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
Nausicaa MalissenDidier Bessis

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 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. 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 prot...Continue Reading

Citations

Aug 29, 2018·Pediatric Dermatology·Michael A Cardis, Cynthia M C DeKlotz
Sep 27, 2018·American Journal of Medical Genetics. Part C, Seminars in Medical Genetics·Angela PeronAglaia Vignoli
Jul 25, 2019·JAMA Dermatology·Qiong WuDirk M Elston
Jan 14, 2020·Pediatric Blood & Cancer·Priscila BadiaAdrienne Hammill
Apr 17, 2020·European Journal of Hospital Pharmacy. Science and Practice·Guillaume Le GuyaderMuriel Paul
May 26, 2018·JAMA Dermatology·Thomas N Darling
May 13, 2020·Journal of the European Academy of Dermatology and Venereology : JEADV·R Happle, A Torrelo
Aug 31, 2019·Dermatologic Clinics·Benjamin Becker, Roy E Strowd
Oct 9, 2018·Journal of the American Academy of Dermatology·Pablo García-MonteroÁngel Vera-Casaño
Mar 29, 2021·The Journal of Investigative Dermatology·Xiong CaiJoel Moss
Jun 2, 2021·Pediatric Dermatology·Lars BrichtaCynthia Marie Carver DeKlotz
Jul 3, 2021·International Journal of Molecular Sciences·Masashi MizuguchiAtsushi Sato
Aug 17, 2021·Pediatric Neurology·Hope NorthrupUNKNOWN International Tuberous Sclerosis Complex Consensus Group

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