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How might luspatercept benefit patients with myelofibrosis?

By Jennifer Reilly

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Aaron GerdsAaron Gerds

Aug 2, 2024

Learning objective: After reading this article, learners will be able to explain the latest data on how to better identify patients who could benefit the most from luspatercept.


Test your knowledge! Take our quick quiz before and after you read this article to find out if you improved your knowledge. Results help us to improve content and continually provide open-access education.

Question 1 of 2

What percentage of patients with myelofibrosis will become anemic within the first year of diagnosis?

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B

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D

The MPN Hub was pleased to speak to Aaron Gerds, Cleveland Clinic Taussig Cancer Institute, Cleveland, US. We asked, How might luspatercept benefit patients with myelofibrosis (MF)?

How might luspatercept benefit patients with myelofibrosis?

Aaron Gerds opens by discussing the prevalence and issue of anemia in patients with MF, noting that almost all patients will become anemic at some point in the course of their disease. Gerds discusses luspatercept as a treatment option in this indication, sharing the latest clinical trial data from the phase II ACE-536-MF-001 study of luspatercept for the treatment of anemia in patients with myelofibrosis; emphasizing the benefit of a reduction in transfusion dependency for patients treated with luspatercept. This interview closes with a look to ongoing clinical trials, including the INDEPENDENCE (NCT04717414) study and the implications of these data on future management strategies for anemic myelofibrosis.

During the interview Gerds made the following key points:

  • Approximately 40% of patients with myelofibrosis are anemic at the time of diagnosis and 60% will become anemic within the first year of diagnosis.1
  • Anemia typically results in poorer outcomes for patients and can lead to transfusion dependency, which requires regular transfusions.
  • Luspatercept is an erythroid maturation agent that is approved by the U.S. Food and Drug Administration (FDA) to treat anemia in certain patients with myelodysplastic syndromes and beta thalassemia.
  • Luspatercept is currently being investigated as part of the phase II ACE-536-MF-001 study and the phase III INDEPENDENCE study.
  • In the ACE-536-MF-001 study, amongst patients who were transfusion dependent, approximately 50% of patients experienced at least a 50% reduction in transfusion burden from baseline.2
  • In the ame trial, following treatment with luspatercept, one in four patients who were getting regular transfusions, no longer required these transfusions.2
  • The NCCN guidelines now endorse luspatercept as a treatment option for patients with anemic myelofibrosis.

Listen to the interview as a podcast

How might luspatercept benefit patients with myelofibrosis?

This educational resource is independently supported by BMS. All content is developed by SES in collaboration with an expert steering committee; funders are allowed no influence on the content of this resource.

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