Deferiprone versus deferoxamine in sickle cell disease: results from a 5-year long-term Italian multi-center randomized clinical trial

Blood Cells Mol Dis. 2014 Dec;53(4):265-71. doi: 10.1016/j.bcmd.2014.04.004. Epub 2014 May 9.

Abstract

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.

Keywords: Chelation in Sickle-Cell-Disease; DFP versus DFO in Sickle-Cell-Disease; Effectiveness of Deferiprone in Sickle-Cell-Disease.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Sickle Cell / blood
  • Anemia, Sickle Cell / drug therapy*
  • Anemia, Sickle Cell / mortality
  • Anemia, Sickle Cell / pathology
  • Blood Transfusion
  • Child
  • Deferiprone
  • Deferoxamine / therapeutic use*
  • Female
  • Ferritins / blood
  • Humans
  • Iron / blood
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / blood
  • Iron Overload / drug therapy*
  • Iron Overload / mortality
  • Iron Overload / pathology
  • Italy
  • Linear Models
  • Male
  • Middle Aged
  • Pyridones / therapeutic use*
  • Survival Analysis

Substances

  • Iron Chelating Agents
  • Pyridones
  • Deferiprone
  • Ferritins
  • Iron
  • Deferoxamine