Efficacy of propranolol between 6 and 12 months of age in high-risk infantile hemangioma

Propranolol has been established as the first-line therapy for infantile hemangioma (IH), but its safety and efficacy specifically in high-risk IH populations had not been demonstrated. Researchers in Poland, France, and Spain sought to close this knowledge gap through a Phase III study involving 45 participants with high-risk IG.

Propranolol has been established as the first-line therapy for infantile hemangioma (IH), but its safety and efficacy specifically in high-risk IH populations had not been demonstrated. Researchers in Poland, France, and Spain sought to close this knowledge gap through a Phase III study involving 45 participants with high-risk IG. The participants, aged roughly 1–5 months, received oral propranolol twice daily for a period of 6 months. If the therapy was successful at that point, patients were managed another 3 months with no propranolol and re-treated for up to 6 more months in the event of rebound growth. If, on the other hand, the therapy was unsuccessful in the initial treatment period, the intervention was extended until achieving the desired outcome or until the patient reached age 1 year—whichever occurred first. The results favored the described protocol in children with high-risk IH, which can be disfiguring, debilitating, or even deadly. The success rate after 6 months of propranolol treatment was 47%, and 76% when treatment was prolonged to up to age 12 months. In the subset of enrollees who benefited during the first 6 months, 68% sustained success during the non-treatment phase; and only 24% required re-treatment. Adverse events were common and expected, but mild in nature.