Luspatercept combined with roxadustat in the treatment of refractory myelodysplastic neoplasms with ring sideroblasts: a prospective, randomized, single-center study
10.3760/cma.j.cn121090-20241126-00484
- VernacularTitle:罗特西普联合罗沙司他治疗难治性骨髓增生异常肿瘤伴环状铁粒幼细胞的随机对照前瞻性单中心研究
- Author:
Xinyi LU
1
;
Zhuxin ZHANG
1
;
Ziwei LIU
1
;
Chen YANG
1
;
Miao CHEN
1
;
Bing HAN
1
Author Information
1. 中国医学科学院北京协和医院血液内科，北京 100730
- Publication Type:Journal Article
- Keywords:
Myelodysplastic neoplasms;
Roxadustat;
Luspatercept;
Erythroid response;
Prospective randomized controlled trial
- From:
Chinese Journal of Hematology
2025;46(7):625-630
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To evaluate the efficacy and safety of luspatercept combined with roxadustat in patients with refractory low-risk myelodysplastic neoplasms with ring sideroblasts (MDS-RS) patients.Methods:In this single-center, prospective, randomized controlled trial, patients with refractory MDS-RS were randomly assigned in a 1:2 ratio to receive either combination therapy (luspatercept + roxadustat) or luspatercept monotherapy. The primary endpoint was erythroid response at 12 weeks, while secondary endpoints included erythroid response at 24 weeks, achievement of transfusion independence ≥8 weeks within the first 12 weeks, and other hematologic indicators.Results:The combination therapy and monotherapy groups included 16 and 32 patients, respectively. Baseline demographic characteristics, laboratory tests, IPSS-R risk classification, transfusion burden, EPO levels, and previous treatment history were comparable between the two groups ( P>0.05). With similar doses of luspatercept and follow-up durations, no significant differences were observed between the groups at either 12 or 24 weeks in terms of erythroid response, transfusion independence, or other clinical indicators (all P-values>0.05). The incidence of adverse events was similar in both groups (all P-values>0.05) . Conclusion:Luspatercept combined with roxadustat shows comparable efficacy and safety to luspatercept monotherapy in the treatment of refractory low-risk MDS-RS. Clinical trial register: Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing (K3697)
