Targeting the BRAF pathway in haematological diseases

Abstract Since the recognition of BRAF V600E mutations in the majority of cases of hairy cell leukaemia, Erdheim–Chester disease and Langerhans cell histiocytosis, the targeted oral kinase inhibitors dabrafenib and vemurafenib have been adapted for their treatment. Like other targeted agents, these drugs produce high response rates and predictable but unique side effects. Physician familiarity is essential for the effective use of these agents. We review the Australian experience of BRAF/MEK inhibitor therapy in these rare haematological cancers.