Oral Versus High-Dose Pulse Corticosteroids for Problematic Infantile Hemangiomas: A Randomized, Controlled Trial

University of Toronto, Section of Dermatology, Hospital for Sick Children, 555 University Ave, Toronto, Ontario, Canada M5G 1X8.
PEDIATRICS (Impact Factor: 5.47). 07/2007; 119(6):e1239-47. DOI: 10.1542/peds.2006-2962
Source: PubMed


Oral systemic corticosteroids are the mainstay of treatment for problematic hemangiomas; however, current information is based on anecdotal experience and retrospective studies. We aimed to determine whether systemic steroids are efficacious in proliferating hemangioma and to compare the efficacy and safety of 2 corticosteroid treatment modalities.
Twenty patients with problematic hemangiomas of infancy were randomly assigned to either daily oral prednisolone or monthly intravenous pulses of methylprednisolone. Their clinical outcomes (improvement using a visual analog score) and adverse events were compared at 3 months from baseline and 1 year of age. Data on possible surrogate markers of angiogenesis were available for the first 3 months.
At 3 months, orally treated patients had a median visual analog score of 70 compared with 12 in the intravenous group. This response pattern was similar at the patients' first birthday: 50.0 vs -1.5. Additional treatment beyond 3 months was needed for 65% of the patients (7 in the intravenous and 6 in the oral group). Six of 8 patients with impaired vision at enrollment had an improved function at 1 year (4 patients in the intravenous group and 3 patients in the oral group). Of the 4 surrogate markers of angiogenesis measured (plasma basic fibroblast growth factor, vascular endothelial growth factor, vascular cellular adhesion molecule 1, endoglin, and urine basic fibroblast growth factor), the only 2 that decreased over time were vascular cellular adhesion molecule 1 and endoglin. Patients in the oral group had a higher rate of adverse effects, such as hypertension (18.6% vs 13.1%), abnormal cortisol (78% vs 60%), and growth retardation.
Systemic corticosteroids are efficacious in stopping the proliferation of hemangiomas. The oral corticosteroids offered more clinical and biological benefit than the pulse steroids with higher risk of adverse effects.

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Available from: Sylvain Baruchel, Oct 05, 2015
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    • "Kasabach-Merritt syndrome, a platelet-trapping thrombocytopenic coagulopathy, and hepatic hemangiomas have a mortality rate of 30%–50%. Corticosteroid therapy worked for 30% of hemangiomas [59]. Radiation, cyclophosphamide treatment, and embolization were also tried and showed favorable outcomes; however, sometimes they showed toxicity. "
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    • "Systemic side effects are reported in the literature to occur in 27%–44%14,15 of cases when corticosteroids are used for prolonged treatment of periorbital hemangiomas. These include behavioral changes, insomnia, cushingoid appearance, and hypertension, which were all avoided in our study. "
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    • "Thus, treatment often begins during the proliferative phase as rapid growth can lead to worsening function, obstruction, and ⁄ or esthetics. Historic treatment options for infantile hemangiomas include systemic or intralesional corticosteroids, chemotherapeutic agents (vincristine, alpha-interferon), surgery , lasers, or a combination of these therapies (Bauman et al, 1997; Fledelius et al, 2001; Perez et al, 2002; Fawcett et al, 2004; Jalil et al, 2006; Pope et al, 2007; Buckmiller et al, 2008). Each treatment option has limited therapeutic benefit with its own side-effect profile and risks (Bauman et al, 1997; Adams, 2001; Goyal et al, 2004; Deboer and Boston, 2008). "
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