Oral versus high-dose pulse corticosteroids for problematic infantile hemangiomas: a randomized, controlled trial.
OBJECTIVE
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.


METHODS
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.


RESULTS
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.


CONCLUSIONS
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.
Pope
Elena
E
University of Toronto, Section of Dermatology, Hospital for Sick Children, 555 University Ave, Toronto, Ontario, Canada M5G 1X8. elena.pope@sickkids.ca
Krafchik
Bernice R
BR
Macarthur
Colin
C
Stempak
Diana
D
Stephens
Derek
D
Weinstein
Miriam
M
Ho
Nhung
N
Baruchel
Sylvain
S
D000284:Administration, Oral; D000305:Adrenal Cortex Hormones / Q000008:administration & dosage* / Q000009:adverse effects; D015415:Biomarkers / Q000097:blood; D005260:Female; D006391:Hemangioma / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D006973:Hypertension / Q000139:chemically induced; D007223:Infant; D007275:Injections, Intravenous; D008297:Male; D010147:Pain Measurement / Q000187:drug effects; D012128:Respiratory Distress Syndrome / Q000139:chemically induced; D019010:Vascular Cell Adhesion Molecule-1 / Q000097:blood
D000305:Adrenal Cortex Hormones; D015415:Biomarkers; D019010:Vascular Cell Adhesion Molecule-1