Myelofibrosis: the present and the future-a review article

Abstract

Currently, there is no drug option that is curative. The goal for most patients is to relieve symptoms, reduce an enlarged spleen, improve blood cell counts and minimize the risk of complications. Myelofibrosis is a disease with few approved treatment options, but many new agents are being investigated and developed. For highrisk disease, stem cell transplant is often considered. The standard of care for myelofibrosis is ruxolitinib (Jakafi, Incyte), which is the only therapy approved by the US Food and Drug Administration for the disease. Ruxolitinib inhibits JAK1 and JAK2, and it can be effective regardless of which molecular mutations a patient has. The data show that ruxolitinib will improve splenomegaly in most patients. In approximately half of patients, this improvement will meet the response criteria from the International Working Group for Myelofibrosis. Most patients will also experience improvement in other symptoms. Additionally, clinical trials demonstrate that ruxolitinib increases survival, when accounting for crossover. That being said, ruxolitinib is not considered a curative therapy. A limitation of ruxolitinib is that it typically does not improve anemia or thrombocytopenia. Ruxolitinib has been tried in at least 10 different combinations to date. Those studies are largely ongoing, so there are no final conclusions. At this time, there are no clear data to support the use of a particular combination outside of a clinical trial.