Chelation therapy in beta-thalassemia: the benefits and limitations of desferrioxamine.
In summary, long-term studies of DFO therapy in multiply-transfused patients with beta-thalassemia major have clearly shown it to be generally safe and effective. Appropriate use of DFO can remove excess iron, prevent iron-induced organ damage, and improve survival in thalassemia patients. Patients who begin treatment at a young age can be protected from the lethal complications of iron overload for at least two decades, but chelation therapy may not always prevent or ameliorate late growth failure and/or delayed or absent puberty. Those with iron damage to the heart and possibly other organs may experience stability or improvement in function with intense chelation. High-dose intravenous DFO produces a rapid decrease in hepatic iron content and improved cardiac function but can also cause severe toxicity, as can normal doses in patients with a low iron burden. Continuing studies of DFO are necessary to help further define its long-term efficacy and toxicity. In particular, significant attention should be paid to new strategies aimed at fostering improved compliance with its use.
Giardina
P J
PJ
Department of Pediatrics, Cornell University Medical Center, New York, NY 10021, USA.
Grady
R W
RW
D000293:Adolescent; D000328:Adult; D002614:Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D015913:Chelation Therapy / Q000009:adverse effects; D002648:Child; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D006130:Growth Disorders / Q000209:etiology / Q000517:prevention & control; D034381:Hearing Loss / Q000139:chemically induced; D006331:Heart Diseases / Q000209:etiology / Q000401:mortality / Q000517:prevention & control; D006432:Hemochromatosis / Q000209:etiology / Q000628:therapy*; D006486:Hemosiderosis / Q000209:etiology / Q000628:therapy; D006801:Humans; D007501:Iron; D007674:Kidney Diseases / Q000139:chemically induced; D008171:Lung Diseases / Q000139:chemically induced; D013789:Thalassemia / Q000150:complications / Q000628:therapy*; D065227:Transfusion Reaction; D014786:Vision Disorders / Q000139:chemically induced
D002614:Chelating Agents; D007501:Iron; D003676:Deferoxamine