Most deaths in thalassemia major (TM) result from cardiac complications due to iron overload. No data are available in literature about possible different changes in cardiac and liver iron in TM patients treated with sequential deferipron-deferoxamine (DFP-DFO) vs. deferasirox (DFX). Magnetic Resonance (MR) is the unique non invasive suitable technique to evaluated quantitatively this issue. Aims. The aim of this multi-centre study was to assess prospectively in the clinical practice the efficacy of the DFP-DFOvs. DFX in a cohort of TM patients by quantitative MR. Methods. Among the first 739 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, 253 patients performed a MR follow up study at 18±3 months according to the protocol. We evaluated prospectively the 25 patients treated with DFP-DFOvs. the 44 patients treated with DFX between the 2 MR scans. Myocardial and liver iron concentrations were measured by T2* multislice multiecho technique. Results. The doses of the sequential treatment were DFP 70±14 mg/kg/d for 4 d/w and DFO 42±8 mg/kg/d for 3 d/w, the dose of DFX was 26±6 mg/kg/d. Excellent/good levels of compliance were similar in the 2 groups (DFP-DFO 96%vs. DFX 100%; P=0.36). At baseline the 2 groups were homogeneous for cardiac and liver iron. Among the patients with no significant myocardial iron overload at baseline (global heart T2* ≥ 20 ms), there were no significant differences between groups to maintain the patients without myocardial iron overload (DFP-DFO 95%vs. DFX 96%; P=1.0). Among the patients with myocardial iron overload at baseline (global heart T2* <20 ms), only in the DFX group there was a significant improvement in the global heart T2* value (11±5 ms at baseline vs. 16±8 at 18±3 months, P=0.0001) and in the number of segment with a normal T2* value (P=0.003). The improvement in the global heart T2* was not significantly difference in the DFP-DFO vs. the DFX group (mean difference global heart T2* 2.2±4.1 ms vs. 4.6±4.8 P=0.2). The changes in the mean serum ferritin level were not significantly different between groups. In patients with liver iron overload at baseline (liver T2* <5.1 ms), the change in the liver T2* was not significant between groups (mean difference liver T2* 0.9±2.1 ms vs. 2.4±5.2; P=0.3).
A T2* MRI PROSPECTIVE SURVEY ON HEART AND LIVER IRON IN THALASSEMIA MAJOR PATIENTS TREATED WITH SEQUENTIAL DEFERIPRON-DESFERRIOXAMINE VERSUS DEFERASIROX
BORGNA, Caterina
2010
Abstract
Most deaths in thalassemia major (TM) result from cardiac complications due to iron overload. No data are available in literature about possible different changes in cardiac and liver iron in TM patients treated with sequential deferipron-deferoxamine (DFP-DFO) vs. deferasirox (DFX). Magnetic Resonance (MR) is the unique non invasive suitable technique to evaluated quantitatively this issue. Aims. The aim of this multi-centre study was to assess prospectively in the clinical practice the efficacy of the DFP-DFOvs. DFX in a cohort of TM patients by quantitative MR. Methods. Among the first 739 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, 253 patients performed a MR follow up study at 18±3 months according to the protocol. We evaluated prospectively the 25 patients treated with DFP-DFOvs. the 44 patients treated with DFX between the 2 MR scans. Myocardial and liver iron concentrations were measured by T2* multislice multiecho technique. Results. The doses of the sequential treatment were DFP 70±14 mg/kg/d for 4 d/w and DFO 42±8 mg/kg/d for 3 d/w, the dose of DFX was 26±6 mg/kg/d. Excellent/good levels of compliance were similar in the 2 groups (DFP-DFO 96%vs. DFX 100%; P=0.36). At baseline the 2 groups were homogeneous for cardiac and liver iron. Among the patients with no significant myocardial iron overload at baseline (global heart T2* ≥ 20 ms), there were no significant differences between groups to maintain the patients without myocardial iron overload (DFP-DFO 95%vs. DFX 96%; P=1.0). Among the patients with myocardial iron overload at baseline (global heart T2* <20 ms), only in the DFX group there was a significant improvement in the global heart T2* value (11±5 ms at baseline vs. 16±8 at 18±3 months, P=0.0001) and in the number of segment with a normal T2* value (P=0.003). The improvement in the global heart T2* was not significantly difference in the DFP-DFO vs. the DFX group (mean difference global heart T2* 2.2±4.1 ms vs. 4.6±4.8 P=0.2). The changes in the mean serum ferritin level were not significantly different between groups. In patients with liver iron overload at baseline (liver T2* <5.1 ms), the change in the liver T2* was not significant between groups (mean difference liver T2* 0.9±2.1 ms vs. 2.4±5.2; P=0.3).I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
