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Herombopag for the treatment of persistent thrombocytopenia following hematopoietic stem cell transplantation

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Abstract

Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) stands as a pivotal treatment for hematologic malignancies, often considered the sole effective treatment option. A frequent complication following allo-HSCT is poor graft function (PGF), with one of its primary manifestations being persistent thrombocytopenia (PT), comprising prolonged isolated thrombocytopenia (PIT) and secondary failure of platelet recovery (SFPR). Conventional treatment methods have had poor efficacy and a high transplantation-associated mortality rate. In recent years, the efficacy of eltrombopag has been reported in the treatment of post-transplantation PT, and additional thrombopoietin receptor agonists (TPO-RA) have been developed. Herombopag is a next-generation TPO-RA which has strong proliferation-promoting effects on human TPO-R-expressing cells (32D-MPL) and hematopoietic progenitor cells in vitro. We reviewed eighteen patients with transplantation-associated thrombocytopenia who received herombopag when eltrombopag was ineffective or poorly tolerated and evaluated its efficacy including effects on survival. Herombopag was administered at a median time of 197 days post-transplantation. Six patients achieved complete response (CR), with a median time to CR of 56 days. Five patients achieved partial response (PR), and the median time to PR was 43 days. Seven patients were considered to have no response (NR). The overall response (OR) rate was 61.1%, and the cumulative incidence (CI) of OR was 90.2%. No patients developed herombopag-associated grade 3–4 toxicity. The median follow-up period was 6.5 months. Twelve patients survived and six patients died, with an overall survival rate of 66.7%. This is the first study to demonstrate the efficacy and safety of herombopag in transplantation-associated thrombocytopenia after failing eltrombopag, introducing a new approach in the treatment of PT following allo-HSCT.

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Funding

The study is funded by the Ningbo Social Welfare Research (No. 2022S032) and the Ningbo Natural Science Foundation (No. 2023J384).

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Authors and Affiliations

  1. Department of Hematology, The First Affiliated Hospital of Ningbo University, No. 59 Liuting Street, Ningbo, Zhejiang, 315000, People’s Republic of China

    Miao Zhou, Tongyu Li, Ping Zhang, Yanli Lai, Lixia Sheng & Guifang Ouyang

  2. Ningbo Clinical Research Center for Hematologic Malignancies, The First Affiliated Hospital of Ningbo University, No. 59 Liuting Street, Ningbo, Zhejiang, 315000, People’s Republic of China

    Miao Zhou, Tongyu Li, Ping Zhang, Yanli Lai & Lixia Sheng

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  1. Miao Zhou
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Contributions

Miao Zhou was responsible for acquiring data, its subsequent analysis and interpretation, and the manuscript’s composition. Tongyu Li was responsible for interpreting the data, and composing the manuscript. Yanli Lai and Ping Zhang were responsible for the data collection and interpretation. Lixia Sheng and Guifang Ouyang were responsible for the study’s design, as well as the analysis and interpretation of data. The final version of this study has been approved and endorsed by all participating authors.

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Correspondence to Guifang Ouyang.

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Zhou, M., Li, T., Zhang, P. et al. Herombopag for the treatment of persistent thrombocytopenia following hematopoietic stem cell transplantation. Ann Hematol 103, 1697–1704 (2024). https://doi.org/10.1007/s00277-024-05711-1

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