Conflict of interest The authors have no conflicts of interest to declare.
Sustained clinical effectiveness and favorable safety profile of topical sirolimus for tuberous sclerosis – associated facial angiofibroma
Article first published online: 11 AUG 2011
© 2011 The Authors. Journal of the European Academy of Dermatology and Venereology © 2011 European Academy of Dermatology and Venereology
Journal of the European Academy of Dermatology and Venereology
Volume 26, Issue 10, pages 1315–1318, October 2012
How to Cite
Salido, R., Garnacho-Saucedo, G., Cuevas-Asencio, I., Ruano, J., Galán-Gutierrez, M., Vélez, A. and Moreno-Giménez, J.C. (2012), Sustained clinical effectiveness and favorable safety profile of topical sirolimus for tuberous sclerosis – associated facial angiofibroma. Journal of the European Academy of Dermatology and Venereology, 26: 1315–1318. doi: 10.1111/j.1468-3083.2011.04212.x
Funding sources None.
- Issue published online: 10 SEP 2012
- Article first published online: 11 AUG 2011
- Received: 17 February 2011; Accepted: 18 July 2011
Background Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous disorder characterized by the development of multisystem hamartomatous tumours. Facial angiofibroma appears in up to 80% of patients and has a considerable psychological impact. Various invasive procedures have been used, although they show limited effectiveness and potential adverse effects.
Objectives To evaluate the sustained clinical benefits and safety profile of topical sirolimus applied to treat facial angiofibromas.
Methods This study was a non-blinded, uncontrolled case-series comprising 10 patients with TSC-associated facial angiofibroma that was treated with 0.4% sirolimus ointment 3 times a week for 9 months. Patients were clinically evaluated at baseline and at 6, 12, 24 and 36 weeks. Plasma levels of sirolimus were determined.
Results A sustained improvement was observed in erythema and in the size and extension of the lesions as early as the first weeks of treatment. Sirolimus plasma levels remained below detection limits (0.3 ng/mL) in all cases. The formula was well-tolerated with no local or systemic adverse effects.
Conclusions Topical sirolimus seems to be an effective and safe medical alternative to surgery or laser-based treatments in patients with TSC-associated facial angiofibromas.