First, in 2008, pediatric dermatologists Bernard Cohen and Kate Puttgen experienced firsthand what some French doctors had discovered serendipitously in treating infants with heart problems who also happened to have infantile hemangiomas – the hemangiomas swiftly faded after the infants received the hypertension drug propranolol. After administering the drug to their first patient, who had large hemangiomas threatening her airway and vision, Cohen and Puttgen reported a similar effect.
“Within 48 hours, the hemangiomas became softer,” Puttgen says.
“The hemangiomas were just a fraction of their initial size, and the eye complication completely resolved,” adds Cohen.
Parlaying their findings, Cohen and Puttgen collected more evidence showing the efficacy of propranolol for infants with function-threatening or severely disfiguring hemangiomas. In their retrospective analysis of 70 infants treated with propranolol, 51 patients had fair to marked improvement in their hemangiomas, whereas 17 percent had minimal improvement. The single reported complication, hypoglycemia, occurred in a patient with a viral illness who recovered. It seemed the two pediatric dermatologists had found a solid alternative for the standard therapy at the time, steroids, known for their serious side-effects, including elevated blood sugar and vulnerability to infection.
But there was another issue the two pediatric dermatologists had to deal with – the requirement that infants under 1 year of age be admitted to the hospital for administration of the drug and monitored because propranolol poses the risk of lowering their blood pressure. But after finding only two cases with complications among some 450 infants treated with propranolol in the past five years, Cohen and Puttgen moved ahead with an outpatient protocol for infants 2 months and older. Newborns under 2 months of age, as well as those with medical comorbidities, they note, still need to be admitted.
Under the outpatient protocol, Cohen explains, the child receives the first dose of propranolol in clinic and vital signs are checked before, during and after administration of the drug. Parents, meanwhile, get instructions on how to give the drug and monitor the child for side effects as the dosage is gradually increased over 10 days. The protocol was launched eight months ago. The results?
“We’ve had no problems. Zero. None of the kids had symptoms and there was no further drop in blood pressure after the first dose,” says Cohen. “The response to treatment is good or better than systemic corticosteroids, which was the standard of care before 2008.”
So, does all this mean propranolol is the new standard of care for severe and/or disfiguring hemangiomas?
Noting that there is no official standard of care – no U.S. Food and Drug Administration approved agents – for treating infantile hemangiomas, Cohen suggests the door is now open for propranolol, an already FDA-approved drug.
“We’re not quick to adopt new treatments but we were one of the first centers in the United States to initiate this treatment and now we’re very comfortable doing this,” says Cohen. “This is a big change in how we approach complex and severely disfiguring hemangiomas, and a big deal for pediatricians, patients and their families. In my mind, this has become the standard of care.”