Heinrich-Heine-University, Düsseldorf, Germany.
Leuk Res. 2010 Sep;34(9):1143-50. doi: 10.1016/j.leukres.2010.03.009. Epub 2010 May 6.
The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500ng/mL, approximately 50% were chelation-naïve. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naïve or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reduction in serum ferritin (p<0.0001). This large dataset prospectively confirms the efficacy and well characterizes the safety profile of deferasirox in MDS.
前瞻性的 1 年 EPIC 研究纳入了 341 例骨髓增生异常综合征（MDS）患者；尽管基线铁负荷 >2500ng/ml，但约 50%的患者是螯合治疗初治者。总体而言，中位血清铁蛋白在 1 年内显著下降（p=0.002）。无论患者是否为螯合治疗初治者或既往接受过螯合治疗，下降均发生；变化取决于剂量调整和持续的铁摄入。观察到稳定的血浆游离铁减少。停药率（48.7%）和不良事件谱与 MDS 中以前报告的地拉罗司数据一致。丙氨酸氨基转移酶水平显著降低；变化与血清铁蛋白的降低显著相关（p<0.0001）。这项大型数据集前瞻性地证实了地拉罗司在 MDS 中的疗效，并很好地描述了其安全性特征。
