Authors :
Presenting Author: Carla Schad, MD – Biocodex
Fábio Nascimento, MD – Washington University School of Medicine
Carly Katterman, PharmD – Biocodex
Andrea Calvert, PharmD, BCPPS – Biocodex
Kelly Gwin, PharmD – Biocodex
Rationale:
Dravet syndrome (DS) is a severe developmental and epileptic encephalopathy characterized by drug-resistant seizures, which often requires polytherapy for adequate seizure control. Despite the availability and international consensus recommendations for DS-approved antiseizure medications (ASMs), stiripentol (STP), fenfluramine (FFA), and cannabidiol (CBD), a recent analysis found that these ASMs have been underutilized in DS patients, with only 7% of patients prescribed STP, 16% FFA, and 28% CBD.1,2 A US multi-center survey and advisory board aimed to explore ASM selection, barriers to DS-specific ASM use, and strategies to improve alignment with international consensus recommendations.2 Methods:
A voluntary survey was completed by 38 clinicians from 27, Level IV National Association of Epilepsy Centers (NAECs). The survey assessed 1st and 2nd line ASM selections for patients with DS, along with rationale for treatment approaches. Upon completion of the survey, a virtual ad-board was held with 8 pediatric neurologists to further analyze results with subsequent discussion surrounding barriers to ASM usage and strategies for mitigation. Results:
The survey identified valproate (47%) or clobazam (CLB) (24%) as the most common 1st line ASMs considered when a patient is diagnosed with DS, with levetiracetam as a common ASM already included in a patient’s regimen. LB (47%) and FFA (24%) were found to be the 2nd most common ASMs considered, followed by CBD (11%) and STP (8%). Respondents cited lack of familiarity, access, and side effects as leading barriers to utilization of DS-specific therapies. Despite limited use, 58% of respondents agreed that DS-approved therapies provide better efficacy compared to other ASMs utilized. In addition, the majority (71%) agreed that seizure freedom is possible when treating patients with DS. While analyzing the differences of practice sites, most providers (89%) agreed that the location where patients receive care impacts ASM selection, however, there was a discrepancy between utilization and comfort level based on institution/provider type. Advisory board discussions reinforced these findings emphasizing the need for expanded medical education on DS-specific ASMs.
Conclusions:
This analysis confirms underutilization of DS-approved ASM therapies in DS management, highlighting a gap between consensus recommendations and real-world practice. Perceptions of limited access, dosing, drug-drug interactions, and lack of exposure were consistently cited as barriers to usage. Targeted education, increased familiarity, and streamlined access are critical for early adoption of DS-specific ASM to optimize seizure control and improve long-term outcomes in this high-risk population. Funding: Biocodex