Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA.
Hematology Am Soc Hematol Educ Program. 2011;2011:451-8. doi: 10.1182/asheducation-2011.1.451.
Whereas RBC transfusion therapy is lifesaving in thalassemia, obligatory iron loading accompanies such treatment and chelation therapy to remove and detoxify iron resulting from these chronic transfusions must therefore be administered. Morbidity and mortality in thalassemia is linked closely to the adequacy of chelation. Three chelators are currently available worldwide-deferoxamine, deferasirox, and deferiprone, although the latter is available in North America only in research protocols and compassionate use programs. These chelators can be used as monotherapy or in combination, although only the combination of deferiprone and deferoxamine has been extensively studied to date. Several factors, including chelator availability and its properties, drug tolerability, degree of organ-specific iron loading, ongoing transfusional iron burden, and patient preference, must be considered in the design of optimal, individualized chelation regimens, and these factors must periodically be reviewed and chelation adjusted accordingly. Ultimately, comparative effectiveness trials may help to determine the ideal strategy (eg, intensification of monotherapy or combined therapy including agents and doses) for treating various scenarios of organ-specific iron loading.
虽然红细胞输血治疗对地中海贫血症具有救命作用，但这种治疗会伴随强制性的铁负荷，因此必须进行螯合疗法以去除和解毒因这些慢性输血而产生的铁。地中海贫血症的发病率和死亡率与螯合治疗的充分性密切相关。目前全球有三种螯合剂——去铁胺、地拉罗司和去铁酮，尽管后者仅在北美可用于研究方案和同情用药计划。这些螯合剂可以单独使用或联合使用，尽管迄今为止只有去铁酮和去铁胺的联合使用得到了广泛研究。在设计最佳个体化螯合方案时，必须考虑包括螯合剂的可用性及其特性、药物耐受性、特定器官铁负荷程度、持续的输血铁负荷以及患者偏好等多种因素，并且必须定期审查这些因素并相应调整螯合治疗。最终，比较疗效试验可能有助于确定针对特定器官铁负荷的各种情况的理想治疗策略（例如，强化单一疗法或联合疗法，包括药物和剂量）。
