Bone Marrow Transplant

Overview

Hematopoietic transplantation, previously referred to as bone marrow transplantation, is a medical procedure that is used in certain circumstances to treat a variety of diseases. Hematopoietic transplantation was initially developed in the 1950s and 1960s to treat aggressive leukemias, bone marrow failure, and inherited immunodeficiency, but in recent years, its use has extended to the treatment of some autoimmune diseases, such as multiple sclerosis, and some genetic diseases.  Hematopoietic transplant may be allogeneic, or autologous.

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Allogeneic Hematopoietic Transplantation

In an allogeneic hematopoietic transplant, high doses of anti-cancer chemotherapy are given to the patient, and, following the chemotherapy, the patient is given an intravenous infusion of hematopoietic cells from a donor. In this case, the donor must be a close match with the patient for a set of genes on human chromosome 6, called the HLA gene cluster. The donor can be a brother or sister or an unrelated donor (there are approximately 9 million donors in the International Bone Marrow Transplant Registry), or the donor cells can be taken from the blood of a placenta that has been stored for this purpose (a cord blood transplant).

Diseases Treated by Allogeneic Hematopoietic Transplantation

Allogeneic hematopoietic transplantation is used primarily to treat acute myeloid leukemia, a related disorder called myelodysplasia, and acute lymphoid leukemia. Less often, allogeneic hematopoietic transplantation is used to treat other leukemias and sometimes lymphomas.

Autologous Hematopoietic Transplantation

In an autologous hematopoietic transplant, the patient has hematopoietic stem cells taken from their bloodstream using a device called an apheresis machine, which is similar to a kidney dialysis machine. The apheresis machine allows collection of hematopoietic stem cells, which are frozen until needed. Once a sufficient number of hematopoietic stem cells have been collected and frozen, the patient then receives very high doses of anticancer chemotherapy followed by an intravenous infusion of the thawed stem cells that were collected previously.

Diseases Treated by Allogeneic Hematopoietic Transplantation

Autologous hematopoietic transplant is performed most often to treat the multiple myeloma. Autologous hematopoietic transplant is also used to treat aggressive lymphoma that has relapsed after initial treatment. In each of these conditions, transplant is the standard of care and is not considered experimental or investigational. Autologous transplant is of benefit in certain severe cases of autoimmune diseases, particularly in multiple sclerosis, and in some non-hematologic cancers, such as neuroblastoma and poor-prognosis testicular cancer. Autologous transplantation is also used for gene therapy.