Bumetanide for autism: Open-label trial in six children.
Bumetanide for autism: Open-label trial in six children.
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DOI：
10.1111/apa.15723
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发表时间：
2021-05
期刊：
影响因子：
--
通讯作者：
Gillberg C
中科院分区：
文献类型：
--
作者：
Fernell E;Gustafsson P;Gillberg C
布美他尼是一种利尿剂，可减少细胞内氯离子，从而加强GABA能抑制，据报道可改善儿童孤独症的核心症状。鉴于法国布美他尼治疗自闭症儿童的试验报告的积极结果，我们决定在一项更大规模的随机对照研究（RCT）之前，在一项小规模的试点研究中评估其效果。这是一项为期三个月的开放标签布美他尼试验，试验对象为6名3-14岁的自闭症儿童（5名男孩）。在4周和12周后，根据父母满意度调查（PASS）进行评分，以评估症状变化。监测血液电解质状态。在PASS领域“沟通和认知能力”的改善是显着或非常显着的四个孩子，两个有一些改善。很少有不良副作用的报道。我们的小队列对布美他尼反应良好，特别是在“沟通和认知能力”方面。根据来自大规模RCT的证据，我们建议应考虑将布美他尼纳入伦理上批准的自闭症儿童治疗/管理试验，并在大规模RCT中进行严格的随访。
Bumetanide, a diuretic agent, that reduces intracellular chloride—thereby reinforcing GABAergic inhibition—has been reported to improve core symptoms of autism in children. Given the positive results reported from French trials of bumetanide in children with autism, we decided to evaluate its effects in a small‐scale pilot study, in advance of a larger randomised controlled study (RCT). This was an open‐label three‐month trial of bumetanide on six children (five boys), aged 3–14 years with autism. Ratings according to the Parental Satisfaction Survey (PASS) were used after four and twelve weeks to assess symptom change. Blood electrolyte status was monitored. Improvement in the PASS domain “Communicative and cognitive abilities” was marked or very marked in four children, and two had some improvements. Few negative side effects were reported. Our small cohort responded well to bumetanide, particularly with regard to “Communicative and cognitive abilities”. Taken with the evidence from larger‐scale RCTs, we suggest that bumetanide should be considered for inclusion in ethically approved treatment/management trials for children with autism, subject to rigorous follow‐up in large‐scale RCTs.
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