Radzikowska Elżbieta
III Department of Lung Diseases, National Tuberculosis and Lung Diseases Research Institute, Ul. Płocka 26, 01-138, Warsaw, Poland,
Lung. 2015 Aug;193(4):467-75. doi: 10.1007/s00408-015-9742-6. Epub 2015 May 17.
Lymphangioleiomyomatosis (LAM) is a rare multisystem disease, occurs in women, usually premenopausal, caused by the proliferation of neoplastic smooth muscle-derived cells. Mutations in the tuberous sclerosis complex genes, lead to the activation of mammalian target of rapamycin kinase (mTOR), results in proliferation of LAM cells, its increasing motility, and survival. Polycystic lung destruction, extensive involvement of lymphatic channels, chylothorax, chyloperitoneum, and renal angiomyolipomas can develop in LAM patients. The new, promising treatment strategies have been recently introduced due to discovery of the genetic and molecular mechanisms of LAM. Comprehension of the disease pathogenesis has resulted in the implementation of other therapeutic agents such as mTOR inhibitors, VEGF-D inhibitors, statins, interferon, chloroquine analogs, cyclin-dependent kinase inhibitors, matrix metalloproteinase inhibitors, aromatase inhibitors, and their combinations. The mTOR inhibitors appear to be the most important, and the efficacy of sirolimus in LAM treatment has been proved. The article discussed the new control studies with mTOR inhibitors, doxycycline, simvastatin, and combination of them in LAM patients.
淋巴管平滑肌瘤病（LAM）是一种罕见的多系统疾病，发生于女性，通常为绝经前女性，由肿瘤性平滑肌来源的细胞增殖引起。结节性硬化复合基因的突变导致雷帕霉素激酶哺乳动物靶点（mTOR）的激活，从而导致LAM细胞增殖、运动性增加和存活。LAM患者可出现多囊性肺破坏、淋巴管广泛受累、乳糜胸、乳糜腹和肾血管平滑肌脂肪瘤。由于LAM遗传和分子机制的发现，最近引入了新的、有前景的治疗策略。对疾病发病机制的理解导致了其他治疗药物的应用，如mTOR抑制剂、VEGF-D抑制剂、他汀类药物、干扰素、氯喹类似物、细胞周期蛋白依赖性激酶抑制剂、基质金属蛋白酶抑制剂、芳香化酶抑制剂及其联合应用。mTOR抑制剂似乎是最重要的，西罗莫司在LAM治疗中的疗效已得到证实。本文讨论了mTOR抑制剂、强力霉素、辛伐他汀及其联合应用于LAM患者的新对照研究。
