Five Cases of Fenfluramine Therapy for Dravet Syndrome

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  • Dravet症候群に対してfenfluramine療法を行った5例

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<p>Objective: The efficacy of fenfluramine therapy for Dravet syndrome has been reported in Europe and the United States. However, no studies of fenfluramine therapy for Dravet syndrome have been performed in Japan. Herein, we evaluated the efficacy and safety of fenfluramine therapy for Dravet syndrome.</p><p>Methods: We retrospectively examined 5 patients with Dravet syndrome who received fenfluramine at Saitama Children's Medical Center between November 2022 and August 2023. Information on patient background, seizure types, fenfluramine dose, efficacy, and adverse effects was collected from the medical records; seizure frequency and safety up to 6 months after fenfluramine initiation was evaluated.</p><p>Results: Focal to bilateral tonic-clonic seizures (FBTCS) were the predominant seizure type in all patients, with a median frequency of 4 (2-12) seizures/month before fenfluramine therapy. The median age at fenfluramine initiation was 7.6 (2.4-10.9) years, and the median initial dose of fenfluramine was 0.18 (0.09-0.19) mg/kg/day. The monthly FBTCS frequency decreased to 0 (0-3) seizures/month at 6 months after treatment. The median longest FBTCS-free interval was 51 (30-180) days. No serious adverse effects were observed.</p><p>Conclusion: Fenfluramine is safe and effective for FBTCS in Dravet syndrome. Fenfluramine therapy should be prioritized in patients refractory to conventional antiseizure medications.</p>

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