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.
Natalizumab in pediatric multiple sclerosis: results of a cohort of 55 cases / Ghezzi, A; Zaffaroni, M; Bianchi, A; Pozzilli, C; Prosperini, L; Borriello, G; Filippi, M; Moiola, L; Gerevini, S; Rocca, Ma; Martinelli, V; Comi, G; BRESCIA MORRA, Vincenzo; Lanzillo, Roberta; Grimaldi, Lm; Bucello, S; Lus, G; Rinaldi, F; Gallo, P; Trojano, M; Provinciali, L; Pucci, E; Bortolon, F; Capra, R; Coniglio, G; Gasperini, C; Lugaresi, A; Pietrolongo, E; Farina, D; Di Ioia, M; Milani, N; Rottoli, Mr; Sarchielli, P.. - In: MULTIPLE SCLEROSIS. - ISSN 1352-4585. - (2013). [10.1177/1352458512471878]
Natalizumab in pediatric multiple sclerosis: results of a cohort of 55 cases.
Martinelli, V;BRESCIA MORRA, VINCENZO;LANZILLO, ROBERTA;
2013
Abstract
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
