Rufinamide in refractory childhood epileptic encephalopathies other than Lennox-Gastaut syndrome.

Abstract

Background: To report on the first multicenter Italian experience with rufinamide as adjunctive drug in children, adolescents and young adults with refractory childhoodonset epileptic encephalopathies other than Lennox–Gastaut syndrome. Methods: Thirty-eight patients (19 males, 19 females), aged between 4 and 34 (mean 13.7 ± 8.3, median 12.5), all affected by different types of childhood-onset refractory epileptic encephalopathies other than Lennox–Gastaut syndrome, were treated with rufinamide as adjunctive drug for a mean period of 11.4 months (range 3–26 months). Results: Fifteen of 38 patients (39.5%) had a ‡50% seizure reduction in countable seizures. Complete seizure freedom was achieved in one of these patients (2.6%). Three patients (7.9%) had a 25–49% seizure reduction, whilst seizure frequency remained unchanged in 15 (39.5%) and increased in five patients (13.1%). Eleven patients (28.9%) reported adverse side effects. Vomiting was reported in five patients (13.1%); drowsiness, decreased appetite and irritability with migraine manifested in other four patients. They were transient and mild in all cases. Conclusion: Rufinamide may be an effective and well-tolerated adjunctive drug for the treatment of refractory childhood-onset epileptic encephalopathies other than Lennox–Gastaut syndrome. Rufinamide was most effective in patients with dropattacks and (bi)frontal spike–wave discharges. Introduction Rufinamide is a structurally triazole-derivative (1-[2,6- difluorophenyl)methyl]-1hydro-1,2,3-triazole-carboxamide) novel antiepileptic drug, structurally unrelated to the existing antiepileptic drugs, and approved by the Food and Drug Administration for the treatment of Lennox–Gastaut syndrome in patients aged 4 and over, and for the treatment of partial seizures in adults and adolescents. The proposed mechanism of action is the limitation of excessiv