Advancing Pediatric Heart Disease Research Through the Pediatric Heart Network

Our Heart Institute is working on some exciting research with the Pediatric Heart Network (PHN), a consortium of leading hospitals focused on children and adults living with congenital heart disease (CHD), as well as children affected by acquired heart disease.

Funded by the National Institutes of Health, the PHN is a consortium of nine core sites, including Children’s Hospital Colorado. The network supports large, multi-site pediatric cardiology clinical studies and serves as the umbrella for several current research efforts. Accepted as a core site in 2024, our hospital is integral in planning and executing PHN research. Our Heart Institute is actively working on two important research projects within the PHN: The FIORE and the WE BEAT CHD studies.

The FIORE study: Can finerenone work for children?

Shelley Miyamoto, MD, is the chair of the FIORE steering committee, helping to lead a multinational clinical trial evaluating the effectiveness of finerenone for treating pediatric heart failure. Finerenone is a medication used for adults with heart failure and has been shown to be effective in treating adults with heart failure symptoms.

“We have so few treatments for children with heart failure that are proven to be effective in kids,” says Dr. Miyamoto. “Pediatric cardiologists largely treat children with heart failure the same as they treat adults — we take the drugs that are used to treat heart failure in adults, decrease the dose, and use that same medication to treat children with heart failure. But we're lacking concrete evidence that the treatment is effective.”

The FIORE study will dive deeper into how effective finerenone is for children with heart failure and in what ways. As chair of the steering committee, one of Dr. Miyamoto’s responsibilities is to enlist pediatric heart centers from across the U.S. and around the world to take part. All participating centers must have pediatric heart failure experts, clinical trial capabilities, specialized pharmacy and lab staff, and rigorous echocardiogram procedures.

What do we know about heart failure in children?

Children with heart failure actually have worse outcomes than adults with heart failure — the risk of death or need for a heart transplant is higher for children than adults. In fact, within five years of being diagnosed with heart failure, about half of all children will either die or need a heart transplant — compared to only about one in 10 adults with the same diagnosis. Based on these outcomes, the current treatments don’t appear to be equally effective in adults and children. Several factors contribute to this, and this study may be one step toward understanding why.

“The advantage of this trial,” says Dr. Miyamoto, “is it's an opportunity to see if we can gather evidence in a very controlled and rigorous way to determine if this medication can be effective in the treatment of children. If it's not, there's benefit in knowing that as well — we shouldn't be exposing children to medications that are not effective. Regardless of the result of the trial, we’re going to gain useful knowledge that will benefit children with heart failure.”

The WE BEAT CHD study: How can we improve psychosocial care for teens with congenital heart disease (CHD)?

The WE BEAT CHD study is a psychologist-led group intervention conducted across 14 PHN heart centers and designed to improve resiliency and support teens as they navigate congenital heart disease and their transition to adult care. As more children born with heart disease receive better care, they’re living longer. Dr. Miyamoto explains that as these children grow up to be adults, we are learning of other co-morbidities doctors must address.

Children born with single ventricle heart disease, for example, get three surgeries, starting soon after birth, to reroute blood flow. This trio of surgeries, the last known as the Fontan procedure, has made it possible for thousands of babies to not only survive, but grow into teenagers and adults. But now we are learning that Fontan circulation, along with the underlying heart disease, impacts individuals across multiple organ systems as they age, including increasing their risk of developmental and learning challenges, and liver and lung disease. These difficulties require multidisciplinary care, such as the care we provide at our Fontan Multidisciplinary Clinic, and can impact the quality of life of patients and their families.

Understanding the mental health of children with congenital heart disease

The WE BEAT CHD study is looking at one aspect of care for these kids — mental health. Growing up as a child with CHD and managing the ongoing care as a teen and adult can be overwhelming. Lifelong treatment and management of congenital heart disease can create a great deal of stress and anxiety, so it’s not a surprise that it impacts mental health. There is a strong need and desire for psychosocial care for CHD, but access to cardiac-specific mental health services is limited. The research team wants to understand the best way to address the stress and anxiety of living with a heart condition.

WE BEAT CHD is a virtual intervention for those between 12 and 17 years old with moderate to complex congenital heart disease, including teens with Fontan circulation. It focuses on improving resiliency through psychologist-led group sessions. Sarah Kelly, PsyD, plays a key role as our hospital’s study lead, and within the PHN as one of four interventionists across three core sites facilitating this group wellbeing education program. Megan SooHoo, MD, Jessica Church, APN, Sara Mackie, NP, Mackenzie Jackson, Kelsey Maloney and Samantha Tellez from the Children’s Colorado Single Ventricle Program are developing outreach and recruitment strategies to reach the teens who need this care. WE BEAT CHD moves beyond merely identifying mental and emotional health challenges. The study offers a proactive intervention to address these issues and the gap in care these patients often experience during their teenage years. It will also gather information on quality of life, psychosocial health and biomarkers of stress to better understand the mental and cardiac health of those with CHD – and how to help.

“One thing that’s happened over the past couple decades is that patients are surviving at a much higher rate than they did in the past,” says Miyamoto. “Now we’re recognizing the extent of the challenges they’re facing beyond their heart issues. It is time to do something to help support these teenage patients as they emerge into adulthood so they can live full and productive lives.”

The Heart Institute and the Pediatric Heart Network make a fruitful partnership for research

As the only hospital in Colorado and one of just nine core sites in the Pediatric Heart Network, Children's Colorado is at the forefront of a nationwide research collaboration — one that spans 13 ancillary sites and numerous additional pediatric cardiology programs across the country. Together, this network is driving the next evolution in care for children and adults with congenital heart disease, building on the remarkable advances of recent decades.