(518) Effectiveness and Tolerability of Perampanel in Epilepsy Patients Treated in Routine Clinical Practice: a Global Pooled Analysis Study

Rationale: Perampanel (PER) is indicated in the U.S. for the treatment of focal-onset seizures in patients aged ≥ 4 years, and as adjunctive therapy for generalized-onset tonic-clonic seizures in patients aged ≥ 12 years. Real-world clinical practice data complement evidence from clinical trials by providing information on patients who are more diverse in terms of clinical characteristics than those recruited for clinical trials. The purpose of this study was to assess the real-world effectiveness, safety and tolerability of PER when used in everyday clinical practice.

Methods: 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 three, six, and 12 months of PER treatment. Effectiveness was assessed by seizure type (focal, generalized, status epilepticus) at the last visit. In patients with focal and/or generalized seizures, effectiveness assessments were seizure freedom rate (no seizures since at least the prior visit), 50% responder rate (≥ 50% seizure frequency reduction) and the proportions of patients with unchanged or worsening seizure frequency; in those with status epilepticus, effectiveness was assessed as responder rate (seizures under control). Safety and tolerability were assessed by evaluating adverse events (AEs), AEs leading to discontinuation, psychiatric AEs, and psychiatric AEs leading to discontinuation.  

Results: Data from 3,496 patients were included (50.5% female; mean age 41.0 years; mean epilepsy duration 25.4 years; mean number of previous antiepileptic drugs, 5.9). Seizure types at baseline were focal only (74.9%), generalized only (12.2%), focal and generalized (11.3%), and status epilepticus (1.6%). Most patients were treated with PER as adjunctive therapy: 3.2% and 2.3% of patients were treated with PER monotherapy at baseline and last visit, respectively. Mean (standard deviation) PER dosage was 2.4 (1.2) mg/day at initiation and 6.5 (2.6) mg/day at the last visit. Effectiveness and safety/tolerability were assessed for 2991 and 3006 patients, respectively. At three, six, and 12 months, overall retention rates were 90.7% (2893/3188), 78.6% (2484/3160) and 61.6% (1844/2994), respectively. Mean (95% confidence interval) time under PER treatment was 10.4 (10.3–10.6) months. At last visit, seizure freedom rates in patients with focal and generalized seizures were 13.9% and 47.6%, respectively, and 36.5% of patients with status epilepticus had responded to treatment (Table 1). AEs were reported for 55.4% of patients; the most frequently reported AEs were dizziness/vertigo (17.1%) and behavioral AEs (16.3%) (Table 2). Overall, 14.4% of patients discontinued due to AEs. Psychiatric AEs were reported for 22.5% of patients and led to discontinuation of 9.1% (Table 2). 

Conclusions: PER was effective and generally well tolerated when used to treat a large cohort of patients with focal and generalized epilepsy under everyday clinical practice conditions.

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