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Randomized experiments (a.k.a. A/B tests) are a powerful tool for estimating treatment effects, to inform decisions making in business, healthcare and other applications. In many problems, the treatment has a lasting effect that evolves…

Machine Learning · Computer Science 2022-10-17 Ziyang Tang , Yiheng Duan , Stephanie Zhang , Lihong Li

Stroke remains a leading cause of death and disability worldwide, yet effective prediction of stroke risk using large-scale population data remains challenging due to data imbalance and high-dimensional features. In this study, we develop…

Applications · Statistics 2025-10-28 Jinbo Niu

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Recent observations, especially in cancer immunotherapy clinical trials with time-to-event outcomes, show that the commonly used proportial hazard assumption is often not justifiable, hampering an appropriate analyse of the data by hazard…

Methodology · Statistics 2021-02-23 Marc Ditzhaus , Menggang Yu , Jin Xu

Background: An early diagnosis together with an accurate disease progression monitoring of multiple sclerosis is an important component of successful disease management. Prior studies have established that multiple sclerosis is correlated…

Audio and Speech Processing · Electrical Eng. & Systems 2021-09-28 Emil Svoboda , Tomáš Bořil , Jan Rusz , Tereza Tykalová , Dana Horáková , Charles R. G. Guttman , Krastan B. Blagoev , Hiroto Hatabu , Vlad I. Valtchinov

One of the notorious issues for Reinforcement Learning (RL) is poor sample efficiency. Compared to single agent RL, the sample efficiency for Multi-Agent Reinforcement Learning (MARL) is more challenging because of its inherent partial…

Machine Learning · Computer Science 2024-04-16 Linjie Xu , Zichuan Liu , Alexander Dockhorn , Diego Perez-Liebana , Jinyu Wang , Lei Song , Jiang Bian

A popular setting in medical statistics is a group sequential trial with independent and identically distributed normal outcomes, in which interim analyses of the sum of the outcomes are performed. Based on a prescribed stopping rule, one…

Statistics Theory · Mathematics 2018-04-03 Ben Berckmoes , Anna Ivanova , Geert Molenberghs

Motivated by the Acute Respiratory Distress Syndrome Network (ARDSNetwork) ARDS respiratory management (ARMA) trial, we developed a flexible Bayesian machine learning approach to estimate the average causal effect and heterogeneous causal…

Applications · Statistics 2024-10-29 Xinyuan Chen , Michael O. Harhay , Guangyu Tong , Fan Li

Multiple myeloma (MM) is preceded by a long preclinical phase spanning decades, yet scalable, non-specialist tools to identify individuals at elevated risk before end-organ damage are lacking. In a prospective analysis of 299,035…

Applications · Statistics 2026-04-07 Bingjie Li , Jiadai Xu , Yiqing Sun , Feiyue Pan , Shing-Tung Yau , Peng Liu , Zhigang Yao

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…

Amyotrophic lateral sclerosis (ALS) is a degenerative disorder of the motor neurons that causes progressive paralysis in patients. Current treatment options aim to prolong survival and improve quality of life. However, due to the…

Quantitative Methods · Quantitative Biology 2025-11-20 Christian Marius Lillelund , Sanjay Kalra , Russell Greiner

In this study we investigate the Percolation Hypothesis for Multiple Sclerosis Progression. The methodology relies on cross-reference analysis centered around a question: What is the evidence for a Percolation/phase-transition hypothesis in…

Disordered Systems and Neural Networks · Physics 2026-02-10 Nikola Mirkov , Dušan S. Radivojević , Slobodan Maletić

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Robins (1998) introduced marginal structural models (MSMs), a general class of counterfactual models for the joint effects of time-varying treatment regimes in complex longitudinal studies subject to time-varying confounding. He established…

Methodology · Statistics 2018-09-17 Eric J Tchetgen Tchetgen , Haben Michael , Yifan Cui

INTRODUCTION: Heterogeneity in the progression of Alzheimer's disease makes it challenging to predict the rate of cognitive and functional decline for individual patients. Tools for short-term prediction could help enrich clinical trial…

Quantitative Methods · Quantitative Biology 2021-01-22 Neda Shafiee , Mahsa Dadar , Simon Ducharme , D. Louis Collins

Alzheimer's disease (AD) is a progressive and irreversible brain disorder that unfolds over the course of 30 years. Therefore, it is critical to capture the disease progression in an early stage such that intervention can be applied before…

Machine Learning · Computer Science 2024-09-02 Yipei Wang , Bing He , Shannon Risacher , Andrew Saykin , Jingwen Yan , Xiaoqian Wang

A targeted learning (TL) framework is developed to estimate the difference in the restricted mean survival time (RMST) for a clinical trial with time-to-event outcomes. The approach starts by defining the target estimand as the RMST…

Methodology · Statistics 2026-01-21 Man Jin , Yixin Fang

Personalized head and neck cancer therapeutics have greatly improved survival rates for patients, but are often leading to understudied long-lasting symptoms which affect quality of life. Sequential rule mining (SRM) is a promising…

Human-Computer Interaction · Computer Science 2023-09-28 Carla Floricel , Andrew Wentzel , Abdallah Mohamed , C. David Fuller , Guadalupe Canahuate , G. Elisabeta Marai
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