TY - JOUR T1 -小儿视神经脊髓炎谱系障碍的治疗反应JF -神经学JO -神经学SP - e985 LP - e994 DO - 10.1212/WNL.00000000002半岛投注体育官网01625六世- 100 - 9盟Raffaella Pizzolato Umeton AU -迈克尔·华尔兹盟-格雷戈里·s . Aaen AU -莱斯利·本森AU -马克戈尔曼盟马努Goyal AU -珍妮弗·s .坟墓AU -尤兰达哈里斯盟-劳伦·克虏伯盟-蒂莫西·e·Lotze AU -尼基塔·m·舒克拉盟Soe Mar盟杰恩洛克非盟-玛丽Rensel盟泰瑞称盟Jan-Mendelt Tillema AU -雪莱Roalstad盟摩西·罗德里格斯AU - John Rose盟Emmanuelle Waubant盟比安卡Weinstock-Guttman AU -查尔斯·卡斯珀盟Tanuja Chitnis AU -背景和目的视神经脊髓炎谱系障碍(NMOSD)是一种罕见的自身免疫性疾病，可导致严重的残疾，高达3%-5%的病例具有儿科起病。半岛投注体育官网目前有有限的研究指导医生选择NMOSD儿童的疾病修饰治疗(DMT)。方法:这项回顾性队列研究评估了在美国儿科多发性硬化中心网络的12个诊所随访的NMOSD患儿。病例分类为水通道蛋白-4抗体阳性(AQP4+)， AQP4+和髓鞘少突胶质细胞糖蛋白抗体阴性(MOG)时为双血清阴性(DS)。采用负二项回归方法评估初始DMTs(包括利妥昔单抗、霉酚酸盐、硫唑嘌呤和IV免疫球蛋白)对年化复发率(ARR)的影响。到残疾进展的时间(EDSS评分增加≥1.0分)采用Cox比例风险模型建模。结果共检出91例NMOSD患儿，其中AQP4+患儿77例，DS患儿14例(85.7%为女性;白人43.2%，非洲裔46.6%)。81例患者开始接受DMT治疗，10例在分析时未接受治疗。 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 ER -