The news has energized pediatric dermatologists worldwide, many of whom are now trying propranolol on their tiny patients -- with what they describe as remarkable success. Others are more cautious about the treatment, waiting for formal clinical trials before committing themselves.
The first report of this use for propranolol came in a letter from French physicians that appeared in the June 12 issue of the New England Journal of Medicine.
- Explain to parents that propranolol, a beta-blocker normally used for hypertension, appears to help shrink infant hemangiomas.
- Note that, although it hasn't been thoroughly tested in infants, propranolol has a much more benign safety profile than the steroids typically used to treat hemangiomas.
Christine LÃ©autÃ©-LabrÃ¨ze, M.D., of Bordeaux Children's Hospital, and colleagues used the drug to treat an infant with cardiomyopathy and another with increased cardiac output, both of whom also had hemangiomas. Unexpectedly, the lesions began to fade.
They subsequently tried propranolol on nine other children with hemangiomas -- all with the same success.
The news convinced researchers at Johns Hopkins to develop a protocol for the beta-blocker as a first-line treatment for the skin disorder. There was good reason for their enthusiasm, according to Bernard A. Cohen, M.D., director of pediatric dermatology at the Johns Hopkins Children's Center.
The most common treatment for hemangioma today involves steroids such as prednisone, which carry the risk of side effects that include elevated blood sugars, water retention, osteopenia, gastritis, reduced resistance to infection, and retarded growth. "We don't like to use them unless we absolutely have to," Dr. Cohen said.
On the other hand, side effects of propranolol, including hypotension and hypoglycemia, are typically few and short-lived.
"It has a safety profile that's light years better than steroids," Dr. Cohen said.
So far, Dr. Cohen and Katherine Puttgen, M.D., also at Johns Hopkins, say they have treated 20 patients with propranolol. Working with cardiologists, they decided to hospitalize the infants for the first two days of treatment to monitor for possible side effects such as hypotension or hypoglycemia. (They have seen none so far.)
The children receive 1 mg/kg of propranolol on the first day, divided over three doses, and 2 mg/kg -- also divided in thirds -- after that.
"Sometimes after just the first dose you can see the hemangioma darken and flatten," Dr. Cohen said. "Within 24 hours, you can usually see real improvement."
Hemangiomas typically appear within a few weeks after birth and peak within three months. Up to 75% shrink to insignificance by the time the youngster reaches school age, Dr. Cohen said.
Researchers aren't sure why they occur, but Dr. Cohen noted that 75 percent occur in girls. They're also more common in white babies than African-American children, and more likely to appear in babies who were premature or whose mothers had complicated pregnancies.
Although they're usually harmless, Cohen noted that severe hemangiomas can be permanently disfiguring, interfere with breathing, vision, or excretion, or put dangerous pressure on internal organs. That's why doctors are anxious for a treatment to speed up the shrinking process.
Some pediatric dermatologists say it's premature to consider propranolol as a first line treatment in light of scattered reports of problems with the drug -- including babies who had to be hospitalized for hypotension and hypoglycemia after receiving the drug as outpatients.
"None of this information is published as of yet, but rather information shared between [members of the] academic pediatric community," noted Mary Wu Chang, M.D., of the University of Connecticut. "So, it appears that we do need further study of the use of propranolol in infants, that it may not be as safe as it is in older children and adults."
But Dr. Cohen and Dr. Puttgen said they believe propranolol is so much safer than the steroid alternatives that it's worth using as a first line treatment even while they're lining up support and funding for a formal clinical trial to prove the drug's safety and determine an optimal dose.
One problem may be funding: propranolol itself has been off patent for years and is relatively inexpensive, so there's little incentive for pharmaceutical companies to help finance the trial.
Cohen said his team's insistence on starting children as inpatients -- which raises the cost of treatment -- is necessary to deal with possible side effects and questions that arise from the lack of clinical trial data on the use of propranolol in young children. But he's still optimistic about the ultimate findings.
"I've been doing pediatric dermatology for 25 years, and this is the fastest I've ever seen a new therapy change the way we do things," he said.