“Background and objectives: C5 inhibitors have been approved for treatment of generalized Myasthenia Gravis (gMG). Objective of our study is to describe the clinical response in a real-life setting.

Materials and methods: We included AChR + gMG patients, receiving either eculizumab, ravulizumab or zilucoplan. Primary outcomes for efficacy evaluation were: MG-ADL and QMG. These scores were calculated before treatment initiation and every week for the first month, then every two weeks for the first three months.

Results: We enrolled 13 patients (F/M 11/2), 6 treated with eculizumab and 2 with ravulizumab (administered with intravenous infusion); 5 patients were treated with zilucoplan (administered with subcutaneous injection). Considering the entire cohort, after one month of therapy, we observed a mean reduction of 4.6 points both in MG-ADL (5.7 ⌖ 4.1; -48.2%; ranging from -10 to +1) and QMG scores (11.5 ⌖ 4.6; -26.8%; ranging from -13 to 0) compared to baseline (MG-ADL 10.4 ⌖ 2.7; QMG 16.2 ⌖ 6.3).
MG-ADL decreased by 4.3 ⌖ 3.9 points for eculizumab and 5.0 ⌖ 2.6 points for zilucoplan; the QMG decreased by 4.8 ⌖ 2.8 for eculizumab and 4.4 ⌖ 5.0 for zilucoplan. All patients reported a significant improvement in the quality of daily life, with return to work.

Conclusions: After one month of therapy, C5 inhibitors led to a clinically significant reduction of MG-ADL and QMG scores, proving to be rapid and effective in a real-world setting. (No safety concerns were detected). A longer observation period is needed to confirm our data.”