Topical rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: follow up of a pilot study and promising future directions.

Abstract

One of the most visible and potentially disfiguring cutaneous manifestations of tuberous sclerosis complex is the development of multiple facial angiofibromas, present in over 80% of patients. Topical rapamycin has been shown in many reports to be a safe and effective treatment for facial angiofibromas. In February 2012 we reported the results of a pilot study of four patients undertaken at a paediatric tertiary hospital in Australia. Since then, we have continued to refine the optimal formulation and concentration of topical rapamycin and expanded our selection of patients. We present an update on our current cohort of treated patients, discuss the optimal formulation of topical rapamycin and include a literature review on all published cases to date. Although topical rapamycin is not a curative treatment, we have demonstrated that its early institution significantly reduces both the vascularity and palpability of angiofibromas and prevents their progression with age. It is well tolerated and now a cost effective option.

• Authors: Jenny Tu, Rachael S Foster, Lewis J Bint, Anne R Halbert
• Journal: The Australasian journal of dermatology (Australas J Dermatol) Vol. 55 Issue 1 Pg. 63-9 (Feb 2014) ISSN: 1440-0960 [Electronic] Australia
• PMID: 24359523 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2013 The Australasian College of Dermatologists.

Chemical References

• Antibiotics, Antineoplastic
• Sirolimus

Topics

• Administration, Topical
• Adolescent
• Angiofibroma (drug therapy, etiology)
• Antibiotics, Antineoplastic (administration & dosage)
• Child
• Female
• Follow-Up Studies
• Humans
• Male
• Pilot Projects
• Sirolimus (administration & dosage)
• Tuberous Sclerosis (complications)
• Young Adult