Imran Farhan, Phatak Pradyumna
Department of Medicine, Rochester General Hospital, 1425 Portland Avenue, Rochester, NY 14621, USA.
Expert Rev Pharmacoecon Outcomes Res. 2009 Aug;9(4):297-304. doi: 10.1586/erp.09.26.
Deferasirox is a once-daily, orally administered, tridentate iron chelator that is indicated in the treatment of iron overload resulting from regular packed red blood cell transfusions in patients with transfusion-dependent anemias, such as beta-thalassemia, sickle cell disease, myelodysplastic syndrome and other rare anemias. Randomized, controlled trials have established its efficacy to reduce liver iron concentration and serum ferritin levels to be comparable to the historic standard iron chelator, deferoxamine, which is administered as a parenteral infusion. However, deferasirox may be more effective than deferoxamine in actual clinical practice owing to the improvement in quality of life and, hence, increased compliance associated with the oral route of administration. The higher acquisition cost of deferasirox may be counterbalanced by savings in the administration cost, as well as the treatment of complications of iron overload that result from noncompliance with therapy attributable to the parenteral mode of administration. Deferasirox may also have potential as an important supplement and even an alternative to phlebotomies in nontransfusional, genetic iron overload disorders, such as hereditary hemochromatosis.
地拉罗司是一种每日服用一次的口服三齿铁螯合剂,适用于治疗因依赖输血的贫血(如β地中海贫血、镰状细胞病、骨髓增生异常综合征和其他罕见贫血)患者定期输注浓缩红细胞导致的铁过载。随机对照试验已证实其降低肝脏铁浓度和血清铁蛋白水平的疗效与历史标准铁螯合剂去铁胺相当,后者通过胃肠外输注给药。然而,由于生活质量的改善以及口服给药途径带来的依从性提高,地拉罗司在实际临床实践中可能比去铁胺更有效。地拉罗司较高的购置成本可能会被给药成本的节省以及因胃肠外给药方式导致的治疗不依从引起的铁过载并发症治疗成本所抵消。在非输血性遗传性铁过载疾病(如遗传性血色素沉着症)中,地拉罗司也可能作为放血疗法的重要补充甚至替代疗法具有潜力。