Advances and Clinical Applications of Thrombopoietic Agents

Abstract

Thrombocytopenia remains a formidable clinical challenge across a broad spectrum of medical conditions, as it substantially elevates hemorrhagic risk and complicates established therapeutic protocols. Although platelet transfusions have traditionally served as the cornerstone of management, their utility is frequently constrained by transient efficacy and inherent logistical burdens. The emergence of thrombopoietic agents, particularly second-generation thrombopoietin receptor agonists (TPO-RAs), has shifted the treatment landscape by offering a more direct and targeted way to stimulate platelet production. This review evaluates the mechanisms and clinical utility of contemporary therapies, spanning from recombinant human thrombopoietin (rhTPO) and established TPO-RAs (eltrombopag, romiplostim, and herombopag) to emerging classes such as spleen tyrosine kinase (SYK) and Bruton tyrosine kinase (BTK) inhibitors. We delineate their specific roles in the management of immune thrombocytopenia (ITP), chemotherapy-induced thrombocytopenia (CIT), chronic liver disease (CLD), and aplastic anemia (AA). Despite these advancements, challenges including inconsistent response rates, long-term safety considerations, and accessibility barriers persist. Looking ahead, the field is pivoting toward personalized management strategies and next-generation agents to further refine patient care and optimize clinical outcomes.

Keywords

Thrombocytopenia / Recombinant human thrombopoietin (rhTPO) / Thrombopoietin receptor agonist (TPO-RA) / Immune thrombocytopenia (ITP) / Chemotherapy-induced thrombocytopenia (CIT) / Aplastic anemia (AA) / Clinical management

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Funding

the Joint Fund for Innovation and Development of Natural Science of Hubei Province(2025AFD824)

he National High Technology Research and Development Program of China (2021YFA1101504)

Anhui Provincial Key Research and Development Plan(2022BCA017)

RIGHTS & PERMISSIONS

The Author(s), under exclusive licence to the Huazhong University of Science and Technology