CASE REPORT article
Front. Immunol.
Sec. Autoimmune and Autoinflammatory Disorders: Autoinflammatory Disorders
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1581954
The Utilization of Efgartigimod in the Treatment of Acute Cerebellar Ataxia: A Case Report
Provisionally accepted
- 1Department of Neurology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
- 2Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
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Background: Acute cerebellar ataxia (ACA) is the most common cause of acute ataxia in children and adolescents. It is a cerebellar disorder with multifactorial pathogenesis, often triggered by viral or bacterial infections, as well as autoimmune mechanisms. The clinical course of ACA can vary widely, ranging from a benign, self-limiting condition to a severe, life-threatening illness. There is no universally accepted consensus on the optimal management of ACA in children. While steroids and immunoglobulins are commonly used, some patients may not respond adequately to these treatments. Efgartigimod, a novel immune modulator, has not been previously reported in the treatment of ACA, but its unique mechanism of action suggests potential therapeutic benefits. Case Presentation: We present a case of an 11-year-old girl with ACA who was treated with efgartigimod and showed significant improvements. The patient presented with acute ataxia, slurred speech, vomiting, diarrhea, abdominal pain, dizziness, and altered mental status. Initial investigations, including routine blood tests, specific autoantibodies related to cerebellitis in both serum and cerebrospinal fluid (CSF), and brain magnetic resonance imaging (MRI) revealed no abnormalities. Despite treatment with neurotrophic drugs and dexamethasone, the patient showed minimal improvement. A trial of efgartigimod (10 mg/kg) resulted in rapid symptom alleviation within three days. Conclusion: This case highlights the potential role of efgartigimod in the treatment of ACA, particularly in cases refractory to conventional therapies. Further studies are needed to validate the efficacy and safety of Efgartigimod in pediatric patients with ACA.
Keywords: Acute cerebellar ataxia, Autoimmune, antibody, efgartigimod, neonatal Fc receptor, Children
Received: 12 Apr 2025; Accepted: 12 Aug 2025.
Copyright: © 2025 Yang, Ruan, Cai and Xia. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Liu L. Yang, Department of Neurology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
Yuan Peng Xia, Department of Neurology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.