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Luspatercept: From Bench to Bedside and Beyond in the Management of Ineffective Erythropoiesis

Year: 2026

Type: article

Abstract: Luspatercept is a novel erythroid maturation agent that has emerged as a significant advancement in the management of ineffective erythropoiesis. By targeting the transforming growth factor-β superfamily signaling pathway, Luspatercept enhances late-stage erythroid differentiation. This review provides an in-depth exploration of its mechanism of action, pharmacologic properties, and clinical efficacy across multiple hematologic disorders. We summarize trial outcomes in lower-risk myelodysplastic syndromes, transfusion-dependent β-thalassemia, and myelofibrosis, highlighting improvements in erythroid response and transfusion independence. The article also discusses adverse event profiles and future directions, including ongoing trials and potential expansion of indications. Luspatercept represents a promising targeted erythroid therapy with benefits across several hematologic diseases.

Source: Journal of Hematology

DOI: 10.14740/jh2163

Authors: Dhara Popat, Rajan Desai, Sheikh Abdullah, Siddhant Jain, Poornima Ramadas

Institutions: Louisiana State University, Louisiana State University Health Sciences Center Shreveport, Feist-Weiller Cancer Center

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

OpenAlex ID: W7151094460

FWCI: 4.054

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Cited by: 1

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