Topical mTOR inhibitors safe, effective in angiofibromas
Topical use of mammalian target of rapamycin (mTOR) inhibitors is effective for angiofibromas linked to tuberous sclerosis complex, with mild to moderate side effects only, according to the results of a recent systematic review.
“This systematic review supports the efficacy of topical sirolimus for angiofibromas linked to tuberous sclerosis complex, with only local side effects reported,” the authors said. “Other indications require further research.”
A literature search was conducted in January 2017 to provide an overview of the topical use of mTOR inhibitors in dermatologic conditions and evaluate their efficacy and safety. All studies that investigated the use of topical mTOR inhibitors in any dermatologic disease were included. The exclusion criteria were systemic use and mucosal administration.
A total of 40 studies comprising 262 patients were included in the analysis. There were 11 dermatologic conditions found, with angiofibromas linked to tuberous sclerosis complex (157 patients) being the most frequent.
Compared with placebos, topical mTOR inhibitors were significantly more effective for angiofibromas (relative risk, 2.52; 95 percent CI, 1.27–5.00; I2, 0 percent). The median concentration of sirolimus was 0.1 percent, and the median treatment duration was 12 weeks.
Topical mTOR inhibitors were found safe, with only mild or moderate local side effects reported. Furthermore, there was no blood level of sirolimus detected in majority of the patients (90 percent).
This systematic review was limited by high heterogeneity in most studies.
“Systemic mTOR inhibitors are currently used in many dermatologic indications. Their topical use is recent and poorly codified,” the authors noted.