Journal
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
Volume 38, Issue 3, Pages E129-E132Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/MPH.0000000000000514
Keywords
Gorham-Stout; sirolimus; zoledronic acid; lymphatic malformation
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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.
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