bone marrow transplantation

Bone marrow (or stem cell) transplantation is now a standard curative treatment for about 75 diseases. About 15,000 transplants are performed each year worldwide.

The first successful transplant was performed in 1968 on a child with Severe Combined Immunodeficiency syndrome (SCID). The procedure is now used in several major areas of disease:

  • congenital immunodeficency syndromes
  • malignancies
  • bone marrow failure syndromes such as aplastic anemia
  • disorders of hemoglobin production (hemoglobinopathies) such as sickle cell anemia
  • inborn errors of metabolism

The ideal donors are identical HLA-matched siblings.

Bone marrow transplantation used to be viewed as a last resort measure. Now BMT is considered very early on in the course of a number of diseases. Preparation for the procedure is rigorous and involves massive doses of chemotherapy and radiation to eradicate the defective marrow.

Chances for BMT success are greatest when the procedure is done early, while the patient is stronger and has not suffered the ravages of his disease for too long. After the procedure, the greatest threat to success is more often not rejection of the transplanted bone marrow stem cells, but so-called graft versus host disease, in which the new immune cells from the donor recognize the host tissues as foreign to them and "attack" or reject them.

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