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.
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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)
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Chemical References |
- Pyridones
- Siderophores
- Deferiprone
- Ferritins
- Iron
- Deferoxamine
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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)
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