A Randomized, Controlled Trial of Oral Propranolol in Infantile
Hemangioma

J 2015

A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma

LEAUTE-LABREZE, C.; P. HOEGER; J. MAZEREEUW-HAUTIER; L. GUIBAUD; E. BASELGA et al.

Základní údaje

Originální název

A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma

Autoři

LEAUTE-LABREZE, C.; 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; A. KROL; O. BOCCARA; R. FOELSTER-HOLST; M. I. FEBRER BOSCH; J. SU; Hana BUČKOVÁ; 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 a J-J VOISARD

Vydání

New England Journal of Medicine, Waltham, MASSACHUSETTS MEDICAL SOC, 2015, 0028-4793

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30209 Paediatrics

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 59.558

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/15:00082536

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

TREATMENT SELECTION; NATURAL-HISTORY; INFANCY; CORTICOSTEROIDS; MANAGEMENT; ANOMALIES; CHILDREN; THERAPY; DESIGNS

Štítky

EL OK

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 28. 5. 2015 13:49, Soňa Böhmová

Anotace

V originále

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.

Citovat

@article{1230641,
   author = {LeauteandLabreze, C. and Hoeger, P. and MazereeuwandHautier, J. and Guibaud, L. and Baselga, E. and Posiunas, G. and Phillips, R. J. and Caceres, H. and Lopez Gutierrez, J. C. and Ballona, R. and Friedlander, S. F. and Powell, J. and Perek, D. and Metz, B. and Barbarot, S. and Maruani, A. and Szalai, Z. Z. and Krol, A. and Boccara, O. and FoelsterandHolst, R. and Febrer Bosch, M. I. and Su, J. and Bučková, Hana and Torrelo, A. and Cambazard, F. and Grantzow, R. and Wargon, O. and Wyrzykowski, D. and Roessler, J. and BernabeuandWittel, J. and Valencia, A. M. and Przewratil, P. and Glick, S. and Pope, E. and Birchall, N. and Benjamin, L. and Mancini, A. J. and Vabres, P. and Souteyrand, P. and Frieden, I. J. and Berul, C. I. and Mehta, C. R. and Prey, S. and Boralevi, F. and Morgan, C. C. and Heritier, S. and Delarue, A. and Voisard, JandJ},
   article_location = {Waltham},
   article_number = {8},
   doi = {https://doi.org/10.1056/NEJMoa1404710},
   keywords = {TREATMENT SELECTION; NATURAL-HISTORY; INFANCY; CORTICOSTEROIDS; MANAGEMENT; ANOMALIES; CHILDREN; THERAPY; DESIGNS},
   language = {eng},
   issn = {0028-4793},
   journal = {New England Journal of Medicine},
   title = {A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma},
   volume = {372},
   year = {2015}
}

TY  - JOUR
ID  - 1230641
AU  - Leaute-Labreze, 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, A. - Boccara, O. - Foelster-Holst, R. - Febrer Bosch, M. I. - Su, J. - Bučková, Hana - 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
PY  - 2015
TI  - A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma
JF  - New England Journal of Medicine
VL  - 372
IS  - 8
SP  - 735-746
EP  - 735-746
PB  - MASSACHUSETTS MEDICAL SOC
SN  - 00284793
KW  - TREATMENT SELECTION
KW  - NATURAL-HISTORY
KW  - INFANCY
KW  - CORTICOSTEROIDS
KW  - MANAGEMENT
KW  - ANOMALIES
KW  - CHILDREN
KW  - THERAPY
KW  - DESIGNS
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<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.
ER  -

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