Department of Neurology, Mayo Clinic, Rochester, MN, USA.
Department of Neurology, Mayo Clinic, 13400 East Shea Boulevard, Scottsdale, AZ, 85259, USA.
BioDrugs. 2021 Jan;35(1):7-17. doi: 10.1007/s40259-020-00460-9. Epub 2020 Dec 10.
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune, inflammatory disorder of the central nervous system that typically presents with recurrent episodes of optic neuritis, longitudinally extensive myelitis, brainstem, diencephalic, and cerebral syndromes. Up to 80% of NMOSD patients have a circulating pathogenic autoantibody that targets the water channel aquaporin-4 (AQP4-IgG). The discovery of AQP4-IgG transformed our understanding of the pathogenesis of the disease and its possible treatment targets. Monoclonal antibodies targeting terminal complement (eculizumab), CD19 (inebilizumab), and the interleukin-6 receptor (satralizumab) have demonstrated efficacy in NMOSD attack prevention in recent phase 3 trials and have gained subsequent regulatory approval in the USA and other countries. We aim to review the evidence supporting the efficacy of these new drugs.
视神经脊髓炎谱系疾病（NMOSD）是一种中枢神经系统自身免疫性、炎症性疾病，通常表现为复发性视神经炎、长节段横贯性脊髓炎、脑干、间脑和大脑综合征。多达 80%的 NMOSD 患者存在一种针对水通道蛋白 4（AQP4-IgG）的循环致病性自身抗体。AQP4-IgG 的发现改变了我们对疾病发病机制及其可能治疗靶点的认识。最近的 3 期临床试验表明，靶向末端补体（依库珠单抗）、CD19（依那西普单抗）和白细胞介素 6 受体（萨替鲁单抗）的单克隆抗体在预防 NMOSD 发作方面具有疗效，并随后在美国和其他国家获得监管批准。我们旨在回顾支持这些新药疗效的证据。
