Gillberg Neuropsychiatry Centre, Institute of Neuroscience and Physiology, Sahlgrenska Academy, Gothenburg University, Gothenburg, Sweden.
Child Neuropsychiatry Clinic, Sahlgrenska University Hospital, Gothenburg, Sweden.
Acta Paediatr. 2021 May;110(5):1548-1553. doi: 10.1111/apa.15723. Epub 2021 Jan 21.
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.
布美他尼是一种利尿剂，可减少细胞内氯离子，从而增强 GABA 能抑制，据报道可改善儿童自闭症的核心症状。鉴于法国对自闭症儿童布美他尼试验的阳性结果，我们决定在更大规模的随机对照试验 (RCT) 之前，先进行小规模的试点研究，评估其疗效。
这是一项为期三个月的开放性标签布美他尼试验，纳入 6 名（5 男）年龄 3-14 岁的自闭症儿童。治疗 4 周和 12 周后，采用父母满意度调查 (PASS) 进行评分，以评估症状变化。监测血液电解质状态。
4 名儿童的 PASS 领域“交流和认知能力”明显或非常明显改善，2 名儿童有一些改善。报告的不良反应很少。
我们的小队列对布美他尼反应良好，尤其是在“交流和认知能力”方面。结合更大规模 RCT 的证据，我们建议应考虑在伦理批准的自闭症儿童治疗/管理试验中纳入布美他尼，但需在更大规模 RCT 中进行严格随访。
