Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study)
P Joy Ho, Lay Tay, Juliana Teo, Paula Marlton, Andrew Grigg, Tim St Pierre, Greg Brown, Caro-Anne Badcock, Robert Traficante, Othon L Gervasio, Donald K Bowden
EUROPEAN JOURNAL OF HAEMATOLOGY | WILEY | Published : 2017
OBJECTIVES: To assess the effect of iron chelation therapy with deferasirox on cardiac iron and function in patients with transfusion-dependent thalassemia major, sickle cell disease (SCD), and myelodysplastic syndromes (MDS). METHODS: This phase IV, single-arm, open-label study over 53 wk evaluated the change in cardiac and liver iron load with deferasirox (up to 40 mg/kg/d), measured by magnetic resonance imaging (MRI). RESULTS: Cardiac iron load (myocardial T2*) significantly improved (P = 0.002) overall (n = 46; n = 36 thalassemia major, n = 4 SCD, n = 6 MDS). Results were significant for patients with normal and moderate baseline cardiac iron (P = 0.017 and P = 0.015, respectively), but..View full abstract
The authors thank Iris Alhassid, Jane Lawson (Novartis Pharmaceuticals Australia), and Gillian Ryan (formerly of Novartis Pharmaceuticals Australia) for ongoing clinical support. We also thank Dr Ann Solterbeck for the statistical support (Statistical Revelations Pty Ltd, Melbourne); Joanne Zacharopoulos, Mary Nteris, and Barbara Hicks for medical editorial assistance (INC Research); Rebecca Helson for editorial assistance (Mudskipper Business Ltd); and Dr Stephen Worthley for the LVEF assessments (Royal Adelaide Hospital). The MILE study and work presented here were sponsored and funded by Novartis Pharmaceuticals Corporation. Financial support for editorial assistance was provided by Novartis Pharmaceuticals Corporation.