Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: A prospective study of 25 patients.
J Am Acad Dermatol
; 77(3): 464-472.e3, 2017 Sep.
Article
en En
| MEDLINE
| ID: mdl-28502378
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.Palabras clave
Texto completo:
1
Base de datos:
MEDLINE
Asunto principal:
Enfermedades de la Piel
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Esclerosis Tuberosa
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Sirolimus
Tipo de estudio:
Guideline
/
Observational_studies
/
Risk_factors_studies
Idioma:
En
Revista:
J Am Acad Dermatol
Año:
2017
Tipo del documento:
Article