Aplastic Anemia

Also known as Bone Marrow Failure
Aplastic anemia is a rare but serious blood disorder that occurs when your bone marrow cannot make enough new blood cells for your body to work normally.
Overview

Aplastic anemia occurs because of damage to stem cells inside bone marrow, which is the sponge-like tissue within your bones. Many diseases and conditions can damage the stem cells in bone marrow. As a result, the bone marrow makes fewer red blood cells, white blood cells, and platelets.

The most common cause of bone marrow damage is from your immune system attacking and destroying the stem cells in your bone marrow, which is a kind of autoimmune disorder. The genes you inherit from your parents, some medicines, and certain toxins in the environment may also cause aplastic anemia.

Aplastic anemia can develop suddenly or slowly, and it can be mild or severe. Signs and symptoms of aplastic anemia include fatigue, infections that last a long time, and easy bruising or bleeding. The low levels of blood cells also increase your risk for complications such as bleeding, leukemia, or other serious blood disorders. Without treatment, aplastic anemia can lead to serious medical conditions such as arrhythmia and heart failure. To diagnose aplastic anemia, your doctor will order tests to determine whether you have low numbers of cells in your bone marrow and blood.

Treatments may include medicines to suppress your immune system, blood transfusions, or a blood and bone marrow transplant. A blood and bone marrow transplant may cure the disorder in some people. Removing a known cause of aplastic anemia, such as exposure to a toxin, may also cure the condition. Because people who have aplastic anemia are more likely to develop blood disorders, your doctor will monitor your condition and screen you for blood disorders regularly. If you take medicine to suppress your immune system, you will also need to take steps to prevent infection and the flu, including taking medicine and getting vaccines such as an annual flu shot.

Visit Aplastic Anemia for more information about this topic.

Participate in NHLBI Clinical Trials

We lead or sponsor many studies on aplastic anemia. See whether you or someone you know is eligible to participate in our clinical trials.

Are you considering a blood stem cell transplant?

This study is investigating a new method for collecting blood stem cells from donors to see if it reduces transplant complications, such as rejection, in patients who have blood diseases. To participate in this study, you must have a well-matched donor; you and your donor must be between 4 and 80 years old; and you must not be a candidate for immunosuppressive therapy. This study is located in Bethesda, Maryland.

Do you or a family member have Diamond-Blackfan anemia?

This registry seeks to collect information from patients who have Diamond-Blackfan anemia (DBA), in order to help researchers and doctors learn more about the condition. The registry will also help connect patients with DBA to research and clinical studies, and possible treatment options. The registry accepts all patients diagnosed with DBA.
View more information about Diamond-Blackfan Anemia Registry (DBAR).

Have you or your child been diagnosed with aplastic anemia but not been treated yet?

This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. Participants in this study must be 2 years or older and have severe aplastic anemia that has not yet been treated. This study is located in Bethesda, Maryland.

Do you or your child require care for a blood disorder?

This study provides training for NHLBI blood doctors in the evaluation and care of patients who have blood disorders. Participants in this study must be 2 years or older. This study is located in Bethesda, Maryland.

Are you younger than 55 and in need of a blood and bone marrow transplant?

This study is investigating the use of lower doses of radiation and chemotherapy before a blood and bone marrow transplant from a related, half-match donor. To participate in this study, you must be younger than 55, have severe aplastic anemia, and have no suitably matched related or unrelated donors. This study is located in Nashville, Tennessee, and Seattle, Washington.

Have you received a stem cell transplant?

This study aims to monitor the long-term health of patients who have received a donor stem cell transplant from the NIH Clinical Center. To participate in this study, you must be between 10 and 80 years old and had your transplant more than 3 years ago. This study is located in Bethesda, Maryland.
Some people who have aplastic anemia have very short telomeres, which protect the ends of DNA in chromosomes. This study is testing whether low doses of the medicine danazol help prevent telomeres from getting shorter and reduce signs of damage from aplastic anemia or related conditions. Participants in this study must be 3 years or older and have a telomere disease and signs of aplastic anemia, lung disease such as pulmonary fibrosis, or liver disease. This study is located in Bethesda, Maryland.

Do you lack a well-matched donor for a blood and bone marrow transplant?

This study aims to determine whether a transplant of umbilical cord blood from an unrelated donor or a blood and bone marrow transplant from a partial match donor are suitable alternatives for people who do not have a well-matched donor. To participate in this study, you must be younger than 75, have severe aplastic anemia, have tried immunosuppressants without success, and not have a well-matched donor. This study is located in 28 cities across the United States.

Would you like to participate in studies on blood disorders?

This study is collecting medical information from health exams and routine tests and procedures to see whether participants may be able to enroll in other studies on blood disorders. Participants in this study must be 8 years or older with or without a blood disorder. This study is located in Bethesda, Maryland.

Are you or your child taking cyclosporine for aplastic anemia?

This study is testing whether the medicine sirolimus helps prevent the return of aplastic anemia after stopping cyclosporine. Participants in this study must be 2 years or older and have severe aplastic anemia. This study is located in Bethesda, Maryland.

Are you interested in new approaches to cord blood stem cell transplants?

This study is investigating whether a blood stem cell transplant using a mix of umbilical cord blood from an unrelated donor and blood cells from a related, half-match donor is better than a single-donor cord blood transplant. To participate in this study, you must have severe aplastic anemia or myelodysplastic syndrome, not have a well-matched donor, and be between 4 and 55 years of age. This study is located in Bethesda, Maryland.

Are you scheduled for a stem cell transplant with cord blood?

This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.
More Information

After reading our Aplastic Anemia Health Topic, you may be interested in additional information found in the following resources.

Non-NHLBI resources

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