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采用大麻二酚治疗 Dravet 综合征和 Lennox-Gastaut 综合征的新方法。

New therapeutic approach in Dravet syndrome and Lennox-Gastaut syndrome with cannabidiol.

机构信息

Hospital Universitario y Politécnico La Fe, Valencia, España.

Hospital Clínico y Provincial de Barcelona, Barcelona, España.

出版信息

Rev Neurol. 2021 Apr 30;72(S01):S1-S10. doi: 10.33588/rn.72S01.2021017.

Abstract

INTRODUCTION

Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are two serious epileptic syndromes with paediatric onset which are refractory to therapy and are associated with an important increase in mortality rates and comorbidities compared to the general population. These pathologies have a strong impact on the lives of patients and their families, because they undergo multiple pharmacological therapies (many of them without specific indication), with frequent changes due to poor efficacy and associated adverse effects. The specialists who care for these patients highlight unmet needs and the lack of specific, safe and effective treatments for better management of the syndrome.

DEVELOPMENT

A group of four neurologists specializing in epilepsy has met to review the scientific literature and evaluate the efficacy and safety of oral solution cannabidiol in the treatment of these syndromes, both in randomized clinical trials (CT) and in some observational studies.

CONCLUSIONS

Cannabidiol is positioned as an innovative therapy that allows better control of epileptic seizures and comorbidities of DS and LGS, furthermore its efficacy and safety have been evaluated in more than 700 patients. In CTs, cannabidiol significantly reduced the percentage of convulsive seizures and drop seizures compared to placebo in patients with DS and LGS respectively, which could improve their quality of life and that of their family members. The most frequent adverse effects reported were somnolence and decreased appetite. Elevated liver aminotransferase levels were also reported, especially in patients given concomitant sodium valproate. This therapy may allow better control of the epileptic seizures associated with these syndromes.

摘要

简介

婴儿痉挛症(DS)和 Lennox-Gastaut 综合征(LGS)是两种严重的癫痫综合征,发病于儿童期,对治疗有抗性,与一般人群相比,死亡率和合并症的发生率显著增加。这些疾病对患者及其家属的生活有很大的影响,因为他们经历了多种药物治疗(其中许多没有特定的适应证),由于疗效不佳和相关不良反应,经常需要改变治疗方案。照顾这些患者的专家强调存在未满足的需求,以及缺乏针对这些综合征的具体、安全和有效的治疗方法,以更好地管理这些疾病。

进展

一组四位专门研究癫痫的神经病学家开会审查了科学文献,并评估了口服溶液型大麻二酚在治疗这些综合征中的疗效和安全性,包括随机对照试验(CT)和一些观察性研究。

结论

大麻二酚是一种创新的治疗方法,可更好地控制 DS 和 LGS 的癫痫发作和合并症,此外,已有超过 700 名患者评估了其疗效和安全性。在 CT 中,与安慰剂相比,大麻二酚分别显著降低了 DS 和 LGS 患者的惊厥性发作和跌倒发作的百分比,从而提高了他们和其家庭成员的生活质量。报告的最常见不良反应是嗜睡和食欲下降。还报告了肝氨基转移酶水平升高,尤其是在同时给予丙戊酸钠的患者中。这种治疗方法可能可以更好地控制这些综合征相关的癫痫发作。

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