Myeloproliferative neoplasms

Treatment

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.

Symptom A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. Some examples of symptoms are headache, fatigue, nausea and pain. Treatment plan A detailed plan with information about a patient’s disease, the goal of treatment, the treatment options for the disease and the possible side effects and expected length of treatment. A detailed plan with information about a patient’s disease, the goal of treatment, the treatment options for the disease and the possible side effects and expected length of treatment. A treatment plan may also include information about how much the treatment is likely to cost and about regular follow-up care after treatment ends.

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.

Watchful waiting Closely watching a patient’s condition but not giving treatment unless symptoms appear or change. During watchful waiting, patients may be given certain tests and exams. Closely watching a patient’s condition but not giving treatment unless symptoms appear or change. Watchful waiting is sometimes used in conditions that progress slowly. It is also used when the risks of treatment are greater than the possible benefits. During watchful waiting, patients may be given certain tests and exams. Watchful waiting is sometimes used in prostate cancer. It is a type of expectant management.

Treatment types

Treatment looks different for different people depending on your diagnosis. We tailor your treatment plan to you. Learn more about the treatment types offered at Fred Hutchinson Cancer Center.

Treatment plan A detailed plan with information about a patient’s disease, the goal of treatment, the treatment options for the disease and the possible side effects and expected length of treatment. A detailed plan with information about a patient’s disease, the goal of treatment, the treatment options for the disease and the possible side effects and expected length of treatment. A treatment plan may also include information about how much the treatment is likely to cost and about regular follow-up care after treatment ends.

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.

Red blood cell A type of blood cell that carries oxygen in the body. Red blood cell A type of blood cell that carries oxygen in the body. Symptom A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. Some examples of symptoms are headache, fatigue, nausea and pain.

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.

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.

Bone marrow The soft, spongy material in the center of your bones that produces all your blood cells, such as white blood cells, red blood cells and platelets. Chemotherapy Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. It may be given alone or with other treatments. Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy may be given by mouth, injection, infusion or on the skin, depending on the type and stage of the cancer being treated. It may be given alone or with other treatments, such as surgery, radiation therapy or biologic therapy. Interferon A natural substance that helps the body’s immune system fight infection and other diseases, such as cancer. Interferons are made in the body but can also be made in the laboratory to use as treatments. A natural substance that helps the body’s immune system fight infection and other diseases, such as cancer. Interferons are made in the body by white blood cells and other cells, but they can also be made in the laboratory to use as treatments for different diseases. In cancer therapy, interferons may help keep cancer cells from growing and may help kill cancer cells. There are three main types of interferons: interferon-alfa, interferon-beta and interferon-gamma. An interferon is a type of cytokine and a type of immunomodulating agent. Platelet A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets help form blood clots to slow or stop bleeding and to help wounds heal. A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets are pieces of very large cells in the bone marrow called megakaryocytes. They help form blood clots to slow or stop bleeding and to help wounds heal. Having too many or too few platelets, or having platelets that do not work as they should, can cause problems. Checking the number of platelets in the blood may help diagnose certain diseases or conditions. Platelet A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets help form blood clots to slow or stop bleeding and to help wounds heal. A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets are pieces of very large cells in the bone marrow called megakaryocytes. They help form blood clots to slow or stop bleeding and to help wounds heal. Having too many or too few platelets, or having platelets that do not work as they should, can cause problems. Checking the number of platelets in the blood may help diagnose certain diseases or conditions. Red blood cell A type of blood cell that carries oxygen in the body.

Cell-reducing medications

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.

Platelet A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets help form blood clots to slow or stop bleeding and to help wounds heal. A tiny, disc-shaped piece of a cell that is found in the blood and spleen. Platelets are pieces of very large cells in the bone marrow called megakaryocytes. They help form blood clots to slow or stop bleeding and to help wounds heal. Having too many or too few platelets, or having platelets that do not work as they should, can cause problems. Checking the number of platelets in the blood may help diagnose certain diseases or conditions. Red blood cell A type of blood cell that carries oxygen in the body. Symptom A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. A physical or mental problem that a person experiences that may indicate a disease or condition. Symptoms cannot be seen and do not show up on medical tests. Some examples of symptoms are headache, fatigue, nausea and pain.

Supportive care

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.

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.

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 Hutch.

Transplantation

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).

Gene The functional and physical unit of heredity passed from parent to offspring. Genes are pieces of DNA, and most genes contain the information for making a specific protein. Remission A decrease in, or disappearance of, signs and symptoms of cancer. A decrease in, or disappearance of, signs and symptoms of cancer. In partial remission, some (but not all) signs and symptoms of cancer have disappeared. In complete remission, all signs and symptoms of cancer have disappeared, although cancer still may be in the body.

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