title: Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies.
abstract: BACKGROUND
  Studies on the efficacy and tolerability of rufinamide in infants and young children are scarce. Here we report on an open, retrospective, and pragmatic study about safety and efficacy of rufinamide in children aged less than four years, in terms of seizures types and epilepsy syndromes.
  
  
  METHODS
  Forty children (mean age 39.5 months; range 22-48) were enrolled in the study. The mean follow-up period was 12.2 months (range 5-21). Rufinamide was initiated at a mean age of 26.7 months (range 12-42). Final rufinamide mean dosage was 31.5 mg/kg/day if associated with valproic acid and 44.2 mg/kg/day if not.
  
  
  RESULTS
  The highest seizure reduction rate was observed in the epileptic spasms (46%) and drop attacks (42%) groups. Seizure reduction was also observed in tonic seizures (35%) and in the focal seizure (30%) groups. In terms of epilepsy syndrome, rufinamide was effective in Lennox-Gastaut syndrome. Results were very poor for those affected by Dravet's syndrome. Globally, responder rate was 27.5%, including two (5%) patients seizure-free. Adverse reactions occurred in 37.5% of children and were mainly represented by vomiting, drowsiness, irritability, and anorexia. Discontinuation rate due to treatment-emergent adverse events was 15%.
  
  
  CONCLUSIONS
  The present study concludes that rufinamide may be a safe and effective drug for a broad range of seizures and epilepsy syndromes in infants and young children and represents a valid therapeutic option in this population.
authors.affiliation: Pediatric Neurology-Immunology and Endocrinology Unit, Department of Pediatrics, University of Siena, Siena, Italy; Department of Pediatrics, University of Siena, Siena, Italy. Electronic address: grosso@unisi.it.
authors.forename: Salvatore
authors.initials: S
authors.lastname: Grosso
authors.affiliation: Institute of Child and Adolescence Neuropsychiatry, University of Salerno, Salerno, Italy.
authors.forename: Giangennaro
authors.initials: G
authors.lastname: Coppola
authors.affiliation: Child Neuropsychiatry Unit, University of Pavia, Pavia, Italy.
authors.forename: Serena Donetti
authors.initials: SD
authors.lastname: Dontin
authors.affiliation: Child Neurology Unit, Ospedale Bellaria, Bologna, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Gobbi
authors.affiliation: Child & Adolescent Neuropsychiatry, Azienda Ospedaliero-Universitaria, Cagliari, Italy.
authors.forename: Dario
authors.initials: D
authors.lastname: Pruna
authors.affiliation: Child Neuropsychiatric Unit, Civile Hospital, Brescia, Italy.
authors.forename: Patrizia
authors.initials: P
authors.lastname: Accorsi
authors.affiliation: Clinical Pediatrics, University of Perugia, Perugia, Italy.
authors.forename: Alberto
authors.initials: A
authors.lastname: Verrotti
authors.affiliation: Child Neurology, Chair of Pediatrics, NESMOS Department, Faculty of Medicine and Psychology, "Sapienza University", Rome, Italy.
authors.forename: Pasquale
authors.initials: P
authors.lastname: Parisi
authors.affiliation: Department of Pediatrics, University of Siena, Siena, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Balestri
mesh_terms: D000927:Anticonvulsants / Q000627:therapeutic use*; D002675:Child, Preschool; D004569:Electroencephalography; D004827:Epilepsy / Q000188:drug therapy*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; C079703:rufinamide
keywords: Antiepileptic drugs; Drug-resistant epilepsy; Epilepsy syndromes; Epileptic encephalopathy; Pediatrics