Evidence map›Paper›PMID 23233021›Full record

Trial reportTranslational psychiatry2012

A randomised controlled trial of bumetanide in the treatment of autism in children.

E Lemonnier, C Degrez, M Phelep, R Tyzio, F Josse, M Grandgeorge, N Hadjikhani, Y Ben-Ari

Registry-linked trialOpen access · goldAbstract readRandomized Controlled TrialVideo-Audio Media
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Translational psychiatry, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06465823 (A Phase 2 Double Blind Placebo Controlled Study on the Efficacy of Bumetanide for Cognitive Improvement in Children and Adolescents With Down Syndrome), which is not on this map. Cited by 154 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
154citing papers in PubMed, 7 pooled it
10.3field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06465823 phase2recruitingnot on this mapstarted 2023, after this paper: background citation

A Phase 2 Double Blind Placebo Controlled Study on the Efficacy of Bumetanide for Cognitive Improvement in Children and Adolescents With Down Syndrome

TypeinterventionalSponsorStefano VicariRan2023 to 2026Enrolled64ConditionsDown SyndromeArmsBumetanide, Placebo
3 · Its place in the literature

Who cites it

154 citing papers in PubMed, 7 syntheses or guidelines pooled it, 303 citations in OpenAlex.

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  20. Role of GABAMolecular biology reports · 2026
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94 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 3 institutions in 3 countries.

E LemonnierCentre de Ressources Autisme de Bretagne, CHRU Brest Hôpital Bohars, Route de Ploudalmezeau, Bohars, France. eric.lemonnier@chu-brest.fr
C Degrez
M Phelep
R Tyzio
F Josse
M Grandgeorge
N Hadjikhani
Y Ben-Ari
Centre Hospitalier Régional Universitaire de Brest · FRInserm · FRÉcole Polytechnique Fédérale de Lausanne · CH

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gamma aminobutyric acid (GABA)-mediated synapses and the oscillations they orchestrate are altered in autism. GABA-acting benzodiazepines exert in some patients with autism paradoxical effects, raising the possibility that like in epilepsies, GABA excites neurons because of elevated intracellular concentrations of chloride. Following a successful pilot study,(1) we have now performed a double-blind clinical trial using the diuretic, chloride-importer antagonist bumetanide that reduces intracellular chloride reinforcing GABAergic inhibition. Sixty children with autism or Asperger syndrome (3-11 years old) received for 3 months placebo or bumetanide (1 mg daily), followed by 1-month wash out. Determination of the severity of autism was made with video films at day 0 (D0) and D90 by blind, independent evaluators. Bumetanide reduced significantly the Childhood Autism Rating Scale (CARS) (D90-D0; P<0.004 treated vs placebo), Clinical Global Impressions (P<0.017 treated vs placebo) and Autism Diagnostic Observation Schedule values when the most severe cases (CARS values above the mean ± s.d.; n=9) were removed (Wilcoxon test: P-value=0.031; Student's t-test: P-value=0.017). Side effects were restricted to an occasional mild hypokalaemia (3.0-3.5 mM l(-1) K(+)) that was treated with supplemental potassium. In a companion study, chronic bumetanide treatment significantly improved accuracy in facial emotional labelling, and increased brain activation in areas involved in social and emotional perception (Hadjikhani et al., submitted). Therefore, bumetanide is a promising novel therapeutic agent to treat autism. Larger trials are warranted to better determine the population best suited for this treatment.

Indexed as

Asperger SyndromeAutistic DisorderBumetanideChildChild, PreschoolDouble-Blind MethodFemaleGABA ModulatorsHumansMaleSeverity of Illness IndexTreatment OutcomeBumetanideGABA Modulators

Identifiers

PMID23233021
PMCPMC3565189
OpenAlexW2112163143

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-SA
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.