Vasani Resham J
Department of Dermatology, K J Somaiya Hospital and Research Centre, Sion, Mumbai, Maharashtra, India.
Indian J Dermatol. 2015 Mar-Apr;60(2):165-9. doi: 10.4103/0019-5154.152516.
Facial angiofibromas are the most visible and unsightly of all the cutaneous manifestations of tuberous sclerosis (TSC). A 17-year-old female, a known case of TSC, presented for the treatment of cosmetically disfiguring facial angiofibromas. She was started on twice daily application of 0.1% sirolimus ointment prepared from crushed tablets of sirolimus compounded in white soft paraffin. After 3 months of use, there was visible decrease in the erythema and the size of the angiofibromas. In an attempt to accelerate the response, the concentration was further increased to 1% sirolimus which was used for a month, resulting in a decrease not only in the size and redness but also in the number of the angiofibromas. The patient did not experience any cutaneous or systemic complications related to therapy. Sirolimus belongs to a novel class of anticancer drugs known as mTOR (mammalian target of Rapamycin) inhibitors. Sirolimus has been used as a targeted therapy for the renal and neurological manifestations of TSC. Topical preparation of sirolimus is not commercially available till date and hence preparations from crushed tablets or oral solution of sirolimus have been used with beneficial effects in treatment of angiofibromas especially in younger patients with flatter lesions. Randomized controlled trials are necessary to enable us to confirm the efficacy, long-term safety, the optimal dosage and possibility of reappearance once the drug is withdrawn. This is possibly the first case report of the use of topical sirolimus in India.
面部血管纤维瘤是结节性硬化症（TSC）所有皮肤表现中最明显且最难看的。一名17岁女性，已知患有TSC，前来治疗对面部容貌有毁损性的血管纤维瘤。开始每日两次涂抹由西罗莫司碾碎片与白软石蜡混合配制而成的0.1%西罗莫司软膏。使用3个月后，血管纤维瘤的红斑和大小有明显减小。为加快疗效，浓度进一步增至1%西罗莫司并使用了一个月，结果不仅血管纤维瘤的大小和发红程度减轻，数量也减少了。患者未出现与治疗相关的任何皮肤或全身并发症。西罗莫司属于一类新型抗癌药物，称为mTOR（雷帕霉素哺乳动物靶点）抑制剂。西罗莫司已被用作治疗TSC肾脏和神经表现的靶向疗法。西罗莫司的外用制剂迄今尚无商业供应，因此碾碎片或西罗莫司口服溶液制剂已用于治疗血管纤维瘤并取得有益效果，尤其适用于皮损较扁平的年轻患者。需要进行随机对照试验，以便我们能够确认其疗效、长期安全性、最佳剂量以及停药后复发的可能性。这可能是印度使用外用西罗莫司的首例病例报告。
