[Emerging treatment approaches targeting the molecular pathogenesis of myelofibrosis].

Myeloproliferative neoplasms (MPNs) are a group of disorders characterized by the activation of the JAK/STAT signaling pathway at the level of hematopoietic stem cells. In one subtype, myelofibrosis (MF), JAK inhibitors have become the standard therapy for high-risk patients who are not eligible for hematopoietic stem cell transplantation. While current JAK inhibitors can alleviate disease-related symptoms, they have not yet been shown to modify the disease course or achieve a cure. In contrast, a watchful waiting approach is generally adopted for asymptomatic low-risk MF. Recently, advances in our understanding of the molecular pathogenesis of MPNs have led to the development of novel therapeutic strategies that could alter disease progression in addition to managing symptoms and complications. This article reviews the latest basic research on the pathogenesis and progression of MPNs, and provides an overview of current and emerging treatment strategies, with a particular focus on MF.