Publication date: Jun 26, 2025

Bumetanide, a diuretic, influences neuronal chloride homeostasis and potentially restores GABAergic inhibition and neuronal signalling balance. This mechanism may contribute to improvements in autism-related symptoms. The present study builds on the preclinical research and on previous clinical research on bumetanide treatment for children with autism. Fifteen children (10 boys, 5 girls), aged 4-12 years with a clinically confirmed autism diagnosis, with and without intellectual disability and with and without ADHD were enrolled in this 9-month randomised waitlist-control study. Participants were randomly assigned to either begin bumetanide treatment immediately or wait 3 months before starting. Parents completed rating scales assessing symptoms, behaviours and functioning at baseline and after 3, 6 and 9 months. Two children had to be excluded in the first treatment period with bumetanide due to emerging behavioural problems. Of the remaining 13, 4 discontinued after the 4-6 months period with bumetanide, and 9 completed the full 9-month study. According to parent reports, 4 of these 9 children experienced significant clinical improvements, leading parents to opt for continued bumetanide treatment for their child after study completion. This small, waitlist-control study provides further support that bumetanide may have beneficial effects for some children with autism.

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