Aplastic anemia impacts about 3 out of every 1 million people in the United States each year. The disease appears more often in eastern Asian countries, where it affects about 15 out of every 1 million people. Aplastic anemia is a condition where bone marrow does not produce sufficient new cells to replenish blood cells: it stops making enough red blood cells, white blood cells and platelets for the body. Any blood cells the marrow does make are normal, but there are not enough of them. Aplastic anemia can be moderate, severe or very severe. People with severe or very severe aplastic anemia are at risk for life-threatening infections or bleeding. Aplastic anemia leaves you feeling fatigued and at higher risk of infections and uncontrolled bleeding. A rare and serious condition, aplastic anemia can develop at any age, though it’s most common in younger people. Treatment for aplastic anemia may include medications, blood transfusions or a bone marrow transplant. Once considered nearly always fatal, aplastic anemia has a much better prognosis today, thanks to advances in treatment.

In most cases, doctors do not know the cause of aplastic anemia. Certain inherited conditions (about 20% of cases) can damage the stem cells and lead to aplastic anemia. Examples include Fanconi anemia, Shwachman-Diamond syndrome, dyskeratosis congenita, and Diamond-Blackfan anemia.. Aplastic anemia may also be caused by high doses of radiation or certain chemicals or viruses. There is evidence that in many people aplastic anemia is an autoimmune disease.


The severity of a person’s symptoms depends on the number of blood cells he or she has.

  • Low numbers of red blood cells can cause a person to feel tired or weak, be short of breath and look pale.
  • Low numbers of white blood cells can lead to frequent or severe infections.
  • Low numbers of platelets can lead to easy bleeding or bruising and tiny red spots under the skin (petechiae), or bleeding that is hard to stop.

For a person with severe or very severe aplastic anemia, infections or bleeding can be life-threatening.


Blood tests. Normally, red blood cell, white blood cell and platelet levels stay within a certain range. Your doctor may suspect aplastic anemia when all three of these blood cell levels are very low. Many conditions can cause low blood cell counts, but usually of just one type of blood cell. For example, other types of anemia cause a decrease in red blood cells. If you have an infection, your white blood cell count alone may be low.

Bone marrow biopsy. To confirm a diagnosis, you’ll need to undergo a bone marrow biopsy. In this procedure, a doctor uses a needle to remove a small sample of bone marrow from a large bone in your body, such as your hipbone. There are two types of samples that can be collected from the bone marrow: A bone marrow aspiration uses a needle to extract a small amount of liquid marrow. A bone marrow biopsy uses a special needle to remove an intact core of marrow. The bone marrow sample is examined under a microscope to rule out other blood-related diseases. In aplastic anemia, bone marrow contains fewer blood cells than normal. The few cells that are present, however, are normal. In certain diseases such as leukemia and myelodysplastic syndrome, the bone marrow is full of abnormal blood cells.

Once you’ve received a diagnosis of aplastic anemia, you may need additional tests to determine an underlying cause.

National Heart Lung and Blood Institute
National Marrow Donor Program

Diagnosed at Birth with Aplastic Anemia
Titus Chang: Cord Blood Stem Cell Transfusion Saves 3-Year Old

Treatment options for aplastic anemia depend on how severe the disease is. A person with moderate aplastic anemia may not need any treatment. Instead, doctors will check blood counts regularly to watch for signs the disease is getting worse. A person with severe or very severe aplastic anemia needs treatment as soon as possible. For severe and very severe aplastic anemia, standard treatment options include:

  • Supportive care to keep a patient stable until other treatment can begin or take effect.
  • Immunosuppressive therapy — drugs that suppress (weaken) the immune system.
  • Bone marrow or cord blood transplant (also called BMT).

All patients with severe aplastic anemia will need supportive care, but the only treatments that can cure the disease or manage it for the long-term are a transplant or immunosuppressive therapy. A transplant is the preferred standard treatment for many patients, while immunosuppressive therapy is the first choice for others. Research into treatment of aplastic anemia is ongoing, so other, newer treatment options may also be available. Whichever treatment you and your doctor decide on, you may choose to be part of a clinical trial. Even standard treatments continue to be studied in clinical trials. These studies help doctors improve treatments so that more patients can have better results.

Cord Blood Registry

Umbilical cord blood is one of three sources for the blood-forming cells used in transplants. The other two sources are bone marrow and peripheral (circulating) blood. The first cord blood transplant was done in 1988. Cord blood plays an important role in transplant today. Doctors are still learning about the ways cord blood transplants are similar to and different from marrow or peripheral blood transplants.

Umbilical cord blood is collected from the umbilical cord and placenta after a baby is born. This blood is rich in blood-forming cells. The donated cord blood is tested, frozen and stored at a cord blood bank for future use. The stored cord blood is called a cord blood unit.

Stem Cell Harvest
(Picture from Advanced Cell Technology, via Scientific American.)

The Be The Match Registry, operated by the National Marrow Donor Program (NMDP), includes nearly 90,000 cord blood units. Doctors search the registry of adult marrow or peripheral blood cell donors and cord blood units to find a suitable HLA match for their patients who need a transplant. If selected, the matching cord blood is transplanted to a patient. The transplant process is the same as for marrow and peripheral blood cell transplants.

Hope is at Hand for Man Living With Severe Aplastic Anemia

James Ilari’s Website on Aplastic Anemia

~ by marbleroad on April 30, 2009.

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