title: Sustained improvements in myocardial T2* over 2 years in severely iron-overloaded patients with beta thalassemia major treated with deferasirox or deferoxamine.
abstract: 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).
authors.affiliation: NIHR Cardiovascular Biomedical Research Unit, Royal Brompton Hospital, London, United Kingdom.
authors.forename: Dudley J
authors.initials: DJ
authors.lastname: Pennell
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Antonio
authors.initials: A
authors.lastname: Piga
authors.forename: Yong-Rong
authors.initials: YR
authors.lastname: Lai
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
authors.forename: Mohsen
authors.initials: M
authors.lastname: Elalfy
authors.forename: Akif
authors.initials: A
authors.lastname: Yesilipek
authors.forename: Yurdanur
authors.initials: Y
authors.lastname: Kilinç
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Khaled M
authors.initials: KM
authors.lastname: Musallam
authors.forename: Junwu
authors.initials: J
authors.lastname: Shen
authors.forename: Yesim
authors.initials: Y
authors.lastname: Aydinok
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use*; D015913:Chelation Therapy; D002648:Child; D000077588:Deferasirox; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007501:Iron / Q000378:metabolism*; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology / Q000378:metabolism / Q000473:pathology; D008099:Liver / Q000378:metabolism / Q000473:pathology; D008297:Male; D009206:Myocardium / Q000378:metabolism* / Q000473:pathology; D011446:Prospective Studies; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000378:metabolism / Q000473:pathology / Q000628:therapy
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D003676:Deferoxamine; D000077588:Deferasirox