Rituximab as a sole steroid-sparing agent in generalized myasthenia gravis: Long-term outcomes.

Autor: Kefalopoulou ZM; Department of Neurology, University Hospital of Patras, 26504, Patras, Rio, Greece. zkefalopoulou@gmail.com.; Neuromuscular Centre, University Hospital of Patras, 26504, Patras, Rio, Greece. zkefalopoulou@gmail.com., Veltsista D; Department of Neurology, University Hospital of Patras, 26504, Patras, Rio, Greece.; Neuromuscular Centre, University Hospital of Patras, 26504, Patras, Rio, Greece., Germeni A; Department of Neurology, University Hospital of Patras, 26504, Patras, Rio, Greece., Lykouras D; Department of Respiratory Medicine, University Hospital of Patras, 26504, Patras, Rio, Greece., Tsiamaki E; Department of Neurology, University Hospital of Patras, 26504, Patras, Rio, Greece., Chroni E; Department of Neurology, University Hospital of Patras, 26504, Patras, Rio, Greece.; Neuromuscular Centre, University Hospital of Patras, 26504, Patras, Rio, Greece.
Jazyk: angličtina
Zdroj: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology [Neurol Sci] 2024 Mar; Vol. 45 (3), pp. 1233-1242. Date of Electronic Publication: 2023 Oct 13.
DOI: 10.1007/s10072-023-07082-3
Abstrakt: Background: Rituximab, a B-cell depleting monoclonal antibody, represents an option for the treatment of refractory myasthenia gravis (MG). Its use is more established in muscle-specific tyrosine kinase positive (MuSK +) patients, while its role in managing acetylcholine receptor positive (AChR +), or double seronegative (DSN) patients, remains less clear. This study evaluates the long-term effectiveness and safety of rituximab in MG of various serotypes.
Methods: We conducted an open-label study of MG patients receiving rituximab. Adults with generalized refractory MG, either anti-AChR + or DSN, and anti-MuSK + , refractory or not, who had follow-up > 12 months were selected. Change in quantitative myasthenia gravis (QMG) score at last follow-up, compared with baseline was a primary outcome, as well as factors affecting response to treatment. Secondary outcomes included, long-term safety, the steroid-sparing effect and relapse rates post-rituximab.
Results: Thirty patients (16 anti-AChR + , 6 anti-MuSK + , 8 DSN) followed for a mean of 33.3 months were included. Mean scores pre-rituximab compared to last follow-up significantly decreased (p < 0.001), from 11 ± 4.1 to 4.3 ± 3.8, and from 1.9 to 0.3 regarding QMG and relapse rate per patient/year, respectively, while in 93.1% a daily steroid dose ≤ 10 mg was achieved. Antibody status was the only factor independently influencing several endpoints. Throughout the study period no crises or deaths occurred.
Conclusion: The present study supports that rituximab is an effective and well tolerated treatment for refractory anti-AChR + and DSN MG patients, while anti-MuSK + remains the group experiencing the greater benefits.
(© 2023. Fondazione Società Italiana di Neurologia.)
Databáze: MEDLINE
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