Efgartigimod in the treatment of Guillain–Barré syndrome

• Published in: Journal of neurology Vol. 271; no. 6; pp. 3506 - 3511
• Main Authors: Zhang, Huiqiu, Ma, Jing, Feng, Yingna, Ma, Hui, Liu, Dan, Pang, Xiaomin, Chang, Xueli, Zhao, Rongjuan, Wang, Juan, Guo, Junhong, Zhang, Wei
• Format: Journal Article
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.06.2024; Springer Nature B.V
• Subjects: Adult; Autoantibodies; Clinical trials; Female; Guillain-Barre syndrome; Guillain-Barre Syndrome - drug therapy; Guillain-Barre Syndrome - physiopathology; Humans; Immobilization; Immunoglobulin G; Male; Medicine; Medicine & Public Health; Middle Aged; Muscle strength; Nerve conduction; Neurology; Neuroradiology; Neurosciences; Original Communication; Paralysis; Patients; Peripheral neuropathy; Treatment Outcome; IgG; Guillain–Barré syndrome; Efgartigimod; FcRn inhibitor
• ISSN: 0340-5354; 1432-1459; 1432-1459
• DOI: 10.1007/s00415-024-12321-4

Background Guillain–Barré Syndrome (GBS) is caused by immunoglobulin G (IgG) autoantibodies. Efgartigimod, a human IgG antibody Fc fragment that acts as a natural ligand for the FcRn, can increase IgG degradation, which thus may be a promising therapeutic drug for GBS. Case presentation The two patients presented with postinfectious and acute flaccid paralysis. On admission, they were bedridden. Nerve conduction studies indicated peripheral neuropathy. GBS was suspected and they are treated with two doses of efgartigimod (10 mg/kg) within 5 days. Their muscle strength improved gradually and 4 weeks after the initial dose, they could walk independently. Following the first dose, Patient 1 complaint of muscle soreness, which subsided the next morning. Patient 2 was intubated due to respiratory failure the day after the initial dose, and did not report other adverse effects. Discussion In GBS patients, two doses of efgartigimod (10 mg/kg) were effective in rapidly improving muscle strength, with a satisfactory safety profile. The findings suggest a potential role for efgartigimod in modifying the disease process in GBS patients. Conclusion Efgartigimod seems effective and safe in the treatment of GBS. This study indicates the potential role of efgartigimod as a novel treatment option for GBS. Well-designed clinical trials should be conducted.