Abstracts

Rationale: Currently there are more than 30 anti-epileptic drugs (AED) available on the market. New generation AEDs such as Perampanel have a promising outlook with novel mechanisms of action but clinical data, especially in the pediatric population, are scarce. Perampanel is FDA approved as an add-on therapy for focal seizures with or without secondary generalization and for primary generalized tonic-clonic seizures in patients with epilepsy aged ≥12 years. Perampanel is the first-in-class AED that antagonizes the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor. Reported efficacy rate in the literature is very variable (9%-89%).  Adverse events include dizziness, fatigue, headache, somnolence, irritability and psychiatric and behavioral symptoms, including hostility and homicidal/suicidal ideation. The objective of this study was to assess the efficacy, tolerability, and side effect profile of Perampanel in pediatric and young adult patients with epilepsy at our institution. Methods: We retrospectively reviewed the records of patients with refractory epilepsy treated with Perampanel over a period of 4 years. Efficacy and safety were evaluated based on parental reports and physician assessment in the electronic medical record.  Results: A total of 19 patients were included (12 F, 7 M). Age range at initiation of therapy was 5-19 years (mean 13.4 years). Seven (37%) were < 12 years old.  All patients had cognitive deficits ranging from mild to severe. Eight (42%) had generalized epilepsy, 4 (21%) mixed type, and 7 (37%) focal. Average number of concurrent AEDs at the start of Perampanel therapy was 2.8. Starting dose was 2 mg daily. Average maximum dose was 6.6 mg/day or 0.14 mg/kg/day. Average duration of Perampanel treatment was 8.8 months. Follow-up information was collected on 18 (95%) patients. Seven (37%) had >50% reduction in seizures. One (5%) experienced worsening of seizure frequency. Most commonly observed side effects were change in behavior/aggression (26%) and sedation/fatigue (21%). Most frequent cause of discontinuation was lack of response (47%). One patient (5%) had visual and tactile hallucinations. There was no statistical significance in efficacy or side effect rate between patients under and over 12 years of age. There were no cases of suicidal/homicidal ideation Conclusions: Based on our small series we conclude that Perampanel is an effective treatment of refractory epilepsy in pediatric patients, including those < 12 years. Behavior and psychiatric side effects should be monitored during therapy. Recent retrospective publications suggest the same findings, however data regarding efficacy, tolerability, and safety is still lacking in the medical literature, especially in children. Therefore, larger randomized studies in pediatric populations are necessary.  Funding: None