Management of thalassemia major (Cooley's anemia).

Cooley's anemia patients, treated with a combined regimen of blood transfusions and chelation therapy, can look forward today to near-normal survival and excellent quality of life. Transfusion therapy should be directed at maintaining a hemoglobin baseline (greater than or equal to 10 g/dL) adequate to prevent hypoxia and its consequences: bone marrow expansion and cardiomegaly. Chelation therapy should aim at a very reduced Fe overload, to be monitored by the serum ferritin level. Several oral Fe chelating drugs currently being developed appear promising; none, however, is ready for general use as yet. Transplantation of bone marrow from a compatible sibling can cure Cooley's anemia; however, this therapy is currently only applicable to a small percentage of patients.