Article
High-risk LCH in infants is serially transplantable in a xenograft model but responds durably to targeted therapy.
Blood advances - 25 Feb 2020
Lee Lynn H, Krupski Christa, Clark Jason, Wunderlich Mark, Lorsbach Robert B, Grimley Michael S, Burwinkel Matthew, Nelson Adam, Kumar Ashish R
Abstract excerpt
Langerhans cell histiocytosis (LCH) is a rare hematologic neoplasm characterized by a clonal proliferation of Langerhans-like cells. Genomic profiling has identified recurrent somatic activating mutations in the mitogen-activated protein kinase pathway, which are targetable by small-molecule inhibitors. However, key questions such as the curative potential of targeted therapy and the cell of origin remain...
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