Contributions
Abstract: EP1314
Type: E-Poster Presentation
Session title: Thalassemias
Background
The combination therapy with deferiprone (DFP) and desferrioxamine (DFO) was associated with an overall improvement in glucose metabolism disorders in β‐thalassaemia, but no data are available about its efficacy in removing pancreatic iron.
Aims
The aim of this multi-center study, conducted in a large clinical observational setting of thalassemia major (TM) patients, was to prospectively evaluate the effects of combined DFO+DFP therapy versus both oral iron-chelators in monotherapy on pancreatic iron over a follow-up of 18 months.
Methods
Among the first 1176 TM patients consecutively enrolled in the E-MIOT (Extension-Myocardial Iron Overload in Thalassemia) network, 416 patients had a FU at 18±3 months. We selected those who had been received combined regimen (N=28) or DFP (N=61) and deferasirox-DFX (N=159) monotherapies between the two MRI scans. Pancreatic iron overload was quantified by multi-slice multiecho T2* technique; measurements were performed over pancreatic head, body and tail and global value was the mean.
Results
The comparison was restricted to patients with a normal baseline global pancreatic cut off value (T2*<26 ms): 28 in the combined DFO+DFP group, 54 in the DFP group and 129 in the DFX group.
The dosages were: 1) DFP in combined regimen 84.8±14.9 mg/kg body weight per day with a frequency of 6.9±0.6 days/week and DFO in combined regimen 37.9±7.8 mg/kg body weight per day with a frequency of 3.9±1.7 days/week; 2) DFP in monotherapy 84.5±14.3 mg/kg body per day; 3) DFX in monotherapy 24.9±7.1 mg/kg body per day if in dispersible tablets (N=98) and 18.3±4.4 mg/kg body per day if in film-coated tablets (N=31). The percentage of patients with excellent/good levels of compliance to the active chelation treatment was significantly lower in the combined DFP+DFO group versus both the DFP group (82.1% vs 98.1%; P=0.016) and the DFX group (82.1% vs 99.2%; P=0.001).
Global pancreatic T2* values were significantly lower in the combined DFO+DFP group compared to both DFP and DFX groups. A significant negative association was detected between changes in global pancreas T2* values and baseline global pancreas T2* values (R=-0.192; P=0.005). So, the % changes in global pancreas T2* values, normalized for the baseline values, were considered.
In no treatment group there was a correlation between the % changes in global pancreas T2* values and the iron chelator dosage.
The % changes in global pancreas T2* values were significantly higher in the combined DFO+DFP group versus both DFP (P=0.036) and DFX groups (P=0.030) (Figure 1).
Conclusion
Prospectively in TM patients at the dosages used in the real world, combined DFP+DFO was significantly more effective in reducing pancreatic iron versus both DFP and DFX in monotherapy.
Keyword(s): Chelation, Iron overload, Pancreas, Thalassemia
Abstract: EP1314
Type: E-Poster Presentation
Session title: Thalassemias
Background
The combination therapy with deferiprone (DFP) and desferrioxamine (DFO) was associated with an overall improvement in glucose metabolism disorders in β‐thalassaemia, but no data are available about its efficacy in removing pancreatic iron.
Aims
The aim of this multi-center study, conducted in a large clinical observational setting of thalassemia major (TM) patients, was to prospectively evaluate the effects of combined DFO+DFP therapy versus both oral iron-chelators in monotherapy on pancreatic iron over a follow-up of 18 months.
Methods
Among the first 1176 TM patients consecutively enrolled in the E-MIOT (Extension-Myocardial Iron Overload in Thalassemia) network, 416 patients had a FU at 18±3 months. We selected those who had been received combined regimen (N=28) or DFP (N=61) and deferasirox-DFX (N=159) monotherapies between the two MRI scans. Pancreatic iron overload was quantified by multi-slice multiecho T2* technique; measurements were performed over pancreatic head, body and tail and global value was the mean.
Results
The comparison was restricted to patients with a normal baseline global pancreatic cut off value (T2*<26 ms): 28 in the combined DFO+DFP group, 54 in the DFP group and 129 in the DFX group.
The dosages were: 1) DFP in combined regimen 84.8±14.9 mg/kg body weight per day with a frequency of 6.9±0.6 days/week and DFO in combined regimen 37.9±7.8 mg/kg body weight per day with a frequency of 3.9±1.7 days/week; 2) DFP in monotherapy 84.5±14.3 mg/kg body per day; 3) DFX in monotherapy 24.9±7.1 mg/kg body per day if in dispersible tablets (N=98) and 18.3±4.4 mg/kg body per day if in film-coated tablets (N=31). The percentage of patients with excellent/good levels of compliance to the active chelation treatment was significantly lower in the combined DFP+DFO group versus both the DFP group (82.1% vs 98.1%; P=0.016) and the DFX group (82.1% vs 99.2%; P=0.001).
Global pancreatic T2* values were significantly lower in the combined DFO+DFP group compared to both DFP and DFX groups. A significant negative association was detected between changes in global pancreas T2* values and baseline global pancreas T2* values (R=-0.192; P=0.005). So, the % changes in global pancreas T2* values, normalized for the baseline values, were considered.
In no treatment group there was a correlation between the % changes in global pancreas T2* values and the iron chelator dosage.
The % changes in global pancreas T2* values were significantly higher in the combined DFO+DFP group versus both DFP (P=0.036) and DFX groups (P=0.030) (Figure 1).
Conclusion
Prospectively in TM patients at the dosages used in the real world, combined DFP+DFO was significantly more effective in reducing pancreatic iron versus both DFP and DFX in monotherapy.
Keyword(s): Chelation, Iron overload, Pancreas, Thalassemia