A randomized controlled 1-year study of daily deferiprone plus twice weekly desferrioxamine compared with daily deferiprone monotherapy in patients with thalassemia major.

Abstract

BACKGROUND AND OBJECTIVES:

The aim of this prospective, randomized, 1-year study was to compare the efficacy and safety of oral deferiprone (DFP) with those of combinations of parenteral desferrioxamine (DFO) with oral DFP.

DESIGN AND METHODS:

A total of 24 patients with thalassemia major were randomized to receive one of the following two treatments; DFP given at a daily dose of 75 mg/kg in combination with DFO (40-50 mg/kg twice weekly) (n=12) or as single agent (n=12). In addition, 12 patients treated with 40-50 mg/kg DFO 5 days weekly were included as a reference group without randomization. Changes in liver iron concentration (LIC) and serum ferritin (SF) were assessed; total iron excretion (TIE), urinary iron excretion (UIE) and iron balance were calculated. Cardiac function and toxicity were also examined.

DESIGN AND METHODS:

SF and LIC were significantly reduced after 1 year of combination therapy (p=0.01 and 0.07, respectively). A decrease of LIC was observed in all but one patient (87.5%) following the combination therapy but in only 42% of patients treated with DFP monotherapy. In the DFO reference group, a statistically significant decrease in LIC (p=0.01) associated with a substantial decrease in SF (p=0.08) was observed after 1 year. The combination regimen resulted in greater TIE compared to DFP monotherapy (p=0.08) and was the regimen associated with the highest iron balance compared to DFP monotherapy (p=0.04) or standard DFO treatment (p=0.006).

INTERPRETATIONS AND CONCLUSIONS:

The addition of subcutaneous DFO twice weekly to oral DFP 75 mg/kg is a highly efficacious and safe chelation therapy providing superior chelation activity to that of DFP and likely has an efficacy profile comparable to that of standard DFO.

• Authors: Yesim Aydinok, Zulal Ulger, Deniz Nart, Aysen Terzi, Nurten Cetiner, Gareth Ellis, Arthur Zimmermann, Chantal Manz
• Journal: Haematologica (Haematologica) Vol. 92 Issue 12 Pg. 1599-606 (Dec 2007) ISSN: 1592-8721 [Electronic] Italy
• PMID: 18055982 (Publication Type: Comparative Study, Journal Article, Randomized Controlled Trial)

Chemical References

• Pyridones
• Siderophores
• Deferiprone
• Ferritins
• Iron
• Deferoxamine

Topics

• Adolescent
• Adult
• Child
• Deferiprone
• Deferoxamine (administration & dosage)
• Female
• Ferritins (blood)
• Humans
• Iron (blood, urine)
• Liver (metabolism)
• Male
• Pyridones (administration & dosage)
• Siderophores (administration & dosage)
• Time Factors
• beta-Thalassemia (blood, drug therapy, urine)