Stiripentol safety profile and efficacy in cases of SCN1A-related Dravet syndrome, multi-center experience, Saudi Arabia.

Abstract

Objectives: To evaluate the effectiveness and safety of Stiripentol (STP) in individuals with Dravet syndrome resulting from SCN1A gene mutation METHODS: A retrospective study with a descriptive focus was carried out in 2023, involving 44 patients diagnosed with Dravet Syndrome, with data collected by chart review and interviews of caregivers based on a pre-structured data extraction sheet. Data included age of seizure onset, SCN1A gene variant, duration of seizures before STP initiation, age at the time of starting STP administration, change in seizure frequency or duration after STP initiation, and at 6 months following initiation, and concomitant anti-seizure medication.

Results: 25 patients experienced a significant reduction in the frequency of their seizures, while 12 showed a mild to moderate reduction. Almost all patients taking Stiripentol concomitant with Valproate and Clobazam had marked seizure reduction. Adverse reactions to Stiripentol were observed in 34 patients, but none were serious. Somnolence was the highest reported (20 patients), followed by behavioral changes, agitation, irritability, and hyperactivity (18 patients) CONCLUSION: Our study showed that over half of our patients with Dravet on Stiripentol had a marked reduction in seizure frequency and duration. The study revealed no serious adverse events. Minor adverse events included somnolence and behavioral changes.