Myelodysplastic/Myeloproliferative Diseases Treatment (PDQ®)

Contents of This Summary

General Information About Myelodysplastic/Myeloproliferative Diseases

Myelodysplastic/myeloproliferative diseases are diseases of the blood and bone marrow. 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. 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.
• White blood cells that fight infection and disease.
• Platelets that help prevent bleeding by causing blood clots to form.

In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets.

In myeloproliferative diseases, a greater than normal number of blood stem cells develop into one or more types of blood cells and the total number of blood cells slowly increases.

This summary is about diseases that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases:

• Myelodysplastic Syndromes Treatment
• Chronic Myeloproliferative Disorders Treatment
• Chronic Myelogenous Leukemia Treatment

The 3 main types of myelodysplastic/myeloproliferative disease include the following:

• Chronic myelomonocytic leukemia (CMML).
• Juvenile myelomonocytic leukemia (JMML).
• Atypical chronic myelogenous leukemia (aCML).

When a myelodysplastic/myeloproliferative disease does not match any of these types, it is called myelodysplastic/myeloproliferative disease, unclassifiable (MDS/MPD-UC).

Myelodysplastic/myeloproliferative diseases may progress to acute 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 and liver. A history of the patient’s health habits and past illnesses and treatments will also be taken.
• Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following:
  • The number of red blood cells and platelets.
  • The number and type of white blood cells.
  • 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 produces it.
• Peripheral blood smear: A procedure in which a sample of blood is checked for the presence of blast cells, number and kinds of white blood cells, the number of platelets, and changes in the shape of blood cells.
• 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. The cancer cells in myelodysplastic/myeloproliferative diseases do not contain the Philadelphia chromosome that is present in chronic myelogenous leukemia.
• Bone marrow aspiration and biopsy: The removal of a small piece of bone and bone marrow by inserting a needle into the hipbone or breastbone. A pathologist views both the bone and bone marrow samples under a microscope to look for abnormal cells.

Chronic Myelomonocytic Leukemia

In CMML, the body tells too many bloodstem cells to develop into two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.

Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following:

• Older age.
• Being male.
• Being exposed to certain substances at work or in the environment.
• Being exposed to radiation.
• Past treatment with certain anticancer drugs.

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

• Fever for no known reason.
• Infection.
• Feeling very tired.
• Weight loss for no known reason.
• Easy bruising or bleeding.
• Pain or a feeling of fullness below the ribs.

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

• The number of white blood cells or platelets in the blood or bone marrow.
• Whether the patient is anemic.
• The amount of blasts in the blood or bone marrow.
• The amount of hemoglobin in red blood cells.
• Whether there are certain changes in the chromosomes.

Juvenile Myelomonocytic Leukemia

Juvenile myelomonocytic leukemia is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of developing juvenile myelomonocytic leukemia.

In JMML, the body tells too many bloodstem cells to develop into two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.

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

• Fever for no known reason.
• Having infections, such as bronchitis or tonsillitis.
• Feeling very tired.
• Easy bruising or bleeding.
• Skin rash.
• Painless swelling of the lymph nodes in the neck, underarm, stomach, or groin.
• Pain or a feeling of fullness below the ribs.

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

• The age of the child at diagnosis.
• The number of platelets in the blood.
• The amount of a certain type of hemoglobin in red blood cells.

Atypical Chronic Myelogenous Leukemia

In atypical chronic myelogenous leukemia (aCML), the body tells too many bloodstem cells to develop into a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow.

The leukemiacells in aCML and chronic myelogenous leukemia (CML) look alike under a microscope. However, in aCML a certain chromosome change, called the "Philadelphia chromosome" is not present.

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

• Shortness of breath.
• Pale skin.
• Feeling very tired and weak.
• Easy bruising or bleeding.
• Petechiae (flat, pinpoint spots under the skin caused by bleeding).
• Pain or a feeling of fullness below the ribs on the left side.

