Authors
Mohsen S Elalfy, Mona Hamdy, Amira Adly, Fatma SE Ebeid, Noemi Toiber Temin, Anna Rozova, David Lee, Caroline Fradette, Fernando Tricta
Publication date
2023/9
Journal
American Journal of Hematology
Volume
98
Issue
9
Pages
1415-1424
Publisher
John Wiley & Sons, Inc.
Description
Children with transfusion‐dependent thalassemia (TDT) require regular blood transfusions that, without iron‐chelation therapy, lead to iron‐overload toxicities. Current practice delays chelation therapy (late‐start) until reaching iron overload (serum ferritin ≥1000 μg/L) to minimize risks of iron‐depletion. Deferiprone's distinct pharmacological properties, including iron‐shuttling to transferrin, may reduce risks of iron depletion during mild‐to‐moderate iron loads and iron overload/toxicity in children with TDT. The early‐start deferiprone (START) study evaluated the efficacy/safety of early‐start deferiprone in infants/young children with TDT. Sixty‐four infants/children recently diagnosed with beta‐thalassemia and serum ferritin (SF) between 200 and 600 μg/L were randomly assigned 1:1 to receive deferiprone or placebo for 12 months or until reaching SF‐threshold (≥1000 μg/L at two consecutive visits …
Total citations
2023202411