Current role of rufinamide in the treatment of childhood epilepsy: literature review and treatment guidelines

Eur J Paediatr Neurol. 2014 Nov;18(6):685-90. doi: 10.1016/j.ejpn.2014.05.008. Epub 2014 May 28.


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.

Keywords: Antiepileptic drug interactions; Epileptic encephalopathy; Focal seizures; Lennox–Gastaut syndrome; Paediatric epilepsy; Rufinamide.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Anticonvulsants / pharmacokinetics
  • Anticonvulsants / standards*
  • Anticonvulsants / therapeutic use*
  • Epilepsy / drug therapy*
  • Female
  • Guidelines as Topic*
  • Humans
  • Male
  • Triazoles / pharmacokinetics
  • Triazoles / standards*
  • Triazoles / therapeutic use*


  • Anticonvulsants
  • Triazoles
  • rufinamide