How Is Aplastic Anemia Treated?

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.

In some cases, a cure may be possible. Blood and marrow stem cell transplants may cure the disorder in 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.

Who Needs Treatment

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

People who have aplastic anemia may need blood transfusions to keep their 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.

Blood and Marrow Stem Cell Transplants

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 often cure aplastic anemia in people who are eligible for this type of transplant. 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 to your doctor about whether a bone and marrow stem cell transplant is an option for you.

Medicines

If you have aplastic anemia, your doctor may prescribe medicines to:

• Stimulate your bone marrow
• Suppress your immune system
• Prevent and treat infections

Medicines To Stimulate Bone Marrow

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 (e-RITH-ro-PO-e-tin) 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.

Medicines To Suppress the Immune System

Research suggests that some cases of aplastic anemia may 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 can allow your bone marrow to start making blood cells again. These medicines 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 its complications.

These medicines often are used for people who aren’t eligible for a bone and marrow stem cell transplant or who are waiting for a transplant.

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-a; MDS). Leukemia is a cancer of the blood cells. MDS is a condition in which the bone marrow makes too many faulty blood cells.

Medicines To Prevent and Treat Infections

People who have aplastic anemia may be at risk for infections due to a low number of white blood cells. Your doctor may prescribe antibiotic and antiviral medicines to prevent and treat infections.