Abstracts

Rationale: Perampanel (PER) has been approved by FDA as an adjunctive therapy for the treatment of partial-onset seizures and primary generalized tonic-clonic seizures in patients with epilepsy who are 12 years and older. However, post-marketing information on the effect of PER on seizure frequency and its safety profile are scarce. The current study aims to evaluate the effectiveness and safety of PER in pediatric epilepsy patients. Methods: We retrospectively reviewed medical records of patients from January 2014 until September 2016 who were treated with PER at Boston Children’s Hospital. Seizure frequency before and after treatment was collected. Response to treatment was defined as more than 50% reduction in seizure frequency compared to baseline frequency. Other variables included in data collection were: demographic data, PER administration date, adverse events, dosage information, EEG and imaging findings, etc. Patients who did not have seizure frequency data at baseline visit, or did not have seizure frequency data for at least one follow up visit after PER administration were excluded from the analysis. Results: 57 patients were identified primarily, from whom 5 patients were excluded because PER was not administered for them, and 14 patients were excluded from the analysis due to pending follow up data. Therefore 38 patients (20 females, 52%) were included in the analysis. Age ranged from 1 to 24 years (median=15 years, IQR= 9-20). 25 (65%) patients had generalized epilepsy, 5 (13%) had focal epilepsy and 8 (21%) had combined epilepsy types. Median follow-up duration was 8.1 months. Seizure frequency decreased significantly after treatment with PER (p=0.003, Table1). 13 patients (34%) responded to treatment, with mean seizure reduction of 74.6% among responders (range= 52%-100%). Sub-group analysis shows significant reduction in seizure frequency for patients with generalized epilepsy, and for patients with genetic etiology (Table 1). Complete seizure freedom was achieved in 5 patients (13%). 17 (32%) patients discontinued PER during the follow up period due to adverse effects (8 patients, 15%), unsatisfactory response (7, 13%) or a combination of both (2, 4%). Most common adverse effect was behavioral change (10, 19%), followed by somnolence (8, 15%), and dizziness (4, 7%). Conclusions: Treatment with PER in pediatric patients with epilepsy is effective, and overall well-tolerated. Further sub-analysis in larger subpopulations are needed to evaluate differential response to different seizure types. Funding: This study was funded by Eisai Inc.