Article
Treatment of severe Kaposiform lymphangiomatosis positive for NRAS mutation by MEK inhibition.
Pediatric research - 1 Dec 2023
Chowers Guy, Abebe-Campino Gadi, Golan Hana, Vivante Asaf, Greenberger Shoshana, Soudack Michalle, Barkai Galia, Fox-Fisher Ilana, Li Dong, March Michael, Battig Mark R, Hakonarson Hakon, Adams Denise, Dori Yoav, Dagan Adi
Abstract excerpt
BACKGROUND: Kaposiform lymphangiomatosis (KLA) is a complex lymphatic anomaly involving most commonly the mediastinum, lung, skin and bones with few effective treatments. In recent years, RAS-MAPK pathway mutations were shown to underlie the pathogenesis of several complex lymphatic anomalies. Specifically, an activating NRAS mutation (p.Q61R) was found in the majority of KLA patients. Recent reports demonstrated...
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