Monica Margoni1,2, Francesca Rinaldi3, Alice Riccardi3, Silvia Franciotta3, Paola Perini3, Paolo Gallo3,4. 1. Multiple Sclerosis Centre of the Veneto Region (CeSMuV), University Hospital of Padua, Padua, Italy. monicamargoni@hotmail.com. 2. Padova Neuroscience Centre (PNC), University of Padua, Padua, Italy. monicamargoni@hotmail.com. 3. Multiple Sclerosis Centre of the Veneto Region (CeSMuV), University Hospital of Padua, Padua, Italy. 4. Department of Neurosciences, Medical School, University of Padua, Padua, Italy.
Abstract
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.
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.
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