title: Rufinamide in children and adults with Lennox-Gastaut syndrome: first Italian multicenter experience.
abstract: This is the first multicenter Italian experience with rufinamide as an adjunctive drug in children, adolescents and adults with Lennox-Gastaut syndrome. The patients were enrolled in a prospective, add-on, open-label treatment study from 11 Italian centers for children and adolescent epilepsy care. Forty-three patients (26 males, 17 females), aged between 4 and 34 years (mean 15.9 ± 7.3, median 15.0), were treated with rufinamide for a mean period of 12.3 months (range 3-21 months). Twenty patients were diagnosed as cryptogenic and 23 as symptomatic. Rufinamide was added to the baseline therapy at the starting dose of 10mg/kg body weight, evenly divided in two daily doses and then increased by 10mg/kg approximately every 3 days up to a maximum of 1000 mg/day in children aged ≥4 years with a body weight less than 30 kg. In patients more than 30 kg body weight, rufinamide could be titrated up to 3200 mg/day. After a mean follow-up period of 12.3 months (range 3-21 months), the final mean dose of rufinamide was 33.5mg/kg/24h (range 11.5-60) if combined to valproic acid, and of 54.5mg/kg/24h (range 21.8-85.6) without valproic acid. The response rate (≥50% decrease in countable seizures) was 60.5% (26 of 45 patients) in total; 51.1% experienced a 50-99% reduction in seizure frequency and complete seizure control was achieved in the last 4 weeks follow-up by 9.3% of patients. Two patients (4.7%) had a 25-50% seizure reduction, while seizure frequency remained unchanged in 13 (30.2%) and increased in 2 (4.7%). Reliable data for atypical absence seizures and myoclonic seizures were not available, as these are usually impossible to count. Ten patients (23.2%) reported adverse side effects, while taking rufinamide. They were generally mild and transient and most frequently included vomiting, drowsiness, irritability and loss of appetite. In conclusion, rufinamide as an adjunctive therapy reduced the number of drop attacks and major motor seizures in about 60% of patients with Lennox-Gastaut syndrome and produced only mild or moderate adverse side effects.
authors.affiliation: Clinic of Child Neuropsychiatry, Second University of Naples, Naples, Italy. gianegennaro.coppola@unina2.it
authors.forename: Giangennaro
authors.initials: G
authors.lastname: Coppola
authors.forename: Salvatore
authors.initials: S
authors.lastname: Grosso
authors.forename: Emilio
authors.initials: E
authors.lastname: Franzoni
authors.forename: Pierangelo
authors.initials: P
authors.lastname: Veggiotti
authors.forename: Nelia
authors.initials: N
authors.lastname: Zamponi
authors.forename: Pasquale
authors.initials: P
authors.lastname: Parisi
authors.forename: Alberto
authors.initials: A
authors.lastname: Spalice
authors.forename: Francesco
authors.initials: F
authors.lastname: Habetswallner
authors.forename: Antonio
authors.initials: A
authors.lastname: Fels
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Capovilla
authors.forename: Alberto
authors.initials: A
authors.lastname: Verrotti
authors.forename: Salvatore
authors.initials: S
authors.lastname: Mangano
authors.forename: Alberto
authors.initials: A
authors.lastname: Balestri
authors.forename: Paolo
authors.initials: P
authors.lastname: Curatolo
authors.forename: Antonio
authors.initials: A
authors.lastname: Pascotto
mesh_terms: D000293:Adolescent; D000328:Adult; D000927:Anticonvulsants / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008607:Intellectual Disability / Q000188:drug therapy; D007558:Italy; D065768:Lennox Gastaut Syndrome; D008297:Male; D013036:Spasms, Infantile / Q000188:drug therapy; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D014635:Valproic Acid / Q000008:administration & dosage / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; D014635:Valproic Acid; C079703:rufinamide