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Updated: Jul 14, 2025
Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric
Mona Hamdy1, Amal El-Beshlawy2, Mônica P A Veríssimo3
1Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt.
Insights
Deferiprone and deferoxamine are comparable in reducing iron overload in children with sickle cell disease. This oral iron chelation therapy option shows no new safety concerns and may improve adherence.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Children with sickle cell disease (SCD) often require iron chelation therapy due to chronic transfusions.
- Limited comparative data exist for iron chelators deferiprone and deferoxamine in pediatric SCD patients.
Purpose of the Study:
- To compare the efficacy and safety of deferiprone versus deferoxamine in reducing iron overload in pediatric patients with SCD.
- To analyze post hoc data from the FIRST trial (NCT02041299) for children aged 17 years and younger.
Main Methods:
- Post hoc analysis of a randomized, open-label, phase 3b/4 study (FIRST).
- Included pediatric patients (≤17 years) with SCD or other anemias receiving deferiprone or deferoxamine.
- Evaluated changes in liver iron concentration, cardiac T2* MRI, and serum ferritin levels at 12 months.
Main Results:
- Both deferiprone and deferoxamine demonstrated comparable reductions in liver iron concentration (mean change: -3.3 vs -3.4 mg/g dw; p=0.8216).
- Cardiac T2* MRI showed similar relative mean changes (1.02 vs 0.95; p=0.0717).
- Common deferiprone adverse events included abdominal pain, vomiting, pyrexia, neutropenia, and elevated ALT/AST, all of which resolved.
Conclusions:
- Deferiprone is comparable to deferoxamine in reducing iron overload in pediatric SCD patients, consistent with adult findings.
- No new safety concerns were identified for deferiprone.
- Deferiprone offers an oral iron chelation option that may enhance adherence and improve outcomes in children.
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 cardiac T2* magnetic resonance imaging was 1.02 (21.8%) with deferiprone and 0.95 (19.5%) with deferoxamine (p = .0717); and the mean (standard error) change in serum ferritin levels was -133.0 (200.3) μg/L with deferiprone and -467.1 (244.1) μg/L with deferoxamine (p = .2924). The most common deferiprone-related adverse events (AEs) were upper abdominal pain (20.2%), vomiting (13.8%), pyrexia (9.6%), decreased neutrophil count (9.6%), increased alanine aminotransferase (ALT; 9.6%), and increased aspartate aminotransferase (AST; 9.6%). All cases of increased ALT, increased AST, and neutropenia resolved, most without intervention.
Conclusions:
This post hoc analysis of pediatric patients from FIRST corroborated previous findings in adults that deferiprone is comparable to deferoxamine in reducing iron overload. No new safety concerns were observed. Deferiprone is an oral chelation option that could improve adherence and outcomes in children.
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