Giangennaro Coppola1, Frank Besag2, Raffaella Cusmai3, Olivier Dulac4, Gerhard Kluger5, Romina Moavero6, Rima Nabbout7, Marina Nikanorova8, Francesco Pisani9, Alberto Verrotti10, Celina von Stülpnagel11, Paolo Curatolo12. 1. Child and Adolescent Neuropsychiatry, Medical School, University of Salerno, Italy. 2. South Essex Partnership University NHS Foundation Trust, Bedfordshire, United Kingdom. 3. Neurology Unit, "Bambino Gesù" Children's Hospital, IRCCS, Rome, Italy. 4. Inserm U1129, Neuropediatrics Department, Necker-Enfants Malades Hospital, APHP, Paris Descartes University, CEA, Orsay, France. 5. Clinic for Neuropediatrics and Neurological Rehabilitation, Epilepsy-Center for Children and Adolescents, Vogtareuth, Germany; Paracelsus Medical University, Salzburg, Austria. 6. Department of Systems Medicine, Child Neurology and Psychiatry Unit, Tor Vergata University Hospital of Rome, Italy. 7. Centre de Reference Epilepsies Rares, Inserm U1129, Neuropediatrics Department, Necker-Enfants Malades Hospital, APHP, Paris Descartes University, CEA, Orsay, France. 8. Danish Epilepsy Centre, Dianalund, Denmark. 9. Department of Neurosciences, University of Messina, Italy. 10. Department of Pediatrics, University of Perugia, Italy. 11. Clinic for Neuropediatrics and Neurological Rehabilitation, Epilepsy-Center for Children and Adolescents, Vogtareuth, Germany. 12. Department of Systems Medicine, Child Neurology and Psychiatry Unit, Tor Vergata University Hospital of Rome, Italy. Electronic address: curatolo@uniroma2.it.
Abstract
PURPOSE: The literature on the efficacy and safety of rufinamide in childhood-onset epilepsy syndromes currently includes approximately 600 paediatric patients. This paper summarizes the views of a panel of experienced European epileptologists with regard to the current role of rufinamide in the treatment of childhood epilepsies. RESULTS: Rufinamide is effective in decreasing the seizure frequency in the Lennox-Gastaut syndrome (LGS), especially tonic and atonic seizures. It might consequently be preferred to other drugs as a second-line treatment for LGS when drop-attacks are frequent. The mean responder rate in the published studies is 38% with seizure freedom achieved in 2.4% of patients. Rufinamide has shown some efficacy in epileptic encephalopathies other than LGS. It can be also effective as adjunctive therapy in children and adolescents with drug-resistant partial seizures. The available data suggest that rufinamide has an acceptable risk/benefit ratio with quite a low risk of aggravating seizures. Common adverse effects (somnolence, nausea and vomiting) are usually mild and self-limiting; they are more frequently observed during titration than in the maintenance phase, suggesting that low escalation rates might be associated with fewer adverse effects. Rufinamide appears to have a favourable cognitive profile compared with other antiepileptic drugs. CONCLUSION: Rufinamide is only approved for adjunctive treatment of seizures associated with LGS in children 4 years of age and older. There are very few data on rufinamide treatment at the onset of LGS or early in the course of the disorder; whether early treatment will improve outcome has yet to be determined.
PURPOSE: The literature on the efficacy and safety of rufinamide in childhood-onset epilepsy syndromes currently includes approximately 600 paediatric patients. This paper summarizes the views of a panel of experienced European epileptologists with regard to the current role of rufinamide in the treatment of childhood epilepsies. RESULTS:Rufinamide is effective in decreasing the seizure frequency in the Lennox-Gastaut syndrome (LGS), especially tonic and atonic seizures. It might consequently be preferred to other drugs as a second-line treatment for LGS when drop-attacks are frequent. The mean responder rate in the published studies is 38% with seizure freedom achieved in 2.4% of patients. Rufinamide has shown some efficacy in epilepticencephalopathies other than LGS. It can be also effective as adjunctive therapy in children and adolescents with drug-resistant partial seizures. The available data suggest that rufinamide has an acceptable risk/benefit ratio with quite a low risk of aggravating seizures. Common adverse effects (somnolence, nausea and vomiting) are usually mild and self-limiting; they are more frequently observed during titration than in the maintenance phase, suggesting that low escalation rates might be associated with fewer adverse effects. Rufinamide appears to have a favourable cognitive profile compared with other antiepileptic drugs. CONCLUSION:Rufinamide is only approved for adjunctive treatment of seizures associated with LGS in children 4 years of age and older. There are very few data on rufinamide treatment at the onset of LGS or early in the course of the disorder; whether early treatment will improve outcome has yet to be determined.
Authors: Martin J Brodie; Frank Besag; Alan B Ettinger; Marco Mula; Gabriella Gobbi; Stefano Comai; Albert P Aldenkamp; Bernhard J Steinhoff Journal: Pharmacol Rev Date: 2016-07 Impact factor: 25.468