Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children

被引：86
|
作者：
Strouse, John J. [1 ,2 ]
Heeney, Matthew M. [3 ,4 ]
机构：
[1] Johns Hopkins Univ, Sch Med, Div Pediat Hematol, Dept Pediat, Baltimore, MD 21205 USA
[2] Johns Hopkins Univ, Sch Med, Dept Med, Div Hematol, Baltimore, MD 21205 USA
[3] Childrens Hosp, Div Hematol Oncol, Dept Med, Boston, MA 02115 USA
[4] Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA 02115 USA
来源：
PEDIATRIC BLOOD & CANCER | 2012年 / 59卷 / 02期
基金：
美国国家卫生研究院;
关键词：
children; efficacy; hydroxyurea; sickle cell disease; FUNCTIONAL-ANALYSIS; YOUNG-CHILDREN; ANEMIA; TRIAL; HEMOGLOBIN; HYDROXYCARBAMIDE; PREVENTION; THERAPY; STROKE; HEALTH;
D O I：
10.1002/pbc.24178
中图分类号：
R73 [肿瘤学];
学科分类号：
100214 ;
摘要：
Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children. Pediatr Blood Cancer 2012;59:365371. (c) 2012 Wiley Periodicals, Inc.
引用
收藏
页码：365 / 371
页数：7
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