Efficacy Study of the Use of Sequential DFP-DFO Versus DFP

Overview

Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published. This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.

The trial was designed as a multicentre randomised open-label trial with blinded data management and data analyses, to assess whether either treatment was superior to the other. The trial was performed on behalf of the Italian Society for the study of Thalassemia and Haemoglobinopathies (SoSTE). The investigators initiated, carried out, and controlled the trial, which was conducted without influence of the non-commercial sponsor.16

Conditions