Neurologist Epilepsy Centre, Department of Neuroscience, Odontostomatological and Reproductive Sciences, Federico II University of Naples, Naples, Italy
Rationale: Patients with epilepsy with tumor etiology are typically excluded from clinical trials. Real-world evidence from clinical practice studies is therefore required in order to inform treatment decisions in this patient group. Perampanel (PER) is indicated in the U.S. for the treatment of focal-onset seizures in patients aged ≥ 4 years, and as adjunctive therapy in the treatment of generalized-onset tonic-clonic seizures in patients aged ≥ 12 years. The purpose of this study was to assess the real-world effectiveness, safety and tolerability of PER when used in everyday clinical practice to treat patients with epilepsy with a tumor etiology. Methods: Patients with epilepsy with tumor etiology were identified from an interim pooled analysis of 18 clinical practice studies/work groups in which patients with focal and generalized seizures 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 the last visit. Effectiveness assessments included 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 AEs leading to discontinuation. Results: A total of 51 patients with focal and/or generalized seizures with a tumor etiology were identified (mean age, 49.7 years; 50.0% male; mean duration of epilepsy, 6.9 years). Seizure types at baseline were focal only (82.4%), generalized only (5.9%), and focal and generalized (11.8%); six of these patients (11.8%) were treated because of status epilepticus. All patients received PER as adjunctive therapy. Mean (standard deviation) PER dosage was 3.0 (1.9) mg/day at baseline and 6.3 (2.2) mg/day at the last visit. Effectiveness was assessed for 49 patients; safety/tolerability was assessed for 39 patients. At 3, 6 and 12 months, retention rates were 99.7% (43/44), 90.5% (38/42) and 79.5% (31/39), respectively. Mean (95% confidence interval) time under PER treatment was 10.7 (9.9–11.6) months. At the last visit, seizure freedom rates in patients with focal and generalized seizures were 37.5% and 33.3%, respectively, and corresponding values for 50% responder rate were 85.0% and 100%, respectively (Figure 1). Of the 6 patients with status epilepticus, 2 (33.3%) responded to treatment. AEs were reported for 38.5% of patients; the most frequently reported AEs were dizziness/vertigo (12.8%) and somnolence (12.8%); 6.8% of patients discontinued due to AEs (Table 1). Psychiatric AEs were reported for 10.3% of patients (Table 1). Conclusions: PER was effective and generally well tolerated when used to treat patients with epilepsy with tumor etiology in clinical practice.