Gombolay Grace Y, Chitnis Tanuja
Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, 55 Fruit Street, ACC708, Boston, MA, 02114, USA.
Partners Multiple Sclerosis Center, Brigham and Women's Hospital, 60 Fenwood Road, Boston, MA, USA.
Curr Treat Options Neurol. 2018 May 2;20(6):19. doi: 10.1007/s11940-018-0502-9.
Neuromyelitis optica spectrum disorders (NMOSDs) are a group of inflammatory and demyelinating disorders of the central nervous system that can occur in children and adults. The classic presentation of NMOSD is characterized by optic neuritis and transverse myelitis, but other presentations are also recognized, expanding the disease as NMO spectrum disorders. The purpose of this review is to discuss the clinical features, along with management and treatment options, including potential future therapeutic options, in pediatric NMOSD.
The aquaporin-4 antibody (AQP4 ab) is specific for NMOSD; however, recently another antibody, the myelin oligodendrocyte glycoprotein (MOG ab) has been found in a subset of AQP4 ab-negative patients including in children. Most treatment studies are reported in adults, but retrospective studies on efficacy and safety of mycophenolate mofetil, azathioprine, and rituximab in pediatric NMOSD are available. While some pediatric NMOSD-specific treatment studies are available, more research is needed in the mechanisms of early onset and specific treatment options in children, including whether different treatment considerations are needed for AQP4 ab as opposed to MOG ab-positive disease.
视神经脊髓炎谱系障碍(NMOSDs)是一组可发生于儿童和成人的中枢神经系统炎性脱髓鞘疾病。NMOSD的典型表现为视神经炎和横贯性脊髓炎,但也认识到其他表现形式,从而将该疾病扩展为视神经脊髓炎谱系障碍。本综述的目的是讨论儿童NMOSD的临床特征以及管理和治疗选择,包括潜在的未来治疗选择。
水通道蛋白4抗体(AQP4 ab)对NMOSD具有特异性;然而,最近在包括儿童在内的一部分AQP4 ab阴性患者中发现了另一种抗体,即髓鞘少突胶质细胞糖蛋白(MOG ab)。大多数治疗研究报告于成人,但有关于霉酚酸酯、硫唑嘌呤和利妥昔单抗在儿童NMOSD中的疗效和安全性的回顾性研究。虽然有一些针对儿童NMOSD的治疗研究,但在儿童早期发病机制和特定治疗选择方面仍需要更多研究,包括与MOG ab阳性疾病相比,AQP4 ab阳性疾病是否需要不同的治疗考量。