Retrospective study: Rapamycin or rapalog 0.1% cream for facial angiofibromas in Tuberous Sclerosis Complex: Evaluation of treatment effectiveness and cost

Abstract

The use of topical rapamycin or rapalog, an mTOR inhibitor, has shown promising results in treating facial angiofibromas (FA) in children with Tuberous Sclerosis Complex (TSC). Nevertheless, several issues remain: the effectiveness, stability and tolerability of ingredients; and the cost - the higher the concentration of rapamycin the more expensive the product. In this retrospective comparative study, children with TSC and FA were treated consecutively with three different topical rapalog compounds of 0.1%, the lowest concentration with crushed tablets to demonstrate an effect.

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