Topical rapamycin for angioifbromas in patients with tuberous sclerosis:how does it work in clinical

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Aim: Topical rapamycin for angioifbromas 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 angioifbromas with improvement in both erythema and papules even if only applied every second to third day, but continuous treatment is needed.
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