Effect of Topical Rapamycin 1% on Multiple Trichoepitheliomas

Dreyfus, I.; Onnis, G L; Tournier, É.; Dereure, O.; Mazereeuw‐Hautier, J.

doi:10.2340/00015555-3116

Cited by 3 publications

(7 citation statements)

References 9 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Adverse effects from treatment with topical sirolimus included mild skin irritation and headache. 24 Conversely, two articles reported no adverse effects. 13,25 Treatment with imiquimod cream was associated with either no adverse effects 26 or local skin irritation, 15 mild local skin reactions (itching, erythema, scaling), moderate local skin reactions (intense erythema, crusting, vesicles, erosion) and systemic reactions (nausea, headache, flu-like symptoms).…”

Section: Resultsmentioning

confidence: 99%

“…Adverse effects associated with pharmacological therapies for multiple trichoepithelioma were reported in seven of the 10 included articles. Adverse effects from treatment with topical sirolimus included mild skin irritation and headache 24 . Conversely, two articles reported no adverse effects 13,25 .…”

Section: Resultsmentioning

confidence: 99%

“…13 The results presented by Dreyfus et al suggest that topical sirolimus may reduce the density of trichoepitheliomas as well as the occurrence of new tumours. 24 In this study, five patients with multiple familial trichoepithelioma were treated twice daily with topical rapamycin 1% cream. Efficacy and tolerance were evaluated using a visual analogical scale from zero to 10.…”

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

“…24 Furthermore, there may be other signalling pathways, such as the Hedgehog, that play a more pertinent role in the pathogenesis of trichoepitheliomas. 24,29 Measures to optimise the efficacy of topical sirolimus may include occlusion, modifications to the formulation to increase the absorption and bioavailability of the drug, or the use of concomitant therapies including prior ablative laser to help reduce the thickness of lesions. 13,24 Imiquimod Imiquimod, an immunomodulating agent, is approved as a topical treatment for human papillomavirus infection, actinic keratosis and basal cell carcinoma.…”

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

“…24,29 Measures to optimise the efficacy of topical sirolimus may include occlusion, modifications to the formulation to increase the absorption and bioavailability of the drug, or the use of concomitant therapies including prior ablative laser to help reduce the thickness of lesions. 13,24 Imiquimod Imiquimod, an immunomodulating agent, is approved as a topical treatment for human papillomavirus infection, actinic keratosis and basal cell carcinoma. Imiquimod stimulates the secretion of tumour necrosis factor-alpha and interferon-gamma via activation of Toll-like receptor 7, resulting in the development of a Th1 lymphocyte-mediated immune response in the areas where the drug is applied.…”

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

See 4 more Smart Citations

Management of multiple trichoepithelioma: A review of pharmacological therapies

2021

View full text Add to dashboard Cite

Trichoepithelioma is a rare benign adnexal neoplasm that can occur in various forms including solitary, multiple, familial or nonfamilial. Multiple facial trichoepithelioma can be associated with significant psychosocial burden. Conventional treatment modalities such as surgical excision and ablative laser have variable results and can be associated with unacceptable complications and tumour regrowth. Pharmacological interventions such as topical and systemic agents are potentially effective but clinical data are limited and treatments are poorly standardised. We review the available evidence to determine the role of pharmacological therapies in the management of multiple trichoepithelioma. Demographic and clinical data were retrospectively collected from the available English literature. Majority of cases treated with pharmacological therapies (93.75%) had a positive treatment outcome, achieving partial lesion response. Adverse effects associated with pharmacological therapies were generally well tolerated and did not interrupt treatment. There are limitations as to how our results can be interpreted owing to the paucity of good quality evidence, spectrum of disease severity, and diversity of study designs utilised in the included articles. Nonetheless, the results of our study indicate that while most pharmacological interventions for multiple trichoepithelioma produce a partial response, they can be employed as effective suppressive therapies, either alone or in conjunction with conventional treatments. The current evidence for pharmacological therapies remains largely anecdotal justifying the need for further clinical studies in this area.

