title: First European long-term experience with the orphan drug rufinamide in childhood-onset refractory epilepsy.
abstract: OBJECTIVE
  Recently, we published the first postmarketing European experience with rufinamide (RUF) in a retrospective 12-week observational study. This follow-up report summarizes the long-term effectiveness and tolerability of RUF after 18 months for the same patient sample.
  
  
  METHODS
  In total, 52 of 60 initially included patients from eight centers in Germany and Austria (45 children and 15 adults aged 1-50 years) with various severe and inadequately controlled epilepsy syndromes continued treatment with RUF after the initial 3-month observation period (mean final dose: 38.2+/-17.3mg/kg/day). Efficacy was assessed by seizure frequency evaluated by comparison with baseline frequency. Tolerability was evaluated by analysis of parental report of adverse events and laboratory tests. Responders were defined as patients who achieved a 50% or greater decrease in countable seizures within 18 months of initiating RUF therapy.
  
  
  RESULTS
  Mean overall duration of RUF treatment was 14.5 months (range: 3-18 months). Retention rate, defined as the percentage of patients still taking RUF after 18 months, was 41.7% (n=25/60). The overall response rate after 18 months was 26.7% (16/60 patients). The highest response rates were found in the subgroup of patients with Lennox-Gastaut syndrome (LGS, 35.5%) and in patients with other generalized epilepsy syndromes. Complete seizure control was maintained in one patient (1.6%). A total of 73 adverse events were reported in 37 of 60 patients. The most frequently occurring adverse events were fatigue (18.3%), vomiting (15.0%), and loss of appetite (10.0%). Only 4 new adverse events were reported after week 12. No serious adverse events were observed.
  
  
  CONCLUSIONS
  The present data suggest that RUF is efficacious and well tolerated in the long-term treatment of children and adults with various epilepsy syndromes and difficult-to-control seizures.
authors.affiliation: Neuropediatric Department, BHZ Vogtareuth, Vogtareuth, Germany. gkluger@schoen-kliniken.de
authors.forename: Gerhard
authors.initials: G
authors.lastname: Kluger
authors.forename: Edda
authors.initials: E
authors.lastname: Haberlandt
authors.forename: Gerhard
authors.initials: G
authors.lastname: Kurlemann
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; D017668:Age of Onset; D000927:Anticonvulsants / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004827:Epilepsy / Q000188:drug therapy*; D005060:Europe; D005260:Female; D006801:Humans; D007223:Infant; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D009965:Orphan Drug Production; D011358:Product Surveillance, Postmarketing; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; C079703:rufinamide