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首页> 外文期刊>Drugs of today: Medicamentos de actualidad >Fenfluramine for treatment-resistant epilepsy in Dravet syndrome and other genetically mediated epilepsies
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Fenfluramine for treatment-resistant epilepsy in Dravet syndrome and other genetically mediated epilepsies

机译：在Dravet综合征和其他遗传介导的癫痫中的治疗癫痫发育抗氟胺

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摘要

Fenfluramine hydrochloride, initially utilized as a weight loss drug in the 1970s and later removed from the market for adverse cardiopulmonary side effects, has since been repurposed as an antiseizure medicine (ASM). The potential antiseizure effects of fenfluramine were first identified in patients with photosensitive epilepsy in the 1980s but it was not rigorously explored as a treatment option until 30 years later. Compared with other ASMs, fenfluramine offers a novel mechanism by acting on serotonin and sigma 1 receptors, demonstrated in vitro and in vivo in animal models of Dravet syndrome. Results from a large double-blind, placebo-controlled trial demonstrated robust efficacy for seizure reduction in patients with Dravet syndrome, and met its primary endpoint with the 0.7 mg/kg/day fenfluramine treatment group experiencing a 62.3% or greater reduction in mean monthly convulsive seizure frequency (MCSF) compared with placebo. Here we provide a comprehensive review of the preclinical and clinical activity of fenfluramine, a recently approved drug for treatment of epilepsy in patients with Dravet syndrome.
机译：芬氟拉明盐酸盐最初在20世纪70年代用作减肥药，后来因不良的心肺副作用而从市场上退出，此后被重新用作抗利尿药（ASM）。芬氟拉明的潜在抗癫痫作用于20世纪80年代首次在光敏性癫痫患者中发现，但直到30年后才被严格探索作为一种治疗选择。与其他ASM相比，芬氟拉明通过作用于血清素和sigma 1受体提供了一种新的机制，这在Dravet综合征的动物模型中得到了体外和体内证明。一项大型双盲安慰剂对照试验的结果显示，与安慰剂相比，0.7 mg/kg/天芬氟拉明治疗组的月平均惊厥发作频率（MCSF）降低62.3%或更高，对Dravet综合征患者的癫痫发作减少有显著疗效，并达到其主要终点。在这里，我们对芬氟拉明的临床前和临床活性进行了全面综述。芬氟拉明是最近批准用于治疗德拉韦特综合征患者癫痫的药物。

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