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

• DOI: 10.1016/j.scib.2021.01.008
• Publication Date: 2021-01-18T07:37:00Z
• Authors: Yuan Dai; Lingli Zhang; Juehua Yu; Xin Zhou; Hua He; Yiting Ji; Kai Wang; Xiujuan Du; Xin Liu; Yun Tang; Shining Deng; Christelle Langley; Wei-Guang Li; Jun Zhang; Jianfeng Feng; Barbara J. Sahakian; Qiang Luo; Fei Li
• Keywords: Bumetanide; Tolerability

With the current limited drug therapy for core symptoms of autism spectrum disorder (ASD), we herein report a randomized, double-blind, placebo-controlled trial to investigate efficacy, safety, and potential neural mechanism bumetanide in children with ASD aged 3−6 years old. A total 120 were enrolled into study randomly assigned either 0.5 mg or placebo. In final sample, 119 received at least one dose (59 children) placebo (60 included analysis. The primary outcome was reduction Childhood Autism Rating Scale (CARS) score, secondary outcomes Clinical Global Impressions (CGI) -Global Improvement (CGI-I) score 3 months change from baseline 3-month Diagnostic Observation Schedule (ADOS). Magnetic resonance spectroscopy (MRS) used measure γ-aminobutyric acid (GABA) glutamate neurotransmitter concentrations insular cortex (IC) before after treatment. As compared placebo, treatment significantly better reducing severity. No patient withdrew due adverse events. superiority GABA, measured using MRS, demonstrated. clinical improvement associated decrease GABA group. conclusion, this large group young predominantly moderate severe demonstrated that is safe effective improving ASD. However, significance remains uncertain, future multi-center trials are required replicate these findings confirm variety measures.