Key concepts

© Hematopoietic stem cell transplantation (HSCT) is a procedure used mainly to treat hematologic malignancies via high-dose chemotherapy and/or a graft-versus-tumor effect.

An autologous HSCT involves the infusion of a patient's own hematopoietic cells and allows for the administration of higher doses of chemotherapy, radiation, or both to treat the malignancy. Infusion of another's hematopoietic cells is an allogeneic HSCT; these cells can be from donors related or unrelated to the recipient.

Umbilical cord blood, peripheral blood progenitor cells (PBPCs), and bone marrow can serve as the source of hematopoietic cells. The optimal cell source differs based on the donor and recipient characteristics.

^^ A myeloablative preparative regimen involves the administration of sublethal doses of chemotherapy to the recipient in order to eradicate residual malignant disease. The recipient will not regain his or her own hematopoiesis and will be at risk for substantial life-threatening nonhematologic toxicity.

O A nonmyeloablative preparative regimen is less toxic than a myeloablative regimen in hopes of being able to offer the benefits of an allogeneic HSCT to more patients. A nonmyeloablative HSCT is based on the concept of donor immune response having a graft-versus-tumor effect.

© Nonhematologic toxicity differs based upon the preparative regimen administered.

^^ Engraftment is the reestablishment of functional hematopoiesis. It is commonly defined as the point at which a patient can maintain a sustained absolute neutrophil count (ANC) of greater than 500 cells/mm (0.5 x 10 /L) and a sustained platelet

count of greater 20,000/mm (20 x 10 /L) lasting for three or more consecutive days without transfusions.

® Graft-versus-host disease (GVHD) is caused by the activation of donor lymphocytes leading to immune damage to the skin, gut, and liver in the recipient. An immunosuppressive regimen is administered to prevent GVHD in recipients of an allogeneic graft; this regimen is based on the type of preparative regimen and the source of the graft.

O Recipients of HSCT are at higher risk of bacterial, viral, and fungal infections and usually receive a prophylactic or preemptive regimen to minimize the morbidity and mortality owing to infectious complications.


-term survivors of HSCT should be monitored closely, particularly for infections and secondary malignant neoplasms.

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