Propranolol was found comparable to steroids for efficacy and safety as a first-line treatment for infantile hemangioma, according to the results of a randomized clinical trial published in JAMA Dermatology.
The noninferiority study, which was conducted at a single academic hospital in the Republic of Korea, equally randomized 34 patients (15 boys, 19 girls, mean age 3.3 months) to one of the two treatment groups.
Patients in the propranolol group were admitted to the hospital, observed for adverse effects for three days after initiation of treatment of 2 mg/kg/d, and then released and treated as outpatients for the remainder of 16 weeks.
The steroid group received outpatient treatment from the beginning, also at 2 mg/kg/d for a total of 16 weeks. Hemangioma lesion volume was evaluated by magnetic resonance imaging (MRI). At 16 weeks, the treatment response rate was 95.65% for propranolol and 91.94% for steroid. There were also no safety differences between the two groups.
Prednisolone was first-line therapy for hemangiomas for many years before propranolol was recognized as a treatment for infantile hemangiomas, according to Kimberly Morel, M.D., an associate professor of dermatology and pediatrics at Columbia University Medical Center (CUMC), New York, who was not part of the study.
“At the time the current study was initiated in 2013, clinicians were gaining experience with propranolol as a treatment option,” she says.
In fact, the first FDA-approved treatment for infantile hemangiomas, propranolol hydrochloride (Hemangeol, Pierre Fabre), was approved in March 2014, during the course of the study.
“What is surprising is that the methods in the study include an MRI to assess hemangioma volume before treatment and after therapy,” Dr. Morel says. “Although an objective measure, MRI is usually reserved for differentiating deep hemangiomas from other lesions under the skin surface, or for evaluating for central nervous system vascular abnormalities that may be seen in patients with segmental hemangiomas.”
In addition, hemangiomas are often treated for longer than 16 weeks, according to Dr. Morel. A randomized, controlled trial of oral propranolol for infantile hemangiomas in the New England Journal of Medicine in 2015 showed more effect on hemangiomas after six months of therapy compared to three months. Overall, 60% of just over 100 patients assigned to propranolol 3 mg/kg/d had complete or near complete resolution of their hemangioma at week 24. Therefore, “a longer treatment period in the current study may have detected a more significant difference between the groups.”
The authors of the current study note that 3 mg/kg/day of propranolol has been shown to be superior to 1 mg/kg/day. “However, in practice, hemangiomas often respond well to 2 mg/kg/day, so comparing to a higher dose may not have made a significant difference,” Dr. Morel says.