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Published on: September 20, 2024
Short-term efficacy and tolerability of rufinamide adjunctive therapy in children with refractory generalised
Se Hee Kim1, Jeong Ho Lee, Hye Won Ryu
1Department of Pediatrics, Pediatric Clinical Neuroscience Center, Seoul National University Children's Hospital, South Korea.
Insights
Rufinamide adjunctive therapy showed efficacy in children with refractory generalised epilepsies, particularly Lennox-Gastaut syndrome. However, response rates varied among epilepsy types, with Dravet syndrome and myoclonic seizures showing less improvement.
Area of Science:
- Pediatric Neurology
- Clinical Pharmacology
- Epileptology
Background:
- Refractory generalised epilepsy in children presents significant treatment challenges.
- Adjunctive therapies are crucial for managing difficult-to-treat epilepsy syndromes.
- Lennox-Gastaut syndrome and Dravet syndrome are severe forms of childhood epilepsy.
Purpose of the Study:
- To evaluate the efficacy and tolerability of rufinamide as an adjunctive therapy.
- To assess treatment response in children with various refractory generalised epilepsy types.
- To identify patient subgroups that may benefit most or least from rufinamide.
Main Methods:
- A cohort study involving 53 children with refractory generalised epilepsy.
- Rufinamide was administered as adjunctive therapy.
- Seizure reduction of over 50% at three and six months defined treatment responders.
Main Results:
- Overall response rates were 37.7% at three months and 34.0% at six months.
- Patients with Lennox-Gastaut syndrome and epilepsy with spasms/tonic seizures demonstrated higher response rates.
- Subgroups with Dravet syndrome and epilepsy with myoclonic seizures showed lower responsiveness to rufinamide.
Conclusions:
- Rufinamide is an effective and well-tolerated adjunctive treatment for many children with refractory generalised epilepsies.
- Treatment outcomes vary significantly based on the specific epilepsy syndrome.
- Further research is needed to optimize rufinamide use in specific pediatric epilepsy populations.
Abstract:
We evaluated the efficacy and tolerability of rufinamide adjunctive therapy in children with refractory generalised epilepsy. The study cohort consisted of 20 patients with Lennox-Gastaut syndrome, 5 with Dravet syndrome, and 28 with unclassified refractory generalised epilepsy. Patients with more than 50% seizure reduction at three and six months were defined as responders. The overall response rate was 37.7% at three months and 34.0% at six months. At three months, patients with Lennox-Gastaut syndrome (40.0%) and epilepsy with spasms/tonic seizures (38.5%) showed higher response rates than those with Dravet syndrome (20.0%) and epilepsy with myoclonic seizures (20.0%). High response rates in patients with Lennox-Gastaut syndrome (30.0%) and epilepsy with spasms/tonic seizures (38.5%) were sustained throughout the six-month study. The accuracy of, and differences between, responder rates should, however, be interpreted with caution due to the small number of patients. Overall, rufinamide appeared to be effective and reasonably well tolerated in this group of children with refractory generalised epilepsies, although a subgroup of patients with Dravet syndrome and epilepsy with myoclonic seizures were less responsive to rufinamide treatment.
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