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Rituximab in AChR and MusK Myasthenia Gravis: long-term follow-up with different therapeutic protocols
C Erra1, D Ricciardi1, G Abbadessa2
1Clinical Neurophysiopathology Unit, AORN Cardarelli, Naples, Italy.
Journal of the Neurological Sciences
|July 23, 2026
Summary
Rituximab shows long-term efficacy and safety in generalized Myasthenia Gravis (gMG). Patients with anti-Musk antibodies had higher success rates and faster steroid reduction compared to anti-AChR antibody patients.
Area of Science:
- Neurology
- Immunology
- Pharmacology
Background:
- Generalized Myasthenia Gravis (gMG) is a rare autoimmune disorder affecting neuromuscular transmission.
- Refractory gMG or inadequate disease control necessitates exploring novel therapeutic strategies.
- Rituximab, an anti-CD20 monoclonal antibody, has emerged as a potential treatment for gMG.
Purpose of the Study:
- To evaluate the long-term efficacy and safety of rituximab in a real-world cohort of generalized Myasthenia Gravis patients.
- To compare treatment outcomes between patients with anti-acetylcholine receptor (AChR) and anti-MuSK antibodies.
- To assess the impact of rituximab on steroid dependency and daily living activities.
Main Methods:
- A single-center retrospective study of 44 generalized Myasthenia Gravis patients treated with rituximab from January 2009 to May 2025.
- Patients were stratified into anti-AChR (n=22) and anti-MusK (n=22) antibody subgroups.
- Primary endpoint: Myasthenia Gravis Foundation of America post-intervention status success; Secondary endpoints: changes in activities of daily living, steroid dose reduction/discontinuation.
Main Results:
- At 60 months, 33.3% of AChR and 66.7% of MusK patients achieved success.
- Steroid dose reduction to ≤10 mg/day was achieved by 76.2% (AChR) and 90.9% (MusK) of patients.
- Stable steroid discontinuation was possible in 42.9% (AChR) and 68.2% (MusK) of patients.
- Anti-MusK serotype was the sole significant predictor of treatment success; no serious adverse events were reported.
Conclusions:
- Rituximab demonstrates sustained efficacy and a favorable safety profile for long-term management of generalized Myasthenia Gravis.
- Patients with anti-MusK antibodies exhibit superior treatment response and steroid-sparing effects compared to anti-AChR antibody patients.
- Real-world data supports rituximab as a valuable therapeutic option for refractory generalized Myasthenia Gravis.
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