Article: Myelodysplastic Syndromes Treatment


What are myelodysplastic syndromes?

Myelodysplastic syndromes, also called pre-leukemia or “smoldering” leukemia, are diseases in which the bone marrow does not function normally and not enough normal blood cells are made. The bone marrow is the spongy tissue inside the large bones in the body. The bone marrow makes red blood cells (which carry oxygen and other materials to all tissues of the body), white blood cells (which fight infection), and platelets (which make the blood clot). Normally, bone marrow cells called blasts develop (mature) into several different types of blood cells that have specific jobs in the body.

Myelodysplastic syndromes occur most often in older people, but they can occur in younger people. The most common sign is anemia, which means there are too few mature red blood cells to carry oxygen. There may also be too few white blood cells in the blood to fight infections. If the number of platelets in the blood is lower than normal, this may cause people to bleed or bruise more easily. A doctor should be seen if a person bleeds without any reason, bruises more easily than normal, has an infection that won't go away, or feels tired all the time.

If there are symptoms, a doctor may order blood tests to count the number of each kind of blood cell. If the results of the blood test are not normal, the doctor may do a bone marrow biopsy. During this test, a needle is inserted into a bone and a small amount of bone marrow is taken out and looked at under the microscope. The doctor can then determine the kind of disease and plan the best treatment.

A myelodysplastic syndrome may develop following treatment with drugs or radiation therapy for other diseases, or it may develop without any known cause. The myelodysplastic syndromes may change into acute myeloid leukemia, a form of cancer in which too many white blood cells are made.

Myelodysplastic syndromes are grouped together based on how the bone marrow cells and blood cells look under a microscope. There are five types of myelodysplastic syndromes: refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia.

Stage Explanation

Stages of myelodysplastic syndromes

There is no staging for the myelodysplastic syndromes. Treatment depends on whether or not the disease developed following other treatments, or whether the patient has been treated for the myelodysplastic syndrome. Myelodysplastic syndromes are grouped as follows:

De novo myelodysplastic syndromes

De novo myelodysplastic syndromes develop without any known cause. The patient has not received radiation therapy or chemotherapy for other diseases.

Secondary myelodysplastic syndromes

Secondary myelodysplastic syndromes develop following treatment with radiation therapy or chemotherapy for other diseases.

Previously treated myelodysplastic syndromes

Previously treated myelodysplastic syndrome means the disease has been treated but has gotten worse.

Treatment Option Overview

How myelodysplastic syndromes are treated

There are treatments for all patients with myelodysplastic syndromes. Often the main treatment is giving red blood cells or platelets by a needle in a vein (transfusion) to control anemia or bleeding. Vitamins or other drugs may also be given to treat anemia.

Chemotherapy and biological therapy are being tested in clinical trials. Chemotherapy uses drugs to treat disease. Chemotherapy may be taken by pill, or it may be put into the body by a needle in the vein or muscle. Chemotherapy is called a systemic treatment because the drug enters the bloodstream, travels through the body, and affects cells throughout the body. Biological therapy tries to get the body to fight disease. It uses materials made by the body or made in a laboratory to boost, direct, or restore the body's natural defenses against disease. Biological therapy is sometimes called biological response modifier (BRM) therapy or immunotherapy.

Bone marrow transplantation is a newer type of treatment that uses high doses of chemotherapy and/or radiation therapy (high doses of x-rays or other high-energy rays) to destroy all of the bone marrow in the body, then transplants healthy bone marrow back into the body. Healthy marrow is then taken from another person (a donor) whose tissue is the same or almost the same as the patient's. The donor may be a twin (the best match), a brother, sister, or other relative, or another person not related. The healthy marrow is given to the patient through a needle in the vein, and the marrow replaces the marrow that was destroyed. A bone marrow transplant using marrow from a relative or person not related to the patient is called an allogeneic bone marrow transplant.

Treatment by stage

The choice of treatment depends on the type of myelodysplastic syndrome, and the patient's age and general 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 myelodysplastic syndromes 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 patients with myelodysplastic syndromes. 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.

De Novo Myelodysplastic Syndrome

Treatment may be one of the following:

  1. Treatment to relieve symptoms of the disease, such as anemia or bleeding.
  2. Allogeneic bone marrow transplantation.
  3. Clinical trials of chemotherapy or biological therapy.

Secondary Myelodysplastic Syndrome

Patients will probably receive treatment to relieve symptoms of the disease, such as anemia or bleeding. They may also choose to take part in a clinical trial of chemotherapy or biological therapy.

Previously Treated Myelodysplastic Syndrome

Patients will probably receive treatment to relieve symptoms of the disease, such as anemia or bleeding. They may also choose to take part in a clinical trial of chemotherapy or biological therapy.

Changes to This Summary (06/06/2003)

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.

To Learn More


For more information, U.S. residents may call the National Cancer Institute's (NCI's) Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 9:00 a.m. to 4:30 p.m. Deaf and hard-of-hearing callers with TTY equipment may call 1-800-332-8615. The call is free and a trained Cancer Information Specialist is available to answer your questions.

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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 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.

Source: National Cancer Institute
Cache Date: December 10, 2004