Real-world effectiveness and safety of perampanel for children and adolescents with epilepsy: A meta-analysis with at least 1-year follow-up

Background

Perampanel, the first third-generation anti-seizure medication targeting the AMPA receptor, has been used in the treatment of patients with focal seizures, with or without secondary generalized seizures, and primary generalized tonic-clonic seizures. This study focused on the effectiveness and safety of perampanel for pediatric patients with at least 1-year follow-up in real-world settings.

Methods

We systematically searched PubMed, EMBASE, and Web of Science for real-world studies published before April 27, 2024. The data of interest were extracted and analyzed using the R software (version 4.2.1).

Results

From 1181 retrieved citations, 25 records involved a total of 2985 individuals were included in the meta-analysis. The 50 % responder rate pooled from the 22 studies yielded an overall 55.0 % (95 % CI: 46.1–63.8 %), with significant evidence of between-study heterogeneity (I² = 93 %, P < 0.01, τ² = 0.038). The seizure-free rate pooled from 22 studies yielded an overall rate of 28.9 % (95 % CI: 19.6–39.1 %). Twenty studies reported the retention rate of perampanel treatment with a pooled proportion was 71.1 % (95 % CI: 61.1–80.2 %). The estimate of the adverse events incidence rate pooled from the 23 studies yielded an overall 29.0 % (95 % CI: 23.4–34.9 %). Subgroup analyses were conducted based on follow-up time (12 months or ≥ 24 months).

Conclusion

Perampanel is generally well tolerated and effective in the treatment of epilepsy in children and adolescents with mid-long-term follow-up. The 50 % responder rate in children and adolescents improved with time. The retention rate and the seizure-free rate during at least 24 months of follow-up were not as sustained as those in 12 months of follow-up. Adverse events, particularly psychiatric and behavioral, should be monitored during clinical practice administration.