A pilot dose finding study of pioglitazone in autistic children.
Lucia Capano, Annie Dupuis, Jessica Brian and 8 others
PMID 30498564WHAT IT FOUND
In 28 autistic children, pioglitazone at 0.75 mg/kg was tolerated without serious adverse events; parent and clinician ratings improved on several behavior scales, but without a control group this cannot show the drug worked.
Key findings
01The maximum tolerated dose was 0.75 mg/kg once daily, and no serious adverse events occurred at 0.25, 0.5, or 0.75 mg/kg.
02The changes in ABC social withdrawal, irritability, and hyperactivity scores, CY-BOCS scores, and RBS-R total scores remained significant after adjustment, with effect sizes from 0.43 to 0.9.
03IL-6 decreased and IL-10 increased with treatment, but change in IL-6 did not correlate with outcome measures, and baseline IL-10 did not correlate with change in any outcome measure.
STILL TO COME
How it was doneWhat they foundWhat it means for SLPs
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What it does not show
This was a pilot maximum tolerated dose study, not a trial designed to prove that pioglitazone works. There was no placebo control group, so the study cannot compare pioglitazone with no active treatment. Clinicians who rated outcomes were not blinded. The sample was small: 28 children started and 25 completed. Some children were also receiving non-pharmacologic educational, behavioral, or dietary interventions, so the contribution of pioglitazone alone cannot be separated. Blood cytokine levels may not reflect immune activity in the brain. The study excluded children with many medical or genetic conditions, so it may not apply to broader autistic children. One child who responded to placebo was removed from analyses.
Declared interests
Funded by Physicians' Services Incorporated Foundation and the University of Toronto Dean's Fund. The supplied text does not include a conflicts-of-interest declaration.
The easy way to misread this
Do not conclude that pioglitazone improves autism symptoms. This was a single-arm pilot with no control group, and some children also had ongoing non-pharmacologic educational, behavioral, or dietary interventions, so the contribution of pioglitazone alone cannot be separated.