A randomised, multi-centre, open-label, Phase II study evaluating the safety of EXJADE DT and EXJADE FCT formulations in patients with transfusion-dependent thalassaemia or an IPSS score of very-low-, low- or intermediate-risk MDS.
Overall, 173 patients were randomised in a 1:1 ratio where 86 patients received EXJADE DT and 87 patients received EXJADE FCT.
Key inclusion criteria1
- ≥10 years of age
- Patients with transfusion-dependent thalassaemia or an IPSS score of very-low-, low-, or intermediate-risk MDS
- Chelation-naïve or pre-treated patients
- Transfusion history of ≥20 pRBC units
- Anticipated transfusion requirements of ≥8 units/year during study
- Serum ferritin >1000 ng/mL
EXJADE treatment regimen1
- For chelation-naïve patients, the starting dose was 20 mg/kg/day with EXJADE DT or 14 mg/kg/day with EXJADE FCT
For previously treated patients, a washout period of 5 days was required
- All pre-treated patients previously treated with EXJADE DT, deferoxamine or deferiprone were requested to start on an EXJADE DT or EXJADE FCT dose equivalent to their pre-washout dose
Dose adjustments were used to improve treatment response based on serum ferritin levels and investigator’s judgment. Dose adjustments were recommended to be:
- Every 4 weeks for chelation-naïve patients
- Every 3 months for pre-treated patients
- In increments of 5–10 mg/kg/day for patients receiving DT
- In increments of 3.5–7 mg/kg/day for patients receiving FCT
- Up to a maximum dose of 40 mg/kg/day for DT and 28 mg/kg/day for FCT
- Dose adjustments based on safety and dose reductions for patients unable to tolerate the protocol-specified dosing schedule were allowed at any time during the study
- The overall safety of EXJADE DT and EXJADE FCT, measured by frequency and severity of adverse events, and changes in laboratory values from BL over 24 weeks
- Pharmacokinetics of EXJADE DT and EXJADE FCT
- Patient-reported outcomes of EXJADE DT and EXJADE FCT
Abbreviations: BL, baseline; DT, dispersible tablets; FCT, film-coated tablets; ICT, iron chelation therapy; IPSS, International Prognostic Scoring System; MDS, myelodysplastic syndromes; pRBC, packed red blood cells.
- Taher A, et al. New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, Phase II ECLIPSE study. Am J Hematol 2017;92(5):420–428.