Vascular Malformations, Tumors, and Hemangiomas Pediatric Research and Clinical Trials

Clinical research conducted by investigators in the Vascular Anomalies Center has resulted in new, more effective treatments. For example, the center's investigators were the first to use interferon alpha in the treatment of children with large life- or vision-threatening hemangiomas. Dr. Mulliken has described a simple technique for removal of hemangioma leaving a smaller scar than with conventional methods. In addition, Dr. Fishman has developed novel approaches to control intestinal bleeding caused by certain colorectal vascular malformations, such as blue rubber bleb nevus syndrome.

Research Underway

Some institutions have started treating hemangiomas with a beta blocker called propranolol; however, its effectiveness and risks compared to corticosteroid have not been studied. To find out more, Children's Vascular Anomalies Center is currently recruiting patients for a prospective, randomized trail to determine the safety and efficacy of propranolol versus corticosteroid.

Research by Joyce Bischoff, PhD, Research Associate for Boston Children's Vascular Biology Program, is revealing some causes of infantile hemangioma. Her research indicates that hemangioma growth may be due to an in utero mutation in a stem cell destined to become an endothelial cell. The mutation then causes the endothelial cells to multiply at an abnormal rate leading to the hemangioma. Dr. Bischoff and colleagues are currently working to identify potential medication to target this cell, thereby halting the growth or preventing its enlargement altogether.

Vascular anomalies—birthmarks caused by abnormal development of lymph or blood vessels—are usually relatively stable. But sometimes they progress rapidly, requiring aggressive treatment to save the child's health or vision. Boston Children's is investigating how urine testing can help predict when an anomaly is about to become a serious threat, and raises the possibility of new drug treatments.