TREATMENT

Aplastic anemia treatment options.

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 (all discussed further below) 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.

Supportive care — blood transfusions, growth factors, infection prevention

Patients with severe or very severe aplastic anemia will need supportive care. Supportive care such as blood transfusions, growth factors and infection prevention are not a cure. However, supportive care can manage symptoms until other treatments can begin or take effect.

Some people may also choose supportive care as their only treatment. People who are older or who have other health problems may be unable to tolerate stronger treatment. Other people weigh the possible risks and benefits of different treatment options and choose supportive care. Supportive care does not offer the possibility of curing or controlling severe aplastic anemia for the long-term, but it may provide a person with a higher quality of life.

Blood transfusions

People with severe aplastic anemia usually need blood transfusions.
· Platelet transfusions reduce the risk of life-threatening bleeding caused by very low numbers of platelets.
· Red blood cell transfusions reduce problems with being very tired and short of breath.

Transfusions are an important treatment to manage the symptoms of aplastic anemia. If you have severe aplastic anemia, your doctor will determine when you need transfusions and manage the possible risks. For patients with severe aplastic anemia, doctors try to give as few transfusions as possible to:

· Limit the amount of iron that builds up in the body from red blood cell transfusions. Large amounts of iron in the body cause organ damage. Patients who need many red blood cell transfusions may receive additional treatment to remove iron from the body (iron chelation therapy).
· Reduce risks the immune system will develop antibodies (immune cells) that attack transfused platelets.
· Prepare for the possibility of a transplant. For patients treated with transplant, having many transfusions increases transplant risks.

In addition, it is common to treat the blood with radiation and to filter the white blood cells out of the blood before it is given in a transfusion. These steps help reduce the risks of an immune system reaction against transfused platelets and risks of a potential transplant, as well as risks of other possible complications.
If there is any chance a patient may receive a transplant, the doctor will avoid giving the patient blood donated by a family member. If that family member is later found to be a suitable donor for a transplant, the patient's immune system may react against the transplant.

Growth factors

Growth factors are drugs that help the body make more blood cells. A person with aplastic anemia may be given growth factors to try to reduce the need for red blood cell transfusions. However, in many cases of aplastic anemia, the marrow does not respond to growth factors.

Growth factors may also be given after treatment with immunosuppressive therapy or transplant. In this case, growth factors often are effective. They can help speed up new blood cell production, reducing a person's need for transfusions and reducing infection risks.