Uganda Heart Institute launches findings from the Goal clinical trial results

Uganda Heart Institute launches findings from the Goal trial
The study was led by an international panel of cardiac experts from institutions including UHI

Uganda Heart Institute on Wednesday (Nov.17) launched the findings from Goal clinical trials revealing that Penicillin, a widely available and affordable antibiotic, may be one key to turning the tide on the deadly impacts of rheumatic heart disease (RHD) for children in developing nations. This is per the new findings of a large-scale, randomized controlled trial completed in Uganda and published in the New England Journal of Medicine.

RHD is serious and irreversible heart damage caused by rheumatic fever — a condition that results from poorly treated, repeated infections from streptococcus bacteria, also known as strep throat. Though widely eradicated in nations such as the United States due to the swift detection and treatment of strep throat, rheumatic fever remains prevalent in developing regions like sub-Saharan Africa. Current estimates are that 40.5 million people worldwide live with rheumatic heart disease and that it kills 306,000 people every year. Most of those affected are children, adolescents, and young adults under age 25.

“Our study found a cheap and easily available penicillin can prevent progression of latent rheumatic heart disease into more severe, irreversible valve damage that is commonly seen in our hospitals with little or no access to valve surgery,” said the Principal Investigator, Dr. Emmy Okello, Head of Cardiology at the Uganda Heart Institute and Principal Investigator on this landmark study.

“This study is the first large-scale clinical trial to show that early detection coupled with prophylactic treatment with penicillin is feasible and can prevent rheumatic heart disease from progressing and causing further damage to a child’s heart.”

Dr. Joselyn Rwebembera, Cardiologist at Uganda Heart Institute and Co-Investigator on the clinical trial said, “The results of this study provide a fresh ray of hope for the millions of children who are born and grow up in developing nations. To know that we can do something in the early, asymptomatic stages of the disease to prevent progression to the late, devastating stages is rewarding,”

“We can prevent the devastating complications and costs of advanced RHD care, including premature death – on average death below 28 years of age,” she added.

According to Andrea Beaton, M.D., Associate Professor of Cardiology at Cincinnati Children’s and co-lead author, this is the first contemporary randomized controlled trial in rheumatic heart disease.

“The results are incredibly important on their own, but also demonstrate that high-quality clinical trials are feasible to address this neglected cardiovascular disease,” she said.

The investigator team named the trial Gwoko Adunu pa Lutino (GOAL), which means “protect the heart of a child.”

The study enrolled 818 Ugandan children and adolescents ages 5 to 17 years old who were diagnosed with latent rheumatic heart disease to see if an injection of penicillin was effective at preventing their heart condition from worsening.

“There are many challenges with recruitment and retention of trial participants in areas like our study region in Uganda,” said Dr. Okello.

“But it is critical to work together and overcome barriers because we must study these treatments in the people most affected by the condition to understand how they, and others like them, may benefit from the findings.”

Of the 799 participants who completed the trial, the group receiving a prophylactic injection of penicillin (399 volunteers) had three participants show evidence of worsened rheumatic heart disease on repeat echocardiogram after two years.

In contrast, 33 of the 400 volunteers in the control group, who received no treatment, showed similar progression on echocardiogram results.

The study was led by an international panel of cardiac experts from institutions including Uganda Heart Institute, Children’s National, Cincinnati Children’s Medical Center, and Murdoch Children’s Research Institute.

Funding was rendered by the Thrasher Pediatric Research Fund among other organizations. Ministry of Health – Uganda, Makerere University School of Medicine Research Ethics Committee, Uganda National Council for Science and Technology, and the National Drug Authority provided technical support and oversight.

Winnie Nantongo

Editor-in-Chief, The Postdale Daily

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