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

被引:2
作者
Forni, Gian Luca [1 ]
Kattamis, Antonis [2 ]
Kuo, Kevin H. M. [3 ]
Maggio, Aurelio [4 ]
Sheth, Sujit [5 ]
Taher, Ali T. [6 ]
Viprakasit, Vip [7 ,8 ]
机构
[1] ForAnemia Fdn, Via Garibaldi 7,3C, I-16124 Genoa, Italy
[2] Natl & Kapodistrian Univ Athens, Dept Pediat 1, Athens, Greece
[3] Univ Toronto, Div Hematol, Toronto, ON, Canada
[4] AOOR Villa Sofia V Cervello, Campus Haematol Franco & Piera Cutino, Palermo, Italy
[5] Weill Cornell Med, Dept Pediat, Div Hematol & Oncol, New York, NY USA
[6] Amer Univ Beirut, Med Ctr, Dept Internal Med, Beirut, Lebanon
[7] Mahidol Univ, Siriraj Hosp, Fac Med, Dept Pediat, Bangkok, Thailand
[8] Mahidol Univ, Siriraj Hosp, Fac Med, Thalassemia Ctr, Bangkok, Thailand
关键词
ferritin; heart; iron chelation; iron overload; liver; thalassemia; PEDIATRIC-PATIENTS; OPEN-LABEL; DEFERASIROX; EFFICACY; SAFETY; DEFERIPRONE; DESFERRIOXAMINE; TOLERABILITY; MULTICENTER; OVERLOAD;
D O I
10.1002/pbc.31035
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
In this review, we provide a summary of evidence on iron overload in young children with transfusion-dependent beta-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.
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页数:7
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