UMC Utrecht Brain Centre, University Medical Centre Utrecht, The Netherlands.
UMC Utrecht Brain Centre, University Medical Centre Utrecht, The Netherlands; Amsterdam UMC, Vrije Universiteit Amsterdam, N=You centre, Amsterdam Neuroscience, Amsterdam Reproduction and Development, The Netherlands.
J Am Acad Child Adolesc Psychiatry. 2021 Aug;60(8):938-939. doi: 10.1016/j.jaac.2020.12.034. Epub 2021 Jan 12.
Before we elaborate on the postulated discrepancies between our trial and previous bumetanide in autism spectrum disorder (ASD) trials, we would like to acknowledge the crucial pioneering work on the γ-aminobutyric acid (GABA) developmental sequence by Dr. Ben-Ari and colleagues. Chloride dysregulation and altered GABA polarity have been implicated in neurological and neurodevelopmental disorders, including some forms of ASD. Etiologies underlying ASD are profoundly heterogeneous, and an important challenge is to link the optimal treatment to individual patients. Indeed, ASD animal models indicate reversed GABA polarity as a treatment target in some, but not all, studies. The aim of the Bumetanide in Autism Medication and Biomarker (BAMBI) trial was to replicate previous trial findings and to develop stratification biomarkers that may help to understand expected variability in treatment response.
在详细阐述我们的试验与之前在自闭症谱系障碍（ASD）试验中使用布美他尼之间假定的差异之前，我们想先感谢 Dr. Ben-Ari 和同事们在γ-氨基丁酸（GABA）发育序列方面的开创性工作。氯离子失调和 GABA 极性改变与神经和神经发育障碍有关，包括某些形式的 ASD。ASD 的病因非常复杂，一个重要的挑战是将最佳治疗方法与个体患者联系起来。事实上，ASD 动物模型表明，在某些但不是所有研究中，反转 GABA 极性是一种治疗靶点。布美他尼在自闭症药物和生物标志物（BAMBI）试验的目的是复制以前的试验结果，并开发分层生物标志物，以帮助了解治疗反应的预期变异性。
