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

摘要

Highlights ? Our study is the largest case series on children treated with Perampanel. ? 96 patients from 11 tertiary paediatric neurology centres in the UK were included. ? Perampanel was effective in children with both focal and generalised seizures. ? Children under 12 years of age had a similar responder rate to older children. ? The adverse events leading to discontinuation of PER were seen in 1/3 of patients. Abstract Purpose Perampanel is one of the latest antiepileptic drugs (AEDs) approved for the treatment of focal and generalised epilepsy in individuals with epilepsy aged 12 years and older. There is sparse data on the use of Perampanel in children under 12. We conducted a study amongst paediatric neurologists in the United Kingdom to investigate its effectiveness and tolerability as an adjunctive therapy in children of all ages with refractory epilepsy. Methods Data was collected via an online questionnaire sent to paediatric neurologists in the UK. Data gathered, prospective in 62 (64.5%) and retrospective in 34 (35.5%) patients, included changes in seizure frequency from baseline and unwanted effects at 3, 6 and 12 months follow-up. Only patients with a minimum follow-up of six months were included. Results Ninety six patients (48 females) with refractory epilepsy from 11 of 29 tertiary centres were included. Median [IQR] (range) age was 14 years 11 months [12 years, 16 years 6 months] (11 months–24 years 5 months). Seventy three (76%) had focal epilepsy, sixteen (17%) generalised, and seven (7%) patients both generalised and focal epilepsy. The responder rate, ≥50% seizure reduction from baseline, was 19% for all seizure types at both 6 and 12 months, 19% and 24% for focal seizures, and 25% and 7% for generalised seizures at these time points respectively. The retention rate was 42% at 12 months. Treatment was discontinued due to unwanted effects in 29 (36.7%) of the 79 patients with follow-up data available up to 12 months: 30% due to challenging behaviour, 14% dizziness, and 7.6% somnolence. Conclusion 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.