Efficacy, safety, and tolerability of adjunctive perampanel in the treatment of pediatric patients aged 4–18 years with epilepsy: A single-center, retrospective, observational real-world study

Abstract

Purpose

To observe the efficacy, safety, and tolerability of perampanel (PER) as add-on therapy in children aged 4–18 years with epilepsy in a real-world environment.

Methods

A single-center, retrospective, observational study was conducted at the First Affiliated Hospital of Fujian Medical University enrolling children with epilepsy aged 4–18 years who received PER as add-on therapy from January 2021 to November 2022 with 12 months of follow-up. Outcomes included 3-, 6- and 12-month retention, seizure freedom, responder rates, and adverse events (AEs) throughout follow-up.

Results

Seventy-eight children were included, of whom three were lost to follow-up. The responder rate at follow-up of 12 months was 54.7 %, the seizure-free rate was 32.0 % and the retention rate was 81.3 %. The number of seizures at baseline was a factor influencing the efficacy of the PER. Nine children reported AEs, with dizziness, drowsiness, and irritability being common.

Conclusions

PER is safe, effective, and well tolerated for the treatment of children aged 4–18 years with epilepsy in clinical practice and is a potential option for refractory epilepsy. Patients with lower baseline seizure frequencies are more likely to exhibit a favorable response to PER.