Beta-Thalassemia, Thalassemia Major
Conditions
Keywords
thalassemia major, chelation treatment, secondary hemochromatosis
Brief summary
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.
Detailed 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
Interventions
DRUGDeferiprone (DFP) and Deferoxamine (DFO)
Sequential treatment including DFP at 75 mg/kg for four days per week and DFO by subcutaneous infusions (8-12h) at 50mg/kg/day for the remaining three days per week
DFP alone at 75 mg/kg divided into three oral daily doses
Sponsors
Azienda Ospedaliera V. Cervello
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
13 Years to 60 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| difference between multiple observations of the serum ferritin concentrations | five-year treatment |
Secondary
| Measure | Time frame |
|---|---|
| the difference between the T2* signal at magnetic resonance imaging (MRI) of heart and liver at T0 and T1 time (see below); survival analysis; adverse events; treatment failures; and costs. | five years |
Countries
Italy
Outcome results
None listed