Abstracts

Rationale:
Real-world studies complement the evidence collected in clinical trials by providing data on a drug when used in everyday clinical practice, outside the relative restrictions of 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. In this pooled analysis of real-world studies, we evaluated the effectiveness and safety profile of PER in adult epilepsy patients treated in clinical practice.
Method:
An interim pooled analysis was conducted of real-world studies that have collected data on patients treated with PER for focal and/or generalized seizures. Retention rate was evaluated at 3, 6 and 12 months. Effectiveness was assessed by evaluating seizure freedom rate (no reported seizures since at least the prior visit) and 50% responder rate (≥ 50% reduction in seizure frequency); in patients with status epilepticus, effectiveness was assessed as responder rate (seizures under control). Tolerability was assessed by evaluating adverse events (AEs) and AEs leading to discontinuation. Here we report the results for the subgroup of adult patients aged ≥ 18 to < 65 years.
Results:
Data from 18 studies/work groups were pooled: of the 3496 patients included in the interim analysis, 2922 were aged ≥ 18 to < 65 years (50.8% female; mean age 39.5 years). Patients had focal seizures (74.7%), generalized seizures (12.2%), both focal and generalized seizures (12.2%) and status epilepticus (0.9%). At treatment initiation the majority of patients were on 2 or 3 concomitant AEDs (only 1.3% initiated PER as monotherapy). The mean (standard deviation) PER dosage was 2.3 (1.1) mg/day at baseline and 6.6 (2.6) mg/day at last visit. Effectiveness was assessed for 2579 patients; safety/tolerability was assessed for 2570 patients. Retention rates at 3, 6, and 12 months were 91.2% (2504/2747), 79.4% (2160/2721) and 62.3% (1613/2588), respectively. Mean retention time on PER treatment was 10.3 months. Overall, 995 (36.2%) patients discontinued PER: 7.9% due to lack of efficacy, 13.9% due to AEs and 3.4% due to both lack of efficacy and AEs. Among patients with focal seizures only, seizure freedom and responder rates at last visit were 12.3% and 43.4%, respectively. Among patients with generalized seizures only, seizure freedom and responder rates at last visit were 47.4% and 76.0%, respectively (Figure 1). Among patients with status epilepticus, 40.7% responded to treatment. A total of 1425 patients (55.4%) experienced AEs. The most common AEs were dizziness/vertigo (17.6%), behavioral AEs (15.7%), and somnolence (12.3%). Psychiatric AEs were reported for 22.1% of patients and led to discontinuation in 8.7% of patients (Table 1).
Conclusion:
In this analysis of real-world studies, PER was effective in controlling focal and generalized seizures in adult patients. PER was also generally well tolerated in this patient population.
Funding:
:Study supported by Eisai
FIGURES
Figure 1