Successful treatment of pediatric patient with Dravet syndrome with cenobamate: Case report.

Abstract

Dravet syndrome, also known as severe myoclonic epilepsy of infancy, is an epileptic encephalopathy characterized by severe epilepsy accompanied by impaired psychomotor and neurologic development. Onset is in the first year of life in apparently healthy infants. Seizures in Dravet syndrome are highly pharmacoresistant, and the SUDEP mortality rate is high. Here, we present the first case of the successful use of cenobamate in a pediatric patient (9-year-old female) with Dravet syndrome and pharmacoresistant epilepsy despite the prior use and failure of typical medications for Dravet syndrome, including fenfluramine, valproate, and clobazam, among others. At 6-month follow-up, the frequency of generalized tonic-clonic seizures decreased from 8 per month to 1 per month upon reaching a daily dose of 50 mg (1.9 mg/kg/day), which represents an 87.5% reduction. No side effects were reported. Given the single-patient case report nature of this study, further investigations are needed to extrapolate the usefulness of cenobamate across different age groups and genetic variants.