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Bumetanide Treatment in 15 Children With Autism: A Randomised Waitlist-Control Study
Elisabeth Fernell1,2, Sara Landberg1, Carmela Miniscalco1
1Gillberg Neuropsychiatry Centre, University of Gothenburg, Gothenburg, Sweden.
Insights
Bumetanide treatment showed potential benefits for some children with autism, with 4 out of 9 participants experiencing significant improvements. This diuretic influences neuronal chloride homeostasis, potentially aiding autism symptom management.
Area of Science:
- Neuroscience
- Pharmacology
- Developmental Pediatrics
Background:
- Bumetanide, a diuretic, modulates neuronal chloride homeostasis.
- This mechanism may restore GABAergic inhibition and balance neuronal signaling.
- Preclinical and prior clinical research suggests potential benefits for autism spectrum disorder (ASD).
Purpose of the Study:
- To evaluate the efficacy of bumetanide in children with autism.
- To build upon existing preclinical and clinical findings regarding bumetanide for ASD.
- To assess the impact of bumetanide on autism-related symptoms and behaviors.
Main Methods:
- A 9-month randomized waitlist-control study involving 15 children (aged 4-12) diagnosed with autism.
- Participants were randomized to immediate bumetanide treatment or a 3-month delayed start.
- Parent-completed rating scales assessed symptoms, behaviors, and functioning at multiple time points.
Main Results:
- Two children were excluded early due to behavioral issues.
- Four of the 13 participants who started bumetanide discontinued treatment between 4-6 months.
- Four of the 9 children who completed the 9-month study showed significant clinical improvements, opting for continued treatment.
Conclusions:
- This small study suggests bumetanide may offer benefits for a subset of children with autism.
- Further research is warranted to confirm these findings in larger cohorts.
- Bumetanide's potential role in managing autism symptoms warrants continued investigation.
Aim:
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.
Methods:
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.
Results:
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.
Conclusion:
This small, waitlist-control study provides further support that bumetanide may have beneficial effects for some children with autism.
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