地拉罗司对伴有铁过载的再生障碍性贫血患者的祛铁疗效及安全性——一项单臂、多中心、前瞻性临床研究  

Efficacy and safety of deferasirox in aplastie anemia patients with iron overload： a single arm, multicenter, prospective study in China

作　　者：施均 常红[2] 张莉 邵英起 聂能 张静[1] 黄金波 张丽[2] 唐旭东[3] 全日成 郑春梅[3] 肖海燕[3] 胡明辉[4] 胡令彦[4] 刘锋[3] 周永明[4] 郑以州 张凤奎 

机构地区：[1]中国医学科学院、北京协和医学院血液学研究所、血液病医院,天津300020 [2]四川大学华西医院 [3]中国中医科学院西苑医院 [4]上海中医药大学附属岳阳中西医结合医院

出　　处：《中华血液学杂志》2016年第1期1-6,共6页Chinese Journal of Hematology

摘　　要：目的研究地拉罗司对伴有铁过载的再生障碍性贫血（AA）患者的祛铁疗效及安全性。方法分析64例伴铁过载的AA患者经地拉罗司治疗12个月后的祛铁疗效，并进行安全性评估。结果所有患者地拉罗司的起始剂量为20．0mg·kg-1·d-1，平均剂量为（18．6±3．60）mg·kg～·d-1。经12个月治疗后，中位血清铁蛋白（SF）水平由基线的4924（2718-6765）μg／L（64例）降到3036（1474～5551）gg／L（23例），降幅达38％，SF降低量的中位数为651（126～2125）μg／L；23例完成12个月治疗的患者sF中位水平由基线的5271（3420～8278）μg／L降到3036（1474～5551）μg／L，降幅达到42％，SF降低量的中位数为1167（580-4806）μg／L。血肌酐增高（40．98％）、胃肠道不适（40．98％）是地拉罗司治疗期间最主要的不良事件，其次为肝脏转氨酶增高（ALT：21．31％；AST：13．11％）、蛋白尿（24．59％）。血肌酐增高呈可逆性、非进行性。对于合并使用环孢素的38例患者，12例（31．8％）连续2次肌酐值〉正常值上限（ULN），10例（26．3％）连续2次肌酐值〉1．33基线值，仅1例（2．6％）血清肌酐升高超过1．33基线值并超过ULN。对于AST和ALT，整个研究中都没有患者发生两次基线后值〉5xULN或〉10xULN。对于基线PLT水平低于50x10^9／L的患者，地拉罗司治疗期间中位PLT未降低。结论地拉罗司治疗伴有铁过载的AA患者可获得较好祛铁疗效，药物耐受性良好，无临床不可控的严重不良事件。Objective To explore the efficacy and safety of deferasirox in aplastic anemia （AA） patients with iron overload. Methods A single arm, multi-center, prospective, open-label study was conducted to evaluate absolute change in serum ferritin （SF）from baseline to 12 months of deferasirox administration, initially at a dose of 20 mg· kg-1·d-1, and the safety in 64 AA patients with iron overload. Results All patients started their deferasirox treatment with a daily dose of 20 nag· kg-1·d-1. Themean actual dose was （18.6±3.60）mg·kg-1·d-1. The median SF decreased from 4 924 （2 718-6 765）μg/L at baseline （n=64） to 3 036（1 47±5 551）μg/L at 12 months （n=23） with the percentage change from baseline as 38%. A median SF decrease of 651 （ 126-2 125） p.g/L was observed at the end of study in 23 patients who completed 12 months＇ treatment, the median SF level decreased by 1 167 （580-4 806）μg/L[5 271（3 420-8 278）μg/L at baseline; 3 036（ 1 474-5 551 ）μg/L after 12 months＇ treatment; the percentage change from baseline as 42% ] after 12 months of deferasirox treatment. The most common adverse events （AEs） were increased serum creatinine levels （40.98%）, gastrointestinal discomfort （40.98%）, elevated liver transaminase （ALT： 21.31%; AST： 13.11% ） and proteinuria （24.59%）. The increased serum creatinine levels were reversible and non-progressive. Of 38 patients with concomitant cyclosporine use, 12 （31,8%） patients had two consecutive values 〉ULN, 10 （26.3%） patients had two consecutive values 〉1.33 baseline values, but only 1 （2.6%） patient＇s serum creatinine increased more than 1.33 baseline values and exceeded ULN. For both AST and ALT, no patients experienced two post-baseline values 〉5 x ULN or 〉10 x ULN during the whole study. In AA patients with low baseline PLT count （less than 50x 10^9/L）, there was no decrease for median PLT level during 12 months＇ treatment period. Conclusions AA patients with iron overload could

关 键 词：贫血 再生障碍性 铁超负荷 地拉罗司 祛铁螯合疗法 

分 类 号：R556.5[医药卫生—血液循环系统疾病]