TY -的T1 -英里群的分析揭示了决定因素的疾病进展和治疗反应lymphangioleiomyomatosis JF -欧洲呼吸杂志》乔和J - 10.1183/13993003.02066 -2018欧元六世- 53 - 4 SP - 1802066 AU -古普塔,作者Nishant AU -李,Hye-Seung盟——年轻,丽莎·r·盟——奇怪,查理AU -莫斯,乔尔盟——歌手,丽芬妮g . AU -醒来时,Koh盟——巴克,艾伦·f . AU -查普曼,杰弗里·t . AU -黑雁,Mark l . AU -股票,James m . AU -布朗AU - Lynch, Joseph P. AU - Goldberg, Hilary J. AU - Downey, Gregory P. AU - Taveira-DaSilva, Angelo M. AU - Krischer, Jeffrey P. AU - Setchell, Kenneth AU - Trapnell, Bruce C. AU - Inoue, Yoshikazu AU - McCormack, Francis X. A2 -，西罗莫司(MILES)的多中心国际淋巴血管平滑肌瘤症(LAM)疗效试验显示，西罗莫司稳定了中度LAM患者的肺功能。本研究的目的是进一步研究MILES队列在LAM的种族、人口统计学、临床和生理特征对疾病进展和治疗反应的影响。方法根据绝经期状况(绝经前/绝经后)、种族(亚洲人/高加索人)、支气管扩张剂反应性(存在/不存在)、1 s内初始用力呼气量(FEV1;51-70%对≤50%预测)和结节性硬化症(TSC)相关性(yes/no)。采用线性混合效应模型比较坡度差异，采用非参数检验比较各地层处理组间的中位数和比例。结果在MILES安慰剂组中，绝经前患者下降速度比绝经后患者快5倍(平均±se FEV1斜率- 17±3 vs - 3±3 mL·月- 1;p = 0.003)。经西罗莫司治疗后，绝经前(- 17±3 vs - 1±2 mL·月- 1;和绝经后患者(- 3±3 vs 6±3 mL·月- 1; p=0.04) exhibited a beneficial response in mean±se FEV1 slope compared with the placebo group. Race, LAM subtype, bronchodilator responsiveness or baseline FEV1 did not impact the rate of disease progression in the placebo group or treatment response in the sirolimus group. Menopausal status and race had differential effects on the adverse event profile of sirolimus. Baseline serum vascular endothelial growth factor (VEGF)-D >600 pg·mL−1 identified subgroups of patients who were more likely to decline on placebo and respond to treatment with sirolimus.Conclusions In LAM patients, treatment with sirolimus is beneficial regardless of menopausal status, race, bronchodilator responsiveness, baseline FEV1 or TSC association. Serum VEGF-D and menopausal status can help inform therapeutic decisions.Menopausal status and serum vascular endothelial growth factor-D levels are clinically useful variables that should be taken into consideration when making therapeutic decisions and designing clinical trials for patients with lymphangioleiomyomatosis http://ow.ly/ijGB30nrNCB ER -