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.
To evaluate safety and efficacy of natalizumab (NTZ) in naïve POMS.
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.
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.
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.
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Conflicts of interest
Monica Margoni reports grants from Teva, grants from Genzyme Sanofi, grants from Merck Serono, grants from Biogen Idec, grants and personal fees from Novartis, during the conduct of the study. Francesca Rinaldi reports grants from Almirall, grants and personal fees from Teva, grants and personal fees from Genzyme Sanofi, grants and personal fees from Merck Serono, grants and personal fees from Biogen Idec, grants from Novartis, during the conduct of the study. Alice Riccardi has nothing to disclose. Silvia Franciotta has nothing to disclose. Paola Perini reports grants and personal fees from Merck Serono, grants and personal fees from Biogen Idec, grants and personal fees from Genzyme Sanofi, grants and personal fees from Bayer Schering Pharma, grants and personal fees from Novartis, grants and personal fees from Teva, during the conduct of the study. Paolo Gallo reports grants and personal fees from Merck Serono, grants and personal fees from Biogen Idec, grants and personal fees from Genzyme Sanofi, grants and personal fees from Bayer Schering Pharma, grants and personal fees from Novartis, grants and personal fees from Teva, grants from University of Padua, Department of Neurosciences DNS, grants from Veneto Region of Italy, grants from Italian Association for Multiple Sclerosis (AISM), grants from Italian Ministry of Public Health, during the conduct of the study.
This study was approved by the local Ethics Committee and was performed according to the Declaration of Helsinki. Participants received an explanatory statement and gave their written informed consent to participate in the study.
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Margoni, M., Rinaldi, F., Riccardi, A. et al. No evidence of disease activity including cognition (NEDA-3 plus) in naïve pediatric multiple sclerosis patients treated with natalizumab. J Neurol 267, 100–105 (2020). https://doi.org/10.1007/s00415-019-09554-z
- Pediatric-onset multiple sclerosis
- NEDA-3 plus