Real-World, Single-Center Experience of Luspatercept in Lower-Risk MDS

Treatment with luspatercept yielded high rates of durable transfusion independence in patients with heavily-pretreated, lower-risk myelodysplastic syndromes (MDS), according to a retrospective analysis.

The lead author of the study, Marisa Kometas, MD, of the UT Southwestern Medical Center in Dallas, Texas, presented the findings at the Society of Hematologic Oncology 2024 Annual Meeting.

Dr. Kometas and colleagues noted that luspatercept response was associated with baseline serum erythropoietin levels below 100 mU/mL (P=.02), but not with baseline transfusion burden. Among 22% of responders who lost transfusion independence, 50% restored independence following dose increases.

The analysis included 37 patients with lower-risk MDS treated with luspatercept at an academic tertiary referral center from January 2020 to December 2023. Baseline characteristics of interest included prior therapies, ring sideroblast status, karyotype, gene mutations, and transfusion burden. Treatment outcomes of interest included erythroid hematologic improvement after 16 or more weeks of therapy and progression-free survival (PFS).

Among 31 patients with evaluable response data, 52% achieved erythroid improvement. Additionally, 48% of transfusion-dependent patients achieved 16 or more weeks of transfusion independence.

According to the report, the median duration of transfusion independence was 48 weeks and did not differ significantly between patients with high or low baseline transfusion burden. Luspatercept doses of 1.00, 1.33, and 1.75 mg/kg yielded equal rates of transfusion independence.

Adverse effects included shortness of breath, falls, fatigue, and hypertension. Adverse effects led to discontinuation in 8% of patients. Overall, 11% of patients progressed to high-risk MDS or to acute myeloid leukemia. Median leukemia-free overall survival was not reached and did not differ between responders and non-responders, the researchers reported.

“As luspatercept gains prominence as first-line therapy, further investigation is required to define predictors of response,” Dr. Kometas and colleagues concluded.

Reference

Kometas M, Hyak J, Hoff F, et al. Real world efficacy of luspatercept in patients with lower-risk myelodysplastic syndromes/neoplasms (LR-MDS); a single center study in a heavily pretreated cohort. Abstract #MDS-199. Presented at the Society of Hematologic Oncology 2024 Annual Meeting; September 4-7, 2024; Houston, Texas.