title: Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.
abstract: BACKGROUND
  Prospective data on cardiac iron removal are limited beyond one year and longer-term studies are, therefore, important.
  
  
  METHODS
  Seventy-one patients in the EPIC cardiac substudy elected to continue into the 3(rd) year, allowing cardiac iron removal to be analyzed over three years.
  
  
  RESULTS
  Mean deferasirox dose during year 3 was 33.6 ± 9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms ± 39.1% at baseline to 17.1 ms ± 62.0% at end of study (P<0.001), corresponding to a decrease in cardiac iron concentration (based on ad hoc analysis of T2*) from 2.43 ± 1.2 mg Fe/g dry weight (dw) at baseline to 1.80 ± 1.4 mg Fe/g dw at end of study (P<0.001). After three years, 68.1% of patients with baseline T2* 10 to <20 ms normalized (≥ 20 ms) and 50.0% of patients with baseline T2* >5 to <10 ms improved to 10 to <20 ms. There was no significant variation in left ventricular ejection fraction over the three years. No deaths occurred and the most common investigator-assessed drug-related adverse event in year 3 was increased serum creatinine (n = 9, 12.7%).
  
  
  CONCLUSIONS
  Three years of deferasirox treatment along with a clinically manageable safety profile significantly reduced cardiac iron overload versus baseline and normalized T2* in 68.1% (32 of 47) of patients with T2* 10 to <20 ms.
authors.affiliation: Royal Brompton Hospital, London, UK.  d.pennell@ic.ac.uk.
authors.forename: Dudley J
authors.initials: DJ
authors.lastname: Pennell
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Maria Domenica
authors.initials: MD
authors.lastname: Cappellini
authors.forename: Lee Lee
authors.initials: LL
authors.lastname: Chan
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
authors.forename: Yesim
authors.initials: Y
authors.lastname: Aydinok
authors.forename: Hishamshah
authors.initials: H
authors.lastname: Ibrahim
authors.forename: Chi-Kong
authors.initials: CK
authors.lastname: Li
authors.forename: Vip
authors.initials: V
authors.lastname: Viprakasit
authors.forename: Mohsen S
authors.initials: MS
authors.lastname: Elalfy
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.forename: Gillian
authors.initials: G
authors.lastname: Smith
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Gabor
authors.initials: G
authors.lastname: Domokos
authors.forename: Bernard
authors.initials: B
authors.lastname: Roubert
authors.forename: Ali
authors.initials: A
authors.lastname: Taher
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000008:administration & dosage / Q000627:therapeutic use*; D001803:Blood Transfusion; D009202:Cardiomyopathies / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology / Q000517:prevention & control; D015913:Chelation Therapy; D002648:Child; D000077588:Deferasirox; D004334:Drug Administration Schedule; D006321:Heart / Q000187:drug effects* / Q000503:physiopathology; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D019190:Iron Overload / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D008137:Longitudinal Studies; D018810:Magnetic Resonance Angiography; D014230:Triazoles / Q000008:administration & dosage / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D000077588:Deferasirox