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Improved symptoms following bumetanide treatment in children aged 3 to 6 years with autism spectrum disorder: a randomized, double-blind, placebo-controlled trial

By Yuan Dai, Lingli Zhang, Juehua Yu, Xin Zhou, Hua He, Yiting Ji, Kai Wang, Xiujuan Du, Xin Liu, Jing Tang, Shining Deng, Christelle Langley, Wei-Guang Li, Jun Zhang, Jianfeng Feng, Barbara J Sahakian, Qiang Luo, Fei Li

Posted 22 Sep 2020
medRxiv DOI: 10.1101/2020.09.18.20197640

With the drug therapy for the core symptoms of autism spectrum disorder (ASD) currently limited, here we reported a randomised, double-blind, placebo-controlled trial to investigate the efficacy, safety, and potential neural mechanism of bumetanide in children with ASD aged 3 to 6 years old. There were 120 children entered into the study and randomly assigned to either 0.5mg bumetanide or placebo. In the final sample, 119 received at least one dose of bumetanide (59 children) or placebo (60 children). The primary outcome was the score reduction of Childhood Autism Rating Scale (CARS) and the secondary outcomes were the score of Clinical Global Impressions Scale (CGI) -Global Improvement (CGI-I) at 3 months and the change from baseline to 3-month in Autism Diagnostic Observation Schedule (ADOS). Magnetic resonance spectroscopy (MRS) was used to measure {gamma}-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 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 the 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.

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