[Pubmed] Bridging the Heterogeneity of Myasthenia Gravis Scores as a Foundational Step Towards the Construction of a Dig

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Biomedicines. 2025 Nov 28;13(12):2920. doi: 10.3390/biomedicines13122920.

ABSTRACT

Background/Objectives: 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." Methods: 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. Results: 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. Conclusions: 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.

PMID:41462932 | DOI:10.3390/biomedicines13122920


Source: https://pubmed.ncbi.nlm.nih.gov/4146293 ... 22+67771e2