Department of Pediatrics and Radiology, Divisions of Cardiology and Radiology, Children's Hospital of Los Angeles, CA.
Am J Hematol. 2010 Oct;85(10):818-9. doi: 10.1002/ajh.21830.
The trial CICL670AUS04 was a single-arm, open-label study of the cardiac efficacy of 18 months of deferasirox monotherapy [1]. Cardiac response in this study was related to the degree of liver siderosis. Patients with mild to moderate liver siderosis improved their cardiac T2* while more severely siderotic patients did not, regardless of initial cardiac iron burden. In this letter, we report 2-year data in those patients who completed a 6-month extension phase (N 5 10). Cardiac and liver iron improved steadily during the 24-month period, with final cardiac T2* and LIC improving 37% and 27%, respectively, in this cohort. Serum ferritin and LVEF were not statistically different at anytime-point. When the extension phase (18-24 months) was considered in isolation, serum ferritin, liver iron concentration, and left ventricular ejection fraction were nearly identical to 18 month results. Despite this, cardiac T2* continued to trend higher, increasing 12.7% from 9.5 ms to 10.7 ms (P 5 0.06). Thus defersirox continued to demonstrate cardiac efficacy in patients with mild to moderate hepatic siderosis throughout 2 years of therapy.
试验 CICL670AUS04 是一项为期 18 个月的地拉罗司单药治疗心脏疗效的单臂、开放标签研究[1]。该研究中心脏反应与肝脏铁沉积程度有关。轻度至中度肝脏铁沉积的患者改善了他们的心脏 T2*，而更严重的铁沉积患者则没有，无论初始心脏铁负荷如何。在这封信中，我们报告了完成 6 个月扩展阶段的患者的 2 年数据（N 5 10）。在 24 个月期间，心脏和肝脏铁持续稳定改善，最终心脏 T2和 LIC 分别改善了 37%和 27%。在任何时间点，血清铁蛋白和 LVEF 均无统计学差异。当单独考虑扩展阶段（18-24 个月）时，血清铁蛋白、肝脏铁浓度和左心室射血分数与 18 个月的结果几乎相同。尽管如此，心脏 T2继续呈上升趋势，从 9.5ms 增加到 10.7ms（P 5 0.06）。因此，地拉罗司在 2 年的治疗中继续显示出对轻度至中度肝脏铁沉积患者的心脏疗效。
