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<p>Eculizumab as C5 complement protein inhibitor has been approved for the maintenance treatment of neuromyelitis optica spectrum disorder (NMOSD). However, evidence regarding its efficacy in the acute phase remain limited. This study enrolled six patients with aquaporin-4 (AQP4)-IgG-seropositive neuromyelitis optica, including five cases of optic neuritis and one case of long-segment transverse myelitis. These patients exhibited no or partial symptomatic improvement following glucocorticoid pulse therapy, and two patient experienced further deterioration. Subsequently, eculizumab was added to the treatment regimen, and all patients achieved rapid symptom improvement without adverse events reported post-infusion. The addition of eculizumab to glucocorticoid therapy during the acute phase may significantly ameliorate symptoms in AQP4-IgG-positive NMOSD patients with rapid onset of action and favorable safety profile. This may provide a new treatment option for patients with poor response to glucocorticoids in the acute phase, while mitigating glucocorticoid-related adverse effects to a certain degree.</p>

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Eculizumab as a rescue therapy in acute-phase aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder

  • 期刊论文

  • 发表日期：

    2026年1月9日

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  • Keyi Zeng,
  • Wei Chen,
  • Aiyu Lin

摘要

Eculizumab as C5 complement protein inhibitor has been approved for the maintenance treatment of neuromyelitis optica spectrum disorder (NMOSD). However, evidence regarding its efficacy in the acute phase remain limited. This study enrolled six patients with aquaporin-4 (AQP4)-IgG-seropositive neuromyelitis optica, including five cases of optic neuritis and one case of long-segment transverse myelitis. These patients exhibited no or partial symptomatic improvement following glucocorticoid pulse therapy, and two patient experienced further deterioration. Subsequently, eculizumab was added to the treatment regimen, and all patients achieved rapid symptom improvement without adverse events reported post-infusion. The addition of eculizumab to glucocorticoid therapy during the acute phase may significantly ameliorate symptoms in AQP4-IgG-positive NMOSD patients with rapid onset of action and favorable safety profile. This may provide a new treatment option for patients with poor response to glucocorticoids in the acute phase, while mitigating glucocorticoid-related adverse effects to a certain degree.