Haematology-Oncology Division, Department of Paediatrics and Thalassaemia Center, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand.
Hospital Kuala Lumpur, Kuala Lumpur, Malaysia.
Int J Hematol. 2011 Mar;93(3):319-328. doi: 10.1007/s12185-011-0789-8. Epub 2011 Mar 4.
Although thalassaemia is highly prevalent in the Asia-Pacific region, clinical data on efficacy and safety profiles of deferasirox in patients from this region are rather limited. Recently, data from the multicentre Evaluation of Patients' Iron Chelation with Exjade (EPIC) study in 1744 patients with different anaemias has provided an opportunity to analyse 1115 thalassaemia patients, of whom 444 patients were from five countries in the Asia-Pacific region (AP) for whom thalassaemia management and choice of iron chelators were similar. Compared to the rest of the world (ROW), baseline clinical data showed that the AP group appeared to be more loaded with iron (3745.0 vs. 2822.0 ng/ml) and had a higher proportion on deferoxamine monotherapy prior to the study (82.9 vs. 58.9%). Using a starting deferasirox dose based on transfusional iron intake and tailoring it to individual patient response, clinical efficacy based on serum ferritin reduction in AP and ROW thalassaemia patients was similar. Interestingly, the AP group developed a higher incidence of drug-related skin rash compared to ROW (18.0 vs. 7.2%), which may indicate different pharmacogenetic backgrounds in the two populations. Our analysis confirms that, with appropriate adjustment of dose, deferasirox can be clinically effective across different regions, with manageable side effects.
虽然地中海贫血在亚太地区非常普遍,但有关该地区患者使用地拉罗司的疗效和安全性数据相当有限。最近,来自多中心评估患者铁螯合治疗 Exjade(EPIC)研究的 1744 名不同贫血患者的数据提供了一个机会,可以分析来自亚太地区(AP)的 1115 名地中海贫血患者的数据,其中 444 名患者来自亚太地区的五个国家,这些国家的地中海贫血管理和铁螯合剂选择相似。与世界其他地区(ROW)相比,基线临床数据显示,AP 组的铁负荷似乎更高(3745.0 vs. 2822.0ng/ml),且在研究前接受单独使用去铁胺治疗的比例更高(82.9 vs. 58.9%)。AP 和 ROW 地中海贫血患者的临床疗效是基于血清铁蛋白的降低,使用基于输血铁摄入量的起始地拉罗司剂量,并根据个体患者的反应进行调整,两组的疗效相似。有趣的是,与 ROW 相比,AP 组出现药物相关皮疹的发生率更高(18.0% vs. 7.2%),这可能表明两个人群的药物遗传学背景不同。我们的分析证实,通过适当调整剂量,地拉罗司在不同地区都能具有临床疗效,且副作用可管理。