Prospective, 1-year, multinational, open-label, Phase III clinical trial evaluating the efficacy and safety of EXJADE in patients aged ≥2 years with transfusional haemosiderosis from various types of anaemia.
1,744 patients were enrolled with the following conditions: thalassaemia (n=1,115), MDS (n=341), aplastic anaemia (n=116), SCD (n=80) and rare anaemias (n=43).
Key inclusion criteria1,2
- ≥2 years of age
- Serum ferritin ≥1,000 ng/mL, or <1,000 ng/mL with a history of at least >20 transfusions or 100 mL/kg of RBCs
- LIC of ≥2 mg/g dw
EXJADE treatment regimen1
The recommended initial dose of 20 mg/kg/day EXJADE DT in patients receiving approximately 2–4 units of pRBC per month. Dose was adjusted based on serum ferritin response:1
Please note: the EPIC study was conducted with EXJADE DT. EXJADE FCTs have a higher bioavailability and the dose is 30% lower than EXJADE DT.1,3,4
- To evaluate if fixed starting doses of EXJADE, based on transfusion history and subsequent dose titration, can provide clinically acceptable chelation as measured by serum ferritin from BL to Week 52
- The safety and tolerability profile of treated patients from BL to Week 521,2,5
- The relationship between dose adjustment regimens and transfusional iron overload1,5
- Evaluate the relationship between serum ferritin in each disease cohort for potential surrogate markers1,2,5
*A 20 mg/kg/day EXJADE® DT dose is equivalent to 14 mg/kg/day EXJADE® FCT; a 30 mg/kg/day EXJADE® DT dose is equivalent to 21 mg/kg/day EXJADE FCT.3,4
Abbreviations: BL, baseline; DT, dispersible tablets; dw, dry weight; FCT, film-coated tablets; ICT, iron chelation therapy; LIC, liver iron concentration; MDS, myelodysplastic syndrome; pRBC, packed red blood cells; RBC, red blood cell; SCD, sickle cell disease.
- 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.
- 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.
- EXJADE® dispersible tablets summary of product characteristics.
- EXJADE® film-coated tablets summary of product characteristics.
- National Institutes of Health. A study assessing the efficacy and safety of deferasirox in patients with transfusion-dependent iron overload. Available at: https://clinicaltrials.gov/ct2/show/study/NCT00171821. Date accessed: May 2020.