ASH 2018 — Iron chelation therapy boosts EFS in lower-risk MDS


  • Univadis
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Takeaway

  • Iron chelation therapy (ICT) with deferasirox prolonged event-free survival (EFS) vs placebo in patients with lower-risk myelodysplastic syndromes (MDS) and iron overload.

Why this matters

  • Most patients with low- or intermediate-1-risk MDS eventually require chronic red blood cell transfusions because of impaired hematopoiesis, resulting in iron overload that contributes to shortened survival.

Study design

  • TELESTO randomized, double-blind, phase 2 trial of 225 adults diagnosed with low or intermediate-1-risk MDS.
  • Patients were assigned in a 2:1 ratio to deferasirox (10-40 mg/kg/day) or placebo.
  • Funding: Novartis

Key results

  • Deferasirox was associated with a significantly longer median EFS vs placebo: 1440 vs 1091 days (HR, 0.64; P=.01).
  • 3-year estimated EFS rate was 61.5% with deferasirox vs 47.3% with placebo.
  • Median OS was not significantly different between the deferasirox and placebo groups (HR, 0.83; P=.2).
  • The improvement in outcomes occurred without an increase in toxicity.

Limitation

  • Possible difference in OS between the groups may have been obscured by the effects of ICT after discontinuation of study treatment.