Background: Pediatric-onset multiple sclerosis (POMS) is characterized by high inflammatory activity, aggressive course and early development of physical and cognitive disability. A highly effective early treatment must be considered in POMS.
Objective: To evaluate safety and efficacy of natalizumab (NTZ) in naïve POMS.
Methods: 20 naïve POMS (13F, 7 M; mean age: 13.8 ± 2.7 years) were treated with NTZ for at least 24 months (mean number of infusions: 42 ± 20). No evidence of disease activity (NEDA)-3 plus status, i.e., no relapse, no disease progression (EDSS score), no radiological activity and no cognitive decline, was evaluated.
Results: After 2 years of NTZ treatment, a significant reduction in the mean EDSS score (p < 0.0001) was observed in the whole cohort. During the follow-up, evidence of disease activity on MRI was observed in two patients (10%) and a mild decline in cognition was observed in other two. No patient had clinical relapse. At the time of last visit NEDA-3 plus status was maintained in 16 (80%) patients. No major adverse event was observed.
Conclusion: Early treatment of aggressive POMS with NTZ proved to be highly effective in achieving and maintaining the NEDA-3 plus status. Our data support the use of NTZ as first treatment choice in POMS.
Keywords: NEDA-3 plus; Natalizumab; Pediatric-onset multiple sclerosis.