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Neurology:这个镇咳药治疗Rett综合征效果喜人

2017-10-13 吴星 环球医学

2017年9月,发表在《Neurology》一项由美国科学家进行的右美沙芬在Rett综合征中的随机开放性试验表明,右美沙芬在年轻Rett综合征女孩中的使用是安全的。

2017年9月,发表在《Neurology》一项由美国科学家进行的右美沙芬在Rett综合征中的随机开放性试验表明,右美沙芬在年轻Rett综合征女孩中的使用是安全的。

目的:旨在确定Rett综合征的女孩中,右美沙芬在规范电尖峰、临床癫痫、行为和认知功能中的安全性,并对有效性的剂量效应进行初步评估。

方法:研究者在右美沙芬快速代谢者中进行了前瞻性、随机、开放性试验,旨在检测右美沙芬对Rett综合征的核心临床特征的影响。发作间期棘波活动和临床癫痫使用EEG和父母报告的方式进行确定。认知功能数据使用早期学习和Vineland适应行为量表的Mullen量表获取,而行为数据通过父母完成的清单、异常行为量表的社区版、社交互动筛查等获取。人体测量数据根据全国健康和营养检查调查获取。Rett综合征严重程度量表提供了右美沙芬对临床严重程度作用的临床总体印象。

结果:3~15岁的Rett综合征女孩使用右美沙芬是安全的。35名女孩在6个月期间,接受了3种右美沙芬给药剂量中的1种。临床癫痫、接受语言、行为多动等方面,改善情况存在统计学显着性的剂量效应。Rett综合征严重程度量表测量的总体临床严重程度没有显着性改善。

结论:右美沙芬是一种有效的非竞争性NMDA受体通道的拮抗剂,在Rett综合征年轻女孩患者中的使用是安全的。初步证据表明,右美沙芬可改善Rett综合征的一些核心特征。

原始出处:

Smith-Hicks CL, Gupta S, Ewen JB, et al. Randomized open-label trial of dextromethorphan in Rett syndrome. Neurology. 2017 Sep 20. pii: 10.1212/WNL.0000000000004515. doi: 10.1212/WNL.0000000000004515. [Epub ahead of print]

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    2017-11-19 yinhl1978
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    2018-01-05 ysjykql
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    2017-10-13 hhh678

    henhao

    0

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    2017-10-13 changjiu

    学习了.谢谢

    0

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