Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload

Haematologica. 2011 Jan;96(1):48-54. doi: 10.3324/haematol.2010.031468. Epub 2010 Nov 11.


Background: The efficacy of cardiac iron chelation in transfusion-dependent patients has been demonstrated in one-year prospective trials. Since normalization of cardiac T2* takes several years, the efficacy and safety of deferasirox was assessed for two years in patients with β-thalassemia major in the cardiac sub-study of the EPIC trial.

Design and methods: Eligible patients with myocardial T2* greater than 5 to less than 20 ms received deferasirox, with the primary endpoint being the change in T2* from baseline to two years.

Results: Baseline myocardial T2* was severe (> 5 to < 10 ms) in 39 patients, and moderate-to-mild (10 to < 20 ms) in 62 patients. Mean deferasirox dose was 33.1 ± 3.7 mg/kg/d in the one-year core study increasing to 36.1 ± 7.7 mg/kg/d during the second year of treatment. Geometric mean myocardial T2* increased from a baseline of 11.2 to 14.8 ms at two years (P < 0.001). In patients with moderate-to-mild baseline T2*, an increase was seen from 14.7 to 20.1 ms, with normalization (≥ 20 ms) in 56.7% of patients. In those with severe cardiac iron overload at baseline, 42.9% improved to the moderate-to-mild group. The incidence of drug-related adverse events did not increase during the extension relative to the core study and included (≥ 5%) increased serum creatinine, rash and increased alanine aminotransferase.

Conclusions: Continuous treatment with deferasirox for two years with a target dose of 40 mg/kg/d continued to remove iron from the heart in patients with β-thalassemia major and mild, moderate and severe cardiac siderosis. ( identifier: NCT 00171821).

Trial registration: NCT00171821.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Benzoates / therapeutic use*
  • Child
  • Deferasirox
  • Female
  • Ferritins / blood
  • Humans
  • Iron / metabolism*
  • Iron Chelating Agents / therapeutic use
  • Iron Overload / chemically induced
  • Iron Overload / drug therapy*
  • Magnetic Resonance Imaging*
  • Male
  • Middle Aged
  • Myocardium / pathology*
  • Prognosis
  • Prospective Studies
  • Triazoles / therapeutic use*
  • Ventricular Function, Left / drug effects*
  • Young Adult
  • beta-Thalassemia / complications
  • beta-Thalassemia / drug therapy*


  • Benzoates
  • Iron Chelating Agents
  • Triazoles
  • Ferritins
  • Iron
  • Deferasirox

Associated data