Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: A prospective study of 25 patients

Abstract

Data on long-term topical sirolimus treatment of the cutaneous manifestations of tuberous sclerosis complex are rare. To evaluate the long-term benefit and tolerance of topical 1% sirolimus in tuberous sclerosis complex. In this 18-month prospective single-center study, 1% sirolimus cream was applied daily to facial angiofibromas (FAs), fibrous cephalic plaques (FCPs), shagreen patches, hypomelanotic macules, and ungual fibromas. After complete clearance (CC) of FAs, we evaluated a maintenance protocol of 3 applications weekly. Twenty-five patients were enrolled. Fifty percent obtained CC of FAs within 9months. Of 7 patients with CC (58%) who were following the maintenance protocol, 6 relapsed within 7months and 1 was still responding at 1year. Of 16 patients with FCPs, 7 (44%) remained stable at 12months and 9 (56%) improved after 3 to 9months of treatment. Only 1 of 5 patients treated for shagreen patches showed improvement at 12months. Treatment was well tolerated with no serious adverse events. The small number of patients was a limitation. Topical 1% sirolimus applied daily produced positive responses in treatment of FAs, FCPs, and facial hypomelanotic macules and was well tolerated. A 3-times-weekly maintenance protocol did not prevent FA relapses.