TY - JOUR
T1 - Real-life effectiveness and tolerability of perampanel in pediatric patients aged 4 years or older with epilepsy
T2 - A korean national multicenter study
AU - Hwang, Su Kyeong
AU - Lee, Yun Jin
AU - Nam, Sang Ook
AU - Kim, Won Seop
AU - Kim, Jon Soo
AU - Kim, Sun Jun
AU - Lee, Yun Jeong
AU - Kwon, Soonhak
N1 - Publisher Copyright:
© 2020 Korean Neurological Association.
PY - 2020/1
Y1 - 2020/1
N2 - Background and Purpose The US Food and Drug Administration approval for perampanel has only recently been expanded to patients as young as 4 years, and so there have been few real-life studies of the effects of perampanel in pediatric patients. The aim of this study was to determine the long-term efficacy, factors affecting treatment response, and tolerability of perampanel as an add-on therapy in pediatric patients aged 4 years or older with epilepsy. Methods This multicenter retrospective observational study collected data from pediatric epilepsy centers of four Korean national universities. Changes in the seizure frequency from baseline, adverse events, and retention rates were obtained at 3, 6, and 12 months. Adverse events and discontinuation profiles were obtained to assess tolerability. Results This study included 220 children and adolescents (117 males and 103 females) aged 4 to 20 years. The overall response rate was 43.6%, and the seizure-freedom rate was 17.7%. Factors affecting a good treatment response were the absence of intellectual disability, small number of concomitant antiepileptic drugs, and low baseline seizure frequency. Eighty-eight patients (40%) experienced adverse events, but they mostly were of mild severity and resolved after the dose reduction or discontinuation of perampanel. The retention rates at 3, 6, and 12 months were 85.0%, 71.8%, and 50.5%, respectively. Conclusions Adjunctive treatment with perampanel was efficacious and tolerated in pediatric patients aged 4 years or older with epilepsy. Early perampanel treatment may help to reduce the burden of their seizures and improve their quality of life.
AB - Background and Purpose The US Food and Drug Administration approval for perampanel has only recently been expanded to patients as young as 4 years, and so there have been few real-life studies of the effects of perampanel in pediatric patients. The aim of this study was to determine the long-term efficacy, factors affecting treatment response, and tolerability of perampanel as an add-on therapy in pediatric patients aged 4 years or older with epilepsy. Methods This multicenter retrospective observational study collected data from pediatric epilepsy centers of four Korean national universities. Changes in the seizure frequency from baseline, adverse events, and retention rates were obtained at 3, 6, and 12 months. Adverse events and discontinuation profiles were obtained to assess tolerability. Results This study included 220 children and adolescents (117 males and 103 females) aged 4 to 20 years. The overall response rate was 43.6%, and the seizure-freedom rate was 17.7%. Factors affecting a good treatment response were the absence of intellectual disability, small number of concomitant antiepileptic drugs, and low baseline seizure frequency. Eighty-eight patients (40%) experienced adverse events, but they mostly were of mild severity and resolved after the dose reduction or discontinuation of perampanel. The retention rates at 3, 6, and 12 months were 85.0%, 71.8%, and 50.5%, respectively. Conclusions Adjunctive treatment with perampanel was efficacious and tolerated in pediatric patients aged 4 years or older with epilepsy. Early perampanel treatment may help to reduce the burden of their seizures and improve their quality of life.
KW - Adverse drug reactions
KW - Children
KW - Treatment efficacy
KW - Perampanel
UR - http://www.scopus.com/inward/record.url?scp=85078277628&partnerID=8YFLogxK
U2 - 10.3988/jcn.2020.16.1.53
DO - 10.3988/jcn.2020.16.1.53
M3 - Article
AN - SCOPUS:85078277628
SN - 1738-6586
VL - 16
SP - 53
EP - 59
JO - Journal of Clinical Neurology (Korea)
JF - Journal of Clinical Neurology (Korea)
IS - 1
ER -