A novel topical rapamycin cream for the treatment of facial angiofibromas in tuberous sclerosis complex

J Child Neurol. 2013 Jul;28(7):933-6. doi: 10.1177/0883073813488664. Epub 2013 May 16.

Abstract

Facial angiofibromas are dermatological manifestations of tuberous sclerosis complex, a neurocutaneous disorder characterized by excess cell growth and proliferation. Oral rapamycin has been used to treat visceral tuberous sclerosis-related tumors; however, the side effect profile of this medicine precludes its routine use in patients lacking significant internal involvement. The authors formulated a novel rapamycin cream that is easy to compound and apply, does not cause local or systemic side effects, and results in a dramatic improvement of facial angiofibromas.

Keywords: facial angiofibroma; rapamycin; treatment; tuberous sclerosis complex.

Publication types

  • Case Reports

MeSH terms

  • Angiofibroma / drug therapy*
  • Angiofibroma / etiology
  • Angiofibroma / pathology
  • Antibiotics, Antineoplastic / administration & dosage*
  • Child
  • Facial Neoplasms / drug therapy*
  • Facial Neoplasms / etiology
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Sirolimus / administration & dosage*
  • Skin Cream / administration & dosage
  • Time Factors
  • Tuberous Sclerosis / complications

Substances

  • Antibiotics, Antineoplastic
  • Sirolimus