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Sirolimus Ointment for Facial Angiofibromas in Individuals with Tuberous Sclerosis Complex.

Amin, S; Lux, A; Khan, A; O'Callaghan, F; (2017) Sirolimus Ointment for Facial Angiofibromas in Individuals with Tuberous Sclerosis Complex. Int Sch Res Notices , 2017 , Article 8404378. 10.1155/2017/8404378. Green open access

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Abstract

BACKGROUND: Facial angiofibromas affect most patients with tuberous sclerosis complex. They tend to progress, can cause recurrent bleeding and facial disfigurement, and have significant psychological effects. We reviewed the effectiveness and safety of topical sirolimus ointment 0.1%. We also assessed the effect of treatment on quality of life. METHODS: We report our experience in using sirolimus ointment in 14 patients with TSC (9 children and 5 adults). The impact of sirolimus ointment was monitored with digital photography, dermatological review using a validated Facial Angiofibroma Severity Index (FASI), and quality of life assessments using the questionnaires PedsQL for children and SF36 for adults. RESULTS: The FASI scores were improved in 12/14 cases after six months' treatment, and improvement was more likely in children (median FASI scores of improvement after treatment were 3 points for children and 1 for adults). Proxy-reported PedsQL scores for the total psychosocial domain improved significantly in the children in the cohort with treatment. CONCLUSIONS: Sirolimus ointment 0.1% administered once a day was effective in treating facial angiofibromas. It appears to be safe and well tolerated and to have a positive impact on patients' quality of life. It appeared to be most beneficial when started in childhood.

Type: Article
Title: Sirolimus Ointment for Facial Angiofibromas in Individuals with Tuberous Sclerosis Complex.
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1155/2017/8404378
Publisher version: https://doi.org/10.1155/2017/8404378
Language: English
Additional information: Copyright © 2017 S. Amin et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
UCL classification: UCL > School of Life and Medical Sciences
UCL > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Child Health
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URI: http://discovery.ucl.ac.uk/id/eprint/10041278
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