When Elfriede Pahl, MD, FACC, completed medical school in 1983, Ronald Reagan was in his first term as president and few pediatric patients with heart failure (HF) reached an age to vote in elections. Today, as she delivers the Dan G. McNamara Keynote, Pahl will look back at the medical advances over the last four decades that have added decades to the lives of those children.

“I will be touching on some of the highlights in treating children who have HF and what still needs to be done going forward,” she notes. “There have been so many changes and advancements over the last few decades that have improved care, yet there is a paucity of evidence-based treatments specific to children, who are not merely small adults and have unique mechanisms of HF.”

Pahl, professor emerita at Northwestern Feinberg School of Medicine, underscores the significance of pediatric HF, saying “this topic is critically important because these children often have the longest and most complex hospital stays with limited options. Improved therapies can impact young lives for decades and affect quality of life for the whole family.” Genetic research and ventricular assist devices (VADs) stand out today as the greatest advances in treating pediatric cardiovascular patients, which were developed with knowledge gained through registries and early pediatric clinical trials. The discovery of biomarkers and improvements in heart transplant procedures have further enhanced outcomes.

“The take-home message is that collaboration is important,” Pahl says. “The more we can do together as the key centers in the field to combine our resources, perform clinical trials and work with industry and funding agencies, the further we will be able to go to enhance the care of these very sick patients.”

Here are what she considers the seven greatest developments in the care of children with HF over the last four decades:

1. Creation of Registries
Most articles about pediatric HF published in 1983 were based on data from only 10 to 15 cases at a single institution, Pahl says. When those institutions pooled their resources by developing registries with much more data, advances followed.

2. Use of VADs in Pediatric Patients
Use of VADs was first expanded to include adolescents in the 1990s, and there has been an “explosion” of VAD use in the last 10 years, Pahl says. VADs are now regularly used to stabilize pediatric patients until they can get heart transplants or, in rare cases, as a bridge to recovery. A key success in this development was the Berlin Heart Trial that led to U.S. Food and Drug Administration approval of the Berlin Heart EXCOR pediatric VAD, which can be used in even the smallest of patients.

3. Advances in Imaging
Pahl says imaging was in its “dinosaur age” when she finished medical school. The ensuing 20 to 30 years saw advances in echocardiography, cardiac CT scans, MRIs and nuclear imaging that fueled the development of better diagnostic algorithms and screenings, which often allowed for avoidance of invasive procedures.

4. Development of Pediatric CV Clinical Trials
The first pediatric randomized clinical trial for a HF treatment was for carvedilol, published in in 2007, followed more recently by the PANORAMA trial. The TEAMMATE trial in heart transplant patients, completed in 2023 with results expected to be published soon, is the first study comparing the use of two immunosuppressants – everolimus with low-dose tacrolimus and standard tacrolimus with mycophenolate mofetil.

5. Discovery of Biomarkers
Biomarkers, often from a blood test or other body sample, such as natriuretic peptides or troponin, can aid in risk stratification after diagnosis, look for rejection after heart transplantation and monitor graft functions. According to Pahl, biomarkers have revolutionized treatment for HF over the last 30 years.

6. Explosion of Cardiac Genetics
Pahl highlights the use of genetics, which allows detection, family screening and treatments that may make cures possible for select genetic cardiomyopathies. The Human Genome Project was completed in 2003 and the first gene therapies for select cardiomyopathies were developed in 2020. Some gene therapies have recently been expanded for use in pediatric patients.

7. Improvements in Heart Transplant Survival
Improved monitoring as well as medical and surgical techniques now available to high-risk patients with antibodies and comorbidities have led to greater early and long-term survival following heart transplantation. “Now, when a child gets a heart transplant, greater than 95% can expect to survive the operation and many more are living longer than before, but we still have a long way to go,” Pahl says.

Clinical Topics: Heart Failure and Cardiomyopathies, Acute Heart Failure

Keywords: Cardiology Magazine, ACC Publications, ACC25, ACC Annual Scientific Session, Heart Failure, Pediatric Cardiology