Findings from a literature review published in Annals of Pharmacotherapy suggest low-dose oral bumetanide may be beneficial for patients with moderate to severe autism spectrum disorder (ASD) when behavioral therapies are unavailable. 

Study authors searched PubMed and Ovid MEDLINE for relevant randomized, controlled, clinical trials from 1946 to October 2018 and found oral bumetanide was evaluated in 208 patients aged 2-18 years with ASD. The studies measured the effect of bumetanide on core behavioral features through different autism assessment scales. 

Using the Childhood Autism Rating Scale to assess improvement at day 90, only 1 trial demonstrated statistical significance with oral bumetanide. Statistically significant improvements in the Clinical Global Impressions Scale was observed in 2 of the 3 trials studied. A statistically significant benefit with bumetanide was seen in 2 trials that used the Autism Behavioral Checklist and Social Responsiveness Scales for assessment. 

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Behavioral features such as social communication, interactions, and restricted interest were most improved with bumetanide treatment; however, the authors reported no dose-effect relationship in the dose-ranging trial. 

Higher doses of bumetanide resulted in more frequent hypokalemia and polyuria. Withdrawal due to adverse effects ranged from 17% to 43%. Bumetanide 0.5mg given twice daily proved to be the best tolerated dose as well as the most commonly studied dose in the studies. 

As there are no FDA-approved treatments for core ASD symptoms, “this review presents preliminary safety and efficacy data for bumetanide in ASD,” the authors concluded. 

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