An Open-Label Single-Arm Clinical Trial of Propranolol Cream in Infantile Hemangioma

Abstract

Infantile hemangioma (IH) is a common tumor in infants that gradually resolves and is often followed up for observation. However, for cosmetic reasons, parents often opt for treatment. Oral propranolol, the first-line therapy for IH, shows several side effects, including hypotension, bradycardia, and hypoglycemia. No clinical studies on topical propranolol have been conducted using standardized procedures. We evaluated the efficacy and safety of topical propranolol in patients with IH. This multicenter, open-label phase II study was conducted from June 2019 to December 2020 and involved 8 Japanese infants aged 35–150 days with proliferating IH. Patients were treated with 5% propranolol cream twice daily. We examined the efficacy rate based on central evaluation (complete or near-complete healing of the target hemangioma) at weeks 24 and 12, respectively, compared to baseline values. The efficacy rate at week 24 was 68.8% (95% confidence interval: 44.1–85.9%). The surface area, maximum diameter, and color intensity of the target IH decreased over time. Adverse event and drug-related adverse event rates were 87.5% and 0%, respectively. Propranolol cream (5%) is effective and safe in Japanese patients with IH and may be considered a first-choice treatment for small and superficial IHs in cosmetically problematic areas.Trial registration numberThe trial is registered at the Japan Registry of Clinical Trials (jRCTs 041190041).Full date of first registration: 11/06/2019Web link: https://jrct.niph.go.jp/en-latest-detail/jRCTs041190041