Efficacy and safety of roxadustat in the treatment of refractory non-severe aplastic anemia
10.3760/cma.j.cn121090-20230902-00101
- VernacularTitle: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
- Author:
Lu XU
1
;
Qinglin HU
;
Chen YANG
;
Miao CHEN
;
Bing HAN
Author Information
1. 北京协和医院血液内科，北京 100730
- Keywords:
Roxadustat;
Non-severe aplastic anemia;
Refractory;
Efficacy;
Safety
- From:
Chinese Journal of Hematology
2024;45(3):264-270
- CountryChina
- Language:Chinese
-
Abstract:
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 grade Ⅰ-Ⅱ and 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.
