Myelofibrosis Overview
Myelofibrosis (MF) is a chronic malignant hematologic disease for which there are currently no approved treatment options in the United States. It is part of a related group of conditions referred to as myeloproliferative neoplasms (MPNs), which also include polycythemia vera (PV), essential thrombocythemia (ET), and chronic myeloid leukemia.1 MF may present as a primary condition or may occur upon progression of PV or ET (secondary MF).2 MF is uncommon, affecting about 1.5 out of 100,000 people annually.3
MF can affect both men and women, and although it is usually diagnosed in patients who are older than 50 years, it can occur at any age.4 While a diagnosis can be made when a routine examination reveals an enlarged spleen, about 30% of patients are diagnosed based on the results of blood cell counts without determined symptoms.1 The symptoms of MF can adversely affect patient quality of life and may include fatigue, weakness, shortness of breath, weight loss, night sweats, and bone pain.3 To control the disease and its symptoms, patients may receive blood transfusions, radiation, chemotherapy, or stem cell transplantation. In addition, splenomegaly occurs frequently, causing discomfort and potential complications such as low platelet count, severe anemia, or portal hypertension in case a splenectomy is indicated.4 The median survival time for MF patients from the time of diagnosis is currently approximately 6 years.5
There is no FDA-approved treatment for MF. Therefore, novel approaches to treat MF are an important medical need to address. Among these, JAK pathway inhibitors are a promising class of agents that are currently being assessed for their impact on the disease.1