Treatment with ruspatercept improves red blood cell counts in patients with myelodysplastic syndrome

Treatment with ruspatercept improves red blood cell counts and red blood cell responses compared to treatment with epoetin alfa in patients with myelodysplastic syndrome (MDS), eliminating the need for routine blood transfusions in the majority of patients. Results from the Phase III COMMANDS trial, led by researchers at The University of Texas MD Anderson Cancer Center, were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting.

This study evaluated the efficacy and safety of first-line treatment with luspatercept, which promotes red blood cell maturation, compared with epoetin alfa, a commonly used treatment for blood cell count reduction, when blood cell concentrations were very low. It was evaluated in transfusion-dependent patients with anemia because of the low His MDS at intermediate risk.

In this interim analysis, 58.5% of patients receiving ruspatercept achieved the primary endpoint of independence from red blood cell transfusions, compared with 31.2% of patients receiving epoetin alfa. Within his first 24 weeks of therapeutic transfusion, 47.6% of his ruspatercept patients achieved transfusion independence, compared with 29.2% of his patients receiving epoetin alfa. Additionally, 74.1% of patients receiving ruspatercept had hematologic improvement in red blood cell response over his 8 weeks compared to 51.3% of patients receiving epoetin alfa.

Patients with myelodysplastic syndrome often experience anemia that requires frequent red blood cell transfusions. In this study, significant improvements in patient red blood cell counts were observed with luspatercept, representing a promising advance in extending life in these patients. ”

Guillermo García Manero, M.D., Professor of Leukemia, Principal Investigator of the Study

Myelodysplastic syndromes are a group of disorders in which the bone marrow does not produce enough healthy blood cells, including red blood cells. Patients with MDS often experience symptoms such as anemia, fatigue, shortness of breath, and increased vulnerability to infections.

Anemia is common, so most people require regular red blood cell transfusions. Some cases of MDS can progress to acute myeloid leukemia (AML). Ruspatercept is a novel agent that enables late maturation of red blood cells. Ruspatercept helps restore normal red blood cell production by targeting the TGF-β signaling pathway.

The trial enrolled 301 patients at 226 centers. Patients were randomized to receive subcutaneous luspatercept every 3 weeks or subcutaneous epoetin alfa every 24 weeks. Patient characteristics were balanced in both treatment groups.

All-grade treatment-related adverse events occurred in 30.3% of patients in the ruspatercept group and 17.6% of patients in the epoetin alfa group. Eight patients (4.5%) who received ruspatercept discontinued treatment due to treatment-related adverse events. AML progression was reported in 4 patients who received ruspatercept and 5 patients who received epoetin alfa. The safety profile was consistent with previous studies of this drug.

“These results demonstrate for the first time that an innovative treatment is superior to epoetin alfa in efficacy,” said Garcia-Manero. “These results are encouraging as ruspatercept is an innovative treatment that has the potential to become the new standard of care for patients with transfusion-dependent myelodysplastic syndrome.”

Patients in this study will continue to be followed long-term to determine overall survival, transfusion independence, and frequency of progression to AML.

This study was funded by Bristol-Myers Squibb. Garcia Manero serves as a consulting/advisor to Bristol-Myers Squibb and receives research support.


University of Texas MD Anderson Cancer Center

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