Randomised controlled trial: Can topical timolol maleate prevent complications for small superficial infantile haemangiomata in high-risk areas?

• Published in: Pediatric research Vol. 88; no. 5; pp. 756 - 760
• Main Authors: Cheng, James W. C. H., Lam, Ying-Yin, Fung, Genevieve P. G., Sin, Conor, Luk, David C. K., Chan, Bill H. B., Chiu, Wa-Keung
• Format: Journal Article
• Language: English
• Published: New York Nature Publishing Group US 01.11.2020; Nature Publishing Group
• Subjects: Babies; Blood vessels; Clinical Research Article; Clinical trials; Hemangioma; Medicine; Medicine & Public Health; Pediatric Surgery; Pediatrics; Preventive medicine; Topical medication; Tumors
• ISSN: 0031-3998; 1530-0447
• DOI: 10.1038/s41390-020-0917-3

Background To define the role of topical timolol maleate (TTM) in the treatment of infantile haemangiomata (IH). Methods In this single-centre randomised controlled trial, we included all <1-year-old infants within a 13-month period presenting with small (<2 cm) superficial IH located at high-risk areas (i.e. tip of ears, tip of nose, eyelids, acral areas, facial areas, scalp, neck, buttocks, perineum and axilla). Patients either received 12 months of 0.5% TTM solution (study group) or watchful waiting (control group). The primary outcome was IH with development of complications that required additional interventions. The secondary outcomes included side effects of TTM and change in IH size. Results Forty-two children were eligible to the study. Patients who received TTM were noted to have significantly fewer complications than the control group (4.2% versus 29%, odds ratio 9.58 [95% confidence interval: 1.01–91.62], p  = 0.04). Mean IH volume percentage reduction was significantly more for the TTM group and no-TTM group at 3, 6 and 12 months. Conclusions TTM is an effective and safe treatment option to reduce complications, IH volume and the need for further intervention for infants with small superficial IH located at high-risk areas. Impact There is a lack of reliable signs to predict ulceration, disfigurement and other complications for high-risk IH. Treatment options range from watchful waiting to early systemic treatment, with TTM a novel and promising treatment. The exact role of TTM remains unanswered due to a lack of evidence-based research. TTM is effective and safe for infants with superficial IH of <2 cm in high-risk areas. Early TTM use on IH can reduce complications, IH volume and the need for further treatment.