Topical rapamycin for the treatment of facial angiofibromas with tuberous sclerosis complex

Tuberous sclerosis complex (TSC) is a neurocutaneus disease that causes various other tumors including angiofibromas. Although angiofibromas are asymptomatic, they can negatively impact the quality of life. Rapamycine is an mTOR (mammalian target of rapamycin) inhibitor and its oral administration has a beneficial effect on facial angiofibromas. In this patient series, 10 patients with TSC and angiofibromas topically applied rapamycin 1mg/ml solution or rapamycin 0.01% cream once daily. Good results such as decreased erythema and papules were seen in all patients. Side effects of the treatment were mild en were mostly seen at the start of the treatment. The results of our study are similar with previously reported studies. However, future research is necessary to confirm beneficial effects by means of scoring systems, and to investigate the optimal dose, the optimal route of administration, possible longterm side effects and the effect on quality of life.