To describe the baseline characteristics, treatment and safety outcomes of the patients with acetylcholine receptor-positive (AChR+) generalized myasthenia gravis (gMG) treated with eculizumab in our clinic.
Safety and outcomes data on eculizumab for AChR+ gMG in real world clinical practice remain limited.
Patients with refractory AChR+ gMG who received > 6 dose of eculizumab at the Department of Neurology, Istanbul Faculty of Medicine were included in the study. After consent, demographic data, MG Foundation of America (MGFA) classification before eculizumab initiation, MG activities of daily living (MG-ADL) scores, MGFA post intervention status (MGFA-PIS) before and after eculizumab and serious adverse events (SAEs) during the treatment were collected from medical records, retrospectively.
Ten patients were included in the study, six of them were female. Mean age at eculizumab initiation was 44.3 years (range, 29-71). Four had history of thymoma and all patients had thymectomy. Mean follow-up duration after eculizumab was 6 months (range, 2-12 months). After six months of eculizumab treatment, all patients achieved improved status (7/7) and five of patients (5/7) had minimal manifestation status. Mean MG-ADL decreased from 9 to 1.2 at 1 month. All of our patients discontinued maintenance IVIG. No meningococcal infections were reported. Two patients had Covid-19 infection with favorable outcome during eculizumab treatment. One patient had mild arthralgia and two other had upper respiratory tract infection during treatment.
Our data provide additional evidence of the safety and effectiveness of eculizumab for refractory gMG treatment in real-world clinical practice.