Retrospective nationwide survey of Japanese patients with transfusion-dependent MDS and aplastic anemia highlights the negative impact of iron overload on morbidity/mortality

Masaaki Takatoku, Takashi Uchiyama, Shinichiro Okamoto, Yuzuru Kanakura, Kenichi Sawada, Masao Tomonaga, Shinji Nakao, Tatsutoshi Nakahata, Mine Harada, Takashi Murate, Keiya Ozawa

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154 Citations (Scopus)


Objective: Myelodysplastic syndromes (MDS) and aplastic anemia (AA) are the most common anemias that require transfusion therapy in Japan. This retrospective survey investigated relationships between iron overload, chelation practices, and morbidity/mortality in patients with these diseases. Method: Medical histories of transfusion-dependent patients were assessed at transfusion onset, chelation onset, and study end. Results: Data were collected from 292 patients with MDS, AA, pure red cell aplasia, myelofibrosis, and other conditions. Patients received a mean of 61.5 red blood cell units during the previous year. Fewer than half (43%) of patients had previously received deferoxamine (DFO) therapy. Only 8.6% received daily/continuous DFO. In all, 75 deaths were reported, with cardiac and liver failure noted in 24.0 and 6.7% of cases. Of these, 97% had ferritin levels >1000 ng/mL. Abnormal cardiac and liver function was observed in 21.9% (14/64) and 84.6% (11/13) of all patients assessed. Effective chelation with DFO resulted in improved serum ferritin, liver enzymes, and fasting blood sugar. Conclusions: Mortality is higher in heavily iron-overloaded patients, with liver and cardiac dysfunction being the primary cause. Daily/continuous chelation therapy was effective at reducing iron burden and improving organ function. Chelation therapy should be initiated once serum ferritin levels exceed 1000 ng/mL.

Original languageEnglish
Pages (from-to)487-494
Number of pages8
JournalEuropean Journal of Haematology
Issue number6
Publication statusPublished - 2007 Jun



  • Aplastic anemia
  • Deferoxamine
  • Iron chelation therapy
  • Myelodysplastic syndromes
  • Refractory anemias

ASJC Scopus subject areas

  • Hematology

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