What is the goal of the PHN?
Through the Pediatric Heart Network (PHN), the NHLBI funds research across a group of hospitals in the United States and Canada to accelerate the discovery of new treatments for congenital and pediatric-acquired heart disease. The PHN is the clinical cornerstone of the larger Bench to Bassinet program, a major effort the NHLBI launched to learn more about how the heart develops and why children are born with heart problems.
The goal of the PHN is to improve outcomes and quality of life for individuals with congenital and pediatric-acquired heart disease. Congenital heart defects occur in approximately 40,000 individuals in the United States each year and are a major cause of infant death.
- The PHN is part of the larger Bench to Bassinet program, which seeks to improve outcomes for individuals with pediatric-acquired and congenital heart disease.
- The network consists of several main clinical centers and dozens of additional research centers.
- The PHN has completed nearly 20 clinical studies and enrolled more than 3,500 patients.
- The PHN’s Single Ventricle Reconstruction Trial was the largest clinical trial to compare surgical treatments for congenital heart disease.
- As survival rates of children with congenital heart defects improve, the PHN has expanded its current research to include adults with congenital heart disease.
How does the PHN contribute to scientific discoveries or improved clinical care?
The PHN has helped create better outcomes for children with heart diseases for more than a decade, by addressing particular research challenges. For example, many hospitals and individual doctors do not see enough children with any one type of heart disease to be able to conduct certain studies that would produce meaningful results. In addition, parents have been unable to enroll their children in studies that are available because they are not aware of them, do not live near a research center, or may not feel at ease about what happens during a study. The PHN was established to address these challenges by encouraging collaboration among multiple research teams that specialize in the care of patients with heart disease and patient advocacy groups.
Major achievements and scientific findings from the PHN include:
- In the Single Ventricle Reconstruction (SVR) Trial, the largest clinical trial to compare surgical treatments for congenital heart disease, researchers found that infants born with a severely underdeveloped heart who undergo a newer surgical procedure were more likely to survive their first year and not require a heart transplant than those who had a more traditional surgical procedure. But when children were evaluated after 12 months of age, no differences were found in transplant-free survival. This group of children is continuing to be studied a decade later to help researchers understand the long-term effects of the different surgical procedures.
- Although effective in treating adults with heart failure, angiotensin-converting enzyme (ACE) inhibitors are not effective in children with congenital heart disease and heart failure. This finding prompted important research into the unique mechanisms of heart failure in children.
- The PHN Nursing Research Program was developed to support studies led by nurse investigators. PHN nurse investigators ask questions that may not be the primary focus of their physician investigator colleagues, such as those related to quality of life, emotional adaptation of families, or feeding and growing difficulties in children with severe forms of congenital heart disease.
The NHLBI also helped support the development of the Children and Clinical Studies website. Children have often had to accept medicines and treatments based on what is known to work in adults. To improve clinical care of children, more studies are needed focusing on children's health, with the goal to develop treatments, drugs, and devices specific to children. This website answers questions and features parents, researchers, and children themselves sharing their thoughts and experiences about clinical research.
As a whole, the PHN provides researchers with a powerful platform for advancing the health of individuals affected by congenital or pediatric-acquired heart disease. Perhaps most importantly, it has brought together a group of people for sustained collaboration over a significant period of time. The PHN has become such an established enterprise that every year U.S. News and World Report uses participation in the PHN as one of the criteria when judging hospitals in its annual issue on the Best Children’s Hospitals.
Investigators are encouraged to use the PHN’s available data sets and resources in their research. The PHN is also focused on training the next generation of researchers and establishing productive collaborations with foundations and patient advocacy groups.
How does the PHN work?
The PHN is a cooperative effort between participating medical centers (nine main research sites), a data coordinating center, an independent Network chair, and the NHLBI. The PHN proposes important research ideas, develops protocols, performs research, monitors patient safety and data, and disseminates the research findings. When studies need more patients than the main centers have, other skilled centers may be trained to conduct one or more of those studies. This helps families across the country have access to a center closer to them.
The studies are conducted with research teams that specialize in the care of patients with heart disease. All of the centers carefully follow a study protocol and treat patients in similar ways so that the study results are accurate. Patient safety is a high priority when a study is being planned and conducted. PHN nurses and doctors have had special training in doing research in ways that help protect patients in a study. They also have had training in how to conduct the specific PHN studies and are experienced with caring for families and their sick children.