Abstracts

Rationale:
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. Perampanel (PER) is indicated in the US 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 as monotherapy in everyday clinical practice.
Method:
Patients treated with PER monotherapy for focal and/or generalized seizures were identified from an 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 the last visit. Effectiveness assessments comprised seizure freedom rate (no seizures since at least the prior visit), responder rate (≥ 50% seizure frequency reduction), and proportions of patients with unchanged or worsening seizure frequency. Safety and tolerability were assessed by evaluating adverse events (AEs), AEs leading to discontinuation, psychiatric AEs, and psychiatric AEs leading to discontinuation.  
Results:
A total of 111 patients treated with PER monotherapy at baseline (first line or conversion to monotherapy) for focal and/or generalized seizures 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%). The mean (standard deviation [SD]; range) number of antiepileptic drugs (AEDs) patients received prior to initiating PER monotherapy was 4.0 (4.0; 0–15). Mean (SD) PER dosage was 2.0 (0.0) mg/day at baseline and 6.5 (2.5) mg/day at the last visit. At the last visit, 54.2% of patients were being treated with concomitant AEDs. 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% (17/18), 88.9% (16/18) and 55.6% (10/18), respectively. Mean (95% confidence interval) time under PER treatment was 9.5 (7.8–11.2) months. At last visit, seizure freedom rates in patients with focal and generalized seizures were 20.0% and 47.6%, respectively, and corresponding 50% responder rates were 50.0% and 100.0%, respectively (Figure 1). AEs were reported for 60.5% of patients; the most frequently reported AEs were behavioral AEs (aggression/anger/irritability; 26.3%) and dizziness/vertigo (21.1%) (Table 1). Overall, 11.1% of patients discontinued due to AEs. Psychiatric AEs were reported for 28.9% of patients and led to discontinuation of one patient (2.6%) (Table 1). 
Conclusion:
PER was effective and generally well tolerated when used as monotherapy to treat patients with focal and/or generalized seizures in everyday clinical practice. PER was particularly effective in treating generalized seizures.
Funding:
:Study supported by Eisai.