Bone marrow transplantation in the treatment of thalassemia.

Early trials with the analysis of results in patients less than 16 years old have allowed us to identify three classes of risk using the criteria of degree of hepatomegaly, the degree of liver fibrosis, and the quality of chelation treatment given before transplant. The posttransplant disease-free survival for patients in risk classes I, II, and III and adults is today 93%, 85%, 64%, and 82%, respectively. Bone marrow transplantations, from mismatched donors and unrelated phenotypically identical donors are still experimental procedures. Bone marrow transplantation represents a desirable option of cure for severe forms of the disease when an HLA-identical donor is available. The posttransplant clinical follow-up of these patients is of particular interest in managing growth, endocrinal problems, iron overload, and normal quality and expectancy of life posttransplant. This purpose seems attainable particularly for those patients who have received transplants earlier, when histological damage of the liver and endocrine organs is not yet present.