How Is Aplastic Anemia Treated?

Treatments for aplastic anemia include:

• Blood transfusions
• Bone marrow transplants
• Several medicines used to suppress the immune system, stimulate the bone marrow, and prevent and treat infections

In rare cases, no treatment is needed.

In general, treatments for aplastic anemia can limit and prevent complications, relieve symptoms, and improve quality of life. For some, a cure may be possible. Bone marrow transplants can be a cure for those people who are eligible for a transplant. Removing a known cause of aplastic anemia (such as exposure to a toxic chemical) also can cure the disorder. Other treatments can restore blood cell counts to levels high enough so that a person can live a normal life.

Who Needs Treatment

People with mild or moderate aplastic anemia may not need treatment as long as the condition does not get worse. People with severe aplastic anemia need immediate medical treatment to prevent complications from the very low levels of blood cells in their bodies. People with very severe aplastic anemia need emergency medical care and hospitalization or the anemia can be fatal in a short time.

Blood Transfusions

Blood transfusions are used to keep blood counts high enough in people with aplastic anemia. Healthy blood is taken from matched donors and then given to the person with aplastic anemia through an injection in a vein. Blood transfusions help relieve symptoms. They are not an effective long-term treatment.

Red Blood Cells

Red blood cell transfusions help relieve lack of energy, tiredness, and shortness of breath. However, if red blood cells are given often, the immune system in the body can learn to recognize these new cells, develop antibodies against them, and destroy these newly transfused cells. Also, if red blood cells are given often, the body can build up too much iron. Excess iron from transfusions can damage the heart, liver, and other organs. If this happens, treatment may be needed to remove excess iron from the body.

White Blood Cells

White blood cells are not routinely transfused because they live in the bloodstream for less than 1 day. However, they may be used for severe infections that are not being helped by antibiotics.

Platelets

Platelet transfusions reduce the risk of fatal bleeding. Several transfusions a month are often needed because platelets live only a few days. However, in time, the immune system will learn to recognize and destroy newly transfused platelets by making antibodies, just as it does with red blood cells.

Bone Marrow Transplantation

A bone marrow transplant replaces damaged stem cells in bone marrow with healthy stem cells from a donor’s bone marrow. It is the best treatment choice for eligible people because it usually cures aplastic anemia.

A bone marrow transplant works best in children and young adults with severe aplastic anemia who are in good health and who have matched donors. Older people are less able to tolerate the treatments needed to prepare the body for the transplant. They also are more likely to have complications after the transplant. There are age limits for who can receive a bone marrow transplant, but they vary among hospitals.

Finding a Donor

For best results, the donor’s bone marrow must be a close match with the bone marrow of the person with aplastic anemia. A matched donor is found by means of a blood test that looks at proteins on the surface of body cells. These proteins are called human leukocyte antigens (HLA). The donor and the person with aplastic anemia are both tested. The HLA antigens on their cells are compared.

Bone marrow transplants work best if the donated marrow is an exact match and comes from a family member, such as a brother or sister. A person receiving bone marrow that is an exact match usually has fewer complications. Only about 20–35 percent of people with severe aplastic anemia have an HLA-matched family member to donate bone marrow.

If no matching family member is found, the search for bone marrow widens. Millions of volunteer donors are registered with the National Marrow Donor Program. Doctors will look for:

• Donors who are HLA matches but are not family members
• Family members who are not exact HLA matches
• Unrelated donors who are not exact HLA matches
• Umbilical cord blood that is an HLA match

A person who is going to have a bone marrow transplant should not receive blood transfusions from close family members before the transplant. When a person receives blood transfusions, their bodies can develop antibodies against the donated blood. If a person develops antibodies against the blood of a close family member, the person would not be able to receive a bone marrow transplant from that family member.

Receiving the Transplant

Bone marrow transplants are done in special units in hospitals to try to protect the person with aplastic anemia from any infections. Before the transplant, the person receives high doses of chemotherapy, radiation therapy, or both. These treatments destroy the abnormal bone marrow that is not producing blood cells.

A sample of bone marrow is taken from the donor and is given to the recipient through a vein. The donated bone marrow stem cells travel to the recipient’s bone marrow and start to make healthy blood cells. If the transplant is successful, the new stem cells multiply and begin making new, healthy blood cells within 3 to 4 weeks.

Possible Complications

After a bone marrow transplant, the recipient is in the hospital for weeks or months. During this time, he or she is isolated and closely watched for infections and other complications. Complications are most likely to occur in the first 100 days after a transplant. Complications include rejection of the graft and what is called graft-versus-host disease (GVHD).

Graft rejection. Sometimes, the recipient’s immune system destroys the new bone marrow cells. This occurs in 5 to 10 percent of recipients, and it is more likely to happen in people who have had several bone marrow transplants.

GVHD. GVHD happens when the new immune system cells, created by the donated bone marrow, attack the recipient’s body. Signs and symptoms are skin inflammation, diarrhea, and liver disease. GVHD can occur soon after transplant, or it can develop slowly and can last for months or years. GVHD most often happens in older people and those who receive unmatched transplants.

To help prevent GVHD, donor marrow is sometimes treated or people may be given medicines that suppress the immune system after the transplant. Those who develop mild but acute GVHD are treated with medicines such as steroids.

Medicines To Stimulate Bone Marrow

Manmade versions of substances that occur naturally in the body are used to stimulate the bone marrow to produce more blood cells. Bone marrow that can produce more blood cells can help a person with aplastic anemia avoid blood transfusions. An increased number of white cells can help protect a person from infection. Examples of bone marrow stimulation medicines used to treat aplastic anemia are:

• Erythropoietin (e-RITH-ro-PO-e-tin)
• Colony-stimulating factors

These medicines have some risks. Based on your situation, you doctor will decide whether the benefits of the medicines outweigh the risks.

Medicines To Suppress the Immune System

Since research suggests that aplastic anemia may occur because the body’s immune system attacks its own cells by mistake, certain medicines may be used to suppress the body’s immune system. They allow the bone marrow to start making blood cells again, and they help avoid the need for blood transfusions. Medicines that suppress the immune system do not cure aplastic anemia, but they can relieve its symptoms and reduce its complications.

These medicines are often used for people who are not good candidates for a bone marrow transplant or who are waiting for a bone marrow transplant.

There are three medicines—often given together—that will suppress the body’s immune system:

• Antithymocyte globulin (ATG)
• Cyclosporine
• Methylprednisolone (a corticosteroid)

If this treatment is successful, it may take a few months to notice the effects of these medicines. Most often, as blood counts rise, symptoms lessen. Blood counts in people who respond well to these drugs do not usually reach normal levels, but they are often high enough to allow people to take part in their normal activities. People with aplastic anemia may need to take the medicines indefinitely to keep their blood counts high enough to resume normal daily life.

Medicines that suppress the immune system can have side effects. They also may increase the risk of developing leukemia (loo-KE-me-a) or myelodysplasia (MI-e-lo-dis-PLA-ze-a; MDS). Leukemia is a type of cancer in which the number of white blood cells increases. MDS is a condition in which the bone marrow produces too many defective blood cells.

Antibiotics

Antibiotics and antivirals are given to people with aplastic anemia to prevent and treat infections.