Objective: To report the benefits and complications of subcutaneous interfe
ron alfa-2a therapy for hemangiomas in children.
Design: Prospective nonrandomized trial.
Setting: Tertiary care pediatric referral center.
Patients: Twenty-four pediatric patients diagnosed with massive or life-thr
eatening hemangiomas.
Interventions: Each patient received daily subcutaneous injections of inter
feron alfa-2a to a target dose of 3 million U/m(2) of body surface area for
a minimum of 4 months. Nineteen patients completed therapy and have receiv
ed adequate follow-up.
Main Outcome Measures: Clinical and radiographic comparisons before, during
, and after therapy. Reduction in hemangioma size was graded as complete (>
90%), substantial (50%-80%), intermediate (20%-40%), or no response (<10%).
Results: Mean age at institution of therapy was 9.6 months, and mean durati
on of treatment was 10.2 months. Most patients (70%) had not received prior
therapy. Responses were as follows: complete, 8 patients (42%); substantia
l, 3 patients (16%);intermediate, 5 patients (26%); and no response, 3 pati
ents (16%) (n=19). During therapy, 5 patients (26%) developed neurological
abnormalities: 3 had an unsteady gait, and 2 had fine motor deficits. Only
1 of these 5 patients required premature termination from the study, and th
e neurological abnormalities in all 5 patients resolved after treatment was
discontinued. Two of the 4 patients with neurological findings who complet
ed therapy demonstrated complete resolution of their hemangiomas. Patients
who developed neurological abnormalities began interferon alfa-2a therapy a
t an earlier age (4.7 months) than patients without neurological difficulti
es (aged 11.1 months). The mean time from initiation of therapy to the appe
arance of neurological complications was 4.8 months.
Conclusions: In pediatric patients with massive or life-threatening hemangi
omas, interferon alfa-2a therapy is an effective treatment option. However,
neurological evaluation before and during therapy with interferon alfa-2a
should be performed owing to a significant incidence of neurological abnorm
alities (28%). Although all children with neurological findings demonstrate
d neurological recovery after discontinuation of therapy, we have changed o
ur protocol and now more gradually increase the dosage of interferon alfa-2
a up to 3 million U/m(2) per day. The effect of this modification on the de
velopment of neurological abnormalities has not yet been determined.