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ASH 2021 | Long-term follow-up of luspatercept: response duration and progression to AML

The Phase III randomized, placebo-controlled MEDALIST trial (NCT02631070) investigated the use of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) with ring sideroblasts. Results published in 2020 showed that the first-in-class erythroid maturation agent significantly reduced transfusion burden versus placebo, and was subsequently approved by the FDA for the treatment of anemia in adults with lower-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. Uwe Platzbecker, MD, University Clinic Leipzig, Leipzig, Germany, shares an overview of additional findings from the long-term follow-up data of MEDALIST. Overall, the 48-week follow-up results confirmed published findings. Patients remained on treatment longer and had more durable and repeated episodes of transfusion independence compared to placebo. No new safety signals were observed and no difference in progression to acute myeloid leukemia (AML) was seen between the two trial arms. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.