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

摘要/Abstract

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2 083例IDA患者数据，其中分别有63、2 009、11例患者接受异麦芽糖酐铁500、1 000、1 500 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（20.40 μmol·L-1比5.00 μmol·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%（705/2 083）和91.7%（1 911/2 083）。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。

关键词: font-size:medium, ">异麦芽糖酐铁；缺铁性贫血；静脉铁剂；回顾性分析；有效性

Abstract: 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 2 083 IDA patients were enrolled in this study. Among them， 63， 2 009， and 11 patients received 500， 1 000 mg， and 1 500 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 (20.40 μmol·L-1 vs 5.00 μmol·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% (705/2 083) IDA patients achieved normal hemoglobin levels and 91.7% (1 911/2 083) 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.

Key words: font-size:medium, ">Ferric derisomaltose； Iron deficiency anemia； Intravenous iron； Retrospective analysis； Efficacy

中图分类号:

李大鹏1, 肖蓉2, 杨正强3, 秘营昌1, 张凤奎1. 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[J]. 中国药物评价, 2025, 42(1): 54-60.

LI Dapeng1, XIAO Rong2, YANG Zhengqiang3, MI Yingchang1, ZHANG Fengkui1. A Multicenter Retrospective Study on the Efficacy of Ferric Derisomaltose for Treatment of Chinese Iron Deficiency Anemia Patients[J]. CHINESE JOURNAL OF DRUG EVALUATION, 2025, 42(1): 54-60.

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