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A randomized, controlled trial of oral propranolol in infantile hemangioma
Christine Léauté-Labrèze1, Peter Hoeger, Juliette Mazereeuw-Hautier
1The authors' affiliations are listed in the Appendix.
Insights
Oral propranolol effectively treats infantile hemangiomas. A 6-month treatment of 3 mg/kg/day demonstrated significant resolution in infants, with minimal adverse events.
Area of Science:
- Pediatric Dermatology
- Pharmacology
- Clinical Trials
Background:
- Infantile hemangiomas are common vascular tumors in infants.
- Oral propranolol is a recognized treatment, but robust clinical trial data are limited.
- Systemic therapy is indicated for complicated or rapidly growing hemangiomas.
Purpose of the Study:
- To assess the efficacy and safety of a pediatric-specific oral propranolol solution.
- To determine the optimal dosage and duration for treating infantile hemangiomas.
- To provide high-quality evidence for the use of propranolol in this condition.
Main Methods:
- A multicenter, randomized, double-blind, adaptive phase 2-3 trial was conducted.
- Infants (1-5 months) with proliferating infantile hemangioma were randomized to placebo or propranolol regimens.
- The primary endpoint was treatment success at 24 weeks, evaluated via blinded photographic assessment.
Main Results:
- The regimen of 3 mg/kg/day propranolol for 6 months was selected based on interim analysis.
- Treatment success was significantly higher with propranolol (60%) compared to placebo (4%, P<0.001).
- Adverse events were infrequent and similar between propranolol and placebo groups.
Conclusions:
- Oral propranolol at 3 mg/kg/day for 6 months is effective for infantile hemangioma.
- The treatment demonstrated a high rate of resolution and a favorable safety profile.
- This study provides strong evidence supporting propranolol use in infantile hemangioma management.
Background:
Oral propranolol has been used to treat complicated infantile hemangiomas, although data from randomized, controlled trials to inform its use are limited.
Methods:
We performed a multicenter, randomized, double-blind, adaptive, phase 2-3 trial assessing the efficacy and safety of a pediatric-specific oral propranolol solution in infants 1 to 5 months of age with proliferating infantile hemangioma requiring systemic therapy. Infants were randomly assigned to receive placebo or one of four propranolol regimens (1 or 3 mg of propranolol base per kilogram of body weight per day for 3 or 6 months). A preplanned interim analysis was conducted to identify the regimen to study for the final efficacy analysis. The primary end point was success (complete or nearly complete resolution of the target hemangioma) or failure of trial treatment at week 24, as assessed by independent, centralized, blinded evaluations of standardized photographs.
Results:
Of 460 infants who underwent randomization, 456 received treatment. On the basis of an interim analysis of the first 188 patients who completed 24 weeks of trial treatment, the regimen of 3 mg of propranolol per kilogram per day for 6 months was selected for the final efficacy analysis. The frequency of successful treatment was higher with this regimen than with placebo (60% vs. 4%, P<0.001). A total of 88% of patients who received the selected propranolol regimen showed improvement by week 5, versus 5% of patients who received placebo. A total of 10% of patients in whom treatment with propranolol was successful required systemic retreatment during follow-up. Known adverse events associated with propranolol (hypoglycemia, hypotension, bradycardia, and bronchospasm) occurred infrequently, with no significant difference in frequency between the placebo group and the groups receiving propranolol.
Conclusions:
This trial showed that propranolol was effective at a dose of 3 mg per kilogram per day for 6 months in the treatment of infantile hemangioma. (Funded by Pierre Fabre Dermatologie; ClinicalTrials.gov number, NCT01056341.).
