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

The Australasian Journal of Dermatology
Jenny TuAnne R Halbert

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.

References

Aug 30, 2008·Lancet·Paolo CuratoloSergiusz Jozwiak
Jul 21, 2010·Archives of Dermatology·Anna K HaemelJoyce M Teng
Aug 3, 2010·The Journal of Dermatological Treatment·Elizabeth Kaufman McNamaraAlan B Fleischer
Aug 13, 2011·Journal of the European Academy of Dermatology and Venereology : JEADV·R SalidoJ C Moreno-Giménez
Feb 9, 2012·The Australasian Journal of Dermatology·Rachael S FosterAnne R Halbert
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May 18, 2013·Journal of Child Neurology·James W Wheless, Hassan Almoazen

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Citations

Mar 10, 2016·Indian Journal of Dermatology·Vishalakshi ViswanathPoonam Pund
Mar 18, 2015·Pediatric Dermatology·Stephanie K Jacks, Patricia M Witman
Feb 24, 2016·Clinics in Dermatology·Karen A Chernoff, Julie V Schaffer
Sep 2, 2014·Journal of the European Academy of Dermatology and Venereology : JEADV·R BalestriM Magnano
Jun 5, 2016·International Journal of Pharmaceutics·Guillaume BouguéonSandy Vrignaud
Dec 23, 2017·International Scholarly Research Notices·S AminF O'Callaghan
Mar 30, 2017·Archives of Disease in Childhood·Michael A Cardis, Cynthia Marie Carver DeKlotz
Apr 17, 2020·European Journal of Hospital Pharmacy. Science and Practice·Guillaume Le GuyaderMuriel Paul
Mar 20, 2015·Pediatric Dermatology·Emma V PynnJenny Hughes
Apr 7, 2021·The Journal of Dermatological Treatment·Clara Cortell FusterMónica Climente Martí
Feb 13, 2019·Journal of the American Academy of Dermatology·Sophie LeducqAnnabel Maruani
Nov 14, 2020·Journal of Nanoscience and Nanotechnology·Xiaojun XiaoHui Luo

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