Early initiation of treatment with oral propranolol for infantile hemangioma improves success rate.

ABSTRACT

BACKGROUND/OBJECTIVES: Early referral and treatment of infantile hemangioma (IH) is a major challenge for treatment success. However, there is a lack of data supporting a specific threshold for initiating treatment with oral propranolol. The aim of this analysis was to find factors, such as age at treatment initiation, leading to a higher success rate with oral propranolol treatment. METHODS: Based on data from the pivotal phase 2-3 clinical trial of oral propranolol in IH, we used Generalized Additive Model (GAM) charts with Generalized Linear Models (GLM), then a rule discovery algorithm, to identify sub-groups presenting a high probability of occurrence of the predefined outcome (i.e., success [complete or nearly complete resolution of the target hemangioma] at 6 months of treatment). RESULTS: Our analyses identified that patients who started oral propranolol 3 mg/kg/day before the age of 10 weeks had a success rate of 86%, higher than the 60% success rate for all patients that received the same regimen commencing after 10 weeks of age. CONCLUSIONS: Treatment initiation before 10 weeks of age was associated with a significantly higher rate of treatment success with oral propranolol 3 mg/kg/day. Infants with IH requiring treatment should be referred to an expert center and treated as soon as possible.