Literature DB >> 20673154

Successful treatment of angiofibromata of tuberous sclerosis complex with rapamycin.

Elizabeth Kaufman McNamara1, Ashley R Curtis, Alan B Fleischer.   

Abstract

Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous syndrome manifesting as hamartomatous growths in multiple organs. We present two cases of patients with TSC and associated facial angiofibromata treated with topical and oral rapamycin and discuss the role for rapamycin in the treatment of these disfiguring lesions. Our patients demonstrated decreased numbers of angiofibromata and less redness with this treatment. This is the first published report on the use of topical rapamycin for the treatment of angiofibromata.

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Year:  2010        PMID: 20673154     DOI: 10.3109/09546634.2010.489598

Source DB:  PubMed          Journal:  J Dermatolog Treat        ISSN: 0954-6634            Impact factor:   3.359


  11 in total

1.  Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial.

Authors:  Mari Wataya-Kaneda; Yuuki Ohno; Yasuyuki Fujita; Hiroo Yokozeki; Hironori Niizeki; Masaaki Ogai; Kazuyoshi Fukai; Hiroshi Nagai; Yuichi Yoshida; Izumi Hamada; Taihei Hio; Kenji Shimizu; Hiroyuki Murota
Journal:  JAMA Dermatol       Date:  2018-07-01       Impact factor: 10.282

Review 2.  Targeted treatment trials for tuberous sclerosis and autism: no longer a dream.

Authors:  Mustafa Sahin
Journal:  Curr Opin Neurobiol       Date:  2012-05-04       Impact factor: 6.627

3.  Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin.

Authors:  Mary Kay Koenig; Adelaide A Hebert; Joan Roberson; Joshua Samuels; John Slopis; Audrey Woerner; Hope Northrup
Journal:  Drugs R D       Date:  2012-09-01

4.  Topical rapamycin (sirolimus) for facial angiofibromas.

Authors:  Bhushan Madke
Journal:  Indian Dermatol Online J       Date:  2013-01

5.  Facial angiofibromas of tuberous sclerosis treated with topical sirolimus in an Indian patient.

Authors:  Resham J Vasani
Journal:  Indian J Dermatol       Date:  2015 Mar-Apr       Impact factor: 1.494

6.  Successful treatment with the mTOR inhibitor everolimus in a patient with perivascular epithelioid cell tumor.

Authors:  Constantine Gennatas; Vasiliki Michalaki; Paraskevi Vasilatou Kairi; Agathi Kondi-Paphiti; Dionysios Voros
Journal:  World J Surg Oncol       Date:  2012-09-03       Impact factor: 2.754

7.  Fibrous papule of the face, similar to tuberous sclerosis complex-associated angiofibroma, shows activation of the mammalian target of rapamycin pathway: evidence for a novel therapeutic strategy?

Authors:  Jung-Yi Lisa Chan; Kuo-Hsien Wang; Chia-Lang Fang; Wei-Yu Chen
Journal:  PLoS One       Date:  2014-02-18       Impact factor: 3.240

Review 8.  Is mTOR inhibition a systemic treatment for tuberous sclerosis?

Authors:  Romina Moavero; Antonella Coniglio; Francesco Garaci; Paolo Curatolo
Journal:  Ital J Pediatr       Date:  2013-09-17       Impact factor: 2.638

9.  Topical rapamycin as a treatment for fibrofolliculomas in Birt-Hogg-Dubé syndrome: a double-blind placebo-controlled randomized split-face trial.

Authors:  Lieke M C Gijezen; Marigje Vernooij; Herm Martens; Charlene E U Oduber; Charles J M Henquet; Theo M Starink; Martin H Prins; Fred H Menko; Patty J Nelemans; Maurice A M van Steensel
Journal:  PLoS One       Date:  2014-06-09       Impact factor: 3.240

10.  Comparison of the In Vitro and Ex Vivo Permeation of Existing Topical Formulations Used in the Treatment of Facial Angiofibroma and Characterization of the Variations Observed.

Authors:  Guillaume Le Guyader; Bernard Do; Victoire Vieillard; Karine Andrieux; Muriel Paul
Journal:  Pharmaceutics       Date:  2020-11-07       Impact factor: 6.321

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