General Information About Hematopoietic Cell Transplantation (HCT)

Rationale for HCT

Blood and marrow transplantation (BMT) or HCT is a procedure that involves infusion of cells (hematopoietic stem cells; also called hematopoietic progenitor cells) that reconstitute the hematopoietic system of a patient. The infusion of hematopoietic stem cells generally follows a preparative regimen given to the patient consisting of agents designed to do the following:

• Suppress the patient's immune system to prevent rejection.
• Intensively treat malignant cells in patients with cancer.

HCT is currently used in the following three clinical scenarios:

1. Treatment of malignancies.
2. Replacement or modulation of an absent or poorly functioning hematopoietic or immune system.
3. Treatment of genetic diseases in which expression of the affected gene product in circulating hematopoietic cells alone is adequate to produce a clinically meaningful result.