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Bumetanide for autism : Open-label trial in six children

Fernell, Elisabeth ; Gustafsson, Peik LU orcid and Gillberg, Christopher (2021) In Acta Paediatrica, International Journal of Paediatrics 110(5). p.1548-1553
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... (More)

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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author
; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
autism, bumetanide, GABA, gamma-amino-butyric acid, parental satisfaction survey
in
Acta Paediatrica, International Journal of Paediatrics
volume
110
issue
5
pages
6 pages
publisher
Wiley-Blackwell
external identifiers
  • scopus:85099989405
  • pmid:33336381
ISSN
0803-5253
DOI
10.1111/apa.15723
language
English
LU publication?
yes
id
2742de5b-6746-4384-b546-5133d2e8b498
date added to LUP
2022-03-01 14:49:47
date last changed
2024-06-13 11:10:52
@article{2742de5b-6746-4384-b546-5133d2e8b498,
  abstract     = {{<p>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.</p>}},
  author       = {{Fernell, Elisabeth and Gustafsson, Peik and Gillberg, Christopher}},
  issn         = {{0803-5253}},
  keywords     = {{autism; bumetanide; GABA; gamma-amino-butyric acid; parental satisfaction survey}},
  language     = {{eng}},
  number       = {{5}},
  pages        = {{1548--1553}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{Acta Paediatrica, International Journal of Paediatrics}},
  title        = {{Bumetanide for autism : Open-label trial in six children}},
  url          = {{http://dx.doi.org/10.1111/apa.15723}},
  doi          = {{10.1111/apa.15723}},
  volume       = {{110}},
  year         = {{2021}},
}