Short-term efficacy and tolerability of rufinamide adjunctive therapy in children with refractory generalised epilepsy

Abstract

We evaluated the efficacy and tolerability of rufinamide adjunctive therapy in children with refractory generalised epilepsy. The study cohort consisted of 20 patients with Lennox-Gastaut syndrome, 5 with Dravet syndrome, and 28 with unclassified refractory generalised epilepsy. Patients with more than 50% seizure reduction at three and six months were defined as responders. The overall response rate was 37.7% at three months and 34.0% at six months. At three months, patients with Lennox- Gastaut syndrome (40.0%) and epilepsy with spasms/tonic seizures (38.5%) showed higher response rates than those with Dravet syndrome (20.0%) and epilepsy with myoclonic seizures (20.0%). High response rates in patients with Lennox-Gastaut syndrome (30.0%) and epilepsy with spasms/tonic seizures (38.5%) were sustained throughout the six-month study. The accuracy of, and differences between, responder rates should, however, be interpreted with caution due to the small number of patients. Overall, rufinamide appeared to be effective and reasonablywell tolerated in this group of children with refractory generalised epilepsies, although a subgroup of patients with Dravet syndrome and epilepsy with myoclonic seizures were less responsive to rufinamide treatment.