Exploring a First-line Treatment for Low-risk MDS

Luspatercept is a novel erythroid maturation agent that enhances late-stage red blood cell production by modulating TGF-β superfamily signaling, improving hemoglobin and reducing transfusion needs. Pivotal trial data demonstrated significant transfusion-independence and hemoglobin responses in lower-risk MDS patients—findings that supported FDA approval and positioned luspatercept as an important option for those with ring sideroblasts or who are ESA-refractory. Compared with ESAs, luspatercept shows benefit particularly in patients who fail or are unlikely to respond to erythropoietin-based therapy, shifting practice toward earlier consideration in selected subgroups.