Gout medication could be used to treat congenital heart disease

Probenecid, a medication typically used to treat gout, may provide relief for patients with congenital univentricular circulation, according to new research published in Pediatric Cardiology.

The study’s authors tested the effectiveness of probenecid in a randomized double-blind trial including eight pediatric patients. All patients received either the medication or a placebo.

Overall, the team found that probenecid “improved cardiac function and exercise performance” in patients with congenital univentricular circulation.

“Heart function in participants along with their symptoms improved as a result of the pilot study,” lead author Jack Rubinstein, MD, association professor at the University of Cincinnati College of Medicine, said in a statement. “Heart contractility was better. It wasn't a huge increase but enough for us to be able to detect it. They ran better and their heart pumped better.”

The observed changes were “small,” Rubinstein added, but this is believed to be partly due to the limited number of subjects included in the study.

“We can repurpose this medicine, long used to treat gout, to improve how the heart works for kids with univentricular circulation without any adverse effects,” Rubinstein added. “The next step is a larger study to prove we can make it work safely in the long term.”

The full Pediatric Cardiology study is available here.