A Novel Topical Rapamycin Cream for the Treatment of Facial Angiofibromas in Tuberous Sclerosis Complex

Wheless, James W.; Almoazen, Hassan

doi:10.1177/0883073813488664

Cited by 40 publications

(34 citation statements)

References 16 publications

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“…[2][3][4][5][6][7][8][9][10][11][12][13] The articles were limited to those available in the English language and they were read, and any relevant articles referred to in the references were also included in the study. Of these, 11 were case series and one was a review article.…”

Section: Discussionmentioning

confidence: 99%

Topical rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: Follow up of a pilot study and promising future directions

Foster

Bint

et al. 2013

Australasian Journal of Dermatology

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One of the most visible and potentially disfiguring cutaneous manifestations of tuberous sclerosis complex is the development of multiple facial angiofibromas, present in over 80% of patients. Topical rapamycin has been shown in many reports to be a safe and effective treatment for facial angiofibromas. In February 2012 we reported the results of a pilot study of four patients undertaken at a paediatric tertiary hospital in Australia. Since then, we have continued to refine the optimal formulation and concentration of topical rapamycin and expanded our selection of patients. We present an update on our current cohort of treated patients, discuss the optimal formulation of topical rapamycin and include a literature review on all published cases to date. Although topical rapamycin is not a curative treatment, we have demonstrated that its early institution significantly reduces both the vascularity and palpability of angiofibromas and prevents their progression with age. It is well tolerated and now a cost effective option.

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Section: Discussionmentioning

confidence: 99%

Topical rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: Follow up of a pilot study and promising future directions

Foster

Bint

et al. 2013

Australasian Journal of Dermatology

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“…In 2010, Haemel et al [5] reported the first case study of a TSC patient treated with topical sirolimus for facial angiofibromas, which showed a clinically significant improvement. A subsequent group of studies [6][7][8][9][10], although most of them are assessed with relatively small numbers of patients, supported the successful role of topical sirolimus in the dermatological management of TSC patients. A recent publication of a phase 2 randomized clinical trial using topical sirolimus gel on facial angiofibromas in TSC showed consistent results in its efficacy and safety with the previous reports [11].…”

Section: Introductionmentioning

confidence: 99%

Comparative Effects of Topical 0.2% Sirolimus for Angiofibromas in Adults and Pediatric Patients with Tuberous Sclerosis Complex

et al. 2018

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Background: Recent reports have suggested that the topical formulation of sirolimus is effective in treating facial angiofibromas in tuberous sclerosis complex (TSC). Here, we determined the safety and efficacy of 0.2% topical sirolimus for the treatment of facial angiofibroma and compared its effects based on age. Method: This was a retrospective study which involved 36 TSC patients with facial angiofibromas who were treated with 0.2% sirolimus ointment. Its effect was evaluated using the Facial Angiofibroma Severity Index (FASI). In order to observe its comparative effect based on patient age, a subgroup analysis was performed, between the adult group (> 18 years old) and the pediatric group (≤18 years old). Results: The total FASI as well as its subcategories (erythema, size, and extent) showed statistically significant improvements after the topical treatment with 0.2% sirolimus ointment (FASI before treatment: 7.2 ± 1.1, FASI after treatment: 4.4± 1.4, p < 0.001). Among the subcategories of FASI, the erythema was most significantly reduced with the fastest response to the treatment. In a subgroup analysis, the pediatric group showed significantly greater improvements in FASI (improvement of FASI in the pediatric group = 49.7 ± 12.2%, adult group = 27.9 ± 15.6%, p < 0.001). The serial improvement analysis also showed that the pediatric group achieved a consistently greater improvement in FASI at all visits. Its 1-year application in 3 patients demonstrated a continuous maintenance effect. No significant adverse effects were observed. Conclusion: 0.2% sirolimus ointment is safe and effective for facial angiofibromas. Considering its higher efficacy in younger patients, an early initiation of the treatment is recommended.

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“…Any mutation in these genes lead to the dysregulation of this inhibitory process, leading to uncontrolled cell growth, proliferation and protein synthesis that results in multi-organ hamartomas. [2] This disorder has an incidence of 1/6000 births, and mostly affects the skin, kidneys, heart, brain and lungs. [1,3] The cutaneous manifestations of tuberous sclerosis complex include angiofibromas, forehead fibrous plaques, hypomelanotic macules or confetti-like lesions, periungual fibromas and shagreen patches.…”

Section: Discussionmentioning

confidence: 99%

“Tuberous Sclerosis Complex”- With Rare Multisystem Involvements

Petkar¹,

Galani²

2017

jemds

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A Novel Topical Rapamycin Cream for the Treatment of Facial Angiofibromas in Tuberous Sclerosis Complex

Cited by 40 publications

References 16 publications

Topical rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: Follow up of a pilot study and promising future directions

Topical rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: Follow up of a pilot study and promising future directions

Comparative Effects of Topical 0.2% Sirolimus for Angiofibromas in Adults and Pediatric Patients with Tuberous Sclerosis Complex

“Tuberous Sclerosis Complex”- With Rare Multisystem Involvements

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