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.