Immediate Action Required for Pediatric Heart Support Innovations

At the Annual Meeting and Scientific Sessions of the International Society for Heart and Lung Transplantation (ISHLT) held in Boston, experts highlighted a critical need for advancements in mechanical circulatory support (MCS) devices tailored for children facing severe heart conditions.

Angela Lorts, a leading pediatric cardiologist, expressed concern over the lack of research and funding dedicated to pediatric cardiac therapies. She noted that many treatments are adaptations of adult devices, which do not adequately meet the specific requirements of young patients.

Pediatric heart failure can result from both genetic and acquired conditions, as well as congenital heart diseases that cannot be sufficiently repaired. In the United States, around 500 pediatric heart transplants and 300 heart device implants occur each year.

For many children awaiting transplants, MCS devices are essential for maintaining blood circulation. However, the current devices are primarily designed for adults, leading to significant challenges in their application for children. Dr. Lorts emphasized that the limited market for these specialized devices deters investment from manufacturers, resulting in reliance on off-label use of adult devices.

This off-label use not only hampers innovation but also sidesteps the rigorous data collection needed to ensure these devices' safety and effectiveness in younger patients. While a pediatric-specific device exists, it is based on older technology and is associated with high rates of complications. Furthermore, the availability of these devices often fails to meet the increasing demand, leaving many children without timely access to crucial life-saving technology.

A major hurdle is that pediatric patients requiring MCS devices typically remain hospitalized for extended periods, sometimes ranging from six months to a year, while awaiting heart transplants. Dr. Lorts advocates for policies that allow these children to return home with their devices, similar to the protocols established for adult patients.

In response to these challenges, Dr. Lorts and her colleagues founded the ACTION Network, a global initiative comprising 70 medical centers and 1,500 healthcare providers. This network aims to enhance awareness and secure funding for pediatric heart support devices. It collaborates with the U.S. Food and Drug Administration (FDA) to evaluate and broaden the use of certain adult ventricular assist devices (VADs) in pediatric settings.

Several manufacturers have begun to engage positively with the pediatric heart community, and promising new devices are under development. The ACTION Network also seeks to integrate real-world data with clinical trial research to better assess the safety and effectiveness of pediatric heart devices.

Despite some progress, Dr. Lorts stressed the need for innovative funding solutions to support pediatric device initiatives. The systemic issue of inadequate therapies for children persists, often forcing healthcare providers to adapt adult solutions.

As a result, some children currently face life-threatening situations due to the unavailability of appropriate devices. Dr. Lorts concluded that the medical community must strive for improvements in this area to ensure that all children receive the care they deserve.