Treatments for aplastic anemia include blood transfusions, blood and marrow stem cell transplants, and medicines. These treatments can prevent or limit complications, relieve symptoms, and improve quality of life.
Blood and marrow stem cell transplants may cure the disorder in some people who are eligible for a transplant. Removing a known cause of aplastic anemia, such as exposure to a toxin, also may cure the condition.
People who have mild or moderate aplastic anemia may not need treatment as long as the condition doesn't get worse. People who have severe aplastic anemia need medical treatment right away to prevent complications.
People who have very severe aplastic anemia need emergency medical care in a hospital. Very severe aplastic anemia can be fatal if it's not treated right away.
Blood transfusions can help keep blood cell counts at acceptable levels. A blood transfusion is a common procedure in which blood is given to you through an intravenous (IV) line in one of your blood vessels.
Transfusions require careful matching of donated blood with the recipient's blood.
Blood transfusions help relieve the symptoms of aplastic anemia, but they're not a permanent treatment.
A blood and marrow stem cell transplant replaces damaged stem cells with healthy ones from another person (a donor).
During the transplant, which is like a blood transfusion, you get donated stem cells through a tube placed in a vein in your chest. Once the stem cells are in your body, they travel to your bone marrow and begin making new blood cells.
Blood and marrow stem cell transplants may cure aplastic anemia in people who can have this type of treatment. The transplant works best in children and young adults with severe aplastic anemia who are in good health and who have matched donors.
Older people may be less able to handle the treatments needed to prepare the body for the transplant. They're also more likely to have complications after the transplant.
If you have aplastic anemia, talk with your doctor about whether a blood and marrow stem cell transplant is an option for you.
If you have aplastic anemia, your doctor may prescribe medicines to:
Man-made versions of substances that occur naturally in the body can stimulate the bone marrow to make more blood cells. Examples of these types of medicines include erythropoietin and colony-stimulating factors.
These medicines have some risks. You and your doctor will work together to decide whether the benefits of these medicines outweigh the risks. If this treatment works well, it can help you avoid the need for blood transfusions.
Research suggests that aplastic anemia may sometimes occur because the body's immune system attacks its own cells by mistake. For this reason, your doctor may prescribe medicines to suppress your immune system.
These medicines allow your bone marrow to start making blood cells again. They also may help you avoid the need for blood transfusions.
Medicines that suppress the immune system don't cure aplastic anemia. However, they can relieve its symptoms and reduce complications. These medicines often are used for people who can't have blood and marrow stem cell transplants or who are waiting for transplants.
Three medicines—often given together—can suppress the body's immune system. They are antithymocyte globulin (ATG), cyclosporine, and methylprednisolone.
It may take a few months to notice the effects of these medicines. Most often, as blood cell counts rise, symptoms lessen. Blood cell counts in people who respond well to these medicines usually don't reach normal levels. However, the blood cell counts often are high enough to allow people to do their normal activities.
People who have aplastic anemia may need long-term treatment with these medicines.
Medicines that suppress the immune system can have side effects. They also may increase the risk of developing leukemia (lu-KE-me-ah) or myelodysplasia (MI-e-lo-dis-PLA-ze-ah; MDS). Leukemia is a cancer of the blood cells. MDS is a condition in which the bone marrow makes too many faulty blood cells.
If you have aplastic anemia, you might be at risk for infections due to low white blood cell counts. Your doctor may prescribe antibiotic and antiviral medicines to prevent and treat infections.
Clinical Trials for Rare Blood Diseases (Neal Young, M.D.)
Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. To find clinical trials that are currently underway for Aplastic Anemia, visit www.clinicaltrials.gov.
July 4, 2012
NIH Media Availability: Platelet drug shows clinical benefits for severe, unresponsive aplastic anemia
Eltrombopag, a drug that was designed to stimulate production of platelets from the bone marrow and thereby improve blood clotting, can raise blood cell levels in some people with severe aplastic anemia who have failed all standard therapies.
The NHLBI updates Health Topics articles on a biennial cycle based on a thorough review of research findings and new literature. The articles also are updated as needed if important new research is published. The date on each Health Topics article reflects when the content was originally posted or last revised.