布美他尼,一种能帮助自闭症谱系障碍儿童的利尿剂。

IF 2.7 4区 医学 Q3 NEUROSCIENCES CNS & neurological disorders drug targets Pub Date : 2024-01-01 DOI:10.2174/1871527322666230404114911
Esraa Shaker, Osama El Agami, Abeer Salamah
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引用次数: 0

摘要

背景:自闭症谱系障碍(ASD)是一种常见的儿童神经发育障碍,其发病机制尚未完全明了。到目前为止,还没有针对自闭症核心症状的行之有效的治疗方法。然而,一些证据表明,这种疾病与 GABA 能信号之间存在重要联系,而 GABA 能信号在 ASD 中发生了改变。布美他尼是一种利尿剂,能减少氯化物,使γ-氨基丁酸(GABA)从兴奋状态转为抑制状态,可能在治疗ASD方面发挥重要作用:本研究旨在评估布美他尼治疗ASD的安全性和有效性:这项双盲、随机对照研究共纳入了80名年龄在3-12岁、经儿童自闭症评定量表(CARS)诊断为自闭症的儿童。第一组接受布美他尼治疗,第二组接受安慰剂治疗,为期 6 个月。在治疗前和治疗 1、3、6 个月后,采用 CARS 评分量表进行随访:结果:第一组使用布美他尼在较短时间内改善了 ASD 的主要症状,不良反应极小且可耐受。治疗 6 个月后,第 1 组与第 2 组相比,CARS 及其 15 个项目中的大多数项目均有统计学意义上的显著下降(P 值 结论:布美他尼在改善 ASD 核心症状方面具有重要作用:布美他尼在治疗 ASD 核心症状方面具有重要作用。
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Bumetanide, a Diuretic That Can Help Children with Autism Spectrum Disorder.

Background: Autism Spectrum Disorder (ASD) is a common child neurodevelopmental disorder, whose pathogenesis is not completely understood. Until now, there is no proven treatment for the core symptoms of ASD. However, some evidence indicates a crucial link between this disorder and GABAergic signals which are altered in ASD. Bumetanide is a diuretic that reduces chloride, shifts gamma-amino-butyric acid (GABA) from excitation to inhibition, and may play a significant role in the treatment of ASD.

Objective: The objective of this study is to assess the safety and efficacy of bumetanide as a treatment for ASD.

Methods: Eighty children, aged 3-12 years, with ASD diagnosed by Childhood Autism Rating Scale (CARS), ⩾ 30 were included in this double-blind, randomized, and controlled study. Group 1 received Bumetanide, Group 2 received a placebo for 6 months. Follow-up by CARS rating scale was performed before and after 1, 3, and 6 months of treatment.

Results: The use of bumetanide in group 1 improved the core symptoms of ASD in a shorter time with minimal and tolerable adverse effects. There was a statistically significant decrease in CARS and most of its fifteen items in group 1 versus group 2 after 6 months of treatment (p-value <0.001).

Conclusion: Bumetanide has an important role in the treatment of core symptoms of ASD.

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来源期刊
CiteScore
5.10
自引率
3.30%
发文量
158
审稿时长
6-12 weeks
期刊介绍: Aims & Scope CNS & Neurological Disorders - Drug Targets aims to cover all the latest and outstanding developments on the medicinal chemistry, pharmacology, molecular biology, genomics and biochemistry of contemporary molecular targets involved in neurological and central nervous system (CNS) disorders e.g. disease specific proteins, receptors, enzymes, genes. CNS & Neurological Disorders - Drug Targets publishes guest edited thematic issues written by leaders in the field covering a range of current topics of CNS & neurological drug targets. The journal also accepts for publication original research articles, letters, reviews and drug clinical trial studies. As the discovery, identification, characterization and validation of novel human drug targets for neurological and CNS drug discovery continues to grow; this journal is essential reading for all pharmaceutical scientists involved in drug discovery and development.
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