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接受艾加莫德治疗的抗谷氨酸脱羧酶相关自身免疫性脑炎患者的病情缓解

Resolution of anti-GAD-associated autoimmune encephalitis in patients treated with efgartigimod.

作者信息

Chen Min, Bi Zhuajin, Kang Wenzhong, Liu Ruihan, Hongbo Liu, Jiang Yan, Wang Qun

机构信息

Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China.

Xiangya School of Medicine, Central South University, Changsha, China.

出版信息

Front Neurol. 2025 Apr 28;16:1550023. doi: 10.3389/fneur.2025.1550023. eCollection 2025.

DOI:10.3389/fneur.2025.1550023
PMID:40356636
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12066553/
Abstract

INTRODUCTION

Temporal lobe epilepsy (TLE) is a significant clinical phenotype of anti-glutamic acid decarboxylase (GAD)-associated disease, which is characterized by disturbances in GABAergic inhibitory neurotransmission. Efgartigimod, a neonatal crystallizable fragment receptor antagonist, controls the trafficking and recycling of pathogenic anti-GAD immunoglobulin G, showing promise as a therapeutic target.

METHODS

We present a case report involving the treatment of three patients affected by GAD-seropositive autoimmune encephalitis with efgartigimod. The patients' overall disability was assessed using the modified Rankin scale.

RESULTS

After 4 weeks of efgartigimod treatment, the patients demonstrated substantial improvements, including no dementia or behavioral abnormalities and well-controlled seizures.

DISCUSSION

Our findings suggest that efgartigimod is a potential candidate drug for the treatment of anti-GADassociated autoimmune encephalitis, particularly in patients presenting with TLE.

摘要

引言

颞叶癫痫(TLE)是抗谷氨酸脱羧酶(GAD)相关疾病的一种重要临床表型,其特征是γ-氨基丁酸(GABA)能抑制性神经传递紊乱。艾加莫德是一种新生儿可结晶片段受体拮抗剂,可控制致病性抗GAD免疫球蛋白G的转运和再循环,显示出作为治疗靶点的潜力。

方法

我们报告一例使用艾加莫德治疗3例GAD血清阳性自身免疫性脑炎患者的病例。使用改良Rankin量表评估患者的整体残疾情况。

结果

艾加莫德治疗4周后,患者有显著改善,包括无痴呆或行为异常,癫痫得到良好控制。

讨论

我们的研究结果表明,艾加莫德是治疗抗GAD相关自身免疫性脑炎的潜在候选药物,尤其是对伴有TLE的患者。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fbe5/12066553/23d057fa8ea3/fneur-16-1550023-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fbe5/12066553/23d057fa8ea3/fneur-16-1550023-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fbe5/12066553/23d057fa8ea3/fneur-16-1550023-g001.jpg

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本文引用的文献

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J Neurol. 2024 Sep;271(9):5911-5915. doi: 10.1007/s00415-024-12556-1. Epub 2024 Jul 9.
2
Efgartigimod as rescue treatment in acute phase of neuromyelitis optica spectrum disorder: A Case Report.艾加莫德作为视神经脊髓炎谱系障碍急性期的挽救治疗:一例报告
Heliyon. 2024 Apr 26;10(9):e30421. doi: 10.1016/j.heliyon.2024.e30421. eCollection 2024 May 15.
3
Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome.
依氟鸟氨酸治疗重症肌无力之外:FcRn 靶向治疗在僵人综合征中的作用。
J Neurol. 2024 Jan;271(1):254-262. doi: 10.1007/s00415-023-11970-1. Epub 2023 Sep 8.
4
Updated consensus statement: Intravenous immunoglobulin in the treatment of neuromuscular disorders report of the AANEM ad hoc committee.更新的共识声明:静脉注射免疫球蛋白治疗神经肌肉疾病——AANEM 特别委员会的报告。
Muscle Nerve. 2023 Oct;68(4):356-374. doi: 10.1002/mus.27922. Epub 2023 Jul 11.
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LGI1 antibody encephalitis: acute treatment comparisons and outcome.LGI1 抗体脑炎:急性期治疗比较和结局。
J Neurol Neurosurg Psychiatry. 2022 Mar;93(3):309-315. doi: 10.1136/jnnp-2021-327302. Epub 2021 Nov 25.
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