Rationale: Stiripentol (STP) is approved for the treatment of seizures associated with Dravet Syndrome (DS) in patients taking clobazam who are 6 months of age and older. While STP has been available for more than a decade, evidence describing its prescription patterns in the United States (US) remains limited. Given the critical role of DS specific therapies, it is important to understand how STP is prescribed in practice. It was hypothesized that STP may be underutilized in the US relative to global practice.
Methods: A physician survey was conducted, hosted by REDCap, to assess STP prescribing practices in DS. The questionnaire collected information on provider demographics, prescription frequency, dosing/ titration strategies, observed DDIs, and perceived clinical effectiveness. Recruitment was conducted through the DSF. Participation was voluntary and anonymous. Responses were analyzed using descriptive statistics.
Results:
To date, 28 physicians have completed the comprehensive survey. Respondents were primarily pediatric epileptologists (89.3%) practicing in the US (92.9%), with additional responses from Brazil and Russia. 96.4% reported prescribing STP, exclusively for DS. Among prescribers, 63.0% had treated 1–5 patients and 29.6% had treated >20 patients. STP was most prescribed in combination with clobazam (85.7%), though 32.1% reported using microdoses of clobazam to satisfy insurance requirements. 64.3% co-prescribed sodium valproate. Half prescribed STP in conjunction with fenfluramine (FFA), 75.0% with cannabidiol (CBD) with most (85.7%) adjusting the FFA dose when used in combination.
When initiating therapy, providers prioritized seizure frequency (53.6%), seizure length (35.7%), and seizure character (10.7%). Most providers described relying on clinical features such as seizure frequency, severity, and side effects to guide therapy, while a minority reported using levels selectively for toxicity assessment, titration, or establishing individualized baselines. Independent of clobazam, STP effectiveness was rated as good or very good by 53.6% of providers, while 17.9% rated outcomes as poor or very poor.
Adverse effects, most commonly sedation, decreased appetite, and cognitive slowing, were reported by most providers, with 42.9% indicating that ≤25% of patients experienced side effects. Discontinuation was required in 7–10% of patients. Side effects were most often observed at higher doses, particularly in pediatric patients. Overall, 60.7% agreed that STP reduced rescue medication use, 64.3% reported decreased seizure-related hospitalizations, and 60.7% noted behavioral changes after initiation.
Conclusions:
Preliminary findings from this survey highlight a modest uptick in STP utilization in DS across experienced pediatric epilepsy specialists. While most providers reported experiencing clinical benefit, adverse effects, and insurance-related barriers were common. These findings suggest STP is utilized but variably implemented in practice, with opportunities to refine dosing strategies and address systemic barriers to optimize outcomes for patients with DS.
Funding: This study is supported by Biocodex.