title: Deferasirox improves hematologic and hepatic function with effective reduction of serum ferritin and liver iron concentration in transfusional iron overload patients with myelodysplastic syndrome or aplastic anemia.
abstract: BACKGROUND
  Transfusional iron overload and its consequences are challenges in chronically transfused patients with myelodysplastic syndromes (MDSs) or aplastic anemia (AA).
  
  
  METHODS
  This was a prospective, multicenter, open-label study to investigate the efficacy of deferasirox (DFX) by serial measurement of serum ferritin (S-ferritin) level, liver iron concentration (LIC) level using relaxation rates magnetic resonance imaging, and other laboratory variables in patients with MDS or AA.
  
  
  RESULTS
  A total of 96 patients showing S-ferritin level of at least 1000 ng/mL received daily DFX for up to 1 year. At the end of the study, S-ferritin level was significantly decreased in MDS (p=0.02366) and AA (p=0.0009). LIC level was also significantly reduced by more than 6.7 mg Fe/g dry weight from baseline. Hemoglobin level and platelet counts were significantly increased from baseline (p=0.002 and p=0.025, respectively) for patients showing significant anemia or thrombocytopenia. Elevated alanine aminotransferase was also significantly decreased from baseline.
  
  
  CONCLUSIONS
  This study shows that DFX is effective in reducing S-ferritin and LIC level in transfusional iron overload patients with MDS or AA and is well tolerated. In addition, positive effects in hematologic and hepatic function can be expected with DFX. Iron chelation treatment should be considered in transfused patients with MDS and AA when transfusion-related iron overload is documented.
authors: Department of Internal Medicine, Yonsei University, Seoul, Korea.
authors: June-Won
authors: JW
authors: Cheong
authors: Hyeoung-Joon
authors: HJ
authors: Kim
authors: Kyoo-Hyung
authors: KH
authors: Lee
authors: Sung-Soo
authors: SS
authors: Yoon
authors: Jae Hoon
authors: JH
authors: Lee
authors: Hee-Sook
authors: HS
authors: Park
authors: Ho Young
authors: HY
authors: Kim
authors: Hyeok
authors: H
authors: Shim
authors: Chu-Myung
authors: CM
authors: Seong
authors: Chul Soo
authors: CS
authors: Kim
authors: Jooseop
authors: J
authors: Chung
authors: Myung Soo
authors: MS
authors: Hyun
authors: Deog-Yeon
authors: DY
authors: Jo
authors: Chul Won
authors: CW
authors: Jung
authors: Sang Kyun
authors: SK
authors: Sohn
authors: Hwi-Joong
authors: HJ
authors: Yoon
authors: Byung Soo
authors: BS
authors: Kim
authors: Young-Don
authors: YD
authors: Joo
authors: Chi-Young
authors: CY
authors: Park
authors: Yoo Hong
authors: YH
authors: Min
mesh_terms: D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000628:therapy*; D001565:Benzoates / Q000627:therapeutic use*; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000378:metabolism*; D019190:Iron Overload / Q000188:drug therapy*; D008099:Liver / Q000187:drug effects / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D011446:Prospective Studies; D065227:Transfusion Reaction; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox