罗沙司他治疗难治性非重型再生障碍性贫血的疗效与安全性

Efficacy and safety of roxadustat in the treatment of refractory non-severe aplastic anemia

目的评估罗沙司他在难治性非重型再生障碍性贫血(NSAA)患者中的疗效与安全性。方法回顾性收集2020年10月至2022年8月在北京协和医院连续使用罗沙司他至少3个月,并在使用后随访超过6个月的难治性NSAA患者的临床资料,收集其人口学信息、临床资料、疗效、不良反应及转归,并分析可能影响疗效的因素。结果共纳入41例患者。男女比例为16∶25,中位年龄52(18~84)岁,罗沙司他中位治疗时间5(3~20)个月,中位随访时间15(6~26)个月。1、2、3、6、12个月的血液学改善-红系(HI-E)率分别为12.2%、29.3%、46.3%、43.9%和30.3%。治疗后3、6、12个月摆脱输血依赖比例分别为28.5%、38.1%、33.3%。治疗后部分患者HGB恢复正常。不良事件发生率为22%,均为Ⅰ~Ⅱ级,可恢复。未发现影响HI-E的因素。至随访期末,有45%(9/20)的患者复发,中位复发时间为7(3~12)个月。未观察到克隆演变。1例患者死于肺部感染。结论罗沙司他可有效改善难治性NSAA的贫血,安全性好。

Objective To evaluate the efficacy and safety of roxadustat in patients with refractory non-severe aplastic anemia(NSAA).Methods The clinical data of patients with refractory NSAA who had been treated with roxadustat continuously for at least 3 months and followed up for more than 6 months at Peking Union Medical College Hospital from October 2020 to August 2022 were retrospectively collected.The demographic information,clinical data,treatment efficacy,adverse reactions,and outcomes were evaluated,and the factors influencing efficacy were analyzed.Results A total of 41 patients were included.The male-to-female ratio was 16∶25,and the median age was 52(18–84)years.The median duration of roxadustat treatment was 5(3–20)months,and the median follow-up was 15(6–26)months.Hematologic improvement-erythroid(HI-E)was 12.2%,29.3%,46.3%,43.9%,and 30.3%at 1,2,3,6,and 12 months,respectively.The rate of transfusion independence was 28.5%,38.1%,and 33.3%at 3,6,and 12 months,respectively.Hemoglobin returned to normal in some patients after treatment with roxadustat.The incidence of adverse events was 22%,all of which were gradeⅠ–Ⅱand recoverable.No factors that could affect HI-E were identified.By the end of follow-up,45%of the patients relapsed,with a median time to relapse of 7(3–12)months.No clonal evolution was observed,and one patient died.Conclusion Roxadustat effectively improved anemia with good tolerance in patients with refractory NSAA.