A randomized, controlled trial of oral propranolol in infantile hemangioma.

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. (Funded by Pierre Fabre Dermatologie; ClinicalTrials.gov number, NCT01056341.).

[Spanish consensus on infantile haemangioma]. / Consenso español sobre el hemangioma infantil.

INTRODUCTION: Infantile haemangiomas are benign tumours produced by the proliferation of endothelial cells of blood vessels, with a high incidence in children under the age of one year (4-10%). It is estimated that 12% of them require treatment. This treatment must be administered according to clinical practice guidelines, expert experience, patient characteristics and parent preferences. METHODS: The consensus process was performed by using scientific evidence on the diagnosis and treatment of infantile haemangiomas, culled from a systematic review of the literature, together with specialist expert opinions. The recommendations issued were validated by the specialists, who also provided their level of agreement. RESULTS: This document contains recommendations on the classification, associations, complications, diagnosis, treatment, and follow-up of patients with infantile haemangioma. It also includes action algorithms, and addresses multidisciplinary management and referral criteria between the different specialities involved in the clinical management of this type of patient. CONCLUSIONS: The recommendations and the diagnostic and therapeutic algorithms of infantile haemangiomas contained in this document are a useful tool for the proper management of these patients.

Consenso español sobre el hemangioma infantil / Spanish consensus on infantile haemangioma

INTRODUCCIÓN: Los hemangiomas infantiles son tumores benignos producidos por la proliferación de células endoteliales de vasos sanguíneos, con una alta incidencia en niños menores de un año (4-10%) y se estima que un 12% de ellos requiere tratamiento. Dicho tratamiento debe realizarse según las guías de práctica clínica y la experiencia de los especialistas, las características de los pacientes y las preferencias de sus progenitores. MÉTODOS: El proceso de consenso se realizó utilizando evidencias científicas sobre el diagnóstico y tratamiento de los hemangiomas infantiles, extraídas mediante una revisión sistemática de la literatura, junto con el juicio experto de los especialistas. Las recomendaciones formuladas fueron validadas por los especialistas, aportando su grado de acuerdo. RESULTADOS: El presente documento recoge recomendaciones sobre la clasificación, las asociaciones, las complicaciones, el diagnóstico, el tratamiento y el seguimiento de los pacientes con hemangioma infantil. Además, se incluyen algoritmos de actuación y se aborda el manejo multidisciplinario y criterios de derivación entre los distintos especialistas que participan en el manejo clínico de este tipo de pacientes. CONCLUSIONES: Las recomendaciones y los algoritmos diagnóstico y terapéutico de los hemangiomas infantiles recogidos en este documento son una herramienta útil en el manejo adecuado de estos pacientes

INTRODUCTION: Infantile haemangiomas are benign tumours produced by the proliferation of endothelial cells of blood vessels, with a high incidence in children under the age of one year (4-10%). It is estimated that 12% of them require treatment. This treatment must be administered according to clinical practice guidelines, expert experience, patient characteristics and parent preferences. METHODS: The consensus process was performed by using scientific evidence on the diagnosis and treatment of infantile haemangiomas, culled from a systematic review of the literature, together with specialist expert opinions. The recommendations issued were validated by the specialists, who also provided their level of agreement. RESULTS: This document contains recommendations on the classification, associations, complications, diagnosis, treatment, and follow-up of patients with infantile haemangioma. It also includes action algorithms, and addresses multidisciplinary management and referral criteria between the different specialities involved in the clinical management of this type of patient. CONCLUSIONS: The recommendations and the diagnostic and therapeutic algorithms of infantile haemangiomas contained in this document are a useful tool for the proper management of these patients