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ASH 2020 | Phase II trial of luspatercept in myelofibrosis-associated anemia

Ruben Mesa, MD, UT Health San Antonio MD Anderson Cancer Center, San Antonio, TX, discusses the results of a trial (NCT03194542) of luspatercept to treat anemia in myelofibrosis (MF). Myelofibrosis is characterized by progressive bone marrow failure, with approximately 60% of patients developing anemia within a year of diagnosis. These patients, who typically rely on transfusions, have a worse quality of life and poor survival outcomes. To address the unmet need for MF-associated anemia therapy, luspatercept, an erythroid maturation agent, was trialed in the Phase II ACE-536-MF-001 trial. The treatment was well tolerated and there was evidence of durable activity in patients with anemia, either as a single agent or in combination with ruxolitinib. These longer-term results support the use of luspatercept for MF-associated anemia. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.