title: Safety and retention rate of rufinamide in 300 patients: a single pediatric epilepsy center experience.
abstract: OBJECTIVE
  Reports of studies evaluating rufinamide as an add-on therapy in children and adolescents with refractory epilepsy are restricted to a few publications. Prospective multicenter studies including children and adults have yielded important information about several types of epilepsies and syndromes. We evaluated the use of rufinamide in a single pediatric center with a large cohort and long-term follow-up period.
  
  
  METHODS
  We retrospectively included patients taking rufinamide from November 2008 to March 2013. Response was defined by a seizure reduction of ≥50% compared to baseline.
  
  
  RESULTS
  Three hundred patients with a median age of 9.1 years (range 0.4-29.6 years) were reviewed. Median follow-up was 9 months (range 1-37 months). Epilepsy etiology was classified as genetic (23.7%), structural/metabolic (41%), and unknown cause (35.3%). Overall, rufinamide treatment led to a median seizure frequency reduction of 59.2% from responders to baseline. Seizure reduction was greater in patients with genetic etiology compared to structural/metabolic (66.2% vs. 45.5% responders, p = 0.005). Rufinamide was discontinued in 110 (36.7%) of 300 patients: 63 (21%) due to unsatisfactory response, 47 (15.7%) due to side effects, and in 18 (6%) of those due to both. Most common adverse effects were sleepiness, vomiting, mood changes, nausea, and loss of appetite. Median time to loss of efficacy was 11.6 months (range 3-28 months).
  
  
  CONCLUSIONS
  Rufinamide provides satisfactory seizure reduction as an adjunctive treatment in refractory epilepsy. Results need to be interpreted in the setting of data acquisition, including inherent biases of retrospective studies. Patients with a known genetic etiology may have better responses than patients with structural/metabolic etiology.
authors: Division of Epilepsy and Clinical Neurophysiology, Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, U.S.A; Department and Institute of Psychiatry, Faculty of Medicine, University of São Paulo, São Paulo, Brazil.
authors: Sigride
authors: S
authors: Thome-Souza
authors: Navah E
authors: NE
authors: Kadish
authors: Sriram
authors: S
authors: Ramgopal
authors: Iván
authors: I
authors: Sánchez Fernández
authors: Ann M
authors: AM
authors: Bergin
authors: Jeffrey
authors: J
authors: Bolton
authors: Chellamani
authors: C
authors: Harini
authors: Mark
authors: M
authors: Libenson
authors: Heather
authors: H
authors: Olson
authors: Jurriaan
authors: J
authors: Peters
authors: Annapurna
authors: A
authors: Poduri
authors: Alexander
authors: A
authors: Rotenberg
authors: Masanori
authors: M
authors: Takeoka
authors: Sanjeev V
authors: SV
authors: Kothare
authors: Kush
authors: K
authors: Kapur
authors: Blaise F D
authors: BF
authors: Bourgeois
authors: Tobias
authors: T
authors: Loddenkemper
mesh_terms: D000293:Adolescent; D000328:Adult; D000927:Anticonvulsants / Q000008:administration & dosage* / Q000009:adverse effects*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004359:Drug Therapy, Combination; D004827:Epilepsy / Q000175:diagnosis / Q000188:drug therapy* / Q000523:psychology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D010349:Patient Compliance / Q000523:psychology; D011446:Prospective Studies; D012189:Retrospective Studies; D012893:Sleep Wake Disorders / Q000139:chemically induced / Q000175:diagnosis; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects*; D014839:Vomiting / Q000139:chemically induced / Q000175:diagnosis; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; D014230:Triazoles; C079703:rufinamide
keywords: Epilepsy; Follow-up; Pediatric; Refractory epilepsy; Retention rate; Rufinamide