Efficacy Study of the Use of Sequential DFP-DFO Versus DFP (SEQDFPDFO)

Azienda Ospedaliera V. Cervello

Status and phase: Completed; Phase 4

Conditions: Beta-Thalassemia; Thalassemia Major

Treatments: Drug: Deferiprone (DFP) and Deferoxamine (DFO); Drug: Deferiprone (DFP)

Study type: Interventional

Funder types: Other

Identifiers

NCT00733811

AOVCervello

Details and patient eligibility

About

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.

Full description

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

Enrollment: 213 patients

Sex: All

Ages: 13 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age

Exclusion criteria

• Known intolerance to one of the trial treatments
• Platelet count < 100,000/mm3 or or leukocyte count < 3,000/mm3
• Severe liver damage indicated by ascites
• Heart failure

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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213 participants in 2 patient groups

1

Experimental group

Description:

Sequential treatment including DFP at 75 mg/kg, divided into three oral daily doses, for four days per week and DFO by subcutaneous infusions (8-12h) at 50 mg/kg/day for the remaining three days per week

Treatment:

Drug: Deferiprone (DFP) and Deferoxamine (DFO)

2

Active Comparator group

Description:

Deferiprone alone at 75 mg/kg divided into three oral daily doses

Treatment:

Drug: Deferiprone (DFP)