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Related papers: Explaining temporal trends in annualized relapse r…

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Background: Recent systematic reviews of randomised controlled trials (RCTs) in relapsing multiple sclerosis (RMS) revealed a decrease in placebo annualized relapse rates (ARR) over the past two decades. Furthermore, regression to the mean…

Applications · Statistics 2015-09-03 Christian Röver , Richard Nicholas , Sebastian Straube , Tim Friede

Prognosis on the occurrence of relapses in individuals with Relapsing-Remitting Multiple Sclerosis (RRMS), the most common subtype of Multiple Sclerosis (MS), could support individualized decisions and disease management and could be…

BACKGROUND: An important goal of chronic obstructive pulmonary disease (COPD) treatment is to reduce the frequency of exacerbations. Some observations suggest a decline in exacerbation rates in clinical trials over time. A more systematic…

Applications · Statistics 2019-08-20 Stefan Andreas , Christian Röver , Judith Heinz , Sebastian Straube , Henrik Watz , Tim Friede

Multiple Sclerosis (MS) is a chronic autoimmune and inflammatory neurological disorder characterised by episodes of symptom exacerbation, known as relapses. In this study, we investigate the role of environmental factors in relapse…

Mixed model repeated measures (MMRM) is the most common analysis approach used in clinical trials for Alzheimer's disease and other progressive diseases measured with continuous outcomes measured over time. The model treats time as a…

Multiple sclerosis (MS) is a chronic autoimmune disease that affects the central nervous system. The progression and severity of MS varies by individual, but it is generally a disabling disease. Although medications have been developed to…

Applications · Statistics 2013-03-06 Joyce C. Ho , Joydeep Ghosh , KP Unnikrishnan

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

Methodology · Statistics 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

Multiple Sclerosis is a degenerative condition of the central nervous system that affects nearly 2.5 million of individuals in terms of their physical, cognitive, psychological and social capabilities. Researchers are currently…

Machine Learning · Statistics 2016-12-05 Samuele Fiorini , Andrea Tacchino , Giampaolo Brichetto , Alessandro Verri , Annalisa Barla

Multiple myeloma is managed through sequential lines of therapy over years to decades, with each decision depending on cumulative disease history distributed across dozens to hundreds of heterogeneous clinical documents. Whether LLM-based…

Objectives: To examine the distribution, temporal associations, and age/sex-specific patterns of multiple long-term conditions (MLTCs) in adults with intellectual disability (ID). Study Design: Observational study using longitudinal…

Background and Objectives: Multidrug Resistance (MDR) is a critical global health issue, causing increased hospital stays, healthcare costs, and mortality. This study proposes an interpretable Machine Learning (ML) framework for MDR…

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

Methodology · Statistics 2025-05-26 Huan Wang , Fei Wu , Yeh-Fong Chen

Amyotrophic lateral sclerosis (ALS) is a severe disease with a typical survival of 3-5 years after symptom onset. Current treatments offer only limited life extension, and the variability in patient responses highlights the need for…

Machine Learning · Computer Science 2025-11-05 Guillaume Tejedor , Veronika Peralta , Nicolas Labroche , Patrick Marcel , Hélène Blasco , Hugo Alarcan

Suppression of disability progression is an important goal in the treatment of multiple sclerosis (MS). Randomized clinical trials in MS frequently use the time to the first confirmed disability progression (CDP) on the ordinal Expanded…

Applications · Statistics 2021-11-04 Alexandra Būhler

A higher Mediterranean diet score has been associated with lower likelihood of multiple sclerosis. However, evidence regarding its association with disease activity and progression is limited. Using data from the AusLong Study, we tested…

Treatment effects vary across different patients and estimation of this variability is important for clinical decisions. The aim is to develop a model to estimate the benefit of alternative treatment options for individual patients. Hence,…

The restricted mean survival time (RMST) is the mean survival time in the study population followed up to a specific time point, and is simply the area under the survival curve up to the specific time point. The difference between two RMSTs…

Methodology · Statistics 2025-09-18 Peter Zhang , Brent Logan , Michael Martens

Sepsis is a deadly condition affecting many patients in the hospital. Recent studies have shown that patients diagnosed with sepsis have significant mortality and morbidity, resulting from the body's dysfunctional host response to…

Machine Learning · Computer Science 2022-12-14 Ronald Moore , Rishikesan Kamaleswaran

In confirmatory Phase 3 clinical trials with recruitment over the years, the underlying placebo effect may follow an unknown temporal trend. Taking a clinical trial on Hidradenitis Suppurativa (HS) as an example, fluctuations or…

Methodology · Statistics 2025-06-03 Tianyu Zhan , Yihua Gu

While randomized controlled trials (RCTs) are the gold standard for estimating treatment effects in medical research, there is increasing use of and interest in using real-world data for drug development. One such use case is the…

Methodology · Statistics 2021-10-11 Devin Incerti , Michael T Bretscher , Ray Lin , Chris Harbron
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