Leiden University Scholarly Publications

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Remijn-Nelissen; L. (2026)

Symptomatic treatment of myasthenia gravis and its role in the era of emerging immunomodulatory treatments

Doctoral Thesis
This thesis provides an overview of the therapeutic landscape for myasthenia gravis (MG), focusing on symptomatic therapies and immunomodulatory treatments.

Part I evaluated symptomatic treatment. A literature review highlighted that pyridostigmine remains the cornerstone of symptomatic therapy despite limited high-quality evidence, while the roles of amifampridine, sympathomimetics, and emerging therapies remain uncertain. A cross-sectional study of 410 patients showed that pyridostigmine was widely used and moderately effective but associated with frequent side effects, with 91% of current users reporting at least one adverse effect and 26% discontinuing treatment because of side effects. The randomized, placebo-controlled IMPACT-MG trial demonstrated that pyridostigmine significantly improved disease severity, quality of life, treatment satisfaction, and was highly cost-effective. In contrast, adding amifampridine to pyridostigmine did not improve clinical...Show more This thesis provides an overview of the therapeutic landscape for myasthenia gravis (MG), focusing on symptomatic therapies and immunomodulatory treatments.

Part I evaluated symptomatic treatment. A literature review highlighted that pyridostigmine remains the cornerstone of symptomatic therapy despite limited high-quality evidence, while the roles of amifampridine, sympathomimetics, and emerging therapies remain uncertain. A cross-sectional study of 410 patients showed that pyridostigmine was widely used and moderately effective but associated with frequent side effects, with 91% of current users reporting at least one adverse effect and 26% discontinuing treatment because of side effects. The randomized, placebo-controlled IMPACT-MG trial demonstrated that pyridostigmine significantly improved disease severity, quality of life, treatment satisfaction, and was highly cost-effective. In contrast, adding amifampridine to pyridostigmine did not improve clinical outcomes but increased adverse events.

Part II examined immunomodulatory therapy. Registry data demonstrated persistent long-term use of pyridostigmine and corticosteroids, with substantial use of IVIg and plasma exchange during the disease course. Finally, a retrospective cohort of patients with refractory MG showed that efgartigimod resulted in favorable clinical outcomes in over half of patients and enabled reduction of other MG treatments in 80%, while individualized dosing intervals were commonly adopted in clinical practice.
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Myasthenia gravis
Symptomatic treatment
Pyridostigmine
Amifampridine
Immunomodulatory treatment
Auto-immune diseases
Neuromuscular diseases
All authors
Remijn-Nelissen; L.
Supervisor
Verschuuren, J.J.G.M.; Gelder, T. van
Co-supervisor
Tannemaat; M.R.
Committee
Maarel, S.M. van der; Guchelaar, H.J.; Barnett-Tapia, C.; Dekker, A.M.
Qualification
Doctor (dr.)
Awarding Institution
Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
Date
2026-09-03
ISBN (print)
9789465345413