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Prediction performance of a risk scoring system needs to be carefully assessed before its adoption in clinical practice. Clinical preventive care often uses risk scores to screen asymptomatic population. The primary clinical interest is to…

Methodology · Statistics 2018-06-22 Yan Yuan , Qian M. Zhou , Bingying Li , Hengrui Cai , Eric J. Chow , Gregory T. Armstrong

Two different approaches to analysis of data from diagnostic biomarker studies are commonly employed. Logistic regression is used to fit models for probability of disease given marker values, while ROC curves and risk distributions are used…

Applications · Statistics 2013-12-02 Ying Huang , Margaret S. Pepe , Ziding Feng

The purpose of this paper is to construct confidence intervals for the regression coefficients in the Fine-Gray model for competing risks data with random censoring, where the number of covariates can be larger than the sample size. Despite…

Methodology · Statistics 2019-04-10 Jue Hou , Jelena Bradic , Ronghui Xu

Recent advances in causal mediation analysis have formalized conditions for estimating direct and indirect effects in various contexts. These approaches have been extended to a number of models for survival outcomes including accelerated…

Methodology · Statistics 2017-01-11 Isabel R. Fulcher , Eric Tchetgen Tchetgen , Paige L. Williams

Risk stratification is a key tool in clinical decision-making, yet current approaches often fail to translate sophisticated survival analysis into actionable clinical criteria. We present a novel method for unsupervised machine learning…

Recent advances in interrupted time series analysis permit characterization of a typical non-linear interruption effect through use of generalized additive models. Concurrently, advances in latent time series modeling allow efficient…

Applications · Statistics 2025-11-11 RJ Waken , Fengxian Wang , Sarah A. Eisenstein , Tim McBride , Kim Johnson , Karen Joynt-Maddox

We study explained variation under the additive hazards regression model for right-censored data. We consider different approaches for developing such a measure, and focus on one that estimates the proportion of variation in the failure…

Applications · Statistics 2020-09-02 Denise Rava , Ronghui Xu

An important task in health research is to characterize time-to-event outcomes such as disease onset or mortality in terms of a potentially high-dimensional set of risk factors. For example, prospective cohort studies of Alzheimer's disease…

Methodology · Statistics 2024-01-12 Harrison T. Reeder , Sebastien Haneuse , Kyu Ha Lee

In studies of time-to-event outcomes with unmeasured heterogeneity, the hazard ratio for treatment is known to have a complex causal interpretation. Accelerated failure time (AFT) models, which assess the effect on the survival time ratio…

Methodology · Statistics 2026-03-18 Mari Brathovde , Hein Putter , Morten Valberg , Richard A. J. Post

Active surveillance (AS) is a suitable management option for newly-diagnosed prostate cancer (PCa), which usually presents low to intermediate clinical risk. Patients enrolled in AS have their tumor closely monitored via longitudinal…

Active surveillance (AS) using repeated biopsies to monitor disease progression has been a popular alternative to immediate surgical intervention in cancer care. However, a biopsy procedure is invasive and sometimes leads to severe side…

Methodology · Statistics 2024-05-21 Muxuan Liang , Yingqi Zhao , Daniel W. Lin , Matthew Cooperberg , Yingye Zheng

We present a new estimator of the restricted mean survival time in randomized trials where there is right censoring that may depend on treatment and baseline variables. The proposed estimator leverages prognostic baseline variables to…

Statistics Theory · Mathematics 2016-08-22 Iván Díaz , Elizabeth Colantuoni , Daniel F. Hanley , Michael Rosenblum

Augmented inverse probability weighting and G-computation with canonical generalized linear models have become increasingly popular for estimating average treatment effects (ATEs) in randomized experiments. These methods leverage outcome…

Methodology · Statistics 2026-03-13 Muluneh Alene , Stijn Vansteelandt , Kelly Van Lancker

In cancer research, profiling studies have been extensively conducted, searching for genes/SNPs associated with prognosis. Cancer is a heterogeneous disease. Examining similarity and difference in the genetic basis of multiple subtypes of…

Methodology · Statistics 2013-04-18 Jin Liu , Jian Huang , Yawei Zhang , Qing Lan , Nathaniel Rothman , Tongzhang Zheng , Shuangge Ma

Accurately assessing a patient's risk of a given event is essential in making informed treatment decisions. One approach is to stratify patients into two or more distinct risk groups with respect to a specific outcome using both clinical…

Methodology · Statistics 2015-03-17 Karen Lostritto , Robert Strawderman , Annette Molinaro

Motivated by the need to analyze continuously updated data sets in the context of time-to-event modeling, we propose a novel nonparametric approach to estimate the conditional hazard function given a set of continuous and discrete…

Methodology · Statistics 2025-07-03 Daphné Aurouet , Valentin Patilea

Mixed-effect models are very popular for analyzing data with a hierarchical structure, e.g. repeated observations within subjects in a longitudinal design, patients nested within centers in a multicenter design. However, recently, due to…

Methodology · Statistics 2019-05-09 Abhik Ghosh , Magne Thoresen

In clinical trials, an experimental treatment is sometimes added on to a standard of care or control therapy in multiple treatment phases (e.g., concomitant and maintenance phases) to improve patient outcomes. When the new regimen provides…

Methodology · Statistics 2020-11-19 Sudipta Bhattacharya , Jyotirmoy Dey

Functional data is a powerful tool for capturing and analyzing complex patterns and relationships in a variety of fields, allowing for more precise modeling, visualization, and decision-making. For example, in healthcare, functional data…

Methodology · Statistics 2023-04-26 Xiyuan Gao , Jiayi Wang , Guanyu Hu , Jianguo Sun

The analysis of randomized trials with time-to-event endpoints is nearly always plagued by the problem of censoring. As the censoring mechanism is usually unknown, analyses typically employ the assumption of non-informative censoring. While…

Methodology · Statistics 2020-07-17 Kelly Van Lancker , Oliver Dukes , Stijn Vansteelandt