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新疗法在发育性和癫痫性脑病管理中的作用:新概念和结果。

The role of new medical treatments for the management of developmental and epileptic encephalopathies: Novel concepts and results.

机构信息

Program for Pharmacy, Department of Life Sciences and Health, Faculty of Health Sciences, Oslo Metropolitan University, Oslo, Norway.

National Center for Epilepsy, Oslo University Hospital, Oslo, Norway.

出版信息

Epilepsia. 2021 Apr;62(4):857-873. doi: 10.1111/epi.16849. Epub 2021 Feb 27.

Abstract

Developmental and epileptic encephalopathies (DEEs) are among the most challenging of all epilepsies to manage, given the exceedingly frequent and often severe seizure types, pharmacoresistance to conventional antiseizure medications, and numerous comorbidities. During the past decade, efforts have focused on development of new treatment options for DEEs, with several recently approved in the United States or Europe, including cannabidiol as an orphan drug in Dravet and Lennox-Gastaut syndromes and everolimus as a possible antiepileptogenic and precision drug for tuberous sclerosis complex, with its impact on the mammalian target of rapamycin pathway. Furthermore, fenfluramine, an old drug, was repurposed as a novel therapy in the treatment of Dravet syndrome. The evolution of new insights into pathophysiological processes of various DEEs provides possibilities to investigate novel and repurposed drugs and to place them into the context of their role in future management of these patients. The purpose of this review is to provide an overview of these new medical treatment options for the DEEs and to discuss the clinical implications of these results for improved treatment.

摘要

发育性和癫痫性脑病 (DEE) 是所有癫痫中最难治疗的,因为其发作类型极其频繁且通常严重,对传统抗癫痫药物有抗药性,并且存在许多合并症。在过去的十年中,人们致力于为 DEE 开发新的治疗选择,其中一些已在美国或欧洲获得批准,包括用于 Dravet 和 Lennox-Gastaut 综合征的大麻二酚作为孤儿药,以及依维莫司作为一种可能的抗癫痫发生和精准药物用于结节性硬化症,其对哺乳动物雷帕霉素靶蛋白途径有影响。此外,一种老药芬氟拉明被重新用作治疗 Dravet 综合征的新疗法。对各种 DEE 病理生理过程的新见解的发展为研究新型和再利用药物提供了可能性,并将它们置于这些患者未来治疗中的作用背景下。本文旨在概述这些 DEE 的新的医疗治疗选择,并讨论这些结果对改善治疗的临床意义。

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