Literature DB >> 22301052

Facial angiofibromas treated with topical rapamycin: an excellent choice with fast response.

Teresa Truchuelo1, Blanca Díaz-Ley, Luis Ríos, Javier Alcántara, Pedro Jaén.   

Abstract

Tuberous sclerosis (TS) is the second most common genodermatosis in our country and one of its main characteristics is the presence of facial angiofibromas. These benign tumors can be really bothersome for some patients and there is not a gold-standard treatment. Laser therapy has been used with good responses but it is a painful option and recurrence is guaranteed. TS develops as a result of a mutation of one of two genes, TSC1 or TSC2, which encode for hamartin and tuberin, respectively. TSC1 and TSC2 are tumor suppressors that inhibit mTOR, which if mutated results in mTOR activation, leading to an increase in protein translation. This eventually induces formation of hamartomatous tumors in patients with TSC. Oral rapamycin had been reported to be effective for the treatment of various tumors, apparently because of its action of inhibiting the m-TOR complex. Recently it has been suggested that the drug may be effective when applied topically. We report the 6th case of facial AF treated with topical rapamycin, 1 percent, once per day. An excellent response was achieved surprisingly rapidly. We propose this option as a safe and effective therapy.

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Year:  2012        PMID: 22301052

Source DB:  PubMed          Journal:  Dermatol Online J        ISSN: 1087-2108


  15 in total

Review 1.  Developmental disease and cancer: biological and clinical overlaps.

Authors:  Alfonso Bellacosa
Journal:  Am J Med Genet A       Date:  2013-10-07       Impact factor: 2.802

2.  Correlative imaging of ionic transport and electronic structure in nano Li0.5FePO4 electrodes.

Authors:  Mi Lu; Fuda Yu; Yongfeng Hu; Karim Zaghib; Steen B Schougaard; Zhenbo Wang; Jigang Zhou; Jian Wang; John Goodenough; T K Sham
Journal:  Chem Commun (Camb)       Date:  2019-12-20       Impact factor: 6.222

3.  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

4.  Rapamycin has a beneficial effect on controlling epilepsy in children with tuberous sclerosis complex: results of 7 children from a cohort of 86.

Authors:  Mehmet Canpolat; Huseyin Per; Hakan Gumus; Ali Yikilmaz; Ekrem Unal; Turkan Patiroglu; Levent Cinar; Ali Kurtsoy; Sefer Kumandas
Journal:  Childs Nerv Syst       Date:  2013-06-07       Impact factor: 1.475

Review 5.  Rapamycin and rapalogs for tuberous sclerosis complex.

Authors:  Teguh H Sasongko; Nur Farrah Dila Ismail; Zamh Zabidi-Hussin
Journal:  Cochrane Database Syst Rev       Date:  2016-07-13

6.  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

7.  Topical rapamycin (sirolimus) for facial angiofibromas.

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

Review 8.  Site-specific immunosuppression in vascularized composite allotransplantation: prospects and potential.

Authors:  Jonas T Schnider; Matthias Weinstock; Jan A Plock; Mario G Solari; Raman Venkataramanan; Xin Xiao Zheng; Vijay S Gorantla
Journal:  Clin Dev Immunol       Date:  2013-02-13

Review 9.  Rapamycin and its analogues (rapalogs) for Tuberous Sclerosis Complex-associated tumors: a systematic review on non-randomized studies using meta-analysis.

Authors:  Teguh Haryo Sasongko; Nur Farrah Dila Ismail; Nik Mohamad Ariff Nik Abdul Malik; Z A M H Zabidi-Hussin
Journal:  Orphanet J Rare Dis       Date:  2015-08-12       Impact factor: 4.123

10.  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

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