A randomized, controlled trial of oral propranolol in infantile hemangioma

C. Léauté-Labrèze, P. Hoeger, J. Mazereeuw-Hautier, L. Guibaud, E. Baselga, G. Posiunas, R. J. Phillips, H. Caceres, J. C. Lopez Gutierrez, R. Ballona, S. F. Friedlander, J. Powell, D. Perek, B. Metz, S. Barbarot, A. Maruani, Z. Z. Szalai, Alfons Krol, O. Boccara, R. Foelster-HolstM. I. Febrer Bosch, J. Su, H. Buckova, A. Torrelo, F. Cambazard, R. Grantzow, O. Wargon, D. Wyrzykowski, J. Roessler, J. Bernabeu-Wittel, A. M. Valencia, P. Przewratil, S. Glick, E. Pope, N. Birchall, L. Benjamin, A. J. Mancini, P. Vabres, P. Souteyrand, I. J. Frieden, C. I. Berul, C. R. Mehta, S. Prey, F. Boralevi, C. C. Morgan, S. Heritier, A. Delarue, J. J. Voisard

Research output: Contribution to journalArticle

270 Citations (Scopus)

Abstract

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 (US)
Pages (from-to)735-746
Number of pages12
JournalNew England Journal of Medicine
Volume372
Issue number8
DOIs
StatePublished - Feb 19 2015

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Hemangioma
Propranolol
Randomized Controlled Trials
Placebos
Therapeutics
Random Allocation
Treatment Failure
Body Weight
Pediatrics
Safety

ASJC Scopus subject areas

  • Medicine(all)

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Léauté-Labrèze, C., Hoeger, P., Mazereeuw-Hautier, J., Guibaud, L., Baselga, E., Posiunas, G., ... Voisard, J. J. (2015). A randomized, controlled trial of oral propranolol in infantile hemangioma. New England Journal of Medicine, 372(8), 735-746. https://doi.org/10.1056/NEJMoa1404710

A randomized, controlled trial of oral propranolol in infantile hemangioma. / Léauté-Labrèze, C.; Hoeger, P.; Mazereeuw-Hautier, J.; Guibaud, L.; Baselga, E.; Posiunas, G.; Phillips, R. J.; Caceres, H.; Lopez Gutierrez, J. C.; Ballona, R.; Friedlander, S. F.; Powell, J.; Perek, D.; Metz, B.; Barbarot, S.; Maruani, A.; Szalai, Z. Z.; Krol, Alfons; Boccara, O.; Foelster-Holst, R.; Febrer Bosch, M. I.; Su, J.; Buckova, H.; Torrelo, A.; Cambazard, F.; Grantzow, R.; Wargon, O.; Wyrzykowski, D.; Roessler, J.; Bernabeu-Wittel, J.; Valencia, A. M.; Przewratil, P.; Glick, S.; Pope, E.; Birchall, N.; Benjamin, L.; Mancini, A. J.; Vabres, P.; Souteyrand, P.; Frieden, I. J.; Berul, C. I.; Mehta, C. R.; Prey, S.; Boralevi, F.; Morgan, C. C.; Heritier, S.; Delarue, A.; Voisard, J. J.

In: New England Journal of Medicine, Vol. 372, No. 8, 19.02.2015, p. 735-746.

