Myelofibrosis is a rare form of bone marrow cancer that effects the production of blood cells that is caused by mutations in bone marrow stem cell DNA. Researchers today are still trying to determine why these mutations occur. The mutated blood cells continue to produce until they influence the production of red blood cells, platelets, and white blood cells. There are less than 20,000 patients in the United States with Myelofibrosis.
Myelofibrosis is classified as a type of chronic leukemia, which effects blood forming tissues in the body. This disease also belongs to a group called myeloproliferative disorders which contain diseases of the bone marrow and blood, have no known causes and are unique in their symptoms and prognosis.
What are the symptoms of Myelofibrosis?
Because the disease develops slowly, many myelofibrosis patients do not experience symptoms in the early stages. Those with a more aggressive case of Myelofibrosis typically experience the following symptoms:
- Anemia from bone marrow scarring that causes weakness and fatigue
- Enlarged spleen, which causes pain on left side of body below the ribs
- Bone pain
- Fever and night sweats
- Loss of platelets which causes Easy bruising and bleeding
Affected individuals receive frequent checkups to detect progression of the illness. In major myelofibrosis cases there are often low ranges of circulating red blood cells, a condition known as anemia. White blood cells and platelets are also decreased. However, there are sometimes too many white blood cells produced. Blood cells are initially affected, which leads to complications throughout the body.
There isn't any single prognosis for individuals who suffer from myelofibrosis, the prognosis is different for every patient. While some individuals live for a few years without having major symptoms, others find that the disease progresses rapidly and is aggressive. Physicians evaluate a wide range of factors earlier than making an analysis of myelofibrosis. Since every case of myelofibrosis is completely different, a medical history, a physical examination, laboratory checks and a bone marrow examination are usually required to diagnose the illness.
What are the risk factors of Myelofibrosis?
Age is the main risk factor for Myelofibrosis, most people who are diagnosed are over the age of 50. Other risk factors include expose to radiation, chemicals, or other blood disorders such as essential thrombocythemia or polycythemia vera.
When myelofibrosis happens by itself, it is referred to as major myelofibrosis. If it occurs as the results of a separate disease, it is known as secondary myelofibrosis (e.g. scar tissue in the bone marrow as a complication of an autoimmune illness).
Ruxolitinib (Jakafi; Incyte) is a small-molecule inhibitor of JAK1/2 that has proven to be effective at reducing splenomegaly and ameliorating symptoms in myeloproliferative neoplasms. Based on the results of 2 pivotal randomized phase III clinical trials, ruxolitinib has become the first therapeutic drug to be approved by the U.S. Food and Drug Administration for treatment of patients with myelofibrosis. Ruxolitinib offers a well-tolerated oral therapeutic option for patients with myelofibrosis with symptomatic splenomegaly and debilitating disease-related symptoms, but it does not seem to be effective at eliminating the underlying hematological malignancy.
Myelofibrosis Clinical Trials
There are currently roughly 400 total clinical trials for myelofibrosis patients. Clinical trials provide early access to cutting edge therapies and treatments that are otherwise inaccessible.