Successful treatment of angiofibromata of tuberous sclerosis complex with rapamycin

Abstract

Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous syndrome manifesting as hamartomatous growths in multiple organs. We present two cases of patients with TSC and associated facial angiofibromata treated with topical and oral rapamycin and discuss the role for rapamycin in the treatment of these disfiguring lesions. Our patients demonstrated decreased numbers of angiofibromata and less redness with this treatment. This is the first published report on the use of topical rapamycin for the treatment of angiofibromata.

Keywords:

• angiofibroma
• rapamycin
• tuberous sclerosis

Acknowledgements

The Center for Dermatology Research is supported by an educational grant from Galderma Laboratories, L.P.

Declaration of interest: Alan B. Fleischer, Jr., reports the following conflicts of interest (applicable to a 5-year-period). Advisory Board member: Allergan, Amgen, Astellas, Galderma, GSK, Ortho Dermatologics, Stiefel; consultant for: Allergan, Astellas, Asubio, Combe, Galderma, Gerson Lehrman, Intendis, Kikaku America International, Merz, Novartis, Serentis, Taisho; investigator for: 3M, Abbott, Amgen, Astellas, Asubio, BioCryst, Biogen, Centocor, Coria, Dow, Galderma, Genentech, GSK, Intendis, Medicis, Novartis, Ortho Dermatologics, Pfizer, Serentis, Stiefel, Taisho; Speaker Bureau: Amgen, Astellas, Galderma, Intendis, Medicis, Novartis, Stiefel, Upsher-Smith; stockholder: none.