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Management of iron overloaded, transfusion-dependent SCD patients1–3

Transfusional iron accumulation leading to iron overload can result in organ damage4,5

Patients repeatedly receiving RBC transfusions are at risk for chronic iron overload, which often occurs in patients with transfusion-dependent anaemias, including SCD.5,6

In patients with SCD and transfusion-dependent thalassaemia receiving RBC transfusions, iron overload contributes to:

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Cardiac complications

  • Heart failure4,7,8
  • Cardiomyopathy4,7
  • Arrhythmias8

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Hepatic complications

  • Liver fibrosis4,7
  • Liver cirrhosis4,7

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Endocrine complications

  • Hypogonadotropic hypogonadism4,7
  • Osteoporosis4,9
  • Diabetes mellitus4,10
  • Growth retardation4,7
  • Abnormal sexual maturity4,7

The EXTENSION study was the largest evaluation of long-term efficacy and safety of an iron chelator in patients with transfusion-dependent SCD to date:3

  • A significant reduction in serum ferritin from BL (–591 μg/L; p=0.027; n=67) was observed after ≥4 years of EXJADE treatment for all patients with SCD who completed the long-term study3

The efficacy and safety of EXJADE was evaluated for up to 5 years in transfusion–dependent SCD patients

All patients3

Graph showing the median change in serum ferritin and mean EXJADE dose in all patients from baseline to month 60

  • Based on LOCF analysis, the overall median serum ferritin levels in patients with at least one EXJADE dose decreased from BL by 22 μg/L (n=185)3

LOCF analysis of patients with a ≥10% reduction in serum ferritin from the start of EXJADE treatment to the end of the study3

Table showing the proportion of patients with a ≥10% reduction in serum ferritin from initiation of EXJADE to study completion by mean overall iron take

The EPIC study was the largest ever investigation conducted for an iron chelator, evaluating the efficacy and safety of EXJADE:11,12

  • A total of 1,744 patients participated, of which 80 patients had SCD11,12
  • Based on LOCF analysis, overall median serum ferritin levels in patients with SCD decreased after 1 year of EXJADE treatment from BL (-225 μg/L, p=0.2588, n=78)11


Abbreviations: BL, baseline; DT, dispersible tablets; ICT, iron chelation therapy; LOCF, last-observation carried forward; RBC, red blood cell; SCD, sickle cell disease; SD, standard deviation.


  1. EXJADE® dispersible tablets summary of product characteristics.
  2. EXJADE® film-coated tablets summary of product characteristics.
  3. Vichinsky E, et al. Long-term safety and efficacy of deferasirox (EXJADE®) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease. Br J Haematol 2011;154(3):387–397.
  4. Lambing A, et al. The dangers of iron overload: Bring in the Iron Police. J Am Acad Nurse Pract 2012;24(4):175–183.
  5. Andrews NC. Disorders of iron metabolism. N Engl J Med 1999;341(26):1986–1995.
  6. Eckes JE. Chelation therapy for iron overload: nursing practice implications. J Infus Nurs 2011;34(6):374–380.
  7. Porter JB, Garbowski M. The pathophysiology of transfusional iron overload. Hematol Oncol Clin North Am. 2014;28(4):683–701.
  8. Hobrand AV, et al. How I treat transfusional iron overload. Blood. 2012;120(18):3657–3669.
  9. Rossi F, et al. Iron overload causes osteoporosis in thalassemia major patients through interaction with transient receptor potential vanilloid type 1 (TRPV1) channels. Haematologica. 2014;99(12):1876–84.
  10. Simcox JA, McClain DA. Iron and diabetes risk. Cell Metab 2013;17(3):329–341.
  11. 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.
  12. Gattermann N, et al. Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study. Leuk Res 2010;1143–1150.
  13. Ataga KI, et al. N Engl J Med 2017; 376:429-439.
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UK | August 2021 | 136208

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