A Novel Application of Topical Rapamycin Formulation, an Inhibitor of mTOR, for Patients With Hypomelanotic Macules in Tuberous Sclerosis Complex

Wataya‐Kaneda, Mari

doi:10.1001/archderm.148.1.138

Cited by 44 publications

(37 citation statements)

References 6 publications

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“…Gel formulation was proposed by 2 studies at 0.2% concentration. 9,11 In one of these, a comparative in vitro percutaneous absorption study between 0.2% rapamycin ointment and 0.2% gel was performed to evaluate the best skin penetration. The results showed that the 0.2% gel had a greater skin penetration than the ointment without being irritant like the solution.…”

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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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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“…Rapamycin may alter factors implicated in melanogenic gene expression, explaining its effectiveness in treating hypomelanotic macules. 14 We did not pursue the use of topical rapamycin for hypomelanotic macules as they are mostly not located on cosmetically sensitive sites.…”

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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“…Recent case reports and small case series have demonstrated that a topical rapamycin formulation might be efficacious, [18][19][20][21][22][23][24][25][26][27] but prior reports have consisted of small series without placebo arms. The primary goal of this study was to determine whether our topical formulation of rapamycin was safe for topical use in the treatment of facial angiofibromas in patients with TSC.…”

Section: Discussionmentioning

confidence: 99%

Topical Rapamycin Therapy to Alleviate the Cutaneous Manifestations of Tuberous Sclerosis Complex

Koenig

Hebert

Roberson³

et al. 2012

Drugs R D

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Background and Objectives: Facial angiofibromas are disfiguring facial lesions, present in up to 80% of patients with tuberous sclerosis complex. Recent elucidation of the complex cell signaling pathways that are disrupted in tuberous sclerosis indicates that rapamycin may be successful in alleviating the appearance of these lesions. The objectives of the current study were to evaluate the safety of topically applied rapamycin in patients with tuberous sclerosis complex and to determine its potential effectiveness in treatment of facial angiofibromas.Patients and Methods: The study was a prospective, randomized, doubleblind, placebo-controlled study performed at the University of Texas Health Science Center at Houston. Study subjects were recruited from the patient populations at the University of Texas Tuberous Sclerosis Center of Excellence. All subjects were over the age of 13 years and had a diagnosis of

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A Novel Application of Topical Rapamycin Formulation, an Inhibitor of mTOR, for Patients With Hypomelanotic Macules in Tuberous Sclerosis Complex

Cited by 44 publications

References 6 publications

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

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

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

Topical Rapamycin Therapy to Alleviate the Cutaneous Manifestations of Tuberous Sclerosis Complex

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