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Safety considerations selecting antiseizure medications for the treatment of individuals with Dravet syndrome

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EXPERT OPINION ON DRUG SAFETY
卷 20, 期 5, 页码 561-576

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TAYLOR & FRANCIS LTD
DOI: 10.1080/14740338.2021.1890025

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Cannabidiol; fenfluramine; stiripentol; adverse events; scn1a; manipulations; off-labels

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Management of individuals with Dravet Syndrome has seen significant progress in understanding the pathophysiology and outcomes of treatments. Antiseizure medications in infancy and childhood are crucial for safety considerations due to the active phase of seizures in early years. Recent treatments show promise in efficacy and safety, but individual assessment is still necessary to balance benefits and risks.
Introduction: Management of individuals with Dravet Syndrome has evolved significantly over the past 10 years. Progress has been made in understanding the pathophysiology, the long-term outcome and possible consequences of inappropriate therapies, new drugs have been approved by the regulatory authorities and patients and families expressed their needs beyond seizures' control. Areas covered: The authors aimed at providing an overview of the main antiseizure medications used in Dravet syndrome with a particular focus on safety considerations. As the highly active phase of seizures takes place before the age of 5 years, the characteristics of antiseizure medications in infancy and childhood have also been considered due to their impact on antiseizure medication safety. Expert opinion: Recent treatments, evaluated via randomized clinical trials, are promising in terms of efficacy and safety in individuals with DS. However, the balance between expected benefits and risks taken must be accurately assessed on an individual basis. There is a lack of data to understand the needs of patients and families, a major point particularly in this population, where the evaluation of efficacy and safety beyond seizures is difficult due to cognitive delay and behavioral disorders and where this evaluation is coming almost exclusively from caregivers.

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