Nelke, Christopher, Schroeter, Christina B., Stascheit, Frauke ORCID: 0000-0001-5306-7880, Pawlitzki, Marc, Regner-Nelke, Liesa, Huntemann, Niklas, Arat, Ercan, Oeztuerk, Menekse, Melzer, Nico ORCID: 0000-0002-2420-701X, Mergenthaler, Philipp, Gassa, Asmae, Stetefeld, Henning, Schroeter, Michael, Berger, Benjamin ORCID: 0000-0003-2654-2898, Totzeck, Andreas, Hagenacker, Tim, Schreiber, Stefanie ORCID: 0000-0003-4439-4374, Vielhaber, Stefan, Hartung, Hans-Peter, Meisel, Andreas ORCID: 0000-0001-7233-5342, Wiendl, Heinz, Meuth, Sven G. and Ruck, Tobias (2022). Eculizumab versus rituximab in generalised myasthenia gravis. J. Neurol. Neurosurg. Psychiatry, 93 (5). S. 548 - 555. LONDON: BMJ PUBLISHING GROUP. ISSN 1468-330X

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Abstract

Objective Myasthenia 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. Methods In 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. Results Both 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. Interpretation This 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.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Nelke, ChristopherUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schroeter, Christina B.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stascheit, FraukeUNSPECIFIEDorcid.org/0000-0001-5306-7880UNSPECIFIED
Pawlitzki, MarcUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Regner-Nelke, LiesaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Huntemann, NiklasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Arat, ErcanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Oeztuerk, MenekseUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Melzer, NicoUNSPECIFIEDorcid.org/0000-0002-2420-701XUNSPECIFIED
Mergenthaler, PhilippUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gassa, AsmaeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stetefeld, HenningUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schroeter, MichaelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Berger, BenjaminUNSPECIFIEDorcid.org/0000-0003-2654-2898UNSPECIFIED
Totzeck, AndreasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hagenacker, TimUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schreiber, StefanieUNSPECIFIEDorcid.org/0000-0003-4439-4374UNSPECIFIED
Vielhaber, StefanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hartung, Hans-PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Meisel, AndreasUNSPECIFIEDorcid.org/0000-0001-7233-5342UNSPECIFIED
Wiendl, HeinzUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Meuth, Sven G.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ruck, TobiasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-692058
DOI: 10.1136/jnnp-2021-328665
Journal or Publication Title: J. Neurol. Neurosurg. Psychiatry
Volume: 93
Number: 5
Page Range: S. 548 - 555
Date: 2022
Publisher: BMJ PUBLISHING GROUP
Place of Publication: LONDON
ISSN: 1468-330X
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
COMPLEMENT; EFFICACY; SAFETYMultiple languages
Clinical Neurology; Psychiatry; SurgeryMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/69205

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