Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study

Br J Haematol. 2017 Jun;177(5):741-750. doi: 10.1111/bjh.14621. Epub 2017 Apr 17.


Iron chelation is controversial in higher risk myelodysplastic syndromes (HR-MDS), outside the allogeneic transplant setting. We conducted a retrospective, multicentre study in 51 patients with transfusion-dependent, intermediate-to-very high risk MDS, according to the revised international prognostic scoring system, treated with the oral iron chelating agent deferasirox (DFX). Thirty-six patients (71%) received azacitidine concomitantly. DFX was given at a median dose of 1000 mg/day (range 375-2500 mg) for a median of 11 months (range 0·4-75). Eight patients (16%) showed grade 2-3 toxicities (renal or gastrointestinal), 4 of whom (8%) required drug interruption. Median ferritin levels decreased from 1709 μg/l at baseline to 1100 μg/l after 12 months of treatment (P = 0·02). Seventeen patients showed abnormal transaminase levels at baseline, which improved or normalized under DFX treatment in eight cases. One patient showed a remarkable haematological improvement. At a median follow up of 35·3 months, median overall survival was 37·5 months. The results of this first survey of DFX in HR-MDS are comparable, in terms of safety and efficacy, with those observed in lower-risk MDS. Though larger, prospective studies are required to demonstrate real clinical benefits, our data suggest that DFX is feasible and might be considered in a selected cohort of HR-MDS patients.

Keywords: International Prognostic Scoring System; deferasirox; iron chelation; myelodyslastic syndromes; revised-International Prognostic Scoring System.

Publication types

  • Multicenter Study

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Aged, 80 and over
  • Benzoates / therapeutic use*
  • Chelation Therapy / methods*
  • Deferasirox
  • Erythrocyte Transfusion / statistics & numerical data
  • Female
  • Ferritins / metabolism
  • Humans
  • Iron Chelating Agents / therapeutic use*
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / drug therapy*
  • Retrospective Studies
  • Treatment Outcome
  • Triazoles / therapeutic use*


  • Benzoates
  • Iron Chelating Agents
  • Triazoles
  • Ferritins
  • Deferasirox