Barcelona, Spain—Administering oral propranolol beyond 6 months and up to a year increases the success rate in treatment of high-risk infantile hemangioma (IH), a new study reports.

Background information in the article notes that the benign vascular tumors, characterized by endothelial-like cell proliferation, have an estimated incidence of 4% to 5%.

Propranolol is the first-line therapy in IH treatment, and its efficacy and safety were previously demonstrated in a placebo-controlled study (complete and/or near complete resolution in 60% of patients after 6 months of treatment at 3 mg/kg per day) in which high-risk IH were excluded.

The article in the journal Pediatrics points out, however, the current lack of a consensus on optimal treatment duration for propranolol in IH when the condition doesn’t improve.

“Although most IHs have an uncomplicated clinical course, some are associated with complications that can be life altering or life- and function threatening,” wrote the study authors, who an international group of researchers. “Permanent sequelae can be psychologically distressing. In a large U.S. cohort study involving 1,058 children with IH, 24% experienced complications, with the most common being ulceration and, to a lesser extent, threat to vision and airway obstruction.”

Seeking to improve treatment success rates, the study team evaluated the efficacy and safety of oral propranolol solution administered for a minimum of 6 months up to a maximum of 12 months of age in high-risk IH. The researchers conducted a single-arm, open-label, phase III study in patients aged 35 to 150 days with high-risk IH in 10 hospitals between 2015 and 2017. Patients had a 6-month initial treatment period (ITP) and then continuation up to a year if total success was not achieved. A follow-up and retreatment period came next in patients where that was necessary.

Results indicated that the success rate after 6 months of treatment was 47%, increasing to 76% at the end of the initial trial period. Of the children in whom treatment was successful, 68% sustained that success for 3 months without treatment and 24% required re-treatment. All but one of the eight patients re-treated achieved success.

The infants received oral propranolol twice daily at 3 mg/kg per day. Adverse events, reported in 80% of patients, were mild, and all were expected in this population or were known propranolol side effects.

“Oral propranolol administered beyond 6 months and up to 12 months of age meaningfully increases the success rate in high-risk IH,” the study authors conclude. “Success was sustained in most patients up to three months after stopping treatment. Retreatment was efficacious, and the safety profile satisfactory.”

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