Real-world Outcomes of Efgartigimod in AChR-Positive Generalized Myasthenia Gravis: Response Patterns, Predictors, and Treatment Strategies
Lior Fuchs1, Ifat Vigiser2, Hadar Kolb2, Keren Regev2, Vivian Drory2, Amir Dori3, David Magalashvili3, Tal Caller3, Gilad Kenan4, Yana Mechnik Steen5, Mark Hellmann6, Adi Wilf-Yarkoni6, Tal Friedman Korn7, Adi Vaknin Dembinsky7, Alaa Bsoul8, Shahar Shelly8, Arnon Karni2
1Tel Aviv University, 2Neurology, Tel Aviv Solurasky Medical Center, 3Sheba Medical Center, 4Shamir Medical Center, 5Soroka Medical Center, 6Rabin Medical Center, 7Hadassah Medical Center, 8Rambam Medical Center
Objective:

To evaluate long-term clinical outcomes, treatment patterns, and predictors of efficacy of efgartigimod in acetylcholine receptor antibody–positive generalize myasthenia gravis (gMG).

Background:

Efgartigimod, a neonatal Fc receptor antagonist, lowers pathogenic IgG and has shown efficacy in gMG. Real-world data on long-term outcomes, steroid-sparing, bridging strategies, and predictors of response remain limited. 

Design/Methods:

This multicenter retrospective study included 46 adult patients treated across 7 tertiary centers between April 2022 and March 2025. Data on demographics, clinical features, treatments, and outcomes were collected. The primary endpoints were improvement in MG-ADL scores and achievement of minimal symptom expression (MSE). Secondary endpoints included corticosteroid dose reduction, successful use of efgartigimod as short-term bridging therapy for disease stabilization, and safety outcomes.

Results:

The patients received 197 cycles (mean 4.3 per patient) at a mean interval of 9.8 weeks. 40 (86.9%) patients achieved a ≥2-point improvement in MG-ADL and 20 (43.5%) patients reached MSE after the first cycle, while 24 (52.2%) achieved MSE at any point. Overall 36 (78.3%) patients maintained a ≥2-point improvement in MG-ADL, with response patterns ranging from cyclical benefit to sustained remission, although 14 (35.9%) patients discontinued treatment due to insufficient efficacy. The prednisone dose was reduced in 17 of the 29 (58.6%) treated patients (from a mean of 30.9 to 16.8 mg/day, p=0.001), with a shorter disease duration independently predicting success. Bridging therapy was successful in a subset of 5 patients, particularly among the those receiving azathioprine or mycophenolate (OR=15.0, p=0.040). Adverse events were mild and occurred in 10 (21.7%) patients, only 1 of whom discontinued therapy.

Conclusions:
In real-world practice, efgartigimod was effective and well-tolerated in AChR-positive gMG. Distinct response patterns, a steroid-sparing effect linked to shorter disease duration, and successful bridging therapy in the presence of immunosuppressant support its role in personalized treatment strategies. Prospective validation of its use is warranted.
10.1212/WNL.0000000000212884
Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.