Response of refractory residual ocular symptoms to efgartigimod in generalized myasthenia gravis: A real-world case series.

J Neuroimmunol

Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China; Huashan Rare Disease Center, Huashan Hospital, Fudan University, Shanghai, China; National Center for Neurological Diseases, Shanghai, China. Electronic address:

Published: February 2025

Objective: To evaluate the efficacy of efgartigimod (EFG) in treating residual ocular symptoms in myasthenia gravis (MG) patients with acetylcholine receptor antibodies (AChR-Ab).

Methods: Five MG patients with refractory residual ocular symptoms treated with EFG at Huashan Hospital were included. The demographic and clinical information was collected, and MG Activities of Daily Living (MG-ADL) scores and Quantitative Myasthenia Gravis (QMG) scores was elevated weekly during the 8-week follow up period. The time to reach minimal symptom expression (MSE) was also recorded.

Results: After a single cycle of EFG infusion, all five patients showed response in MG-ADL (≥2 points reduction), and three patients in QMG score (≥3 points reduction). The mean ± SD MG-ADL scores decreased significantly from 5.0 ± 1.0 at baseline to 1.8 ± 1.1 at weak 4 (p = 0.0027) and 1.8 ± 0.5 at weak 6 (p = 0.0027). The mean ± SD QMG score decreased from 5.8 ± 0.5 at baseline to 2.4 ± 1.7 at week 4 (p = 0.1357) and 1.0 ± 0.7 at week 6 (p = 0.0076). The proportions of patients reaching MSE at week 4, 6 and 8 were 20 % (1/5), 20 % (1/5), and 60 % (3/5), respectively.

Conclusions: AChR-Ab+ MG patients with residual and refractory ocular symptoms could benefit from EFG treatment, while the duration of efficacy varied in individuals.

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http://dx.doi.org/10.1016/j.jneuroim.2025.578558DOI Listing

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