Key Points for This Section

There are different types of treatment for patients with myelodysplastic syndromes. Treatment for myelodysplastic syndromes aims to relieve symptoms, slow progression, and improve quality of life. Three types of standard treatment are used: Chemotherapy Supportive care Chemotherapy with stem cell transplant New types of treatment are being tested in clinical trials. Patients may want to think about taking part in a clinical trial. Patients can enter clinical trials before, during, or after starting their treatment. Follow-up tests may be needed.

There are different types of treatment for patients with myelodysplastic syndromes.

Different types of treatments are available for patients with myelodysplastic syndromes. 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 a myelodysplastic syndrome. 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.

Treatment for myelodysplastic syndromes aims to relieve symptoms, slow progression, and improve quality of life.

Treatment options for patients with myelodysplastic syndromes range from supportive care that helps relieve symptoms to aggressive treatment that may slow or prevent progression of the disease.

Problems caused by low blood cell counts, such as fatigue and infections, may be treated with transfusions of blood products or the use of growth factors.

Chemotherapy may be used to delay progression of the disease. Other drug therapy may be used to lessen the need for transfusions. Certain patients may benefit from aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.

Three types of standard treatment are used:

Chemotherapy

In myelodysplastic syndromes, chemotherapy is a treatment that uses drugs to stop the growth of immature blood 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 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 disease being treated.

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

• Transfusion therapy

  Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or treatment. Patients who receive frequent red blood cell transfusions may have their tissues and organs damaged from the buildup of extra iron. Iron chelation therapy is a treatment that uses drugs that attach to the extra iron. The drug and the iron are removed from the body in the urine.

  Platelet transfusions are usually given when the patient is bleeding or is having a procedure that may cause bleeding.




• Growth factor therapy

  Erythropoietin may be given to increase the number of red blood cells and lessen the effects of anemia. Sometimes granulocyte colony-stimulating factor (G-CSF) is given with erythropoietin to help the treatment work better.




• Drug therapy

  Deferoxamine may be used to treat the build-up of too much iron in the blood of patients receiving blood transfusions. It is sometimes given with vitamin C.

  Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndrome caused by a specific chromosome change.

  Antithymocyte globulin (ATG) may also be used to lessen the need for transfusions in patients with a certain form of myelodysplastic syndrome.

  Antibiotics may be given to fight infections.

Chemotherapy with stem cell transplant

Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of 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. These reinfused stem cells grow into (and restore) the body's blood cells.

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI Web site ¹.

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 medical research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for disease 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 diseases 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 treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose disease has not gotten better. There are also clinical trials that test new ways to stop a disease from recurring (coming back) or reduce the side effects of 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 clinical trials database.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the disease 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.

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 disease has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

Glossary Terms

The area of the body that contains the pancreas, stomach, intestines, liver, gallbladder, and other organs.

A quickly growing cancer.

A condition in which the number of red blood cells is below normal.

A drug used to treat infections caused by bacteria and other microorganisms.

A protein used to reduce the risk of or to treat graft-versus-host disease.

A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.

A test to check the number of red blood cells, white blood cells, and platelets in a sample of blood. Also called complete blood count and CBC.

The administration of blood or blood products into a blood vessel.

The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.

A hollow area or hole. It may describe a body cavity (such as the space within the abdomen) or a hole in a tooth caused by decay.

The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.

Treatment with drugs that kill cancer cells.

Part of a cell that contains genetic information. Except for sperm and eggs, all human cells contain 46 chromosomes.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called a clinical study.

An iron-chelating agent that removes iron from tumors by inhibiting DNA synthesis and causing cancer cell death. It is used in conjunction with other anticancer agents in pediatric neuroblastoma therapy.

The process of identifying a disease, such as cancer, from its signs and symptoms.

Any substance, other than food, that is used to prevent, diagnose, treat or relieve symptoms of a disease or abnormal condition. Also refers to a substance that alters mood or body function, or that can be habit-forming or addictive, especially a narcotic.

A substance that is naturally produced by the kidneys, and that stimulates the bone marrow to make red blood cells. When erythropoietin is made in the laboratory, it is called epoetin alfa or epoetin beta.

