What is the most effective treatment of children with severe aplastic anemia who lack a matched sibling donor?

Abstract

The most effective treatment currently available for children with severe aplastic anemia is bone marrow transplantation using a sibling donor who is completely matched with the patient at the major histocompatibility complex. Only a minority of patients, however, will have such a donor and other treatments which have been used include the use of single agent immunosuppression, multi-agent immunosuppression, multi-agent immunosuppression with hemopoietic growth factors, and the use of allogeneic stem cell transplantation using mismatched family members, unrelated individuals or placental blood. Of these approaches the most promising appears to be the use of combination immunosuppressive therapy plus hemopoietic growth factors. This paper reviews the current results obtained in using these various methods of treating children with severe aplastic anemia who lack a matched sibling donor.