新型铁螯合剂地拉罗司治疗骨髓增生异常综合征铁过载的临床研究  

Efficacy of deferasirox in iron overload patients secondary to myelodysplastic syndrome

作　　者：金香淑[1] 徐绎涵[1] 靖彧[1] 韩晓蘋[1] 李红华[1] 姚子龙[1] 于力[1] 朱海燕[1] 

机构地区：[1]解放军总医院血液科

出　　处：《中华老年多器官疾病杂志》2016年第11期815-818,共4页Chinese Journal of Multiple Organ Diseases in the Elderly

基　　金：中国博士后科学基金面上资助一等资助(2014M552583)~~

摘　　要：目的：分析新型铁螯合剂地拉罗司治疗骨髓增生异常综合征（ MDS ）继发性铁过载的临床疗效及不良反应。方法回顾性分析了解放军总医院2012年1月至2014年4月期间应用新型铁螯合剂地拉罗司治疗MDS继发性铁过载患者的临床资料，观察治疗前后血清铁蛋白（ SF）、红细胞输注量、血红蛋白及药物不良反应。结果共有8例MDS继发性铁过载患者服用地拉罗司，其中男性7例，女性1例，中位年龄52（38～71）岁。治疗3个月后，疗效评价为完全反应（CR）3例，微小反应（MiR）3例，稳定铁过载（SIL）2例，总反应率为75．0％（6／8），中位红细胞输注量为2（1～3）u／月。治疗1年后，疗效评价为CR 5例，MiR 2例，SIL1例，总反应率为87．5％（7／8）。与治疗前相比，患者治疗1年后SF显著降低[（871．0±584．2） vs （2164．9±1233．6）ng／ml]、血红蛋白显著升高[（101．5±34．59） vs （65．37±21．35）g／L]，差异均具有统计学意义（P＜0．05）。1年后5例患者脱离输血，其余3例中位红细胞输注量分别为0．5 u／月、1．5 u／月及2．0 u／月。治疗1年后，仅1例患者死亡。服药后出现恶心、呕吐者3例，腹泻1例。结论地拉罗司治疗MDS继发性铁过载安全有效。Objective To analyze the efficacy of a new iron chelator , deferasirox, in treatment of iron overload secondary to myelo-dysplastic syndrome （ MDS） and investigate its adverse reactions .Methods Clinical data of all the iron overload patients secondary to MDS who receiving deferasirox therapy in our hospital from January 2012 to April 2014 were collected and retrospectively analyzed . Their serum ferritin （ SF） level, amount of red blood cell transfusion , and hemoglobin level were measured and recorded before and after treatment, and adverse reactions were observed .Results There were totally 8 cases of MDS secondary iron overload enrolled in this study.They were 7 males and 1 female, and at a median age of 52 （ranging from 38 to 71） years.After 3 months’ treatment, complete response （CR） was obtained in 3 cases, minor response （MiR） in 3 cases, and stable iron load （SIL） in 2 cases.The overall response rate was 75.0%（6/8）, and the median amount of red blood cell transfusion was 2 （1-3） u/month.In 1 year after treatment, SF level was significantly decreased [（871.0 ±584.2） vs （2164.9 ±1233.6） ng/ml], while that of hemoglobin was obviously increased[（101.5 ±34.59） vs （65.37 ±21.35） g/L], with statistical differences （P 〈0.05）.At this time point, 5 patients were out of red blood cell transfusion , and the amount of red blood cell transfusion was 0.5, 1.5 and 2.0 u/month respectively, for the other 3 patients.After 1 year treatment, only 1 patient died, 3 patients experienced nausea and vomiting, and 1 patient diarrhea .Conclusion Deferasirox therapy is safe and effective for MDS secondary iron overload patients .

关 键 词：骨髓增生异常综合征 铁过载 地拉罗司 

分 类 号：R551.3[医药卫生—血液循环系统疾病]