The U.S. Food and Drug Administration's Office of Orphan Products Development has awarded Seattle Children's Research Institute a $1.6 million grant to lead a 4-year, multi-site clinical trial aimed at improving long-term health in infants born with a heart defect.
Dr. Michael Portman, director of Pediatric Cardiovascular Research at Seattle Children's Hospital and an investigator in Seattle Children's Research Institute's Center for Developmental Therapeutics, will be investigating whether treating infants on cardiopulmonary bypass with thyroid hormone supplement may help them recover more quickly and with fewer side effects.
"The societal cost of congenital heart disease is huge," Portman said. "These are children who suffer lifelong effects. If we can use a medication that's already proven to be safe to help them recover from heart surgery then that could have a major impact on their lives."
The trial will begin enrolling participants next month and will include patients from Seattle Children's, Stanford University Lucille Packard Children's Hospital and Los Angeles Children's Hospital.
Portman previously discovered the benefit of thyroid supplementation for children on bypass in a study published in 2010 in the American Heart Association's academic journal Circulation.
Knowing thyroid hormone levels decrease in infants and children while on cardiopulmonary bypass, Portman led a team of researchers to test the safety of thyroid hormone supplementation in infants and children after cardiac surgery to determine whether it might improve patient outcomes.
The randomized clinical trial, which included children under 2 years old, revealed that giving patients thyroid hormone was safe for all ages and improved clinical outcomes in patients under 5 months old. A second study looking specifically at patients under 5 months of age is required to modify the labeling of thyroid hormone supplement, which would impact the current standard of care.
"I anticipate this study will confirm the results of our previous research so that clinicians may begin prescribing this supplement to those patients that would benefit most," Portman said.
Congenital heart disease occurs in approximately 32,000 infants per year and approximately 38percent of these patients will have one or more surgical procedures in their lifetime. Over the past 20 years, more and more have undergone cardiopulmonary bypass. While the surgical procedures themselves have improved, little research has been done to investigate whether medications might be beneficial to patients after surgery. Infants are especially likely to spend prolonged time on mechanical ventilation due to poor heart function after surgery and fluid build-up in the lungs.
Portman believes these effects are worsened because cardiopulmonary bypass causes an inflammatory response that suppresses normal thyroid hormone production. Still, relatively few randomized clinical trials have evaluated agents, like thyroid supplement, provided to these infants after cardiopulmonary bypass.
"Seattle Children's is focused on using cutting-edge research to offer the best in clinical care,"
Portman said. "If this study is successful, patients will spend less time on a ventilator, less time in cardiac intensive care, and less time in hospital. They will have the chance to grow up and live longer, healthier lives."
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