Effectiveness and safety of perampanel in Chinese paediatric patients (2‐14 years) with refractory epilepsy: a retrospective, observational study

Abstract

Objective. We report our clinical experience of the effectiveness and tolerability of adjunctive perampanel treatment in a Chinese paediatric population with refractory epilepsy. We also compare the effectiveness and tolerability of perampanel with or without concomitant oxcarbazepine or levetiracetam. Methods. This retrospective observational study was conducted from September 2019 to September 2020 in the paediatric neurology clinics of two tertiary hospitals in the Chinese mainland. We reviewed the data obtained from 96 paediatric patients aged 2‐14 years whose seizures were pharmacoresistant and who could be followed up for a minimum of six months. The effectiveness was estimated by the perampanel response rate at 6‐ and 12‐month follow‐up evaluations. Adverse events were also recorded. Patients were stratified by age (2‐7 and 7‐14 years), and with/without concomitant oxcarbazepine or levetiracetam. Results. The population comprised 96 patients with refractory epilepsy. The retention rate was 84.4% and 81.0% at 6 and 12 months, respectively. The most common dose used was 4 mg (48.5%). Corresponding 50% responder and seizure freedom rates were as follows: 46.9% and 20.8% at six months, and 51.2% and 27.4% at 12 months, respectively. Adverse effects were reported in 22 patients (22.9%). The most common adverse effects were irritability, somnolence, and dizziness. The 50% and 100% responder rates and adverse effects were greater in patients aged 7‐14 years than in those aged 2‐7 years. The proportion of responder and seizure‐free rates and adverse effects were similar with or without oxcarbazepine. Perampanel was more effective in patients concomitantly treated with levetiracetam, however, this did not result in significantly more adverse events, including aggression. Significance. The present study suggests that perampanel, with or without concomitant oxcarbazepine or levetiracetam, is generally safe, well‐tolerated, and efficacious in paediatric patients with uncontrolled epilepsy in a clinical setting.

Therapeutic drug monitoring of perampanel in children diagnosed with epilepsy: Focus on influencing factors on the plasma concentration‐to‐dose ratio

“…In pediatric epilepsy, previous studies found that adjunctive PER therapy appeared effective and tolerable 3–5 . A good efficacy was also seen in PER monotherapy, evidenced by a multicenter, retrospective, observational study 6 .…”

“…2 In pediatric epilepsy, previous studies found that adjunctive PER therapy appeared effective and tolerable. [3][4][5] A good efficacy was also seen in PER monotherapy, evidenced by a multicenter, retrospective, observational study. 6 However, daily clinical experiences have shown large interindividual variability in the relationship between plasma PER levels and its clinical effects.…”

Efficacy, Tolerability, and Safety of Treatment With Perampanel in Pediatric Patients With Epilepsy Aged ≥4 Years: A Real-Life Observational Study

“…However, the recent approval of perampanel means that few real-life studies on using perampanel in Chinese patients have been reported. Only Qu et al 11 found that perampanel was safe, tolerated, and effective in treating pediatric patients with refractory epilepsy aged 2-14 years in China, with a 50% responder and seizure freedom rate of 51.2% and 27.4%, respectively.…”

“…Clinical data on the safety and efficacy of perampanel in the clinical setting involving Chinese pediatric patients are limited, [11][12][13] as perampanel has only recently been approved for use in China. Qu et al 11 found that perampanel, with or without concomitant oxcarbazepine or levetiracetam, is safe, well tolerated, and efficacious in pediatric patients aged 2-14 years with refractory epilepsy in a clinical setting. Liao et al 12 found that adjunctive perampanel (up to 12 mg/d) may be a suitable treatment for Chinese patients (aged ≥12 years) with focal seizures, with or without focal to bilateral tonic-clonic seizures, or generalized tonic-clonic seizures, with similar efficacy and safety or tolerability compared with non-Chinese patients.…”

LC-MS/MS assay for the therapeutic drug monitoring of perampanel in children with drug-resistant epilepsy

“…However, plasma concentrations of PER were not measured in the study, and the author concerned that they could not precisely assess the influence of concomitant enzyme inducers on the plasma levels of PER associated with seizure control and AEs [41]. Another retrospective observational study has suggested the effectiveness and safety of PER in Chinese pediatric patients (2-14 years) with refractory epilepsy [42]. But the author did investigate the plasma concentration of PER in these children, too.…”