Conditions & Treatments
- A to Z Guide

Marfan syndrome

Disease Information

Research & Innovation

Children’s Hospital Boston is constantly looking for ways to prevent and treat diseases and disorders, including Marfan syndrome.

Problem: Marfan syndrome affects one in 5,000 Americans and weakens the aorta (the blood vessel that carries blood away from the heart to the body), often causing the vessel wall to bulge, forming an aneurysm.

Innovative solution: Traditional treatment for Marfan syndrome often includes beta blockers to lower blood pressure and reduce stress on the aorta. But researchers at Children’s may have found a better solution: We’re testing the blood pressure drug losartan, which may strengthen the aorta and help prevent aortic aneurysms.

Losartan inhibits signaling by transforming growth factor-beta, a protein now known to be overactive in Marfan syndrome, and blocks a cascade of cellular events that weaken aortic tissue. In a landmark 2006 study, losartan completely prevented aortic aneurysms in mice with Marfan syndrome—and even reversed existing aortic damage.

A Phase III trial, one of the largest Marfan studies ever done andco-led by Ronald Lacro, MD, director of Children’s Cardiovascular Genetics Program, has enrolled about 600 patients from 6 months to 25 years of age, as patients in this age group tend to experience the greatest incidence of cardiovascular-related complications.The patients have been randomly assigned to receive either losartan or the beta-blocker atenolol, and are being closely followed for three years.

History of innovation
In 1938, Children’s cardiac surgeon Robert Gross, MD, performed the world’s first successful surgery to correct a child’s heart defect. Since then, we’ve gained recognition around the globe for our leadership in pediatric cardiology, and we continue to make critical advances in the field. In 2010, U.S. News & World Report named Children’s cardiology and cardiac surgery programs the best of any pediatric hospital in the country.

History of innovation

In 1938, Children’s cardiac surgeon Robert Gross, MD, performed the world’s first successful surgery to correct a child’s heart defect. Since then, we’ve gained recognition around the globe for our leadership in pediatric cardiology, and we continue to make critical advances in the field. In 2010, U.S. News & World Report named Children’s cardiology and cardiac surgery programs the best of any pediatric hospital in the country.