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

PER is indicated in the US for treatment of focal-onset seizures (patients aged ≥4 years) and as adjunctive therapy for generalized-onset tonic-clonic seizures (patients aged ≥12 years). Information on using PER monotherapy in clinical practice is currently limited.

Patients treated with PER monotherapy for focal and/or generalized seizures were identified from interim pooled analysis of data from 18 clinical practice studies/work groups. Retention was assessed after 3, 6 and 12 months of PER treatment. Effectiveness was assessed by seizure type at last visit. Effectiveness assessments included seizure freedom rate (no seizures since at least prior visit) and responder rate (≥50% seizure frequency reduction). Safety and tolerability were assessed by evaluating adverse events (AEs).  

A total of 111 patients treated with PER monotherapy at baseline were identified (51.4% male; mean age, 32.1 years; mean epilepsy duration, 14.3 years). Seizure types at baseline were focal only (54.7%), generalized only (42.2%), and focal and generalized (3.1%). At last visit, 54.2% of patients were being treated with concomitant antiseizure medications. Effectiveness was assessed for 31 patients; safety and tolerability were assessed for 38 patients. At 3, 6 and 12 months, retention rates were 94.4%, 88.9% and 55.6%, respectively. Mean time under PER treatment was 9.5 months. At last visit, seizure freedom rates in patients with focal and generalized seizures were 20.0% and 47.6%, respectively; corresponding 50% responder rates were 50.0% and 100.0%, respectively. AEs were reported for 60.5% of patients; most common AEs were behavioral AEs (aggression/anger/irritability; 26.3%) and dizziness/vertigo (21.1%). Overall, 11.1% of patients discontinued due to AEs. 

PER monotherapy was effective and generally well tolerated in patients with focal and/or generalized seizures treated in clinical practice.