Researchers discovered that deferiprone and deferoxamine both have acceptable efficacy and safety profiles for treating transfusional iron overload in sickle cell disease (SCD) and other anemias, as published in Blood Advances.
Many patients with SCD and other anemias are treated with chronic blood transfusions. The risk of this form of treatment is iron overload, which is then treated with chelation therapy since the body does not have a natural mechanism to excrete excess iron.
In thalassemia, the iron chelator deferiprone is often used, but data for its use in SCD is limited. Deferoxamine is an iron chelator approved for use in the US to treat chronic transfusional iron overload in SCD and other rare anemias. However, this drug needed to be administered parenterally over 8 to 12 hours per day, which some patients find to be a significant treatment burden.
The researchers thus decided to compare the 2 iron chelators for use in patients with SCD. They conducted a late-phase, multicenter, randomized, and open-label study at 34 centers in 8 countries. Eligible participants (n=300) were randomly assigned to receive either deferiprone (n=200) or deferoxamine (n=100) for up to 12 months in a 2:1 ratio.
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The safety of both drugs was assessed, including parameters such as the frequency and duration of any adverse events. “The primary efficacy endpoint was the change from baseline to 12 months in [liver iron content] as measured by R2* MRI [magnetic resonance imaging],” the research team wrote in assessing their efficacy.
“Secondary efficacy endpoints were the change from baseline to 12 months in cardiac iron as assessed by cardiac T2* MRI, serum ferritin levels, and patient-reported quality of life assessed by the [36-item Short Form] and [Child Health Questionnaire–Parent Form 50] questionnaires, respectively, for adults and parents/guardians of minors.”
The results demonstrated that the efficacy of deferiprone was not inferior to deferoxamine in all 3 measures of iron load (liver iron concentration, cardiac iron, and serum ferritin). In addition, deferiprone was generally well-tolerated, and the overall rate of adverse events was the same in both treatment groups. No significant difference in the quality of life surveys was reported in both treatment cohorts.
“Deferiprone provides a new treatment option for patients with SCD and other rare anemias with transfusional iron overload. This oral iron chelator effectively reduced transfusional iron overload, and the effect was noninferior to subcutaneously infused deferoxamine,” the researchers concluded.
Kwiatkowski JL, Hamdy M, El Beshlawy A, et al. Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label, noninferiority study. Blood Adv. Published online December 2, 2021. doi:10.1182/bloodadvances.2021004938