Utilization of Stiripentol in Children With Developmental and Epileptic Encephalopathies: Considerations for Clinical...

Developmental and epileptic encephalopathies (DEEs) are a group of pediatric seizure syndromes, accompanied by developmental delay or regression and cognitive, psychiatric, and/or behavioral impairment. Seizures can be frequent and are typically refractory to treatment with antiseizure medications. In addition to the high burden of disease endured by patients, DEEs are also associated with negative psychosocial impacts and reduced quality of life for parents and caregivers. Stiripentol is an antiseizure medication approved for the treatment of seizures associated with Dravet syndrome in children 6 months of age and older currently taking clobazam. Early studies in children with drug-resistant seizures provided preliminary evidence of the efficacy and safety of adjunctive stiripentol in seizure syndromes other than Dravet syndrome. These studies have since been followed by additional studies in cohorts of children with different DEEs, as well as studies in children with a specific non-Dravet DEE diagnosis, which have demonstrated association of add-on stiripentol treatment with improvements in seizure control, increased rates of seizure freedom, reductions in rates of status epilepticus, and, in a few studies, improvements in cognitive outcomes. Given stiripentol's favorable safety and tolerability profile, these results suggest a potential role of stiripentol in the treatment of refractory patients with DEE. The purpose of this paper is to review the available evidence of the efficacy and safety of stiripentol in children with non-Dravet DEEs. Practical considerations regarding the use of stiripentol in clinical practice to treat pediatric patients with DEE will also be discussed.