Medical Affairs Biocodex, Inc San Mateo, California
Rationale: Dravet syndrome is a rare, catastrophic, infantile-onset epileptic encephalopathy associated with global developmental delays and intractable epilepsy characterized by multiple seizure types. Stiripentol (STP) is indicated for the treatment of seizures associated with Dravet syndrome in patients two years and older taking clobazam (CLB). The recommended target dose of STP is 50 mg/kg/day. This abstract reports the results of a survey of providers regarding the use of STP in the U.S. Dravet syndrome population to evaluate prescriptive behavior and perceived efficacy. Methods: A total of 52 physicians from 22 U.S. centers, selected based on their experience treating patients with Dravet syndrome, were invited to complete an online survey between April and May 2020. Twenty-seven (52%) responded, of whom 13 also treat adults, who reported a total of 192 patients (mean of 7 patients per physician). Data collected included patient age group, starting dose, target dose, titration increment, titration interval, perceived percent seizure reduction, and any taper of concomitant antiepileptic drugs. This abstract reports on starting and target doses, and perceived percent seizure reduction. Results: Reported typical starting and target doses for pediatric and adult patients are listed in Table 1. The most common STP titration increment was 10 mg/kg/day (38.5%) and 84.1% of physicians reported they titrate STP once a week. Figure 1 presents percentage seizure reduction in all ages, reported as 25% (n=2), 50% (n=13), 75% (n=8), and other (n=3). Reduction of concomitant CLB dose was reported by 73% of respondents with a reduction of 25% (n=11), 50% (n=3), or other (n=5). A total of 58% of respondents reported reducing concomitant VPA dose by 15% (n=2), 30% (n=5), 50% (n=1) or other (n=3). Conclusions: In this online survey of 27 physicians (from 22 U.S. centers) experienced using STP in the treatment of seizures in individuals with Dravet syndrome, 81% of providers reported at least 50% seizure reduction and 31% reported 75% seizure reduction with the use of STP. These data are very close to the data collected in the pivotal Phase 3 trials in Europe and in Japanese patients, with a responder rate even slightly higher (a responder being defined as a patient with a ≥50% seizure reduction). Although 37% of physicians titrated stiripentol to at least the recommended target dose of 50 mg/kg/day in their pediatric patients, 7.7% did so in their adult patients. The present data may indicate that U.S. physicians are aware of the difference in pediatric versus adult STP pharmacokinetics and of the need for an adaptive dosing strategy for adult patients.1,2 Overall, this survey indicates that both the prescription habits of US physicians and the efficacy reported in U.S. patients are very close to those collected in the other countries where stiripentol has been marketed for many years. Funding: Please list any funding that was received in support of this abstract.: Biocodex, Inc Click here to view image/table
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