Real-World Analysis Evaluates Luspatercept in LR-MDS

By Cecilia Brown - Last Updated: June 8, 2023

Most patients with lower-risk myelodysplastic syndromes (LR-MDS) who had a low to moderate transfusion burden and received luspatercept achieved transfusion independence for at least eight weeks, according to results from a real-world analysis.

Sudipto Mukherjee, MD, PhD, MPH, of the Cleveland Clinic and colleagues conducted the study and presented their findings at the 2023 American Society of Clinical Oncology Annual Meeting.

They assessed clinical outcomes of patients who had LR-MDS with ring sideroblasts or MDS with ring sideroblasts and thrombosis who received luspatercept in US community practices. They used physician-abstracted chart data from community oncology clinics to conduct the retrospective, observational cohort study. It included adults who were diagnosed with LR-MDS on or after January 1, 2015. All patients in the study received luspatercept for at least three months with at least three months of follow-up data.

The researchers defined transfusion burden by the number of sessions received in the eight weeks prior to luspatercept, and the lowest number of transfusions for any consecutive period of at least eight weeks during weeks one through 24 of treatment. They categorized patients as being transfusion independent, having a low transfusion burden (one to three sessions) or having a moderate transfusion burden (four to five sessions).

The study included 253 patients. Nearly all (87%) had one prior line of treatment before luspatercept and 12% had at least two lines of therapy before luspatercept. Most patients (83%) received an erythroid stimulating agent as a first-line therapy. Lack of anemia improvement was the “most common reason for discontinuing initial MDS” therapy in patients who received at least one prior therapy before luspatercept.

Most patients (82%) had a low transfusion burden at baseline, while 10% had a moderate transfusion burden and 8% were transfusion independent. The median duration of luspatercept treatment was 10.8 months.

Nearly all (95%) of patients remained transfusion independent during weeks one through 24 of therapy, and 87% who had low or moderate transfusion burden at baseline achieved transfusion independence for at least eight weeks.

“Most [patients] with LR-MDS received [first-line erythroid stimulating agents] and over half discontinued the first [treatment] prior to luspatercept due to lack of anemia improvement, highlighting the need for alternative options to improve clinical outcomes,” Dr. Mukherjee and colleagues concluded. “Consistent with data from the MEDALIST trial, 87% of [patients] with [low or moderate transfusion burden] who received luspatercept achieved [transfusion independence] for [at least eight weeks], supporting its clinical benefit in routine clinical practice.”


Mukherjee S, Brown-Bickerstaff C, Falkenstein A, et al.Hematologic and transfusion outcomes in patients (pts) with lower-risk myelodysplastic syndromes (LR-MDS) receiving luspatercept: real-world assessment in the community practice setting. Abstract #7057. Presented at the 2023 American Society of Clinical Oncology Annual Meeting; June 2-6, 2023; Chicago, Illinois.

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