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After the diagnosis of a disease, one major objective is to predict cumulative probabilities of events such as clinical relapse or death from the individual information collected up to a prediction time, including usually biomarker repeated…

Applications · Statistics 2018-08-08 Loïc Ferrer , Hein Putter , Cécile Proust-Lima

Joint models for longitudinal and survival data have gained a lot of attention in recent years, with the development of myriad extensions to the basic model, including those which allow for multivariate longitudinal data, competing risks…

Methodology · Statistics 2020-03-09 Katya Mauff , Ewout Steyerberg , Isabella Kardys , Eric Boersma , Dimitris Rizopoulos

One primary goal of precision medicine is to estimate the individualized treatment rules (ITRs) that optimize patients' health outcomes based on individual characteristics. Health studies with multiple treatments are commonly seen in…

Methodology · Statistics 2025-05-07 Xuqiao Li , Qiuyan Zhou , Ying Wu , Ying Yan

In applications such as biomedical studies, epidemiology, and social sciences, recurrent events often co-occur with longitudinal measurements and a terminal event, such as death. Therefore, jointly modeling longitudinal measurements,…

Machine Learning · Statistics 2024-04-08 Zhiyue Zhang , Yao Zhao , Yanxun Xu

We consider the estimation of treatment effects in settings when multiple treatments are assigned over time and treatments can have a causal effect on future outcomes or the state of the treated unit. We propose an extension of the…

Econometrics · Economics 2021-06-18 Greg Lewis , Vasilis Syrgkanis

Interfractional geometric uncertainties can lead to deviations of the actual delivered dose from the prescribed dose distribution. To better handle these uncertainties during treatment, the authors propose a dynamic framework for robust…

Medical Physics · Physics 2019-05-16 Michelle Böck , Kjell Eriksson , Anders Forsgren

In many clinical trials studying neurodegenerative diseases such as Parkinson's disease (PD), multiple longitudinal outcomes are collected to fully explore the multidimensional impairment caused by this disease. If the outcomes deteriorate…

Applications · Statistics 2017-05-18 Jue Wang , Sheng Luo , Liang Li

We consider Dynamic Treatment Regimes (DTRs) with One Sided Noncompliance that arise in applications such as digital recommendations and adaptive medical trials. These are settings where decision makers encourage individuals to take…

Econometrics · Economics 2025-05-06 Ravi B. Sojitra , Vasilis Syrgkanis

Longitudinal observational patient data can be used to investigate the causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for controlling for the time-dependent confounding that…

Methodology · Statistics 2021-10-08 Ruth H. Keogh , Jon Michael Gran , Shaun R. Seaman , Gwyneth Davies , Stijn Vansteelandt

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

Methodology · Statistics 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

Public policies and medical interventions often involve dynamic treatment assignments, in which individuals receive a sequence of interventions over multiple stages. We study the statistical learning of optimal dynamic treatment regimes…

Methodology · Statistics 2025-05-21 Shosei Sakaguchi

Precision medicine leverages patient heterogeneity to estimate individualized treatment regimens, formalized, data-driven approaches designed to match patients with optimal treatments. In the presence of competing events, where multiple…

In oncology clinical trials, tumor burden (TB) stands as a crucial longitudinal biomarker, reflecting the toll a tumor takes on a patient's prognosis. With certain treatments, the disease's natural progression shows the tumor burden…

Methodology · Statistics 2024-09-24 Ethan M. Alt , Yixiang Qu , Emily Damone , Jing-ou Liu , Chenguang Wang , Joseph G. Ibrahim

The goal of precision medicine is to provide individualized treatment at each stage of chronic diseases, a concept formalized by Dynamic Treatment Regimes (DTR). These regimes adapt treatment strategies based on decision rules learned from…

Methodology · Statistics 2025-06-09 Sophia Yazzourh , Nicolas Savy , Philippe Saint-Pierre , Michael R. Kosorok

In modern biomedical and econometric studies, longitudinal processes are often characterized by complex time-varying associations and abrupt regime shifts that are shared across correlated outcomes. Standard functional data analysis (FDA)…

Methodology · Statistics 2026-01-28 Baolin Chen , Mengfei Ran

Dynamic treatment regimes (DTR) are a statistical paradigm in precision medicine which aim to optimize patient outcomes by individualizing treatments. At its simplest, a DTR may require only a single decision to be made; this special case…

Applications · Statistics 2021-09-06 Larry Dong , Erica E. M. Moodie , Laura Villain , Rodolphe Thiébaut

Patients with chronic diseases often receive treatments at multiple time points, or stages. Our goal is to learn the optimal dynamic treatment regime (DTR) from longitudinal patient data. When both the number of stages and the number of…

Statistics Theory · Mathematics 2025-05-26 Nilanjana Laha , Nilson Chapagain , Victoria Cicherski , Aaron Sonabend-W

Dynamic treatment regimes (DTRs) are sequences of functions that formalize the process of precision medicine. DTRs take as input patient information and output treatment recommendations. A major focus of the DTR literature has been on the…

Methodology · Statistics 2024-02-21 Dylan Spicker , Michael P. Wallace , Grace Y. Yi

Dynamic treatment regimes (DTRs) aim to formalize personalized medicine by tailoring treatment decisions to individual patient characteristics. G-estimation for DTR identification targets the parameters of a structural nested mean model…

Methodology · Statistics 2017-04-27 M. P. Wallace , E. E. M. Moodie , D. A. Stephens
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