title: Improvement in liver pathology of patients with β-thalassemia treated with deferasirox for at least 3 years.
abstract: OBJECTIVE
  Most data on the effects of iron chelation therapy for patients with liver fibrosis come from small studies. We studied the effects of the oral iron chelator deferasirox on liver fibrosis and necroinflammation in a large population of patients with iron overload β-thalassemia.
  
  
  METHODS
  We studied data from 219 patients with β-thalassemia, collected from histologic analyses of biopsy samples taken at baseline and after at least 3 years of treatment with deferasirox. Treatment response was assessed from liver iron concentrations at baseline and the end of the study. Liver fibrosis, necroinflammation, and markers of iron overload and liver enzymes were recorded. Patients were also assessed, by serologic analysis at baseline, for hepatitis C virus infection.
  
  
  RESULTS
  By the end of the study, stability of Ishak fibrosis staging scores (change of -1, 0, or +1) or improvements (change of ≤-2) were observed in 82.6% of patients; Ishak necroinflammatory scores improved by a mean value of -1.3 (P<.001). Improvements in fibrosis stage and necroinflammation were independent of hepatitis C virus exposure or reduction in liver iron concentration defined by the response criteria. Absolute changes in concentrations of liver iron by the end of the study did not correlate with improved Ishak fibrosis or necroinflammatory scores.
  
  
  CONCLUSIONS
  Deferasirox treatment for 3 or more years reversed or stabilized liver fibrosis in 83% of patients with iron-overloaded β-thalassemia. This therapeutic effect was independent of reduced concentration of liver iron (defined by the response criteria) or previous exposure to hepatitis C virus.
authors: Liver Disease Unit and Inserm U-991, Department of Pathology, University Hospital Pontchaillou, Rennes, France. yves.deugnier@univ-rennes1.fr
authors: Yves
authors: Y
authors: Deugnier
authors: Bruno
authors: B
authors: Turlin
authors: Martine
authors: M
authors: Ropert
authors: M Domenica
authors: MD
authors: Cappellini
authors: John B
authors: JB
authors: Porter
authors: Vanessa
authors: V
authors: Giannone
authors: Yiyun
authors: Y
authors: Zhang
authors: Louis
authors: L
authors: Griffel
authors: Pierre
authors: P
authors: Brissot
mesh_terms: D000293:Adolescent; D000328:Adult; D000410:Alanine Transaminase / Q000097:blood; D001565:Benzoates / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood; D001706:Biopsy; D002648:Child; D002675:Child, Preschool; D018592:Cross-Over Studies; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006526:Hepatitis C / Q000473:pathology; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008099:Liver / Q000187:drug effects* / Q000378:metabolism / Q000473:pathology; D008103:Liver Cirrhosis / Q000097:blood / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D013997:Time Factors; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult; D017086:beta-Thalassemia / Q000097:blood / Q000150:complications / Q000188:drug therapy* / Q000473:pathology
chemical_list: D001565:Benzoates; D015415:Biomarkers; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000410:Alanine Transaminase; D000077588:Deferasirox