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.
Piomelli
S
S
Columbia University College of Physicians and Surgeons, New York, New York.
Loew
T
T
D001803:Blood Transfusion; D016026:Bone Marrow Transplantation; D015913:Chelation Therapy; D003676:Deferoxamine / Q000627:therapeutic use; D006801:Humans; D007501:Iron; D013789:Thalassemia / Q000628:therapy*
D007501:Iron; D003676:Deferoxamine