Stanford/Packard Team Seeks Infants with Hemangiomas for Trial of New Drug Treatment

For Release: April 28, 2010

PALO ALTO, Calif. — Researchers from the Stanford University School of Medicine are seeking infants with non-cancerous blood vessel tumors called hemangiomas for a study of a new treatment to shrink the tumors. Hemangiomas are overgrowths of blood vessels that cause red or purple blotches on the skin of about 3 percent of all infants. In most cases, the blotches grow for a few months, then shrink and disappear. However, some hemangiomas cause lasting facial deformities, interfere with development of vision or block breathing. Large hemangiomas may also ulcerate and become infected. The target of the new trial is to identify hemangiomas early and head off lasting disfigurement and complications. 

"Our goal is to find a safe, easy way to treat these tumors early in a child's life so that we can prevent deformity," said Alfred Lane, MD, chair of pediatric dermatology at Lucile Packard Children's Hospital and a professor of dermatology and of pediatrics at Stanford. Many children with serious hemangiomas now receive cortisone, a steroid drug, to shrink the tumors. But cortisone has undesirable side effects: It can increase blood pressure, cause obesity and stunt a child's linear growth. A second option, surgery, has the potential disadvantage of leaving children with disfiguring scars. So researchers are searching for a better treatment. The new study will investigate whether propranolol, a drug that is already FDA-approved to treat heart problems and high blood pressure, can safely and effectively treat infants' hemangiomas. Physicians in Europe noticed that hemangiomas shrank among children already receiving propranolol for heart problems. Lane's team is part of an international effort to confirm and extend these findings. Four hundred and seventy infants with hemangiomas are now being recruited to join the propranolol trial at 40 study sites in Europe and the United States. 

The Stanford/Packard team is recruiting 20 infants with facial hemangiomas, both boys and girls, to join the investigation. Prospective study subjects must be 35 to 150 days old and must have a hemangioma at least three-quarters of an inch in diameter on the face. The subjects will be randomized to receive three or six months of propranolol treatment, at one of two doses, or a placebo. All subjects will be tracked for two years to see what happens to their hemangiomas after drug treatment ends. The subjects will have 15 check-ups at Packard Children's Hospital during the trial. The research is funded by Pierre Fabre Dermatologie, a French company that makes propranolol and is providing the drug for the study. The pharmaceutical manufacturer is paying for the time and effort of the research team. The Stanford/Packard researchers have no financial stake or involvement in the company's operations.