Timolol for Infantile Hemangiomas under Review

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.

Under the protocol thought leadership of Dr. Beth Drolet, Principal Investigator, the PTN’s Timolol study will enroll 100 infants between the ages of ≥32 to <50 weeks postmenstrual age. The infants will be treated with timolol for 28 days then randomized into 2 groups. One group will continue timolol treatment while the other will be withdrawn from treatment. Both groups will remain on the study in their respective groups for up to 120 days. The study will begin to enroll in the early summer of 2016 in approximately 10 US sites, and will end in the fall of 2017. The data received from the study will be presented to the FDA to support the use of timolol for the treatment of IH.