Iron chelation therapy (ICT) in patients with lower-risk myelodysplastic syndromes (MDS) has not been evaluated in randomised studies.
The multicentre, randomised, double-blind, placebo-controlled TELESTO trial evaluated event-free survival (EFS) and safety of ICT in 225 iron-overloaded (serum ferritin >2247 pmol/L; prior recipients of 15-75 packed red blood cell units) patients with low or intermediate-1 risk MDS. Patients were enrolled from 60 centres across 16 countries.
Participants received deferasirox dispersible tablets 10-40 mg/kg/day (n=149) or placebo (n=76).
Median EFS was prolonged by approximately one year with deferasirox versus placebo (3.9 years verses 3.0 years; hazard ratio [HR] 0.64; 95% CI 0.42-0.96).
Adverse events occurred in 97.3 per cent of deferasirox recipients and 90.8 per cent of placebo recipients. Exposure-adjusted incidence rates of adverse events (≥15 events/100 patient treatment-years) were 24.7 versus 23.9 for diarrhoea, 21.8 versus 18.7 for pyrexia, 16.7 versus 22.7 for upper respiratory tract infection, and 15.9 versus 0.9 for increased serum creatinine concentration.
The authors say the findings support ICT in iron-overloaded patients with low to intermediate-1 risk MDS, with longer EFS compared with placebo and a clinically manageable safety profile.