A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma

BackgroundOral propranololhas been used to treat complicated infantile hemangiomas, although data from randomized, controlled trials to inform its use are limited. MethodsWe performed a multicenter, randomized, double-blind, adaptive, phase 2–3 trial assessing the efficacy and safety of a pediatric-specific oral propranololsolution in infants 1 to 5 months of age with proliferating infantile hemangiomarequiring systemic therapy. Infants were randomly assigned to receive placebo or one of four propranololregimens (1 or 3 mg of propranololbase per kilogramof body weight per day for 3 or 6 months). A preplanned interim analysiswas 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. ResultsOf 460 infants who underwent randomization, 456 received treat...