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西罗莫司治疗淋巴管肌瘤病专家共识（2018）
中华医学会呼吸病学分会间质性肺疾病学组
淋巴管肌瘤病共识专家组
中国医学科学院罕见病研究中心
中国研究型医院学会罕见病分会
作者及单位信息
·
DOI： 10.3760/cma.j.issn.1001-0939.2019.02.002
Consensus Statement: sirolimus (rapamycin) as therapy for lymphangioleiomyomatosis (2018)
Interstitial Lung Disease Group, Chinese Thoracic Society, Chinese Medical Association
Expert Consensus Group of Lymphangioleiomyomatosis
Rare Diseases Research Center, Chinese Academy of Medical Sciences
Rare Diseases Society, Chinese Research Hospital Association
Authors Info & Affiliations
Interstitial Lung Disease Group, Chinese Thoracic Society, Chinese Medical Association
Expert Consensus Group of Lymphangioleiomyomatosis
Rare Diseases Research Center, Chinese Academy of Medical Sciences
Rare Diseases Society, Chinese Research Hospital Association
·
DOI： 10.3760/cma.j.issn.1001-0939.2019.02.002
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摘要

淋巴管肌瘤病（lymphangioleiomyomatosis，LAM），又称淋巴管平滑肌瘤病，是一种以双肺弥漫性囊性变为主要特征的、罕见的多系统低度恶性肿瘤性疾病，主要发生于女性。LAM可导致肺功能逐渐下降，并可反复发生气胸、乳糜胸等并发症。近年来的研究发现，TSC1/TSC2基因突变是LAM发病中的关键机制，并研发了第一个治疗LAM的靶向治疗药物西罗莫司（又称雷帕霉素）。本共识内容包括西罗莫司治疗LAM的适应证、安全性和临床使用的建议。

引用本文

中华医学会呼吸病学分会间质性肺疾病学组,淋巴管肌瘤病共识专家组,中国医学科学院罕见病研究中心,等. 西罗莫司治疗淋巴管肌瘤病专家共识（2018）[J]. 中华结核和呼吸杂志,2019,42(2)：92-97.

DOI:10.3760/cma.j.issn.1001-0939.2019.02.002

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淋巴管肌瘤病（lymphangioleiomyomatosis，LAM），又称淋巴管平滑肌瘤病，是一种以双肺弥漫性囊性变为主要特征的、罕见的多系统低度恶性肿瘤性疾病，主要发生于女性 [ 1 , 2 ]。LAM可导致肺功能逐渐下降，并可反复发生气胸、乳糜胸等并发症。近年来的研究发现，TSC1/TSC2基因突变是LAM发病的关键机制，并研发了第一个治疗LAM的靶向治疗药物西罗莫司（又称雷帕霉素）。西罗莫司虽然在多个国家和地区被批准用于治疗LAM，但在我国尚未获得国家食品药品监督管理局的批准 [ 3 ]。为了规范西罗莫司的使用，促进相关临床研究，推动西罗莫司尽早在我国获得治疗LAM的适应证，特制定本专家共识，内容包括西罗莫司的适应证、安全性和临床使用的建议。
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备注信息
A
徐凯峰，中国医学科学院北京协和医院呼吸与危重症医学科，北京100730，Email： nc.defhcabmupfkux
B
所有作者均声明不存在利益冲突
C
国家重点研发计划 (2016YFC0901502)
北京市科技计划 (Z151100003915126)
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