Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, 3501 Civic Center Boulevard, Clinical Hub Building, Room 13547, Philadelphia, PA 19104, USA.
Hematol Oncol Clin North Am. 2023 Apr;37(2):379-391. doi: 10.1016/j.hoc.2022.12.013.
Conventional therapy for severe thalassemia includes regular red cell transfusions and iron chelation therapy to prevent and treat complications of iron overload. Iron chelation is very effective when appropriately used, but inadequate iron chelation therapy continues to contribute to preventable morbidity and mortality in transfusion-dependent thalassemia. Factors that contribute to suboptimal iron chelation include poor adherence, variable pharmacokinetics, chelator adverse effects, and difficulties with precise monitoring of response. The regular assessment of adherence, adverse effects, and iron burden with appropriate treatment adjustments is necessary to optimize patient outcomes.
常规治疗严重地中海贫血症包括定期输注红细胞和铁螯合治疗,以预防和治疗铁过载并发症。铁螯合治疗在适当使用时非常有效,但在输血依赖型地中海贫血症中,铁螯合治疗不足仍然导致可预防的发病率和死亡率。导致铁螯合治疗不理想的因素包括依从性差、药代动力学变化、螯合剂不良反应以及精确监测反应的困难。定期评估依从性、不良反应和铁负荷,并进行适当的治疗调整,是优化患者结局所必需的。