Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: A prospective study of 25 patients - Université de Montpellier Accéder directement au contenu
Article Dans Une Revue Journal of The American Academy of Dermatology Année : 2017

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

Résumé

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.
Fichier non déposé

Dates et versions

hal-01983965 , version 1 (16-01-2019)

Identifiants

Citer

Nausicaa Malissen, Laurence Vergely, Marguerite Simon, Agathe Roubertie, Marie-Claire Malinge, et al.. Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: A prospective study of 25 patients. Journal of The American Academy of Dermatology, 2017, 77 (3), pp.464-472.e3. ⟨10.1016/j.jaad.2017.04.005⟩. ⟨hal-01983965⟩
48 Consultations
0 Téléchargements

Altmetric

Partager

Gmail Mastodon Facebook X LinkedIn More