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A core component of precision medicine research involves optimizing individualized treatment rules (ITRs) based on patient characteristics. Many studies used to estimate ITRs are longitudinal in nature, collecting outcomes over time. Yet,…

统计方法学 · 统计学 2024-05-29 Lanqiu Yao , Thaddeus Tarpey

This paper focuses on the problem of modeling and estimating interaction effects between covariates and a continuous treatment variable on an outcome, using a single-index regression approach. The primary motivation is to estimate an…

统计方法学 · 统计学 2021-02-02 Hyung Park , Eva Petkova , Thaddeus Tarpey , R. Todd Ogden

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…

统计方法学 · 统计学 2020-06-12 Zekun Xu , Eric Laber , Ana-Maria Staicu , Emanuel Severus

There is tremendous interest in precision medicine as a means to improve patient outcomes by tailoring treatment to individual characteristics. An individualized treatment rule formalizes precision medicine as a map from patient information…

机器学习 · 统计学 2020-05-28 Daniel J. Luckett , Eric B. Laber , Michael R. Kosorok

While studying response trajectory, often the population of interest may be diverse enough to exist distinct subgroups within it and the longitudinal change in response may not be uniform in these subgroups. That is, the timeslope and/or…

统计方法学 · 统计学 2020-06-30 Madan Gopal Kundu , Jaroslaw Harezlak

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…

统计方法学 · 统计学 2024-11-14 Christina W. Zhou , Nikki L. B. Freeman , Katharine L. McGinigle , Michael R. Kosorok

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…

统计方法学 · 统计学 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

The use of observational time series data to assess the impact of multi-time point interventions is becoming increasingly common as more health and activity data are collected and digitized via wearables, social media, and electronic health…

统计方法学 · 统计学 2020-12-01 Roy Adams , Suchi Saria , Michael Rosenblum

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

统计方法学 · 统计学 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

Precision medicine has the potential to tailor treatment decisions to individual patients using machine learning (ML) and artificial intelligence (AI), but it faces significant challenges due to complex biases in clinical observational data…

Intensive longitudinal biomarker data are increasingly common in scientific studies that seek temporally granular understanding of the role of behavioral and physiological factors in relation to outcomes of interest. Intensive longitudinal…

统计方法学 · 统计学 2024-01-17 Mingyan Yu , Zhenke Wu , Margaret Hicken , Michael R. Elliott

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

统计方法学 · 统计学 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

In cohort studies, non-random medication use can pose barriers to estimation of the natural history trend in a mean biomarker value (namely, the association between a predictor of interest and a biomarker outcome that would be observed in…

统计方法学 · 统计学 2021-01-25 Andrew J. Spieker , Joseph A. C. Delaney , Robyn L. McClelland

The individual data collected throughout patient follow-up constitute crucial information for assessing the risk of a clinical event, and eventually for adapting a therapeutic strategy. Joint models and landmark models have been proposed to…

机器学习 · 统计学 2024-07-17 Anthony Devaux , Robin Genuer , Karine Pérès , Cécile Proust-Lima

Longitudinal processes often unfold concurrently where the growth of two or more longitudinal outcomes are associated. Additionally, if the study under investigation is long, the growth curves may exhibit nonconstant change with respect to…

统计方法学 · 统计学 2022-08-23 Jin Liu , Robert A. Perera

Prediction of the future trajectory of a disease is an important challenge for personalized medicine and population health management. However, many complex chronic diseases exhibit large degrees of heterogeneity, and furthermore there is…

机器学习 · 统计学 2016-08-17 Joseph Futoma , Mark Sendak , C. Blake Cameron , Katherine Heller

Individualized treatment rules, cornerstones of precision medicine, inform patient treatment decisions with the goal of optimizing patient outcomes. These rules are generally unknown functions of patients' pre-treatment covariates, meaning…

统计方法学 · 统计学 2025-04-23 Philippe Boileau , Ning Leng , Sandrine Dudoit

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

统计方法学 · 统计学 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

Estimating the effect of a treatment on a given outcome, conditioned on a vector of covariates, is central in many applications. However, learning the impact of a treatment on a continuous temporal response, when the covariates suffer…

机器学习 · 计算机科学 2019-06-11 Guangyi Zhang , Reza Ashrafi , Anne Juuti , Kirsi Pietiläinen , Pekka Marttinen

In recent years, the growing availability of biomedical datasets featuring numerous longitudinal covariates has motivated the development of several multi-step methods for the dynamic prediction of survival outcomes. These methods employ…

统计方法学 · 统计学 2026-01-14 Mirko Signorelli , Sophie Retif
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