Research output: Contribution to journalArticle

Léauté-Labrèze, C, Hoeger, P, Mazereeuw-Hautier, J, Guibaud, L, Baselga, E, Posiunas, G, Phillips, RJ, Caceres, H, Lopez Gutierrez, JC, Ballona, R, Friedlander, SF, Powell, J, Perek, D, Metz, B, Barbarot, S, Maruani, A, Szalai, ZZ, Krol, A, Boccara, O, Foelster-Holst, R, Febrer Bosch, MI, Su, J, Buckova, H, Torrelo, A, Cambazard, F, Grantzow, R, Wargon, O, Wyrzykowski, D, Roessler, J, Bernabeu-Wittel, J, Valencia, AM, Przewratil, P, Glick, S, Pope, E, Birchall, N, Benjamin, L, Mancini, AJ, Vabres, P, Souteyrand, P, Frieden, IJ, Berul, CI, Mehta, CR, Prey, S, Boralevi, F, Morgan, CC, Heritier, S, Delarue, A & Voisard, JJ 2015, 'A randomized, controlled trial of oral propranolol in infantile hemangioma', New England Journal of Medicine, vol. 372, no. 8, pp. 735-746. https://doi.org/10.1056/NEJMoa1404710
Léauté-Labrèze C, Hoeger P, Mazereeuw-Hautier J, Guibaud L, Baselga E, Posiunas G et al. A randomized, controlled trial of oral propranolol in infantile hemangioma. New England Journal of Medicine. 2015 Feb 19;372(8):735-746. https://doi.org/10.1056/NEJMoa1404710
Léauté-Labrèze, C. ; Hoeger, P. ; Mazereeuw-Hautier, J. ; Guibaud, L. ; Baselga, E. ; Posiunas, G. ; Phillips, R. J. ; Caceres, H. ; Lopez Gutierrez, J. C. ; Ballona, R. ; Friedlander, S. F. ; Powell, J. ; Perek, D. ; Metz, B. ; Barbarot, S. ; Maruani, A. ; Szalai, Z. Z. ; Krol, Alfons ; Boccara, O. ; Foelster-Holst, R. ; Febrer Bosch, M. I. ; Su, J. ; Buckova, H. ; Torrelo, A. ; Cambazard, F. ; Grantzow, R. ; Wargon, O. ; Wyrzykowski, D. ; Roessler, J. ; Bernabeu-Wittel, J. ; Valencia, A. M. ; Przewratil, P. ; Glick, S. ; Pope, E. ; Birchall, N. ; Benjamin, L. ; Mancini, A. J. ; Vabres, P. ; Souteyrand, P. ; Frieden, I. J. ; Berul, C. I. ; Mehta, C. R. ; Prey, S. ; Boralevi, F. ; Morgan, C. C. ; Heritier, S. ; Delarue, A. ; Voisard, J. J. / A randomized, controlled trial of oral propranolol in infantile hemangioma. In: New England Journal of Medicine. 2015 ; Vol. 372, No. 8. pp. 735-746.
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abstract = "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",
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AU - Léauté-Labrèze, C.

AU - Hoeger, P.

AU - Mazereeuw-Hautier, J.

AU - Guibaud, L.

AU - Baselga, E.

AU - Posiunas, G.

AU - Phillips, R. J.

AU - Caceres, H.

AU - Lopez Gutierrez, J. C.

AU - Ballona, R.

AU - Friedlander, S. F.

AU - Powell, J.

AU - Perek, D.

AU - Metz, B.

AU - Barbarot, S.

AU - Maruani, A.

AU - Szalai, Z. Z.

AU - Krol, Alfons

AU - Boccara, O.

AU - Foelster-Holst, R.

AU - Febrer Bosch, M. I.

AU - Su, J.

AU - Buckova, H.

AU - Torrelo, A.

AU - Cambazard, F.

AU - Grantzow, R.

AU - Wargon, O.

AU - Wyrzykowski, D.

AU - Roessler, J.

AU - Bernabeu-Wittel, J.

AU - Valencia, A. M.

AU - Przewratil, P.

AU - Glick, S.

AU - Pope, E.

AU - Birchall, N.

AU - Benjamin, L.

AU - Mancini, A. J.

AU - Vabres, P.

AU - Souteyrand, P.

AU - Frieden, I. J.

AU - Berul, C. I.

AU - Mehta, C. R.

AU - Prey, S.

AU - Boralevi, F.

AU - Morgan, C. C.

AU - Heritier, S.

AU - Delarue, A.

AU - Voisard, J. J.

PY - 2015/2/19

Y1 - 2015/2/19

N2 - 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

AB - 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

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