Long-term controlled studies are needed to inform on the clinical benefit of
chelation therapy for myocardial
iron removal in transfusion-dependent
beta thalassemia patients. In a 1-year nonrandomized extension to the CORDELIA study, data collected from patients with myocardial
siderosis provided additional information on
deferasirox or
deferoxamine (DFO) efficacy and safety. Myocardial (m)T2* increased from baseline 11.6 to 15.9 ms in patients receiving
deferasirox for 24 months (n = 74; geometric mean [Gmean ] ratio of month 24/baseline 1.38 [95% confidence interval 1.28, 1.49]) and from 10.8 to 14.2 ms in those receiving DFO (n = 29; Gmean ratio 1.33 [1.13, 1.55]; P = 0.93 between groups). Improved mT2* with
deferasirox was evident across all subgroups evaluated irrespective of baseline myocardial (mT2* < 10 vs. ≥ 10 ms) or liver (LIC <15 vs. ≥15 mg Fe/g dw)
iron burden. Mean LVEF was stable and remained within normal limits with
deferasirox or DFO. Liver
iron concentration decreased from high baseline values of 30.6 ± 18.0 to 14.4 ± 16.6 mg Fe/g dw at month 24 in
deferasirox patients and from 36.8 ± 15.6 to 11.0 ± 12.1 mg Fe/g dw in DFO patients. The long-term safety profile of
deferasirox or DFO was consistent with previous reports; serious
drug-related AEs were reported in 6.8% of
deferasirox and 6.9% of DFO patients. Continued treatment of severely
iron-overloaded
beta thalassemia patients with
deferasirox or DFO led to sustained improvements in myocardial
iron irrespective of high or low baseline myocardial or liver
iron burden, in parallel with substantial improvements in liver
iron (Clinicaltrials.gov identifier: NCT00600938).