Aplastic Anemia | Diagnosis & Treatment

How is aplastic anemia diagnosed?

In addition to a complete medical history and physical examination, aplastic anemia can only be diagnosed accurately by a full evaluation of the blood and bone marrow, which includes some or all of the following tests:

Blood Tests

A complete blood count (CBC) can help your child’s doctor to understand the cause of the anemia. In aplastic anemia, this test shows an abnormally low number of all blood cells.

  • Samples of the fluid (aspiration) and solid (biopsy) portions of bone marrow are withdrawn by needle under local or general anesthesia. This allows your child’s doctor to look for marrow abnormalities. This test is necessary to make a definitive diagnosis of aplastic anemia.
  • Additional blood tests and genetic testing may be ordered to rule out certain inherited types of anemia and other disorders, such as myelodysplastic syndrome.

Additional blood tests and genetic testing may be ordered to rule out certain inherited types of anemia and other disorders such as myelodysplastic syndrome.

After we complete all necessary tests, our experts meet to review and discuss what they have learned about your child's condition. Then, we meet with you and your family to discuss the results and outline the best treatment options for your child’s aplastic anemia.

How is aplastic anemia treated?

Specific treatment for aplastic anemia will be determined by your child’s physician based on your child’s age and overall health, the extent of their condition, and tolerance for specific medications and therapies.

Stem Cell (Bone Marrow) Transplant

Presently, this is the only cure for aplastic anemia. Bone marrow transplant involves the replacement of diseased bone marrow with another person’s healthy bone marrow. Unfortunately it may not be an option for everyone. The success of a transplant depends on many factors, such as how close the match is between child and donor and the patient's age. If the child has a sibling, there is a 1-in-4 chance they will be a donor match. The degree to which your child's bone marrow has failed also factors into the discussion whether to pursue a transplant. The decision to proceed with bone marrow transplant should be discussed with your child’s hematologist and a stem cell transplant team.

Immunosuppressive Therapy

This is the standard drug therapy for aplastic anemia and is based on the theory that aplastic anemia is caused by the immune system reacting against the bone marrow. Immunosuppressive drugs “suppress” the immune response and allow the bone marrow to make blood again. The drugs used, anti-thymocyte globulin (ATG) and cyclosporine (CsA), may also have other effects that allow the bone marrow to make blood effectively again. ATG/CsA therapy for aplastic anemia is not a fast process and can take three to six months for the treatment to have an effect. During that time, your child will need frequent supportive care, such as red blood cell or platelet transfusions and antibiotics. Children with aplastic anemia treated with immunosuppressive therapy also need life-long follow-up care from a hematologist.

Treating an underlying disorder

In rare cases aplastic anemia is caused from a previous illness or disorder. If this problem can be identified and treated, there are cases in which the aplastic anemia will improve. Additional treatment alternatives are currently being studied and may be available through clinical trials.

Supportive Care

In addition to treatment, supportive care helps the symptoms of aplastic anemia (fatigue, bleeding, infections, etc.). Examples include blood transfusion (both red cells and platelets), preventative antibiotic therapy, medications that stimulate the body to make more white blood cells, and behavioral interventions like thorough hand washing and avoiding large crowds or people who are known to have fevers or illness.