Treatment of Aplastic Anemia & Myelodysplastic Syndromes

How do health care professionals treat aplastic anemia?

Health care professionals treat aplastic anemia by first treating any underlying condition that might be triggering the aplastic anemia, if an underlying condition is suspected. This may include removing exposure to a chemical, stopping a certain medicine, or treating a viral infection.

Your care team may recommend one or more of the following treatment options

  • blood transfusion
  • bone marrow stem-cell transplants
  • immunosuppressive therapy
  • medicines

Treatments for aplastic anemia can ease your symptoms, improve your quality of life, and, in some cases, provide a cure for the disorder. Your treatment will depend on your age, general health, cause and severity of the disease, and availability of a stem-cell donor. Mild or moderate aplastic anemia may not need immediate treatment.

Blood transfusions

Blood transfusions can temporarily relieve symptoms such as anemia, fatigue, and bleeding by increasing the amount of red blood cells or platelets or both in your blood.

Repeat transfusions are common but can lead to other health problems.

  • Iron from transfused red blood cells can build up in the body and damage organs, called iron overload or hemochromatosis. Your health care professional can prescribe treatments that remove extra iron.
  • Over time, the body may develop antibodies that damage or destroy the donor blood cells. Your health care professional may prescribe medicines to prevent or manage this reaction.

Blood and bone marrow stem-cell transplants

Blood and bone marrow transplant, also called a stem-cell transplant, uses a donor’s healthy stem cells to replace your body’s own damaged stem cells. The donor’s cells must closely match yours for the best outcomes. Close relatives such as siblings are more likely to be close matches, but unrelated donors can also be close matches. If a matched donor isn’t found in your family, your doctor will search the National Marrow Donor Program, which has millions of potential donors.

Stem-cell transplant is the only possible cure for aplastic anemia. Talk with your health care professional about the risk and benefits of a stem-cell transplant and if the procedure is right for you.

Immunosuppressive therapy

Immunosuppressive therapy may be used for people who cannot have a stem-cell transplant or to control aplastic anemia in people who are waiting for a stem-cell transplant. Immunosuppressants, such as antithymocyte globulin (ATG) and cyclosporine, suppress your body’s immune system and slow or stop damage to your bone marrow. This is not a cure though. Your health care professional may use a medicine called eltrombopag in combination with an immunosuppressant to increase the number of blood cells in your body.

Medicines

Low white blood cell counts can make it hard for your body to fight off infections. Your care team may give you medicine to kill bacteria, fungi, or viruses that cause infection.

How do health care professionals treat MDS?

Treatment for MDS is similar to treatment for aplastic anemia. However, because most people with MDS are older adults and may have other health problems, treatment for MDS focuses on supportive care to ease your symptoms, improve your quality of life, and prevent bleeding and infections rather than curing the condition. Some people live for many years with supportive care alone.

People who have few or no symptoms may not need treatment. If you develop symptoms, have worsening symptoms, or have very low blood cell counts, treatment will be tailored to your age, general health, symptoms, and cause and severity of the disease. A stem-cell transplant may offer a chance for a cure for certain people.

Blood transfusions

Blood transfusions increase the number of healthy red blood cells or platelets in your body. You may need a transfusion occasionally or possibly as often as every week or two.

Medicines

Medicines to stimulate bone marrow. Medicines called growth factors may ease your symptoms by helping your bone marrow make more blood cells. These medicines are copies of substances found naturally in your body, such as erythropoietin and granulocyte colony-stimulating factor.

Medicines to treat or prevent infections. Fewer white blood cells make it harder for your body to fight infections. Antibiotics may be used to kill bacteria that cause infections.

Hypomethylating agents. Medicines that help prevent the growth of abnormal bone marrow stem cells and increase the number of healthy blood cells in your body may improve your quality of life and lower your chances of developing leukemia. Your health care professional may ask you to take a hypomethylating agent such as azacitidine or decitabine.

Immune modulators. Your health care professional may prescribe the immune modulator lenalidomide to slow the growth of abnormal blood cells and lessen your need for blood transfusions.

Immunosuppressive therapy

In some people with cases of MDS that are thought to be more similar to aplastic anemia, medicines that calm the body’s immune system can slow or stop damage to bone marrow. These immunosuppressant medicines, including ATG and cyclosporin, may relieve your symptoms and help you avoid blood transfusions.

Blood and bone marrow stem-cell transplants and chemotherapy

Stem-cell transplant and chemotherapy medicine can be used separately or together to treat MDS. These treatments are more aggressive and may improve blood counts more quickly than less intensive treatments, reduce the risk of death from MDS, and possibly provide a cure. But these treatments have greater risks of complications so they are used in patients in whom the risk and benefits have been clearly assessed.

For people with more advanced MDS or those at greater risk of developing acute myeloid leukemia, your care team may prescribe traditional chemotherapy medicine to help your body get rid of the abnormal cells. Traditional chemotherapy medicines include cytarabine, daunorubicin, and idarubicin. This treatment may be too toxic for some older people or those with other health problems.

A patient talking with a health care professional.
Treatments for aplastic anemia and MDS depend on how severe the disorder is, your age, and other factors. Talk with your health care professional about the treatment options that might be best for you.
July 2020
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This content is provided as a service of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health. The NIDDK translates and disseminates research findings to increase knowledge and understanding about health and disease among patients, health professionals, and the public. Content produced by the NIDDK is carefully reviewed by NIDDK scientists and other experts.