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Aplastic anemia

Aplastic anemia is a bone marrow failure syndrome characterized by peripheral pancytopenia and marrow hypoplasia. Paul Ehrlich, MD, introduced the concept of aplastic anemia in 1888 when he studied the case of a pregnant woman who died of bone marrow failure.

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Aplastic 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 symptoms by providing blood cells that your bone marrow isn't producing.

Transfusions of red blood cells raise red blood cell counts. This helps relieve anemia and fatigue. Transfusions of platelets help prevent bleeding symptoms.

Red blood cells are easiest to replace by transfusion. There are only four major blood types, so matching blood types between donor and recipient is usually easy, and transfused cells may remain in the body for a month or longer.

Platelets are collected from a donor through a process called hemapheresis. Blood is drawn from a vein in a donor's arm and is circulated through a blood-separating machine that removes platelets before returning the blood to the donor. The platelets removed are then given to the person with aplastic anemia.

Unfortunately, white blood cells can't be easily transfused.

There's no limit necessarily to the number of blood cell transfusions you can have. However, some complications can arise with multiple transfusions.

Red blood cell transfusions contain iron that accumulates in the body and can damage vital organs if not treated. Over time, your body may develop antibodies to transfused blood cells, making them less effective at relieving symptoms. Blood transfusions may contain viruses or other infections that get passed on to the recipient. However, the blood supply today is safer than it has ever been.

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 for treatment. You don't want the infection to become severe, which could prove life-threatening. If you have severe aplastic anemia, your doctor may give you antibiotics to help prevent infections.

Immune-suppressing drugs
Aplastic anemia may be due to an autoimmune disorder that's causing your body's immune system to attack and damage cells in your bone marrow. To prevent this from continuing, doctors sometimes treat aplastic anemia with drugs that alter or suppress the immune system.

Drugs such as cyclosporine (Neoral, Sandimmune) and anti-thymocyte globulin (Thymoglobulin) are examples. These drugs, given separately or in combination, suppress the activity of immune cells that are damaging your bone marrow. This helps your bone marrow recover and generate new blood cells.

Corticosteroids, such as methylprednisolone (Medrol, Solu-Medrol), are often given at the same time as these drugs to lessen their side effects.

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 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.

Bone marrow transplantation from a well-matched donor can treat aplastic anemia — without recurrence — in about 80 percent of younger people and in about 40 percent to 70 percent of older people. However, this procedure carries risk. There's a chance that the 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 and may begin generating new blood cells in about two 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 of bone marrow transplantation. Additionally, doctors avoid transfusions from relatives because such transfusions also can increase the risk of complications of bone marrow transplantation.

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 disease relapses when they stop taking the drugs — for safety reasons — during pregnancy.

If all treatments fail, aplastic anemia can be fatal. Fortunately, new therapies are continually being developed to treat aplastic anemia of all causes. Talk to your doctor about what experimental treatments may be available. Treatments under study include:

  • Growth factors. These drugs, such as granulocyte-macrophage colony-stimulating factor (GM-CSF), granulocyte colony-stimulating factor (G-CSF) and cytokines, may help stimulate the bone marrow to produce new blood cells — especially white blood cells. Growth factors may work well in combination with each other or with immune-suppressing drugs to relieve the signs and symptoms of aplastic anemia. However, the safety of long-term use of growth factors is unknown.

  • Male hormones. Researchers are investigating a synthetic version of the male hormone androgen as a treatment for aplastic anemia. The drug, which also stimulates blood cell production, may offer some benefit to people with mild to moderate aplastic anemia.

  • Peripheral stem cell transplants. Stem cells are taken from the blood of a donor, rather than from their own bone marrow, in this procedure. Research is still being done to see how effective this treatment may be for people with aplastic anemia.

  • Autologous transplant. For this type of transplant, which is considered experimental, stem cells are retrieved from the bone marrow of people with aplastic anemia. The stem cells then undergo chemotherapy to destroy the unhealthy cells. The remaining cells are then reimplanted.


Most cases of aplastic anemia can't be prevented. However, avoiding exposure to insecticides, herbicides, organic solvents, paint removers and other toxic chemicals may lower your risk of the disease. This is especially important if you've already had aplastic anemia. Exposure to the same compound a second time may cause the disease to return.


If you have aplastic anemia, it's important to:

  • Avoid excessive exercise. Anemia causes fatigue and shortness of breath with even mild exertion. It may be best to conserve your energy. Long-term anemia can be hard on your heart, as your heart pumps more blood to compensate for a lack of oxygen in the blood.

  • Avoid contact sports. Because of the risk of bleeding associated with a low platelet count, avoid activities that may result in a major cut or fall.

  • Avoid infections. Protect yourself from infections by frequent hand washing and by avoiding sick people. If you develop a fever or other indicators of an infection, see your doctor for treatment.

Coping skills

As with any serious and chronic disease, you may experience many emotional side effects living with aplastic anemia. Tips to help you and your family better cope with your illness include:

  • Research your disease. The more you know, the better prepared you'll be to make treatment decisions.

  • Ask questions. Be sure to ask your doctor about anything related to your disease or treatment that you don't understand. It may help you to tape-record or write down what your doctor tells you.

  • Be vocal. Don't be afraid to express any concerns you have to your doctor or any other health care professional treating you.

  • Seek support. Ask family and friends for emotional support. Ask them to consider becoming blood donors or bone marrow donors. Consider joining an aplastic anemia support group. It may be helpful to talk to others coping with the disease.

  • Take care of yourself. Proper nutrition and sleep are important to optimize blood production.

Complementary and alternative medicine

Herbal treatments and vitamins aren't effective treatment for aplastic anemia and may actually worsen the condition. Talk to your doctor before trying any such therapy.


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