Onkológia 5/2018
Primary myelofibrosis – current view of pathophysiology, prognosis and treatment
Myelofibrosis (MF) is a Philadelphia chromosome-negative myeloproliferative neoplasm (Ph-MPN). It can be sub-categorized into primary myelofibrosis, post polycythemia vera myelofibrosis and post essential thrombocythemia myelofibrosis. MF is a life-threatening hematologic malignancy characterized by dysregulation of the Janus associated kinase (JAK)/signal transducer and activator of transcription (STAT) signaling network. We cover the pathogenesis, clinical features, new prognostic models, current treatment of MF and discuss agents in development. There are many ongoing clinical trials evaluating novel therapeutic approaches, including selective JAK inhibitors, histone deacetylase/DNA methyltransferase inhibitors, PI3K-inhibitors, anti-fibrotic agents, immunomodulators, monoclonal antibodies and immune checkpoint inhibitors. The development of biomarker driven clinical trials of mechanism based novel therapeutics will usher in a new era of advances in the treatment of this chronic and progressive myeloid malignancy.
Keywords: myelofibrosis, ruxolitinib, bone marrow transplant, HDP90, PI3K-inhibitors, immunomodulators
