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.

Publication types

  • Multicenter Study

MeSH terms

  • Administration, Topical
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Immunosuppressive Agents / administration & dosage*
  • Infant
  • Lymphatic Abnormalities / drug therapy*
  • Lymphatic Abnormalities / pathology
  • Male
  • Retrospective Studies
  • Sirolimus / administration & dosage*
  • Treatment Outcome
  • Vascular Malformations / drug therapy*
  • Vascular Malformations / pathology
  • Young Adult


  • Immunosuppressive Agents
  • Sirolimus