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Chronic Myelogenous Leukemia Treatment (PDQ®)

Chronic myelogenous leukemia is a disease in which the bone marrow makes too many white blood cells.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children.

Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:

  • Red blood cells that carry oxygen and other materials to all tissues of the body.
  • Platelets that help prevent bleeding by causing blood clots to form.
  • Granulocytes (white blood cells) that fight infection and disease

In CML, too many blood stem cells develop into a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They may also be called leukemic cells. The leukemic cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur.

This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia:

  • Adult Acute Lymphoblastic Leukemia Treatment
  • Childhood Acute Lymphoblastic Leukemia Treatment
  • Adult Acute Myeloid Leukemia Treatment
  • Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment
  • Chronic Lymphocytic Leukemia Treatment
  • Hairy Cell Leukemia Treatment
Possible signs of chronic myelogenous leukemia include tiredness, night sweats, and fever.

These and other symptoms may be caused by CML. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:

  • Feeling very tired.
  • Weight loss for no known reason.
  • Night sweats.
  • Fever.
  • Pain or a feeling of fullness below the ribs on the left side.

Sometimes CML does not cause any symptoms at all.

Most people with CML have a gene mutation (change) called the Philadelphia chromosome.

Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making an enzyme, called tyrosine kinase, that causes too many stem cells to develop into white blood cells (granulocytes or blasts).

The Philadelphia chromosome is not passed from parent to child.

Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myelogenous leukemia.

The following tests and procedures may be used:

  • Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease such as an enlarged spleen. A history of the patient’s health habits and past illnesses and treatments will also be taken.
  • Complete blood count (CBC): A procedure in which a sample of blood is drawn and checked for the following:
    • The number of red blood cells, white blood cells, and platelets.
    • The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
    • The portion of the sample made up of red blood cells.

  • Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it.
  • Cytogenetic analysis: A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes, such as the Philadelphia chromosome.
  • Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells.
Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis (chance of recovery) and treatment options depend on the following:

  • The patient’s age.
  • The phase of CML.
  • The amount of blasts in the blood or bone marrow.
  • The size of the spleen at diagnosis.
  • The patient’s general health.
After chronic myelogenous leukemia has been diagnosed, tests are done to find out if the cancer has spread.

Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myelogenous leukemia (CML). Instead, the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The following tests and procedures may be used to find out the phase:

  • Cytogenetic analysis: A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes, such as the Philadelphia chromosome.
  • Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells.
Chronic myelogenous leukemia has 3 phases.

As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of symptoms determine the phase of the disease.

Chronic phase

In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells.

Accelerated phase

In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells.

Blastic phase

In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis.

In relapsed CML, the number of blast cells increases after a remission.

There are different types of treatment for patients with chronic myelogenous leukemia.

Different types of treatment are available for patients with chronic myelogenous leukemia (CML). 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 for patients with cancer. 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 cancer treatment is a decision that ideally involves the patient, family, and health care team.

Six types of standard treatment are used:
Tyrosine kinase inhibitor therapy

A drug called imatinib mesylate is used as initial treatment for certain types of chronic myelogenous leukemia in newly diagnosed patients. It blocks an enzyme called tyrosine kinase that causes stem cells to develop into more white blood cells (granulocytes or blasts) than the body needs. Another tyrosine kinase inhibitor called dasatinib is used to treat patients with certain types of CML that have progressed, and is being studied as an initial 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 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 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.

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.

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.

Donor lymphocyte infusion (DLI)

Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them.

Surgery

Splenectomy is surgery to remove the spleen.

New types of treatment are being tested in clinical trials.

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

Treatment of chronic phase chronic myelogenous leukemia may include the following:

  • Drug therapy with a tyrosine kinase inhibitor.
  • High-dose chemotherapy with donor stem cell transplant.
  • Biologic therapy (interferon) with or without chemotherapy.
  • Chemotherapy.
  • Splenectomy.
  • A clinical trial of lower-dose chemotherapy with donor stem cell transplant.
  • A clinical trial of a new treatment.

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.

Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with chronic phase chronic myelogenous leukemia.

Treatment of accelerated phase chronic myelogenous leukemia may include the following:

  • Stem cell transplant.
  • Drug therapy with imatinib mesylate.
  • Biologic therapy (interferon) with or without chemotherapy.
  • High-dose chemotherapy.
  • Chemotherapy.
  • Transfusion therapy to replace red blood cells, platelets, and sometimes white blood cells, to relieve symptoms and improve quality of life.
  • A clinical trial of a new treatment.

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

Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with accelerated phase chronic myelogenous leukemia.

Treatment of blastic phase chronic myelogenous leukemia may include the following:

  • Drug therapy with imatinib mesylate.
  • Chemotherapy using one or more drugs.
  • High-dose chemotherapy.
  • Donor stem cell transplant.
  • Chemotherapy as palliative therapy to relieve symptoms and improve quality of life.
  • A clinical trial of a new treatment.

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

Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with blastic phase chronic myelogenous leukemia.

Treatment of relapsed chronic myelogenous leukemia may include the following:

  • Drug therapy with a tyrosine kinase inhibitor.
  • Donor stem cell transplant.
  • Donor lymphocyte infusion.
  • Biologic therapy (interferon).
  • A clinical trial of biologic therapy, combination chemotherapy, or other drug therapy.

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.

Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with relapsing chronic myelogenous leukemia.

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PDQ is the National Cancer Institute's (NCI's) comprehensive cancer information database. Most of the information contained in PDQ is available online at NCI's Web site. PDQ is provided as a service of the NCI. The NCI is part of the National Institutes of Health, the federal government's focal point for biomedical research.

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PDQ also contains information on clinical trials.

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." 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.

Listings of clinical trials are included in PDQ and are available online at NCI's Web site. Descriptions of the trials are available in health professional and patient versions. Many cancer doctors who take part in clinical trials are also listed in PDQ. For more information, call the Cancer Information Service 1-800-4-CANCER (1-800-422-6237); TTY at 1-800-332-8615.

2008-02-22