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- Title
The Efficacy of Everolimus for Facial Angiofibromas in Tuberous Sclerosis Complex Patients Treated for Renal Angiomyolipoma/Subependymal Giant Cell Astrocytoma.
- Authors
Wei, Chang-Ching; Hsiao, Yu-Ping; Gau, Shuo-Yan; Wu, Yue-Ting; Wu, Chi-Tang; Wu, Meng-Hsuan; Tsai, Jeng-Dau
- Abstract
<bold>Background: </bold>Facial angiofibromas may be present since early childhood in individuals with tuberous sclerosis complex (TSC), causing substantial cosmetic disfigurement. Current therapies are partially effective, but they are uncomfortable, produce scarring, and are especially expensive.<bold>Objective: </bold>The aim of the present study was to evaluate the efficacy of oral everolimus for TSC-associated angiofibromas.<bold>Methods: </bold>This retrospective study included TSC patients being treated with oral everolimus for subependymal giant cell astrocytomas (SEGAs) and angiomyolipomas (AMLs). We recorded the changes in facial angiofibromas. Changes in the Angiofibroma Grading Scale (AGS) indicators were recorded according to erythema, average lesion size, lesion density, and percent involvement on the forehead, nose, cheeks, and chin. The scores were recorded before and after the administration of oral everolimus.<bold>Results: </bold>Twenty-one patients being treated with oral everolimus were enrolled in this study. The mean age was 20.5 years (range 11-44 years, 4 males, and 17 females). The mean dose of oral everolimus was 3.6 mg/day. Clinically meaningful and statistically significant improvement was observed in erythema (p = 0.001), average lesion size (p < 0.001), lesion density (p < 0.001), and percent involvement (p < 0.001). Changes in the AGS findings were statistically significant on the forehead (p = 0.001), nose (p < 0.001) cheeks (p < 0.001), and chin (p = 0.004).<bold>Conclusion: </bold>Everolimus shows evident improvement and is approved for TSC-associated SEGAs and AMLs. The current study demonstrated the efficacy of oral everolimus in reducing facial angiofibromas, showing the parallel benefits of the treatment protocol for TSC.
- Subjects
TUBEROUS sclerosis; EVEROLIMUS; ANGIOMYOLIPOMA; ASTROCYTOMAS; FACIAL care; THERAPEUTIC use of antineoplastic agents; BLOOD-vessel tumors; ADIPOSE tissue tumors; GLIOMAS; RETROSPECTIVE studies; FACE diseases; KIDNEY tumors; TUMORS; DISEASE complications
- Publication
Dermatology (10188665), 2021, Vol 237, Issue 3, p444
- ISSN
1018-8665
- Publication type
journal article
- DOI
10.1159/000510222