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治疗抵抗性 Dravet 综合征:新型药物的考虑因素。

Treatment-Refractory Dravet Syndrome: Considerations for Novel Medications.

机构信息

Juan Carlos Lopez, PGY-1 Pharmacy Resident, Department of Pharmacy, Boston Medical Center, Boston, MA.

Jasmine R. Pare, PGY-2 Pharmacy Resident, Department of Pharmacy, UCSF Benioff Children's Hospital, San Francisco, CA.

出版信息

J Pediatr Health Care. 2022 Sep-Oct;36(5):479-488. doi: 10.1016/j.pedhc.2022.05.003.

Abstract

Before 2018, there were no U.S. Food and Drug Administration-approved medications for managing seizures in Dravet syndrome (DS). Common agents used in the antiepileptic drug regimens of patients with DS included clobazam, valproic acid, topiramate, and levetiracetam, among others; however, these agents alone rarely provide adequate seizure control. Management of seizures in DS changed in recent years with the approval of cannabidiol and stiripentol in 2018 and fenfluramine in 2020. This continuing education article summarizes available efficacy and safety data involving cannabidiol, stiripentol, and fenfluramine and provides a practical review of dosing strategies, pharmacokinetics, and monitoring interventions relevant to their use.

摘要

在 2018 年之前,美国食品和药物管理局尚未批准任何药物可用于治疗德拉维综合征(Dravet syndrome,DS)的癫痫发作。DS 患者的抗癫痫药物方案中常用的药物包括氯巴占、丙戊酸、托吡酯和左乙拉西坦等;然而,这些药物单独使用时很少能充分控制癫痫发作。近年来,随着 2018 年批准使用大麻二酚和司替戊醇以及 2020 年批准使用芬氟拉明,DS 癫痫发作的治疗方法发生了变化。这篇继续教育文章总结了涉及大麻二酚、司替戊醇和芬氟拉明的现有疗效和安全性数据,并提供了与这些药物使用相关的剂量策略、药代动力学和监测干预的实用综述。

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