Universidade de São Paulo, Faculdade de Medicina, Hospital das Clínicas, Departamento de Neurologia, São Paulo SP, Brazil.
Pontifícia Universidade Católica do Rio Grande do Sul, Instituto do Cérebro do Rio Grande do Sul, Porto Alegre RS, Brazil.
Arq Neuropsiquiatr. 2023 Feb;81(2):201-211. doi: 10.1055/s-0043-1761432. Epub 2023 Mar 22.
Neuromyelitis optica spectrum disorder (NMOSD) is a rare and severe inflammatory disorder of the central nervous system (CNS). It is strongly associated with anti-aquaporin 4 antibodies (AQP4-IgG), and it mainly affects young women from non-white ethnicities. However, ∼ 5 to 10% of all cases have onset during childhood. Children and adolescents share the same clinical, radiologic, and laboratory presentation as adults. Thus, the same NMOSD diagnostic criteria are also applied to pediatric-onset patients, but data on NMOSD in this population is still scarce. In seronegative pediatric patients, there is a high frequency of the antibody against myelin oligodendrocyte glycoprotein (MOG-IgG) indicating another disease group, but the clinical distinction between these two diseases may be challenging. Three drugs (eculizumab, satralizumab, and inebilizumab) have been recently approved for the treatment of adult patients with AQP4-IgG-positive NMOSD. Only satralizumab has recruited adolescents in one of the two pivotal clinical trials. Additional clinical trials in pediatric NMOSD are urgently required to evaluate the safety and efficacy of these drugs in this population.
视神经脊髓炎谱系疾病（NMOSD）是一种罕见且严重的中枢神经系统（CNS）炎症性疾病。它与抗水通道蛋白 4 抗体（AQP4-IgG）强烈相关，主要影响来自非白种人群的年轻女性。然而，约 5%至 10%的病例发生在儿童期。儿童和青少年与成人具有相同的临床、影像学和实验室表现。因此，同样的 NMOSD 诊断标准也适用于儿科发病的患者，但该人群中 NMOSD 的数据仍然有限。在血清阴性的儿科患者中，有一种针对髓鞘少突胶质细胞糖蛋白（MOG-IgG）的抗体的频率很高，表明存在另一种疾病群体，但这两种疾病的临床鉴别可能具有挑战性。三种药物（依库珠单抗、satralizumab 和 inebilizumab）最近已被批准用于治疗 AQP4-IgG 阳性 NMOSD 的成年患者。只有 satralizumab 在两项关键临床试验中的一项中招募了青少年。迫切需要在儿科 NMOSD 中开展额外的临床试验，以评估这些药物在该人群中的安全性和疗效。
