ising 模型的非凹惩罚复合条件似然估计

The Ising model is a useful tool for studying complex interactions within a system. The estimation of such a model, however, is rather challenging, especially in the presence of high-dimensional parameters. In this work, we propose efficient procedures for learning a sparse Ising model based on a penalized composite conditional likelihood with nonconcave penalties. Nonconcave penalized likelihood estimation has received a lot of attention in recent years. However, such an…

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A Perturbation Method for Inference on Regularized Regression Estimates

正则化回归估计推断的扰动方法

Analysis of high dimensional data often seeks to identify a subset of important features and assess their effects on the outcome. Traditional statistical inference procedures based on standard regression methods often fail in the presence of high-dimensional features. In recent years, regularization methods have emerged as promising tools for analyzing high dimensional data. These methods simultaneously select important features and provide stable estimation of their…

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Nonparametric Evaluation of Biomarker Accuracy Under Nested Case-Control Studies

嵌套病例对照研究中生物标志物准确性的非参数评价

To evaluate the clinical utility of new risk markers, a crucial step is to measure their predictive accuracy with prospective studies. However, it is often infeasible to obtain marker values for all study participants. The nested case-control (NCC) design is a useful cost-effective strategy for such settings. Under the NCC design, markers are only ascertained for cases and a fraction of controls sampled randomly from the risk sets. The outcome dependent sampling generates a…

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Time-Dependent Predictive Values of Prognostic Biomarkers With Failure Time Outcome

生存结局下预后生物标志物的时间依赖预测值

In a prospective cohort study, information on clinical parameters, tests and molecular markers is often collected. Such information is useful to predict patient prognosis and to select patients for targeted therapy. We propose a new graphical approach, the positive predictive value (PPV) curve, to quantify the predictive accuracy of prognostic markers measured on a continuous scale with censored failure time outcome. The proposed method highlights the need to consider both…

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Evaluating Prediction Rules for t -Year Survivors With Censored Regression Models

利用删失回归模型评估 t 年生存者预测规则

Suppose that we are interested in establishing simple but reliable rules for predicting future t-year survivors through censored regression models. In this article we present inference procedures for evaluating such binary classification rules based on various prediction precision measures quantified by the overall misclassification rate, sensitivity and specificity, and positive and negative predictive values. Specifically, under various working models, we derive consistent…

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On the accelerated failure time model for current status and interval censored data

现状及区间删失数据的加速失效时间模型

This paper introduces a novel approach to making inference about the regression parameters in the accelerated failure time model for current status and interval censored data. The estimator is constructed by inverting a Wald-type test for testing a null proportional hazards model. A numerically efficient Markov chain Monte Carlo based resampling method is proposed for obtaining simultaneously the point estimator and a consistent estimator of its variance-covariance matrix.…

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Semiparametric Box–Cox power transformation models for censored survival observations

删失生存观测的半参数 Box–Cox 幂变换模型

The accelerated failure time model specifies that the logarithm of the failure time is linearly related to the covariate vector without assuming a parametric error distribution. In this paper, we consider the semiparametric Box–Cox transformation model, which includes the above regression model as a special case, to analyse possibly censored failure time observations. Inference procedures for the transformation and regression parameters are proposed via a resampling…

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Semiparametric Receiver Operating Characteristic Analysis to Evaluate Biomarkers for Disease

评价疾病生物标志物的半参数ROC分析

The receiver operating characteristic (ROC) curve is a popular method for characterizing the accuracy of diagnostic tests when test results are not binary. Various methodologies for estimating and comparing ROC curves have been developed. One approach, due to Pepe, uses a parametric regression model ROCx(u) = g(h0(u) + θ'0x) with the baseline function h0(u) specified up to a finite-dimensional parameter. In this article we extend the regression models by allowing arbitrary…

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