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Aim: Topical rapamycin for angiofibromas has been reported to be a new promising treatment. This study aims to report the outcome in clinical practice. Methods: A retrospective clinical follow-up on twenty-three patients who had been prescribed an oral solution of 0.1% rapamycin, to be applied on facial lesions once a day. Results: Seventeen of 23 patients continued the treatment. Papules and nodules were improved in 8 patients (47%) and erythema in 12 (70%). Side effects, such as stinging and redness were reported in 35% of patients. Blood samples were taken from 5 patients and no rapamycin could be detected. All patients who paused the treatment relapsed. Conclusion: Topical rapamycin has a positive effect on angiofibromas with improvement in both erythema and papules even if only applied every second to third day, but continuous treatment is needed.

Topical rapamycin for angiofibromas in patients with tuberous sclerosis: how does it work in clinical practice?