Pioneering Translational Research
The Heart Institute at Cincinnati Children’s Hospital Medical Center is currently conducting a Pediatric Heart Network trial titled “Marfan Trial of Randomized Beta Blocker Therapy (atenolol) vs. Angiotensin II Receptor Blocker (ARB) Therapy (losartan)” that is as close to translational research as can be found in pediatric cardiology. The Heart Institute is conducting this trial as a PHN core site. Cincinnati Children's is one of eight centers with experienced research teams specializing in the care of patients with heart disease that was chosen to become part of the PHN.
The Marfan trial compares the effect of atenolol to that of losartan on the rates of aortic growth and progression of aortic regurgitation in patients with Marfan Syndrome (MFS). MFS is a systemic disorder of connective tissue due to mutation in the Fibrillin-1 gene. Cardiovascular pathology, including aortic root dilation, dissection and rupture is the leading cause of mortality in patients with MFS.
Several studies have shown that beta blockers reduce the rate of aortic growth, but these effects have been small. Recent studies in a Fibrillin 1-deficient mouse model of MFS with a susceptibility to aortic dilation and dissection similar to that seen in humans with MFS demonstrated that treatment with losartan normalized aortic root growth and architecture, preventing aortic aneurysms and premature death. Preliminary data from a small pilot trial suggests potential benefit of losartan use in humans with MFS.
Research On The Horizon
In addition to the Marfan trial, a great deal of translational research has been discussed among the world-class team assembled at Cincinnati Children’s. A current example that has been discussed extensively is the early intervention into the cardiac dysfunction of Duchenne Muscular Dystrophy. With the progress being made by the CMR group under William M. Gottliebson, MD, at Cincinnati Children's, a clinical trial appears probable in the next few years.
Contact Us About the Marfan Trial
For more information about the Marfan trial, email our Research Coordinator, Michelle Hamstra at michelle.hamstra@cchmc.org or call PHN Marfan Trial site Principal Investigator, D. Woodrow Benson, MD, PhD at 513-636-3891.
