Steagall Wendy K, Stylianou Mario, Pacheco-Rodriguez Gustavo, Yu Zu Xi, Moss Joel
Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
ERJ Open Res. 2023 Aug 14;9(4). doi: 10.1183/23120541.00305-2023. eCollection 2023 Jul.
Lymphangioleiomyomatosis (LAM) is a multisystem disease affecting primarily women, characterised in the lung by proliferation of LAM cells, abnormal smooth muscle-like cells with dysfunctional tuberous sclerosis complex genes. This dysfunction results in activation of mechanistic target of rapamycin (mTOR), leading to LAM cell proliferation. Sirolimus (rapamycin) is the only United States Food and Drug Administration-approved treatment for pulmonary LAM, resulting in decreased LAM cell growth/size and stabilised lung function [1].
淋巴管平滑肌瘤病（LAM）是一种主要影响女性的多系统疾病，其肺部特征为LAM细胞增殖，LAM细胞是具有功能失调的结节性硬化复合物基因的异常平滑肌样细胞。这种功能失调导致雷帕霉素靶蛋白（mTOR）激活，进而导致LAM细胞增殖。西罗莫司（雷帕霉素）是美国食品药品监督管理局批准的唯一用于治疗肺部LAM的药物，可使LAM细胞生长/大小减小并使肺功能稳定[1]。
