Rituximab as a first-line preventive treatment in pediatric NMOSDs: Preliminary results in 5 children

Giulia Longoni; Brenda Banwell; Massimo Filippi; E Ann Yeh

doi:10.1212/NXI.0000000000000046

Rituximab as a first-line preventive treatment in pediatric NMOSDs: Preliminary results in 5 children

Neurol Neuroimmunol Neuroinflamm. 2014 Dec 11;1(4):e46. doi: 10.1212/NXI.0000000000000046. eCollection 2014 Dec.

Authors

Giulia Longoni¹, Brenda Banwell¹, Massimo Filippi¹, E Ann Yeh¹

Affiliation

¹ Neuroimaging Research Unit, Institute of Experimental Neurology, and Department of Neurology, Division of Neuroscience (G.L., M.F.), San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy; Department of Pediatrics (G.L., E.A.Y.), University of Toronto, and Division of Neurology (G.L., E.A.Y.), The Hospital for Sick Children, Toronto, Ontario, Canada; and Division of Neurology (B.B.), Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PA.

Abstract

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.