Background

Treatment of neuromyelitis optica spectrum disorder (NMOSD) has so far been based on retrospective case series. The results of six randomized clinical trials including five different monoclonal antibodies targeting four molecules and three distinct pathophysiological pathways have recently been published.

Methods

Literature search on clinical trials and case studies in NMOSD up to July 10. 2020.

Results

We review mechanism of action, efficacy and side effects, and consequences for reproductive health from traditional immunosuppressants and monoclonal antibodies including rituximab, inebilizumab, eculizumab, tocilizumab and satralizumab.

Conclusion

In NMOSD patients with antibodies against aquaporin 4, monoclonal antibodies that deplete B cells (rituximab and inebilizumab) or interfere with interleukin 6 signaling (tocilizumab and satralizumab) or complement activation (eculizumab) have superior efficacy compared to placebo. Tocilizumab and rituximab were also superior to azathioprine in head-to-head studies. Rituximab, tocilizumab and to some extent eculizumab have well-known safety profiles for other inflammatory diseases, and rituximab and azathioprine may be safe during pregnancy.

中文翻译：

---

视神经脊髓炎谱系障碍维持治疗的最新进展

背景

迄今为止，视神经脊髓炎谱系障碍 (NMOSD) 的治疗基于回顾性病例系列。最近发表了六项随机临床试验的结果，包括针对四种分子和三种不同病理生理途径的五种不同单克隆抗体。

方法

截至 2020 年 7 月 10 日，NMOSD 临床试验和案例研究的文献检索。

结果

我们回顾了传统免疫抑制剂和单克隆抗体（包括利妥昔单抗、inebilizumab、eculizumab、tocilizumab 和 satralizumab）的作用机制、疗效和副作用以及对生殖健康的影响。

结论

在具有抗水通道蛋白 4 抗体的 NMOSD 患者中，与安慰剂相比，消耗 B 细胞（利妥昔单抗和inebilizumab）或干扰白细胞介素 6 信号（托珠单抗和 satralizumab）或补体激活（依库珠单抗）的单克隆抗体具有更好的疗效。在头对头研究中，托珠单抗和利妥昔单抗也优于硫唑嘌呤。利妥昔单抗、托珠单抗和某种程度的依库珠单抗对其他炎症性疾病具有众所周知的安全性，而利妥昔单抗和硫唑嘌呤在怀孕期间可能是安全的。