ORIGINAL RESEARCH article
Front. Immunol.
Sec. Autoimmune and Autoinflammatory Disorders: Autoinflammatory Disorders
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1645401
This article is part of the Research TopicClinical and Immunological Phenotypic Characterization to better understand Pathogenesis and Response to Therapies in Systemic Autoimmune DiseasesView all 6 articles
Eculizumab for the acute attack of neuromyelitis optica spectrum disorder
Provisionally accepted- 1Wenzhou Central Hospital, Wenzhou, China
- 2Fudan University Huashan Hospital Department of Neurology, Shanghai, China
- 3Huashan Hospital Fudan University, Shanghai, China
- 4Fudan University Eye Ear Nose and Throat Hospital Department of Ophthalmology, Shanghai, China
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To assess clinical outcomes in patients treated with eculizumab for acute attacks of aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD).We retrospectively analyzed prospectively collected data from the Huashan NMOSD registry cohort, and included patients who received eculizumab within 30 days of attack onset. Eculizumab was administered at 900 mg weekly for four weeks, followed by an eight-week observation period. Primary outcomes included visual acuity and visual field for optic neuritis (ON) and muscle strength, assessed using the Medical Research Council (MRC) scale for longitudinally extensive transverse myelitis (LETM). Serum neurofilament light chain (sNfL) and glial fibrillary acidic protein (sGFAP) levels were also monitored.Nine patients (seven with ON, two with LETM) were included. All patients received high-dose intravenous methylprednisolone prior to eculizumab treatment. Following eculizumab, six of the seven ON patients showed significant improvements in visual acuity and visual fields, with five recovering to near-normal or pre-attack vision. Visual field mean deviation improved from -22.4 dB to -2.0 dB (p = 0.008). Among LETM patients, one regained substantial lower limb strength (MRC grade 0 to 3 proximally, 4 distally), while the other showed improvement in distal strength (MRC grade 0 to 3).Serum sGFAP decreased from 278.0 to 130 pg/mL (p = 0.027), while sNfL levels transiently increased before stabilizing. LETM patients developed urinary tract infections, and one had Klebsiella pneumoniae pneumonia; all infections were effectively treated.Eculizumab may yield favorable outcomes in acute NMOSD attacks, with infection monitoring being particularly important in severe cases.
Keywords: Eculizumab, Neuromyelitis optica spectrum disorder, acute attack, Neurofilament light, Glial Fibrillary Acidic Protein
Received: 11 Jun 2025; Accepted: 01 Sep 2025.
Copyright: © 2025 Zhuang, Huang, Zhangbao, Wang, ZHOU, Li, Fan, Wang, Xiao, Yu, Wang, Tan and Quan. 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:
Hongmei Tan, Fudan University Huashan Hospital Department of Neurology, Shanghai, China
Chao Quan, Fudan University Huashan Hospital Department of Neurology, Shanghai, China
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