Supportive treatment for aplastic anemia

Transfusion

While your blood counts are low, you may need transfusions of red blood cells or platelets. Although blood transfusions are generally safe, their long-term use creates problems. The main problem is that the red cells contain iron. The transfused cells only live a few weeks and iron from these cells builds up in the body. Eventually this will result in a high level of body iron, which may be toxic. Although the buildup of iron can be treated with drugs, these aren’t always successful and some organs, mainly the liver and heart, can become damaged. Also, getting transfusions before a stem cell transplant increases the chance that the transplant won’t work. This is why doctors avoid giving transfusions when a transplant is planned.

See our document Blood Transfusion and Donation to learn more about this topic.

Antibiotics

Because of your low white blood cell count, you might develop an infection and need antibiotics. Infection is the major cause of death from aplastic anemia. Treatment with antibiotics should be started as soon as infection is suspected. Transfusing white blood cells is not generally practical. They survive only a very short time, and it’s not possible to get enough white blood cells from normal donors to raise a person’s white blood cell count.

Growth factors

Drugs can be given to increase the white blood cell count. These are called filgrastim (also called G-CSF or Neupogen^®), pegfilgrastim (Neulasta^®), and sargramostim (also called GM-CSF or Leukine^®). These work only slightly for most patients with aplastic anemia.

Androgens

Some patients with early or mild aplastic anemia can be treated with androgens (instead of stem cell transplantation or ATG). Androgens are also often the first treatment given to patients with inherited forms of aplastic anemia (like Fanconi anemia and dyskeratosis congenita). Androgens are male sex hormones that also stimulate blood production. They are the reason that men have higher red blood cell counts than do women. Although they can be effective in improving blood counts, they do not cure aplastic anemia. Also, using androgens for a long time has been linked to liver tumors and liver cancer. Androgens are male hormones, so women taking this medicine can develop masculine characteristics such as facial hair, balding, deepening voice, etc.

Treatment to lower iron levels

Blood transfusions can cause excess iron to build up in the body. This extra iron can deposit in the liver and heart, causing the organs to function poorly. This buildup of iron is only seen in people who receive many transfusions over a period of years. Drugs called chelating agents (substances that bind with metal so that the body can get rid of it) can be used in patients who may develop iron overload from transfusions. The most commonly used drug is desferoxamine. This drug helps treat and prevent iron overload. This is given intravenously or as an injection under the skin. It is inconvenient because the injection must be given slowly (over several hours) 5 to 7 times per week. In some patients, treatment continues for years. Deferasirox (Exjade^®) is a newer drug that is taken by mouth once a day to treat iron overload. Although it has been used more for patients with certain congenital anemias (like thalassemia), it can also help patients with aplastic anemia and iron overload.