Treatment of superficial vascular anomalies with topical sirolimus: A multicenter case series

Pediatr Dermatol. 2020 Mar;37(2):272-277. doi: 10.1111/pde.14104. Epub 2020 Jan 19.


Background: Systemic sirolimus (rapamycin) has recently been found effective in treating complex vascular anomalies by reducing the size and associated complications. Many vascular anomalies have a cutaneous component, and thus, we sought to determine whether topical administration of sirolimus may be an effective therapy, as data on the use of topical sirolimus are limited.

Objective: We reviewed the efficacy and tolerability of topical formulations of sirolimus in the treatment of various simple and combined vascular malformations and tumors.

Methods: Eighteen patients with any vascular anomaly treated exclusively with topical sirolimus were retrospectively reviewed.

Results: Eleven patients had combined venous lymphatic malformations, three had tufted angiomas, two had a lymphatic malformation, one had a venous malformation, and one had a verrucous venous malformation. All (100%) patients reported some degree of improvement and 50% of patients reported marked improvement in one or more symptoms, most commonly blebs and lymphatic drainage, and bleeding.

Limitations: The retrospective nature, small number of patients, and differences in topical preparations limit the broad application of the results.

Conclusion: Topical sirolimus appears to be a safe and useful non-invasive therapy that is well-tolerated in the treatment of the cutaneous portion of a variety of vascular anomalies.

Keywords: genetic diseases/mechanisms; hemangiomas/vascular tumors; therapy topical; vascular malformation.