Dravet syndrome (DS) is a debilitating, epileptic encephalopathy of childhood for which few treatment options were available in the United States prior to 2018. Because, until recently, treatment options were limited, most patients retained a high seizure burden even with polypharmacy, with little positive impact on non-seizure-related outcomes. Novel treatment options, however, provide an unprecedented level of seizure control with prolonged
intervals of seizure freedom and ≥75% seizure reduction in as many as 50% of patients, with evidence emerging that the robust reduction in seizure frequency also improves cognitive outcomes. Future investigations will show if treatment with novel agents can translate into DS patients having a greater likelihood of better long-term neurodevelopmental outcomes. This symposium will review DS and its clinical diagnosis and management, with a practical focus on rational therapy choices that optimize patient management and long-term outcomes.
AGENDA
Introduction
Elizabeth A. Thiele, MD, PhD (Program Chair)
Harvard Medical School
Massachusetts General Hospital
Dravet Syndrome: Clinical Updates and Therapeutic Options
Elizabeth A. Thiele, MD, PhD
Seizure Management: From Clinical Trials to Clinical Care
Elaine C. Wirrell, MD, FRCPC
Mayo Clinic
Secondary Outcomes: Real-World Perspectives on Long-Term Outcomes and Clinical Expectations
Joseph E. Sullivan, MD
University of California, San Francisco (UCSF)
UCSF Benioff Children’s Hospital Pediatric Epilepsy Center of Excellence
Q&A
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