Real world efficacy of luspatercept in patients with lower-risk myelodysplastic syndromes/neoplasms (MDS); a single center study in a heavily pretreated cohort

Anemia leads to transfusion dependence and decreases quality of life in LR-MDS patients. Our study retrospectively evaluates the efficacy and safety of luspatercept in the real-world treatment of anemia in LR-MDS, and the impact of patient and disease characteristics on hematologic improvement erythroid (HI-E). Baseline patient and disease characteristics, and transfusion burden (TB) were captured. HI-E was assessed in patients with ≥16 weeks of therapy per IWG 2018 criteria. Luspatercept achieved high rates (58.1%) of transfusion independence (TI) in heavily pretreated LR-MDS patients (median 58.3 weeks). Response was associated with serum EPO <100 mU/ml, but not with any other baseline characteristic. Adverse effects, including shortness of breath, falls, fatigue, and hypertension, precipitated discontinuation in 8%. Eleven percent progressed to HR-MDS or AML. Median overall survival was not reached and did not differ between responders and nonresponders (p=NS).