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Treatments and drugs
By Mayo Clinic staffAplastic anemia can be mild, moderate or severe. Severe aplastic anemia, in which your blood cell counts are extremely low, is life-threatening and requires immediate hospitalization for treatment. Mild or moderate aplastic anemia is still serious, but usually doesn't require hospitalization to treat.
Treatments for aplastic anemia may include observation for mild cases, blood transfusions, medications and, in severe cases, bone marrow transplantation.
Blood transfusions
Most people with aplastic anemia require multiple blood transfusions — transfusions of red blood cells or platelets, or both. Blood transfusions aren't a cure for aplastic anemia. But they do relieve signs and symptoms by providing blood cells that your bone marrow isn't producing.
- Red blood cells. Transfusions of red blood cells raise red blood cell counts. This helps relieve anemia and fatigue. Red blood cells are easiest to replace by transfusion.
- Platelets. Transfusions of platelets help prevent excessive bleeding.
- White blood cells. Because they're so short-lived, white blood cells can't be easily transfused.
While there's generally no limit to the number of blood cell transfusions you can have, complications can sometimes arise with multiple transfusions. Transfused red blood cells contain iron that can accumulate in your body and can damage vital organs if an iron overload isn't treated. Another possible complication is that over time, your body may develop antibodies to transfused blood cells, making them less effective at relieving symptoms.
Bone marrow transplantation
Bone marrow transplantation — replacing diseased bone marrow with healthy bone marrow from a donor — may offer the only successful treatment option for people with severe aplastic anemia. This is generally the treatment of choice for people who are younger and have a matching sibling donor.
Bone marrow transplantation from a related, matched donor can treat aplastic anemia — without recurrence — in about four out of five cases. Up to about half the people with matched, but unrelated, donors can expect to have successful bone marrow transplants.
This procedure carries risk. There's a chance that your body may reject the transplant, leading to life-threatening complications. In addition, not everyone is a candidate for transplantation or can find a suitable donor.
If a donor is found, the diseased bone marrow in the person with aplastic anemia is first depleted with radiation or chemotherapy. Healthy bone marrow from the donor is extracted through a surgical technique. The healthy marrow is injected intravenously into the bloodstream of the person with aplastic anemia, where it migrates to the bone marrow cavities and may begin generating new blood cells in about three to four weeks. The procedure requires a lengthy hospital stay. After the transplant, you'll receive drugs to help prevent rejection of the donated marrow.
If no suitable bone marrow donor is available, treatment of severe aplastic anemia is with immune-suppressing drugs. If you're a candidate for bone marrow transplantation, your doctor may limit the number of blood transfusions you have before the procedure because they can increase the risks associated with bone marrow transplantation.
Immunosuppressants
Aplastic anemia may be due to an autoimmune disorder that's causing your immune system to attack and damage cells in your bone marrow. To prevent this from continuing, doctors may treat aplastic anemia with drugs that alter or suppress your immune system.
Drugs such as cyclosporine (Gengraf, Neoral, Sandimmune) and anti-thymocyte globulin (Thymoglobulin) are examples. These drugs suppress the activity of immune cells that are damaging your bone marrow. This helps your bone marrow recover and generate new blood cells. Cyclosporine and anti-thymocyte globulin are often used in combination. This option is usually the treatment of choice for older people with aplastic anemia and for those without a matching donor for bone marrow transplant or those with additional health conditions.
Corticosteroids, such as methylprednisolone (Medrol, Solu-Medrol), are often given at the same time as these drugs.
Immune-suppressing drugs can be very effective at treating aplastic anemia. The downside is that these drugs further weaken your immune system. It's also possible that after you stop taking these drugs, aplastic anemia may return.
Bone marrow stimulants
Certain drugs — including granulocyte-macrophage colony-stimulating factor, such as sargramostim (Leukine); granulocyte colony-stimulating factor, such as filgrastim (Neupogen); and epoetin alfa (Epogen, Procrit) — may help stimulate the bone marrow to produce new blood cells. Growth factors aren't considered effective on their own, but may be beneficial when used in combination with immune-suppressing drugs.
Antibiotics, antivirals
Having aplastic anemia weakens your immune system. You have fewer white blood cells in circulation to fight off germs. This leaves you susceptible to all kinds of infections — everything from colds to more-serious illnesses.
At the first sign of infection, such as a fever, see your doctor. You don't want the infection to become severe, because it could prove life-threatening. If you have severe aplastic anemia, your doctor may give you antibiotics or antiviral medications to help prevent infections.
Other treatments
Aplastic anemia caused by radiation and chemotherapy treatments for cancer usually improves once you complete those treatments. The same is true for most other drugs that induce aplastic anemia. You usually can expect a full recovery once you stop taking the drug.
Some cases of pregnancy-related aplastic anemia improve once the pregnancy ends. If that doesn't happen, treatment is still necessary. Some women who have aplastic anemia that responds to immune-suppressing drugs before pregnancy may find the disorder returns when they stop taking the drugs — for safety reasons — during pregnancy.
If all treatments fail, aplastic anemia can be fatal. Ask your doctor about what experimental treatments may be available if standard treatments aren't working.
- Aplastic anemia. National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/health/dci/Diseases/aplastic/aplastic_all.html. Accessed Feb. 5, 2009.
- Aplastic anemia. The Merck Manuals: The Merck Manual for Healthcare Professionals. http://www.merck.com/mmpe/sec11/ch130/ch130f.html#sec11-ch130-ch130e-91. Accessed Feb. 5, 2009.
- Young NS. Aplastic anemia. Current Opinion in Hematology. 2008;15:162.
- Schrier SL. Aplastic anemia: Pathogenesis; clinical manifestations; and diagnosis. http://www.uptodate.com/home/index.html. Accessed Feb. 5, 2009.
- Schrier SL. Aplastic anemia: Prognosis and treatment. http://www.uptodate.com/home/index.html. Accessed Feb. 5, 2009.
- Shih RD. Plants, mushrooms, and herbal medications. In: Marx JA, et al. Rosen's Emergency Medicine: Concepts and Clinical Practice. 6th ed. Philadelphia, Pa.: Mosby Elsevier; 2006. http://www.mdconsult.com/das/book/body/119296292-3/801134565/1365/515.html#4-u1.0-B0-323-02845-4..50167-0--cesec32_8318. Accessed Feb. 6, 2009.
