卡波西样血管内皮瘤（KHE）是一种血管肿瘤，被认为是边缘性或局部侵袭性的，可能导致致命的结果。对于 KHE 患者来说，手术和栓塞等传统疗法可能不足或在技术上不可能。西罗莫司（或雷帕霉素）是雷帕霉素机制靶点的特异性抑制剂，最近被证明在 KHE 的治疗中非常有用。在这里，我们重点介绍西罗莫司对 KHE 影响的最新实质性进展，并讨论西罗莫司治疗这种疾病的潜在作用机制。预防血小板活化和炎症，以及抗血管生成作用、抑制淋巴管生成、减轻纤维化或所有这些作用的组合，可能是西罗莫司的治疗效果的原因。此外，还讨论了一些 KHE 患者对西罗莫司耐药的机制。最后，我们回顾了最近在 KEH 病变中发现的体细胞突变，并基于对 KHE 细胞和​​分子发病机制的进一步了解讨论了新治疗靶点的潜力。© 2024 UICC。

Kaposiform hemangioendotheliomas (KHEs) are vascular tumors that are considered borderline or locally aggressive and may lead to lethal outcomes. Traditional therapies, such as surgery and embolization, may be insufficient or technically impossible for patients with KHE. Sirolimus (or rapamycin), a specific inhibitor of mechanistic target of rapamycin, has recently been demonstrated to be very useful in the treatment of KHEs. Here, we highlight recent substantial progress regarding the effects of sirolimus on KHEs and discuss the potential mechanisms of action of sirolimus in treating this disease. The prevention of platelet activation and inflammation, along with antiangiogenic effects, the inhibition of lymphangiogenesis, the attenuation of fibrosis, or a combination of all these effects, may be responsible for the therapeutic effects of sirolimus. In addition, the mechanism of sirolimus resistance in some KHE patients is discussed. Finally, we review the somatic mutations that have recently been identified in KEH lesions, and discuss the potential of novel therapeutic targets based on these further understandings of the cellular and molecular pathogenesis of KHE.© 2024 UICC.