Combined Treatment of Disfiguring Facial Angiofibromas in Tuberous Sclerosis Complex With Surgical Debulking and Topical Sirolimus

Patterson, Jessica L.; Iyengar, Sanjana; Catasus, Carson; Kolodney, Joanna; Zinn, Zachary

doi:10.12788/cutis.0137

Cited by 3 publications

(5 citation statements)

References 6 publications

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“…In one case the systemic rapamycin was added after 3 months of topical therapy 2 . The remaining 7 cases received rapamycin in combination with a physical therapy: 6 laser ablation 12,16,31,39 and 1 cold knife surgery 37 …”

Section: Resultsmentioning

confidence: 99%

“…Seven studies combined topical rapamycin with other therapies, for a total of 67 patients (Table 1). 7,12,16,31,35,37,39 …”

Section: Resultsmentioning

confidence: 99%

“…Seven studies combined topical rapamycin with other therapies, for a total of 67 patients (Table 1). 7,12,16,31,35,37,39 Most of them (50 patients) received a rapamycin-calcitriol combination ointment, developed by mixing 1 g of rapamycin powder and 1 g of calcitriol 03% ointment with 998 g of petrolatum. 35 In 9 patients, rapamycin was dispersed in tacrolimus ointment.…”

Section: Combined Therapymentioning

confidence: 99%

“…2 The remaining 7 cases received rapamycin in combination with a physical therapy: 6 laser ablation 12,16,31,39 and 1 cold knife surgery. 37…”

Section: Combined Therapymentioning

confidence: 99%

“…The use of topical mammalian target of rapamycin (mTOR) inhibitors in the treatment of facial angiofibromas (FA) in Tuberous sclerosis complex (TSC) is a topic of growing interest following the first report published in 2011, 1 and numerous articles and studies have since been published 2–39 …”

Section: Introductionmentioning

confidence: 99%

See 4 more Smart Citations

Analysis of current data on the use of topical mTOR inhibitors in the treatment of facial angiofibromas in tuberous sclerosis complex—An update

Balestri¹,

Rizzoli²,

Pedrolli³

et al. 2022

Acad Dermatol Venereol

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Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous syndrome causing hamartomatous growths in multiple organs. Facial angiofibromas occur in up to 80% of patients and can be highly disfiguring. Treatment for these lesions is challenging. Recently, topical rapamycin has been proposed as an effective option to treat angiofibromas but a commercially available compound has not yet been developed in Europe. We conducted a retrospective review with the aim to update the current data on the use of topical rapamycin in the treatment of angiofibromas in TSC, focusing on the optimal concentration and trying to establish which vehicle should be preferred. Thirty‐nine reports describing the use of topical rapamycin in the treatment of angiofibromas in TSC were considered, involving a total of 483 patients. An improvement of the lesions has been shown in over 90% of subjects, particularly if the treatment was started at early stages. Several different formulations (ointment, gel, solution and cream) with a wide range of concentrations (0.003%–1%) were proposed, of which a pharmacological analysis has also been performed. Topical rapamycin can be considered an effective and safe option for the treatment and the prevention of facial angiofibromas in younger patients, but the best formulation has yet to be established. Our review demonstrates that ointment and gel should be preferred, but it is not clear which concentration is optimal. However, according to this study, the 0.1% concentration represents the first choice. Long‐term and comparative studies between topical rapamycin formulations are required in order to establish which treatment has a better outcome and lower recurrence rate.

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Section: Resultsmentioning

confidence: 99%

“…Seven studies combined topical rapamycin with other therapies, for a total of 67 patients (Table 1). 7,12,16,31,35,37,39 …”

Section: Resultsmentioning

confidence: 99%