An update on the treatment of hemangiomas in children with interferon alfa-2a.

OBJECTIVE: To report the benefits and complications of subcutaneous interferon 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-threatening hemangiomas.

INTERVENTIONS: Each patient received daily subcutaneous injections of interferon alfa-2a to a target dose of 3 million U/m2 of body surface area for a minimum of 4 months. Nineteen patients completed therapy and have received 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 duration of treatment was 10.2 months. Most patients (70%) had not received prior therapy. Responses were as follows: complete, 8 patients (42%); substantial, 3 patients (16%), intermediate, 5 patients (26%); and no response, 3 patients (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 the neurological abnormalities in all 5 patients resolved after treatment was discontinued. Two of the 4 patients with neurological findings who completed therapy demonstrated complete resolution of their hemangiomas. Patients who developed neurological abnormalities began interferon alfa-2a therapy at an earlier age (4.7 months) than patients without neurological difficulties (aged 11.1 months). The mean time from initiation of therapy to the appearance of neurological complications was 4.8 months.

CONCLUSIONS: In pediatric patients with massive or life-threatening hemangiomas, 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 abnormalities (28%). Although all children with neurological findings demonstrated neurological recovery after discontinuation of therapy, we have changed our protocol and now more gradually increase the dosage of interferon alfa-2a up to 3 million U/m2 per day. The effect of this modification on the development of neurological abnormalities has not yet been determined.