Efficacy of eculizumab in acute refractory pediatric neuromyelitis optica: A case report

Michael Enriquez , Scott Rosenthal , Loren A. McLendon , Jeffrey L. Bennett , Amanda L. Piquet , Ryan Kammeyer
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Abstract

Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system caused by autoantibodies against the aquaporin-4 (AQP4) water channel. Inflammatory injury is often severe and focused on the optic nerves, spinal cord, and other CNS regions with high AQP4 expression. Acute management includes pulse corticosteroids and plasmapheresis; however, many patients have incomplete recovery. We describe the successful use of eculizumab in treating an acute severe refractory brainstem syndrome in a pediatric patient with NMOSD, highlighting the potential utility of eculizumab as a potent, acute therapy in pediatric NMOSD.

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依库珠单抗对急性难治性小儿神经脊髓炎视网膜病变的疗效:病例报告
神经脊髓炎视网膜谱系障碍(NMOSD)是一种中枢神经系统自身免疫性炎症性疾病,由针对水通道蛋白-4(AQP4)的自身抗体引起。炎症损伤通常很严重,主要集中在视神经、脊髓和其他 AQP4 高表达的中枢神经系统区域。急性期的治疗包括脉冲皮质类固醇激素和血浆置换术;然而,许多患者无法完全康复。我们介绍了使用依库珠单抗成功治疗一名急性重症难治性脑干综合征儿科 NMOSD 患者的情况,突出了依库珠单抗作为一种有效的急性疗法在儿科 NMOSD 中的潜在作用。
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