Salvatore Grosso1, Giangennaro Coppola2, Serena Donetti Dontin3, Giuseppe Gobbi4, Dario Pruna5, Patrizia Accorsi6, Alberto Verrotti7, Pasquale Parisi8, Paolo Balestri9. 1. 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. 2. Institute of Child and Adolescence Neuropsychiatry, University of Salerno, Salerno, Italy. 3. Child Neuropsychiatry Unit, University of Pavia, Pavia, Italy. 4. Child Neurology Unit, Ospedale Bellaria, Bologna, Italy. 5. Child & Adolescent Neuropsychiatry, Azienda Ospedaliero-Universitaria, Cagliari, Italy. 6. Child Neuropsychiatric Unit, Civile Hospital, Brescia, Italy. 7. Clinical Pediatrics, University of Perugia, Perugia, Italy. 8. Child Neurology, Chair of Pediatrics, NESMOS Department, Faculty of Medicine and Psychology, "Sapienza University", Rome, Italy. 9. Department of Pediatrics, University of Siena, Siena, Italy.
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%. CONCLUSION: 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.
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 epilepticspasms (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%) patientsseizure-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%. CONCLUSION: 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.