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

June Won Cheong, Hyeoung Joon Kim, Kyoo Hyung Lee, Sung Soo Yoon, Jae Hoon Lee, Hee Sook Park, Ho Young Kim, Hyeok Shim, Chu Myung Seong, Chul Soo Kim, Jooseop Chung, Myung Soo Hyun, Deog Yeon Jo, Chul Won Jung, Sang Kyun Sohn, Hwi Joong Yoon, Byung Soo Kim, Young Don Joo, Chi Young Park, Yoo Hong Min

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28 Citations (Scopus)

Abstract

Background Transfusional iron overload and its consequences are challenges in chronically transfused patients with myelodysplastic syndromes (MDSs) or aplastic anemia (AA). Study Design and 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 levelof at least 1000ng/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 6.7mg 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.

Original languageEnglish
Pages (from-to)1542-1551
Number of pages10
JournalTransfusion
Volume54
Issue number6
DOIs
Publication statusPublished - 2014 Jun

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Hematology

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    Cheong, J. W., Kim, H. J., Lee, K. H., Yoon, S. S., Lee, J. H., Park, H. S., Kim, H. Y., Shim, H., Seong, C. M., Kim, C. S., Chung, J., Hyun, M. S., Jo, D. Y., Jung, C. W., Sohn, S. K., Yoon, H. J., Kim, B. S., Joo, Y. D., Park, C. Y., & Min, Y. H. (2014). 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. Transfusion, 54(6), 1542-1551. https://doi.org/10.1111/trf.12507