Baseline erythropoietin level predicts Luspatercept response in Chinese patients with myelodysplastic syndrome: a propensity score-matched analysis

Abstract

Background

Luspatercept is a novel erythroid maturation agent approved for treating transfusion-dependent anemia in myelodysplastic syndrome (MDS). However, predictive biomarkers and real-world data in Asian populations remain limited.

Methods

We conducted a retrospective cohort study of 66 MDS patients from two Chinese centers, including 22 patients treated with luspatercept and 44 matched controls. Propensity score matching (1:2) was used to adjust for age, sex, risk category, baseline hemoglobin, transfusion burden, and erythropoietin (EPO) levels. Primary endpoints were red blood cell transfusion independence (RBC-TI) ≥8 weeks and hematologic improvement–erythroid (HI-E).

Results

By week 12, 54.5 % of luspatercept-treated patients achieved RBC-TI, compared to 29.5 % in the control group (P < 0.05). Patients with baseline EPO <500 IU/mL had higher RBC-TI rates than those with EPO ≥500 IU/mL (63.6 % vs. 45.5 %). Multivariate analysis identified baseline EPO <500 IU/mL as an independent predictor of response (OR=2.36, 95 % CI: 1.24–4.52, P = 0.008). Safety was acceptable, with no treatment discontinuation due to adverse events.

Conclusion

This first matched real-world study of luspatercept in Chinese MDS patients confirms its efficacy in reducing transfusion needs and identifies baseline erythropoietin level as a clinically meaningful predictor of response. These findings support the integration of EPO-guided selection in personalized treatment strategies.