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Risk Projection for Time-to-Event Outcome Leveraging Summary Statistics With Source Individual-Level Data

结合目标总体汇总统计与源总体个体数据的事件发生风险外推

Predicting risks of chronic diseases has become increasingly important in clinical practice. When a prediction model is developed in a cohort, there is a great interest to apply the model to other cohorts. Due to potential discrepancy in baseline disease incidences between different cohorts and shifts in patient composition, the risk predicted by the model built in the source cohort often under- or over-estimates the risk in a new cohort. In this article, we assume the…

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Nonparametric Maximum Likelihood Estimators of Time-Dependent Accuracy Measures for Survival Outcome Under Two-Stage Sampling Designs

两阶段抽样下生存结局时变准确性指标的非参数极大似然估计

Large prospective cohort studies of rare chronic diseases require thoughtful planning of study designs, especially for biomarker studies when measurements are based on stored tissue or blood specimens. Two-phase designs, including nested case–control and case-cohort sampling designs, provide cost-effective strategies for conducting biomarker evaluation studies.Existing literature for biomarker assessment under two-phase designs largely focuses on simple inverse probability…

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Estimating Risk With Time-to-Event Data: An Application to the Women’s Health Initiative

事件发生时间数据的风险估计:妇女健康倡议应用

Accurate and individualized risk prediction is critical for population control of chronic diseases such as cancer and cardiovascular disease. Large cohort studies provide valuable resources for building risk prediction models, as the risk factors are collected at the baseline and subjects are followed over time until disease occurrence or termination of the study. However, for rare diseases the baseline risk may not be estimated reliably based on cohort data only, due to…

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Resampling Procedures for Making Inference Under Nested Case–Control Studies

嵌套病例对照研究中的推断重抽样程序

The nested case-control (NCC) design have been widely adopted as a cost-effective solution in many large cohort studies for risk assessment with expensive markers, such as the emerging biologic and genetic markers. To analyze data from NCC studies, conditional logistic regression (Goldstein and Langholz, 1992; Borgan et al., 1995) and maximum likelihood (Scheike and Juul, 2004; Zeng et al., 2006) based methods have been proposed. However, most of these methods either cannot…

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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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Application of Multidimensional Selective Item Response Regression Model for Studying Multiple Gene Methylation in SV40 Oncogenic Pathways

用多维选择性项目反应回归研究 SV40 致癌通路中的多基因甲基化

Alteration of gene methylation patterns has been reported to be involved in the early onsets of many human malignancies. Many exogenous risk factors, such as cigarette smoke, dietary additives, chemical exposures, radiation, and biologic agents including viral infection, are involved in the methylation pathways of cancers. We propose a multidimensional selective item response regression model to describe and test how a risk factor may alter molecular pathways involving…

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