title: Topical sirolimus for the treatment of angiofibromas in tuberous sclerosis.
abstract: BACKGROUND
  The skin is one of the most affected organs in tuberous sclerosis complex and angiofibromas are seen in almost 80% of such patients. These benign tumors impose a great psycho-social burden on patients.
  
  
  OBJECTIVE
  The aim of the study was to evaluate the effectiveness and tolerability of topical sirolimus for facial angiofibromas in patients with tuberous sclerosis complex.
  
  
  METHODS
  This was a prospective, single-blinded, cross-over study which involved twelve patients. We investigated the effect and safety of topical 0.1% sirolimus, which was obtained by crushing sirolimus tablets and mixing it with petrolatum. The patients were asked to apply the cream to one side of their face, and vaseline to the other side. The effect of topical sirolimus was evaluated using the "facial angiofibroma severity index."
  
  
  RESULTS
  There was a significant improvement in the redness and extension of the tumors on the sides to which the active ingredient was applied. Some side effects such as itching and irritation occurred in three patients, which were treated with topical hydrocortisone cream.
  
  
  CONCLUSIONS
  Topical sirolimus appears to be a promising, fairly well tolerated treatment for facial angiofibromas in patients with tuberous sclerosis complex. Although its efficacy diminishes with time, repetitive usage is effective.
authors.affiliation: Department of Dermatology and Venereology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Salih Levent
authors.initials: SL
authors.lastname: Cinar
authors.affiliation: Department of Dermatology and Venereology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Demet
authors.initials: D
authors.lastname: Kartal
authors.affiliation: Department of Pediatric Neurology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Ayşe Kacar
authors.initials: AK
authors.lastname: Bayram
authors.affiliation: Department of Pediatric Neurology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Mehmet
authors.initials: M
authors.lastname: Canpolat
authors.affiliation: Department of Dermatology and Venereology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Murat
authors.initials: M
authors.lastname: Borlu
authors.affiliation: Department of Dermatology and Venereology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Ayten
authors.initials: A
authors.lastname: Ferahbas
authors.affiliation: Department of Pediatric Neurology, Erciyes University Faculty of Medicine, Kayseri, Turkey.
authors.forename: Hüseyin
authors.initials: H
authors.lastname: Per
mesh_terms: D000287:Administration, Topical; D018322:Angiofibroma / Q000175:diagnosis* / Q000188:drug therapy*; D000903:Antibiotics, Antineoplastic / Q000008:administration & dosage*; D002648:Child; D018592:Cross-Over Studies; D005260:Female; D006801:Humans; D008297:Male; D011446:Prospective Studies; D016037:Single-Blind Method; D020123:Sirolimus / Q000008:administration & dosage*; D016896:Treatment Outcome; D014402:Tuberous Sclerosis / Q000175:diagnosis* / Q000188:drug therapy*
chemical_list: D000903:Antibiotics, Antineoplastic; D020123:Sirolimus