Myelodysplastic Syndromes

Chicago, IL—Interim results from the phase 3 COMMANDS trial demonstrated that first-line treatment with luspatercept-aamt (Reblozyl) increased hemoglobin levels and enabled nearly twice as many patients with transfusion-dependent, lower-risk myelodysplastic syndromes (MDS) to avoid red blood cell transfusions during the first 24 weeks of the study compared with standard-of-care epoetin alfa.
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The investigational drug pevonedistat in combination with azacitidine injection (Vidaza) leads to longer event-free survival (EFS) and a higher complete response rate than azacitidine alone in patients with high-risk myelodysplastic syndromes (MDS), according to results from a phase 2, open-label, international clinical trial. The findings were presented by Mikkael A. Sekeres, MD, MS, Director, Leukemia Program, Cleveland Clinic, OH, at ASH 2020.
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Patients with anemia and lower-risk myelodysplastic syndromes (MDS) in whom first-line erythropoiesis-stimulating agents (ESAs) are not effective generally become transfusion dependent. Luspatercept-aamt (Reblozyl), a recombinant fusion protein that binds transforming growth factor beta superfamily ligands to reduce SMAD2 and SMAD3 signaling, was approved by the FDA in November 2019 and has been studied in this patient population with promising results.

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San Diego, CA—Luspatercept, a first-in-class erythroid maturation agent, significantly reduced the need for blood transfusions in more than 50% of patients with myelodysplastic syndromes (MDS) who were anemic, required regular red blood cell (RBC) transfusions, and/or had abnormal iron overload.
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San Diego, CA—A phase 2 expansion-cohort clinical trial has shown encouraging results with the combination of oral rigosertib, a novel styryl benzyl sulfone, and azacitidine (Vidaza) in patients with heavily pretreated myelodysplastic syndromes (MDS) compared with azacitidine monotherapy, according to data presented at ASH 2018.
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Traditional myeloablative conditioning remains the standard of care for preparing patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) for transplant, according to a randomized trial from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). The study, presented as a late-breaker at ASH 2015, was halted after a reduced-­intensity conditioning (RIC) regimen proved less effective.
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