Vol.:(0123456789) 1 3 Journal of Neurology https://doi.org/10.1007/s00415-019-09191-6 ORIGINAL COMMUNICATION High efcacy of rituximab for myasthenia gravis: a comprehensive nationwide study in Austria Raf Topakian 1  · Fritz Zimprich 2  · Stephan Iglseder 3  · Norbert Embacher 4  · Michael Guger 5  · Karl Stieglbauer 6  · Dieter Langenscheidt 7  · Jakob Rath 2  · Stefan Quasthof 8  · Philipp Simschitz 9  · Julia Wanschitz 10  · David Windisch 11  · Petra Müller 1  · Dierk Oel 1  · Günther Schustereder 1  · Stefan Einsiedler 1  · Christian Eggers 3  · Wolfgang Löscher 10 Received: 18 November 2018 / Revised: 1 January 2019 / Accepted: 6 January 2019 © Springer-Verlag GmbH Germany, part of Springer Nature 2019 Abstract Background Most patients with myasthenia gravis (MG) need long-term immunosuppressive therapy. However, conventional agents may have intolerable side efects, take too long or fail to achieve disease control. Rituximab (RTX) has emerged as an of-label treatment for refractory MG, but data on its use are still sparse. Methods We conducted a retrospective nationwide study contacting all Austrian neurologists to provide anonymized data of all adult MG patients treated with RTX and minimum follow-up of 3 months. The Myasthenia Gravis Foundation of America Postintervention Status scale was used to assess outcomes. Results 34 (60.7%) of a total of 56 patients were women. Median (IQR) age at diagnosis of MG and start of RTX were 41.5 (24.3; 65.8) and 47.5 (33; 71) years, respectively. Antibodies (ab) against acetylcholine receptor (AchR) and muscle- specifc tyrosine kinase (MuSK) were present in 69.6% and 25% of patients, respectively (seronegative: 5.4%). Before RTX, 47 (83.9%) patients had had plasma exchange, immune adsorption or immunoglobulins. Three months after RTX, 14 of 53 (26.4%) patients were in remission. At last follow-up after a median of 20 (10; 53) months, remission was present in 42.9% of patients and another 25% had minimal manifestations. Remission was more frequent in patients with MuSK ab vs. those with AchR ab (71.4% vs. 35.9%, p = 0.022). RTX was safe. The presence of MuSK ab independently predicted remission after RTX. Conclusion In this retrospective study on RTX for MG, the largest to date, RTX appeared safe, efcacious and fast acting. Beneft from RTX was greatest in MuSK ab + MG. Keywords Myasthenia gravis · Rituximab · Efcacy · Anti-MuSK · Outcome Electronic supplementary material The online version of this article (https://doi.org/10.1007/s00415-019-09191-6) contains supplementary material, which is available to authorized users. * Raf Topakian raf.topakian@hotmail.com 1 Department of Neurology, Academic Teaching Hospital Wels-Grieskirchen, Grieskirchner Str. 42, 4600 Wels, Austria 2 Department of Neurology, Medical University of Vienna, Vienna, Austria 3 Department of Neurology, Krankenhaus Barmherzige Brüder, Linz, Austria 4 Department of Neurology, University Clinic St. Pölten, St. Pölten, Austria 5 Clinic for Neurology 2, Med Campus III, Kepler University Clinic, Linz, Austria 6 Neurologist in Private Practice, Linz, Austria 7 Department of Neurology, Landeskrankenhaus Rankweil, Rankweil, Austria 8 Department of Neurology, Graz Medical University, Graz, Austria 9 Department of Neurology, Klinikum Klagenfurt, Klagenfurt, Austria 10 Department of Neurology, Medical University Innsbruck, Innsbruck, Austria 11 Department of Neurology, Landeskrankenhaus Bruck, Bruck, Austria