Abstracts

Rationale: Clinical practice studies provide information on patients who are more diverse in terms of age and clinical characteristics than those recruited for clinical trials. Perampanel (PER) is a once-daily oral anti-seizure medication for focal-onset seizures, with or without focal to bilateral tonic-clonic seizures, and generalized tonic-clonic seizures. The purpose of this study was to assess PER when used in everyday clinical practice in patients aged < 12 years, by age category (< 4 years, 4–< 7 years and 7–< 12 years).

Methods: Pediatric patients (< 12 years) who were treated with PER were identified from a pooled analysis of 44 clinical practice studies/work groups. Retention was assessed after 3, 6 and 12 months. Effectiveness assessments comprised responder rate (≥50% seizure frequency reduction), seizure freedom rate (no seizures since at least the prior visit), 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. Data were analyzed by age category: < 4, 4–< 7 and 7–< 12 years.

Results: 56 pediatric patients were identified (< 4 years, n=5; 4–< 7 years; n=12; 7–< 12 years, n=39). Retention was assessed for 41 patients (< 4 years, n=2; 4–< 7 years; n=10; 7–< 12 years, n=29); effectiveness for 50 patients (< 4 years, n=3; 4–< 7 years; n=11; 7–< 12 years, n=36); and safety/tolerability for 47 patients (< 4 years, n=3; 4–< 7 years; n=10; 7–< 12 years, n=34). Mean (standard deviation) PER doses at baseline and the last visit (last observation carried forward) in patients aged < 4 years were 1.8 (0.5) and 2.3 (1.5) mg/day, respectively; corresponding values for patients aged 4–< 7 and 7–< 12 years were 1.8 (0.5) and 4.1 (1.8) mg/day, and 2.1 (0.5) and 4.9 (2.9) mg/day, respectively. Retention rates at 3, 6 and 12 months in patients aged < 4 years were 50.0% (1/2), 50.0% (1/2) and 50.0% (1/2), respectively; corresponding rates for patients aged 4–< 7 and 7–< 12 years were 90.0% (9/10), 70.0% (7/10) and 0% (0/5), and 89.7% (26/29), 76.9% (20/26) and 63.6% (14/22), respectively. Mean (95% confidence interval) times under PER treatment were 6.8 (0–14.0), 4.9 (4.1–5.8) and 9.5 (7.9–11.0) months in patients aged < 4, 4–< 7 and 7–< 12 years, respectively. At the last visit, responder and seizure freedom rates in patients aged < 4 years were 66.7% and 33.3%, respectively; corresponding rates for patients aged 4–< 7 and 7–< 12 years were 36.4% and 9.1%, and 58.3% and 27.8%, respectively (Figure). AEs were reported for 0% (0/3), 40.0% (4/10) and 38.2% (13/34) of patients aged < 4, 4–< 7 and 7–< 12 years, respectively (Table). The most frequently reported AEs were irritability (< 4 years, 0% [0/3]; 4–< 7 years, 10% [1/10]; 7–< 12 years, 14.7% [5/34]) and dizziness/vertigo (< 4 years, 0% [0/3]; 4–< 7 years, 0% [0/10]; 7–< 12 years, 8.8% [3/34]).

Conclusions: PER was effective and generally well tolerated when used to treat this small population of patients aged < 4, 4–< 7 and 7–< 12 years in everyday clinical practice.