Stiripentol for Drug-resistant Epilepsy Treatment in Tuberous Sclerosis Complex
Abstract number :
2.249
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2022
Submission ID :
2204735
Source :
www.aesnet.org
Presentation date :
12/4/2022 12:00:00 PM
Published date :
Nov 22, 2022, 05:26 AM
Authors :
Gewalin Aungaroon, MD – Cincinnati Children's Hospital. University of Cincinnati.; Amar Mehta, MD – Midwestern University; David Franz, MD – Cincinnati Children's Hospital, University of Cincinnati
Rationale: Drug-resistant epilepsy (DRE) is common in tuberous sclerosis complex (TSC). While stiripentol (STP) is proven efficacious in controlling seizures in Dravet syndrome, benefits in TSC patients are unknown.
Methods: A retrospective review of TSC patients at Cincinnati Children’s Hospital from 2011 until 2021 was performed to identify patients with DRE receiving STP. Seizure frequency was assessed 1 month before (baseline) and 1, 3, 6, and 12 months after STP initiation.
Results: Of the 1492 TSC patients, 13 (10 males) received STP. The age range was 3.8 to 40 years (median, IQR = 15.2 years, 6.7 - 22.0). STP was initiated a median of 13.5 years (IQR 5.0 - 20.3) after seizure onset. The median treatment duration was 12.8 months, and a median STP dose was 21.1 mg/kg/day (IQR = 12.8 - 34.1) and 750 mg/day (range, 500-2250). The number of patients with > 50% seizure reduction was 6/13 (46.2%), 4/13 (30.8%), 8/11 (72.7%), and 6/8 (75.0%) at 1, 3, 6, and 12 months. Importantly, 6 patients (46.2%) had persistent seizure reduction from 1 through 12 months, with the mean (±SD) percentage of reduction at 1, 3, 6, and 12 months of 68.1% (±22.0), 71.3% (±23.2), 75.7% (±23.5), and 75.7% (±23.5), respectively. Two of 13 (15.4%) reported worsening in seizure frequency with the treatment. Eleven of 13 patients reported side effects with aggression being the most common, and 3 patients discontinued STP due to side effects.
Conclusions: Most TSC patients with DRE treated with STP experienced seizure reduction, with about half having a persistent seizure reduction. About 20% of patients discontinue the treatment due to side effects. This study suggests that STP could be an efficacious seizure treatment in TSC patients. Further studies are necessary to better understand the utility of STP in this population.
Funding: None
Anti-seizure Medications