title: Effect of deferasirox on iron overload in patients with transfusion-dependent haemoglobinopathies.
abstract: BACKGROUND
  Patients with haematopoietic disorders requiring long-term blood transfusions are at risk of iron overload. This study aimed to investigate the efficacy and safety of long-term deferasirox monotherapy in patients with transfusion-dependent anaemia in the routine clinical practice setting.
  
  
  METHODS
  This was a retrospective analysis of patients who commenced deferasirox therapy at the Hospital Bianchi Melacrino Morelli in Reggio Calabria, Italy. Data collected included cardiac and hepatic iron load (assessed by magnetic resonance imaging); left ventricular ejection fraction (LVEF). Patients were divided into two groups for analysis: group A (baseline information collected prior to deferasirox initiation) and group B (baseline information collected after deferasirox initiation).
  
  
  RESULTS
  Forty-six patients were included (group A: n=25; group B: n=21). The overall population was 63% male, with a mean age of 33 years. The majority of patients (65%) had thalassaemia major. In the overall population, cardiac iron levels between the baseline and first follow-up visits improved in both groups A and B (29.2 vs. 32.5 ms; p=0.04 and 28.4 vs. 31.4 ms; p=0.038). Liver iron levels improved significantly from baseline to visit 1 in group A (7.2 vs. 12.1 ms; p<0.004) and from baseline to visit 3 (6.9 vs. 10.7; p=0.049) in group B. Generally, there was no correlation between cardiac and liver iron levels. LVEF remained stable throughout the study period. Deferasirox was well tolerated and was not associated with significant adverse events.
  
  
  CONCLUSIONS
  Long-term treatment with deferasirox is effective and safe in patients with transfusion-dependent haemoglobinopathies monitored in the clinical practice setting.
authors.affiliation: SSD Microcythemia Center, Hospital Bianchi Melacrino Morelli, Reggio Calabria, Italy.
authors.forename: Concetta
authors.initials: C
authors.lastname: Fragomeno
authors.affiliation: SSD Microcythemia Center, Hospital Bianchi Melacrino Morelli, Reggio Calabria, Italy.
authors.forename: Emilio
authors.initials: E
authors.lastname: Roccabruna
authors.affiliation: SSD Microcythemia Center, Hospital Bianchi Melacrino Morelli, Reggio Calabria, Italy. Electronic address: domdascola@alice.it.
authors.forename: Domenico Giuseppe
authors.initials: DG
authors.lastname: D'Ascola
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006453:Hemoglobinopathies / Q000150:complications* / Q000628:therapy; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000209:etiology* / Q000503:physiopathology; D008099:Liver / Q000187:drug effects / Q000378:metabolism / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009206:Myocardium / Q000378:metabolism; D012189:Retrospective Studies; D013318:Stroke Volume; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016277:Ventricular Function, Left; D055815:Young Adult
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox
keywords: Deferasirox; Haemoglobinopathies; Iron overload; Transfusion-dependent