期刊
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
卷 38, 期 3, 页码 E129-E132出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/MPH.0000000000000514
关键词
Gorham-Stout; sirolimus; zoledronic acid; lymphatic malformation
Gorham-Stout disease is a life-threatening disorder often manifested by lymphatic malformation and osteolysis. Unfortunately, available therapies are not uniformly effective and often carry substantial morbidity. We report an 18-year-old male with Gorham-Stout disease manifested by lytic rib lesions and an intractable pleural effusion that responded dramatically to the combination of the mammalian target of rapamycin (mTOR) inhibitor sirolimus and the aminobisphosphonate zoledronic acid after failing interferon therapy. This tolerable therapeutic combination has demonstrated synergism in preclinical cancer models and merits further study in vascular anomalies.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据