Iron chelation in thalassemia: combined or monotherapy? The Egyptian experience

Ann Hematol. 2008 Jul;87(7):545-50. doi: 10.1007/s00277-008-0471-2. Epub 2008 Mar 20.


Patients with thalassemia major requiring regular blood transfusions accumulate iron that is toxic to the heart, liver, and endocrine systems. The following prospective, randomized trial was carried out to determine the effectiveness, in children and young adults, of combined deferiprone (DFP) and deferoxamine (DFO) in reducing transfusional iron overload compared to either drug alone and to assess the safety and tolerability of DFP. Sixty-six patients were randomized into three treatment arms: daily DFP combined with DFO twice weekly; daily DFP only; and DFO only 5 days/week. Fifty-six patients completed the 54 weeks and were assessed by different indices. A significant reduction of liver iron concentration and serum ferritin was observed in all three arms while significant reduction of liver iron score was observed in patients on combination therapy only. Cardiac function did not significantly change in any arm. Compliance improved in patients who received combined therapy. Toxicity of DFP was mild to moderate and acceptable; most commonly, transient arthropathy and nausea/vomiting were observed. Thus, combination therapy has shown to be effective in reducing iron overload in thalassemia patients.

Publication types

  • Comparative Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Chelation Therapy / adverse effects
  • Chelation Therapy / methods*
  • Child
  • Child, Preschool
  • Deferiprone
  • Deferoxamine / administration & dosage
  • Deferoxamine / adverse effects
  • Deferoxamine / therapeutic use*
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Egypt / epidemiology
  • Female
  • Ferritins / analysis
  • Ferritins / blood
  • Humans
  • Iron Chelating Agents / administration & dosage
  • Iron Chelating Agents / adverse effects
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / drug therapy*
  • Iron Overload / etiology
  • Iron Overload / prevention & control
  • Liver / pathology
  • Male
  • Patient Compliance
  • Prospective Studies
  • Pyridones / administration & dosage
  • Pyridones / adverse effects
  • Pyridones / therapeutic use*
  • Thalassemia / complications
  • Thalassemia / drug therapy*
  • Thalassemia / epidemiology
  • Transfusion Reaction


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