Effectiveness and tolerability of Perampanel in children, adolescents and young adults with refractory epilepsy: A UK national multicentre study

Abstract

Perampanel was fairly effective in a heterogeneous group of 96 children and adolescents with very refractory epilepsy. The rate of adverse events leading to discontinuation was considerable in this group.

Efficacy and tolerability of perampanel in children and adolescents with pharmacoresistant epilepsy: The first real-world evaluation in Asian pediatric neurology clinics

“…In contrast, an Italian study found that the proportion of responder patients was higher in patients taking enzyme-inducing antiepileptic drugs than in those taking nonenzyme-inducing antiepileptic drugs. However, the majority of prior observational studies have reported no significant difference in the PER responder rate between patients undergoing concomitant treatment [31,[36][37][38], which is consistent with our observations. The diverse results suggest that the effect of concomitant enzyme-inducing antiepileptic drugs on PER may vary in real-world settings.…”

“…A similar responder rate was observed in 24 adolescents in a Canadian center over a median treatment duration of 59 weeks [26]; however, no patients achieved sustained seizure freedom. Recently, a United Kingdom national multicenter observational study in a heterogeneous group of 96 children and adolescents with highly refractory epilepsy who underwent PER treatment reported an overall responder of 18.8% for all seizure types at both 6-and 12-month follow-ups [31]. Our data were generally consistent with previous reports of broadly similar responder and seizure-free rates for the overall core study population.…”

“…Treatment was discontinued because of lack of efficacy/aggravation in 16 (24.2%) and unwanted side effects in eight (12.1%) of the 66 patients at the end of the current study. With respect to the tolerability and adverse events, patients with pharmacoresistant seizures in our study cohort demonstrated relatively good toleration with a 35.7% rate of self-reported adverse events compared with other series wherein adverse events were noted in 48-67% of patients [26][27][28][29]31]. The leading adverse events of PER were irritability followed by skin rash, dizziness, somnolence, ataxia, and anxiety.…”

“…A recent study conducted in 13 Lennox-Gastaut syndrome patients reported that nine patients (69.2%) were responders, and seven patients (53.8%) were improved in cognitive function and/ or behavior [30]. Other studies also indicated a responder of 40-66.6% in a small group of nine patients [28,29,31]. In contrast, almost all Lennox-Gastaut syndrome patients in our study had a poor response to PER as adjunctive therapy, with the exception of one patient who achieved seizure freedom at the daily dose of 8 mg by the 12-month follow-up evaluation.…”

“…Although the efficacy of PER treatment in Dravet syndrome was only mentioned sporadically in some reports with a limited number of patients, a clinically significant response was observed in half of them (one patient was seizure-free and one experienced aggravation in [29]; one patient with ≥75% seizure reduction in [28]; and lack of efficacy in one [31]). Notably, our results indicated that PER was conspicuously efficacious in patients with Dravet syndrome, achieving clinically meaningful improvement in four of five patients and even seizure freedom in two patients.…”