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Treatment Options

Watch and Wait

People with no symptoms are generally not treated at the time of diagnosis. Some people remain stable and symptom free for many years. It is very important for anyone who has been diagnosed with myelofibrosis to be closely monitored through regular medical check-ups to look for any signs or symptoms of disease progression.

Drug Therapies

Many kinds of drugs are used to treat myelofibrosis. They work in different ways and have different side effects. Ask your doctor or nurse to tell you about possible side effects before starting a new drug.

Chemotherapy

These drugs kill rapidly dividing cancer cells. In doing so, they often harm healthy cells, causing side effects. Chemotherapy drugs may be given by mouth (orally) or into the vein (intravenously).

JAK inhibitors

These new kinds of drugs work by targeting the JAK1 and JAK2 genes that help regulate blood. They can treat and lessen some of the symptoms of myelofibrosis, including an enlarged spleen, night sweats, itching and bone or muscle pain. They can work in both patients that have a JAK mutation and those that do not. The JAK inhibitor is the first Food and Drug Administration-approved therapy for patients with myelofibrosis.

Immunomodulators (IMiDs)

These drugs fight cancer cells by helping the immune system work normally. They sometimes are used to treat symptoms such as anemia, an enlarged spleen, bone pain and high platelet count.

Androgen therapy

This male hormone treatment can increase red blood cell production. These drugs may help with the symptoms of severe anemia.

Recombinant erythropoietin

This treatment helps regulate red blood cell production. An injection is given into a muscle or under the skin.

Glucocorticoids

Also known as steroids, these drugs are used to treat various conditions. In MF, they can help with serious anemia.

Bisphosphonates

These drugs can prevent bone loss in cancer patients, relieve bone pain and increase blood counts in MF patients. They are given through a vein.

Anagrelide hydrochloride

This drug, given orally, may be used in MF patients who have an extremely high platelet count.

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. It is also used to treat bone pain and tumors outside the bone marrow.

Surgical Removal of the Spleen (Splenectomy)

If your spleen is enlarged and causing harmful complications and pain, and other interventions have not been successful, you may benefit from having your spleen surgically removed. Your doctor will consider the risks and benefits of your particular situation and determine if this is a feasible option. If surgery is done, you should be closely monitored for complications.

Stem Cell Transplantation

This is the only type of treatment that has the potential to cure myelofibrosis. It is a very high-risk treatment that depends on the availability of a suitable donor. It is not an option for everyone.

Allogeneic stem cell transplant

In this procedure, a person with myelofibrosis receives high doses of chemotherapy or radiation to destroy the diseased bone marrow. The donor stem cells (from someone other than the patient) are then infused into the patient.

Non-myeloblative stem cell transplant

This transplant uses lower doses of chemotherapy or radiation prior to the infusion of donor cells. This type of transplant can be used in older and sicker patients, who may not be eligible for an allogeneic transplant.

Clinical Trials

Other treatments may be available through clinical trials. Clinical trials can offer the opportunity to benefit from the latest treatments or combinations of treatments while helping advance knowledge of the disease and its treatment options. Ask your health care team if a clinical trial might be right for you.