The prognosis (chance of recovery) for aCML depends on the number of red blood cells and platelets in the blood.

Myelodysplastic/Myeloproliferative Disease, Unclassifiable

In myelodysplastic/myeloproliferative disease, unclassifiable (MDS/MPD-UC), the body tells too many bloodstem cells to develop into red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets.

MDS/MPD-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known.

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

• Fever or frequent infections.
• Shortness of breath.
• Feeling very tired and weak.
• Pale skin.
• Easy bruising or bleeding.
• Petechiae (flat, pinpoint spots under the skin caused by bleeding).
• Pain or a feeling of fullness below the ribs.

Stages of Myelodysplastic/Myeloproliferative Diseases

Staging is the process used to find out how far the cancer has spread. There is no standard staging system for myelodysplastic/myeloproliferative diseases. Treatment is based on the type of myelodysplastic/myeloproliferative disease the patient has. It is important to know the type in order to plan treatment.

When cancer cells spread outside the blood, a solid tumor may form. This process is called metastasis. The three ways that cancer cells spread in the body are:

• Through the blood. Cancer cells travel through the blood, invade solid tissues in the body, such as the brain or heart, and form a solid tumor.
• Through the lymph system. Cancer cells invade the lymph system, travel through the lymph vessels, and form a solid tumor in other parts of the body.
• Through solid tissue. Cancer cells that have formed a solid tumor spread to tissues in the surrounding area.

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.

Treatment Option Overview

Different types of treatments are available for patients with myelodysplastic/myeloproliferative diseases. 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 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. 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.

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. Combination chemotherapy is treatment using more than one anticancer drug.

13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act.

Stem cell transplant is a method of replacing blood-forming cells that are destroyed by chemotherapy. 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.

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusiontherapy or drug therapy, such as antibiotics to fight infection.

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 cancer treatment that uses drugs or other substances to attack cancer cells without harming normal cells. Farnesyltransferase inhibitors are one type of targeted therapy that is being studied in the treatment of JMML.

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 clinical trials database.

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.

Treatment Options for Myelodysplastic/Myeloproliferative Diseases

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.

Chronic Myelomonocytic Leukemia

Treatment of chronic myelomonocytic leukemia (CMML) may include the following:

• Chemotherapy with one or more agents.
• Stem cell transplant.
• A clinical trial of a new treatment.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with chronic myelomonocytic 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.

Juvenile Myelomonocytic Leukemia

Treatment of juvenile myelomonocytic leukemia (JMML) may include the following:

• Combination chemotherapy.
• Stem cell transplant.
• 13-cis-retinoic acidtherapy.
• A clinical trial of a new treatment, such as targeted therapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with juvenile myelomonocytic 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.

Atypical Chronic Myelogenous Leukemia

Treatment of atypical chronic myelogenous leukemia (aCML) may include chemotherapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with atypical chronic myeloid 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.

Myelodysplastic/Myeloproliferative Disease, Unclassifiable

Because myelodysplastic/myeloproliferative disease, unclassifiable (MDS/MPD-UC) is a rare disease, little is known about its treatment. Supportive care treatments are used to manage problems caused by the disease such as infection, bleeding, and anemia.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with myelodysplastic/myeloproliferative disease, unclassifiable. 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.

To Learn More About Myelodysplastic/Myeloproliferative Diseases

For more information from the National Cancer Institute about myelodysplastic/myeloproliferative diseases, see the following:

• Myeloproliferative Disorders Home Page
• Understanding Cancer Series: Blood Stem Cell Transplants
• Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation: Questions and Answers

For general cancer information and other resources from the National Cancer Institute, see the following:

• What You Need to Know About™ Cancer - An Overview
• Understanding Cancer Series: Cancer
• Staging: Questions and Answers
• Chemotherapy and You: Support for People With Cancer
• Radiation Therapy and You: Support for People With Cancer
• Coping with Cancer
• Support and Resources
• Cancer Library
• Information For Survivors/Caregivers/Advocates

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