Efficacy in thalassaemia

Management of iron overloaded, thalassaemia patients^1–4

Iron overload may cause organ damage and result in serious clinical consequences⁵

Heart

Increases the risk of heart failure

Liver

Increases the risk of cirrhosis

Pancreas

Increases the risk of multiple endocrine abnormalities

• ICT aims to remove excess iron by increasing iron excretion in the urine and faeces, therefore reducing the risk of organ damage as a result of regular blood transfusions
• Oral ICT may improve patient compliance vs. parenteral therapies, but overall compliance to ICT remains poor

EXJADE: Proven to reduce serum ferritin in iron overloaded, thalassaemia patients^3,4

The EPIC study was the largest ever investigation conducted for an iron chelator, evaluating the efficacy and safety of EXJADE:^3,4

• A total of 1,744 patients participated, of which 1,115 patients had thalassaemia
• During this investigation there was a significant reduction in serum ferritin from BL (-264 μg/L; p<0.0001; n=1,707) after 1-year of EXJADE treatment for all patients
• Based on LOCF analysis, overall median serum ferritin levels in patients with thalassaemia significantly decreased from BL after 1-year of EXJADE treatment (-163 μg/L; p<0.0001; n=1,104)
• After 1-year of EXJADE treatment, the mean LIC in patients with cardiac iron overload was significantly decreased from BL (-6.6 mg Fe/g dw, p<0.001; n=105)

Mean change in LIC from BL

All patients with transfusion-dependent thalassaemia and cardiac iron overload⁴

Abbreviations: BL, baseline; dw, dry weight; ICT, iron chelation therapy; LIC, liver iron concentration; LOCF, last-observation carried forward.

References

1. EXJADE^® dispersible tablets summary of product characteristics.
2. EXJADE^® film-coated tablets summary of product characteristics.
3. Cappellini M, et al. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica 2010;95:557–566.
4. Pennell DJ, et al. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood 2010;115(12):2364–2371.
5. Cappellini MD, et al. Guidelines for the management of transfusion dependent thalassaemia (TDT). 3^rd ed. Nicosia, Cyprus: Thalassaemia International Federation; 2014.