Hydroxyurea for Children with Sickle Cell Disease
Hydroxyurea for Children with Sickle Cell Disease
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DOI：
10.1016/j.hoc.2009.11.002
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发表时间：
2010-02-01
影响因子：
2.4
通讯作者：
Ware, Russell E.
中科院分区：
文献类型：
--
作者：
Heeney, Matthew M.;Ware, Russell E.
羟基脲疗法有望改善镰状细胞病 (SCD) 儿童的临床病程。羟基脲是一种原型治疗选择；它的副作用最小，具有相对较宽的治疗窗，并且具有解决镰状化、血管闭塞、溶血和器官损伤等病理生理途径的作用机制。关于羟基脲预防或减少器官功能障碍的能力的数据有限，并且羟基脲治疗的长期风险仍未完全确定。尽管正在进行解决长期问题的临床试验，但羟基脲仍然是一种有效但未得到充分利用的 SCD 疗法。
Hydroxyurea therapy offers promise for ameliorating the clinical course of children with sickle cell disease (SCD). Hydroxyurea is a prototypic therapeutic option; it can be administered with minimal side effects, has a relatively wide therapeutic window, and has mechanisms of action that address pathophysiologic pathways of sickling, vaso-occlusion, hemolysis, and organ damage. There are limited data regarding hydroxyurea's ability to prevent or diminish organ dysfunction, and the long-term risks of hydroxyurea therapy remain incompletely defined. Although clinical trials are underway to address long-term issues, hydroxyurea remains an effective but underutilized therapy for SCD.