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Updated: May 9, 2026
Murine Full-thickness Skin Transplantation
Published on: January 2, 2017
First left-right comparative study of topical rapamycin vs. vehicle for facial angiofibromas in patients with
M Tanaka1, M Wataya-Kaneda, A Nakamura
1Department of Dermatology, Graduate School of Medicine, Osaka University, 2-2 Yamada-oka, Suita, Osaka, 565-0871, Japan.
Background:
Tuberous sclerosis complex (TSC) is an autosomal dominant disorder causing multiple hamartomas. Treatment of TSC lesions with mammalian target of rapamycin inhibitors is effective. Recently, several reports have shown the efficacy of topical rapamycin (sirolimus) for angiofibromas. However, almost all studies have been case studies and the 0·1% solution caused skin irritation. A comparative study of topical rapamycin and a vehicle has not yet been reported.
Objectives:
To compare the efficacy of topical rapamycin formulation with that of vehicle for angiofibromas.
Methods:
A left-right comparative study between rapamycin 0·2% topical formulation and vehicle was conducted in 11 patients with TSC. Two formulations, an ointment and a gel, were prepared and in vitro percutaneous absorption of rapamycin was determined.
Results:
In vitro percutaneous absorption of rapamycin was significantly greater with the gel compared with the ointment. In the clinical study, the rapamycin-treated cheek showed significant improvements relative to the vehicle-treated cheek in all outcome measures after 12 weeks of treatment. The improvement was particularly remarkable in children aged ≤ 10 years. No side-effects were noted, and rapamycin was not detected in the blood of the patients.
Conclusions:
Topical rapamycin was significantly effective against angiofibromas. Both formulations used were effective and safe. The 0·2% gel is especially useful because of its better skin penetration and low irritancy. Initiation of topical rapamycin therapy in early childhood would be beneficial for patients with TSC.
Insights
Topical rapamycin effectively treats angiofibromas in Tuberous Sclerosis Complex (TSC). The 0.2% gel formulation showed superior skin penetration and safety, especially beneficial when initiated in early childhood.
Area of Science:
- Dermatology
- Genetics
- Pharmacology
Background:
- Tuberous Sclerosis Complex (TSC) is an autosomal dominant disorder characterized by hamartomas.
- Mammalian target of rapamycin (mTOR) inhibitors are effective for TSC lesions.
- Topical rapamycin shows promise for angiofibromas, but comparative studies and irritation potential are concerns.
Purpose of the Study:
- To compare the efficacy and safety of a topical rapamycin formulation versus a vehicle for treating angiofibromas in TSC patients.
- To evaluate the percutaneous absorption of topical rapamycin in different formulations.
Main Methods:
- A left-right comparative study involving 11 TSC patients.
- Evaluation of two topical rapamycin formulations (ointment and gel) at 0.2% concentration.
- In vitro percutaneous absorption studies and clinical assessment of angiofibroma improvement over 12 weeks.
Main Results:
- The rapamycin gel demonstrated significantly greater in vitro percutaneous absorption compared to the ointment.
- Significant clinical improvement in angiofibromas was observed on the rapamycin-treated side versus the vehicle-treated side.
- Treatment was well-tolerated with no detected systemic absorption of rapamycin; notable efficacy in children under 10.
Conclusions:
- Topical rapamycin (0.2% gel) is an effective and safe treatment for angiofibromas associated with TSC.
- The gel formulation offers better skin penetration and lower irritancy.
- Early initiation of topical rapamycin therapy in childhood may provide significant benefits for TSC patients.
