Efficacy of MEK inhibitors in Erdheim-Chester disease: impact of MAPK pathway pathogenic variants

Abstract excerpt

Erdheim Chester disease (ECD) is a rare histiocytic neoplasm characterized by recurrent activating mutations in the mitogen-activated protein kinase – extracellular signal-regulated kinase (MAPK-ERK [ RAS-RAF-MEK-ERK] ) pathway, most commonly BRAF V600E [ 1 ]. Vemurafenib was the first targeted therapy to be approved by the US-FDA for BRAF V600E -mutated ECD due to near-universal responses [ 2 ]. Subsequently, an...

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