Abstracts

Rationale: To review the efficacy and tolerability of stiripentol in the treatment of US children with Dravet syndrome.Methods: US clinicians who had prescribed stiripentol for two or more children with Dravet syndrome between 03/2005 and 03/2012 were contacted to request participation in this retrospective study. Data collected included overall seizure frequency, frequency of prolonged seizures, use of rescue medications and ER/hospital visits in the year preceding stiripentol initiation, and with stiripentol therapy. We separately assessed efficacy in the following treatment groups: Group A: stiripentol without clobazam or valproate, Group B: stiripentol with clobazam but without valproate, Group C: stiripentol with valproate but without clobazam, and Group D: stiripentol with clobazam and valproate. Additionally, adverse effects were recorded.Results: Thirteen of 16 clinicians contacted for study participated and provided data on 82 children. Stiripentol was initiated a median of 6.0 years after seizure onset and 1.2 years after diagnosis of Dravet syndrome. Compared to baseline, overall seizure frequency was reduced in 2/6 in Group A, 28/35 in Group B, 8/14 in Group C and 30/48 in Group D. All children with prolonged seizure frequency greater than quarterly during the baseline period experienced a reduction in this frequency on the various treatment arms with stiripentol. Similarly, 2/4 patients in Group A, 25/25 in Group B, 5/10 in Group C and 26/33 in Group D experienced reduction in frequency of rescue medication usage and 1/1 in Group A, 12/12 in Group B, 3/5 in Group C and 18/19 in Group D had reduction in frequency of ER/hospital visits. Adverse effects were reported in 38, most commonly sedation and reduced appetite. Four patients (5%) discontinued stiripentol for adverse effects and two (2%) for lack of efficacy. Conclusions: Stiripentol is an effective and well-tolerated therapy which markedly reduced frequency of prolonged seizures in Dravet syndrome.