Abstracts

Rationale: To assess the safety and efficacy of stiripentol (STP) as add-on therapy for children with Dravet syndrome (DS). Methods: Five DS patients (age range, 1.3-6.8 years) confirmed with SCN1A mutations, followed by a one month STP dose-adjustment and three months fixed-dose phase after a one month baseline phase. Frequency of generalized tonic-clonic seizures, focal seizures, status epilepticus, use of AEDs and adverse events were recorded. The primary efficacy endpoint was responder rate (proportion of patients with a =50% reduction from baseline phase in seizure frequency over the last one month of fixed-dose treatment). Results: All five patients entered the dose-adjustment phase and completed the study. The responder rate was 60% (3/5) at the endpoint, three patients were seizure free, they were combined with valproate (VPA) and /or clobazam (CLB), and two of them performed strong sensitivity to VPA after the addition of STP (20-40 mg/kg/d). The duration of generalized tonic-clonic seizures, focal seizures or status epilepticus was also reduced in the other two patients, though the overall seizure frequency was not reduced. Before STP initiation, the patients had tried 2-10 kinds (mean 6) of AEDs, but still had 1-2 times or even more seizures per month. The most frequent adverse events of STP were nausea, vomiting, anorexia, drowsiness , weary, ataxia and excitability, which related to a significant increase in plasma concentrations of VPA and CLB after the addition of STP, all relieved with or without symptomatic managements 1 month later. Conclusions: Add-on STP to VPA and/or CLB was well tolerated and significantly reduced seizure frequency in patients with SCN1A related Dravet syndrome. Funding: Key Research Project of the Ministry of Science and Technology of China (Grant No. 2016YFC0904400)