57672
document
A randomised controlled trial of bumetanide in the treatment of autism in children . 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 .
57673
Intervention_Pharmacological
bumetanide
57674
Intervention_Pharmacological
benzodiazepines
57675
Intervention_Pharmacological
bumetanide
57676
Intervention_Control
placebo
57677
Intervention_Pharmacological
bumetanide
57678
Intervention_Pharmacological
Bumetanide
57679
Intervention_Control
placebo
57680
Intervention_Control
placebo
57681
Outcome_Other
effects
57682
Outcome_Physical
Childhood Autism Rating Scale ( CARS )
57683
Outcome_Mental
Clinical Global Impressions
57684
Outcome_Physical
Autism Diagnostic Observation Schedule values
57685
Participant_Age
autism in children .
57686
Participant_Age
Sixty children with autism or Asperger syndrome ( 3-11 years old )