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