BACKGROUND: Severe myoclonic epilepsy in infants (SMEI), also known as Dravet 
syndrome, is a rare, refractory form of epilepsy, for whose treatment 
stiripentol (STP) has been recently licensed for add-on use.
OBJECTIVES: To evaluate the efficacy and tolerability of STP and other 
antiepileptic drug treatments (including ketogenic diet) as therapy for patients 
with SMEI.
SEARCH METHODS: We searched the Cochrane Epilepsy Group Specialised Register (15 
May 2013), the Cochrane Central Register of Controlled Trials (CENTRAL, Issue 4 
of 12, The Cochrane Library, April 2013), MEDLINE (1946 to May 2013) and SCOPUS 
(1823 to May 2013). The online trials registries ClinicalTrials.gov and the WHO 
International Clinical Trials Registry Platform were systematically searched. 
The bibliographies of any identified study were searched for further references. 
We handsearched selected journals and conference proceedings. No language 
restrictions were imposed.
SELECTION CRITERIA: Randomised controlled trials (RCTs) or quasi-randomised 
controlled trials; double- or single-blinded or unblinded trials; and 
parallel-group studies. Administration of at least one antiepileptic drug 
therapy given singly (monotherapy) or in combination (add-on therapy) compared 
with add-on placebo or no add-on treatment.
DATA COLLECTION AND ANALYSIS: Review authors independently selected trials for 
inclusion according to predefined criteria, extracted relevant data and 
evaluated the methodological quality of trials. The following outcomes were 
assessed: at least 50% seizure reduction, seizure freedom, adverse effects, 
proportion of dropouts and quality of life. Outcomes were assessed using a 
Mantel-Haenszel meta-analysis to calculate risk ratio (RR) with 95% confidence 
intervals (95% CIs).
MAIN RESULTS: No RCTs assessing drugs other than STP were found. Two RCTs 
evaluating the use of STP (total of 64 children) were included. Both studies 
were generally at unclear risk of bias. A significantly higher proportion of 
participants had 50% or greater reduction in seizure frequency in the STP group 
compared with the placebo group (22/33 vs 2/31; RR 10.40, 95% CI 2.64 to 40.87). 
A significantly higher proportion of participants achieved seizure freedom in 
the STP group compared with the placebo group (12/33 vs 1/31; RR 7.93, 95% CI 
1.52 to 41.21). No significant difference in the proportion of dropouts was 
found in the STP group compared with the placebo group (2/33 vs 8/31; RR 0.24, 
95% CI 0.06 to 1.03). Only one study explicitly reported the occurrence of side 
effects; higher proportions of participants were reported to experience side 
effects in the STP group compared with the placebo group (100% vs 25%; RR 3.73, 
95% CI 1.81 to 7.67).
AUTHORS' CONCLUSIONS: Data derived from two small RCTs indicate that STP is 
significantly better than placebo with regards to 50% or greater reduction in 
seizure frequency and seizure freedom. Adverse effects occurred more frequently 
with STP. Further adequately powered studies with long-term follow-up should be 
conducted to unequivocally establish the long-term efficacy and tolerability of 
STP in the treatment of SMEI.