Natalizumab Use in Pediatric Multiple Sclerosis

Background: Natalizumab, a humanized monoclonal antibody raised against alpha(4) integrins, is approved for treatment of active relapsing-remitting multiple sclerosis (RRMS) in adult patients. Objective: To determine the safety, effectiveness, and tolerability of natalizumab use in pediatric patients with MS. Design: Case report. Setting: Center for MS in childhood and adolescents, Gottingen, Germany. Patients: Three pediatric patients with RRMS having a poor response to other immunomodulatory therapies or having intolerable adverse effects. Interventions: Natalizumab given every 4 weeks at a dosage of 3 to 5 mg/kg of body weight. Main Outcome Measures: Cranial magnetic resonance (MR) imaging before treatment and every 6 months thereafter. Results: During 24, 16, and 15 months of treatment, no further relapses occurred in the 3 pediatric patients; all reported significant improvement in their quality of life. Follow-up MR imaging showed no new T2-weighted lesions or gadolinium-enhancing lesions. No adverse events were seen when dosage was adjusted to body weight. Conclusions: Natalizumab treatment was effective and well tolerated in our pediatric patients with RRMS who did not respond to initial immunomodulatory treatments. Therefore, it is a promising second-line therapy for pediatric patients with RRMS.