Abstract

BackgroundDue to the limited data available in literature, the aim of this multi-centre study was to prospectively compare in thalassemia major (TM) patients the efficacy of combined deferiprone (DFP) and deferoxamine (DFO) regimen versus either DFP and DFO in monotherapy by cardiovascular magnetic resonance (CMR) over a follow up of 18 months.MethodsAmong the first 1135 TM patients in the MIOT (Myocardial Iron Overload in Thalassemia) network, we evaluated those who had received either combined regimen (DFO + DFP, N=51) or DFP (N=39) and DFO (N=74) monotherapies between the two CMR scans. Iron overload was measured by T2* multiecho technique. Biventricular function parameters were quantitatively evaluated by cine images.ResultsThe percentage of patients that maintained a normal global heart T2* value was comparable between DFP+DFO versus both monotherapy groups. Among the patients with myocardial iron overload at baseline, the changes in the global heart T2* and in biventricular function were not significantly different in DFP+DFO compared with the DFP group. The improvement in the global heart T2* was significantly higher in the DFP+DFO than the DFO group, without a difference in biventricular function. Among the patients with hepatic iron at baseline, the decrease in liver iron concentration values was significantly higher with combination therapy than with either monotherapy group.ConclusionsIn TM patients at the dosages used in the real world, the combined DFP+DFO regimen was more effective in removing cardiac iron than DFO, and was superior in clearing hepatic iron than either DFO or DFP monotherapy. Combined therapy did not show an additional effect on heart function over DFP.

Highlights

  • Due to the limited data available in literature, the aim of this multi-centre study was to prospectively compare in thalassemia major (TM) patients the efficacy of combined deferiprone (DFP) and deferoxamine (DFO) regimen versus either DFP and DFO in monotherapy by cardiovascular magnetic resonance (CMR) over a follow up of 18 months

  • No long term data are available in literature about observational prospective comparisons of the effects on cardiac iron and function and liver iron in TM patients treated with combined deferiprone and deferoxamine (DFP+DFO) versus DFP in monotherapy

  • Among the first 1135 TM patients enrolled in the Myocardial Iron Overload in Thalassemia (MIOT) project, 392 performed a CMR follow up study at 18 ± 3 months, according to the protocol

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Summary

Introduction

Due to the limited data available in literature, the aim of this multi-centre study was to prospectively compare in thalassemia major (TM) patients the efficacy of combined deferiprone (DFP) and deferoxamine (DFO) regimen versus either DFP and DFO in monotherapy by cardiovascular magnetic resonance (CMR) over a follow up of 18 months. In the same population a prospective study showed that combined chelation therapy DFP+DFO effectively reduced myocardial iron and improved cardiac function in patients with severe myocardial siderosis and impaired LV function [5]. Another small sized one-year study using changes in liver iron concentration (LIC) assessed by biopsies showed the superiority of the combined DFP+DFO therapy with respect to the DFP alone [6]. No long term data are available in literature about observational prospective comparisons of the effects on cardiac iron and function and liver iron in TM patients treated with combined DFP+DFO versus DFP in monotherapy

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