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Huntington disease (HD) is a fatal autosomal dominant neurocognitive disorder that causes cognitive disturbances, neuropsychiatric symptoms, and impaired motor abilities (e.g., gait, speech, voice). Due to its progressive nature, HD…

Audio and Speech Processing · Electrical Eng. & Systems 2020-10-20 Amrit Romana , John Bandon , Noelle Carlozzi , Angela Roberts , Emily Mower Provost

Joint models are used in ageing studies to investigate the association between longitudinal markers and a time-to-event, and have been extended to multiple markers and/or competing risks. The competing risk of death must be considered in…

Huntington disease (HD) is a neurodegenerative disease with progressively worsening symptoms. Accurately modeling time to HD diagnosis is essential for clinical trial design. Langbehn's model, the CAG-Age Product (CAP) model, the Prognostic…

Background: Huntington's disease (HD) is a rare, genetically determined brain disorder that limits the life of the patient, although early prognosis of HD can substantially improve the patient's quality of life. Current HD prognosis methods…

Signal Processing · Electrical Eng. & Systems 2023-02-09 Sucheer Maddury

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…

Machine Learning · Statistics 2024-07-17 Anthony Devaux , Robin Genuer , Karine Pérès , Cécile Proust-Lima

Dementia currently affects about 50 million people worldwide, and this number is rising. Since there is still no cure, the primary focus remains on preventing modifiable risk factors such as cardiovascular factors. It is now recognized that…

Methodology · Statistics 2024-08-14 Léonie Courcoul , Catherine Helmer , Antoine Barbieri , Hélène Jacqmin-Gadda

This article considers the joint modeling of longitudinal covariates and partly-interval censored time-to-event data. Longitudinal time-varying covariates play a crucial role in obtaining accurate clinically relevant predictions using a…

Methodology · Statistics 2024-12-05 Annabel Webb , Nan Zou , Serigne Lo , Jun Ma

Longitudinal biomarker data and health outcomes are routinely collected in many studies to assess how biomarker trajectories predict health outcomes. Existing methods primarily focus on mean biomarker profiles, treating variability as a…

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

Huntington's disease (HD) is an inherited neurodegenerative disorder caused by an expanded CAG repeat in the coding sequence of the huntingtin protein. Initially, it predominantly affects medium-sized spiny neurons (MSSNs) of the corpus…

Within-individual variability of health indicators measured over time is becoming commonly used to inform about disease progression. Simple summary statistics (e.g. the standard deviation for each individual) are often used but they are not…

Non-terminal events can represent a meaningful change in a patient's life. Thus, better understanding and predicting their occurrence can bring valuable information to individuals. In a context where longitudinal markers could inform these…

Methodology · Statistics 2025-01-16 Juliette Ortholand , Stanley Durrleman , Sophie Tezenas du Montcel

Often in Phase 3 clinical trials measuring a long-term time-to-event endpoint, such as overall survival or progression-free survival, investigators also collect repeated measures on biomarkers which may be predictive of the primary…

Methodology · Statistics 2022-11-30 Abigail J. Burdon , Lisa V. Hampson , Christopher Jennison

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

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

Across health applications, researchers model outcomes as a function of time to an event, but the event time is right-censored for participants who exit the study or otherwise do not experience the event during follow-up. When censoring…

Methodology · Statistics 2025-11-21 Jesus E. Vazquez , Yanyuan Ma , Karen Marder , Tanya P. Garcia

Motivated by recent findings that within-subject (WS) visit-to-visit variabilities of longitudinal biomarkers can be strong risk factors for health outcomes, this paper introduces and examines a new joint model of a longitudinal biomarker…

While right-censored time-to-event outcomes have been studied for decades, handling time-to-event covariates, also known as right-censored covariates, is now of growing interest. So far, the literature has treated right-censored covariates…

Methodology · Statistics 2024-09-10 Jesus E. Vazquez , Marissa C. Ashner , Yanyuan Ma , Karen Marder , Tanya P. Garcia

Disease progression modeling provides a robust framework to identify long-term disease trajectories from short-term biomarker data. It is a valuable tool to gain a deeper understanding of diseases with a long disease trajectory, such as…

To select outcomes for clinical trials testing experimental therapies for Huntington disease, a fatal neurodegenerative disorder, analysts model how potential outcomes change over time. Yet, subjects with Huntington disease are often…

Methodology · Statistics 2023-03-06 Kyle F. Grosser , Sarah C. Lotspeich , Tanya P. Garcia
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