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

From the Ege University, Faculty of Medicine, Department of Paediatric Haematology, Izmir, Turkey (YA, AT); Ege University, Faculty of Medicine, Department of Cardiology, Izmir, Turkey (ZU), Ege University, Faculty of Medicine, Department of Pathology, Izmir, Turkey (DN), Ege University, Faculty of Medicine, Research Laboratory, Izmir, Turkey (NC); Royal Free Hospital, Haematology Department, Hampstead, London, United Kingdom (GE); University of Berne, Institute of Pathology, Switzerland (AZ), Clinical R & D, Lipomed AG, Arlesheim, Switzerland (CM)

Correspondence: Yesim Aydinok, Ege University Faculty of Medicine, Department of Pediatric Hematology 35100 Bornova, Izmir, Turkey. E-mail: yesim.aydinok{at}ege.edu.tr

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.

Key words: thalassemia, deferiprone, desferrioxamine, combination therapy, iron overload, iron balance, liver iron concentration.