A condition marked by extreme tiredness and inability to function due lack of energy. Fatigue may be acute or chronic.

G-CSF. A colony-stimulating factor that stimulates the production of neutrophils (a type of white blood cell). It is a cytokine that is a type of hematopoietic (blood-forming) agent. Also called G-CSF and filgrastim.

A substance made by the body that functions to regulate cell division and cell survival. Some growth factors are also produced in the laboratory and used in biological therapy.

Invasion and multiplication of germs in the body. Infections can occur in any part of the body and can spread throughout the body. The germs may be bacteria, viruses, yeast, or fungi. They can cause a fever and other problems, depending on where the infection occurs. When the body’s natural defense system is strong, it can often fight the germs and prevent infection. Some cancer treatments can weaken the natural defense system.

A method of putting fluids, including drugs, into the bloodstream. Also called intravenous infusion.

Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."

An important mineral the body needs to make hemoglobin, a substance in the blood that carries oxygen from the lungs to tissues throughout the body. Iron is also an important part of many other proteins and enzymes needed by the body for normal growth and development. It is found in red meat, fish, poultry, lentils, beans, and foods with iron added, such as cereal.

A drug that is similar to thalidomide, and is used to treat multiple myeloma and certain types of anemia. It is also being studied in the treatment of other types of cancer. Lenalidomide belongs to the family of drugs called angiogenesis inhibitors. Also called CC-5013 and Revlimid.

A group of diseases in which the bone marrow does not make enough healthy blood cells. Also called preleukemia and smoldering leukemia.

A part of the body that performs a specific function. For example, the heart is an organ.

A type of blood cell that helps prevent bleeding by causing blood clots to form. Also called a thrombocyte.

In medicine, action taken to decrease the chance of getting a disease or condition. For example, cancer prevention includes avoiding risk factors (such as smoking, obesity, lack of exercise, and radiation exposure) and increasing protective factors (such as getting regular physical activity, staying at a healthy weight, and having a healthy diet).

In medicine, the course of a disease, such as cancer, as it becomes worse or spreads in the body.

To come back or to return.

RBC. A cell that carries oxygen to all parts of the body. Also called RBC and erythrocyte.

Treatment with anticancer drugs directed to a specific area of the body.

A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

The bones, muscles, tendons, and other tissues that reach from the base of the skull to the tailbone. The spinal column encloses the spinal cord and the fluid surrounding the spinal cord. Also called spine, backbone, and vertebral column.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with standard therapy. Also called standard of care or best practice.

A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.

A method of replacing immature blood-forming cells that were destroyed by cancer treatment. The stem cells are given to the person after treatment to help the bone marrow recover and continue producing healthy blood cells.

Care given to improve the quality of life of patients who have a serious or life-threatening disease. The goal of supportive care is to prevent or treat as early as possible the symptoms of a disease, side effects caused by treatment of a disease, and psychological, social, and spiritual problems related to a disease or its treatment. Also called palliative care, comfort care, and symptom management.

An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.

Treatment with anticancer drugs that travel through the blood to cells all over the body.

Treatment.

A group or layer of cells that work together to perform a specific function.

The infusion of components of blood or whole blood into the bloodstream. The blood may be donated from another person, or it may have been taken from the person earlier and stored until needed.

Fluid containing water and waste products. Urine is made by the kidneys, stored in the bladder, and leaves the body through the urethra.

A blood vessel that carries blood to the heart from tissues and organs in the body.

A key nutrient that the body needs to fight infection, heal wounds, and keep tissues healthy, including the blood vessels, cartilage, ligaments, tendons, bones, muscle, skin, teeth, and gums. It is an antioxidant that helps prevent tissue damage caused by free radicals. The body does not make or store vitamin C, so it must be taken in every day. It is found in many fruits and vegetables, especially green peppers, citrus, strawberries, tomatoes, broccoli, leafy greens, potatoes, and cantaloupe. Also called ascorbic acid.

Refers to a blood cell that does not contain hemoglobin. White blood cells include lymphocytes, neutrophils, eosinophils, macrophages, and mast cells. These cells are made by bone marrow and help the body fight infections and other diseases. Also called WBC.