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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
Author(s) -
WatayaKaneda M.,
Tanaka M.,
Nakamura A.,
Matsumoto S.,
Katayama I.
Publication year - 2011
Publication title -
british journal of dermatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.304
H-Index - 179
eISSN - 1365-2133
pISSN - 0007-0963
DOI - 10.1111/j.1365-2133.2011.10471.x
Subject(s) - tuberous sclerosis , tacrolimus , medicine , sirolimus , angiofibroma , calcineurin , adverse effect , dermatology , pharmacology , gastroenterology , surgery , pathology , transplantation
Summary 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.