Investigational drugs in early phase trials for myelofibrosis

Introduction Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by bone marrow fibrosis, cytopenias, and organomegaly. Four JAK inhibitors are US-FDA approved for treatment of MF. While these drugs reduce symptom burden and spleen size to varying degrees, they do not affect the natural disease course or decrease the risk of leukemic transformation. Therefore, there is a strong need for newer therapies to further advance the field and improve the outcomes of MF. In this review, we cover novel therapies for MF currently in early stages of development.