Efficacy and safety of Roxadustat in the treatment of refractory NSAA and low-risk MDS-related anemia
10.3969/j.issn.1006-5725.2024.12.018
- VernacularTitle: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
- Author:
Qinglin HU
1
;
Ziqi WAN
;
Chen YANG
;
Miao CHEN
;
Bing HAN
Author Information
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)
- Keywords:
Roxadustat;
refractory;
non-severe aplastic anemia;
low risk myelodysplastic syndromes
- From:
The Journal of Practical Medicine
2024;40(12):1719-1724
- CountryChina
- Language:Chinese
-
Abstract:
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 haemato-logical 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.
