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地拉罗司:口服,每日一次铁螯合剂——专家意见。

Deferasirox: oral, once daily iron chelator--an expert opinion.

机构信息

Bombay Hospital Institute of Medical Sciences, Marine Lines, Mumbai, India.

出版信息

Indian J Pediatr. 2010 Feb;77(2):185-91. doi: 10.1007/s12098-010-0030-4. Epub 2010 Feb 23.

Abstract

Iron overload is a serious and potentially fatal condition that results from multiple blood transfusions required over a long period of time to treat certain types of anemias such as, that caused by beta-thalassemia, sickle cell disease and myelodysplastic syndrome. Deferoxamine, which has been used since four decades as an iron chelator has limited efficacy due to its demanding therapeutic regimen, leading to poor compliance. Deferasirox, once daily oral iron chelator provides an effective alternative to Deferoxamine in the treatment of transfusional hemosiderosis. In this review, the role of Deferasirox as an ideal iron chelator has been discussed. Pubmed searches on Deferasirox were carried out for the same. Several studies demonstrated the safety and efficacy of Deferasirox in reducing iron burden in iron-overloaded patients with beta-thalassemia, sickle cell anemia and myelodysplastic anemia. Thus, convenient, effective and tolerable chelation therapy with oral Deferasirox is likely to be a significant development in the treatment of transfusional iron overload, due to its ability to provide constant chelation coverage and the potential to improve compliance.

摘要

铁过载是一种严重且潜在致命的病症,它是由于治疗某些类型的贫血症(如β-地中海贫血、镰状细胞病和骨髓增生异常综合征)需要多次长期输血引起的。自四十年前以来,去铁胺一直被用作铁螯合剂,但由于其治疗方案要求苛刻,导致患者顺应性差,疗效有限。地拉罗司,一种每日一次的口服铁螯合剂,为治疗输血性血色素沉着症提供了一种替代去铁胺的有效方法。在这篇综述中,讨论了地拉罗司作为一种理想的铁螯合剂的作用。在 PubMed 上对地拉罗司进行了检索。多项研究表明,地拉罗司在降低β-地中海贫血、镰状细胞贫血和骨髓增生异常综合征铁过载患者的铁负荷方面具有安全性和有效性。因此,口服地拉罗司具有方便、有效和耐受性好的螯合治疗作用,可能是治疗输血性铁过载的一个重要进展,因为它能够提供持续的螯合作用,并有可能提高顺应性。

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