01:50pm Wednesday 23 August 2017

SLU Researchers Test New Drug for Common Infant Skin Condition

Elaine Siegfried, M.D.

A hemangioma is a non-cancerous tumor made of tiny blood vessels, that affects approximately one in 10 babies within the first three months of life. While many hemangiomas will heal on their own, a significant number of babies are at risk for complications, including disfigurement and ulceration.

Hemangiomas most often develop on the face and scalp. The appearance can vary from a flat pink patch to a massive, red, raised plaque. Most hemangiomas are not noticeable at birth, but appear within the first month as a faint pink discoloration or a web of fine blood vessels. Hemangiomas may grow very fast, but rarely continue to enlarge beyond 12 months. Fortunately, 50 percent of hemangiomas will be completely gone by age 5, and 90 percent by age 9.

Researchers are studying the safety and effectiveness of propranolol, a 40-year-old medication most often used to treat high blood pressure, in shrinking hemangiomas. The drug was serendipitously discovered while treating a child who had a severe hemifacial hemangioma that was not responsive to high-dose corticosteroids. When the child was given propranolol to treat steroid-induced cardiomyopathy, the hemangioma also shrank.

“For more than half of affected infants, hemangiomas eventually fade away. In these cases, there is no need to interfere,” said Elaine Siegfried, M.D., chief of pediatric dermatology at Saint Louis University and SSM Cardinal Glennon Children’s Medical Center, and principal investigator of the study at SLU.

“However, more than one third of affected infants have a hemangioma that grows very fast, is located in a cosmetically sensitive area, interferes with the child’s vision or breathing or is at risk of ulcerating. Treatment may be necessary to prevent or minimize serious complications for these infants.”

Currently, there is no FDA-approved treatment for the condition, though options include corticosteroids or surgery.

“The initial report of propranolol treatment for hemangioma, published in the New England Journal of Medicine, was promising. More information is needed about the safety and effectiveness of the drug, but if this study is successful, it will pave the way for approval of a less invasive treatment with few side effects,” Siegfried said.

SLU is the only research site in Missouri. Locally, researchers hope to enroll up to 30 babies in the Stage II/III study. Study participants will be randomly assigned to one of five groups to compare four regiments of propranolol to a placebo group.

To qualify for the study, participants must be under 5 months of age and have a hemangioma the size of a dime or larger anywhere on their body, other than in the diaper area.

The study will last up to two years, and will include 24 weeks of medication and regular visits to Cardinal Glennon Children’s Medical Center. Participants will receive study-related medication, evaluation and laboratory tests at no cost.

For more information about the study, call Terry Rhodes at (314) 678-5457 or e-mail trhodes3@slu.edu

Sara Savat
314.977.8018

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