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Myelodysplastic/Myeloproliferative Diseases: Treatment

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:


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:



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:


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:


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

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



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 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:


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

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



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:


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:



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.