Abstract

Objective Limited data are available on natalizumab in pediatric multiple sclerosis. This is to report the sustained therapeutic benefit of natalizumab over 32 months follow up in a 12-year old child with a breakthrough activity disease. Methods In February 2009, the diagnosis of childhood onset relapsing remitting multiple sclerosis was confirmed at age 9, based on the “International Pediatric Multiple Sclerosis Study Group”. A disease breakthrough activity with suboptimal response was considered after 26 months of a well tolerated treatment with beta-interferon-1b; 6 relapses, rapid neurological deterioration and an increase by 4 new plaques on average per year with at least one showing enhancement after gadolinium injection were observed on MRI follow up. Monthly natalizumab 300 mg intravenous infusion was initiated on February 2012, after written consent. Negative JCV antibodies from serum samples were confirmed using the “STRATIFT JCV” test before treatment initiation and at 6 months intervals; clinical evaluation and biological tests were performed on routine base every 3 months. Results The patient's neurological status improved dramatically with rapid recovery after the second natalizumab infusion. Presently, this 13-year-old patient has received a total of 32 infusions of natalizumab and is still asymptomatic with no occurrence of any clinical relapse or no new lesion. Treatment is still well tolerated. Conclusion This report highlights the tolerability and sustained therapeutic efficacy of natalizumab as second-line therapy in childhood multiple sclerosis with breakthrough disease activity when critical decision of shifting from first-line therapy has to be made in children with potential greater risk in reaching progressive neurological disability at younger age.

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