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Topical 0.2% rapamycin for angiofibromas due to tuberous sclerosis complex (TSC)
( Young In Lee ) , ( Tae Gwang Kwon ) , ( Dae Suk Kim ) , ( Do Young Kim ) , ( Juhee Lee ) , ( Ki Yang Chung ) , ( Min Geol Lee ) , ( Kwang Hoon Lee ) , ( Jung U Shin )
프로그램북 68권 1호 293-293(1pages)
UCI I410-ECN-0102-2017-510-000081623
이 자료는 4페이지 이하의 자료입니다.

Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous syndrome which causes hamartomatous growth in multiple organs. In TSC, the mammalian target of rapamycin(mTOR), a protein kinase that has a diverse functions in protein syntheses, is aberrantly activated, causing an abnormal cellular proliferations. Among the hamartomatous skin lesions, facial angiofibormas occur in 80% of patients. Although angiofibromas are usually asymptomatic, they can significantly affect the quality of life of the patients. Rapamycin is an mTOR inhibitor involved in correcting aberrant signaling pathways regulating cell growth and apoptosis, therefore known to inhibit tumorigenesis activity. Recently, topical formulation of rapamycin has been proposed as an effective option to treat angiofibromas in TSC patients without critical side effects. Over the past 6 months, we have treated a total of 20 patients with topical rapamycin through the dermatology outpatient clinic of Severance Hospital, Yonsei University College of Medicine. Patients had histories of recurrent angiofibromas, and had done several treatments with laser ablation without sufficient improvements. In our study, we found that the topical 0.2% rapamycin ointment was safe and effective for facial angiofibromas with minimal side effects. Nevertheless, few severe adult patients showed limited efficacy with rapamycin treatment alone. Therefore, we suggest that 0.2% topical rapamycin can be considered a safe option for the treatment of facial angiofibromas in Korea, especially for younger patients.

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