Infantile Hemangiomas: Daily Timolol Maleate Slows Growth

Timolol maleate 0.5% gel is safe and effective in treating superficial infantile hemangiomas (IHs) for young infants, significantly reducing lesion volume, proportional growth, and clinical appearance without the worrisome adverse effects associated with other treatments. Hsien Chan, MBBS, from the Department of Pediatric Dermatology at Sydney Children's Hospital in Randwick, Australia, and colleagues report the findings of their randomized, double-blind, placebo-controlled trial in an article published online May 6 in Pediatrics. Among infants, IHs are among the most common tumors, affecting roughly 4% of children. Although case reports have suggested that timolol maleate 0.5% gel is efficacious in treating IHs, safety concerns have lingered. Dr. Chan and colleagues enrolled 41 children with superficial IHs who were aged 5 to 24 weeks. Parents of the 19 children who were in the active study group applied and gently rubbed in a single drop of the timolol maleate gel, an estimated 0.25-mg dose of timolol, twice daily. A placebo was similarly applied to the 22 children in the control group. The infants' blood pressure and heart rate were measured before and after application of the gel or placebo, and 2 measurements of the lesion size were taken before and after treatment. These measurements were repeated at weeks 0, 1, 2, 3, 4, 8, 12, 16, 20, and 24 of the study. Nine children dropped out of the study. "There was a significantly higher proportion of treated IHs that reduced in size by >5% at weeks 20 and 24 (P < .02). The predicted proportion of IH volume change was also significantly less for treated IHs from week 16 onward when compared with placebo (P < .05)," Dr. Chan and coauthors write. Blood pressure and heart rates among infants in the treatment group did not vary significantly from those of the infants in the placebo group. One of the study limitations was that all of the children were enrolled from a single medical facility. The authors note that a larger, multicenter trial could provide additional granularity about efficacy, optimal dosing, and subgroup analysis by age. "The onset of action appears to be slower than oral propranolol chloride with significant improvements in absolute volume reduction, proportional growth, and clinical appearance noted after only 12 to 16 weeks," the authors conclude. "The efficacy of topical timolol maleate 0.5% gel appears to be more pronounced for lesions with a mean diameter of <11.3 mm (ie, <100 mm3 in vol)." Support for this study was provided by the Sydney Children's Hospital and the Touched by Olivia Foundation supported a fellowship position. The authors have disclosed no relevant financial relationships. Pediatrics. Published online May 6, 2013.