Effectiveness and safety of perampanel for pediatric patients with epilepsy: A real-world study from China
Xiaohui Wang1, Taoyun Ji2, Maomao Liu3
1Department of Neurology Beijing Children's Hospital Capital Medical University, National Center for Children's Health Beijing China.
Insights
Perampanel (PER) effectively reduced seizure frequency in Chinese pediatric epilepsy patients, showing good retention rates. The study found PER safe for adjunctive therapy in children with refractory epilepsy.
Area of Science:
- Pediatric Neurology
- Epileptology
- Clinical Pharmacology
Background:
- Limited data exists on perampanel (PER) use in Chinese children, especially those with refractory epilepsy.
- Perampanel is an adjunctive therapy for epilepsy, but its use in pediatric populations is less established than in adults.
Purpose of the Study:
- To evaluate the effectiveness, retention, and safety of perampanel (PER) in Chinese children and adolescents with epilepsy.
- To address the lack of large-scale clinical data for PER in this demographic, particularly for refractory cases.
Main Methods:
- A multicenter, prospective, observational study.
- Enrolled 240 children and adolescents receiving PER as adjunctive therapy.
- Assessed seizure frequency reduction (primary endpoint), retention rates, seizure freedom, and treatment-emergent adverse events (TEAEs) over 6 months.
Main Results:
- 70.4% of patients achieved ≥50% seizure frequency reduction after 6 months.
- 22.1% of patients became seizure-free, with a retention rate of 90.2%.
- 89 patients (37.1%) experienced TEAEs, leading to discontinuation in 7 cases; no severe TEAEs were reported.
Conclusions:
- Perampanel (PER) demonstrates good effectiveness and retention in Chinese pediatric epilepsy patients under routine clinical settings.
- PER is a viable and safe adjunctive treatment option for children and adolescents with epilepsy, including those with refractory epilepsy.
Importance:
Perampanel (PER) is used less extensively in children than in adults. Currently, there is a lack of data from PER clinical studies with large sample sizes in Chinese children and adolescents with epilepsy, especially those with refractory epilepsy.
Objective:
To evaluate the effectiveness, retention, and safety of PER in the treatment of children and adolescents with epilepsy in China.
Methods:
This was a multicenter, prospective, observational study. Children and adolescents with epilepsy who received PER as adjunctive therapy were included. The primary effectiveness endpoint was the proportion of patients achieving a ≥50% reduction in seizure frequency after 6 months of treatment compared to baseline. The secondary effectiveness endpoints included retention and seizure-free rates. The safety outcome was the incidence of treatment-emergent adverse events (TEAEs).
Results:
A total of 240 patients with epilepsy were enrolled in the study. Prior to initiating PER treatment, approximately 87.9% of them took two or more antiseizure medications. After a 6-month treatment regimen with PER, 70.4% of the patients experienced a reduced seizure frequency of at least 50%, and 22.1% achieved complete seizure freedom. The retention rate was 90.2%. TEAEs were reported by 89 patients, leading to the discontinuation of PER in seven cases. No severe TEAEs were observed in this study.
Interpretation:
Under routine clinical conditions, PER demonstrated good effectiveness and retention in Chinese children with epilepsy, particularly in those with refractory epilepsy.
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