Aplastic anemia occurs because of damage to 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, , and .
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 ofdisorder. The 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. 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.and of aplastic anemia include , 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
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.
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Research for Your Health
Improving health with current research
- Aplastic Anemia
Learn about the following ways the NHLBI continues to translate current research into improved health for people with aplastic anemia. Research on this topic is part of the NHLBI’s broader commitment to advancing blood disorders and blood safety scientific discovery.
- Program Helps Protect Blood Transfusion Recipients. The NHLBI’s Recipient Epidemiology and Donor Evaluation Study (REDS) program began in 1989 to protect the Nation’s blood supply and improve the benefits and reduce the risks of transfusions. Now in its third phase, called REDS-III, the program supports research in the United States and around the world.
- Providing Access to NHLBI Biologic Specimens and Data. The Biologic Specimen and Data Repository Information Coordinating Center (BioLINCC) centralizes and integrates biospecimens and clinical data that were once stored in separate repositories. Researchers can find and request available resources on BioLINCC’s secure website, which maximizes the value of these resources and advances heart, lung, blood, and sleep research.
- Supporting Safe Manufacturing of Cell-based Therapies. The NHLBI’s Production Assistance for Cellular Therapies (PACT) program supports translational research on cellular and genetic therapies by increasing the capacity to manufacture cell products that follow current Good Manufacturing Practices (cGMP) regulations. The PACT program is designed to increase the supply and safety of genetically modified cells available for patients who have blood disorders such as aplastic anemia.
- Network Accelerates Research on Blood and Bone Marrow Transplants. The NHLBI and the National Cancer Institute (NCI) launched the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) in 2001 to promote large multi-institutional clinical trials that seek to understand the best possible treatment approaches in blood and marrow transplantation. In the United States, about 20,000 patients receive blood or marrow transplants annually.
Learn about some of the pioneering research contributions we have made over the years that have improved clinical care.
- Improving treatments for people who have aplastic anemia. NHLBI researchers pioneered the use of antithymocyte globulin (ATG) and cyclosporine, greatly increasing the survival rates of patients who have aplastic anemia. This combination treatment is now the standard of care for older patients who have aplastic anemia and those who do not have a matching bone marrow donor.
- New treatments for aplastic anemia. About one-third of people who have aplastic anemia do not respond to medicines that calm the immune system, called . NHLBI studies led to the approval of eltrombopag, which stimulates the body to make more blood cells in people who do not respond to standard immunosuppressants. Studies show that eltrombopag also helps patients who respond well to immunosuppressants.
- Understanding risk factors for aplastic anemia. NHLBI researchers have helped determine some causes of aplastic anemia, such as parvovirus B19 infection, and how in that protect the ends of your can increase the risk of getting the disease.
Advancing research for improved health
- Aplastic Anemia
In support of our mission, we are committed to advancing research on anemia, in part through the following ways.
- We perform research. Our Division of Intramural Research and its Hematology Branch include investigators who are actively engaged in research on anemia, including aplastic anemia. Research in the Hematopoiesis and Bone Marrow Failure Laboratory spans the basic sciences, , and epidemiology, and focuses on blood cell production in healthy individuals and patients with bone marrow failure.
- We fund research. The research we fund today will help improve our future health. The Division of Blood Diseases and Resources supports research on the causes, prevention, and treatment of blood diseases, including aplastic anemia. Search the NIH RePORTER to learn about research the NHLBI is funding on aplastic anemia.
- We stimulate high-impact research. Our Trans-Omics for Precision Medicine (TOPMed) Program includes participants with aplastic anemia, which may help us understand how genes contribute to differences in disease severity and how patients respond to treatment. The NHLBI Strategic Vision highlights ways we may support research over the next decade.
Learn about exciting research areas the NHLBI is exploring for aplastic anemia.
- Biomarkers to help identify aplastic anemia. Diagnosis of aplastic anemia is often challenging. NHLBI researchers identified small molecules in the blood, called microRNAs, that could serve as to help diagnose, monitor, and determine the severity of the disease.
- Improved immunosuppressive treatments for aplastic anemia. The NHLBI continues to sponsor and fund clinical trials to find new and improved combination treatments for patients who have severe aplastic anemia.
- Mutations in bone marrow stem cells in aplastic anemia. NHLBI-funded researchers are looking at mutations in the bone marrow stem cells or early blood cells of people who develop aplastic anemia to understand how the mutations affect patients’ response to treatment or risk for complications.
- New scientific approaches for Diamond-Blackfan anemia (DBA). NHLBI-supported researchers converted skin cells from patients with DBA into early-stage blood cells in order to screen for new treatments. Using these cells, the researchers identified a compound that may be developed to treat DBA, a rare type of anemia, related to aplastic anemia.
- Preventing chromosome damage in aplastic anemia. Some people who have aplastic anemia have very short telomeres, which are structures that protect DNA at the ends of chromosomes. People who have aplastic anemia and have shorter telomeres have a lower survival rate than those with longer telomeres. NHLBI-supported researchers are studying the proteins involved in maintaining telomeres to provide clues on how to reverse or prevent telomere shortening in aplastic anemia.
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.
[At NIH Clinical Center] Blood disorder research
Are you considering a blood stem cell transplant?
Do you or a family member have Diamond-Blackfan anemia?
Have you or your child been diagnosed with aplastic anemia but not been treated yet?
Do you or your child require care for a blood disorder?
Are you younger than 55 and in need of a blood and bone marrow transplant?
Have you received a stem cell transplant?
Do you have short telomeres and a telomere gene mutation?
Do you lack a well-matched donor for a blood and bone marrow transplant?
Are you or your child taking cyclosporine for aplastic anemia?
Are you interested in new approaches to cord blood stem cell transplants?
Are you scheduled for a stem cell transplant with cord blood?
After reading our Aplastic Anemia Health Topic, you may be interested in additional information found in the following resources.