Authors
Mona Hamdy, Amal El‐Beshlawy, Mônica PA Veríssimo, Julie Kanter, Baba Inusa, Suzan Williams, David Lee, Noemi Toiber Temin, Caroline Fradette, Fernando Tricta, Fatma SE Ebeid, Janet L Kwiatkowski, Mohsen S Elalfy
Publication date
2024/1
Journal
Pediatric Blood & Cancer
Volume
71
Issue
1
Pages
e30711
Description
Background
Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of the iron chelator deferiprone versus deferoxamine in children with SCD.
Methods
This post hoc analysis of the phase 3b/4, randomized, open‐label FIRST (Ferriprox in Patients with IRon Overload in Sickle Cell Disease Trial) study (NCT02041299) included patients 17 years and younger with SCD or other anemias receiving deferiprone or deferoxamine.
Results
Overall, 142 patients were evaluated; mean ages were 10.5 and 11.7 years in the deferiprone and deferoxamine groups, respectively. At 12 months: mean change from baseline in liver iron concentration was −3.3 mg/g dry weight (dw) with deferiprone and −3.4 mg/g dw with deferoxamine (p = .8216); relative mean change (coefficient of variation %) in log …
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