Introduction

MDS, RBC, anemia, bone marrow disorders, peripheral cytopenias
MYELODYSPLASTIC SYNDROMES, BONE MARROW DISORDER, ANEMIA, ACUTE MYELOID LEUKEMIA
Myelodysplastic syndromes (MDS) are a heterogenous group of clonal bone marrow disorders characterized by inadequate hematopoiesis; peripheral cytopenias, particularly anemia; and an increased risk of progression to acute myeloid leukemia [Real-world, observational studies have shown an inverse relationship between the burden of ongoing RBC transfusion and survival in patients with anemia due to LR-MDS, even among those with low initial RBC transfusion burden [Eliminating or reducing the need for RBC transfusions could improve the overall health-related quality of life of patients with LR-MDS while also reducing health care resource use and associated costs. However, very few therapeutic options are available to these patients after failure of erythropoiesis-stimulating agents (ESAs). Alternative treatments are a priority, one of which is luspatercept, a first-in-class recombinant fusion protein that binds select transforming growth factor β superfamily ligands thus inhibiting downstream Smad2/3 signaling, which enables late-stage erythroid blast differentiation and erythroid maturation [Translating the findings from the MEDALIST study to clinical and health policy decision-making requires broader considerations of real-world patients and clinical scenarios. It is important to understand how luspatercept may reduce RBC transfusion burden relative to the patient’s level of transfusion burden before initiation of treatment. In addition, the potential clinical benefits of continuing luspatercept treatment beyond week 25, with the aim of achieving RBC-TI or substantially reducing anemia burden (for those who remained transfusion dependent at week 25), are not well understood and would be relevant to clinicians to help guide treatment decision-making.In this post hoc analysis of MEDALIST, we evaluated the impact of luspatercept on cumulative RBC transfusion units and RBC transfusion visits over time for treated patients based on their pretreatment transfusion burden levels. Furthermore, we evaluated the clinical benefits of continuing luspatercept treatment beyond week 25 in patients who had not achieved RBC-TI for ≥ 8 weeks during the first 24 weeks of MEDALIST.