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西罗莫司成功治疗结节性硬化症相关的血管纤维瘤。

Successful treatment of angiofibromata of tuberous sclerosis complex with rapamycin.

机构信息

Department of Dermatology, Wake Forest University School of Medicine, Winston-Salem, North Carolina 27157-1071, USA.

出版信息

J Dermatolog Treat. 2012 Feb;23(1):46-8. doi: 10.3109/09546634.2010.489598. Epub 2010 Aug 1.

DOI:10.3109/09546634.2010.489598
PMID:20673154
Abstract

Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous syndrome manifesting as hamartomatous growths in multiple organs. We present two cases of patients with TSC and associated facial angiofibromata treated with topical and oral rapamycin and discuss the role for rapamycin in the treatment of these disfiguring lesions. Our patients demonstrated decreased numbers of angiofibromata and less redness with this treatment. This is the first published report on the use of topical rapamycin for the treatment of angiofibromata.

摘要

结节性硬化症(TSC)是一种常染色体显性神经皮肤综合征,表现为多个器官的错构瘤生长。我们报告了两例 TSC 患者伴发面部血管纤维瘤,接受了雷帕霉素的局部和口服治疗,并讨论了雷帕霉素在治疗这些毁容病变中的作用。我们的患者在接受这种治疗后,血管纤维瘤数量减少,红色减少。这是第一份关于局部使用雷帕霉素治疗血管纤维瘤的报告。

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Long-Term Exposure and Safety of a Novel Topical Rapamycin Cream for the Treatment of Facial Angiofibromas in Tuberous Sclerosis Complex: Results From a Single-Center, Open-Label Trial.
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