Progress of add-on stiripentol therapy in children with Dravet syndrome

Dravet syndrome(DS), known as severe myoclonic epilepsy of infancy, is a devastating disorder characterized by intractable epilepsy and poor neurodevelopmental outcome.Seizures are refractory to conventional antiepileptic therapy, therefore resulting in heavy psychological pressure and burden.Stiripentol(STP)is a novel antiepileptic drug, which has been proposed to achieve better seizure control in DS.It acts as a direct GABA receptor agonist by increasing the GABAergic transmission and by prolongating the opening period of the receptor dependent chloride channels.In addition, STP inhibits several isoenzymes of the cytochrome P450 system in the liver involved in the metabolism of other antiepileptics, thus potentiating their effects.By summarizing the relevant researches at home and abroad, we describe an on-going work in the anticonvulsant mechanisms and efficacy of STP, to provide an alternative treatment of DS. Key words: Dravet syndrome; Stiripentol; Anticonvulsant Mechanism; Efficacy