Real-World Utilization of Stiripentol in Children Aged 3 Years and Younger: A US Perspective
Abstract number :
3.375
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2025
Submission ID :
275
Source :
www.aesnet.org
Presentation date :
12/8/2025 12:00:00 AM
Published date :
Authors :
Presenting Author: Kelly Gwin, PharmD – Biocodex
Carly Katterman, PharmD – Biocodex
Andrea Calvert, PharmD, BCPPS – Biocodex
Veanna Fong, N/A – Biocodex
Fabiola Garcia, N/A – Biocodex
Carla Schad, MD – Biocodex
Rationale: Early and effective seizure control in Dravet syndrome (DS) may be associated with improved long-term neurodevelopmental outcomes, yet data on real-world treatment patterns in the pediatric population remain limited. Stiripentol’s (STP) indication in the US was expanded from ≥2 years of age to include children down to 6 months of age in 2022, making it the only FDA approved Dravet-specific therapy in this age group. Chiron et al. demonstrated that STP initiation before 2 years of age is associated with a decrease in frequency/ duration of seizures and episodes of status epilepticus (SE)1. STP use in young children has increased, reflecting the expanded indication, evolving clinical practice, and emphasis on earlier diagnosis. This analysis describes earlier treatment approaches in patients who initiated STP at ≤3 years of age.
Methods: A retrospective analysis was performed on US patients who initiated STP at the age of ≤3 years from August 2022 to April 2025 from a singular specialty pharmacy. Data in the analysis included age, weight, STP dosage, SKU, diagnosis code, and concomitant medications. Dosage, SKU, and diagnosis code were collected based off prescriptions dispensed to the patient, while concomitant medications were captured during discussions with patients or caregivers at initiation.
Results: Among patients with DS who initiated STP between August 2022 - April 2025, 27% were ≤3 with an average time on therapy of 11 months. Of these, 96% were started on the sachets for oral suspension vs. the oral capsules. The average prescribed STP dose during the 1st month was 50 mg/kg/day, increasing slightly to an average prescribed maintenance dose of 51 mg/kg/day at 8 months of treatment or beyond (Table 1). At STP initiation, 77% of patients/caregivers reported use of clobazam. Thirty percent of patients had been on cannabidiol (21%) or fenfluramine (9%) prior to starting STP (Table 2). Additionally, 7% of patients reported somnolence; 4% reported weight loss/decreased appetite.
Conclusions: This analysis represents the 1st, US-led real-world perspective of STP use in patients <
Anti-seizure Medications