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Fenfluramine hydrochloride for the treatment of Dravet syndrome
Expert Opinion on Orphan Drugs ( IF 0.8 ) Pub Date : 2020-04-26 , DOI: 10.1080/21678707.2020.1758930
Blandine Dozières-Puyravel 1 , Stéphane Auvin 1, 2
Affiliation  

Introduction: Fenfluramine (3‐trifluoromethyl‐N‐ethylamphetamine), a former anorectic agent, has been successfully repurposed for Dravet syndrome (DS).

Area covered: A systematic review of data on fenfluramine in the treatment of patients with DS has been conducted, with 11 published papers on the use of fenfluramine for DS (six clinical trials, of which two were randomized controlled trials (RCTs), and five preclinical studies). Following clinical observations suggesting the efficacy of fenfluramine, the effect on convulsive seizures has been confirmed by two RCTs. The first RCT demonstrated the efficacy of the two tested doses of 0.2 mg/kg/day and 0.7 mg/kg/day, while the second RCT showed the efficacy of 0.4 mg/kg/day fenfluramine in patients with DS treated with stiripentol.

Expert opinion: Preclinical studies provide insights into the mechanisms of action of fenfluramine. There are still no large real-life studies. Fenfluramine is under investigation for the treatment of other epilepsy syndromes.



中文翻译:

盐酸芬氟拉明用于治疗Dravet综合征

简介:前厌食药芬氟拉明(3-三氟甲基-N-乙基苯丙胺)已成功用于Dravet综合征(DS)。

涵盖的领域:对芬氟拉明治疗DS患者的数据进行了系统的回顾,发表了11篇关于将芬氟拉明用于DS的论文(六项临床试验,其中两项为随机对照试验(RCT),五项为临床试验)临床前研究)。在临床观察表明芬氟拉明有效后,已通过两项RCT证实了对惊厥性癫痫发作的作用。第一个RCT证明了两种测试剂量0.2 mg / kg /天和0.7 mg / kg /天的功效,而第二个RCT显示了0.4 mg / kg /天的芬氟拉明对替瑞替林治疗的DS患者的功效。

专家意见:临床前研究提供了有关芬氟拉明作用机理的见解。仍然没有大型的现实生活研究。芬氟拉明正在研究用于治疗其他癫痫综合征。

更新日期:2020-04-26
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