Now Cohen and Puttgen have more evidence showing the efficacy of propranolol as a first-line therapy for infants with function-threatening or severely disfiguring hemangiomas. In their recent retrospective analysis of 70 infants treated with propranolol, 53 patients were assessed as “good” responders (fair to marked improvement) vs. 17 as “poor” responders (minimal improvement to worsened appearance). There was only one complication, hypoglycemia, in a patient with a viral illness who recovered. Steroids, the standard therapy for hemangiomas, are associated with serious side-effects like elevated blood sugars and reduced resistance to infection. The conclusion?
“Propranolol therapy is largely effective as a primary agent in the treatment of infantile hemangionmas,” says Puttgen. “Nobody got worse, and the dose of 2.0 mg/kg/day was safe with minimal drug related side effects.”
Other findings? The earlier you start therapy, Puttgen says, the better patients responded. Why? Propranolol, Puttgen explains, down-regulates nitric oxide expression and growth factors, decreasing the formation of new blood vessels.
“Theoretically it makes sense that younger kids, who have more of those elevated growth factors because their hemangiomas are growing and growing, would respond more dramatically to the medicine because there is more there to down-regulate,” Puttgen says. “The older you get, the less likely you are to have a good response.”
Because the drug has been shown to decrease the size of the hemangiomas so rapidly, Puttgen adds, it may also reduce the number of hemangioma patients requiring plastic surgery. While most hemangiomas resolve over time, some patients are left with disfiguring scar tissue.
“The difficulty is complete involution doesn’t necessarily mean complete resolution,” Puttgen says. “The proof is going to be when these kids reach school age, but with this treatment I think a lot fewer kids are going to need cosmetic surgery in the future.”
More data and randomized controlled trials are needed, but for now propranolol is being used at Hopkins Children’s as an effective tool against function-threatening and severely disfiguring hemangiomas.
“There’s a lot of variability in how much pediatric dermatologists are using this,” Puttgen says. “We were early adopters thinking this is a great therapeutic option, which has become a first-line therapy.”