Immunosuppressive treatment is effective in most Myasthenia gravis patients, but 10-15% of patients are considered refractory due to inadequate response or intolerance to the therapy.
Eculizumab a humanized monoclonal antibody directet against C5 complement protein has been approved in Italy for the treatment of Ab-AchR generalized refractory MG in October 2022.
A retrospective data analysis was conducted from patients with refractory generalized MG treated between November 2022 and May 2024.
48 treatment-refractory AchR generalized MG patients were identified and 21 patients were treated with eculizumab. 1 patient dropped after ten weeks of treatment due to the risk of impeding myasthenic crisis, 14 patients were identified at the last follow up visit with 12 months or more of therapy.
Clinical assessement was performed monthly throught specific scales (MG ADL – QMG – MGFA – PIS) and steroid sparing effect was recorded.
Patients treated with eculizumab showed improvement in all clinical outcome measures, from the first month. Clinically meaningful reduction in total MG-ADL and QMG scores was achieved and were maintained throughout the first, third, sixth and twelfth month along with concomitant reduction of immunosuppressive treatment.
Intravenous immunoglobulin treatment was discontinued in all patients and the total daily dose of prednisone was significantly reduced.
During the twelve months of treatment, eculizumab improved all clinical outcome measures, leading to remarkable immunosuppressant-sparing benefit.