Guidelines for Pediatric Heart Failure and the Impact of Clinical Trials

Pediatric cardiologists at Children’s Hospital Colorado were among the experts who contributed to recently published guidelines for diagnosing and treating pediatric heart failure. While treating heart failure in adults is based on the results of large clinical trials involving thousands of people, there is a lack of data to guide treating heart failure in children. Therefore, these new guidelines for children are valuable but largely based on what we know is effective in adults. Cardiologists and researchers at Children’s Colorado suggest that children with heart failure require focused study to improve outcomes.

Updated guidelines for managing pediatric heart failure

The International Society for Heart and Lung Transplantation updated their guidelines for  managing pediatric heart failure in 2025 — the first update in 11 years. Authors of the updated guidelines included pediatric cardiologists, Shelley Miyamoto, MD, the Chair of Pediatric Cardiology at Children Colorado, and Stephanie Nakano, MD. Dr. Miyamoto provided insights into why they updated the guidelines.

The update incorporates advancements in pharmacologic therapies and new approaches for evaluating and managing heart failure in children. Because data on pediatric heart failure is sparse, it’s more challenging to provide evidence-based guidance on treating heart failure and its complications in children. Dr. Miyamoto says, “While we know many drugs work really well to treat heart failure in adults, it is challenging to know if these same drugs are as effective in children because we do not have large clinical trial data to inform us.”

Understanding the current approach to treating pediatric heart failure

Dr. Miyamoto, along with pediatric heart transplant and heart failure fellow Benjamin Olsen, MD, discuss their perspective in the article Pediatric Heart Failure: Current Approach and Treatment. Their comprehensive review outlines the current landscape and future direction in diagnosing and treating pediatric heart failure, emphasizing its complexity, heterogeneity and distinct pathophysiology compared to adult heart failure. Despite lower prevalence, pediatric heart failure is linked to a disproportionately higher burden in terms of mortality, resource utilization and long-term impact.

“The word that stands out is equity,” says Dr. Olsen, “and it extends beyond just what we typically would think in terms of ensuring that all people receive the care they deserve — it goes further to ensure that when some have less access to quality care, more effort is put toward ensuring they have sufficient access to address their problem. And this idea extends more broadly into the pediatric heart failure population where again the burden of disease is greater with higher rates of mortality than in the adult population.”

Addressing the inequities in treating children and adults for heart failure

Drs. Miyamoto and Olsen ultimately see the need for more equitable care delivery, improved trial design and targeted research to close the gap in outcomes between children and adults with heart failure.

They also acknowledge that while some treatments may be effective for one population, they may not be effective for all populations with the same issues. For example, while heart muscle failure is a shared pathway in adults and children with heart failure, the mechanisms responsible for heart failure differ between adults and children. This provides potential explanations for why existing heart failure treatment regimens are more effective in adults than children.

Complementary to the new pediatric heart failure guidelines, Dr. Miyamoto and Nadia Chaudhry-Waterman, MD, a graduate of the Pediatric Cardiology Fellowship Program at Children’s Colorado, propose ideas for how to combat the inequity in research in the article, Promoting Advancements in Pediatric and Congenital Cardiology Amid Clinical Trial Challenges. Improvements in diagnosing and treating heart diseases diagnosed in infancy or childhood mean that patients are surviving well into adulthood. However, evidence-based guidance for their care hasn’t caught up to the increased life expectancy.

The article cites historical barriers to the success of studies on children with heart disease, as well as highlights some innovative approaches to pediatric heart disease clinical research, which can act as a model for future success. Since the number of children with each heart condition is very small, it is difficult to adequately power studies. Therefore, collaboration is essential and multiple centers must work together and combine their data to effectively study pediatric heart disease treatments.

There is also an increasing number of registry-based prospective clinical trials in pediatric and congenital heart disease (CHD) that can be an effective approach. The collaborative learning network Advanced Cardiac Therapies Improving Outcomes Network (ACTION) started in 2017 with the mission of improving outcomes for children and adults with heart failure secondary to cardiomyopathies or CHD. Children’s Colorado participates in this international collaborative, which unites key players, including patients, families, clinicians, researchers and industry.