Abstract：
We conducted a retrospective cohort study on 663 transfusion-dependent β-thalassemia patients receiving the same iron chelation monotherapy with deferoxamine, deferiprone, or deferasirox for up to 10 years (median age 31.8 years, 49.9 % females). Patients on all three iron chelators had a steady and significant decline in serum ferritin over the 10 years (median deferoxamine: -170.7 ng/mL, P = 0.049, deferiprone: -236.7 ng/mL, P = 0.001; deferasirox: -323.7 ng/mL, P < 0.001) yet had no significant change in liver iron concentration or cardiac T2*; while noting that patients generally had low hepatic and cardiac iron levels at study start. Median absolute, relative, and normalized changes were generally comparable between the three iron chelators. Patients receiving deferasirox had the highest morbidity and mortality-free survival probability among the three chelators, although the difference was only statistically significant when compared with deferoxamine (P = 0.037). On multivariate Cox regression analysis, there was no significant association between iron chelator type and the composite outcome of morbidity or mortality. In a real-world setting, there is comparable long-term iron chelation effectiveness between the three available iron chelators for patients with mild-to-moderate iron overload.
摘要：
我们对663例输血依赖性β-地中海贫血患者进行了一项回顾性队列研究，这些患者接受了相同的铁螯合单药治疗和去铁胺。去铁酮，或地拉罗司10年(中位年龄31.8岁，49.9%女性）。使用所有三种铁螯合剂的患者在10年内血清铁蛋白稳定且显着下降（中位数去铁胺：-170.7ng/mL，P=0.049，去铁酮：-236.7ng/mL，P=0.001；地拉罗司：-323.7ng/mL，P<0.001）但肝脏铁浓度或心脏T2*没有显着变化；同时注意到患者在研究开始时通常具有较低的肝脏和心脏铁水平。绝对中位数,相对，和归一化变化在三种铁螯合剂之间通常是相当的。在三种螯合剂中，接受地拉罗司的患者具有最高的发病率和无死亡率生存概率。尽管与去铁胺相比差异仅具有统计学意义（P=0.037）。在多元Cox回归分析中，铁螯合剂类型与发病率或死亡率的复合结局之间没有显著关联.在现实世界中,三种铁螯合剂对轻度至中度铁超负荷患者的长期铁螯合效果相当.
