Efficacy and safety of Roxadustat in the treatment of refractory NSAA and low-risk MDS-related anemia
HU Qinglin
WAN Ziqi
YANG Chen
CHEN Miao
HAN Bing
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.
Keywords：Roxadustatrefractorynon-severe aplastic anemialow risk myelodysplastic syndromes
Publication Date：2024-06-25
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：6( 1719-1724 )