National Cancer Institute
Posted Date: Jul 19, 2005
- Treatment Option Overview
Myeloproliferative disorders are diseases in which too many of certain types of blood cells are made in the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. The bone marrow makes red blood cells (which carry oxygen to all the tissues in the body), white blood cells (which fight infection), and platelets (which make the blood clot). (Refer to the PDQ® summary on Chronic Myeloproliferative Disorders Treatment for more information.)
Myelodysplastic syndromes, also called preleukemia or smoldering leukemia, are diseases in which the bone marrow does not function normally and not enough normal blood cells are made. Normally, bone marrow cells called blasts develop (mature) into several different types of blood cells that have specific jobs in the body. (Refer to the PDQ® summary on Myelodysplastic Syndromes Treatment for more information.)
- Chronic myelomonocytic leukemia:
Chronic myelomonocytic leukemia is a disease in which too many myelomonocytes (a type of white blood cell that has properties of both a that has properties of both a monocyte and a and a granulocyte) are in the ) are in the bone marrow, crowding out other normal , crowding out other normal blood cells, such as , such as red blood cells and and platelets. .
- Juvenile myelomonocytic leukemia:
Juvenile myelomonocytic leukemia is a disease in which too many myelomonocytes are in the bone marrow, crowding out other normal , crowding out other normal blood cells. This form of . This form of leukemia is very rare, has unique features that separate it from is very rare, has unique features that separate it from chronic myelomonocytic leukemia, and usually occurs only in very young children. Children , and usually occurs only in very young children. Children diagnosed with with neurofibromatosis 1 are at an increased risk for developing juvenile myelomonocytic leukemia. are at an increased risk for developing juvenile myelomonocytic leukemia.
- Atypical chronic myeloid leukemia:
Atypical chronic myeloid leukemia is a disease in which too many immature bone marrow cells develop into a type of develop into a type of white blood cell called called granulocytes. Some of these bone marrow cells never become mature white blood cells. These are called Some of these bone marrow cells never become mature white blood cells. These are called blasts. Over time, the granulocytes and blasts crowd out the . Over time, the granulocytes and blasts crowd out the red blood cells and and platelets in the bone marrow. in the bone marrow.
- Myelodysplastic/myeloproliferative disease, unclassifiable (MDS/MPD-U):
MDS/MPD-U are diseases of the bone marrow or or blood cells that have similar features or presentations of that have similar features or presentations of myelodysplastic syndromes or or myeloproliferative disorders but do not meet the criteria of the major types of MDS/MPD. but do not meet the criteria of the major types of MDS/MPD.
If there are symptoms, a doctor will order blood tests to count the numbers of each of the different types of cells in the blood. If the results of the tests are not normal, more blood tests may be done. The doctor may also do a bone marrow biopsy. During this test, a needle is inserted into a bone to take out some of the marrow. The marrow is then looked at under a microscope. The doctor can then tell what kind of disease the patient has and plan the best treatment.
There is no staging for these diseases. Treatment depends on the type of myelodysplastic/myeloproliferative disease the patient has.
There are treatments for all patients with myelodysplastic/myeloproliferative diseases. Usually the diseases cannot be cured, but the symptoms can be controlled and the number of blood cells can be reduced with treatment.
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 in the spinal column, a body cavity such as the abdomen, or an organ, the drugs mainly affect cancer cells in those areas. The way the chemotherapy is given depends on the type and stage of the cancer being treated.
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 bone marrow or peripheral blood stem cell transplantation:
A method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood of the patient or a donor and are frozen for storage. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. Over a short time, these reinfused stem cells grow into (and restore) the body's blood cells.
Treatment for myelodysplastic/myeloproliferative diseases depends on the type of myeloproliferative disorder, whether the patient has symptoms or not, and the patient's age and overall health.
Standard treatment may be considered because of its effectiveness in patients in past studies, or participation in a clinical trial may be considered. Most patients with myeloproliferative disorders are not cured with standard therapy and some standard treatments may have more side effects than are desired. For these reasons, clinical trials are designed to find better ways to treat cancer patients and are based on the most up-to-date information. Clinical trials are ongoing in most parts of the country for the myelodysplastic/myeloproliferative diseases. To learn more about clinical trials, call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237); TTY at 1-800-332-8615.
- Bone marrow or peripheral stem cell transplantation.
- Chemotherapy to lower the number of platelets in the blood.
- Biologic therapy.
- Bone marrow or peripheral stem cell transplantation with or without chemotherapy.
- Chemotherapy to lower the number of platelets in the blood.
- Biologic therapy.
Editorial changes were made to this summary and links to the NCI Dictionary of Cancer Terms were added..
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PDQ® also contains information on clinical trials.
Before starting treatment, patients may want to think about taking part in a clinical trial. 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."
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.
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