Chronic Myeloproliferative Disorders: Treatment Option Overview

by National Cancer Institute

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There are different types of treatment for patients with chronic myeloproliferative disorders.

Different types of treatments are available for patients with chronic myeloproliferative disorders. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Before starting treatment, patients may want to think about taking part in a clinical trial. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI Web site. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team.

Ten types of standard treatment are used:

Watchful waiting

Watchful waiting is closely monitoring a patient's condition without giving any treatment until symptoms appear or change.

Phlebotomy

Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative disorders.

Platelet apheresis

Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient's bloodstream.

Transfusion therapy

Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the spinal column, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells. There are two types of radiation therapy. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated.

Radiation therapy to treat myeloproliferative disorders is usually directed at the spleen.

Other drug therapy

Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood.

Surgery

Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged.

Biologic therapy

Biologic therapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Interferon alfa is a biologic agent commonly used to treat some chronic myeloproliferative disorders.

High-dose chemotherapy with stem cell transplant

High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

New types of treatment are being tested in clinical trials.

Information about ongoing clinical trials is available from the NCI Web site.

Treatment Options for Chronic Myeloproliferative Disorders

Chronic Myelogenous Leukemia

Refer to the PDQ summary on Chronic Myelogenous Leukemia Treatment 1 for information.

Polycythemia Vera

The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following:

• Phlebotomy.
• Chemotherapy with or without phlebotomy.
• Biologic therapy using interferon alfa.
• A clinical trial of a new treatment.

Information about ongoing clinical trials is available from the NCI Web site.

Chronic Idiopathic Myelofibrosis

Treatment of chronic idiopathic myelofibrosis in patients without symptoms is usually watchful waiting.

Treatment of chronic idiopathic myelofibrosis in patients with symptoms may include the following:

• Transfusion of red blood cells to relieve symptoms and improve quality of life.
• Biologic therapy using interferon alfa.
• Splenectomy.
• Radiation therapy to the spleen.
• Chemotherapy.
• Donor stem cell transplant.
• A clinical trial of thalidomide.

This summary section refers to specific treatments under study in clinical trials, but it may not mention every new treatment being studied. Information about ongoing clinical trials is available from the NCI Web site.

Essential Thrombocythemia

Treatment of essential thrombocythemia in patients younger than 60 years who have no symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following:

• Chemotherapy.
• Anagrelide therapy.
• Biologic therapy using interferon alfa.
• Platelet apheresis.
• A clinical trial of a new treatment.

Information about ongoing clinical trials is available from the NCI Web site.

Chronic Neutrophilic Leukemia

Treatment of chronic neutrophilic leukemia may include the following:

• Donor bone marrow transplant.
• Chemotherapy.
• Biologic therapy using interferon alfa.
• A clinical trial of a new treatment.

Information about ongoing clinical trials is available from the NCI Web site.

Chronic Eosinophilic Leukemia

Treatment of chronic eosinophilic leukemia may include the following:

• Bone marrow transplant.
• Biologic therapy using interferon alfa.
• A clinical trial of a new treatment.

Information about ongoing clinical trials is available from the NCI Web site.

About the Author

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