Luspatercept vs Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective therapy for hematological malignancies. Nonetheless, poor graft function remains a life-threatening complication after allo-HSCT, characterized by persistent cytopenias despite evidence of complete donor chimerism. Poor erythroid engraftment refers to hemoglobin <70g/L and inability to detach from red blood cell transfusion after 28 days of transplantation, which is associated with increased bleeding events and shorter survival. Current treatment methods such as epoetin or repeated red-cell transfusions are not effective for poor erythroid engraftment, with limited and transient responses. New treatment strategies are needed to enhance the response rate in patients with poor erythroid engraftment.

Luspatercept is a specific activin receptor fusion protein that reduces SMAD2 and SMAD3 signaling by binding specific transforming growth factor β (TGF-β) superfamily ligands, thereby allowing erythrocyte maturation through late-stage erythroblast differentiation. Retrospective studies suggested that luspatercept showed efficacy in patients with anemia post-transplantation or poor erythroid engraftment. To date, there have been no studies comparing luspatercept versus epoetin for the treatment of poor erythroid engraftment. Therefore, we conducted a randomized controlled study to compared the effect of luspatercept versus epoetin in treating poor erythroid engraftment for hematological malignancies.