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RESEARCH PRODUCT
Deferiprone versus Deferoxamine in Sickle Cell Disease: Results from a 5-year long-term Italian multi-center randomized clinical trial
Calvaruso GiusiVitrano AngelaDi Maggio RosarioBallas SamirSteinberg Martin HRigano PaoloSacco MassimilianoTelfer PaulRenda DismaBarone RitaAurelio MaggioCarollo Antonellasubject
AdultMalemedicine.medical_specialtyIron OverloadBlood transfusionAdolescentPyridonesAnemiaIronmedicine.medical_treatmentAnemia Sickle CellDeferoxamineIron Chelating AgentsGastroenterologylaw.inventionchemistry.chemical_compoundBasal (phylogenetics)Randomized controlled triallawInternal medicineHumansMedicineBlood TransfusionDeferiproneChildMolecular Biologybusiness.industryCell BiologyHematologyMiddle Agedmedicine.diseaseSurvival AnalysisSurgeryDeferoxamineItalychemistrySupportive psychotherapyFerritinsCohortLinear ModelsMolecular MedicineFemalebusinessDeferipronemedicine.drugdescription
Blood transfusion and iron chelation currently represent a supportive therapy to manage anemia, vasculopathy and vaso-occlusion crises in Sickle-Cell-Disease. Here we describe the first 5-year long-term randomized clinical trial comparing Deferiprone versus Deferoxamine in patients with Sickle-Cell-Disease. The results of this study show that Deferiprone has the same effectiveness as Deferoxamine in decreasing body iron burden, measured as repeated measurements of serum ferritin concentrations on the same patient over 5-years and analyzed according to the linear mixed-effects model (LMM) (p=0.822). Both chelators are able to decrease, significantly, serum ferritin concentrations, during 5-years, without any effect on safety (p=0.005). Moreover, although the basal serum ferritin levels were higher in transfused compared with non-transfused group (p=0.031), the changes over time in serum ferritin levels were not statistically significantly different between transfused and non-transfused cohort of patients (p=0.389). Kaplan-Meier curve, during 5-years of study, suggests that Deferiprone does not alter survival in comparison with Deferoxamine (p=0.38). In conclusion, long-term iron chelation therapy with Deferiprone was associated with efficacy and safety similar to that of Deferoxamine. Therefore, in patients with Sickle-Cell-Disease, Deferiprone may represent an effective long-term treatment option.
year | journal | country | edition | language |
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2014-03-18 | Blood Cells, Molecules, and Diseases |