Topical timolol treatment for infantile hemangiomas: a phase II multicentre randomized clinical trial

Abstract

Infantile hemangiomas (IHs) are common benign vascular tumours of infancy, although the best approach to their management remains unclear. IHs have a characteristic natural course, with a rapid proliferating phase followed by a spontaneous involuting phase, leaving in many cases residual lesions. The standard approach to uncomplicated IHs is “wait and see” but active intervention may be necessary since they can reach a considerable size and the aesthetic outcome is unpredictable. In addition, IHs can cause psychological distress in affected children and their parents. Recently, topical timolol has emerged as a new therapy option for treating uncomplicated IHs, but literature data are still lacking Objective: We aim to evaluate the efficacy and safety of topical timolol maleate 0,5% solution for treating superficial and mixed IHs Methods: A phase II, multicentre, randomized, double-blind, placebo-controlled, parallelgroup trial will be conducted during an estimated period of 2 years. Infants between 1 and 12 months with a focal, superficial or mixed IH will be recruited and randomly assigned to placebo and treatment groups. Efficacy will be assessed by performing blinded investigator photograph scoring and blinded volume measurements with ultrasound imaging at weeks 0, 1, 2, 3, 4, 8, 12, 16, 20 and 24. Safety will be assessed by measuring timolol in serum at week 4, and measuring heart rate and blood pressure and reporting adverse events at weeks 0, 1, 2, 3, 4, 8, 12, 20 and 24. Follow up will be carried out during 6 months in order to detect rebound