Natalizumab, a humanized monoclonal antibody raised against α4 integrins, is approved for treatment of active relapsing-remitting multiple sclerosis (RRMS) in adult patients.
To determine the safety, effectiveness, and tolerability of natalizumab use in pediatric patients with MS.
Center for MS in childhood and adolescents, Göttingen, Germany.
Three pediatric patients with RRMS having a poor response to other immunomodulatory therapies or having intolerable adverse effects.
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.
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.
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.