CP-173 Topical 0.1% rapamycin for angiofibromas in a paediatric patient with tuberous sclerosis

Abstract

BackgroundFacial angiofibromas (FA) are the most visible of the cutaneous manifestations of tuberous sclerosis. Current treatments include laser and other invasive techniques. Topical rapamycin is a recent and unauthorised option to treat FA (off-label use) but a commercially available compound has not yet been developed.PurposeTo evaluate the efficacy and safety of a pharmaceutical compound of topical rapamycin in a child with FA.Material and methodsA retrospective review of the literature was conducted to select the vehicle, concentration and posology of the topical formulation. Topical 0.1% rapamycin in petrolatum using the powder from the manufacturer was the pharmaceutical compound selected. This concentration was proposed because it is an effective, efficient and safe therapy in pretreated children. The vehicle selected to prepare this topical preparation was petrolatum because treatment with topical rapamycin solution has reportedly caused local adverse side effects, such as irritation. The treatment was authorised by the hospital management, and the child´s parents were informed and provided informed consent. The authors evaluated efficacy through improvement of lesions and safety was evaluated by adverse effects at 3 months.ResultsA 6-year-old patient with FA was selected for treatment with topical 0.1% rapamycin in petrolatum twice daily to the affected areas on the face. In this patient there was an improvement and clearance of the lesions. No local irritation or serious adverse events were described. Rapamycin blood levels at 3 months were 1.02 ng/mL, far below the therapeutic range (5–15 ng/mL) needed for immunosuppression. The posology was reduced to three times a week instead of daily for maintenance.ConclusionTopical 0.1% rapamycin in petrolatum was an effective treatment for FA in this patient. The preparation formulated in petrolatum was well tolerated with no adverse effects. This pharmaceutical compound could be used as an effective option for treatment of FA in paediatric patients without serious adverse effects. It is necessary to establish how long treatment must be continued.References and/or AcknowledgementsBalestri R, Neri I, Patrizi A, et al. Analysis of current data on the use of topical rapamycin in the treatment of FA. J Eur Acad Dermatol Venereol 2015;29:14-20No conflict of interest.