Children’s National Health System to Launch Rheumatic Heart Disease Eradication Effort

6/23/17

New project unites global experts to better diagnose and treat preventable, deadly heart condition caused by untreated strep throat infection

Children’s National Health System has been awarded a $3.7 million grant from the American Heart Association (AHA) to launch a Rheumatic Heart Disease Center, with the goal of developing innovative strategies and economic incentives to improve the prevention and diagnosis of rheumatic heart disease (RHD) in high-risk, financially disadvantaged countries and low-income communities across the United States.

Children’s National was selected as one of four centers in the AHA’s Strategically Focused Children’s Research Network, which is dedicated to improving children’s heart health and reducing the global burden of cardiovascular disease and stroke.

Known as the ‘world’s forgotten disease,’ RHD is caused by untreated streptococcal throat infections that progress into acute rheumatic fever (ARF) and eventually weaken the valves of the heart. It is the most common cardiovascular disease in children and young adults globally – affecting nearly 33 million people and causing 345,000 deaths annually – yet, it is preventable with early detection and access to penicillin.

Over the next four years, the Rheumatic Heart Disease Center, led by Children’s National Heart Institute experts Craig Sable, M.D., associate division chief of cardiology, and Andrea Beaton, M.D., cardiologist, along with RHD leaders around the globe, will develop evidence-based strategies to strengthen the health system’s response to RHD through synergistic basic, clinical and population science research along the entire spectrum of the disease.

“While it’s often thought that we’ve already beaten rheumatic heart disease, data shows there’s nearly no decrease in mortality rates in low-income countries. The disease is endemic in Sub-Saharan Africa, and some poverty-stricken communities in the U.S. are hit nearly as hard,” said Dr. Sable. “We are thrilled to receive this funding from the AHA, which will help us close the research gap for this neglected disease and change the plight of millions of children around the world.”

The basic research project, led by James Dale, M.D., chief of the division of infectious disease at the University of Tennessee in Memphis, will work to better define the immune system response to Group A Streptococcal (GAS) infection, or strep throat, paving the way for vaccine development. In collaboration with a partner site in Cape Town, South Africa, experts will recruit 300 children ages 5-15 to participate for 24 months in a study capturing and classifying various strains of the GAS bacteria. Similar to the common flu, the strains of GAS bacteria vary from region to region and year to year. By identifying immune system targets, or how our bodies fight GAS, the research can inform the creation of effective and long-lasting vaccines.

Dr. Beaton will lead the clinical project that will work to improve understanding and detection of ARF, the precursor to RHD. According to Dr. Beaton, the current, outdated paradigm is that patients with RHD at one point experienced a full-blown episode of ARF – including fever, severe joint pains and rash. These symptoms should be unmistakable and prompt treatment, but in truth the disease remains vastly underdiagnosed in high-risk regions. Through an on-the-ground partnership with experts at Mulago National Referral Hospital in Uganda, the clinical project will work to enroll over 1,000 children ages 3-18 with more subtle symptoms, potentially suggestive of ARF, in order to paint a more accurate picture of the disease in Africa today.

“The gap between the low number of children diagnosed with ARF and the high number of young adults with advanced RHD remains one of the most challenging mysteries and barriers to improved RHD prevention,” said Dr. Beaton. “For the first time, we will systematically characterize the clinical, laboratory and echocardiographic features of ARF in low-resource settings, with the goal of developing a biological signature for ARF that can be translated into a diagnostic test and improve detection.”

Dr. Beaton expects that this research could benefit other related diseases too, such as kidney disease or serious skin infections.

The population research project, led by David Watkins, M.D., M.P.H., an expert in epidemiological and economic modeling at the University of Washington in Seattle, will work to build an economic case for prevention around the world, using the data from the basic and clinical work. The goal is to identify local gaps in delivery of health services for disease prevention and treatment and to measure the cost-effectiveness of RHD interventions, as well as the cost of inaction – especially as patients suffering from advanced RHD are often in the prime of their productive, adult lives. Researchers anticipate the findings will provide effective tools for addressing RHD in other endemic countries too.

AHA selected Children’s National for the grant based on its proven record of global collaboration to solve complex health issues and the potential impact of this research. The program will use Children’s robust telemedicine infrastructure to connect co-collaborators around the world, as well as train the next generation of globally minded cardiovascular researchers.

“The Children’s Strategically Focused Research Network will fulfill an unmet public health need focused on the most vulnerable segments of our population, children, who have the potential to reap the largest societal health dividends free of heart disease and stroke. Our newest SFRN combines the most meritorious programs to prevent rheumatic heart disease, Congenital heart disease, childhood obesity, and strategies to maintain lifelong ideal cardiovascular health,” said Ivor J. Benjamin, M.D., FAHA, FACC, Chair of the AHA Research Committee.

About Children’s National Health System

Children’s National Health System, based in Washington, D.C., has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.

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