Iron chelation therapy for children with transfusion-dependent β-thalassemia: How young is too young?

Autor: Forni GL; ForAnemia Foundation, Genoa, Italy., Kattamis A; First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece., Kuo KHM; Division of Hematology, University of Toronto, Toronto, Ontario, Canada., Maggio A; Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo, Italy., Sheth S; Division of Hematology and Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, New York, USA., Taher AT; Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon., Viprakasit V; Department of Pediatrics & Thalassemia Center, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Jazyk: angličtina
Zdroj: Pediatric blood & cancer [Pediatr Blood Cancer] 2024 Aug; Vol. 71 (8), pp. e31035. Date of Electronic Publication: 2024 May 16.
DOI: 10.1002/pbc.31035
Abstrakt: In this review, we provide a summary of evidence on iron overload in young children with transfusion-dependent β-thalassemia (TDT) and explore the ideal timing for intervention. Key data from clinical trials and observational studies of the three available iron chelators deferoxamine, deferiprone, and deferasirox are also evaluated for inclusion of subsets of young children, especially those less than 6 years of age. Evidence on the efficacy and safety of iron chelation therapy for children ≥2 years of age with transfusional iron overload is widely available. New data exploring the risks and benefits of early-start iron chelation in younger patients with minimal iron overload are also emerging.
(© 2024 Wiley Periodicals LLC.)
Databáze: MEDLINE
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