Can propranolol be used as a first-line treatment for infantile hemangioma (IH)?
In this noninferiority randomized clinical trial of 34 infants, the treatment response rate in the propranolol group was 95.65%, and that of the steroid group was 91.94%. Because the difference in response rate between the groups was 3.71%, propranolol was considered noninferior to steroid in therapeutic effects in IH, and there was no difference between the groups in safety outcomes.
Propranolol can be used as a first-line treatment for IH.
There are limited data from randomized clinical trials comparing propranolol and steroid medication for treatment of infantile hemangioma (IH).
To determine the efficacy and safety of propranolol compared with steroid as a first-line treatment for IH.
Design, Setting, and Participants
This randomized clinical noninferiority trial tested the efficacy and safety of propranolol vs steroid treatment for IH at a single academic hospital. All participants were diagnosed with IH between June 2013 and October 2014, had normal heart function, and had not been previously treated for IH.
The participants were randomly assigned to either the propranolol group or the steroid group. In the propranolol group, the patients were admitted, observed for adverse effects for 3 days after treatment initiation, and then released and treated as outpatients for 16 weeks (2 mg/kg/d). In the steroid group, the patients were seen as outpatients from the beginning and were also treated for 16 weeks (2 mg/kg/d).
Main Outcomes and Measures
The primary efficacy variable was the response to treatment at 16 weeks, which was evaluated by the hemangioma volume using magnetic resonance imaging before and at 16 weeks after treatment initiation. While comparing the effect of medication between the groups, we monitored the adverse effects of both drugs.
A total of 34 patients (15 boys, 19 girls; mean age, 3.3 months; range, 0.3-8.2 months) were randomized to receive either propranolol or steroid treatment (17 in each treatment group). Guardians for 2 patients in the steroid group withdrew their consent, and 1 patient in the propranolol group did not complete the efficacy test. The intention-to-treat analysis, applying multiple imputations, found the treatment response rate in the propranolol group to be 95.65%, and that of the steroid group was 91.94%. Because the difference in response rate between the groups was 3.71%, propranolol was considered noninferior. We found that there was no difference between the groups in safety outcomes.
Conclusions and Relevance
Our trial demonstrated that propranolol was not inferior to steroid with respect to therapeutic effects in IH.
clinicaltrials.gov Identifier: NCT01908972
Kim KH, Choi TH, Choi Y, Park YW, Hong KY, Kim DY, Choe YS, Lee H, Cheon J, Park J, Park KD, Kang HJ, Shin HY, Jeong JH. Comparison of Efficacy and Safety Between Propranolol and Steroid for Infantile HemangiomaA Randomized Clinical Trial. JAMA Dermatol. Published online April 19, 2017. doi:10.1001/jamadermatol.2017.0250