title: Low long-term efficacy and tolerability of add-on rufinamide in patients with Dravet syndrome.
abstract: In this retrospective European multicenter study we evaluated the efficacy and tolerability of rufinamide in patients with Dravet syndrome and refractory seizures. Twenty patients were included; in 16 patients a SCN1A mutation was detected. The responder rate after 6 months was 20%, and after 34 months, 5%. The retention rate was 45% after 6 months and 5% after 34 months. Rufinamide treatment was stopped because of aggravation of seizures (30%), no effect (45%), and side effects (10%). The efficacy and long-term retention rate were low in our patients with Dravet syndrome and refractory seizures, far lower than in patients with Lennox-Gastaut syndrome; one-third of our patients experienced seizure aggravation. Therefore, rufinamide does not seem to be a suitable option for long-term treatment in patients with Dravet syndrome.
authors.affiliation: Neuropediatric Clinic and Clinic for Neurorehabilitation, Epilepsy Center for Children and Adolescents, Schön Klinik Vogtareuth, Vogtareuth, Germany.
authors.forename: A
authors.initials: A
authors.lastname: Mueller
authors.forename: R
authors.initials: R
authors.lastname: Boor
authors.forename: G
authors.initials: G
authors.lastname: Coppola
authors.forename: P
authors.initials: P
authors.lastname: Striano
authors.forename: M
authors.initials: M
authors.lastname: Dahlin
authors.forename: C
authors.initials: C
authors.lastname: von Stuelpnagel
authors.forename: J
authors.initials: J
authors.lastname: Lotte
authors.forename: M
authors.initials: M
authors.lastname: Staudt
authors.forename: G
authors.initials: G
authors.lastname: Kluger
mesh_terms: D000293:Adolescent; D000927:Anticonvulsants / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020190:Myoclonic Epilepsy, Juvenile / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D062550:NAV1.1 Voltage-Gated Sodium Channel; D009419:Nerve Tissue Proteins / Q000235:genetics; D012189:Retrospective Studies; D003294:Seizures, Febrile / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D015222:Sodium Channels / Q000235:genetics; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; D062550:NAV1.1 Voltage-Gated Sodium Channel; D009419:Nerve Tissue Proteins; C568242:SCN1A protein, human; D015222:Sodium Channels; D014230:Triazoles; C079703:rufinamide