PracticeUpdate Neurology February 2019

EDITOR’S PICKS 10

Stiripentol Decreases Seizure Frequency in Dravet Syndrome Journal of Child Neurology Take-home message • In Dravet syndrome, an epileptic encephalopathy of infancy, the neurocognitive outcomes of these patients are related to early control of seizures and status epilepticus. Although there are approved therapies, not all patients respond to the treatments available. Stiripentol is a new anti-epileptic medication with a novel mechanism of action (allosteric modulator of GABA-A receptor), which has shown efficacy in treating seizures in patients with Dravet syndrome in placebo-controlled trials. In this study, the authors retrospectively reviewed their results of adding stiripentol to the anti-epileptic regimens in 21 patients with Dravet syndrome between 2016 and 2017. The mean duration of stiripentol use in this cohort was 41 months. Seizure frequency decreased by more than 50% in more than half of the patients, and 2 patients became seizure-free. Adverse effects included sedation, tremor, and ataxia; 1 patient stopped stiripentol due to increased seizure frequency. • These data, collected over a longer duration of use than what has previously been reported in clinical trials of stiripentol, support the efficacy and relative tolerability of a new adjunctive treatment for Dravet syndrome. Sarah Matteson Kranick MD

Abstract Dravet syndrome is a rare and progressive epileptic encephalopathy of infancy. Stiripen- tol reduces the seizure frequency in patients with Dravet syndrome. We evaluated the clin- ical characteristics of patients with Dravet syndrome and their response to stiripentol. We retrospectively collected the data of 21 patients (11 females; mean age, 8.2 years, range: 5.4-15 years) with Dravet syndrome who were treated with stiripentol in our outpatient clinic between June 2016 and June 2017. Patients with seizure reduction ≥50% were considered responders. Most of our patients had severe (47%) or moder- ate (33%) cognitive disabilities, although 14% had mild cognitive disability. There was a significant difference in both status epilepticus and age between the groups with normal/mild versus severe/moderate neurocognitive prognoses. Of the patients, 85.7% were using stiripentol. The mean duration of stiripentol use was 41.2 months (range: 24-64 months). In 12 patients (57%), the seizure frequency decreased by more than 50%, and 2 of them were seizure-free. Status epilepticus was not recorded after stiripentol treatment in 8 of 11 patients with status epilepti- cus. Despite the small sample size, our results suggest that stiripentol has a favorable efficacy. In addition, considering the absence of status epilepticus after treatment and the negative effects of status epilepticus on cognitive devel- opment, early treatment should be initiated in SD patients, for whom disease control is difficult. Efficacy of Stiripentol and the Clinical Outcome in Dravet Syndrome. J Child Neurol. 2018 Oct 26;[EPub Ahead of Print], E Pembegul Yıldız, " These data… support the efficacy and relative tolerability of a new adjunctive treatment for Dravet syndrome. "

MU Ozkan, TA Uzunhan, et al. www.practiceupdate.com/c/77662

PRACTICEUPDATE NEUROLOGY