Summary
Since first reported in 2008 that propranolol, a beta blocker, was effective in the treatment of Infantile Hemangiomas (IH)—a birthmark that most commonly appears as a rubbery, bright red nodule of extra blood vessels in the skin, commonly called a “strawberry”—this class of drug has been used as the first line of therapy for infants with IH. Timolol, also a beta blocker, is available in topical formulation, and increasingly used off-label for small, non-complicated IH. The popularity of timolol is likely due to its perceived safety as a topical drug. However, data on timolol efficacy, safety and pharmacokinetics are limited.
PTN’s timolol study enrolled 100 infants between the ages of ≥32 to <50 weeks postmenstrual age. The infants were treated with timolol for 28 days then randomized into 2 groups. One group continued timolol treatment while the other was withdrawn from treatment. Both groups remained on the study in their respective groups for up to 120 days. The study began enrolling in summer 2016 at 10 U.S. sites. The data received from the study will be presented to the FDA to support the use of timolol for the treatment of IH.
Publications
- Systemic Timolol Exposure Following Topical Application to Infantile HemangiomasJournal of the American Academy of Dermatology • February 2019. Drolet BA, Boakye-Agyeman F, Harper B, Holland K, Lewandowski A, Stefanko N, Melloni C; Pediatric Trials Network Steering Committee. Off-label ophthalmic timolol has been rapidly adopted for treatment of infantile hemangioma since topical application of beta-blockers was presumed to have an improved safety profile compared to oral ...
OVERVIEW
Status:
Analysis ongoing
ClinicalTrials.gov Identifier:
NCT02913612
Principal Investigators:
Beth Ann Drolet, MD and Kristen Holland, MD
Medical College of Wisconsin
Milwaukee, WI
NEWS
- Timolol for Infantile Hemangiomas under ReviewSince first reported in 2008 that propranolol, a beta blocker, was effective in the treatment of Infantile Hemangiomas (IH)—a birthmark that most commonly appears as a rubbery, bright red nodule of extra blood vessels in the skin, commonly called a “strawberry”—this class of drug has been used as the first line of therapy for infants ...