王森分, 刘元香, 徐子刚. 外用雷帕霉素制剂的应用进展[J]. 罕见病研究, 2023, 2(2): 273-280. DOI: 10.12376/j.issn.2097-0501.2023.02.013
引用本文: 王森分, 刘元香, 徐子刚. 外用雷帕霉素制剂的应用进展[J]. 罕见病研究, 2023, 2(2): 273-280. DOI: 10.12376/j.issn.2097-0501.2023.02.013
WANG Senfen, LIU Yuanxiang, XU Zigang. Progress in the Treatment of Topical Rapamycin Preparations[J]. Journal of Rare Diseases, 2023, 2(2): 273-280. DOI: 10.12376/j.issn.2097-0501.2023.02.013
Citation: WANG Senfen, LIU Yuanxiang, XU Zigang. Progress in the Treatment of Topical Rapamycin Preparations[J]. Journal of Rare Diseases, 2023, 2(2): 273-280. DOI: 10.12376/j.issn.2097-0501.2023.02.013

外用雷帕霉素制剂的应用进展

Progress in the Treatment of Topical Rapamycin Preparations

  • 摘要: 雷帕霉素是哺乳动物雷帕霉素靶蛋白(mTOR)受体抑制剂。临床对mTOR信号通路及其对肿瘤发生和血管增殖下游作用的认识进展,拓宽了mTOR抑制剂在许多具有挑战性疾病中的临床应用。口服雷帕霉素用于治疗肾移植、肺淋巴管平滑肌瘤病、结节性硬化症(TSC)等,但系统治疗具有明显的副作用。为了减轻系统使用雷帕霉素的副作用, 局部治疗已经开展。近年来,雷帕霉素外用剂型的临床应用报道逐渐增多,如TSC面部血管纤维瘤、淋巴管畸形、Kaposi样血管内皮瘤、丛状血管瘤等。外用雷帕霉素可在避免系统性不良反应的同时,进行有效的长期治疗,为皮肤科临床医生提供了新的治疗手段。本文探讨雷帕霉素外用制剂的临床应用进展。

     

    Abstract: Rapamycin is a mammalian target of rapamycin(mTOR) receptor inhibitor. Advances in the understanding of the mTOR signaling pathway and its downstream effects on tumorigenesis and vascular proliferation have broadened the clinical applications of mTOR receptor inhibitors in treating many challenging diseases. Rapamycin is used orally for the treatment of kidney transplantation, lymphatic leiomyomatosis of lung, tuberous sclerosis complex(TSC), and etc. But systemic therapy using the rapamycin has significant side effects. To mitigate the side effects of systemic rapamycin for dermatologic applications, clinicians have used topical therapy. In recent years, research publications on the topical rapamycin in the treatment of a variety of diseases have increased, as on such diseases of facial angiofibroma of tuberous sclerosis complex, lymphatic malformation, Kaposi hemangioendothelioma, tufted angiomas, and etc. Topical rapamycin can be used as an effective long-term therapy while avoiding systemic side effects, providing a new treatment method for dermatologists. This paper discusses the progress in the treatment of topical rapamycin preparations.

     

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