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Abstract Details

Real-world Outcomes of Efgartigimod in AChR-Positive Generalized Myasthenia Gravis: Response Patterns, Predictors, and Treatment Strategies
Neuromuscular and Clinical Neurophysiology (EMG)
S14 - Updates on Myasthenia Gravis (1:12 PM-1:24 PM)
002

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

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. 

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.

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.

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.
Authors/Disclosures
Lior Fuchs, MD
PRESENTER
Dr. Fuchs has nothing to disclose.
Ifat Vigiser (Tel Aviv Medical Center) Ifat Vigiser has nothing to disclose.
Hadar Kolb Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Hadar Kolb has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for MAPI pharma. Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neopharm Scientific. Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Marck. The institution of Hadar Kolb has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MAPI pharma. Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Medison pharma. Hadar Kolb has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Marck.
Keren Regev, MD Dr. Regev has nothing to disclose.
Vivian E. Drory, MD (Tel-Aviv Medical Center) Prof. Drory has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Teva. Prof. Drory has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Prof. Drory has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Prof. Drory has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Prof. Drory has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Medison Pharma. The institution of Prof. Drory has received research support from Neurosense Therapeutics. The institution of Prof. Drory has received research support from AB Science. The institution of Prof. Drory has received research support from Biogen. The institution of Prof. Drory has received research support from Neuralight.
Amir Dori, MD, PhD (Sheba Medical Center) Dr. Dori has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TEVA. Dr. Dori has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda . Dr. Dori has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Dori has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medison Pharma. Dr. Dori has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Medison Pharma. The institution of Dr. Dori has received research support from Biogen. The institution of Dr. Dori has received research support from Pfizer. The institution of Dr. Dori has received research support from Alnylam therapeutics. Dr. Dori has received intellectual property interests from a discovery or technology relating to health care.
David Magalashvili, MD No disclosure on file
Tal Caller, PhD Dr. Caller has nothing to disclose.
Gilad Kenan, MD Dr. Kenan has nothing to disclose.
Yana Mechnik Steen, MD Dr. Mechnik Steen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medison. Dr. Mechnik Steen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Mechnik Steen has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Medison .
Mark Hellman Mark Hellman has nothing to disclose.
Adi Wilf-Yarkoni, MD Dr. Wilf-Yarkoni has nothing to disclose.
Tal Friedman Korn, MD Dr. Friedman Korn has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche.
Adi Vaknin-Dembinsky, MD Dr. Vaknin-Dembinsky has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medison Pharma. Dr. Vaknin-Dembinsky has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for neopharm group. Dr. Vaknin-Dembinsky has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for medisone . Dr. Vaknin-Dembinsky has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for alexion.
Alaa Bsoul, MD Dr. Bsoul has nothing to disclose.
Shahar Shelly, MD (Rambam Medical Center) Dr. Shelly has or had stock in Remepy.
Arnon Karni, MD, PhD (Tel Aviv Sourasky medical center) Prof. Karni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neopharm. Prof. Karni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BMS. Prof. Karni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Prof. Karni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Prof. Karni has received research support from Medison. The institution of Prof. Karni has received research support from BMS. The institution of Prof. Karni has received research support from Novartis.