A tiny heart pump that maintains blood flow in babies and small children with serious heart failure proved effective and life-saving in a pioneering study involving 17 institutions led by Texas Children’s Hospital and Baylor College of Medicine (BCM).
The study looked at the safety and probable benefit of the Berlin Heart EXCOR pediatric ventricular assist device (VAD), the only VAD available for babies and children. Patients who received the Berlin Heart lived longer on the device and were more likely to receive a transplant or recover heart function than children who were maintained on more traditional support using extracorporeal membrane oxygenation (ECMO), according to the study’s results.
“This study is unprecedented and represents broad collaboration among the top pediatric cardiac transplantation centers in North America, really the who’s who in the field,” says Dr. Charles Fraser Jr., surgeon-in-chief at Texas Children’s Hospital and professor of surgery and pediatrics at BCM. Fraser is also the corresponding author of the report and was national principal investigator of the study. “This study is now the gold standard for VAD therapies in children,” he shares. “Everything going forward will be compared to this.”
Before the Berlin Heart
Before the Berlin Heart, physicians used complicated medical therapies to treat children with heart failure, hoping to keep them alive until a suitable donor heart became available. Newborns and small children often died as they waited, since no more than 70 or 80 small donor hearts become available each year. ECMO provided only short-term support when the child’s heart failed completely. The Berlin Heart offers families a new ray of hope.
“With the Berlin Heart, we have a more effective therapy to offer patients earlier in the management of their heart failure,” says Fraser. “When we sit with parents, we have real data to offer, so they can make an informed decision. This is a giant step forward.”
Children on the device can leave the intensive care unit and go to a regular hospital room where they receive rehabilitation and better nutrition while they wait for a transplant. While there are many such devices available for adults, development and approval of devices for children and especially for babies has lagged.
“This prospective trial is unprecedented, because it was not a look back at how children on the device had fared but instead followed their clinical course from the device’s implantation. The results were compared to those of children who received ECMO, the only other method of treatment,” says Fraser, also chief of congenital heart surgery at Texas Children’s.
“We thought we knew a lot about this device and how it worked, but the FDA was insistent that we conduct a trial and they were right,” he adds. “We did not know as much as we should about how the device performs in practice.” Funding for this study came from the U.S. FDA’s Office of Orphan Product Development and Berlin Heart Inc.
About Texas Children’s Hospital
Texas Children’s Hospital, a not-for-profit organization, is committed to creating a community of healthy children through excellence in patient care, education, and research. Consistently ranked among the top children’s hospitals in the nation, Texas Children’s has recognized Centers of Excellence in multiple pediatric subspecialties including the Cancer and Heart Centers, and operates the largest primary pediatric care network in the country. Texas Children's has completed a $1.5 billion expansion, which includes the Jan and Dan Duncan Neurological Research Institute; Texas Children’s Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; and Texas Children’s Hospital West Campus, a community hospital in suburban West Houston. For more information on Texas Children's, go to www.texaschildrens.org.