Analysis of the MILES cohort reveals determinants of disease progression and treatment response in lymphangioleiomyomatosis
Analysis of the MILES cohort reveals determinants of disease progression and treatment response in lymphangioleiomyomatosis
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DOI：
10.1183/13993003.02066-2018
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发表时间：
2019-04-01
影响因子：
24.3
通讯作者：
McCormack, Francis X.
中科院分区：
文献类型：
--
作者：
Gupta, Nishant;Lee, Hye-Seung;McCormack, Francis X.
简介：多中心国际淋巴管平滑肌瘤病（LAM）西罗莫司疗效（MILES）试验显示，西罗莫司可稳定中重度LAM患者的肺功能。本研究的目的是进一步检查MILES队列中的种族、人口统计学、临床和生理患者特征对LAM疾病进展和治疗反应的影响。（绝经前/绝经后），人种（亚裔/高加索人），支气管扩张剂反应性（存在/不存在），1秒内的初始用力呼气量（FEV 1; 51-70% vs. 600 pg.mL（-1）确定了安慰剂组更可能下降并对西罗莫司治疗有反应的患者亚组。在LAM患者中，无论绝经状态、种族、支气管扩张剂反应性、基线FEV 1或TSC相关性如何，西罗莫司治疗均有益。血清VEGF-D和绝经状态可以帮助指导治疗决策。
Introduction: The Multicenter International Lymphangioleiomyomatosis (LAM) Efficacy of Sirolimus (MILES) trial revealed that sirolimus stabilised lung function in patients with moderately severe LAM. The purpose of this study was to further examine the MILES cohort for the effects of racial, demographic, clinical and physiological patient characteristics on disease progression and treatment response in LAM.Methods: MILES subjects were stratified on the basis of menopausal status (pre-menopausal/postmenopausal), race (Asian/Caucasian), bronchodilator responsiveness (present/absent), initial forced expiratory volume in 1 s (FEV1; 51-70% versus 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.