Advanced Cardiomyopathy and Heart Failure Diagnosis and Treatments
The Heart Institute has become an international leader in heart muscle disease (cardiomyopathies) and its associated treatments. We have developed the largest Advanced Pediatric and Young Adult Cardiomyopathy and Heart Failure Program in the US, directed by John Lynn Jefferies, MD, MPH, and Jeffrey Towbin, MD. Our Heart Transplant Program, under the medical direction of Clifford Chin, MD, and surgical leadership of Alistair Phillips, MD, is one of the largest and most successful in the US. We performed 14 transplants in the past year and 10 the previous year, with 100 percent survival. More recently, the Heart Institute has become a leader in mechanical circulatory support with the addition of David Morales, MD, a world-renowned pediatric cardiothoracic surgeon and new chief of cardiothoracic surgery. Eight children with end-stage heart failure received mechanical circulatory support this year; six of whom were successfully transplanted and are doing well. Members of the Heart Institute also published more than 170 manuscripts this year.
Heart Institute Leads an International Research Consortium
Under the leadership of Jeffrey Robbins, PhD, the Heart Institute was awarded one of four grants given worldwide by the Fondation Leducq to study how proteotoxicity – cell malfunctions caused by misfolded proteins – can lead to heart disease and failure. The consortium for this study is led by Cincinnati Children’s and includes laboratories at the University of Texas-Southwest, University of North Carolina at Chapel Hill and premier academic medical centers in Germany, Great Britain and Italy. These proteotoxicity studies were recognized by the Leducq Foundation as having the potential to lead to novel treatments for a variety of heart diseases, with new therapeutic targets being identified in our pediatric population.
Pediatric Heart Network Consortium
The Heart Institute successfully competed for a renewal grant to continue serving as one of nine “core” sites in the Pediatric Heart Network (PHN) through 2016. This national competition was open to academic centers across North America. The National Heart, Lung and Blood Institute created this collaborative network to conduct clinical studies in children with congenital or acquired heart disease. Since its inception, the PHN has completed more than 10 multi-center studies that have had significant impact on the field of pediatric cardiovascular disease. The most recent study, evaluating the use of therapies for individuals with Marfan syndrome, began in 2007 and will have a major impact on how we treat these patients. These complex, randomized trials are the only way of carrying out high quality clinical research in rare diseases. Cincinnati Children’s has been a core site in the PHN since 2006. James Cnota, MD and Catherine Krawczeski, MD, serve as principal investigators.