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Sequential multiple assignment randomized trials (SMARTs) provide a systematic framework for constructing and evaluating dynamic treatment regimens (DTRs). In clinical studies, longitudinal biomarkers are routinely collected to monitor…

Methodology · Statistics 2026-05-06 Zhengxi Chen , Holly Hartman

A survival analysis model for predicting time-to-total knee replacement (TKR) was developed using features from medical images and clinical measurements. Supervised and self-supervised deep learning approaches were utilized to extract…

Image and Video Processing · Electrical Eng. & Systems 2024-11-19 Ozkan Cigdem , Shengjia Chen , Chaojie Zhang , Kyunghyun Cho , Richard Kijowski , Cem M. Deniz

Estimating counterfactual outcomes over time has the potential to unlock personalized healthcare by assisting decision-makers to answer ''what-iF'' questions. Existing causal inference approaches typically consider regular, discrete-time…

Machine Learning · Computer Science 2022-06-17 Nabeel Seedat , Fergus Imrie , Alexis Bellot , Zhaozhi Qian , Mihaela van der Schaar

It is a common practice in randomized clinical trials with the standard survival outcome to follow patients until a prespecified number of events have been observed, a type of trial known as the event-driven trial. The event-driven design…

Methodology · Statistics 2026-02-10 Jingwen Zhang , Satoshi Hattori

Predictive models in acute care settings must be able to immediately recognize precipitous changes in a patient's status when presented with data reflecting such changes. Recurrent neural networks (RNNs) have become common for training and…

Machine Learning · Computer Science 2020-07-30 David Ledbetter , Eugene Laksana , Melissa Aczon , Randall Wetzel

In many important applications of precision medicine, the outcome of interest is time to an event (e.g., death, relapse of disease) and the primary goal is to identify the optimal individualized decision rule (IDR) to prolong survival time.…

Methodology · Statistics 2022-04-11 Yu Zhou , Lan Wang , Rui Song , Tuoyi Zhao

Background: Phase I dose-finding trials increasingly encounter delayed-onset toxicities, especially with immunotherapies and targeted agents. The time-to-event continual reassessment method (TITE-CRM) handles incomplete follow-up using…

Methodology · Statistics 2026-02-24 Robert Amevor , Emmanuel Kubuafor , Dennis Baidoo

We are now witnessing the increasing availability of event stream data, i.e., a sequence of events with each event typically being denoted by the time it occurs and its mark information (e.g., event type). A fundamental problem is to model…

Machine Learning · Computer Science 2017-02-12 Yongqing Wang , Shenghua Liu , Huawei Shen , Xueqi Cheng

Electronic health record (EHR) data are increasingly used to support real-world evidence (RWE) studies. Yet its ability to generate reliable RWE is limited by the lack of readily available precise information on the timing of clinical…

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…

Methodology · Statistics 2025-01-30 Sky Qiu , Jens Tarp , Andrew Mertens , Mark van der Laan

Linear mixed models are commonly used in analyzing stepped-wedge cluster randomized trials (SW-CRTs). A key consideration for analyzing a SW-CRT is accounting for the potentially complex correlation structure, which can be achieved by…

Methodology · Statistics 2024-08-21 Yongdong Ouyang , Monica Taljaard , Andrew B Forbes , Fan Li

Joint models initially dedicated to a single longitudinal marker and a single time-to-event need to be extended to account for the rich longitudinal data of cohort studies. Multiple causes of clinical progression are indeed usually…

Applications · Statistics 2016-01-26 Cécile Proust-Lima , Jean-François Dartigues , Hélène Jacqmin-Gadda

Assessing the causal effect of time-varying exposures on recurrent event processes is challenging in the presence of a terminating event. Our objective is to estimate both the short-term and delayed marginal causal effects of exposures on…

Given the high incidence of cardio and cerebrovascular diseases (CVD), and its association with morbidity and mortality, its prevention is a major public health issue. A high level of blood pressure is a well-known risk factor for these…

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

The task of predicting long-term patient outcomes using supervised machine learning is a challenging one, in part because of the high variance of each patient's trajectory, which can result in the model over-fitting to the training data.…

Machine Learning · Computer Science 2026-02-09 Thomas Frost , Kezhi Li , Steve Harris

Many studies employ the analysis of time-to-event data that incorporates competing risks and right censoring. Most methods and software packages are geared towards analyzing data that comes from a continuous failure time distribution.…

Methodology · Statistics 2025-06-06 Tomer Meir , Malka Gorfine

Timed Transition Models (TTMs) are event-based descriptions for modelling, specifying, and verifying discrete real-time systems. An event can be spontaneous, fair, or timed with specified bounds. TTMs have a textual syntax, an operational…

Software Engineering · Computer Science 2015-06-12 Chen-Wei Wang , Jonathan S. Ostroff , Simon Hudon

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered":…

In many medical studies, patients are followed longitudinally and interest is on assessing the relationship between longitudinal measurements and time to an event. Recently, various authors have proposed joint modeling approaches for…

Applications · Statistics 2010-11-16 Paul S. Albert , Joanna H. Shih