• Christopher Nelke, Christina B Schroeter, Frauke Stascheit, Marc Pawlitzki, Liesa Regner-Nelke, Niklas Huntemann, Ercan Arat, Menekse Öztürk, Nico Melzer, Philipp Mergenthaler, Asmae Gassa, Henning Stetefeld, Michael Schroeter, Benjamin Berger, Andreas Totzeck, Tim Hagenacker, Stefanie Schreiber, Stefan Vielhaber, Hans-Peter Hartung, Andreas Meisel, Heinz Wiendl, Sven G Meuth, and Tobias Ruck.
• Department of Neurology, Heinrich Heine University Düsseldorf, Dusseldorf, Germany.
• J. Neurol. Neurosurg. Psychiatr. 2022 May 1; 93 (5): 548-554.

ObjectiveMyasthenia gravis (MG) is the most common autoimmune disorder affecting the neuromuscular junction. However, evidence shaping treatment decisions, particularly for treatment-refractory cases, is sparse. Both rituximab and eculizumab may be considered as therapeutic options for refractory MG after insufficient symptom control by standard immunosuppressive therapies.MethodsIn this retrospective observational study, we included 57 rituximab-treated and 20 eculizumab-treated patients with MG to compare the efficacy of treatment agents in generalised, therapy-refractory anti-acetylcholine receptor antibody (anti-AChR-ab)-mediated MG with an observation period of 24 months. Change in the quantitative myasthenia gravis (QMG) score was defined as the primary outcome parameter. Differences between groups were determined in an optimal full propensity score matching model.ResultsBoth groups were comparable in terms of clinical and demographic characteristics. Eculizumab was associated with a better outcome compared with rituximab, as measured by the change of the QMG score at 12 and 24 months of treatment. Minimal manifestation of disease was more frequently achieved in eculizumab-treated patients than rituximab-treated patients at 12 and 24 months after baseline. However, the risk of myasthenic crisis (MC) was not ameliorated in either group.InterpretationThis retrospective, observational study provides the first real-world evidence supporting the use of eculizumab for the treatment of refractory, anti-AChR-ab positive MG. Nonetheless, the risk of MC remained high and prompts the need for intensified monitoring and further research effort aimed at this vulnerable patient cohort.© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

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