摘要
Importance:Perampanel(PER)is used less extensively in children than in adults.Currently,there is a lack of data from PER clinical studies with large sample sizes in Chinese children and adolescents with epilepsy,especially those with refractory epilepsy.Objective:To evaluate the effectiveness,retention,and safety of PER in the treatment of children and adolescents with epilepsy in China.Methods:This was a multicenter,prospective,observational study.Children and adolescents with epilepsy who received PER as adjunctive therapy were included.The primary effectiveness endpoint was the proportion of patients achieving a≥50%reduction in seizure frequency after 6 months of treatment compared to baseline.The secondary effectiveness endpoints included retention and seizure-free rates.The safety outcome was the incidence of treatment-emergent adverse events(TEAEs).Results:A total of 240 patients with epilepsy were enrolled in the study.Prior to initiating PER treatment,approximately 87.9%of them took two or more antiseizure medications.After a 6-month treatment regimen with PER,70.4%of the patients experienced a reduced seizure frequency of at least 50%,and 22.1%achieved complete seizure freedom.The retention rate was 90.2%.TEAEs were reported by 89 patients,leading to the discontinuation of PER in seven cases.No severe TEAEs were observed in this study.Interpretation:Under routine clinical conditions,PER demonstrated good effectiveness and retention in Chinese children with epilepsy,particularly in those with refractory epilepsy.
