Children’s Hospital researchers are close to completing a study on the use of a medication called Sildenafil as a complement to a three-stage surgical intervention to treat complex congenital heart disease, such as hypoplastic left heart syndrome (HLHS) — a disease in which babies are born with an underdeveloped left side of their heart. HLHS, and other forms of complex single-ventricle heart disease, are universally fatal without surgery.
To correct this defect, a series of three open heart surgical procedures — pioneered at Children’s Hospital of Philadelphia and now utilized worldwide — are performed to properly direct blood flow to the lungs and body. While these procedures are lifesaving, survivors typically have a diminished capacity to exercise and may develop secondary problems in other organs, such as the lungs and liver. Truly long-term outcomes are unknown at this time.
David Goldberg, MD, along with several other Cardiac Center physician-scientists in a group led by Jack Rychik, MD, is completing a ground-breaking study to improve on these outcomes. Sildenafil, which is currently approved by the Food and Drug Administration to treat high blood pressure in the lungs in adults, is being studied to assess its impact in this complex congenital heart defect. The Cardiac Center staff expects that treatment with Sildenafil will improve the exercise ability and overall quality of life in patients with HLHS and other forms of single-ventricle heart disease.
A significant gift from Mr. and Mrs. Mark Harrington has given Goldberg and collaborators the chance to test this hypothesis in a pilot study here at the Cardiac Center, the first of its kind. Should preliminary information suggest that this new approach to treatment is beneficial, it would lay the groundwork for further large-scale, multi-institutional research. HLHS and other forms of single-ventricle heart disease affect nearly 1,000 children each year, and there are an estimated 20,000 children and young adults who might benefit from this new approach.