NHLBI awarded four contracts to test new devices to help children with congenital heart defects

The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, has awarded four contracts totaling $23.6 million to begin preclinical testing of devices to help children born with congenital heart defects or those who develop heart failure. The four-year program is called Pumps for Kids, Infants, and Neonates (PumpKIN).

Each year in the United States, nearly 1,800 infants die as a result of congenital heart defects and another 350 develop heart disease, which leads to heart failure for many. Approximately 60 infants and children under 5 years old who are placed on the heart transplant waiting list die each year before receiving one. Mechanically assisted circulatory support could be used to sustain these young patients as they seek to recover or wait to receive a heart transplant.

"This research seeks to develop technologies to expand life-saving options for infants and children born with congenital heart defects or those who develop heart failure," said NHLBI Acting Director Susan B. Shurin, M.D., a pediatrician. "The NHLBI is committed to saving the lives of our youngest patients. Well-designed circulatory support devices are expected to substantially improve the outcomes of the infants and young children who need them as they seek to recover or wait to receive a heart transplant."

The options for chronic circulatory support devices for infants and young children are limited, and all have substantial risks for serious adverse events such as infection, stroke, and device failure. With this in mind, the NHLBI launched the Pediatric Circulatory Support Program in 2004 by funding the development of five novel circulatory support devices for infants and young children with congenital and acquired cardiovascular disease.

The PumpKIN program is the next phase of NHLBI support for the development and clinical realization of these devices. The program's goal is to complete the needed animal studies and other tests in artificial environments for the most promising devices in order to gain approval from the FDA to begin clinical testing.

Devices in the program will provide suitable circulatory support for newborns, older infants, and children less than 55 pounds who experience heart failure due to congenital and acquired cardiovascular disease. They are designed to supply adequate blood flow to prevent organ damage while minimizing the risk of blood vessel damage, infection, breakdown of red blood cells, excessive bleeding, brain damage, and dangerous blood clots. The devices are intended to support circulation in pediatric patients for one to six months, be sufficiently small and reasonably portable, and be able to be routinely positioned and functioning in less than one hour, among other specifications.

"Similar devices are used in adults," Shurin noted. "As an adult, your heart is normally about the size of your fist; devices for small children require radically different designs from adult devices to adapt to the differences in the size of the patients."

The program will test ventricular assist devices (VADs) and advanced extracorporeal membrane oxygenator (ECMO) devices. The VADs in the PumpKIN program are very small rotary pumps which are implanted to provide circulatory support for extended periods of use. They work by drawing blood from the heart and pumping it to the body. ECMO devices circulate and supply oxygen to the blood, and are commonly used for patients who need both heart and lung support. For ECMO devices, tubes connecting the patient to the device are placed directly into large blood vessels near the base of the neck. Blood is drawn from the right side of the heart, pumped through the oxygenator, and then returned to the body on the left side of the heart so the oxygen-rich blood can be delivered throughout the body.

The contractors will conduct all preclinical animal testing and analysis in the first three years of the contract. During the third year, they will partner with a data coordinating center (the contract for which is still to be awarded) to complete the necessary activities to seek FDA approval to begin the clinical trial.

PumpKIN contractors are Harvey S. Borovetz, Ph.D., University of Pittsburgh; Mark Gartner, Ph.D., Ension, Inc., Pittsburgh, Pa.; Bartley P. Griffith, M.D., University of Maryland School of Medicine; and Robert Jarvik, M.D., Jarvik Heart, Inc., New York, N.Y.