Stiripentol: Efficacy and tolerability in children with epilepsy

Purpose: Stiripentol (STP) is a new antiepileptic drug (AED) that inhibits cytochrome P-450, resulting in increased plasma concentrations of concomita nt AEDs. The efficacy and tolerability of STI) as an add-on therapy in chil dren were assessed. Methods: Two hundred twelve patients with refractory epilepsy, aged from I month to 20.5 years, received STP either in a single-blind, placebo-control led trial (108 patients) or in a further open trial (104 other patients sel ected by epilepsy syndrome for possible: efficacy based on the results of t he previous trial). Results: Among the 97 patients who could be analyzed for efficacy in the pl acebo-controlled study, the median seizure frequency was lower at 3 months with STP than with the placebo (p < 0.0001); 49% responded to the drug, inc luding 10% who became seizure free. Patients with partial epilepsy had the highest response rate (57%). Results were confirmed in the open study where 68% of the 91 patients receiving STP responded at 3 months. These patients were mainly those with partial epilepsy (73%) who were receiving carbamaze pine (CBZ) (75%) as comedication (p < 0.001). Ten of the 20 children with s evere myoclonic epilepsy in infancy also responded with clobazam (CLB) as c omedication. Efficacy was sustained long term in 74% of the 94 patients sti ll receiving STP at a mean 30-month follow-up. Adverse events were reported in 48% of the 212 patients, mainly anorexia and loss of weight, but these events required STP discontinuation in only nine cases. Side effects were m inimized in the open trial by optimizing the dose of comedication. Conclusions: STP seems to be a promising add-on drug, particularly when com bined with CBZ in patients with partial childhood epilepsy refractory to vi gabatrin (VGB) and with CLB in patients with severe myoclonic epilepsy in i nfancy.