Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: A prospective study of 25 patients - 12/08/17
Abstract |
Background |
Data on long-term topical sirolimus treatment of the cutaneous manifestations of tuberous sclerosis complex are rare.
Objective |
To evaluate the long-term benefit and tolerance of topical 1% sirolimus in tuberous sclerosis complex.
Methods |
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.
Results |
Twenty-five patients were enrolled. Fifty percent obtained CC of FAs within 9 months. Of 7 patients with CC (58%) who were following the maintenance protocol, 6 relapsed within 7 months and 1 was still responding at 1 year. Of 16 patients with FCPs, 7 (44%) remained stable at 12 months and 9 (56%) improved after 3 to 9 months of treatment. Only 1 of 5 patients treated for shagreen patches showed improvement at 12 months. Treatment was well tolerated with no serious adverse events.
Limitations |
The small number of patients was a limitation.
Conclusions |
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.
Le texte complet de cet article est disponible en PDF.Key words : angiofibromas, fibrous cephalic plaque, hypomelanotic macules, shagreen patch, sirolimus, treatment, tuberous sclerosis complex, ungual fibroma
Abbreviations used : CC, FAs, FASI, FCP, PGA, PR, TSC
Plan
| Funding sources: None. |
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| Conflicts of interest: None declared. |
Vol 77 - N° 3
P. 464 - septembre 2017 Retour au numéro
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