Facial angiofibromas associated to tuberous sclerosis treated with topical sirolimus

• Published in: Ugeskrift for læger Vol. 175; no. 43; p. 2569
• Main Authors: Tiedemann Svendsen, Mathias, Bygum, Anette, Hansen, Lars Kjærsgaard, Broesby-Olsen, Sigurd
• Format: Journal Article
• Language: Danish
• Published: Denmark 21.10.2013
• Subjects: Angiofibroma - drug therapy; Angiofibroma - etiology; Angiofibroma - pathology; Antibiotics, Antineoplastic - administration & dosage; Antibiotics, Antineoplastic - therapeutic use; Child, Preschool; Facial Neoplasms - drug therapy; Facial Neoplasms - etiology; Facial Neoplasms - pathology; Humans; Immunosuppressive Agents - administration & dosage; Immunosuppressive Agents - therapeutic use; Male; Ointments - therapeutic use; Sirolimus - administration & dosage; Sirolimus - therapeutic use; Treatment Outcome; Tuberous Sclerosis - complications

We present a five-year-old boy with facial angiofibromas associated to tuberous sclerosis successfully treated with topical sirolimus 0.4% applied three times a week for six months. After six months we observed a nearly complete resolution of facial angiofibromas. The blood levels of sirolimus remained under a detectable limit and no side-effects were observed. Topically applied sirolimus seems to be a safe and effective alternative to surgery or laser therapy.