西罗莫司治疗淋巴管平滑肌瘤病的持久性。

Durability of Sirolimus for Lymphangioleiomyomatosis.

作者信息

Martirossian Alexandra, Shah Shiwan, Carrete Lola, Valle Jose, Valentine Vincent

机构信息

Department of Internal Medicine (AM, LC), University of Texas Medical Branch, Galveston, Texas; Department of Internal Medicine, Division of Pulmonary Critical Care & Sleep Medicine (SS, JV), University of Texas Medical Branch, Galveston, Texas; Department of Internal Medicine, Division of Pulmonary, Allergy, & Critical Care Medicine (VV), University of Alabama at Birmingham, Birmingham, Alabama..

出版信息

Am J Med Sci. 2017 Dec;354(6):603-607. doi: 10.1016/j.amjms.2016.11.028. Epub 2016 Dec 5.

DOI:10.1016/j.amjms.2016.11.028

PMID:29208258

Abstract

Lymphangioleiomyomatosis (LAM), a rare, multisystem disorder primarily affecting women of reproductive age, is characterized by cystic-appearing lung lesions, progressive loss of lung function, chylous effusions and renal angiomyolipomas. Sirolimus, an mammalian target of rapamycin inhibitor, has been shown to stabilize lung function, reduce symptoms and resolve chylous effusions in the short term for patients with LAM. Herein, we report a premenopausal female with LAM who experienced complete and durable resolution of her chylothoraces with significant and sustained improvement in lung function on sirolimus.

摘要

淋巴管平滑肌瘤病（LAM）是一种罕见的多系统疾病，主要影响育龄女性，其特征为肺部出现囊性病变、肺功能进行性丧失、乳糜性胸腔积液和肾血管平滑肌脂肪瘤。西罗莫司是一种雷帕霉素靶蛋白抑制剂，已证明可使LAM患者的肺功能在短期内保持稳定、减轻症状并消除乳糜性胸腔积液。在此，我们报告了一例绝经前LAM女性患者，其服用西罗莫司后乳糜胸完全且持久消退，肺功能得到显著且持续改善。