RT杂志文章SR电子T1治疗反应在小儿神经脊髓炎视神经谱系障碍JF神经学JO神经学FD Lippincott Williams & Wilkins SP e985 OP e994 DO 10.1212/WNL.0000000半岛投注体育官网000201625签证官100 9 A1 Raffaella Pizzolato Umeton A1迈克尔·华尔兹A1格雷戈里·s . Aaen A1莱斯利·本森A1马克戈尔曼A1马努Goyal A1詹妮弗·s .坟墓A1尤兰达哈里斯A1劳伦克虏伯A1盖大肠Lotze A1尼基塔·m·舒克拉A1 Soe Mar A1杰恩洛克A1玛丽Rensel A1泰瑞称A1 Jan-Mendelt Tillema A1雪莱Roalstad A1摩西·罗德里格斯A1 John Rose A1 Emmanuelle Waubant A1比安卡Weinstock-Guttman A1查尔斯·卡斯珀A1 Tanuja Chitnis A1代表美国女士儿科中心网络2023年UL //www.ebmtp.com/content/100/9/e985.abstract AB Background and Objective Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune condition, which can lead to significant disability, and up to 3%–5% of the cases have a pediatric onset. There are limited studies to guide physicians in disease-modifying treatment (DMT) choices for children with NMOSD.Methods This retrospective cohort study evaluated children with NMOSD cases followed at 12 clinics in the US Network of Pediatric MS Centers. Cases were classified as aquaporin-4 antibody positive (AQP4+) and double seronegative (DS) when negative for AQP4+ and for myelin oligodendrocyte glycoprotein (MOG) antibody. The effect of initial DMTs including rituximab, mycophenolate, azathioprine, and IV immunoglobulin (IVIg) on the annualized relapse rate (ARR) was assessed by negative binomial regression. Time to disability progression (EDSS score increase ≥1.0 point) was modeled with a Cox proportional-hazards model.Results A total of 91 children with NMOSD were identified: 77 AQP4+ and 14 DS (85.7% females; 43.2% White and 46.6% African American). Eighty-one patients were started on a DMT, and 10 were treatment naive at the time of the analysis. The ARR calculated in all serogroups was 0.25 (95% CI 0.13–0.49) for rituximab, 0.33 (95% CI 0.19–0.58) for mycophenolate, 0.40 (95% CI 0.13–1.24) for azathioprine, and 0.54 (95% CI 0.28–1.04) for IVIg. The ARR in the AQP4+ subgroup was 0.28 (95% CI 0.14–0.55) for rituximab, 0.39 (95% CI 0.21–0.70) for mycophenolate, 0.41 (95% CI 0.13–1.29) for azathioprine, and 0.54 (95% CI 0.23–1.26) for IVIg. The ARR in the treatment-naive group was 0.97 (95% CI 0.58–1.60) in all serogroups and 0.91 (95% CI 0.53–1.56) in the AQP4+ subgroup. None of the initial DMT had a statistically significant effect on EDSS progression.Discussion The use of DMTs, particularly rituximab, is associated with a lowered annualized relapse rate in children with NMOSD AQP4+.Classification of Evidence This study provides Class IV evidence that use of disease-modifying treatments is associated with a lowered annualized relapse rate in children with NMOSD AQP4+.ARR=annualized relapse rate; DCAC=Data Coordinating and Analysis Center; DMTs=disease-modifying treatments; EDSS=Expanded Disability Status Scale; LETM=longitudinally extensive transverse myelitis; MS=multiple sclerosis; NMOSD=neuromyelitis optica spectrum disorder