A topical combination of rapamycin and tacrolimus for the treatment of angiofibroma due to tuberous sclerosis complex (TSC): a pilot study of nine Japanese patients with TSC of different disease severity.

Abstract

BACKGROUND:

Dysregulation of mTOR signalling by mutations in tuberin and/or hamartin leads to the formation of tuberous sclerosis complex (TSC). Trials to treat TSC using mTOR inhibitors, including rapamycin, have been performed. Although rapamycin improves many TSC lesions, significant side-effects appear after systemic administration. Topical administration has been recommended.

OBJECTIVES:

The efficacy of rapamycin-tacrolimus ointment was examined for TSC-related angiofibroma.

METHODS:

Left-right comparisons of the tacrolimus ointments with/without 0·2% rapamycin was conducted in symmetrical facial angiofibromas in nine patients with definitive TSC. After the 3-month treatment, a cumulative score for redness, flatness and papule size was used to evaluate the efficacy of the treatment. Blood rapamycin levels were analysed by liquid chromatography-electrospray mass spectrometry (LC-ESI/MS).

RESULTS:

At the end of the treatment, all of the scores significantly improved for rapamycin-tacrolimus treatment compared with tacrolimus alone. No adverse reactions were noted and blood levels of rapamycin were below the detection limit in all cases.

CONCLUSIONS:

Topical application of rapamycin-tacrolimus ointment is a safe and useful treatment for TSC-related angiofibroma.

• Authors: M Wataya-Kaneda, M Tanaka, A Nakamura, S Matsumoto, I Katayama
• Journal: The British journal of dermatology (Br J Dermatol) Vol. 165 Issue 4 Pg. 912-6 (Oct 2011) ISSN: 1365-2133 [Electronic] England
• PMID: 21692771 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2011 The Authors. BJD © 2011 British Association of Dermatologists.

Chemical References

• Drug Combinations
• Immunosuppressive Agents
• Ointments
• Pharmaceutical Vehicles
• Sirolimus
• Tacrolimus

Topics

• Administration, Cutaneous
• Adolescent
• Adult
• Aged
• Aged, 80 and over
• Angiofibroma (drug therapy, etiology)
• Child
• Drug Combinations
• Facial Neoplasms (drug therapy, etiology)
• Female
• Humans
• Immunosuppressive Agents (administration & dosage)
• Male
• Middle Aged
• Ointments
• Pharmaceutical Vehicles (administration & dosage)
• Pilot Projects
• Sirolimus (administration & dosage)
• Tacrolimus (administration & dosage)
• Treatment Outcome
• Tuberous Sclerosis (complications)
• Young Adult