Cited by 34Autism Spectrum Disorder ResearchGenetics and Neurodevelopmental DisordersChild Nutrition and Feeding Issues

Bumetanide for autism: open-label trial in six children

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Fernell, Elisabeth, Gustafsson, Peik, Gillberg, Christopher·2021·Acta Paediatrica

Abstract

AIM: 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). METHODS: 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. RESULTS: 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. CONCLUSION: 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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  • An open-label trial of 6 children with severe ASD and intellectual disability showed parent-reported improvement in communicative abilities of all children after 3 months of bumetanide.

    “Another open-label trial of 6 children with severe ASD and intellectual disability showed parent-reported improvement in communicative abilities of all children after 3 months of bumetanide [96].”

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  1. [1]Coleman M, Gillberg C. The autisms. Oxford: Oxford University Press; 2012.

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Authors
Fernell, E., Gustafsson, P., & Gillberg, C.
Affiliations
  • Sahlgrenska University Hospital (SE)
  • Lund University (SE)
Corresponding author
Fernell, Elisabeth
Venue
Acta Paediatrica
Publisher
Wiley
Published
2021-01-21
Volume
110
Issue
5
Pages
1548-1553
ISSN
  • 0365-1436 (ISSN-L)
  • 0803-5253
  • 1651-2227
OpenAlex
W3116942098
Cited by
34
References
21
Citations by year
2026: 3 · 2025: 4 · 2024: 9 · 2023: 4 · 2022: 7 · 2021: 7
FWCI
2.42
Type
journal article
Language
en
Open access
Locations
  • Acta Paediatrica (publishedVersion, cc-by-nc-nd)
  • PubMed Central (publishedVersion, cc-by-nc-nd)
  • PubMed (publishedVersion)
  • Lund University Publications (Lund University) (submittedVersion)
Topics
  • Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
  • Health Sciences › Medicine › Psychiatry and Mental health › Child Nutrition and Feeding Issues
Keywords
bumetanide, autism spectrum disorder, diuretics, chloride reduction, GABAergic inhibition, communication skills, cognitive ability, children, electrolyte monitoring
MeSH terms
Adolescent, Autistic Disorder, Bumetanide, Child, Child, Preschool, Humans, Male, Parents, Pilot Projects

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