Luspatercept Shows Effectiveness in ESA-Naïve Patients with Lower-Risk MDS

By Blood Cancers Today Staff Writers - Last Updated: September 5, 2023

Luspatercept is highly effective in treating erythropoietin-stimulating agent (ESA)-naïve patients with lower-risk myelodysplastic syndromes (LR-MDS), according to research presented at the Eleventh Society of Hematologic Oncology (SOHO) Annual Meeting in Houston, Texas.

In a retrospective observational cohort study, researchers led by presenter Sudipto Mukherjee MD, PhD, of the Cleveland Clinic found that nearly 95% of these patients maintained nontransfusion-dependent status or transfusion independence for at least 12 weeks during luspatercept therapy.

As there’s only limited real-world data available on the effectiveness of luspatercept in ESA-naïve patients with LR-MDS, the researchers sought in their study to describe treatment patterns and transfusion outcomes for this patient population in routine clinical practice. Using physician-abstracted data from US medical records, the researchers retrospectively analyzed data from 33 ESA-naïve adults with LR-MDS who were treated with luspatercept for at least three months on or after January 1, 2015. They excluded patients who had received prior treatment for MDS or who had received luspatercept in a clinical trial.

Of these 33 patients, 52% were male and 79% were White. The mean patient age was 70.1 years. Before receiving luspatercept, 91% of patients received at least one line of therapy; 20 patients received granulocyte-macrophage colony-stimulating factor. Patients were followed up for a median duration of 4.1 months.

In the 32 patients who had a low or moderate transfusion burden at baseline (eight weeks prior to luspatercept treatment), 97% were transfusion independent for at least 8 weeks and 94% were still transfusion dependent for at least 12 weeks during luspatercept treatment. The one patient who was nontransfusion dependent at baseline maintained that status during luspatercept treatment for at least 12 weeks, according to the researchers.

“These results suggest luspatercept, when used among ESA-naïve patients with LR-MDS, may have substantial impact on [transfusion burden],” the authors concluded.

The study was supported by Bristol-Myers Squibb, the manufacturer of luspatercept.


Mukherjee S, Brown-Bickerstaff C, Huggar D, et al. Treatment patterns and transfusion outcomes among erythropoietin-stimulating agent (ESA)-naïve patients with lower-risk myelodysplastic syndromes (LR-MDS) receiving luspatercept in routine clinical practice in the United States (US). Abstract MDS-065. Presented at the Eleventh Annual Meeting of the Society of Hematologic Oncology; September 6-9, 2023; Houston, Texas.

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