Natalizumab in pediatric multiple sclerosis: results of a cohort of 55 cases.

BACKGROUND

Limited information is available on the use of natalizumab (NA) in pediatric multiple sclerosis (ped-MS) patients.

OBJECTIVE

The purpose of this study was to describe the long-term effects of NA in a large cohort of active ped-MS patients.

METHODS

Patients with definite ped-MS were treated with NA if in the previous year they had experienced at least two relapses or a severe relapse with incomplete recovery while on immunomodulating treatment, or at least two relapses and new magnetic resonance imaging (MRI) lesions regardless of any prior treatment.

RESULTS

The study included 55 patients (mean age: 14.4 years, mean number of relapses: 4.4, pre-treatment mean disease duration: 25.5 months). They received a median number of 26 infusions. Three relapses occurred during the follow-up, one female patient continued to deteriorate in cognitive functioning. Mean Expanded Disability Status Scale (EDSS) scores decreased from 2.7 to 1.9 at the last visit (p<0.001). During the follow-up the majority of patients remained free from MRI activity. Transient and mild clinical adverse events occurred in 20 patients. Mild hematological abnormalities occurred in seven patients. Anti-JCV antibodies were detected in 20/51 tested patients.

CONCLUSIONS

NA was well tolerated in all patients. A strong suppression of disease activity was observed in the majority of patients during the follow-up.