Neuromyelitis optica (NMO)/NMO spectrum disorder (NMOSD) is a chronic, recurrent, antibody-mediated, inflammatory demyelinating disease of the central nervous system, characterized by optic neuritis and transverse myelitis. The binding of NMO-IgG with astrocytic aquaporin-4 (AQP4) functions directly in the pathogenesis of >60% of NMOSD patients, and causes astrocyte loss, secondary inflammatory infiltration, demyelination, and neuron death, potentially leading to paralysis and blindness. Current treatment options, including immunosuppressive agents, plasma exchange, and B-cell depletion, are based on small retrospective case series and open-label studies. It is noteworthy that monoclonal antibody (mAb) therapy is a better option for autoimmune diseases due to its high efficacy and tolerability. Although the pathophysiological mechanisms of NMOSD remain unknown, increasingly, therapeutic studies have focused on mAbs, which target B cell depletion, complement and inflammation cascade inactivation, blood-brain-barrier protection, and blockade of NMO-IgG-AQP4 binding. Here, we review the targets, characteristics, mechanisms of action, development, and potential efficacy of mAb trials in NMOSD, including preclinical and experimental investigations.

视神经脊髓炎 (NMO)/NMO 谱系障碍 (NMOSD) 是一种慢性、复发性、抗体介导的中枢神经系统炎性脱髓鞘疾病，以视神经炎和横贯性脊髓炎为特征。NMO-IgG 与星形细胞水通道蛋白 4 (AQP4) 的结合直接在超过 60% 的 NMOSD 患者的发病机制中起作用，并导致星形胶质细胞丢失、继发性炎症浸润、脱髓鞘和神经元死亡，可能导致瘫痪和失明。当前的治疗选择，包括免疫抑制剂、血浆置换和 B 细胞耗竭，都是基于小型回顾性病例系列和开放标签研究。值得注意的是，单克隆抗体（mAb）疗法因其高效和耐受性而成为自身免疫性疾病更好的选择。尽管 NMOSD 的病理生理机制仍然未知，但越来越多的治疗研究集中在 mAb 上，其靶向 B 细胞耗竭、补体和炎症级联失活、血脑屏障保护和阻断 NMO-IgG-AQP4 结合。在这里，我们回顾了 NMOSD 中 mAb 试验的靶点、特征、作用机制、发展和潜在功效，包括临床前和实验研究。