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. 2025 Feb;46(2):951-959.
doi: 10.1007/s10072-024-07861-6. Epub 2024 Nov 4.

Eculizumab in refractory myasthenia gravis: a real-world single-center experience

Dario Ricciardi  1   2 , Carmen Erra  3 , Francesco Tuccillo  1 , Bernardo Maria De Martino  1 , Alessandra Fasolino  1 , Francesco Habetswallner  1
Affiliations

Eculizumab in refractory myasthenia gravis: a real-world single-center experience

Dario Ricciardi et al. Neurol Sci. 2025 Feb.

Abstract

Introduction: Immunosuppressive treatment is effective in most Myasthenia gravis patients, but 10-15% of patients areconsidered refractory due to inadequate response or intolerance to therapy. Eculizumab, a humanized monoclonalantibody directed against C5 complement protein, was approved in Italy to treat Ab-AchR generalized refractoryMG (rMG) in October 2022.

Aim: We aim to describe a real-world Italian experience in a population of refractory myasthenia gravis patients with oneyear follow up.

Methods: A retrospective data analysis was conducted on patients with refractory generalized MG treated with eculizumabbetween November 2022 and May 2024. Clinical assessment through specific scales (MG ADL - QMG - MGFA -PIS), rescue, and background therapy was recorded after one, three, six, and twelve months.

Results: 21 rMG patients were treated with eculizumab with a medium follow up of 10.4 months and 14 patients had at leastone year follow up. A clinically meaningful reduction in total MG-ADL and QMG scores was achieved in the firstmonth. It was maintained throughout the first, third, sixth, and twelfth month along with concomitant reduction ofimmunosuppressive treatments. A drastic reduction of myasthenic exacerbations and crisis was observed duringfollow up and intravenous immunoglobulin treatment was discontinued in all patients except one. The total dailydose of prednisone was significantly reduced.

Discussion: This single-center real-world study confirmes safety and effectiveness of eculizumab. Eculizumab improved rapidlyall clinical outcome measures, leading to discontinuation of intravenous immunoglobulin treatment and remarkable immunosuppressant-sparing benefits.

Keywords: Complement inhibitor; Eculizumab; Myasthenia gravis; Real world evidence.

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Conflict of interest statement

Declarations. Ethical approval: This study was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice. Ethical approval was waived by the local Ethics Committee and all the procedures being performed were part of the routine care. All patients provided written informed consent. We confirm that we have read the Journal’s position on issues involved in ethical publication and affirm that this report is consistent with those guidelines. Conflict of interest: Dario Ricciardi has received travel funding and speaker honoraria from Alexion, UCB, Argenx. This author has no potential conflict of interest to disclose. Carmen Erra has received travel funding and speaker honoraria from Alexion, UCB, Argenx, Anlylam, CLS Bhering. This author has no potential conflict of interest to disclose. Francesco Tuccillo has received travel funding and speaker honoraria from Alexion, UCB, Argenx. This author has no potential conflict of interest to disclose. Bernardo Maria De Martino has received travel funding and speaker honoraria from Alexion, UCB, Argenx. This author has no potential conflict of interest to disclose. Alessandra Fasolino has no financial disclosure. This author has no potential conflict of interest to disclose. Francesco Habetswallner has received travel funding and speaker honoraria from Alexion, UCB, Argenx. This author has no potential conflict of interest to disclose.

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