To assess real-world effectiveness, safety and tolerability of perampanel (PER) in everyday clinical practice.

PER is indicated in the US for treatment of focal-onset seizures (patients aged ≥4 years) and as an adjunctive therapy for generalized-onset tonic-clonic seizures (patients aged ≥12 years). Real-world clinical practice data provide information on patients who are more diverse than those in clinical trials.

An interim pooled analysis was conducted of real-world data from 18 studies/work groups in which patients with focal and generalized epilepsy were treated with PER. Retention was assessed after 3, 6 and 12 months of PER treatment. Effectiveness was assessed by seizure type (focal, generalized, status epilepticus) at last visit. In patients with focal and/or generalized seizures, effectiveness assessments included seizure freedom rate (no seizures since at least prior visit) and 50% responder rate (≥50% seizure frequency reduction); in those with status epilepticus, effectiveness was assessed as responder rate (seizures under control). Safety and tolerability were assessed by evaluating adverse events (AEs).  

Data from 3496 patients were included (50.5% female; mean age 41.0 years; mean epilepsy duration 25.4 years). Seizure types at baseline were focal only (74.9%), generalized only (12.2%), focal and generalized (11.3%), and status epilepticus (1.6%). At 3, 6 and 12 months, overall retention rates were 90.7%, 78.6% and 61.6%, respectively. Mean time under PER treatment was 10.4 months. At last visit, seizure freedom rates were 13.9% (focal seizures) and 47.6% (generalized seizures); 36.5% of patients with status epilepticus had responded to treatment. AEs were reported for 55.4% of patients (most frequent: dizziness/vertigo [17.1%]; behavioral AEs [16.3%]). Overall, 14.4% of patients discontinued due to AEs.    

PER was effective and generally well tolerated in a large cohort of patients with focal and generalized epilepsy treated in clinical practice.