Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
Curr Opin Rheumatol. 2021 May 1;33(3):233-239. doi: 10.1097/BOR.0000000000000791.
This review examines recently published randomized placebo-controlled trials for the treatment of neuromyelitis optica spectrum disorders (NMOSD).
Until recently, treatments for NMOSD were used-off label and had not been subjected to randomized placebo-controlled trials. Increased understanding of the pathophysiology of NMOSD, particularly aquaporin-4-IgG seropositive NMOSD, lead to the investigation of eculizumab, inebilizumab, and satralizumab for maintenance therapy. Eculizumab inhibits the cleavage of the terminal complement protein C5, inebilizumab depletes immune cells of B-lymphocyte lineage, and satralizumab inhibits interleukin-6 receptors. International, phase 3, randomized, placebo-controlled trials have demonstrated that each of these therapies reduces the risk of NMOSD relapse. In some cases, the studied therapies were administered in conjunction with other immunosuppressants. Each therapy has important safety considerations, notably risk of meningococcal infection with eculizumab and risks of infection and hypogammaglobulinemia with inebilizumab. Reviewing trial design highlights future areas of inquiry for the treatment of NMOSD.
Eculizumab, inebilizumab, and satralizumab are effective maintenance therapies approved for the treatment of AQP-4 seropositive NMOSD.
本篇综述考察了最近发表的用于治疗视神经脊髓炎谱系疾病（NMOSD）的随机安慰剂对照试验。
直到最近，NMOSD 的治疗方法都是在没有经过随机安慰剂对照试验的情况下使用的。对 NMOSD 病理生理学的深入了解，特别是水通道蛋白-4-IgG 阳性 NMOSD，促使人们研究依库珠单抗、伊奈利珠单抗和萨特利珠单抗用于维持治疗。依库珠单抗抑制末端补体蛋白 C5 的裂解，伊奈利珠单抗耗尽 B 淋巴细胞谱系的免疫细胞，萨特利珠单抗抑制白细胞介素-6 受体。国际、3 期、随机、安慰剂对照试验表明，这些疗法中的每一种都降低了 NMOSD 复发的风险。在某些情况下，研究中的疗法与其他免疫抑制剂联合使用。每种疗法都有重要的安全注意事项，尤其是依库珠单抗存在脑膜炎球菌感染的风险，以及伊奈利珠单抗存在感染和低丙种球蛋白血症的风险。回顾试验设计突出了 NMOSD 治疗的未来研究领域。
依库珠单抗、伊奈利珠单抗和萨特利珠单抗是批准用于治疗水通道蛋白-4 阳性 NMOSD 的有效维持治疗药物。
