[Luspatercept combined with roxadustat in the treatment of refractory myelodysplastic neoplasms with ring sideroblasts: a prospective, randomized, single-center study]

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).