title: Rufinamide as an adjuvant treatment in children with Lennox-Gastaut syndrome.
abstract: OBJECTIVE
  To evaluate the efficacy of rufinamide as an add-on treatment in children and adolescents with Lennox-Gastaut syndrome (LGS).
  
  
  METHODS
  The study was an open-label, observational clinical trial of rufinamide as an add-on treatment in intractable LGS patients. This intent-to-treat trial included 4 weeks of scheduled titrated doses and a 12-week maintenance phase with a target dose of 20-40 mg/kg rufinamide, adjusted according to its effectiveness and tolerability after a baseline period of 4 weeks. The primary outcome was measured by the seizure-reduction rate according to individual seizure type over the 12-week maintenance period.
  
  
  RESULTS
  One hundred and twenty-eight patients with LGS who were determined to be unresponsive to one or more antiepileptic drugs or dietary therapy were enrolled. Of the 128 patients enrolled, 112 (87.5%) completed the study. After add-on rufinamide treatment, 46 patients (35.9%) achieved a more than 50% reduction in seizure frequency and 10 (7.8%) patients became seizure-free. When we identified those who responded with an at least 50% reduction in seizure frequency, 39.4% of the responders reported reductions in convulsive seizures, 36.4% in drop attacks, 33.3% in myoclonic seizures, and 20.0% in epileptic spasms. Overall, 32.8% of patients reported adverse effects, which were mostly mild and transient in nature. The most common adverse effects were fatigue (15 patients, 11.7%) and poor appetite (9 patients, 7.0%). Twenty-one (16.4%) patients experienced an increased seizure frequency.
  
  
  CONCLUSIONS
  Rufinamide appears to be a safe and effective adjuvant treatment for many cases of intractable LGS.
authors.affiliation: Division of Pediatric Neurology, Pediatric Epilepsy Clinics, Severance Children's Hospital, Epilepsy Research Institute, Yonsei University College of Medicine, Seoul, Republic of Korea.
authors.forename: Shin Hye
authors.initials: SH
authors.lastname: Kim
authors.forename: So-Hee
authors.initials: SH
authors.lastname: Eun
authors.forename: Hoon-Chul
authors.initials: HC
authors.lastname: Kang
authors.forename: Eun Ji
authors.initials: EJ
authors.lastname: Kwon
authors.forename: Jung Hye
authors.initials: JH
authors.lastname: Byeon
authors.forename: Young-Mock
authors.initials: YM
authors.lastname: Lee
authors.forename: Joon Soo
authors.initials: JS
authors.lastname: Lee
authors.forename: Baik-Lin
authors.initials: BL
authors.lastname: Eun
authors.forename: Heung Dong
authors.initials: HD
authors.lastname: Kim
mesh_terms: D000293:Adolescent; D000927:Anticonvulsants / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007223:Infant; D008607:Intellectual Disability / Q000188:drug therapy*; D065768:Lennox Gastaut Syndrome; D008297:Male; D013036:Spasms, Infantile / Q000188:drug therapy*; D014230:Triazoles / Q000008:administration & dosage*
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; C079703:rufinamide