Fu-Man Chang1, Pi-Chaun Fan2, Wen-Chin Weng2, Chin-Hao Chang3, Wang-Tso Lee4. 1. Department of Pediatrics, Taitung MacKay Memorial Hospital, Taitung, Taiwan. 2. Department of Pediatric Neurology, National Taiwan University Children's Hospital, Taipei, Taiwan; Department of Pediatrics, National Taiwan University College of Medicine, Taipei, Taiwan. 3. Center of Statistical Consultation and Research in the Department of Medical Research, National Taiwan University Hospital, Taipei, Taiwan. 4. Department of Pediatric Neurology, National Taiwan University Children's Hospital, Taipei, Taiwan; Department of Pediatrics, National Taiwan University College of Medicine, Taipei, Taiwan; Graduate Institute of Brain and Mind Sciences, National Taiwan University College of Medicine, Taipei, Taiwan. Electronic address: wangtsolee@ntu.edu.tw.
Abstract
PURPOSE: Perampanel, a selective, noncompetitive alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) antagonist, has been approved as an effective and safe drug for the treatment of adults and adolescents with epilepsy. However, only a few studies have reported about the efficacy and tolerability of perampanel in children under 7 years old with refractory epilepsy. In this work, we aimed to describe our clinical experience in this group. METHODS: This single-center retrospective observational study was performed by reviewing the medical records of children below 7 years old with epilepsy and were treated with perampanel. RESULTS: A total 38 patients (19 females, 19 males) were enrolled, with a mean age of 4 ± 1.6 years (range, 0-6 years). The response rates (defined as 50 % seizure reduction) were 44 % and 31 % after 6 and 12 months of treatment, respectively, and the freedom of seizures was reached in 13 % and 10 % after the same treatment periods. The short- and long-term response rate in patients with tuberous sclerosis (TSC) and Dravet syndrome was high (67 %). The response rate in refractory spasms was 40 % at 6 months, but dropped to 13 % after 12 months of treatment. The retention rates were 61.1 % and 51.5 % at 6 and 12 months, respectively. Adverse events were reported in eight patients (22 %), which included emotional change (n = 4), lethargy (n = 2), nasal bleeding (n = 1), and drug allergy (n = 1). CONCLUSIONS: As a real-world pediatric case series, our study demonstrated the efficacy and tolerability of perampanel in young children with intractable epilepsy. In our experience, lower starting and maintenance dose of perampanel would be effective in the pediatric group. We also propose the use of perampanel as an effective therapy for epilepsy in children with TSC and Dravet syndrome.
PURPOSE: Perampanel, a selective, noncompetitive alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) antagonist, has been approved as an effective and safe drug for the treatment of adults and adolescents with epilepsy. However, only a few studies have reported about the efficacy and tolerability of perampanel in children under 7 years old with refractory epilepsy. In this work, we aimed to describe our clinical experience in this group. METHODS: This single-center retrospective observational study was performed by reviewing the medical records of children below 7 years old with epilepsy and were treated with perampanel. RESULTS: A total 38 patients (19 females, 19 males) were enrolled, with a mean age of 4 ± 1.6 years (range, 0-6 years). The response rates (defined as 50 % seizure reduction) were 44 % and 31 % after 6 and 12 months of treatment, respectively, and the freedom of seizures was reached in 13 % and 10 % after the same treatment periods. The short- and long-term response rate in patients with tuberous sclerosis (TSC) and Dravet syndrome was high (67 %). The response rate in refractory spasms was 40 % at 6 months, but dropped to 13 % after 12 months of treatment. The retention rates were 61.1 % and 51.5 % at 6 and 12 months, respectively. Adverse events were reported in eight patients (22 %), which included emotional change (n = 4), lethargy (n = 2), nasal bleeding (n = 1), and drug allergy (n = 1). CONCLUSIONS: As a real-world pediatric case series, our study demonstrated the efficacy and tolerability of perampanel in young children with intractable epilepsy. In our experience, lower starting and maintenance dose of perampanel would be effective in the pediatric group. We also propose the use of perampanel as an effective therapy for epilepsy in children with TSC and Dravet syndrome.
Authors: Reza Maroofian; Jiří Sedmík; Neda Mazaheri; Marcello Scala; Maha S Zaki; Liam P Keegan; Reza Azizimalamiri; Mahmoud Issa; Gholamreza Shariati; Alireza Sedaghat; Christian Beetz; Peter Bauer; Hamid Galehdari; Mary A O'Connell; Henry Houlden Journal: J Med Genet Date: 2020-07-27 Impact factor: 6.318