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Palmetun Ekbäck et al. Plast Aesthet Res 2016;3:328-34 Plastic and
DOI: 10.20517/2347-9264.2016.60
Aesthetic Research

www.parjournal.net
Original Article Open Access

Topical rapamycin for angiofibromas in

patients with tuberous sclerosis: how does

it work in clinical practice?

Maria Palmetun Ekbäck , Desiree Wiegleb Edström 3
1,2
1 Department of Dermatology, Örebro University Hospital, 70185 Örebro, Sweden.
2 Department of Pharmacology and Therapeutic, Region Örebro County Council, 70185 Örebro, Sweden.
3 Department of Medicin Solna, Karolinska Institute and Department of Dermatology, Karolinska University Hospital, 17176 Stockholm, Sweden.
Correspondence to: Dr. Maria Palmetun Ekbäck, Department of Dermatology, Örebro University Hospital, 70285 Örebro, Sweden.
E-mail: maria.palmetun-ekback@regionorebrolan.se
How to cite this article: Palmetun Ekbäck M, Wiegleb Edström D. Topical rapamycin for angiofibromas in patients with tuberous sclerosis: how
does it work in clinical practice? Plast Aesthet Res 2016;3:328-34.

ABSTRACT
Article history: Aim: Topical rapamycin for angiofibromas has been reported to be a new promising treatment.
Received: 31-07-2016 This study aims to report the outcome in clinical practice. Methods: A retrospective clinical
Accepted: 13-10-2016 follow-up on twenty-three patients who had been prescribed an oral solution of 0.1% rapamycin,
Published: 25-10-2016 to be applied on facial lesions once a day. Results: Seventeen of 23 patients continued the
treatment. Papules and nodules were improved in 8 patients (47%) and erythema in 12 (70%).
Key words: Side effects, such as stinging and redness were reported in 35% of patients. Blood samples
Tuberous sclerosis, were taken from 5 patients and no rapamycin could be detected. All patients who paused the
topical rapamycin, treatment relapsed. Conclusion: Topical rapamycin has a positive effect on angiofibromas
angiofibroma, with improvement in both erythema and papules even if only applied every second to third day,
clinical practice but continuous treatment is needed.

INTRODUCTION hamartomas in the brain, retina, skin, heart, kidneys,
and lungs.
Tuberous sclerosis complex (TSC) is a dominant
autosomal disorder that affects multiple organ These patients should be evaluated by a dermatologist
systems. The prevalence of the disease is estimated for facial angiofibromas, fibrous cephalic plaques,
[1]
to 1 in 6,000 live births. Spontaneous or inherited hypomelanotic macules, ungual fibromas and shagreen
mutations in the tumor-suppressor genes TSC1 (9q34) patches.
[2]
or TSC2 (16p13) are found in 85% result in activation
of the mammalian target of rapamycin complex 1 Facial angiofibroma is found in 80-90% of cases
(mTORC1) leading to uncontrolled formation of and consist of vascular and fibrotic tissue, leading

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