Stanford/Packard Team Seeks Infants With Hemangiomas To Test Drug Treatment

For Release: July 19, 2011

STANFORD, Calif. — Researchers from the Stanford University School of Medicine and Lucile Packard Children’s Hospital Stanford are seeking infants with a type of birthmark made up of blood vessels known as infantile hemangiomas (also called strawberry marks) for a study of a drug that may shrink the non-cancerous tumors. The study is investigating whether propranolol, a drug that is approved by the U.S. Food and Drug Administration for use in treating children with heart problems and high blood pressure, can safely and effectively treat infants’ hemangiomas.

Who: The Stanford/Packard team is enrolling 20 infants with hemangiomas at least three-quarters of an inch in diameter, anywhere on their body except for the diaper area. Subjects must be 35 to 150 days (5 months) old to participate.

Why: In the first three months of life, about 10 percent of infants will develop a hemangioma. Most hemangiomas disappear on their own by age 9, but some cause lasting deformities. Large hemangiomas may result in skin breakdown (ulcerate) and become infected. Established treatments such as steroid drugs and surgery have undesirable side effects and are not always effective.

In 2008, reports from a group of French physicians discovered that propranolol might be effective in shrinking hemangiomas. The doctors were treating a girl with a large, disfiguring facial hemangioma. After receiving high doses of steroids, the girl developed heart failure, which then necessitated treatment with propranolol. Her hemangioma quickly began to shrink, prompting an investigation into propranolol’s safety and efficacy for treating infantile hemangiomas. “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  Latanya Benjamin, MD, a pediatric dermatologist at Packard Children’s Hospital and a clinical assistant professor of dermatology and of pediatrics at Stanford. She is leading the Stanford arm of the study, which has over 60 sites in Europe and the United States. Benjamin noted that propranolol is now being used “off-label” to treat infantile hemangiomas; however the safety data, proper dosing and long-term side effects are currently not known. “This study is important as it will help to provide us with this information gathered from infants who are carefully monitored in the study,” she said.

When and where: The infants will receive study medication for six months and be monitored for a total of two years to see what ultimately happens after drug treatment ends. The participants will have 15 checkups at Packard Children’s Hospital during the study, and receive study treatment at no charge. Some infants may be randomized to receive a placebo (medication without active study drug); there is a one out of nine chance of receiving placebo.

How: Those who want more information about the trial may contact Emily Gorell at (650) 721-7166 or egorell@stanford.edu.

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.

Author

Erin Digitale
(650) 724-9175
digitale@stanford.edu