Myelodysplastic/Myeloproliferative Diseases

Summary Type: Treatment
Summary Audience: Patients
Summary Language: English
Summary Description: Expert-reviewed information summary about the treatment of myelodysplastic/myeloproliferative diseases including chronic/juvenile myelomonocytic leukemias and atypical CML.


Myelodysplastic/Myeloproliferative Diseases

General Information About Myelodysplastic/Myeloproliferative Diseases

Myelodysplastic/myeloproliferative diseases are a group of diseases in which the bone marrow makes too many white blood cells.

Myelodysplastic /myeloproliferative diseases are diseases of the blood and bone marrow . Normally, the bone marrow makes stem cells (immature cells ) that become mature blood cells. There are 3 types of mature blood cells:

Myelodysplastic/myeloproliferative diseases have features of both myelodysplastic syndromes and myeloproliferative disorders.

In myelodysplastic diseases , the 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 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
There are different types of myelodysplastic/myeloproliferative diseases.

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

When a myelodysplastic/myeloproliferative disease does not match any of these types, it is called myelodysplastic/myeloproliferative disease, unclassifiable.

Myelodysplastic/myeloproliferative diseases may progress to acute leukemia .

Tests that examine the blood and bone marrow are used to detect (find) and diagnose myelodysplastic/myeloproliferative diseases.

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

Chronic myelomonocytic leukemia is a disease in which too many myelocytes and monocytes (immature white blood cells) are made in the bone marrow.

In CMML , the body tells too many bone marrow stem cells to develop into two types of white blood cells called myelocytes and monocytes. Some of these bone marrow stem cells never become mature white blood cells. These immature white blood cells, called blasts , are unable to do their usual work. 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.

Older age and being male increase the risk of developing chronic myelomonocytic leukemia.

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 .
Possible signs of chronic myelomonocytic leukemia include fever, feeling very tired, and weight loss.

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.
Certain factors affect prognosis (chance of recovery) and treatment options.

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 is a childhood disease in which too many myelocytes and monocytes (immature white blood cells) are made in the bone marrow.

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 bone marrow stem cells to develop into two types of white blood cells called myelocytes and monocytes. Some of these bone marrow stem cells never become mature white blood cells. These immature white blood cells, called blasts , are unable to do their usual work. 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.

Possible signs of juvenile myelomonocytic leukemia include fever, feeling very tired, and weight loss.

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.
Certain factors affect prognosis (chance of recovery) and treatment options.

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

Atypical chronic myelogenous leukemia is a disease in which too many granulocytes (immature white blood cells) are made in the bone marrow.

In atypical chronic myelogenous leukemia (aCML), the body tells too many bone marrow stem cells to develop into a type of white blood cell called granulocyte . Some of these bone marrow 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 leukemia cells 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.

Possible signs of atypical chronic myelogenous leukemia include easy bruising or bleeding and feeling tired and weak.

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.
Certain factors affect prognosis (chance of recovery).

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

Myelodysplastic/Myeloproliferative Disease, Unclassifiable

Myelodysplastic/myeloproliferative disease, unclassifiable, is a disease that has features of both myelodysplastic and myeloproliferative diseases but is not chronic myelomonocytic leukemia, juvenile myelomonocytic leukemia, or atypical chronic myelogenous leukemia.

In myelodysplastic /myeloproliferative disease, unclassifiable (MDS/MPD-U), the body tells too many bone marrow stem cells to develop into red blood cells , white blood cells , or platelets . Some of these bone marrow 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-U is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known.

Possible signs of chronic myelomonocytic leukemia include fever, feeling very tired, and weight loss.

These and other symptoms may be caused by MDS/MPD-U. 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.

Treatment Option Overview

There are different types of treatment for patients with myelodysplastic/myeloproliferative diseases.

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 . 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.

Four types of standard treatment are used:
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. Combination chemotherapy is treatment using more than one anticancer drug.

Other drug therapy

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

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

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

New types of treatment are being tested in clinical trials. These include the following:
Targeted therapy

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 .

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

Treatment Options for Myelodysplastic/Myeloproliferative Diseases

Chronic Myelomonocytic Leukemia

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

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

Juvenile Myelomonocytic Leukemia

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

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

Atypical Chronic Myelogenous Leukemia

Treatment of atypical chronic myelogenous leukemia (aCML) may include chemotherapy . Information about ongoing clinical trials is available from the NCI Web site.

Myelodysplastic/Myeloproliferative Disease, Unclassifiable

Because myelodysplastic /myeloproliferative disease, unclassifiable (MDS/MPD-U) 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 .

Changes to This Summary (06/19/2006)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

Editorial changes were made to this summary.

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PDQ is a comprehensive cancer database available on NCI's Web site.

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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The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries are available in two versions. The health professional versions provide detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions provide current and accurate cancer information.

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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 new treatments, the risks involved, and how well they do or do not work. 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.

2005-10-072006-06-19