Department of Neurology, Akershus University Hospital, Lørenskog, Norway.
Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
J Neurol. 2021 Dec;268(12):4522-4536. doi: 10.1007/s00415-020-10235-5. Epub 2020 Oct 3.
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.
Literature search on clinical trials and case studies in NMOSD up to July 10. 2020.
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.
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.
目前，视神经脊髓炎谱系疾病（NMOSD）的治疗主要基于回顾性病例系列研究。最近，六项随机临床试验的结果已经公布，这些试验包括五种针对四种分子和三种不同病理生理途径的不同单克隆抗体。
对截至 2020 年 7 月 10 日的 NMOSD 临床试验和病例研究进行文献检索。
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、依那西普单抗、依库珠单抗、托珠单抗和 satralizumab）的作用机制、疗效和副作用，以及对生殖健康的影响。
在针对水通道蛋白 4 抗体的 NMOSD 患者中，与安慰剂相比，耗竭 B 细胞的单克隆抗体（利妥昔单抗和依那西普单抗）或干扰白细胞介素 6 信号的单克隆抗体（托珠单抗和 satralizumab）或补体激活的单克隆抗体（依库珠单抗）具有更好的疗效。托珠单抗和利妥昔单抗在头对头研究中也优于硫唑嘌呤。利妥昔单抗、托珠单抗和在一定程度上依库珠单抗在其他炎症性疾病中有良好的安全性，利妥昔单抗和硫唑嘌呤在怀孕期间可能是安全的。
