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布美他尼治疗自闭症:针对特定人群的大型多中心和单中心试验是否具有互补性?

Treating Autism With Bumetanide: Are Large Multicentric and Monocentric Trials on Selected Populations Complementary?

机构信息

Autism Expert Center and Autism Resource Center of Limousin, University Hospital Center, Limoges, France.

Neurochlore, Luminy Scientific Campus, Marseille, France.

出版信息

J Am Acad Child Adolesc Psychiatry. 2021 Aug;60(8):937-938. doi: 10.1016/j.jaac.2020.09.025. Epub 2020 Dec 29.

DOI:10.1016/j.jaac.2020.09.025
PMID:33385505
Abstract

In their article in the Journal, Sprengers et al. conclude that bumetanide does not attenuate autism spectrum disorder (ASD) despite a nominally significant treatment effect in repetitive behaviors, which is a core symptom of ASD but was defined as a secondary measure in this trial. Four earlier studies performed by 3 independent institutes, including 2 studies not mentioned by Sprengers et al., testing a total of 169 children (versus 122 placebo) showed a significant amelioration of ASD symptoms. Bumetanide also significantly attenuated behavioral features of patients with tuberous sclerosis according to another study by Sprengers' same group..

摘要

在他们发表在《期刊》上的文章中,Sprengers 等人得出结论,布美他尼并没有减轻自闭症谱系障碍(ASD),尽管在重复行为方面有明显的治疗效果,这是 ASD 的核心症状,但在这项试验中被定义为次要措施。包括 Sprengers 等人未提及的两项研究在内的三个独立研究所进行的四项早期研究,共测试了 169 名儿童(与 122 名安慰剂组相比),结果显示 ASD 症状有显著改善。根据 Sprengers 同一团队的另一项研究,布美他尼还显著减轻了结节性硬化症患者的行为特征。

相似文献

1
Treating Autism With Bumetanide: Are Large Multicentric and Monocentric Trials on Selected Populations Complementary?布美他尼治疗自闭症:针对特定人群的大型多中心和单中心试验是否具有互补性?
J Am Acad Child Adolesc Psychiatry. 2021 Aug;60(8):937-938. doi: 10.1016/j.jaac.2020.09.025. Epub 2020 Dec 29.
2
Editorial: Targeting the Core Symptoms of Autism Spectrum Disorder With Mechanism-Based Medications.社论:以基于机制的药物靶向自闭症谱系障碍的核心症状。
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Bumetanide for Core Symptoms of Autism Spectrum Disorder (BAMBI): A Single Center, Double-Blinded, Participant-Randomized, Placebo-Controlled, Phase-2 Superiority Trial.布美他尼治疗孤独症谱系障碍核心症状(BAMBI):一项单中心、双盲、随机、安慰剂对照、2 期优效性试验。
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Dr. Sprengers et al. Reply.施普伦杰斯博士等人的回复。
J Am Acad Child Adolesc Psychiatry. 2021 Aug;60(8):938-939. doi: 10.1016/j.jaac.2020.12.034. Epub 2021 Jan 12.
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Bumetanide, a Diuretic That Can Help Children with Autism Spectrum Disorder.布美他尼,一种可帮助自闭症谱系障碍儿童的利尿剂。
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Improved symptoms following bumetanide treatment in children aged 3-6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial.布美他尼治疗3至6岁自闭症谱系障碍儿童后症状改善:一项随机、双盲、安慰剂对照试验。
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Bumetanide for Autism Spectrum Disorder in Children: A Review of Randomized Controlled Trials.布美他尼治疗儿童自闭症谱系障碍的随机对照试验综述。
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Bumetanide oral solution for the treatment of children and adolescents with autism spectrum disorder: Results from two randomized phase III studies.布美他尼口服液治疗儿童和青少年自闭症谱系障碍的疗效:两项随机 III 期研究结果。
Autism Res. 2023 Oct;16(10):2021-2034. doi: 10.1002/aur.3005. Epub 2023 Oct 4.
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Effects of Bumetanide on Neurocognitive Functioning in Children with Autism Spectrum Disorder: Secondary Analysis of a Randomized Placebo-Controlled Trial.布美他尼对自闭症谱系障碍儿童神经认知功能的影响:一项随机安慰剂对照试验的二次分析。
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Symptom improvement in children with autism spectrum disorder following bumetanide administration is associated with decreased GABA/glutamate ratios.在接受布美他尼治疗后,自闭症谱系障碍儿童的症状改善与 GABA/谷氨酸比值降低有关。
Transl Psychiatry. 2020 Jan 27;10(1):9. doi: 10.1038/s41398-020-0692-2.