Enter Note Done
Go to previous page in this tab
Session
  • Presentation | Poster Session | Poster Session 1
  • Poster Session
  • Bookmark Icon
  • Real-World Use and Effectiveness of Stiripentol in U.S. Patients with Dravet Syndrome: Results from the STIRUS Study
    • Schedule
    Notes
  • Set Timezone

Poster Number: 1.386

Rationale:

Stiripentol (STP) is approved in the U.S. for the treatment of seizures associated with Dravet syndrome (DS) in patients aged ≥6 months who are taking clobazam. Since its approval in 2018, real-world data on current STP use have been limited. The STIRUS study aimed to characterize treatment patterns, clinical outcomes, and quality of life following STP initiation in a contemporary U.S. DS population.



Methods:

STIRUS was a retrospective, observational, multicenter review conducted across 10 US epilepsy centers. Patients with a confirmed diagnosis of DS who initiated STP after August 2018 and received it for ≥3 months were included. Data were extracted for three time periods: 3 months prior to STP initiation (baseline), the first 3 months on STP, and the final 3 months on STP (irrespective of treatment discontinuation). Outcomes included seizure frequency (by type), status epilepticus - seizures lasting > 5min - (SE), rescue medication use, emergency room (ER) visits/hospitalizations, and quality of life. Changes from baseline were analyzed using generalized estimating equations; odds ratios (ORs) >1 indicated improvement. Investigators were also asked to evaluate patients and caregivers’ quality of life (subjective description).



Results: A total of 98 patients (55 female) were included, of whom 94 had an SCN1A mutation. At baseline, patients were taking a median of 3 antiseizure medications, most commonly clobazam (n=80), cannabidiol (n=45), valproate (n=42), fenfluramine (n=24), or levetiracetam (n=23). 90% experienced bilateral convulsive seizures (BCS), and 34% had SE, with the longest episode lasting a median of 30 minutes. The median age at DS diagnosis was 15 months, and the median age at STP initiation was 6.9 years. STP significantly reduced BCS frequency (OR=2.16, 95% CI [1.25–3.75]; p< 0.006), and the number of patients experiencing SE decreased from 33 at baseline to 16 and 14 during the first and final 3 months, respectively (ORs >2.8; p< 0.003), with the longest episode lasting a median of 15 minutes. STP also reduced the need for rescue medications (OR=3.5; p< 0.0001) and seizure-related ER visits/hospitalizations (OR=4.0; p< 0.0001 in the final 3 months). Improvements were consistent across age groups and STP dosage and were observed with or without co-treatment with clobazam or valproate. Quality of life improved for 52% of patients and 54% of caregivers. Adverse events were reported in 50% of patients, most commonly somnolence and decreased appetite, with no unexpected safety concerns.

Conclusions:

In this contemporary U.S. DS cohort, the use of stiripentol was associated with a reduction in seizure burden, status epilepticus episodes, and healthcare utilization, with consistent benefits observed across age groups and co-medication profiles. These findings support STP as an effective treatment option beyond its approved use with clobazam.



Funding: This study was funded by Biocodex.

Types
Main Session