Acute eculizumab treatment in a pediatric patient with AQP4-IgG+ NMOSD.

IF 5 2区 医学 Q1 CLINICAL NEUROLOGY Multiple Sclerosis Journal Pub Date : 2025-04-01 Epub Date: 2025-01-22 DOI:10.1177/13524585241283650
Ria H Soni, Mekka Garcia, Eunhye Oak, Eliana J Applbaum, Logi Rajagopalan, Lauren B Krupp, Kimberly A O'Neill
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Abstract

Background: Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disorder that occurs in children and adults.

Case: We report a case of a 10-year-old female with AQP4+ NMOSD who presented with paraparesis from longitudinally extensive transverse myelitis (LETM) from C2 to the conus medullaris. The patient showed gradual improvement in strength and sensation with solumedrol and plasma exchange therapy. Given her severe presentation, eculizumab therapy was also initiated acutely. She had near complete recovery, although she developed a myelitis relapse during transition to rituximab treatment.

Conclusion: This case demonstrates the role of eculizumab as a safe and effective treatment option in treating an acute attack of pediatric AQP4+ NMOSD. More data are needed to understand the risk of relapse if transitioning off of these highly effective medications.

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急性依珠单抗治疗AQP4-IgG+ NMOSD患儿
背景:视神经脊髓炎谱系障碍(NMOSD)是一种罕见的自身免疫性疾病,常见于儿童和成人。病例:我们报告了一例10岁女性AQP4+ NMOSD患者,其表现为从C2到髓圆锥的纵向广泛横断面脊髓炎(LETM)的麻痹。采用舒美地罗和血浆交换治疗后,患者的力量和感觉逐渐改善。鉴于她的严重表现,eculizumab治疗也被急性启动。她几乎完全康复,尽管在过渡到利妥昔单抗治疗期间,她复发了脊髓炎。结论:本病例证明了eculizumab作为一种安全有效的治疗选择,可用于治疗小儿AQP4+ NMOSD急性发作。需要更多的数据来了解在停止使用这些高效药物后复发的风险。
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来源期刊
Multiple Sclerosis Journal
Multiple Sclerosis Journal 医学-临床神经学
CiteScore
10.90
自引率
6.90%
发文量
186
审稿时长
3-8 weeks
期刊介绍: Multiple Sclerosis Journal is a peer-reviewed international journal that focuses on all aspects of multiple sclerosis, neuromyelitis optica and other related autoimmune diseases of the central nervous system. The journal for your research in the following areas: * __Biologic basis:__ pathology, myelin biology, pathophysiology of the blood/brain barrier, axo-glial pathobiology, remyelination, virology and microbiome, immunology, proteomics * __Epidemology and genetics:__ genetics epigenetics, epidemiology * __Clinical and Neuroimaging:__ clinical neurology, biomarkers, neuroimaging and clinical outcome measures * __Therapeutics and rehabilitation:__ therapeutics, rehabilitation, psychology, neuroplasticity, neuroprotection, and systematic management Print ISSN: 1352-4585
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