Bone Marrow Transplant (2)

What is involved in obtaining a bone marrow transplant for thalassemia major? The biggest obstacle is finding the donor for the transplantation. Since beta thalassemia major is no longer considered a fatal disease, criteria for donation differs from the criteria for BMT for a patient with a fatal disease such as cancer. Usually a patient suffering from cancer is given very toxic chemotherapy to kill the cancer cells and then is "rescued" from the chemotherapy with a bone marrow transplant. In the case of thalassemia, the goal of the chemotherapy is to remove most or all of the blood forming cells (stem cells) from the recipient and replace them with donor stem cells in order to repopulate the bone marrow space and make new, normal blood cells. In a cancer patient, the 'best' (genotypic) match is used for the transplant. In a b thalassemia major patient, the match must be the 'perfect' (HLA-identical) match. The closest perfect match is usually a sibling who has the same antigenic makeup (is HLA identical to the recipient) as the individual who has thalassemia. There is only a one in four chance a sibling will have the required immunologic criteria for a bone marrow donation. There are many children who have thalassemia who have no siblings or no siblings who are HLA-identical for donation.

Generally speaking, the younger the recipient, the more optimistic the outcome. The Lucarelli Staging System is based on the amount of liver damage sustained as a consequence of iron overload from chronic transfusion therapy and the adequacy of Desferal chelation therapy. The staging includes evaluations of liver size, the schedule and compliance of desferrioxamine therapy, and liver biopsy (in children over 3 years) to determine whether or not there has been microscopic liver damage. <next>

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