Pediatric Blood Disorders: Acquired Severe Aplastic Anemia

Unlike inherited bone marrow failure syndromes, aplastic anemia is often acquired during a person’s lifetime, either through unknown or environmental causes. Your child can be diagnosed through a series of blood tests that rule out inherited bone marrow failure syndromes and indicate the abnormal production of red and while blood cells as well as platelets.

If your child has been diagnosed with acquired aplastic anemia, Memorial Sloan Kettering’s team of pediatric experts will develop a treatment plan designed to cure the bone marrow failure, address any additional symptoms, and improve your child’s quality of life.

Stem Cell Transplantation from a Matched Sibling

If your child has a sibling who does not have the disease and is a bone marrow match, allogeneic stem cell transplantation is the treatment of choice recommended by our physicians. At Memorial Sloan Kettering, the cure rate of bone marrow failure in children with aplastic anemia is about 90 percent.(1)

In this treatment, another person’s healthy stem cells are transplanted into your child to stimulate the production of red blood cells, white blood cells, and platelets. Allogeneic stem cell transplantation can also be referred to as blood or bone marrow transplantation or cord blood transplantation, depending on the origin of the transplanted cells. The particular type of transplantation used to treat your child depends on the type of syndrome he or she has.

Regardless of the type of transplantation, the goals are the same: to successfully replace the bone marrow stem cells with healthy ones; to limit the risk of graft-versus-host disease, in which the child’s immune system attacks the transplanted cells; to decrease the risk that your child will develop infections during the treatment process; and to reduce the chance that your child will reject the transplanted cells.

If a child with severe aplastic anemia has a sibling who is a bone marrow match, the standard treatment is to perform a stem cell transplant as soon as possible. The conditioning regimen used prior to the transplant involves a mild chemotherapy and immunotherapy to suppress the immune system. This therapy is usually very well-tolerated by young patients.

Suppressing the Immune System

Aplastic anemia results from white blood cells known as T cells preventing the blood stem cells from growing, causing a child to be unable to produce red blood cells, white blood cells, or platelets.

If your child does not have a sibling who is a bone marrow match, our doctors will treat him or her with a treatment known as immunosuppression. This type of therapy suppresses the immune system, and in particular the T cells. In a successful immunosuppression treatment, your child’s T cells will be destroyed and his or her stem cells will be able to produce healthy blood cells.

This treatment often results in a cure, though some children can experience a recurrence of bone marrow failure or develop another blood disease called myelodysplastic syndrome, in which blood cells become abnormally shaped and reproduce uncontrollably. For this reason, patients who undergo treatment with immunosuppression will need to be monitored carefully by a doctor.

Transplantation from an Unrelated or Mismatched Donor

If immunosuppression is not successful in treating your child’s bone marrow failure, our doctors recommend allogeneic stem cell transplantation from a matched donor who is not related to your child, or from a mismatched relative.

This may be achieved at Memorial Sloan Kettering using T-cell depleted transplants to prevent graft-versus-host disease. Our most recent clinical trials also include conditioning treatment using chemotherapy only, without total body irradiation.