Annals of Hematology

, 88:301 | Cite as

Deferasirox in MDS patients with transfusion-caused iron overload—a phase-II study

  • Georgia Metzgeroth
  • Dietmar Dinter
  • Beate Schultheis
  • Alexandra Dorn-Beineke
  • Kira Lutz
  • Oliver Leismann
  • Rüdiger Hehlmann
  • Jan HastkaEmail author
Original Article


Blood transfusions represent a main component of supportive care in myelodysplastic syndromes (MDS). To avoid organ damage caused by transfusion-dependent iron overload, an adequate iron chelation therapy is required. Recently, a new oral iron chelator deferasirox (ICL670, Exjade®) has become available. A study was conducted to demonstrate the efficacy and tolerability of deferasirox in transfusion-dependent iron-overloaded patients with MDS. The efficacy of deferasirox was monitored by changes in serum ferritin, bone marrow iron, and liver iron concentration (LIC), as determined by T2*-weighted magnetic resonance imaging. Twelve patients with MDS of different subtypes (median age 76 years, range 53–91) were enrolled. Deferasirox administered in a once-daily dose of 20–30 mg/kg for 12 months was effective in reducing median ferritin concentration from 1,515 µg/L (range 665–6,900) to 413 µg/L (range 105–3,052). Within the first 4 weeks of treatment before the continuous decline of ferritin levels, the values markedly rose in eight of 12 patients. The median LIC declined from 315 to 230 µmol/g (p = 0.02) at the end of study, accompanied by a reduction of bone marrow siderosis. The most common adverse events were mild and transient gastrointestinal disturbances, skin rash, nonprogressive transient increases in serum creatinine and urine β2-microglobulin, and a temporary reduction of the creatinine clearance. The renal parameters normalized after end of treatment. No hematologic toxicities were observed. Deferasirox proved to be effective in transfusion-dependent iron overload in MDS by mobilizing iron deposits in liver and at least stabilizing iron stores in bone marrow.


Myelodysplastic syndromes Transfusional iron overload Iron chelator Deferasirox LIC assessment by MRI 



myelodysplastic syndromes


liver iron concentration


magnetic resonance imaging


refractory anemia


refractory anemia with ringed sideroblasts


refractory cytopenia with multilineage dysplasia


refractory anemia with excess of blasts


red blood cells


acute myeloid leukemia


alanine aminotransferase


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Copyright information

© Springer-Verlag 2008

Authors and Affiliations

  • Georgia Metzgeroth
    • 1
  • Dietmar Dinter
    • 2
  • Beate Schultheis
    • 1
  • Alexandra Dorn-Beineke
    • 3
  • Kira Lutz
    • 2
  • Oliver Leismann
    • 4
  • Rüdiger Hehlmann
    • 1
  • Jan Hastka
    • 1
    Email author
  1. 1.III. Medizinische UniversitätsklinikMedizinische Fakultät Mannheim der Universität HeidelbergMannheimGermany
  2. 2.Institut für Klinische Radiologie und Nuklearmedizin, Medizinische Fakultät MannheimUniversität HeidelbergMannheimGermany
  3. 3.Institut für Klinische Chemie, Medizinische Fakultät MannheimUniversität HeidelbergMannheimGermany
  4. 4.Novartis PharmaNürnbergGermany

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