罗沙司他治疗难治性NSAA和低风险MDS相关贫血的疗效和安全性  

Efficacy and safety of Roxadustat in the treatment of refractory NSAA and low-risk MDS-related anemia

作　　者：胡青林 万梓琪 杨辰[1] 陈苗[1] 韩冰[1] HU Qinglin;WAN Ziqi;YANG Chen;CHEN Miao;HAN Bing(Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Science and Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医学院、北京协和医院血液内科,北京100730

出　　处：《实用医学杂志》2024年第12期1719-1724,共6页The Journal of Practical Medicine

基　　金：中央高水平医院临床科研基金资助项目(编号:2022-PUMCH-C-026)。

摘　　要：目的探讨罗沙司他治疗难治性非重型再生障碍性贫血(NSAA)和低风险MDS(LR-MDS)的整组及亚组疗效、亚组疗效差异、疗效预测因素及安全性。方法收集2020年8月至2022年12月在北京协和医院血液内科就诊的难治性NSAA和LR-MDS患者,所有患者在罗沙司他治疗前,都接受过一线的标准治疗,并包括至少3个月以上的重组人促红细胞生成素(rhEPO)。患者均使用过罗沙司他2.5 mg/kg隔天1次,至少3个月,并随访至少8个月。分析患者临床特征、罗沙司他的疗效、疗效预测因素、不良反应、复发及疾病克隆演变情况。结果共纳入40例患者,包括24例难治性NSAA和16例LR-MDS。年龄18~81岁,中位年龄56岁,男性占40%。65%的患者有输血依赖。随访9~34个月,中位随访21个月,在第1、2、3、4、5、6个月及随访期末,分别有22.5%、25.0%、47.5%、55.0%、57.5%、60.0%和50.0%的患者达到血液学改善-红系反应(HI-E),未发现影响HI-E的因素。两组患者组间血红蛋白较基线变化在随访期末差异有统计学意义(P<0.05)。50%患者脱离输血依赖。22.5%患者报告了不良反应。28.5%患者在达到HI-E后复发,复发时间4~12个月,复发中位时间为7个月。在随访期末未观察到克隆演变。结论罗沙司他对传统疗法和rhEPO难治的NSAA或LR-MDS患者可能有效,不良反应轻微,复发率较低。难治性NSAA患者血红蛋白改善的程度可能更好。Objective To investigate the overall and subgroup efficacy,subgroup differences,predictors of efficacy and safety of roxadustat in the treatment of refractory non-severe aplastic anemia(NSAA)and low-risk myelodysplastic syndromes(LR-MDS).Methods Patients with refractory NSAA and LR-MDS who were admitted to the Department of Hematology,Peking Union Medical College Hospital from August 2020 to December 2022 were enrolled.All patients received first-line standard treatment,including recombinant human erythropoietin(rhEPO)for at least 3 months before roxadustat treatment.All patients received roxadustat 2.5 mg/kg every other day for at least 3 months,and were followed up for at least 8 months.The clinical characteristics of patients,roxadustat efficacy,predictors of efficacy,adverse effects,relapse and disease clonal evolution were analyzed.Results A total of 40 patients including 24 refractory NSAA and 16 LR-MDS were included.median age was 56(18~81)years and 40% were males.65% of the patients were transfusion dependent.Median follow-up was 21(9~34)months.22.5%,25.0%,47.5%,55.0%,57.5%,60.0% and 50.0% of the patients achieved haematological improvement-erythroid(HI-E)at months 1,2,3,4,5,6,and end of the follow-up period,respectively,and no factors affecting HI-E were identified.The hemoglobin change from baseline was statistically different between the two groups at the end of the follow-up period.50% of patients were relieved from transfusion dependence.Adverse reactions were reported in 22.5% of patients.28.5% of patients relapsed after achieving HI-E,with a median time to relapse of 7(4~12)months.No clonal evolution was observed at the end of the follow-up period.Conclusions Our preliminary findings suggested that Roxadustat may be effective for patients with NSAA or LR-MDS refractory to conventional therapies and rhEPO,with mild adverse effects and low relapse rate.The degree of hemoglobin improvement may be better in the refractory NSAA patients.

关 键 词：罗沙司他 难治性 非重型再生障碍性贫血 低风险骨髓增生异常综合征 

分 类 号：R552[医药卫生—血液循环系统疾病]