Effectiveness and safety of ravulizumab in anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis: a systematic review and meta-analysis.

OBJECTIVE

To evaluate the effectiveness and safety of ravulizumab in patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR-Ab+ gMG).

METHODS

A systematic search was conducted in PubMed, Scopus, Web of Science, and Cochrane CENTRAL up to May 3, 2025. Eligible studies included clinical trials and observational studies assessing ravulizumab in MG. Two independent reviewers performed study selection and data extraction. Outcomes included the Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Activities of Daily Living (MG-ADL) score, Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL15r), corticosteroid dose, clinically meaningful improvement (≥ 2-point reduction in MG-ADL score), and adverse events (AEs). We used random-effects models to calculate pooled mean differences (MD) and proportions with 95% confidence intervals (CIs), and heterogeneity was assessed via I2 statistics.

RESULTS

Six studies involving 321 patients (mean age: 56.27 ± 18.78 years) were included. Ravulizumab significantly improved QMG (MD: -2.91 points, 95% CI: [-4.50 to -1.32]), MG-ADL (MD: -2.64 points, 95% CI: [-3.75 to -1.54]), and MG-QoL15r (MD: -4.61 points, 95% CI: [-8.44 to -0.77]) scores. Clinically meaningful improvement was observed in 69% of patients (95% CI: [42% to 96%]). The corticosteroid dose was reduced by 5.98 mg/day (95% CI: [-7.71 to -4.25]). AEs were reported in 80.52% of patients; 28.5% experienced serious AEs.

CONCLUSIONS

Ravulizumab significantly improved QMG and MG-ADL scores and quality of life in AChR-Ab+ gMG patients, with a minimal steroid-sparing effect. Given the generally favorable safety profile, these findings support ravulizumab as a treatment option for patients with AChR-Ab+ gMG.