异麦芽糖酐铁治疗中国缺铁性贫血患者疗效的多中心回顾性研究  

A Multicenter Retrospective Study on the Efficacy of Ferric Derisomaltose for Treatment of Chinese Iron Deficiency Anemia Patients

作　　者：李大鹏 肖蓉[2] 杨正强 秘营昌 张凤奎 LI Dapeng;XIAO Rong;YANG Zhengqiang;MI Yingchang*;ZHANG Fengkui(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China;Sichuan Academy of Medical Science&Sichuan Provincial People′s Hospital,Sichuan Chengdu 610041,China;Shandong Cancer Hospital and Institute,Shandong First Medical University and Shandong Academy of Medical Sciences,Shandong Jinan 250117,China)

机构地区：[1]中国医学科学院血液病医院/中国医学科学院血液学研究所,实验血液学国家重点实验室国家血液系统疾病临床医学研究中心,天津300020 [2]四川省医学科学院,四川省人民医院,四川成都610041 [3]山东第一医科大学/山东省医学科学院,山东省肿瘤防治研究院/山东省肿瘤医院,山东济南250117

出　　处：《中国药物评价》2025年第1期54-60,共7页Chinese Journal of Drug Evaluation

摘　　要：目的:调查异麦芽糖酐铁在中国缺铁性贫血(IDA)患者中的疗效。方法:收集2021年6月至2022年9月在全国79家医院接受异麦芽糖酐铁治疗的IDA患者数据;使用SAS9.4软件分别采用t检验和Wilcoxon符号秩检验,比较符合正态分布和不符合正态分布血液指标治疗前后的差异,采用混合线性模型进行影响临床疗效[治疗后4周血红蛋白(Hb)相对基线差值]的多因素分析。结果:最终纳入2083例IDA患者数据,其中分别有63、2009、11例患者接受异麦芽糖酐铁500、1000、1500 mg单次静脉输注,平均剂量为(987.5±93.4)mg。接受异麦芽糖酐铁治疗后2周,患者的Hb(92.26 g·L^(-1)比74.28 g·L^(-1))、平均红细胞体积(77.72 fL比70.89 fL)、平均红细胞血红蛋白量(28.51 pg比24.14 pg)、平均红细胞血红蛋白浓度(298.64 g·L^(-1)比275.26 g·L^(-1))、血清铁(20.40μmol·L^(-1)比5.00μmol·L^(-1))、血清铁蛋白(78.00 ng·mL^(-1)比4.50 ng·mL^(-1))和转铁蛋白饱和度(21.40%比5.80%)均较基线显著改善(P<0.001);接受异麦芽糖酐铁治疗后4周,上述指标进一步得到改善;治疗后4周的Hb达标率和贫血改善(Hb上升≥20 g·L^(-1))率分别为33.8%(705/2083)和91.7%(1911/2083)。多因素分析显示,影响接受麦芽糖酐铁治疗患者临床疗效的因素包括年龄、体质量、患者来源、贫血病因、异麦芽糖酐铁给药剂量、输血和基线Hb水平(P<0.05)。结论:异麦芽糖酐铁在我国IDA患者中的疗效确切,单次静脉输注能提升血液指标,显著改善患者贫血。Objective:To investigate the efficacy of ferric derisomaltose in the treatment of Chinese patients with iron deficiency anemia(IDA).Methods:Data of IDA patients who treated with ferric derisomaltose were collected in 79 hospitals across the country from June 2021 to September 2022.SAS 9.4 software was used for statistical analysis.The T-test(normal distribution)and Wilcoxon signed-rank test(non-normal distribution)were used to compare the pre-and post-treatment differences in blood indices.The factors that influencing the clinical efficacy[defined as the change in hemoglobin(Hb)levels at 4 weeks post-treatment relative to baseline]were analyzed using mixed linear model.Results:A total of 2083 IDA patients were enrolled in this study.Among them,63,2009,and 11 patients received 500,1000 mg,and 1500 mg of ferric derisomaltose at a single intravenous infusions,respectively.The mean dose was(987.5±93.4)mg.The blood indexes such as hemoglobin(Hb:92.26 g·L^(-1) vs 74.28 g·L^(-1)),mean corpuscular volume(77.72 fL vs 70.89 fL),mean corpuscular hemoglobin(28.51 pg vs 24.14 pg),mean corpuscular hemoglobin concentration(298.64 g·L^(-1) vs 275.26 g·L^(-1)),Fe(20.40μmol·L^(-1) vs 5.00μmol·L^(-1)),serum ferritin(78.00 ng·mL^(-1) vs 4.50 ng·mL^(-1))and transferrin saturation(21.40%vs 5.80%)were significantly improved at 2 weeks post-treatment(P<0.001).Further improvements in these blood indexes were observed at 4 weeks post-treatment.Among them,33.8%(705/2083)IDA patients achieved normal hemoglobin levels and 91.7%(1911/2083)improved the anemia(Hb increase≥20 g·L^(-1))at 4 weeks post-treatment.Multivariate analysis showed that age,body weight,patient source,etiology of anemia,ferric derisomaltose dosage,blood transfusion,and baseline Hb levels were factors influencing clinical efficacy of patients receiving ferric derisomaltose therapy(P<0.05).Conclusion:Ferric derisomaltose is effective in Chinese patients with IDA,with significant improvements in anemia observed after a single infusion.

关 键 词：异麦芽糖酐铁 缺铁性贫血 静脉铁剂 回顾性分析 有效性 

分 类 号：R973.3[医药卫生—药品]