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依氟鸟氨酸治疗吉兰-巴雷综合征。

Efgartigimod in the treatment of Guillain-Barré syndrome.

机构信息

Department of Neurology, First Hospital, Shanxi Medical University, No.85, Jiefang South Street, Taiyuan, China.

First Clinical Medical College, Shanxi Medical University, Taiyuan, China.

出版信息

J Neurol. 2024 Jun;271(6):3506-3511. doi: 10.1007/s00415-024-12321-4. Epub 2024 Mar 26.

Abstract

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.

摘要

背景

吉兰-巴雷综合征(GBS)是由免疫球蛋白 G(IgG)自身抗体引起的。依氟鸟氨酸(efgartigimod)是一种人 IgG 抗体 Fc 片段,作为 FcRn 的天然配体,可增加 IgG 的降解,因此可能是 GBS 的一种有前途的治疗药物。

病例介绍

两名患者均表现为感染后急性弛缓性瘫痪。入院时,他们卧床不起。神经传导研究提示周围神经病。怀疑为 GBS,给予依氟鸟氨酸(10mg/kg)两剂,在 5 天内给药。他们的肌肉力量逐渐改善,在初始剂量后 4 周,他们可以独立行走。在首次给药后,患者 1 出现肌肉酸痛,次日清晨缓解。患者 2 在初始剂量后第二天因呼吸衰竭行气管插管,未报告其他不良反应。

讨论

在 GBS 患者中,两剂依氟鸟氨酸(10mg/kg)可有效迅速改善肌肉力量,安全性良好。这些发现提示依氟鸟氨酸在改变 GBS 患者疾病进程方面可能具有潜在作用。

结论

依氟鸟氨酸治疗 GBS 有效且安全。本研究表明依氟鸟氨酸可能成为 GBS 的一种新的治疗选择。应进行精心设计的临床试验。

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