Efgartigimod for steroid-resistant autoimmune glial fibrillary acidic protein astrocytopathy: a case report and literature review

IF 2.5 4区医学 Q3 IMMUNOLOGY

Journal of neuroimmunology Pub Date : 2026-04-01 Epub Date: 2026-01-24 DOI:10.1016/j.jneuroim.2026.578866

Peng Lei , Yun Lu , Jie Xia , Tao Wang , Ling Zhong

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引用次数: 0

Abstract

Background

Neonatal Fc receptor (FcRn) inhibitors rapidly and specifically clear serum immunoglobulin G (IgG) levels and, therefore, are increasingly used for the treatment of neurological autoimmune diseases, such as myasthenia gravis. However, whether FcRn inhibitors could alleviate steroid-unresponsive glial fibrillary acidic protein astrocytopathy (GFAP-A) has not been reported.

Case presentation

We report a case of a 68-year-old male patient who presented with gait instability and numbness in the left hand. Cranial magnetic resonance imaging (MRI) revealed multiple demyelinating lesions. The initial clinical diagnosis was demyelinating encephalopathy. After 1 month of intravenous methylprednisolone pulse therapy, followed by sequential oral prednisone, the patient's gait instability did not improve. Upon re-examination at our hospital, cranial MRI revealed no significant changes in the lesions. Testing for central nervous system demyelinating antibodies revealed serum anti-GFAP IgG antibody (titer 1:100), cerebrospinal fluid anti-GFAP IgG antibody (titer 1:1), and negative result for oligoclonal bands in blood and the cerebrospinal fluid. The final diagnosis was GFAP-A. Treatment with the FcRn inhibitor efgartigimod significantly improved clinical symptoms and brain lesions.

Conclusions

This rare case indicates that efgartigimod is a promising treatment option for steroid-unresponsive GFAP-A.

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依加替莫德治疗类固醇抵抗性自身免疫性胶质原纤维酸性蛋白星形细胞病1例报告并文献复习

背景：新生儿Fc受体（FcRn）抑制剂可快速特异性清除血清免疫球蛋白G （IgG）水平，因此越来越多地用于治疗神经自身免疫性疾病，如重症肌无力。然而，FcRn抑制剂是否可以缓解类固醇无反应的胶质原纤维酸性蛋白星形细胞病（GFAP-A）尚未报道。我们报告一例68岁男性患者，其表现为步态不稳和左手麻木。头颅磁共振成像（MRI）显示多发脱髓鞘病变。最初的临床诊断为脱髓鞘性脑病。静脉注射甲基强的松龙脉冲治疗1个月后，随后连续口服强的松，患者的步态不稳定没有改善。在我院复查后，颅脑MRI显示病变无明显变化。中枢神经系统脱髓鞘抗体检测血清抗gfap IgG抗体（滴度1:100），脑脊液抗gfap IgG抗体（滴度1:1），血、脑脊液寡克隆带阴性。最终诊断为gmap - a。使用FcRn抑制剂efgartigimod治疗可显著改善临床症状和脑部病变。结论这一罕见的病例表明依加替莫德是治疗类固醇无反应的GFAP-A的一种有希望的治疗选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of neuroimmunology 医学-免疫学

CiteScore

6.10

自引率

3.00%

发文量

154

审稿时长

37 days

期刊介绍： The Journal of Neuroimmunology affords a forum for the publication of works applying immunologic methodology to the furtherance of the neurological sciences. Studies on all branches of the neurosciences, particularly fundamental and applied neurobiology, neurology, neuropathology, neurochemistry, neurovirology, neuroendocrinology, neuromuscular research, neuropharmacology and psychology, which involve either immunologic methodology (e.g. immunocytochemistry) or fundamental immunology (e.g. antibody and lymphocyte assays), are considered for publication.