Safety and efficacy of natalizumab in children with multiple sclerosis
| Autor: | Mauro Zaffaroni, L.M.E. Grimaldi, F. Bortolon, M. Rottoli, G. Comi, Paola Sarchielli, Ruggero Capra, C. Pozzilli, Lucia Moiola, A. Ghezzi, Massimo Filippi, V. Brescia Morra, Maria A. Rocca |
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| Přispěvatelé: | Ghezzi, A, Pozzilli, C, Grimaldi, Lme, Morra, Vb, Bortolon, F, Capra, R, Filippi, Massimo, Moiola, L, Rocca, Ma, Rottoli, M, Sarchielli, P, Zaffaroni, M, Comi, Giancarlo, Grimaldi, Lm, BRESCIA MORRA, Vincenzo, Filippi, M, Comi, G. |
| Jazyk: | angličtina |
| Rok vydání: | 2010 |
| Předmět: |
Male
medicine.medical_specialty Multiple Sclerosis Adolescent Antibodies Monoclonal Humanized Severity of Illness Index Central nervous system disease Natalizumab Internal medicine medicine Humans Prospective Studies Child Expanded Disability Status Scale business.industry Multiple sclerosis Antibodies Monoclonal Brain Mean age medicine.disease Magnetic Resonance Imaging Surgery Treatment Outcome El Niño T2 lesions Female Neurology (clinical) business After treatment medicine.drug |
| Popis: | OBJECTIVE: To describe the effect of natalizumab in the treatment of subjects with active multiple sclerosis (MS) treated before the age of 18 years. METHODS: Nineteen pediatric subjects with MS (mean age 14.6 +/- 2.2 years, mean number of attacks 5.2 +/- 1.9 during the pretreatment phase of 27.7 +/- 19.7 months, median pretreatment Expanded Disability Status Scale score [EDSS] 2.5, range 1.0-5.0) were treated with natalizumab at the dose of 300 mg every 28 days. After treatment initiation, patients were reassessed clinically every month; brain MRI was performed at baseline and every 6 months. RESULTS: Patients received a median number of 15 infusions (range 6-26). A transient reversible worsening of preexisting symptoms occurred in 1 subject during and following the first infusion. All the patients remained relapse-free during the whole follow-up. The median EDSS decreased from 2.5 to 2.0 at the last visit (p < 0.001). EDSS remained stable in 5 cases, decreased by at least 0.5 point in 6 cases, and decreased by at least 1 point in 8 cases. At baseline, the mean number of gadolinium-enhancing lesions was 4.1 (range 1-20). During the follow-up, no gadolinium-enhancing lesions were detected (p = 0.008); 3 patients developed new T2-visible lesions at month 6 scan but the overall number of T2 lesions remained stable during the subsequent follow-up. Transient and mild side effects occurred in 8 patients. CONCLUSIONS: Natalizumab was well-tolerated in all subjects. A strong suppression of disease activity was observed in all subjects during the follow-up. Classification of evidence: This study provides Class IV evidence that natalizumab, 300 mg IV once every 28 days, decreased EDSS scores in pediatric patients with MS over a mean treatment period of 15.2 months. |
| Databáze: | OpenAIRE |
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