1. Medicines
  2. Haematology
  3. EXJADE® ▼ (deferasirox)
  4. Efficacy in thalassaemia

Prescribing information

 

Efficacy in thalassaemia

    

Management of iron overloaded, thalassaemia patients1–4

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

Icon depicting the heart

Heart

Increases the risk of heart failure

Icon depicting the liver

Liver

Increases the risk of cirrhosis

Icon depicting the pancreas

Pancreas

Increases the risk of multiple endocrine abnormalities

 
Regular blood transfusions in patients with transfusion-dependent thalassaemia will result in iron accumulation5
  • 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 patients3,4

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

Graph showing the mean change in LIC in all patients with transfusion-dependent thalassaemia and cardiac iron overload from baseline to month 12

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). 3rd ed. Nicosia, Cyprus: Thalassaemia International Federation; 2014.
HCP20-C005h June 2020.
Haematology
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