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DOI10.1016/j.scib.2021.01.008
Improved symptoms following bumetanide treatment in children aged 3−6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial
Dai Y.; Zhang L.; Yu J.; Zhou X.; He H.; Ji Y.; Wang K.; Du X.; Liu X.; Tang Y.; Deng S.; Langley C.; Li W.-G.; Zhang J.; Feng J.; Sahakian B.J.; Luo Q.; Li F.
发表日期2021
ISSN20959273
起始页码1591
结束页码1598
卷号66期号:15
英文摘要With the current limited drug therapy for the core symptoms of autism spectrum disorder (ASD), we herein report a randomized, double-blind, placebo-controlled trial to investigate the efficacy, safety, and potential neural mechanism of bumetanide in children with ASD aged 3−6 years old. A total of 120 children were enrolled into the study and randomly assigned to either 0.5 mg bumetanide or placebo. In the final sample, 119 children received at least one dose of bumetanide (59 children) or placebo (60 children) were included in the final analysis. The primary outcome was a reduction in the Childhood Autism Rating Scale (CARS) score, and the secondary outcomes were the Clinical Global Impressions Scale (CGI) -Global Improvement (CGI-I) score at 3 months and the change from baseline to 3-month in the Autism Diagnostic Observation Schedule (ADOS). Magnetic resonance spectroscopy (MRS) was used to measure γ-aminobutyric acid (GABA) and glutamate neurotransmitter concentrations in the insular cortex (IC) before and after the treatment. As compared with the placebo, bumetanide treatment was significantly better in reducing the severity. No patient withdrew from the trial due to adverse events. The superiority of bumetanide to placebo in reducing insular GABA, measured using MRS, was demonstrated. The clinical improvement was associated with a decrease in insular GABA in the bumetanide group. In conclusion, this trial in a large group of young children with predominantly moderate and severe ASD demonstrated that bumetanide is safe and effective in improving the core symptoms of ASD. However, the clinical significance remains uncertain, and future multi-center clinical trials are required to replicate these findings and confirm the clinical significance using a variety of outcome measures. © 2021 Science China Press
关键词Autism spectrum disorderBumetanideChildrenGABARCT
英文关键词Amino acids; Controlled drug delivery; Magnetic resonance; Magnetic resonance spectroscopy; Autism spectrum disorders; Controlled trial; Gamma-aminobutyric acids; Magnetic resonance spectroscopies (MRS); Multi-center clinical trials; Neural mechanisms; Outcome measures; Young children; Diseases
语种英语
来源期刊Science Bulletin
文献类型期刊论文
条目标识符http://gcip.llas.ac.cn/handle/2XKMVOVA/207381
作者单位Department of Developmental and Behavioural Pediatric & Child Primary Care, Brain and Behavioural Research Unit of Shanghai Institute for Pediatric Research and MOE-Shanghai Key Laboratory for Children's Environmental Health, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200092, China; Centre for Experimental Studies and Research, The First Affiliated Hospital of Kunming Medical University, Kunming, 650032, China; Clinical Research Unit, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200092, China; The School of Psychology and Cognitive Science, East China Normal University, Shanghai, 200062, China; Department of Psychiatry and the Behavioural and Clinical Neuroscience Institute, University of Cambridge, Cambridge, CB21TN, United Kingdom; Collaborative Innovation Center for Brain Science, Department of Anatomy and Physiology, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China; Institute of Science and Technology for Brain-...
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GB/T 7714
Dai Y.,Zhang L.,Yu J.,et al. Improved symptoms following bumetanide treatment in children aged 3−6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial[J],2021,66(15).
APA Dai Y..,Zhang L..,Yu J..,Zhou X..,He H..,...&Li F..(2021).Improved symptoms following bumetanide treatment in children aged 3−6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial.Science Bulletin,66(15).
MLA Dai Y.,et al."Improved symptoms following bumetanide treatment in children aged 3−6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial".Science Bulletin 66.15(2021).
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