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

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

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.

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.

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