If you aren't experiencing symptoms and don't show signs of anemia, an enlarged spleen or other complications, treatment usually isn't necessary. Instead, your doctor is likely to monitor your health closely through regular checkups and exams, watching for any signs of disease progression. Some people remain symptom-free for years.
For people with serious symptoms or complications, treatment options typically include:
• Blood transfusions. If you have severe anemia, periodic blood transfusions can increase your red blood cell count and ease anemia symptoms, such as tiredness and weakness. Sometimes, medications can help improve anemia so that you don't need blood transfusions.
• Androgen therapy. Taking a synthetic version of the male hormone androgen combined with a corticosteroid medication, such as prednisone, can promote red blood cell production and may improve severe anemia in some people. People who respond to this treatment after a month of therapy may continue on with the androgen and slowly taper off the prednisone. Androgen therapy does have risks, including liver damage, masculinizing effects in women, and growth of prostate cancer cells.
• Chemotherapy. Hydroxyurea (Hydrea) is the most commonly used chemotherapy drug in treating myelofibrosis. Hydroxyurea can reduce the size of an enlarged spleen, decrease high platelet counts, improve night sweats and weight loss, and possibly reduce bone marrow fibrosis.
• Radiation therapy. Radiation may help a small number of people who have bone pain. It can also help reduce the size of the spleen, when surgical removal isn't an option.
• Thalidomide with steroids. Using thalidomide along with prednisone can help reduce spleen size, improve anemia and white blood cell and platelet counts, and improve other systemic symptoms, such as weakness, fatigue, night sweats and shortness of breath. This therapy can also reduce the need for blood transfusions, but it's still fairly experimental.
• Surgical removal of the spleen (splenectomy). If the size of your spleen becomes painful and begins to cause harmful complications, and if you don't respond to other forms of therapy, you may benefit from having your spleen surgically removed. Risks include infection, excessive bleeding, blood clot formation leading to stroke or pulmonary embolism, and a higher rate of conversion to acute leukemia. After the procedure, some people experience liver enlargement and an abnormal increase in platelet count.
• Stem cell transplantation. Allogeneic stem cell transplantation — stem cell transplantation from a suitable donor — is the only treatment that has the potential to cure myelofibrosis. But it also has a high risk of life-threatening side effects because it requires high doses of chemotherapy and radiation before transplantation to destroy the diseased cells. After the procedure, there's a risk that the new stem cells will react against your body's healthy tissues, causing potentially fatal damage (graft-versus-host disease). Other risks include organ or blood vessel damage, cataracts, and the development of a different cancer later on. Most people with myelofibrosis, because of age, stability of the disease or other health problems, don't qualify for this treatment.
Currently being studied is a reduced-intensity transplant, also called a nonmyeloablative transplant or mini-transplant. Reduced-intensity transplants use lower doses of pre-transplant chemotherapy and radiation, relying instead on the donor's immune system to destroy diseased cells. Although reduced-intensity transplantation has side effects, doctors hope that it will be safer but just