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Study design³

• This prospective, 1-year, phase 2 study evaluated the efficacy of EXJADE in regularly transfused patients with MDS, Diamond-Blackfan anemia, other rare anemias, or β-thalassemia (N=184)

Patient profile³

• Eligible patients were:
  • 2 years of age or older
  • Diagnosed with a transfusion-dependent anemia (with the exclusion of sickle cell anemia)
  • Receiving 8 or more transfusions per year
  • Required to have an LIC ≥2 mg Fe/g dry weight
• Of the 184 patients, 47 had MDS

Results³

• EXJADE had an overall success rate of 78.6% in MDS patients, with success defined by maintenance or reduction of LIC based on patients' baseline LIC
• All patients with MDS had reductions of LIC over 1 year
• Effects on mean serum ferritin paralleled the dose-dependent changes on LIC

"The availability of a convenient, once-daily, oral iron chelator that has predictable, dose-dependent effects on iron balance and a defined, clinically manageable safety profile across a range of transfusion-dependent anemias, including MDS, should allow a greater number of regularly transfused patients to gain benefit from iron chelation therapy.³"

— J Porter et al