Professor Emeritus of Pharmacology, University of Auckland, Auckland, New Zealand and Professor Emeritus of Oral Biology, University of Alberta, Edmonton, Alberta, Canada.
Drugs Today (Barc). 2021 Mar;57(3):209-218. doi: 10.1358/dot.2021.57.3.3251715.
Neuromyelitis optica spectrum disorders (NMOSD) consist of a rare autoimmune disorder in which patients suffer from relapses that affect the optic nerve, spinal cord or brainstem. Few have a full recovery. NMOSD is more common in women, the age of onset being around 30-40 years of age depending on race. The prevalence of the condition varies from 0.5-4.4 per 100,000 population. About 80% of patients have antibodies directed against the aquaporin-4 (AQP4) protein that form membrane-bound water transporters in the central nervous system (CNS). This protein is highly expressed in those areas of the CNS often targeted in NMOSD relapses. Satralizumab is a humanized monoclonal antibody that binds to the interleukin-6 (IL-6) receptor and thus inhibits IL-6 signaling. Two recent phase III studies have demonstrated that satralizumab significantly reduced the relapse rate in NMOSD by 76-79%. This beneficial effect was apparently confined to patients who have anti-AQP4 antibodies and satralizumab did not reduce the rate of pain or fatigue in NMOSD patients.
视神经脊髓炎谱系疾病(NMOSD)是一种罕见的自身免疫性疾病,患者会反复发作,影响视神经、脊髓或脑干。很少有患者能完全康复。NMOSD 在女性中更为常见,发病年龄在 30-40 岁左右,具体取决于种族。这种疾病的患病率在每 10 万人中有 0.5-4.4 人不等。大约 80%的患者有针对水通道蛋白-4(AQP4)蛋白的抗体,该蛋白在中枢神经系统(CNS)中形成膜结合水转运体。这种蛋白在 NMOSD 复发时经常靶向的中枢神经系统区域中高度表达。Satralizumab 是一种人源化单克隆抗体,可与白细胞介素-6(IL-6)受体结合,从而抑制 IL-6 信号传导。两项最近的 III 期研究表明,Satralizumab 可使 NMOSD 的复发率显著降低 76-79%。这种有益的效果显然仅限于具有抗 AQP4 抗体的患者,Satralizumab 并未降低 NMOSD 患者的疼痛或疲劳发生率。