Severe Aplastic Anemia: What Is It & Treatment Options

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What is it?

Aplastic anemia is a blood disorder in which the body’s bone marrow doesn’t make enough new blood cells. The 3 main types of blood cells your body needs are red blood cells (to carry oxygen), white blood cells (to fight infection), and platelets (to control bleeding).

Treatment Options

When stem cell medicine was first used as treatment for severe aplastic anemia, the only option was an allogeneic hematopoietic stem cell transplant: in simpler words, a transplant from another person of the type of stem cells that can turn into blood cells. Standard transplant procedures first use chemotherapy and radiation to destroy the existing cells in the bone marrow. Depending on age, smaller transplants use no or very small amounts of chemotherapy and radiation. HLA typing, which is used to look at the proteins on the cells, is used to match patients to a suitable match.

Currently, the only option for curing aplastic anemia is an allogeneic transplant. Standard transplants use chemotherapy and radiation to destroy the cells

A Clinical Trial and Advancements

A 2013 study conducted by doctors at Children’s Hospital of Orange County successfully transplanted autologous cord blood in a 9-year old male child with acquired severe aplastic anemia, the first successful transplant using the patient’s own cord blood for this condition. The transplant was successful following immunoablative chemotherapy. They concluded that with increasing numbers of cord blood cryopreservations, the use of autologous cord blood in the treatment of severe aplastic anemia might be considered as an initial therapy. This was the first successful application of autologous cord blood hematopoietic cell transplantation in a pediatric patient with acquired severe aplastic anemia using an immunoablative preparative regimen consisting of fludarabine and cyclophosphamide. This was found to be an effective and safe alternative to the complications with the use of matched unrelated donor hematopoietic cell transplantation.

Since then, more research into autologous cord blood transplants has been undertaken that has further demonstrated safe, effective treatment utilizing the patient’s own cord blood.


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