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Original Investigation
April 19, 2017

Comparison of Efficacy and Safety Between Propranolol and Steroid for Infantile HemangiomaA Randomized Clinical Trial

Author Affiliations
  • 1Department of Dermatology, Seoul National University College of Medicine, Seoul, Republic of Korea
  • 2Institute of Human-Environment Interface Biology, Seoul National University Medical Research Center, Seoul, Republic of Korea
  • 3Laboratory of Cutaneous Aging and Hair Research, Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea
  • 4Department of Plastic and Reconstructive Surgery, Institute of Human-Environment Interface Biology, Seoul National University College of Medicine, Seoul, Republic of Korea
  • 5Division of Medical Statistics, Medical Research Collaborating Center, Seoul National University College of Medicine, Seoul National University Hospital, Seoul, Republic of Korea
  • 6Department of Plastic and Reconstructive Surgery, Seoul National University Hospital, Seoul, Republic of Korea
  • 7Department of Radiology, Seoul National University College of Medicine, Seoul, Republic of Korea
  • 8Department of Medicine, Seoul National University School of Medicine (Master Course), Seoul, Republic of Korea
  • 9Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul National University Children’s Hospital, Seoul, Republic of Korea
  • 10Department of Plastic and Reconstructive Surgery, Seoul National University Bundang Hospital, Gyeonggi, Republic of Korea
JAMA Dermatol. Published online April 19, 2017. doi:10.1001/jamadermatol.2017.0250
Key Points

Question  Can propranolol be used as a first-line treatment for infantile hemangioma (IH)?

Findings  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.

Meaning  Propranolol can be used as a first-line treatment for IH.

Abstract

Importance  There are limited data from randomized clinical trials comparing propranolol and steroid medication for treatment of infantile hemangioma (IH).

Objective  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.

Interventions  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.

Results  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.

Trial Registration  clinicaltrials.gov Identifier: NCT01908972

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