Treatments and drugs

By Mayo Clinic Staff

Immediate treatment may not be necessary

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.

Treatments that target gene mutations

Researchers are working to develop medications that target the JAK2 gene mutation that's thought to be responsible for myelofibrosis.

The first of these medications approved by the Food and Drug Administration is ruxolitinib (Jakafi). Ruxolitinib and the other medications being developed and tested in clinical trials have been helpful in decreasing enlarged spleens and reducing symptoms associated with myelofibrosis.

It's not yet clear whether these medications can help people with myelofibrosis live longer. But preliminary indications from clinical trials show promise.

Ruxolitinib works by stopping the action of all JAK-related genes in the body, including those found in both healthy and diseased cells. Because healthy cells are affected, side effects may occur, such as bleeding, infection, bruising, dizziness and headaches.

Treatments for anemia

If myelofibrosis is causing severe anemia, you may consider treatment, such as:

  • Blood transfusions. If you have severe anemia, periodic blood transfusions can increase your red blood cell count and ease anemia symptoms, such as fatigue and weakness. Sometimes, medications can help improve anemia.
  • Androgen therapy. Taking a synthetic version of the male hormone androgen may promote red blood cell production and may improve severe anemia in some people. Androgen therapy does have risks, including liver damage and masculinizing effects in women.
  • Thalidomide and related medications. Thalidomide (Thalomid) and the related drugs lenalidomide (Revlimid) and pomalidomide (Pomalyst) may help improve blood cell counts and may also relieve an enlarged spleen. These drugs may be combined with steroid medications. Thalidomide and related drugs carry a risk of serious birth defects and require special precautions. This type of treatment is being studied in clinical trials.

Treatments for enlarged spleen

If an enlarged spleen is causing complications, your doctor may recommend treatment. Your options may include:

  • Surgical removal of the spleen (splenectomy). If the size of your spleen becomes so large that it causes you pain 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 and blood clot formation leading to stroke or pulmonary embolism. After the procedure, some people experience liver enlargement and an abnormal increase in platelet count.

  • Chemotherapy. Chemotherapy drugs may reduce the size of an enlarged spleen and relieve related symptoms, such as pain.
  • Radiation therapy. Radiation uses high-powered beams, such as X-rays, to kill cells. Radiation therapy can help reduce the size of the spleen, when surgical removal isn't an option.

Stem cell transplant

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.

Most people with myelofibrosis, because of age, stability of the disease or other health problems, don't qualify for this treatment.

Prior to a stem cell transplant, also called a bone marrow transplant, you receive very high doses of chemotherapy or radiation therapy to destroy your diseased bone marrow. Then you receive infusions of stem cells from a compatible donor.

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.

Doctors are studying a reduced-intensity transplant, also called a nonmyeloablative transplant or minitransplant. Reduced-intensity transplants use lower doses of pre-transplant chemotherapy and radiation. Although reduced-intensity transplantation has side effects, doctors hope that it will one day be a safer option for older adults.

Mar. 25, 2014

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