Publication at Faculty of Medicine in Hradec Králové |
2015
Abstract
Out of the group of Philadelphia chromosome-negative(Ph-) diseases, primary myelofibrosis (PMF) has the worst prognosis. Younger patients with higher risk disease should be directed to undergo allogenic haematopoietic cell transplantation still relied on as the only curative option.
JAK2V617F somatic mutation discovery marks another important milestone on the path in understanding of PMF pathogenesis and has actually paved the way for the discovery of JAK1/2 inhibitors. Ruxolitinib became the first representative of this new group to have received approval from Food and Drug Administration (FDA) for the therapy of PMF in 2011.