Article
Langerhans cell histiocytosis with BRAF p.N486_P490del or MAP2K1 p.K57_G61del treated by the MEK inhibitor trametinib.
Pediatric blood & cancer - 1 Dec 2020
Messinger Yoav H, Bostrom Bruce C, Olson Damon R, Gossai Nathan P, Miller Lane H, Richards Michael K
Abstract excerpt
Activating variants of the MAPK pathway have been found in some Langerhans cell histiocytosis (LCH) lesions. Inhibition of the MAPK pathway with trametinib (MEK inhibitor) has been shown to induce responses in LCH patients. Two adolescent males with LCH driven by BRAF p.N486_P490del have received trametinib for >1 year with no reactivation in one and partial response in another (including stable lung disease). A...
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