Myeloproliferative Neoplasms (MPN)

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Treatment Options

Myeloproliferative neoplasms (MPN) may cause only mild symptoms, with little effect on health, or may be severe. Your doctor will base your treatment plan on the severity of your MPN and the type that you have. Treatment is geared toward relieving symptoms and preventing complications, and it can vary greatly from person to person. Your plan will be customized to you.

Watchful Waiting 

If your disease is mild and interferes only a little or not at all with your usual activities, your doctor may recommend watchful waiting. This means you receive no treatment or you only take aspirin to reduce your risk for blood clots, but you visit your doctor on a regular basis (or if you notice changes) to check your health. Some people with MPN go for several years with no major health changes.

Phlebotomy

Phlebotomy means removing blood from the body. In MPN, large-volume phlebotomy (usually one unit of blood, or 500 milliliters) is done to decrease red blood cell (RBC) counts. This may relieve symptoms linked with high levels of RBCs. Phlebotomy is also used along with aspirin to reduce clot complications in people with polycythemia vera and low clot risk.

Cell-Reducing Medications

People who have had or are at high risk for a blood clot and people with very high non-RBC blood counts may need medications that decrease their production of blood cells or the release of blood cells from their bone marrow.

  • The mild chemotherapy drug hydroxyurea (Hydrea) can be used to lower RBCs, white blood cells, and platelets.
  • Interferon-alpha, a human protein, can be used to lower all three types of blood cells.
  • Anagrelide (Agrylin) is a drug that lowers the platelet count. 

Supportive Care

In advanced disease and burn-out phases (when your marrow is not producing enough healthy blood cells), your doctor may recommend steps to reduce symptoms caused by low levels of blood cells. This is called supportive care. For MPN, these measures may include medicines and transfusions.

Medicines used in supportive care for MPN include the following:

  • The growth factor erythropoietin (EPO) to increase your red blood cells
  • The growth factor granulocyte colony-stimulating factor (G-CSF) or granulocyte macrophage colony-stimulating factor (GM-CSF) to increase your white blood cells
  • Antibiotics and antiviral drugs to fight infections that your white blood cells cannot fight on their own

Transfusions are used to add red blood cells or platelets to your bloodstream if medicines do not increase your levels enough.

Chemotherapy

Doctors may use intensive anti-cancer drugs, or chemotherapy, like that used for acute myelogenous leukemia (AML), to control your body’s production of abnormal blood cells. Your doctor may recommend this if your MPN progresses to a phase called a blast crisis—meaning there is an increase in the number of the abnormal stem cells in your bone marrow or blood. Chemotherapy may be given intravenously, by mouth, or directly into your spinal fluid (if you need treatment in your central nervous system, which is rare in patients with MPN). These medicines are distributed throughout your body through your bloodstream. They can help control your body’s production of abnormal blood cells. Read more about chemotherapy for AML.

Side effects are an important concern for many people receiving chemotherapy. Besides killing cancer cells, chemotherapy can damage healthy tissue and cause side effects like fatigue, hair loss, digestive problems, mouth sores, skin irritation, and low levels of healthy blood cells. Your doctor will talk with you about the specific side effects you might develop and what to watch for. We will help you take the best possible care of yourself to prevent, minimize, or relieve side effects so you can focus your energy on your recovery.

Transplantation

Patients with severe or advanced MPN may be treated with bone marrow transplantation (hematopoietic cell transplantation). This is currently the only type of treatment that has the potential to cure MPN.

MPN patients receive allogeneic transplants (using stem cells from a donor). First they have chemotherapy designed to kill diseased stem cells in their marrow. This is called conditioning. Some have myeloablative conditioning, which completely destroys their bone marrow and immune system. Others have low-dose conditioning, called a non-myeloablative transplant or mini-transplant. The low-dose conditioning destroys some of your bone marrow cells. The intent is to suppress your immune system enough that the donor cells are not rejected and they can help to kill remaining abnormal cells in your marrow by an immune attack on those cells.

A transplant is not appropriate for everyone. For instance, a transplant may be too challenging for older people, especially if they have other health problems besides MPN, as many older people do. Though some people have had successful transplants at age 70, it is not the standard treatment in this age group.

For those who cannot withstand a myeloablative transplant, a non-myeloablative transplant may be an option, as it is less toxic and the side effects are generally milder. This option was developed in the late 1990s by Rainer F. Storb, MD, and colleagues at Fred Hutchinson Cancer Research Center.

Read more about our Transplant Program.

New Treatments

With the discovery of specific gene mutations in MPN, medications were designed to inhibit the abnormal proteins related to these mutations. 

  • The drug imatinib (Gleevec) was developed because it can inhibit the abnormal BCR-ABL protein in chronic myeloid leukemia cells, and it has produced a very high rate of remission (no detectable disease). 
  • A drug called ruxolitinib (Jakafi) is a JAK1/JAK2 inhibitor, and it is used to treat intermediate-to-high risk myelofibrosis (including primary myelofibrosis and myelofibrosis related to polycthemia vera or essential thrombocythemia).

Clinical Studies

Many patients with MPN receive treatment through clinical studies because these offer a way to get the best and latest treatments. Clinical studies also help improve care for all patients. Your doctor can identify studies that match your circumstances and talk with you about whether and how to participate. If you are eligible for a study, we strongly recommend you consider this option for yourself. Read more about participating in a study.

References
Leukemia & Lymphoma Society
MPN Research Foundation
National Cancer Institute