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


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.


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).


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.
Aydinok
Yesim
Y
Ege University, Faculty of Medicine, Department of Pediatric Hematology 35100 Bornova, Izmir, Turkey. yesim.aydinok@ege.edu.tr
Ulger
Zulal
Z
Nart
Deniz
D
Terzi
Aysen
A
Cetiner
Nurten
N
Ellis
Gareth
G
Zimmermann
Arthur
A
Manz
Chantal
C
D000293:Adolescent; D000328:Adult; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage*; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000097:blood / Q000652:urine; D008099:Liver / Q000378:metabolism; D008297:Male; D011728:Pyridones / Q000008:administration & dosage*; D017262:Siderophores / Q000008:administration & dosage*; D013997:Time Factors; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000652:urine
D011728:Pyridones; D017262:Siderophores; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron; D003676:Deferoxamine