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NIH picks MUSC for heart disease network by Cindy Abole Public Relations A five-year, multi-million dollar program to begin a Pediatric Heart Disease Clinical Network by the National Institutes of Health, yielded award grants of $2.5 million for six clinical centers, including MUSC. J. Philip Saul, division chief for Pediatric Cardiology and director of the Children's Heart Program of South Carolina, recently learned that MUSC has been chosen from more than 30 applicants as one of the clinical centers. The program is designed to pioneer and coordinate new treatment methods and strategies for children with congenital and acquired heart disease through a national research network. The grant, sponsored by the National Heart, Lung and Blood Institute (NHLBI), is the first of its kind on a national level, and was developed to address the problem of small patient numbers for these diseases at any individual center.  “We are particularly delighted to have the Children’s Heart Program at MUSC chosen as one of the centers because the competition was keen,” said J. Philip Saul, M.D. “Many prominent pediatric centers across the country competed for the grants including Texas Children’s Hospital, the University of California-San Francisco and the University of Michigan.”   Despite the fact that heart disease is the most frequent congenital defect, occurring in about 1 percent of all live births, small patient numbers, very few randomized trials, similar to those that are common with adult heart disease, have ever been performed. These trials are critical to scientifically determine optimum treatment strategies for any disease.  The program will team seven clinical centers to create the Pediatric Heart Disease Clinical Research Network. MUSC is in good company with the other centers including Boston’s Children’s Hospital, Children’s Hospital of Philadelphia, Sick Kids in Toronto, Columbia-Presbyterian in New York, Primary Children’s in Salt Lake and Duke Children’s Hospital and Health Center.  A steering committee, composed of Saul and other participating principal investigators, will decide on which studies will be performed, initially choosing from studies proposed in the original applications. Network partners will then recruit qualified patients, perform the studies and transfer the resulting data to a coordinating center.  Asked how he attributes the award to MUSC’s success, Saul credits the unified efforts of the Children’s Heart Program of South Carolina, which is anchored at MUSC. It  provides comprehensive inpatient and outpatient cardiovascular care to all children of South Carolina through a statewide network of pediatric cardiologists in Charleston, Columbia, Greenville and Florence.  “This kind of cooperation is virtually unique to South Carolina and is a tribute to all the participants, as well as the patients throughout the Palmetto State who reap the benefits,” Saul said. The heart program’s strong three-year research program, excellent clinical outcomes and program components, including the unique surgical expertise and support of colleagues Fred Crawford, M.D., chairman of the Department of Surgery and Scott Bradley, M.D., assistant professor of Surgery, are important factors. Each year in the United States, it is estimated that approximately 32,000 infants are born in the with congenital cardiovascular defects or other malformations. The incidence of congenital heart disease exceeds all childhood cancers and pediatric AIDS together. The medical, social and economic impact relating to the consequences of pediatric heart disease are profound with high medical expenses, often the need for additional procedures and disruption of family life.  Although treatment of pediatric heart disease involves medical, surgical and catheter-based approaches, most treatment decisions are not evidence-based. Since 1975, fewer than 20 randomized clinical trials have been conducted. Barriers to clinical studies concerning pediatric heart disease include heterogeneity of conditions, the small number of patients and the complex treatment approaches.  Another obstacle to research includes the absence of a systematic, centralized database and resources for collaboration on a national level. MUSC’s faculty have participated in more than 20 multi-center clinical trials or registries since Saul’s arrival in 1998. He has been the lead investigator in four of these national trials.  In addition, MUSC faculty have successfully coordinated 11 local clinical research protocols. Such studies will continue and be expanded with the kind of funding needed to sustain them, with the support from the new pediatric heart disease network.  The network will also serve as a platform to train junior investigators in pediatric clinical research and other cost-saving collaborations. Its outcomes should accelerate research in the diagnosis and management of congenital and acquired pediatric heart disease, help standardize existing treatments and evaluate new therapies with an emphasis on clinical studies using optimal diagnosis, monitoring and therapy. “This network ushers in a new era in the care of children with heart disease,” Saul said, citing the enormous progress made during the last 40 years that has allowed more than 95 percent of these children to survive childhood, when cared for in the best centers like MUSC. “Now is the time to optimize and standardize this care for every child in the U.S. We are indeed fortunate to be a part of this coming transformation.”