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

Tu, Jenny; Foster, Rachael S; Bint, Lewis; Halbert, Anne

doi:10.1111/ajd.12125

Cited by 47 publications

(78 citation statements)

References 15 publications

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“…The first sign is reappearance of erythema and then the growth of FA. This fact has already been reported (3,5,7,11,14). Nevertheless, our four cases were highly or very highly satisfied with this topical product.…”

Section: © C I C E D I Z I O N I I N T E R N a Z I O N A L Isupporting

confidence: 84%

“…The effectiveness of topical rapamycin in FA has been widely reported (2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14). A review of existing evidence is Table 3.…”

Section: Discussionmentioning

confidence: 99%

“…2-3 applications per week for 9-12 months) (4,5,14). Also, lower concentrations of rapamycin [0,4% (4), 0,2% (5, 11), 0,03% (10)] are obtaining good results with longer application.…”

Section: © C I C E D I Z I O N I I N T E R N a Z I O N A L Imentioning

confidence: 99%

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Topical rapamycin in facial angiofibromas: patients satisfaction versus objective response, and a literature review

Rodrigo-Nicols¹

2014

CDERM

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SummaryFacial Angiofibromas (FA) are a common cutaneous manifestation of Tuberous Sclerosis (TS). Different therapies have been employed until now (surgery, CO2 laser…) without successful and permanent effects. Recently, topical rapamycin has been reported as a new and effective treatment without significant adverse effects. We present our experience in four adult patients with FA and TS who have been treated with 1% rapamycin ointment for three months. We evaluated the objective response achieved by using the Facial Angiofibromas Severity Index (FASI). On the other hand, in order to know the patients' subjective satisfaction they completed a questionnaire to evaluate this therapy. We noticed a slight reduction of erythema, size and number of FA, lower than published in the literature. Despite this, overall satisfaction was high or very high even if the FA reappeared months after discontinuing the application. We wonder if this subjective response justifies the costs of this treatment nowadays, especially in adult patients in which the established fibrosis in the FA could decrease the efficacy of the ointment. We also present a review of all published cases to date.

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Section: © C I C E D I Z I O N I I N T E R N a Z I O N A L Isupporting

confidence: 84%

“…The effectiveness of topical rapamycin in FA has been widely reported (2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14). A review of existing evidence is Table 3.…”

Section: Discussionmentioning

confidence: 99%

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Topical rapamycin in facial angiofibromas: patients satisfaction versus objective response, and a literature review

Rodrigo-Nicols¹

2014

CDERM

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“…The result was maintained for the next 48 weeks with the continuous use of topical sirolimus. Therefore, we suggest to start the topical treatment at an earlier age in order to achieve its best results, as suggested for the systemic therapies in other TSC-related tumors as well [23].…”

Section: Discussionmentioning

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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“…In fact, 13 patients required a dose augmentation (0.1%-0.5 or 1% formulations). 15 All topical rapamycin formulations nevertheless have a good level of safety regardless of the concentration values. One SAE (ab ingestis pneumonia) has been reported, but, since the blood level of rapamycin was undetectable in this patient, the relationship with the topical treatment was excluded.…”

Section: Discussionmentioning

confidence: 99%

Analysis of current data on the use of topical rapamycin in the treatment of facial angiofibromas in Tuberous Sclerosis Complex

Balestri

Neri

Patrizi

et al. 2014

Acad Dermatol Venereol

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Topical rapamycin can be considered a safe option for the treatment and the prevention of facial angiofibromas in younger patients, but the best formulation has not been established. Our review demonstrates that ointment and gel should be preferred, but it is not clear which concentration is optimal. Long-term and comparative studies between topical rapamycin and ablative techniques are required to establish which treatment has a better outcome and lower recurrence rate.

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

Cited by 47 publications

References 15 publications

Topical rapamycin in facial angiofibromas: patients satisfaction versus objective response, and a literature review

Topical rapamycin in facial angiofibromas: patients satisfaction versus objective response, and a literature review

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

Analysis of current data on the use of topical rapamycin in the treatment of facial angiofibromas in Tuberous Sclerosis Complex

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