Myelofibrosis is a serious bone marrow disorder that disrupts your body's normal production of blood cells. The result is extensive scarring in your bone marrow, leading to severe anemia, weakness, fatigue, and often, an enlarged spleen and liver. Myelofibrosis is an uncommon type of chronic leukemia — a cancer that affects the blood-forming tissues in the body. Myelofibrosis belongs to a group of diseases called myeloproliferative disorders.
Double-blind, randomized, placebo-controlled phase 3 study in 94 sites in 24 countries in which 289 adult patients (≥18 years of age) with intermediate-2 or high-risk primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF were randomly assigned between December 2011 and September 2012 to once-daily oral fedratinib, at a dose of 400 mg or 500 mg, or placebo, for at least 6 consecutive 4-week cycles.The primary end point was spleen response at week 24 and confirmed 4 weeks later.
In the past, the treatment of myelofibrosis has depended on the symptoms and degree of the low blood counts. A long-term remission is possible for some patients with bone marrow transplantation. Such treatment should be considered for younger patients and some others. Other treatment may involve:Blood transfusions and medicines to correct anemia, Radiation and chemotherapy, Medicines to target a genetic mutation, Splenectomy if swelling causes symptoms, or to help with anemia.