Bridging the Heterogeneity of Myasthenia Gravis Scores as a Foundational Step Towards the Construction of a Digital Twin

<b>Background/Objectives</b>: Myasthenia gravis (MG) is a rare autoimmune neuromuscular disease. Clinical trials with rigorously collected data provide valuable opportunities for mathematical modeling of patient outcomes over time. However, for rare diseases such as MG, combining data across multiple trials presents challenges due to heterogeneity in outcome measures. This study aims to address these challenges by investigating relationships among commonly used MG outcome measures to support the development of a standardized "Myasthenia Gravis Portrait." <b>Methods</b>: We integrated three primary data types from multiple clinical studies: (i) laboratory and medication data, (ii) Electronic Health Record (EHR) data (e.g., age, sex, years since diagnosis, BMI), and (iii) disease severity scores. We examined the relationships among several MG-specific scoring systems, including Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), MG Composite (MG-CE), and MG Quality of Life-15 (MGQOL-15), to evaluate consistency and comparability across studies. <b>Results</b>: Preliminary analyses revealed variable correlations among the different scoring systems, indicating that, while some measures capture overlapping aspects of disease progression, others reflect distinct patient- or clinician-centered perspectives. These findings highlight the need for a harmonized framework that captures both functional and clinical dimensions of MG severity. <b>Conclusions</b>: The proposed "Myasthenia Gravis Portrait" provides a standardized approach for representing patient outcomes across diverse clinical datasets. This framework will facilitate the creation of virtual populations of digital twins, enabling a machine-learning-based modeling of MG progression and prediction of individualized disease trajectories.

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Publication Details

Journal
Biomedicines
Published
2025-11-28
DOI
https://doi.org/10.3390/biomedicines13122920
Primary Topic
Myasthenia Gravis and Thymoma
Type
article
Field-Weighted Citation Impact
0.00

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article

Bridging the Heterogeneity of Myasthenia Gravis Scores as a Foundational Step Towards the Construction of a Digital Twin

Quentin Lesport, Henry J. Kaminski, Marc Garbey
Biomedicines
Myasthenia Gravis and Thymoma
article

Bridging the Heterogeneity of Myasthenia Gravis Scores as a Foundational Step Towards the Construction of a Digital Twin

Quentin Lesport, Henry J. Kaminski, Marc Garbey
article en

Abstract

<b>Background/Objectives</b>: Myasthenia gravis (MG) is a rare autoimmune neuromuscular disease. Clinical trials with rigorously collected data provide valuable opportunities for mathematical modeling of patient outcomes over time. However, for rare diseases such as MG, combining data across multiple trials presents challenges due to heterogeneity in outcome measures. This study aims to address these challenges by investigating relationships among commonly used MG outcome measures to support the development of a standardized "Myasthenia Gravis Portrait." <b>Methods</b>: We integrated three primary data types from multiple clinical studies: (i) laboratory and medication data, (ii) Electronic Health Record (EHR) data (e.g., age, sex, years since diagnosis, BMI), and (iii) disease severity scores. We examined the relationships among several MG-specific scoring systems, including Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), MG Composite (MG-CE), and MG Quality of Life-15 (MGQOL-15), to evaluate consistency and comparability across studies. <b>Results</b>: Preliminary analyses revealed variable correlations among the different scoring systems, indicating that, while some measures capture overlapping aspects of disease progression, others reflect distinct patient- or clinician-centered perspectives. These findings highlight the need for a harmonized framework that captures both functional and clinical dimensions of MG severity. <b>Conclusions</b>: The proposed "Myasthenia Gravis Portrait" provides a standardized approach for representing patient outcomes across diverse clinical datasets. This framework will facilitate the creation of virtual populations of digital twins, enabling a machine-learning-based modeling of MG progression and prediction of individualized disease trajectories.

BiomedicinesVol. 13(12)
Centre National de la Recherche Scientifique (FR), George Washington University (US), Constitutional Court of Korea (KR), La Rochelle Université (FR)
National Science Foundation, Rare Diseases Clinical Research Network, National Institutes of Health, National Institute of Neurological Disorders and Stroke, National Center for Advancing Translational Sciences
Openalex Percentile: Top 48%
Myasthenia Gravis and Thymoma
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