Abstracts

Rationale: Real-world clinical practice data complement evidence from clinical trials by providing information on patients who are more diverse than those recruited for clinical trials. Perampanel (PER) is a once-daily oral anti-seizure medication (ASM) for focal-onset seizures, with or without focal to bilateral tonic-clonic seizures, and generalized tonic-clonic seizures. This study assessed the real-world effectiveness, safety and tolerability of PER when used as monotherapy in everyday clinical practice.

Methods: Patients treated with PER monotherapy (first-line or conversion to monotherapy) for focal-onset and/or generalized-onset seizures were identified from a pooled analysis of 44 prospective, retrospective and cross-sectional clinical practice studies/work groups. Retention was assessed after 3, 6 and 12 months of PER treatment. Effectiveness was assessed by seizure type (focal-onset or generalized-onset) at the last visit (last observation carried forward). Effectiveness assessments comprised seizure freedom rate (no seizures since at least the prior visit), responder rate (≥ 50% seizure frequency reduction), and the proportions of patients with unchanged or worsening seizure frequency. Safety and tolerability were assessed by evaluating adverse events (AEs), psychiatric AEs, and AEs leading to discontinuation.

Results: A total of 268 patients were identified who were treated with PER monotherapy at baseline (51.1% female; mean age, 44 years; mean epilepsy duration, 13.2 years). Seizure types at baseline were focal-onset only (75.0%), generalized-onset only (24.5%), and focal-onset and generalized-onset (0.5%). Mean (standard deviation [SD]) PER doses at baseline and last visit were 3.0 (1.4) and 5.5 (2.2) mg/day, respectively. The mean (SD; range) number of ASMs patients received prior to initiating PER monotherapy was 2.7 (2.8; 0–15). At the last visit, 30.8% of patients were being treated with concomitant ASMs. Retention was assessed for 168 patients; effectiveness for 183 patients; and safety and tolerability for 197 patients. At 3, 6 and 12 months, retention rates were 91.1% (153/168), 87.3% (138/158) and 73.3% (96/131), respectively. Mean (95% confidence interval) time under PER treatment was 11.8 (11.2–12.5) months. At last visit, seizure freedom rates in patients with focal-onset and generalized-onset seizures were 64.1% and 69.4%, respectively, and corresponding responder rates were 84.4% and 93.9%, respectively (Figure). AEs were reported for 45.2% of patients; the most frequently reported AEs (≥10% of patients) were dizziness/vertigo (16.8%) and irritability (11.2%) (Table). Overall, 13.7% of patients discontinued due to AEs over 12 months. Psychiatric AEs were reported for 20.8% of patients and led to discontinuation of 10.2% of patients.

Conclusions: PER was effective and generally well tolerated when used as monotherapy in patients with focal-onset and/or generalized-onset seizures in everyday clinical practice. At the last visit, approximately two-thirds of patients were seizure free.

Funding: Please list any funding that was received in support of this abstract.: Supported by Eisai.