Incorporating Newer ASMs in Pediatric Patients With Dravet Syndrome

EP: 1.Symptoms and Disease Progression in Pediatric Patients With Dravet Syndrome

EP: 2.Role of SCN1A Genetic Testing in Diagnosis of Dravet Syndrome

EP: 3.Risk Factors and Risk Mitigation Strategies of SUPED for Patients With Dravet Syndrome

EP: 4.Standard of Care and Dravet Syndrome–Specific ASMs: Mechanisms and Risk Management

EP: 5.Treatment Selection and Treatment Adherence in Dravet Syndrome

EP: 6.Monitoring and Switching Therapies in Patients With Dravet Syndrome

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EP: 7.Incorporating Newer ASMs in Pediatric Patients With Dravet Syndrome

EP: 8.Emerging Disease-Modifying Therapies in Dravet Syndrome

Specialist Practice vs Real-World Implementation

Epilepsy specialists report that the majority, if not all, of their patients with Dravet syndrome are receiving first- through third-line therapies according to current guidelines, including the newer targeted medications cannabidiol, stiripentol, and fenfluramine. When patients in specialist practices are on fourth-, fifth-, or sixth-line therapies, it typically indicates that the evidence-based first-line treatments have already been attempted without success. This represents optimal implementation of current treatment standards within specialized centers where clinicians have developed expertise with these newer medications and feel confident incorporating them into routine practice.

National Claims Data Reveals Treatment Gaps

However, analysis of national claims data using the International Statistical Classification of Diseases, Tenth Revision code for Dravet syndrome reveals significant gaps in real-world implementation of evidence-based therapies. Among newly diagnosed patients, the newer targeted medications (stiripentol, fenfluramine, and cannabidiol) were used in only 7% to 25% of cases within the first year following diagnosis. Conversely, older, nonapproved medications that should be reserved for later-line therapy were prescribed as first-line treatments in more than 50% of newly diagnosed patients. This pattern suggests that many clinicians are still defaulting to traditional approaches rather than utilizing the medications with the strongest evidence base for Dravet syndrome.

Implementation Barriers and Educational Needs

The disconnect between international guidelines and clinical practice appears to stem from practical implementation challenges rather than awareness of the recommendations. While guidelines appropriately identify which medications should be prioritized, they may not adequately address the practical aspects of clinical implementation, such as dosing strategies, titration schedules, and management techniques. This knowledge gap creates barriers for physicians who recognize the importance of using evidence-based therapies but lack the detailed procedural knowledge necessary to confidently incorporate these newer medications into their practice, ultimately impacting patient access to optimal treatment.