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ASH 2018: MEDALIST: Luspatercept in Patients With Myelodysplastic Syndrome Requiring Red Blood Cell Transfusion

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Key Points

  • 37.9% of patients receiving luspatercept and 13.2% percent of those receiving a placebo achieved the primary endpoint of at least 8 weeks without a need for a red blood cell transfusion.
  • 28.1% of patients receiving luspatercept and 78.1% of patients receiving a placebo achieved the secondary endpoint of at least 12 weeks without transfusion.
  • 52.9% of patients experienced either a significant reduction in the number of transfusions required or an increase in hemoglobin levels even without transfusions, compared with 11.8% percent of patients receiving placebo.

In the phase III MEDALIST clinical trial, luspatercept significantly reduced the need for frequent blood transfusions in just over half (53%) of patients with myelodysplastic syndromes (MDS) who were anemic, required regular red blood cell transfusions, and showed abnormal iron overload in red blood cell precursors (a condition known as ring sideroblasts) before the study, which was presented by Fenaux et al at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition (Abstract 1).

Among those with lower-risk MDS, anemia is the most common subtype of low blood count and causes debilitating symptoms for many patients. Available anemia drugs only work for about half of patients with lower-risk MDS-related anemia and about one-quarter of those who are dependent on red blood cell transfusions. Patients who do not respond to these drugs or have adverse reactions to them may require frequent blood transfusions to boost their supply of red blood cells and hemoglobin.

Red blood cell transfusions increase hemoglobin only temporarily, and frequent transfusions are costly and time-consuming. They can also lead to iron overload, especially in the heart and liver.

“Anemia and the chronic need for transfusions is a very big issue for these patients,” said lead study author Pierre Fenaux, MD, PhD, of Hôpital Saint-Louis in Paris. “With low hemoglobin levels, patients are tired all the time and have an increased risk of falls and cardiovascular events. When you can improve hemoglobin levels, you really see a difference in quality of life.”

MEDALIST Methods

The trial enrolled 229 adult patients with low-, very low-, or intermediate-risk MDS with ring sideroblasts. All patients had either failed to respond to available anemia drugs or were ineligible for treatment with such drugs, and required red blood cell transfusions at least every 1 to 2 months. Patients were randomly assigned 2:1 to receive luspatercept or a placebo, both administered via an injection every 3 weeks for at least 6 months.

Results

  • 37.9% of patients receiving luspatercept and 13.2% percent of those receiving a placebo achieved the primary endpoint of at least 8 weeks without a need for a red blood cell transfusion.
  • 28.1% of patients receiving luspatercept and 78.1% of patients receiving a placebo achieved the secondary endpoint of at least 12 weeks without transfusion.
  • 52.9% of patients experienced either a significant reduction in the number of transfusions required or an increase in hemoglobin levels even without transfusions, compared with 11.8% percent of patients receiving placebo.

The most common reported adverse effects with luspatercept treatment included fatigue and muscle pain, though it is difficult to determine whether these effects were related to anemia or to the drug itself. It is also unclear whether the drug would offer benefits for patients with higher-risk MDS or those without lower-risk MDS without ring sideroblasts, researchers noted, since only low- and intermediate-risk patients with ring sideroblasts were included in the trial.

Disclosure: See the study authors’ full disclosures at ash.confex.com.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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