Reducing the iron burden and improving survival in transfusion-dependent thalassemia patients: current perspectives

Abstract

Hypertransfusion regimens for thalassemic patients revolutionized the management of severe thalassemia; transforming a disease which previously led to early infant death into a chronic condition. The devastating effect of the accrued iron from chronic blood transfusions necessitates a more finely tuned approach to limit the complications of the disease, as well as its treatment. A comprehensive approach including carefully tailored transfusion protocol, continuous monitoring and assessment of total body iron levels, and iron chelation are currently the mainstay in treating iron overload. There are also indications for ancillary treatments, such as splenectomy and fetal hemoglobin induction. The main cause of death in iron overload continues to be related to cardiac complications. However, since the widespread use of iron chelation started in the 1970s, there has been a general improvement in survival in these patients.

Keywords: chelators; deferiprone; deferiserox; deferoxamine; hematology; hepatic iron storage; iron chelation therapy; iron overload; liver iron concentration; serum ferritin concentration.

Figure 1

Non-contrast CT scans. Notes: (A) Normal liver and spleen. (B) Diffuse increased attenuation of the liver indicating excess iron deposition in a 47-year old patient with sickle cell disease undergoing multiple blood transfusions. Abbreviation: CT, computed tomography.

Figure 2

Normal axial T2* of liver of a 21-year old male patient with a known case of β-thalassemia on regular blood transfusions. Notes: There is a rise in signal intensity as echo time increases. This patient had a normal liver iron concentration of 0.15 mg/g.

Figure 3

Axial T2* of liver and heart of a 24-year old female patient with a known case of β-thalassemia major on chronic blood transfusions. Notes: (A) Moderate-to-high degree of hemosiderosis in this patient’s liver. T2* measurements of liver demonstrated sever hepatic iron loading of 2.1 ms. Sharp decrease in signal intensity as the echo time increases. Measured liver iron content was moderately high with 13.09 mg/g. (B) Heart – moderate degree of cardiac hemosiderosis demonstrating a gradual decrease in signal intensity as the echo time increases.