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Background In thalassemia intermedia (TI) patients no observational study prospectively evaluated in the real life the efficacy of the desferrioxamine (DFO) therapy in removing or preventing myocardial iron overload. The efficacy endpoint of this study is represented by the changes in cardiac T2* values and left ventricular (LV) function parameters in non-transfusion dependent (NTD) TI patients after 18 months of desferrioxamine therapy.

Background Recently two novels indicators of left ventricular (LV) performance assessed by Cardiovascular Magnetic Resonance (CMR) have been introduced: the LV global function index (LVGFI) and the LV mass/volume ratio (LVMVR). The LVGFI combines LV stroke volume, end-systolic and end diastolic volumes, as well as LV mass, integrating structural as well as mechanical behaviour. Elevated LVMVR is indicative of concentric remodelling. A LVGFI 1 were shown to be associated with the occurrence of cardiovascular events in no-thalassemic populations. This retrospective cohort study aimed to systematically evaluate in a large historical cohort of thalassemia major (TM) in the CMR era whether the LVGFI and the LVMVR were associated with a higher risk of heart failure.

Results Three groups of patients were identified: 27 patients (13 females, mean age 40.12±10.31 years) treated with desferioxamine (DFO mean dosage 37.52±8.69 mg/kg/ die), 23 patients (14 females, mean age 34.73±10.67 years) treated with deferiprone (DFPdosage 71.70 ±14.46mg/kg/die) and 14 patients (9 females, mean age 36.63±10.92 years) treated with deferasirox (DFX mean dosage 27.75±5.04 mg/kg/die). Excellent/good levels of compliance were similar in the DFO (92.6%), DFP (100%) and DFX (100%) groups (P=0.345). The mean starting age of regular transfusion was 14.73 ±15.89 years. At baseline in DFO group two patients (7.4%) showed a global heart T2*

Background There are no prospective studies comparing the effectiveness of the three iron chelators commercially available in preventing or decreasing iron overload in the heart in pediatric thalassemia major (TM) patients. Our aim was to evaluate the changes in cardiac iron and function by quantitative magnetic resonance imaging (MRI) over a follow-up (FU) of 18 months in pediatric TM patients treated with one of the 3 available iron chelators in monotherapy or non-chelated. Methods Among the first 1611 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, we considered pediatric patients who had maintained the same chelation regimen between the two MRI scans. Myocardial iron overload (MIO) was quantified by a multislice multiecho T2* sequence. Biventricular function parameters were evaluated by cine images. Due to the low sample size, no inter-treatment comparisons were performed.

while one showed cardiac iron (global heart T2*=12.3 ms). Due mainly to technical reasons, biventricular function was assesses at both baseline and FU MRIs in 22 patients. At baseline 6 patients showed a reduced left ventricular ejection fraction (LVEF) and 4 of them recovered at the FU. All patients had a baseline global heart T2*>20 ms (one with 2 segmental T2* values