Abstracts

Rationale: To explore the effectiveness and tolerability of perampanel (PER) in a heterogeneous group of patients under 18 years of age with refractory epilepsies.Methods: This non-sponsored observational survey was conducted as a collection of retrospective data from multiple centers in Germany, Austria and Denmark. Clinical course of the first patients treated in these centers with PER was documented. Initial dosage and titration schedule of PER were at the discretion of the treating physician according to medical need. Effectiveness was evaluated by comparing the frequency of seizures between baseline and a 4-week-period 3 months after initiation. Observation is ongoing and planned until an 18-months follow-up. Results: The study population consisted of 47 children (mean age: 10.9 years, range: 2-17, male: 23), with various refractory epilepsies classified as focal epilepsy (n=27), generalized epilepsy (n=13), LGS (n=3), Dravet Syndrome (n=2), and others (n=2). The response rate (50% seizure reduction) after 3 months of therapy was 27.6% (13 of 47 patients) in total. Of all patients, 10.6 % (n=5) experienced a 50-75% reduction and 8.5 % (n=4) experienced a more than 75% reduction in seizure frequency. Complete seizure control was achieved in 8.5 % (n=4). Adverse events during the first 3 months of follow-up were reported in 25 cases, mostly mild to moderate, with remission spontaneously or with decreasing the PER dosage. The most frequent adverse events were fatigue (n=11), agitation (n=10), changes in appetite (n=5), and dizziness (n=4). Severe adverse events (severe behavioral changes, fatigue, psychomotor slowing, diarrhea-vomiting) occurred in 5 cases.Conclusions: These preliminary data suggest that PER may be effective in the treatment of children and adolescents with refractory epilepsies of different etiologies. There is a lack of pharmacokinetic data in young patients. Slower titration rates may reduce side effects. Longer follow-up of our patients and further investigations are needed.