Propranolol may benefit those with severe infantile hemagiomas

Article Outline

• Question
• Design
• Setting
• Participants
• Intervention
• Outcomes
• Main Results
• Conclusions
• Commentary
• Copyright

Sans V, de la Roque ED, Berge J, Grenier N, Boralevi F, Mazereeuw-Hautier J, et al. Propranolol for severe infantile hemangiomas: follow-up report. Pediatrics 2009;124:e423-31.

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Question 

Among children with severe infantile hemangiomas, can the use of propranolol control the rapid growth phase, as well as accelerate involution in older lesions?

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Design 

Observational case series.

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Setting 

France.

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Participants 

32 children (21 girls; mean age at onset of treatment: 4.2 months).

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Intervention 

Propranolol, starting dose of 2 to 3 mg/kg per day, given in 2 or 3 divided doses.

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Outcomes 

Clinical observation and ultrasound measurements were performed after 60 days of treatment.

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Main Results 

Immediate effects on color and growth were noted in all cases and were especially dramatic in cases of dyspnea, hemodynamic compromise, or palpebral occlusion. In ulcerated infantile hemangiomas, complete healing occurred in <2 months. Objective clinical and ultrasound evidence of longer-term regression was seen in 2 months. Systemic corticosteroid treatment could be stopped within a few weeks. Treatment was administered for a mean total duration of 6.1 months. Relapses were mild and responded to retreatment. Late intervention treatment of fully developed lesions also accelerated involution. Side effects were limited and mild. One patient discontinued treatment because of wheezing.

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Conclusions 

Propranolol has a consistent, rapid, therapeutic effect, leading to considerable shortening of the natural course of infantile hemangiomas, with good clinical tolerance.

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Commentary 

“Chance favors the prepared mind.” This was the case when Leaute-Labreze used propranolol to treat corticosteroid-induced hypertension in an infant with a complicated hemangioma and noted a rapid decrease in size of the tumor. The recognition of the potential significance of this serendipitous finding led to the present study. Although flaws exist in the design methodology, the potential impact of the findings is significant. The study was observational and lacked any control or comparative arm. Different drug doses and intervals occurred. Age of enrollment varied, and more than one third of the infants were receiving concurrent corticosteroid therapy. Ultrasonography was available for only one third of the patients. Absent a control group, the significance of the changes noted is hard to ascertain in a disease with natural involution. Propranolol can induce hypoglycemia; no monitoring occurred for this outcome, which may have caused some of the adverse effects noted (agitation, profuse sweats, cold hands). Unfortunately, no FDA-approved drug exists for the treatment of complicated infantile hemangiomas. The most commonly used agents (corticosteroids, interferon, and vincristine) all have significant side effects and inconsistent efficacy. Propranolol offers the possibility of a safer and more effective option, but dose-response data, as well as controlled, prospective, and comparative trials that carefully monitor safety, are ongoing and may confirm these very positive initial findings.