Natalizumab use in pediatric patients with relapsing-remitting multiple sclerosis

Background: Not all pediatric patients with relapsing-remitting multiple sclerosis (MS) may respond to traditional disease-modifying therapies. Natalizumab has been shown to be effective but is currently only approved in adults. Objective: To analyze the safety and efficacy of natalizumab in patients under 18 years of age diagnosed with MS. Method: Data for pediatric patients with MS treated with natalizumab in a compassionate use setting were retrospectively collected and analyzed. Results: Valid data were obtained for nine patients under 18 years from seven different centers (mean age, 15 years 4 months [range 9.8-17.7]; 5 were boys). Patients received a median of 17 infusions of natalizumab (range, 2-31) and eight received at least 12 infusions. For these 8 patients, the median score on the Expanded Disability Status Scale decreased from 3.0 to 1.0 and the median annualized relapse rate decreased from 3.0 to 0. After 12 months, no patients reported gadolinium-enhancing lesions compared to seven at baseline. Four post-baseline adverse events occurred and one patient discontinued due to hypersensitivity reaction. Conclusion: Natalizumab is a highly effective treatment as a second-line option in pediatric patients. In as far as the limited numbers allowed comparisons, the safety and efficacy of natalizumab in children was in line with the experience published in adult populations. (C) 2012 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.