评估盐酸芬氟拉明口服溶液用于治疗与伦诺克斯-加斯塔特综合征相关的癫痫发作。

IF 3.4 2区 医学 Q2 CLINICAL NEUROLOGY Expert Review of Neurotherapeutics Pub Date : 2024-03-01 Epub Date: 2024-02-07 DOI:10.1080/14737175.2024.2313548
Frank M C Besag, Michael J Vasey, Richard F M Chin
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引用次数: 0

摘要

简介伦诺克斯-加斯托特综合征(Lennox-Gastaut Syndrome,LGS)是一种严重的儿童期发育性癫痫脑病,其特征是难治性癫痫发作,包括强直/失张力 "跌倒 "发作、智力障碍和脑电图上的慢尖波放电。芬氟拉明以前曾作为减肥药被处方使用,但后来被撤消,最近在美国、欧盟和英国被批准用于LGS相关癫痫发作的辅助治疗:作者回顾了芬氟拉明治疗 LGS 临床试验的疗效和安全性。然后,作者结合芬氟拉明用于治疗 LGS 难治性癫痫发作的情况,讨论了芬氟拉明可能引起特别关注的不良反应的证据,即心脏异常和体重减轻:专家意见:在短期和长期临床试验中,芬氟拉明对降低LGS患者的癫痫发作频率,尤其是减少跌倒发作具有疗效。在这些研究中使用低剂量(≤26 毫克/天)时,尚未发现瓣膜性心脏病和肺动脉高压的报道,但数据有限。由于芬氟拉明的作用机制新颖,它可能对其他抗癫痫药物反应不佳的 LGS 患者有益。然而,在大多数病例中,包括芬氟拉明在内的所有这些药物都无法实现摆脱癫痫发作的最终目标。
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Evaluating fenfluramine hydrochloride as an oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome.

Introduction: Lennox-Gastaut syndrome (LGS) is a severe childhood-onset developmental and epileptic encephalopathy characterized by treatment-refractory seizures, including tonic/atonic 'drop' seizures, and intellectual impairment and slow spike-wave discharges on the electroencephalogram. Fenfluramine, previously prescribed as a weight-loss drug but then withdrawn, has recently been approved in the US, EU, and UK for the adjunct treatment of seizures associated with LGS.

Areas covered: The authors review the efficacy and safety findings from clinical trials of fenfluramine in LGS. The authors then discuss the evidence for adverse effects that may be of particular concern to fenfluramine, namely cardiac abnormalities, and weight loss, in the context of the use of fenfluramine for the treatment of the refractory seizures in LGS.

Expert opinion: Fenfluramine has demonstrated efficacy in reducing the frequency of seizures in LGS, notably drop seizures, in short-term and long-term clinical trials. Valvular heart disease and pulmonary hypertension have not been reported at the low doses (≤26 mg/day) used in these studies, however, data are limited. Due to its novel mechanism of action, fenfluramine may be of benefit in LGS which has not responded adequately to other antiseizure medications. However, none of these medications, including fenfluramine, achieves the ultimate goal of seizure freedom in most cases.

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来源期刊
Expert Review of Neurotherapeutics
Expert Review of Neurotherapeutics Medicine-Neurology (clinical)
CiteScore
7.00
自引率
2.30%
发文量
61
审稿时长
4-8 weeks
期刊介绍: Expert Review of Neurotherapeutics (ISSN 1473-7175) provides expert reviews on the use of drugs and medicines in clinical neurology and neuropsychiatry. Coverage includes disease management, new medicines and drugs in neurology, therapeutic indications, diagnostics, medical treatment guidelines and neurological diseases such as stroke, epilepsy, Alzheimer''s and Parkinson''s. Comprehensive coverage in each review is complemented by the unique Expert Review format and includes the following sections: Expert Opinion - a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points
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