Saturday, February 16, 2013

"Gattermann et al. [Haematologica, 2012; 97(9): 1364-1371] have recently published
 data on the impact of chelation therapy with deferasirox (Exjade) on hemoglobin levels,
 platelet counts, and neutrophil counts in patients with transfusion-dependent MDS.
To be included in the trial, patients must have:
  1. Been diagnosed with iron overload (serum ferritin > 1,000 μg/L or LIC > 2 mg-Fe/g-dw)
  2. Had a history of multiple blood transfusions (> 20 transfusions or > 100 ml/Kg
  3.  of RBCs)
  4. Not be on a concomitant disease modifying agent for MDS (e.g., Vidaza, Dacogen,
  5.  Revlimid)
Recruited patients were treated with deferasirox for 1 year of active treatment – there
was no control arm in the trial design.  The following table summarizes the main study findings:"
Erythroid analysisPlatelet analysisNeutrophil analysis
Eligible patients24710050
Inclusion criteriaPre-treatment HB < 11 g/dl, OR RBC transfusion requirements > 4 units
per 8 weeks
Pre-treatment platelets
< 100 x 109/L
Pre-treatment ANC
< 1.0 x 109/L
Definition of responseHB increase > 1.5g/dL,
OR reduction of ≥ 4 RBC transfusions per 8 weeks
Increase > 30 x 109/L for patients below 20 x 109/L, or > 100% increase> 100% increase, AND absolute increase
> 0.5 x 109/L
Response rate21.5%13%22%
Median days to response109169226
Mean actual deferasirox dose, mg/kg/day~19.3~19.5~18.8

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