show abstract

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

Section: Sirolimus (Also Known As Rapamycin)mentioning

confidence: 99%

See 3 more Smart Citations

Management of multiple trichoepithelioma: A review of pharmacological therapies

2021

View full text Add to dashboard Cite

show abstract

Topical sirolimus in dermatology: a systematic review

Afzal,

Alazemi,

Ali

et al. 2024

Clinical and Experimental Dermatology

View full text Add to dashboard Cite

Context Topical sirolimus is increasingly utilised off-license to manage various dermatological conditions whilst avoiding typical adverse effects associated with systemic sirolimus. However, widespread use is limited by a highly heterogeneous evidence base of mixed quality. Objective to evaluate the current evidence base for the indications, efficacy and safety profile for topical sirolimus in dermatology. Data sources A literature search was conducted from 2005 to July 4th, 2023, of English language studies, with the following databases consulted: MEDLINE, PubMed, Embase, CENTRAL and EBSCO. Key words included ‘topical’, ‘rapamycin’, ‘sirolimus’ and ‘dermatology’. Data extraction Data on drug efficacy, concentration, side effects, co-interventions and follow up were extracted. Results The search identified 202 studies; 71 studies met the inclusion criteria. Efficacy of topical sirolimus was demonstrated in facial angiofibromas (799 patients) compared to placebo across multiple randomised controlled trials with a predominant concentration of 0.1%. Evidence was mixed for sirolimus use in port-wine stains (61 patients), with evidence of effectiveness in combined sirolimus and pulsed-dye laser. Multiple case reports demonstrated clinical improvement with topical sirolimus use in cutaneous vascular abnormalities (33 patients) at a higher concentration of 1%. Other applications of topical sirolimus were predominantly case reports demonstrating generally favourable outcomes. Topical sirolimus was generally well tolerated – most reported adverse effects were localised irritation and pruritus. Ointment-based preparations and once-daily dosing appeared to confer a better side effect profile. Conclusion Most high-quality data pertain to the efficacy of topical sirolimus in treating facial angiofibromas in tuberous sclerosis. Outcomes are generally promising in other indications and good tolerability, but data quality is mixed.

show abstract

Systematic review of sirolimus in dermatological conditions

2021

View full text Add to dashboard Cite

Sirolimus is a mammalian target of rapamycin inhibitor (mTORI) with anti-proliferative, antiangiogenic and immunosuppressive properties. While approved in Australia as an anti-rejection medication for renal transplant patients, there is mounting evidence regarding the utility of oral and topical sirolimus in treating a plethora of dermatological conditions or conditions with cutaneous manifestations. Our aim was to present an overview of the evidence for current usage and breadth of the application of sirolimus in dermatology. We carried out a systematic review of all the literature published up to 31 August 2019 on oral and topical sirolimus with respect to dermatological conditions or conditions otherwise relevant to dermatology. While 3368 papers were initially produced in our search, 238 papers met our inclusion criteria and were examined in our review. The conditions examined were categorised into genodermatoses (9 conditions), infection (1 condition), inflammatory/autoimmune (10 conditions), neoplasm (3 conditions) and vascular (17 conditions). We extracted data on first author, publication year, journal, characteristics of the study and study patients, condition, drug modalities, drug efficacy, side effects, blood level of mTORI, co-interventions and follow-up. While there is level 1 evidence for the efficacy of sirolimus in conditions such as tuberous sclerosis complex (TSC) and GVHD prophylaxis, for many other conditions, the evidence is limited to level 4 evidence. Regarding oral systemic therapy, dosing regimens varied with the most common for children 0.8mg/m 2 twice daily and for adults 1 mg twice daily. Doses were often adjusted to reach a typical trough level of between 5 and 15 ng/mL, though targets often varied. In the overall majority of cases, side effects were minimal or tolerable, including mucositis, cytopenias, lipid abnormalities and nausea/vomiting, and only a few cases had to stop due to adverse effects. Regarding topical therapy, concentration of formulations varied from 0.1% to 1% and were compounded into creams, ointments or gels and administered typically once or twice per day. The most common side effect was skin irritation. There were a number of limitations to our study. In particular, many of the published studies were case reports or case series with no comparator arm, leading to susceptibility of bias in conclusions drawn, in particular a high likelihood of publication bias. Given the heterogeneity amongst studies, comparisons or aggregation of results was difficult. There continues to be growing use of oral and topical sirolimus in dermatological conditions. It provides new therapeutic options to patients where previous therapies have either failed or are limited due to toxicity. However, further studies are warranted.

show abstract

Effect of Topical Rapamycin 1% on Multiple Trichoepitheliomas

Cited by 3 publications

References 9 publications

Management of multiple trichoepithelioma: A review of pharmacological therapies

Management of multiple trichoepithelioma: A review of pharmacological therapies

Topical sirolimus in dermatology: a systematic review

Systematic review of sirolimus in dermatological conditions

Contact Info

Product

Resources

About