PT - JOURNAL ARTICLE AU - Giulia Longoni AU - Brenda Banwell AU - Massimo Filippi AU - E. Ann Yeh TI - Rituximab as a first-line preventive treatment in pediatric NMOSDs AID - 10.1212/NXI.0000000000000046 DP - 2014 Dec 01 TA - Neurology - Neuroimmunology Neuroinflammation PG - e46 VI - 1 IP - 4 4099 - http://nn.neurology.org/content/1/4/e46.short 4100 - http://nn.neurology.org/content/1/4/e46.full SO - Neurol Neuroimmunol Neuroinflamm2014 Dec 01; 1 AB - Objective: No established therapeutic protocol has been proposed to date for childhood-onset neuromyelitis optica (NMO) spectrum disorders (NMOSDs). We report the response of 5 NMO immunoglobulin (Ig)G–positive pediatric cases to a standardized B-cell–targeted first-line immunosuppressive protocol with rituximab for prevention of relapses.Methods: Retrospective observational cohort study.Results: All patients included in the study showed disease remission after rituximab induction. Relapses always occurred in conjunction with CD19+ B-cell repopulation and appeared less severe than prior to treatment. At the end of follow-up, neurologic disability and MRI findings stabilized or improved in all the patients, with only minor and transient side effects. Oral steroid discontinuation was possible in all the patients.Conclusions: Our protocol is well-tolerated and has provided encouraging results in terms of control of relapses and progression of disability. An early intervention with rituximab might affect the disease course in pediatric NMO-IgG–positive NMOSDs.Classification of evidence: This study provides Class IV evidence that for children with NMOSDs, rituximab is well-tolerated and stabilizes or improves neurologic disability.AQP4=aquaporin-4; EDSS=Expanded Disability Status Scale; Ig=immunoglobulin; NMO=neuromyelitis optica; NMOSD=neuromyelitis optica spectrum disorder