Division of Hematology, Department of Internal Medicine, Seoul St Mary's Hospital, The Catholic University of Korea, Seoul, South Korea.
Blood. 2010 Oct 7;116(14):2448-54. doi: 10.1182/blood-2010-01-261289. Epub 2010 Jun 21.
The prospective 1-year Evaluation of Patients' Iron Chelation with Exjade (EPIC) study enrolled a large cohort of 116 patients with aplastic anemia; the present analyses evaluated the efficacy and safety of deferasirox in this patient population. After 1 year, median serum ferritin decreased significantly from 3254 ng/mL at baseline to 1854 ng/mL (P < .001). Decreases occurred in chelation-naive (3229-1520 ng/mL; P < .001, last-observation-carried-forward analysis), and previously chelated (3263-2585 ng/mL; P = .21, last-observation-carried-forward analysis) patients and were reflective of dose adjustments and ongoing iron intake. Baseline labile plasma iron levels were within normal range despite high serum ferritin levels. The most common drug-related adverse events were nausea (22%) and diarrhea (16%). Serum creatinine increases more than 33% above baseline and the upper limit of normal occurred in 29 patients (25%), but there were no progressive increases; concomitant use of cyclosporine had a significant impact on serum creatinine levels. The decrease in mean alanine aminotransferase levels at 1 year correlated significantly with reduction in serum ferritin (r = 0.40, P < .001). Mean absolute neutrophil and platelet counts remained stable during treatment, and there were no drug-related cytopenias. This prospective dataset confirms the efficacy and well characterizes the tolerability profile of deferasirox in a large population of patients with aplastic anemia. This study was registered at www.clinicaltrials.gov as #NCT00171821.
前瞻性的 EPIC 研究（患者去铁酮螯合治疗的 1 年评估）纳入了 116 例再生障碍性贫血患者，本分析评估了去铁酮在这一患者人群中的疗效和安全性。经过 1 年，中位血清铁蛋白从基线时的 3254ng/ml 显著下降至 1854ng/ml（P＜0.001）。新开始螯合治疗（3229-1520ng/ml；P＜0.001，末次观测值结转分析）和既往螯合治疗（3263-2585ng/ml；P=0.21，末次观测值结转分析）患者的铁蛋白水平均出现下降，这与剂量调整和持续铁摄入有关。尽管血清铁蛋白水平较高，但基础不稳定血浆铁水平仍处于正常范围内。最常见的药物相关不良事件是恶心（22%）和腹泻（16%）。血清肌酐较基线水平升高超过 33%且超过正常值上限的患者有 29 例（25%），但没有进行性升高；环孢素的同时使用对血清肌酐水平有显著影响。治疗 1 年后平均丙氨酸氨基转移酶水平的下降与血清铁蛋白的降低显著相关（r=0.40，P＜0.001）。治疗期间平均绝对中性粒细胞和血小板计数保持稳定，无药物相关的血细胞减少症。这项前瞻性数据集证实了去铁酮的疗效，并很好地描述了其在大量再生障碍性贫血患者中的耐受性特征。这项研究在 www.clinicaltrials.gov 上注册，编号为#NCT00171821。
