Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)

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General Information About Myelodysplastic/Myeloproliferative Neoplasms

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

Myelodysplastic/myeloproliferative neoplasms 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. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.

Myelodysplastic/myeloproliferative neoplasms have features of both myelodysplastic syndromes and myeloproliferative neoplasms.

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 become one or more types of blood cells and the total number of blood cells slowly increases.

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

There are different types of myelodysplastic/myeloproliferative neoplasms.

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

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

Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia.

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

The following tests and procedures may be used:

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 chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become 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.

Older age and being male increase the risk of 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.

Signs and symptoms of chronic myelomonocytic leukemia include fever, weight loss, and feeling very tired.

These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following:

  • 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

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 (JMML) 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 juvenile myelomonocytic leukemia.

In JMML, the body tells too many blood stem cells to become 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.

Signs and symptoms of juvenile myelomonocytic leukemia include fever, weight loss, and feeling very tired.

These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following:

  • 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 (CML), the body tells too many blood stem cells to become 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 leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there.

Signs and symptoms of atypical chronic myelogenous leukemia include easy bruising or bleeding and feeling tired and weak.

These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following:

  • 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 atypical CML depends on the number of red blood cells and platelets in the blood.

Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable

Myelodysplastic/myeloproliferative neoplasm, 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 neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become 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/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known.

Signs and symptoms of myelodysplastic/myeloproliferative neoplasm, unclassifiable, include fever, weight loss, and feeling very tired.

These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following:

  • 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 Neoplasms

There is no standard staging system for myelodysplastic/myeloproliferative neoplasms.

Staging is the process used to find out how far the cancer has spread. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. Treatment is based on the type of myelodysplastic/myeloproliferative neoplasm 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 neoplasms.

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

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

See Drugs Approved for Myeloproliferative Disorders for more information.

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.

Stem Cell TransplantStem cell transplant (Step 1). Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm.Stem cell transplant (Step 2). The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown).Stem cell transplant (Step 3). The patient receives stem cells through a catheter placed into a blood vessel in the chest.

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.

Targeted therapy

Targeted therapy is a cancer treatment that uses drugs or other substances to attack cancer cells without harming normal cells. Targeted therapy drugs called tyrosine kinase inhibitors (TKIs) are used to treat myelodysplastic/myeloproliferative neoplasm, unclassifiable. TKIs block the enzyme, tyrosine kinase, that causes stem cells to become more blood cells (blasts) than the body needs. Imatinib mesylate (Gleevec) is a TKI that may be used. Other targeted therapy drugs are being studied in the treatment of JMML.

See Drugs Approved for Myeloproliferative Disorders for more information.

New types of treatment are being tested in clinical trials.

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

Patients may want to think about taking part in a clinical trial.

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.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

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.

Follow-up tests may be needed.

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 Neoplasms

Chronic Myelomonocytic Leukemia

Treatment of chronic myelomonocytic leukemia (CMML) 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 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. Talk with your doctor about clinical trials that may be right for you. 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:

Check for U.S. clinical trials from NCI's list of cancer clinical trials 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. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI Web site.

Atypical Chronic Myelogenous Leukemia

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

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with atypical chronic myeloid leukemia, BCR-ABL1 negative. 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. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI Web site.

Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable

Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following:

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with myelodysplastic/myeloproliferative neoplasm, 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. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI Web site.

To Learn More About Myelodysplastic/Myeloproliferative Neoplasms

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

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



Cancer Care Services

Clinical Trials