title: Effectiveness and tolerability of rufinamide in children and adults with refractory epilepsy: first European experience.
abstract: OBJECTIVE
  The aim of the study was to explore the effectiveness and tolerability of rufinamide in a heterogeneous group of patients with refractory epilepsies in Europe, immediately after the drug became available as an orphan drug for the adjunctive treatment of Lennox-Gastaut syndrome (LGS).
  
  
  METHODS
  This observational study was conducted as a collection of retrospective data from multiple centers in Germany and Austria. Clinical course in patients treated with rufinamide was documented. Initial dosage and titration schedule of rufinamide were at the discretion of the treating physician according to medical need. The observation period was 12 weeks. Effectiveness was evaluated by comparing the frequency of seizures with limitations to the countability between baseline and the last 4-week period of observation.
  
  
  RESULTS
  The study population consisted of 45 children and 15 adults (mean age: 14.5+/-11.6 years, range: 1-50) with various severe and inadequately controlled epilepsy syndromes, that is, LGS (n=31), idiopathic generalized epilepsy syndromes (n=5), cryptogenic unclassified generalized epilepsy (n=7), and partial epilepsy (n=17). The response rate (50% reduction in countable seizures) was 46.7% (28 of 60 patients) in total; 25.0% experienced a 75% reduction in seizure frequency and 21.7% experienced a 50-75% reduction. Complete seizure control was achieved by 8.3%. The highest response rate was observed in patients with LGS (17/31, 54.8%), and the lowest in patients with partial epilepsy (4/17, 23.5%). Response rate in patients with unclassified generalized epilepsy was 42.8% (3/7 patients). A total of 67 adverse events were reported by 35 of 60 patients. The most frequently occurring adverse events were fatigue (18.3%), vomiting (13.3%), and loss of appetite (10.0%). No serious adverse events were observed.
  
  
  CONCLUSIONS
  These preliminary data suggest that rufinamide may be effective and well tolerated in the treatment of children and adults with various epilepsy syndromes and difficult-to-control seizures. The results of our study suggest that the efficacy of rufinamide in patients with generalized epilepsy might be comparable to that in patients with LGS, whereas rufinamide was less effective in patients with partial epilepsy.
authors.affiliation: Neuropediatric Department, BHZ Vogtareuth, Vogtareuth, Germany. gkluger@schoen-kliniken.de
authors.forename: Gerhard
authors.initials: G
authors.lastname: Kluger
authors.forename: Gerhard
authors.initials: G
authors.lastname: Kurlemann
authors.forename: Edda
authors.initials: E
authors.lastname: Haberlandt
authors.forename: Jan-Peter
authors.initials: JP
authors.lastname: Ernst
authors.forename: Uwe
authors.initials: U
authors.lastname: Runge
authors.forename: Felix
authors.initials: F
authors.lastname: Schneider
authors.forename: Christine
authors.initials: C
authors.lastname: Makowski
authors.forename: Rainer
authors.initials: R
authors.lastname: Boor
authors.forename: Thomas
authors.initials: T
authors.lastname: Bast
mesh_terms: D000293:Adolescent; D000328:Adult; D000927:Anticonvulsants / Q000009:adverse effects* / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004351:Drug Resistance; D004828:Epilepsies, Partial / Q000188:drug therapy; D004827:Epilepsy / Q000145:classification / Q000188:drug therapy*; D004829:Epilepsy, Generalized / Q000145:classification / Q000188:drug therapy; D005060:Europe; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D009965:Orphan Drug Production; D012189:Retrospective Studies; D012640:Seizures / Q000188:drug therapy / Q000453:epidemiology; D016896:Treatment Outcome; D014230:Triazoles / Q000009:adverse effects* / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; C079703:rufinamide