Facial angiofibromas, composed of fibrous tissue and blood vessels appearing on the face,
are closely associated with tuberous sclerosis complex. Historically, oral rapamycin, a
mammalian target of the rapamycin inhibitor of cell proliferation, has been used to treat
visceral tuberous sclerosis–related tumors; however, the side effect profile of this
medicine generally precludes its use in patients lacking significant internal involvement.
The authors developed a novel topical formulation of rapamycin cream to treat the facial
angiofibroma without exposing patients to possible systemic side effects. We followed 11
patients in a long-term, open-label, prospective study to evaluate the safety and
effectiveness of rapamycin cream when used chronically. All of the patients showed an
improvement in the appearance of their facial angiofibroma which was maintained in
long-term follow-up without safety concerns or systemic absorption. The novel rapamycin
cream was tolerated well by all patients and represents a way to address the cutaneous
manifestation of tuberous sclerosis complex.
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