A randomized, controlled trial of oral propranolol in infantile hemangioma

Christine Leaute-Labreze, Peter Hans Hoeger, Juliette Mazereeuw-Hautier, Laurent Guibaud, Eulalia Baselga, Gintas Posiunas, Roderic J Phillips, Hector Caceres, Juan Carlos Lopez Gutierrez, Rosalia Angelica Ballona Chambergo, Sheila Fallon Friedlander, Julie Powell, Danuta Perek, Brandie Jean Metz, Sebastien Barbarot, Annabel Maruani, Zsuzsanna Zsofia Szalai, Alfons L Krol, Olivia Boccara, Regina Foelster-HolstMaria Isabel Febrer Bosch, John Chit Su, Hana Buckova, Antonio Torrelo, Frederic Cambazard, Rainer Grantzow, Orli Wargon, Dariusz Wyrzykowski, Jochen Roessler, Jose Bernabeu-Wittel, Adriana M Valencia-Herrera, Przemyslaw Przewratil, Sharon A Glick, Elena Pope, Nicholas M Birchall, Latanya Benjamin, Anthony J Mancini, Pierre Vabres, Pierre Souteyrand, Ilona J Frieden, Charles I Berul, Cyrus R Mehta, Sorilla Prey, Franck Boralevi, Caroline Claire Morgan, Stephane Roland Heritier, Alain Delarue, Jean-Jacques Voisard

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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
Original languageEnglish
Pages (from-to)735 - 746
Number of pages12
JournalThe New England Journal of Medicine
Issue number8
Publication statusPublished - 2015

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