Deferiprone Is Comparable With Deferoxamine in Pediatric Sickle Cell Disease

Researchers reported that deferiprone safety and efficacy was comparable with deferoxamine for treating iron overload in pediatric patients, according to a post hoc analysis published in Pediatric Blood & Cancer.

Researchers compared the safety and efficacy of deferiprone and deferoxamine in patients aged 17 years or younger with sickle cell disease or other anemias who participated in the phase IIIb/IV FIRST trial. Deferoxamine is injected subcutaneously while deferiprone is an oral agent.

Children who receive frequent blood transfusions to manage sickle cell disease often require iron chelation therapy to reduce iron overload. The researchers suggested that “deferiprone is an oral chelation option that could improve adherence and outcomes in children.”

Deferiprone Outcomes Consistent with Deferoxamine in Pediatric SCD

The analysis included 142 pediatric patients who were treated with deferiprone or deferoxamine in FIRST. The deferiprone group had a mean age of 10.5 years and the deferoxamine group had a mean age of 11.7 years. After 12 months of treatment, the mean change from baseline in liver iron concentration (LIC) in the deferiprone group was -3.3 mg/g dry weight compared with -3.4 mg/g dry weight in the deferoxamine group (P=.8216).

Magnetic resonance imaging data showed the relative mean change in log cardiac T2* was 1.02 (coefficient of variation, 21.8%) with deferiprone versus 0.95 (coefficient of variation, 19.5%) with deferoxamine (P=.0717). Additionally, the mean changes in serum ferritin levels were -133.0 μg/L (standard error, 200.3) and -467.1 μg/L (standard error, 244.1) with deferiprone and deferoxamine, respectively.

Adverse events related to deferiprone were most commonly upper abdominal pain (20.2%), vomiting (13.8%), pyrexia (9.6%), increased alanine aminotransferase (9.6%), increased aspartate aminotransferase (9.6%), and decreased neutrophil count (9.6%). The authors noted most neutrophil, aspartate aminotransferase, and alanine aminotransferase changes were resolved without intervention.

Overall, the authors of the analysis concluded that “this post hoc subgroup analysis of pediatric patients receiving chronic transfusions for [sickle cell disease] or other anemias corroborated previous findings that deferiprone is comparable to deferoxamine in reducing [liver iron concentration].”

Reference

Hamdy M, El-Beshlawy A, Veríssimo MPA, et al. Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: pediatric subgroup analysis of the randomized, open-label FIRST study. Pediatr Blood Cancer. 2023;e30711. doi:10.1002/pbc.30711

Related: Characterizing Infections in Children With Sickle Cell Disease Over Time