Abstracts

Rationale: Stiripentol is a novel anticonvulsant that belongs to a family of aromatic allylic alcohols and is thought to have direct anticonvulsant activity related to effects on gammaaminobutyric acid (GABA). It also potentiates the efficacy of other anticonvulsants as a result of its pharmacokinetic interactions. It was authorised in the European Union in January 2007 for use in conjunction with clobazam and valproate as adjunctive therapy of refractory generalized tonic-clonic seizures in patients with severe myoclonic epilepsy in infancy (SMEI or Dravet's syndrome) whose seizures were not adequately controlled with clobazam and valproate. We conducted a retrospective study to evaluate the efficacy and safety of Stiripentol in management of epilepsy in children with Dravet s syndrome and other intractable epilepsy syndromes. Methods: Retrospective review of electronic case notes of all children treated with Stiripentol from May 2006 to May 2010 in the department of Neurology at the Birmingham Children s Department. The data was supplemented by telephonic questionnaires to parents where required. Results: A total of 25 children were identified, all with intractable epilepsy. This included 12 males and 13 females with age ranging from 8 months to 13.5 yrs at onset of treatment. They had previously been trialled on 2-14 anti-convulsant medications and 9 had been tried with VNS and /or ketogenic diet with poor seizure control. 16 (64%) had a diagnosis of Dravet s Syndrome. Of this 10(62.5%) were SCN1A mutation positive. 2 were negative for the mutation while results were awaited on 4. 2 children experienced single seizure type while the rest had multiple types of seizures.11 experienced < 10 seizures/day while 8 had > 30 seizures/day. In 19 children stiripentol was used in combination with either Sodium Valproate and/ or clobazam. In the SCN1A mutation positive Dravet s group (n=10), 5(50%) experienced significant reduction (50%) in seizure frequency while 4 (40%) showed no benefit and 1 experienced an increase in seizures. Among the SCN1A negative Dravet s (n=2), 1(50%) showed > 50% reduction in seizure frequency while the other child showed no response. In the 4 children in whom the mutation status was not known 3 achieved significant seizure reduction while 1 did not show any response. One child had become seizure free but the benefit was sustained for 4 months only. In the Non- Dravet s group (n=9), 3(33 %) showed > 50% seizure reduction including one child who was seizure free for 7 months. 6(67%) showed no response. 9(36%) experienced side effects secondary to Stiripentol. This was in the form of sedation (4), appetite / weight problems (3), behavioural problems (2) and raised ferritin(1). Stiripentol was continued in 18 (72%) while it was withdrawn in 4 cases owing to no benefit and due to side effects in 3. Conclusions: Stiripentol is effective and safe to use in children with epilepsy. It is effective in reducing seizures not only in children with Dravet s syndrome, but also in other Childhood epilepsy syndromes.