Real-World Effectiveness of Luspatercept on Transfusion Dependence Examined by Analysis Group Researchers

January 3, 2025

Patients with myelodysplastic syndromes (MDS) – disorders that affect the ability to produce healthy blood cells – often depend on blood transfusions. Luspatercept, a novel therapy that assists with the production of red blood cells, has been clinically proven to reduce the need for transfusions among patients with MDS. However, real-world evidence (RWE) corroborating these clinical findings is limited. Given the potentially substantial health care burden associated with blood transfusions, further evaluation of the effectiveness of luspatercept in reducing the need for transfusions is critical for patients with MDS.

To advance scientific knowledge on the subject, an Analysis Group team led by Managing Principal Mihran Yenikomshian, Vice Presidents Lynn Huynh and Enrico Zanardo, and Associate Rui Song collaborated with researchers from The University of New Mexico, Bristol Myers Squibb, Viver Health, the Mayo Clinic College of Medicine and Science, and Carolina Blood and Cancer Care Associates on a real-world study describing the red blood cell transfusion status of patients with MDS who were treated with luspatercept. The researchers examined retrospective claims data and assessed the proportion of patients who did not receive transfusions after luspatercept initiation. They coauthored an article reporting that high proportions of the patients studied either achieved or maintained transfusion independence following their treatment. The researchers concluded that treatment with luspatercept “could prevent the need for [red blood cell transfusion], thereby mitigating the clinical burden associated with transfusion dependence” among patients with MDS. The real-world findings of this study were consistent with prior pivotal clinical trial results.

The article, “Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study,” was published in Leukemia Research. Results from the study were exhibited in an oral podium presentation at the American Society of Hematology (ASH) Annual Meeting and Exposition.

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