Rituximab as a first-line preventive treatment in pediatric NMOSDs

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.