|NCI/PDQ® Patients: Chronic Myeloproliferative Disorders Treatment (PDQ®)|
|National Cancer Institute|
| Last Modified: September 21, 2012
Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:
In myeloproliferative disorders too many blood stem cells become one or more types of blood cells. The disorders usually get worse slowly as the number of extra blood cells increases.
The type of myeloproliferative disorder is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative disorders include the following 6 types:
Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ® summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment.
In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis.
Polycythemia vera often does not cause early symptoms. It is sometimes found during a routine blood test. Symptoms may occur as the number of blood cells increases. Other conditions may cause the same symptoms. Check with your doctor if you have any of the following problems:
In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made).
The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue's ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells.
Primary myelofibrosis often does not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following problems:
Essential thrombocythemia often does not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by essential thrombocytopenia or by other conditions. Check with your doctor if you have any of the following problems:
Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack.
Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia.
Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia.
Chronic eosinophilic leukemia may not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following problems:
Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myeloproliferative disorders. Treatment is based on the type of myeloproliferative disorder the patient has. It is important to know the type in order to plan treatment.
The new (metastatic) tumor is the same type of cancer as the primary cancer. For example, if leukemia cells spread to the brain, the cancer cells in the brain are actually leukemia cells. The disease is metastatic leukemia, not brain cancer.
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. 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. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.
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 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.
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them 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 cerebrospinal fluid, 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.
See Drugs Approved for Myeloproliferative Disorders for more information.
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. 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.
See Drugs Approved for Myeloproliferative Disorders for more information.
Biologic therapy is a treatment that uses the patient's immune system to fight cancer or other diseases. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against disease. This type of treatment is also called biotherapy or immunotherapy. Interferon alfa and pegylated interferon alpha are biologic agents commonly used to treat some chronic myeloproliferative disorders.
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.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI Web site.
Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors are targeted therapy drugs that block signals needed for tumors to grow. JAK2 tyrosine kinase inhibitors are targeted therapy drugs being studied in clinical trials to treat primary myelofibrosis.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.
A link to a list of current clinical trials is included for each treatment section. For some types or stages of cancer, there may not be any trials listed. Check with your doctor for clinical trials that are not listed here but may be right for you.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with polycythemia vera. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with primary myelofibrosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
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:
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with essential thrombocythemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Treatment of chronic neutrophilic leukemia may include the following:
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with chronic neutrophilic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Treatment of chronic eosinophilic leukemia may include the following:
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with chronic eosinophilic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
For more information from the National Cancer Institute about chronic myeloproliferative disorders, see the following:
For general cancer information and other resources from the National Cancer Institute, see the following:
Editorial changes were made to this summary.
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