Targeted therapy- Vemurafinib in BRAF V600E positive Langerhan cell histiocytosis

摘要

Background: Langerhans cell histiocytosis (LCH) is a clonally derived neoplasm due to constitutive activation of the MAP Kinase RAS-RAF-MEK-ERK cell signaling pathway. BRAF is a serine-threonine protein kinase involved in cell survival, proliferation, and differentiation; mutations of which is seen in 57% cases of LCH. Vemurafinib, a BRAF-V600E inhibitor is used in adults for treatment of malignant melanoma. There are case reports with Vemurafenib, as an emerging drug for the first line treatment of BRAF-V600E mutated LCH, here is one in support.