摘要
Abstract
Objective To investigate the efficacy and safety of perampanel(PER)add-on therapy in children with epilepsy of genetic etiology.Methods A retrospective analysis was conducted on the clinical data of 53 children who attended the Department of Neurology,Wuhan Children's Hospital,from November 2020 to April 2023.All children received PER add-on therapy and were diagnosed with epilepsy of genetic etiology based on whole-exome sequencing.The primary outcome measure was the proportion of children with a reduction in seizure frequency of≥50%at month 12 of PER treatment(i.e.,response rate),and the secondary outcome measures were response rates at months 3 and 6 of treatment.The influencing factors for the efficacy of PER add-on therapy in the treatment of epilepsy of genetic etiology were analyzed,and adverse events were recorded.Results The median follow-up duration was 13.10 months.After 12 months of follow-up,42 children were included in the analysis,comprising 25 boys(60%)and 17 girls(40%).The median initial dose of PER was 1.5(1.0,2.0)mg/d,and the median maintenance dose was 4.0(3.0,8.0)mg/d.The response rates to PER at months 3,6,and 12 of treatment were 61%(30/49),54%(25/46),and 48%(20/42),respectively.No significant difference in the efficacy of PER was observed between children with mutations in genes encoding different protein functions(P>0.05).The most common adverse event reported was fatigue,observed in 3 children(6%).Conclusions PER add-on therapy demonstrates good efficacy and safety in children with epilepsy of genetic etiology.No influencing factors for the efficacy of PER have been identified to date.关键词
癫痫/吡仑帕奈/添加治疗/儿童Key words
Epilepsy/Perampanel/Add-on therapy/Child
