Kattamis Antonis
First Department of Pediatrics, University of Athens, Medical School, Aghia Sophia Children's Hospital, Thivon and Levadias, Goudi 115 27, Greece.
Ann N Y Acad Sci. 2005;1054:175-82. doi: 10.1196/annals.1345.020.
Therapy with either deferiprone (DFP) or deferoxamine (DFO) is inadequate in achieving negative iron balance in many patients with thalassemia. There are mounting theoretical, experimental, and clinical evidences of increased efficacy when therapy includes both chelating agents. DFP and DFO chelate excess iron in different ways without affecting each other's metabolism. When both chelators are administered simultaneously, they interact either in an additive or synergistic manner, probably through "shuttling" iron from DFP to DFO. Iron-balance studies have shown that the use of both agents on the same day can induce negative iron balance in all patients. Long-term combined therapy with DFO with DFP results in considerable reduction of both ferritin levels and liver iron concentration as well as significant improvement in cardiac siderosis and function. This therapeutic regimen is well tolerated and safe, even though it may be related to a small increase in the incidence of agranulocytosis compared with DFP monotherapy. Apart from using both agents simultaneously, sequential administration of DFP and DFO has also shown promising results. Combining the available iron chelators offers many therapeutic options that can be tailored to each patient individually. It is an exciting advance in treating hemosiderosis in thalassemic patients.
对于许多地中海贫血患者，去铁酮（DFP）或去铁胺（DFO）治疗在实现负铁平衡方面并不充分。越来越多的理论、实验和临床证据表明，联合使用这两种螯合剂进行治疗时疗效会提高。DFP和DFO以不同方式螯合过量铁，且不影响彼此的代谢。当同时给予两种螯合剂时，它们可能通过将铁从DFP“转运”到DFO以相加或协同的方式相互作用。铁平衡研究表明，同一天使用这两种药物可使所有患者实现负铁平衡。DFO与DFP长期联合治疗可使铁蛋白水平和肝脏铁浓度显著降低，同时心脏含铁血黄素沉着症及功能也有显著改善。这种治疗方案耐受性良好且安全，尽管与DFP单药治疗相比，粒细胞缺乏症的发生率可能会略有增加。除了同时使用这两种药物外，DFP和DFO序贯给药也显示出了有前景的结果。联合使用现有的铁螯合剂提供了许多可针对每个患者量身定制的治疗选择。这是治疗地中海贫血患者铁过载的一项令人兴奋的进展。
