Stiripentol is safe and beneficial in infants with Dravet syndrome younger than 2 years of age, according to a new study published in the journal Developmental Medicine & Child Neurology. The authors of the study reported that the treatment reduced long-lasting seizures, including hospitalizations, status epilepticus, and mortality during the first years of life.
To investigate the safety and efficacy of stiripentol in patients with Dravet syndrome before 2 years of age, the team, led by Rima Nabbout, MD, PhD, from Necker-Enfants Malades Hospital in Paris, France, conducted a 30-year, real-world retrospective study.
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The researchers analyzed data from 131 patients who started stiripentol treatment before 2 years of age. In most patients, the treatment was added to valproate and clobazam at 13 months at a median dose of 50 mg/kg daily.
The researchers reported that with short-term therapy, lasting less than 6 months, the frequency of tonic–clonic seizures lasting longer than 5 minutes decreased. Moreover, status epilepticus lasting longer than 30 minutes was completely abolished in more than half (55%) of patients. Finally, emergency hospitalizations dropped from 91% to 43%.
With long-term therapy, the frequency of long-lasting tonic-clonic seizures continued to decline, and emergency hospitalizations dropped down to 12%.
During the study period, a total of 3 patients died from sudden unexpected death in epilepsy. Another 3 patients discontinued stiripentol due to adverse events.
A little more than half of patients (55%) reported at least 1 adverse event. The most common of these were appetite and weight loss, seen in 21% of patients, and somnolence, seen in 11% of them.
“Data reuse in medicine is a major innovation axis in rare diseases,” the authors concluded. “It can answer important questions that are not addressed by randomized controlled trials:”
Dravet syndrome is a rare type of childhood epilepsy that is hard to control.
Stiripentol is the first therapy indicated for the treatment of seizures in Dravet syndrome in combination with clobazam.
Chiron C, Chemaly N, Chancharme L, Nabbout R. Initiating stiripentol before 2 years of age in patients with Dravet syndrome is safe and beneficial against status epilepticus. Dev Med Child Neurol. Published online May 17, 2023. doi:10.1111/dmcn.15638