Experts report urgent need for pediatric heart support devices

At today’s Annual Meeting and Scientific Sessions of the International Society for Heart and Lung Transplantation (ISHLT) in Boston, Angela Lorts, MD, MBA, issued an urgent call for improved mechanical circulatory support (MCS) devices for children with life-threatening heart conditions.

“Advances in pediatric cardiac disease are underfunded and understudied. Therapies are rarely developed for children. We modify adult therapies to use in pediatrics,” said Dr. Lorts, director of the Ventricular Assist Device Program at Cincinnati Children’s Hospital.

Pediatric hearts fail because of acquired and genetic disorders that affect the heart muscle and structural or congenital heart disease that can’t be adequately repaired. Approximately 500 pediatric transplants are performed, and about 300 heart devices are implanted annually in the United States alone.

Patients awaiting a heart transplant are often implanted with an MCS device, which helps the heart pump oxygen-rich blood to the rest of the body.

“Kids really don’t get the therapies they need because MCS devices are designed for adults,” she said. “The patient population is relatively small, and there is often concern about return on investment for small devices, so companies struggle to invest in them. In many cases, adult devices are used off-label in pediatric patients.”

According to Dr. Lorts, off-label device use in children not only deters innovation but also does not require data collection and analysis for safety and efficacy.

“We’re grateful to have a pediatric-specific device, but it is older-generation engineering and has high adverse event rates,” she said. “Also, the supply of these devices can’t keep up with demand, so children sometimes can’t access the life-saving technology when they need it most.”

Compounding the problem is the fact that children implanted with the pediatric MCS device must remain in the hospital. These patients may be hospitalized for six months to a year before they receive a new heart.

“We need to get these kids home to their houses with their devices,” she said. “That’s what adults get to do, and our children should be in the same position.”

She and colleagues established the ACTION Network, an international collaborative of 70 centers and 1,500 providers to raise awareness and funding for pediatric heart devices. The network is working with the U.S. Food and Drug Administration (FDA) to expand the use and monitor the safety of two adult VAD devices in pediatric patients.

She said several manufacturers have become great partners for the pediatric heart community, and a handful of promising new devices are on the horizon.

The ACTION network, which also includes regulatory organizations, researchers, and industry representatives, is also working to combine real-world data and clinical trial research to develop and monitor the safety and efficacy of pediatric heart devices.

“The device manufacturers are aware of the problem,” Dr. Lorts said. “But we still need alternative ways to allocate funds to support a pediatric device initiative so we can save these kids’ lives and get them home with their devices.”

Dr. Lorts said the lack of novel therapies designed for children is a systemic problem in medicine.

“We always use adult therapies and try to jerry-rig them,” she said. “There are kids who die now because we don’t have the right device. We can do better.”

Provided by
International Society for Heart and Lung Transplantation

At today’s Annual Meeting and Scientific Sessions of the International Society for Heart and Lung Transplantation (ISHLT) in Boston, Angela Lorts, MD, MBA, issued an urgent call for improved mechanical circulatory support (MCS) devices for children with life-threatening heart conditions.

“Advances in pediatric cardiac disease are underfunded and understudied. Therapies are rarely developed for children. We modify adult therapies to use in pediatrics,” said Dr. Lorts, director of the Ventricular Assist Device Program at Cincinnati Children’s Hospital.

Pediatric hearts fail because of acquired and genetic disorders that affect the heart muscle and structural or congenital heart disease that can’t be adequately repaired. Approximately 500 pediatric transplants are performed, and about 300 heart devices are implanted annually in the United States alone.

Patients awaiting a heart transplant are often implanted with an MCS device, which helps the heart pump oxygen-rich blood to the rest of the body.

“Kids really don’t get the therapies they need because MCS devices are designed for adults,” she said. “The patient population is relatively small, and there is often concern about return on investment for small devices, so companies struggle to invest in them. In many cases, adult devices are used off-label in pediatric patients.”

According to Dr. Lorts, off-label device use in children not only deters innovation but also does not require data collection and analysis for safety and efficacy.

“We’re grateful to have a pediatric-specific device, but it is older-generation engineering and has high adverse event rates,” she said. “Also, the supply of these devices can’t keep up with demand, so children sometimes can’t access the life-saving technology when they need it most.”

Compounding the problem is the fact that children implanted with the pediatric MCS device must remain in the hospital. These patients may be hospitalized for six months to a year before they receive a new heart.

“We need to get these kids home to their houses with their devices,” she said. “That’s what adults get to do, and our children should be in the same position.”

She and colleagues established the ACTION Network, an international collaborative of 70 centers and 1,500 providers to raise awareness and funding for pediatric heart devices. The network is working with the U.S. Food and Drug Administration (FDA) to expand the use and monitor the safety of two adult VAD devices in pediatric patients.

She said several manufacturers have become great partners for the pediatric heart community, and a handful of promising new devices are on the horizon.

The ACTION network, which also includes regulatory organizations, researchers, and industry representatives, is also working to combine real-world data and clinical trial research to develop and monitor the safety and efficacy of pediatric heart devices.

“The device manufacturers are aware of the problem,” Dr. Lorts said. “But we still need alternative ways to allocate funds to support a pediatric device initiative so we can save these kids’ lives and get them home with their devices.”

Dr. Lorts said the lack of novel therapies designed for children is a systemic problem in medicine.

“We always use adult therapies and try to jerry-rig them,” she said. “There are kids who die now because we don’t have the right device. We can do better.”

Provided by
International Society for Heart and Lung Transplantation