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The following is a summary of “Real-life experience of luspatercept in transfusion-dependent lower risk myelodysplastic syndrome patients,” published in the May 2025 issue of British Journal of Hematology by Bouchla et al. 

Researchers conducted a retrospective study to evaluate the safety and efficacy of luspatercept in treating anemia among patients with transfusion-dependent (TD) and lower-risk (LR) myelodysplastic syndromes (MDS) following erythroid-stimulated agent (ESA) failure.   

They analyzed data from 98 patients with TD and LR-MDS who received luspatercept after ESA failure. The proportion of patients who stopped luspatercept due to adverse events (AEs) was the same as that observed in the MEDALIST study.   

The results showed that 44.3% of patients with TD and LR-MDS who completed 24 weeks of follow-up achieved transfusion independence for more than 8 weeks, compared to 38% in the MEDALIST trial. This improved outcome was likely influenced by the inclusion of patients with reduced baseline transfusion requirements. All hematologic responses occurred within 8 months of initiating luspatercept, with many responses sustained even among those with a high transfusion burden. Additionally, achieving a response to luspatercept and having fewer than 2 mutations were both independent predictors of prolonged overall survival.   

Investigators concluded the confirmation of luspatercept’s safety and efficacy in patients with TD and LR-MDS who had experienced ESA failure in a real-world setting. 

Source: onlinelibrary.wiley.com/doi/10.1111/bjh.20102