Abstracts

Rationale: The aim of the study was to explore the long-term effectiveness and tolerability of rufinamide (RUF) in a heterogeneous group of patients with refractory epilepsies in Europe. Treatment was started immediately after the drug became available as an orphan drug for the adjunctive treatment of Lennox-Gastaut syndrome (LGS). Methods: This observational study was conducted as a collection of retrospective data from multiple centres in Germany and Austria. Clinical course in patients treated with RUF was documented. Initial dosage and titration schedule of RUF were at the discretion of the treating physician according to medical need. The observation period was 18 months. Safety and tolerability were evaluated by analysis of adverse events. Responders were defined as patients who achieved a 50% or higher decrease in countable seizures within the first 12 weeks after initiating RUF therapy (evaluated by comparison to baseline frequency). Retention rate was defined as the percentage of patients still taking RUF after 18 month. Results: The study population consisted of 45 children and 15 adults (mean age: 14.5 ± 11.6 years, range: 1-50) with various severe and inadequately controlled childhood-onset epilepsy syndromes, that is, LGS (n = 31), idiopathic generalized epilepsy syndromes (n = 5), cryptogenic unclassified generalized epilepsy (n = 7) and partial epilepsy (n = 17). Mean overall duration of RUF treatment was 48 weeks (range 4-72 weeks). The response rate after 12 weeks was 46.7% (28 of 60 patients) and 25.0% (15 of 60 patients) after 18 months. Complete seizure control was achieved by 8.3% after 12 weeks and was still continued in one patient (1.6%) after 18 months. Retention rate after 18 month was 41.7% (n=25). A total of 73 adverse events were reported by 37 of 60 patients. They mostly occurred during titration and subsided in maintenance phase. Adverse events occurring most frequently were fatigue (18.3%), vomiting (15.0%), and loss of appetite (10.0%). No serious adverse events were observed. Conclusions: The present data suggest that RUF may be effective and well tolerated in the long-term treatment of children and adults with various epilepsy syndromes and difficult-to-control seizures of childhood-onset. The results of our study suggest that the efficacy of RUF in patients with generalized epilepsy might be comparable to that in patients with LGS, whereas RUF was less effective in patients with partial epilepsy.