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Infantile hemangiomas (IH) affect up to 10% of infants. A rapid proliferative phase is followed by slow spontaneous involution. Approximately 12% of IH develop complications necessitating intervention. Systemic corticosteroids were standard treatment, but response was variable, and adverse effects were common. In 2008, the serendipitous discovery that propranolol successfully treats IH led to its widespread use, despite lack of controlled trials.
These authors performed a multicenter, randomized, controlled trial of propranolol versus placebo and its long-term safety in 343 patients (age, 1-5 months) with a proliferating IH (>1.5 cm) requiring systemic therapy who completed 96 weeks of follow-up. Patients were randomized to receive placebo or propranolol (1 mg/kg/day for 3 months, 1 mg/kg/day for 6 months, 3 mg/kg/day for 3 months, or 3 mg/kg/day for 6 months).
The primary outcome was success (complete/nearly complete resolution of target IH) or failure at week 24 versus baseline, evaluated by independent, blinded readers of photographs. The highest benefit-to-risk ratio was seen with 3 mg/kg/day for 6 months (success, 60% vs. 4% with placebo). Adverse effects were infrequent and mild to moderate in severity. Significant adverse events were rare: One patient had bradycardia with intercurrent enterocolitis, and one with a pre-existing cardiac condition had second-degree atrioventricular block necessitating discontinuation.
Léauté-Labrèze C et al. A randomized, controlled trial of oral propranolol in infantile hemangioma. N Engl J Med 2015 Feb 19; 372:735. (http://dx.doi.org/10.1056/NEJMoa1404710)
Comment
Propranolol was recently FDA approved for treatment of infantile hemangiomas, but the optimal treatment protocol is not yet known. The commonly used dosing of 2 mg/kg/day was not studied here, but 3 mg/kg/day appears to be similarly tolerated. Partial improvement was not captured, so the treatment value is likely higher. Although rare, pre-existing cardiac conditions should be ruled out before beginning therapy. Propranolol treatment should be managed by a pediatric dermatologist or physician familiar with its use to minimize hypoglycemia, bronchospasm, and other adverse events.