Objective: To evaluate the safety, efficacy and tolerability of natalizumab as a first-line treatment in pediatric multiple sclerosis (MS) patients with highly active disease.
Background: Up to 30% of children and juveniles with MS experience a breakthrough disease requiring therapies beyond traditional first-line agents. Natalizumab, a humanized monoclonal antibody against alpha4 integrin is approved for treatment of active relapsing-remitting MS in adult patients.
Design/Methods: Patients with onset of MS before the age of 21 years who experienced highly active disease were treated with natalizumab as a first line therapy. Highly active disease was defined as: (1) two relapses within 6 months from disease onset, or (2) a severe relapse within 6 months from disease onset with incomplete recovery, and (3) at least three Gd enhancing brain lesions, and at least one spinal cord lesion.
Outcome measures included annualized relapse rate, neurological disability by the Expanded Disability Status Scale (EDSS), number of Gd-enhancing lesions on brain and spinal cord MRI, adverse events and serum JC virus-antibody status.
Results: Twenty-nine highly active pediatric MS patients were included, 17 females, and 12 males. Mean age at initiation of natalizumab treatment was 17.2 +0.61 years (range 9 to 21 years), disease duration 2.3 +0.4 years, and EDSS at treatment initiation 1.6 +0.22. Treatment was well tolerated without serious adverse events (except of one patient that developed Molluscum contagiosum ).
Conclusions: Natalizumab is well tolerated and effective as first-line treatment in highly active pediatric MS patients.
