Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial

Br J Haematol. 2009 Apr;145(2):245-54. doi: 10.1111/j.1365-2141.2009.07609.x. Epub 2009 Feb 19.


A multicentre randomized open-label trial was designed to assess the effectiveness of long-term sequential deferiprone-deferoxamine (DFO-DFP) versus DFP alone to treat thalassaemia major (TM). DFP at 75 mg/kg, divided into three oral daily doses, for 4 d/week and DFO by subcutaneous infusion (8-12 h) at 50 mg/kg per day for the remaining 3 d/week was compared with DFP alone at 75 mg/kg, administered 7 d/week during a 5-year follow-up. The main outcome measures were differences between multiple observations of serum ferritin concentrations. Secondary outcomes were survival analysis, adverse events, and costs. Consecutive thalassaemia patients (275) were assessed for eligibility; 213 of these were randomized and underwent intention-to-treat analysis. The decrease of serum ferritin levels during the treatment period was statistically significant higher in sequential DFP-DFO patients compared with DFP-alone patients (P = 0.005). Kaplan-Meier survival analysis for the two chelation treatments did not show any statistically significant differences (long-rank test, P = 0.3145). Adverse events and costs were comparable between the groups. The trial results show that sequential DFP-DFO treatment compared with DFP alone significantly decreased serum ferritin concentration during treatment for 5 years without significant differences regarding survival, adverse events, or costs.

Trial registration: ClinicalTrials.gov NCT00733811.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Administration, Oral
  • Adolescent
  • Adult
  • Deferiprone
  • Deferoxamine / administration & dosage*
  • Deferoxamine / therapeutic use
  • Drug Therapy, Combination
  • Female
  • Ferritins / blood
  • Follow-Up Studies
  • Humans
  • Infusions, Subcutaneous
  • Iron Chelating Agents / administration & dosage*
  • Iron Chelating Agents / therapeutic use
  • Kaplan-Meier Estimate
  • Male
  • Pyridones / administration & dosage*
  • Pyridones / therapeutic use
  • Thalassemia / blood
  • Thalassemia / drug therapy*
  • Thalassemia / mortality
  • Treatment Outcome
  • Young Adult


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

Associated data

  • ClinicalTrials.gov/NCT00733811