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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

Time-homogeneous Markov chains are often used as disease progression models in studies of cost-effectiveness and optimal decision-making. Maximum likelihood estimation of these models can be challenging when data are collected at a time…

Methodology · Statistics 2022-09-26 Duncan Ermini Leaf

We consider robustness to distribution shifts in the context of diagnostic models in healthcare, where the prediction target $Y$, e.g., the presence of a disease, is causally upstream of the observations $X$, e.g., a biomarker. Distribution…

The regression discontinuity (RD) design is a quasi-experimental design that estimates the causal effects of a treatment by exploiting naturally occurring treatment rules. It can be applied in any context where a particular treatment or…

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

Methodology · Statistics 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

Estimating the causal treatment effects by subgroups is important in observational studies when the treatment effect heterogeneity may be present. Existing propensity score methods rely on a correctly specified propensity score model. Model…

Methodology · Statistics 2024-04-19 Yan Li , Yong-Fang Kuo , Liang Li

Although the notion of diagnostic problem has been extensively investigated in the context of static systems, in most practical applications the behavior of the modeled system is significantly variable during time. The goal of the paper is…

Artificial Intelligence · Computer Science 2013-03-25 Luigi Portinale

Consider the problem of estimating average treatment effects when a large number of covariates are used to adjust for possible confounding through outcome regression and propensity score models. The conventional approach of model building…

Statistics Theory · Mathematics 2018-01-31 Zhiqiang Tan

Observational longitudinal studies are a common means to study treatment efficacy and safety in chronic mental illness. In many such studies, treatment changes may be initiated by either the patient or by their clinician and can thus vary…

Methodology · Statistics 2020-06-12 Zekun Xu , Eric Laber , Ana-Maria Staicu , Emanuel Severus

Introduction: Kidney function is reported using estimates of glomerular filtration rate (eGFR). However, eGFR values are recorded without reference to the creatinine (SCr) assays used to derive them, and newer assays were introduced at…

Quantitative Methods · Quantitative Biology 2014-09-05 Norman Poh , Andrew McGovern , Simon de Lusignan

Any reliable biomarker has to be specific, generalizable, and reproducible across individuals and contexts. The exact values of such a biomarker must represent similar health states in different individuals and at different times within the…

Quantitative Methods · Quantitative Biology 2023-05-12 Madhur Mangalam , Arash Sadri , Junichiro Hayano , Eiichi Watanabe , Ken Kiyono , Damian G. Kelty-Stephen

We propose a nonparametric bivariate time-varying coefficient model for longitudinal measurements with the occurrence of a terminal event that is subject to right censoring. The time-varying coefficients capture the longitudinal…

Methodology · Statistics 2021-11-10 Yue Wang , Bin Nan , Jack D. Kalbfleisch

Parkinson's disease (PD) is a common neurological disorder characterized by gait impairment. PD has no cure, and an impediment to developing a treatment is the lack of any accepted method to predict disease progression rate. The primary aim…

Signal Processing · Electrical Eng. & Systems 2019-11-26 Vyom Raval , Kevin P. Nguyen , Ashley Gerald , Richard B. Dewey , Albert Montillo

Many rare diseases offer limited established treatment options, leading patients to switch therapies when new medications emerge. To analyze the impact of such treatment switches within the low sample size limitations of rare disease…

The paper algorithmizes the problem of regime change point identification for data measured in a system exhibiting impulsive behaviors. This is a fundamental challenge for annotation of measurement data relevant, e.g., for designing…

Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

We consider causal inference in dynamic settings where treatment is assigned by thresholding a state variable that can change over time. There is a large literature on regression-discontinuity methods building on the fact that, in the…

Methodology · Statistics 2026-05-25 Aditya Ghosh , Stefan Wager

Passive and non-obtrusive health monitoring using wearables can potentially bring new insights into the user's health status throughout the day and may support clinical diagnosis and treatment. However, identifying segments of free-living…

Signal Processing · Electrical Eng. & Systems 2019-01-31 Yordan P. Raykov , Luc J. W. Evers , Reham Badawy , Marjan J. Faber , Bastiaan R. Bloem , Kasper Claes , Max A. Little

Hypertension is a major risk factor for stroke, cardiovascular disease, and end-stage renal disease, and its prevalence is expected to rise dramatically. Effective hypertension management is thus critical. A particular priority is…

Computers and Society · Computer Science 2019-07-02 Ramin Mohammadi , Sarthak Jain , Stephen Agboola , Ramya Palacholla , Sagar Kamarthi , Byron C. Wallace

Dynamic prediction of future clinical outcomes based on longitudinally measured predictors plays a crucial role in disease management and patient counseling, particularly when conventional static models are inadequate. Joint modeling of…

Methodology · Statistics 2025-07-30 Wenhao Li , Shikun Wang , Zhe Yin , Brad C. Astor , Wei Yang , Tom H. Greene , Liang Li
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