Prescribing information

 

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Objective1,2

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.

Trial cohort1,2

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).

Study design1

Diagram summarising the design of the EPIC study, including the number of patients enrolled, treatment regimen and primary endpoint

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

Table showing the change in median serum ferritin level at 1 year vs baseline, p-value and mean transfusional iron intake per EXJADE dosage in the EPIC study

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

Primary endpoint1,2,5

  • 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

Secondary endpoints

  • 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.

References

  1. 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.
  2. 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.
  3. EXJADE® dispersible tablets summary of product characteristics.
  4. EXJADE® film-coated tablets summary of product characteristics.
  5. 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.
HCP20-C005d June 2020.
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Adverse events should be reported. Reporting forms and information can be found at yellowcard.mhra.gov.uk. Adverse events should also be reported to Novartis via [email protected] or online through the patient safety information (PSI) tool at https://psi.novartis.com
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