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Dabrafenib and Trametinib Treatment for Erdheim-Chester Disease With Brain Stem Involvement
Erdheim-Chester disease (ECD) is a rare form of non–Langerhans cell histiocytosis characterized by infiltration of organs by CD68+ and CD1a− lipid-laden histiocytes, including the central nervous system in more than a third of patients. Molecular analysis of ECD samples has demonstrated the prevalence of BRAF V600E mutations as high as 54%. Recently, vemurafenib became the only Food and Drug Administration–approved treatment for patients with ECD who carry the BRAF V600E mutation. However, dabrafenib has been suggested to have greater brain distribution.
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