This retrospective study included 17 infants with vision-threatening periocular hemangiomas treated with oral propranolol on an outpatient basis. Low-dose therapy (25 percent of full dose) was initiated between 3 weeks and 12 months of age and slowly increased to full dosage (2 mg/kg daily) over the course of one to two weeks.
Treatment was continued until there was complete resolution or regression to the point of eliminating the visual compromise, or at age 9 to 11 months, depending on the clinician's discretion. Once resolution or regression was achieved, treatment was reduced slowly over the course of a few weeks.
The authors screened infants with large segmental hemangiomas for PHACES, a condition associated with abnormal aortic and cerebral vasculature, which may place them at higher risk for developing adverse effects from the hemodynamic changes induced by propranolol.
The tumors stopped growing in all patients and reduced in size by more than 50 percent in 10 patients. Six patients experienced mild adverse effects, including gastric reflux, fatigue, gastrointestinal upset, and slight ''shakiness'' due to a missed dose. No symptoms were severe enough to discontinue treatment. Only six of the 17 patients had any complaints, all minor.
This apparent low side effect profile coupled with the ability to treat on an outpatient basis make oral propranolol treatment a promising treatment for infantile hemangioma. Current mainstays of hemangioma treatment - intralesional or systemic steroids - have well-known, frequent, and sometimes severe side effects. Other medications, such as vincristine, interferon-alpha, and cyclophosphamide, can have other potential adverse effects and pose additional challenges regarding administration and monitoring. Due to the many real and theoretical benefits of avoiding admission to the hospital, the authors hope this study will encourage others to further explore the safety and effectiveness of this modality of treatment.
While very little is known about the mechanism of action or the ideal dosage and time to start treatment, the authors conclude that carefully administered and observed outpatient oral propranolol can be an effective starting strategy for periocular infantile hemangioma.