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A new treatment regime for facial angiofibromas in patients with tuberous sclerosis complex
Publication year - 2020
Publication title -
british journal of dermatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.304
H-Index - 179
eISSN - 1365-2133
pISSN - 0007-0963
DOI - 10.1111/bjd.19438
Subject(s) - tuberous sclerosis , medicine , dermatology , distress , citation , psychiatry , computer science , clinical psychology , world wide web
Tuberous sclerosis complex (TSC) is a genetic disorder affecting approximately one in every 6,000 to 10,000 people. The disease causes hamartomas, which are non‐cancerous growths, to develop in many parts of the body. Facial growths that are composed of blood vessels and fibrous tissues, called facial angiofibromas (FAs), occur in up to 80% of TSC cases and cause significant distress to the patients. This study, from Taiwan, aimed to find out if a new treatment regime using two topical (applied to the skin) medicines, rapamycin and calcitriol, in combination would be an effective and safe treatment for TSC‐related FAs. We enrolled 52 TSC patients with FAs who were assigned to apply either topical rapamycin (0.1%) or calcitriol (0.0003%) single‐agent therapy (on its own) to one side of the face, and the two medicines as a combined treatment regime to the other side of the face, for 12 weeks. The patients were reassigned to use the ointment which worked best for another 12 weeks, and the treatment was then discontinued for 12 weeks. We evaluated the improvement of FAs by comparing the modified Facial Angiofibroma Severity Index (mFASI; score 0‐10), which was calculated by the sum of erythema, meaning redness (0‐3), papule (a raised area of skin tissue) size (0‐3), papule elevation (0‐3), and extent of lesions (0‐1), at weeks 12 (used as the primary outcome), 24, and 36 from baseline (the start of the trial). We found that the mean reduction of mFASI at week 12 compared to baseline was ‐0.92 and ‐1.09 for rapamycin and rapamycin‐calcitriol combination, respectively. Compared to rapamycin alone, extended use of the rapamycin/calcitriol combination regimen for 24 weeks showed more effectiveness in decreasing papule elevation and could maintain a longer improvement after stopping treatment. We concluded that topical rapamycin/calcitriol combination therapy is an effective and safe regimen for TSC‐related FAs. Linked Article: Chen et al . Br J Dermatol 2020; 183:655–663.