Bone marrow transplantation

The place of marrow transplantation for the treatment of P-thalassaemia is now much clearer. In centres with broad experience of the procedure, and if it is carried out within the first few years of life and before there is iron loading (provided that there is a good HLA match for the donor), the success rate appears to be close to 90%. It is lower if there is already iron loading of the liver or if the transplant is carried out in older patients. The main complications are severe infection during the period of transplantation and either acute or chronic graft-versus-host disease. So far, after over 15 years of surveillance, there have been no reports of secondary bone marrow malignancies in children treated in this way. A number of approaches are being tried to overcome the problem of lack of matched donors. In a few cases, HLA typing has been carried out after in vitro fertilization to attempt to obtain an HLA-matching sibling, although there is still considerable ethical concern about this approach. It is still too early to determine the role of haemopoietic stem cell therapy for the treatment of this disease.

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