title: Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes.
abstract: BACKGROUND
  Reductions in transfusion requirements/improvements in hematologic parameters have been associated with iron chelation therapy in transfusion-dependent patients, including those with myelodysplastic syndromes; data on there reductions/improvements have been limited to case reports and small studies.
  
  
  METHODS
  To explore this observation in a large population of patients, we report a post-hoc analysis evaluating hematologic response to deferasirox in a cohort of iron-overloaded patients with myelodysplastic syndromes enrolled in the Evaluation of Patients' Iron Chelation with Exjade(®) (EPIC) study using International Working Group 2006 criteria.
  
  
  RESULTS
  Two-hundred and forty-seven, 100 and 50 patients without concomitant medication for myelodysplastic syndromes were eligible for analysis of erythroid, platelet and neutrophil responses, respectively. Erythroid, platelet and neutrophil responses were observed in 21.5% (53/247), 13.0% (13/100) and 22.0% (11/50) of the patients after a median of 109, 169 and 226 days, respectively. Median serum ferritin reductions were greater in hematologic responders compared with non-responders at end of study, although these differences were not statistically significant. A reduction in labile plasma iron to less than 0.4 μmol/L was observed from week 12 onwards; this change did not differ between hematologic responders and non-responders.
  
  
  CONCLUSIONS
  This analysis suggests that deferasirox treatment for up to 1 year could lead to improvement in hematologic parameters in some patients with myelodysplastic syndromes.
authors.affiliation: Heinrich-Heine-Universität, Düsseldorf, Germany. gattermann@med.uni-duessel-dorf.de
authors.forename: Norbert
authors.initials: N
authors.lastname: Gattermann
authors.forename: Carlo
authors.initials: C
authors.lastname: Finelli
authors.forename: Matteo
authors.initials: M
authors.lastname: Della Porta
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: Michael
authors.initials: M
authors.lastname: Stadler
authors.forename: Agnes
authors.initials: A
authors.lastname: Guerci-Bresler
authors.forename: Mathias
authors.initials: M
authors.lastname: Schmid
authors.forename: Kerry
authors.initials: K
authors.lastname: Taylor
authors.forename: Dominique
authors.initials: D
authors.lastname: Vassilieff
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Andrea
authors.initials: A
authors.lastname: Marcellari
authors.forename: Bernard
authors.initials: B
authors.lastname: Roubert
authors.forename: Christian
authors.initials: C
authors.lastname: Rose
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000627:therapeutic use*; D001792:Blood Platelets / Q000187:drug effects / Q000473:pathology*; D002648:Child; D000077588:Deferasirox; D005260:Female; D005500:Follow-Up Studies; D006454:Hemoglobins / Q000032:analysis*; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications* / Q000628:therapy; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy / Q000209:etiology / Q000401:mortality; D009504:Neutrophils / Q000187:drug effects / Q000473:pathology*; D011379:Prognosis; D011446:Prospective Studies; D015996:Survival Rate; D065227:Transfusion Reaction; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D001565:Benzoates; D006454:Hemoglobins; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox