“Introduction
Treatment options available for patients with myasthenia gravis have undergone rapid expansion in recent years with the introduction of new drugs. Our study aims to describe the real-life experience of Efgartigimod use in acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG).
Methods
Efgartigimod was administered intravenous infusion at a dose of 10 mg/kg, once a week for 4 weeks, under the Italian PAA program. Efficacy was primarily assessed by using the MG-ADL and QMG scales. The scales were administered both pre- and post-Efgartigimod for each therapeutic cycle. All patients undergoing treatment are classifiable as responders if they experienced a reduction in MG-ADL ≥ 2 points during the first cycle. Information regarding MG history, concomitant treatments, laboratory data, adverse events, and prednisone use were recorded.
Results
We enrolled 8 patients affected by AChR-seropositive gMG treated with Efgartigimod. A reduction in MG-ADL was observed at the end of the second cycle compared to the baseline with a mean change – 6 points (ranging from 15 to 3). 88% of patients completed at least two cycles, with an improvement of at least 3 points, while 1 patient was interrupted for an adverse event during the second cycle.
Conclusion
The decline in scores obtained testifies to the efficacy of treatment. Considering his tolerability, efficacy, rapid onset of action, and manageability, Efgartigimod could in the future eventually replace the use of traditional and more challenging immunosuppressive therapies in terms of side effects or rescue therapy. Further data and longer observations are needed to confirm our data.”