Optimising iron chelation therapy with deferasirox for non transfusion dependent thalassaemia patients: 1 year results from the THETIS study.
Efficacy and safety of iron chelation therapy with deferasirox in iron overloaded non transfusion dependent thalassaemia (NTDT) patients were established in the THALASSA study. THETIS, an open label, single arm, multicentre, Phase IV study, added to this evidence by investigating earlier dose escalation by baseline liver iron concentration (LIC) (week 4: escalation according to baseline LIC; week 24: adjustment according to LIC response, maximum 30mg/kg/day) The primary efficacy endpoint was absolute change in LIC from baseline to week 52. 134 iron overloaded non transfusion dependent anaemia patients were enrolled and received deferasirox starting at 10mg/kg/day. Mean actual dose+ SD over 1year was 14.70+ 5.48mg/kg/day. At week 52, mean LIC+ SD decreased significantly from 15.13+ 10.72mg Fe/g dw at baseline to 8.46+ 6.25mg Fe/g dw (absolute change from baseline, 6.68+ 7.02mg Fe/g dw [95% CI: 7.91, 5.45] P<0.0001) Most common drug related adverse events were gastrointestinal: abdominal discomfort, diarrhoea and nausea (n=6 each) There was one death (pneumonia, not considered drug related) With significant and clinically relevant reductions in iron burden alongside a safety profile similar to that in THALASSA, these data support earlier escalation with higher deferasirox doses in iron overloaded non transfusion dependent anaemia patients.
Blood cells, molecules diseases