Kwiatkowski Janet L
The Children's Hospital of Philadelphia, Division of Hematology and University of Pennsylvania School of Medicine, Philadelphia, PA, USA.
J Blood Med. 2011;2:135-49. doi: 10.2147/JBM.S13065. Epub 2011 Sep 21.
Regular red cell transfusion therapy ameliorates disease-related morbidity and can be lifesaving in patients with various hematological disorders. Transfusion therapy, however, causes progressive iron loading, which, if untreated, results in endocrinopathies, cardiac arrhythmias and congestive heart failure, hepatic fibrosis, and premature death. Iron chelation therapy is used to prevent iron loading, remove excess accumulated iron, detoxify iron, and reverse some of the iron-related complications. Three chelators have undergone extensive testing to date: deferoxamine, deferasirox, and deferiprone (although the latter drug is not currently licensed for use in North America where it is available only through compassionate use programs and research protocols). These chelators differ in their modes of administration, pharmacokinetics, efficacy with regard to organ-specific iron removal, and adverse-effect profiles. These differential properties influence acceptability, tolerability and adherence to therapy, and, ultimately, the effectiveness of treatment. Chelation therapy, therefore, must be individualized, taking into account patient preferences, toxicities, ongoing transfusional iron intake, and the degree of cardiac and hepatic iron loading.
定期红细胞输血疗法可改善与疾病相关的发病率，对于患有各种血液系统疾病的患者而言可能挽救生命。然而，输血疗法会导致铁逐渐蓄积，如果不进行治疗，会导致内分泌病、心律失常和充血性心力衰竭、肝纤维化以及过早死亡。铁螯合疗法用于预防铁蓄积、清除过量蓄积的铁、使铁解毒，并逆转一些与铁相关的并发症。迄今为止，已有三种螯合剂经过了广泛测试：去铁胺、地拉罗司和去铁酮（尽管后一种药物目前在北美未获许可使用，在那里仅通过同情用药计划和研究方案才能获得）。这些螯合剂在给药方式、药代动力学、特定器官铁清除的疗效以及不良反应方面存在差异。这些不同特性会影响对治疗的接受度、耐受性和依从性，并最终影响治疗效果。因此，螯合疗法必须个体化，要考虑患者的偏好、毒性、持续的输血铁摄入量以及心脏和肝脏铁负荷程度。
