Hematopoietic stem cell transplantation

Abstract

Introduced in the early 1970s for treatment of aplastic anemia and leukemia, hematopoietic cell transplantation (HCT) offers treatment for a growing number of patients with complex hematologic malignancies, dysfunctional or absent immune systems, inherited or acquired marrow failure, and selected genetic disorders including hemoglobinopathies and inborn errors of metabolism. The procedure involves a preparative regimen for suppression of the patient’s immune system followed by infusion of hematopoietic progenitor cells. There are two major forms of HCT: allogeneic and autologous. In allogeneic transplant, hematopoietic stem cells (HSC) are obtained from related or unrelated donor source after administration of high-dose cytotoxic therapy. Because of immunologic differences between the donor and recipient, graft-versus-tumor (GVT) or graft-versus-leukemia (GVL) effect can occur. Autologous HCT involves exposing patients to myeloablative doses of cytotoxic therapy followed by infusion of the patient’s previously stored hematopoietic stem cells. Current pediatric indications for autologous transplant include patients with certain lymphomas, neuroblastoma, and brain tumors.