Recombinant Human Thrombopoietin Reduces the Need for Platelet Transfusion in Patients With Chronic Liver Disease and Thrombocytopenia

ABSTRACT Chronic liver disease (CLD)‐related thrombocytopenia can limit the feasibility of invasive procedures. Recombinant human thrombopoietin (rhTPO) has demonstrated a favorable safety profile without hepatotoxicity. We evaluated the efficacy and safety of rhTPO in patients with CLD‐related thrombocytopenia who were undergoing elective invasive procedures. In this multicenter, randomized (2:1), double‐blind, placebo‐controlled phase III trial, 120 adult Chinese patients with CLD‐related thrombocytopenia (platelet count < 50 × 10 9 /L) received rhTPO ( n = 80) or placebo ( n = 40) once daily for up to 5 or 7 days. The primary endpoint was the proportion of patients with sustained platelet counts ≥ 50 × 10 9 /L from 24 h before invasive procedure to 7 days post‐procedure, without requiring emergency bleeding management. The primary endpoint was achieved by 85.0% of patients in the rhTPO group versus 12.5% in the placebo group ( p < 0.0001). Preoperatively, platelet counts ≥ 50 × 10 9 /L were achieved in 92.5% and 20.0% of patients in the rhTPO and placebo groups, respectively ( p < 0.0001). Platelet transfusion was avoided in 92.5% of rhTPO‐treated patients versus 25.0% of placebo‐treated patients ( p < 0.0001). The median duration of platelet counts ≥ 50 × 10 9 /L was significantly longer with rhTPO than with placebo (21.0 vs. 3.0 days, p = 0.0007). Treatment‐related treatment‐emergent adverse events (TEAEs) occurred in 12.5% of patients in both the rhTPO and placebo groups. No treatment‐related serious adverse events were reported. Overall, rhTPO was effective and well tolerated in patients with CLD‐related thrombocytopenia and may represent a viable therapeutic option for those undergoing elective invasive procedures. Trial Registration: www.chinadrugtrials.org.cn : number CTR20230919.

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Journal
American Journal of Hematology
Published
2026-07-28
DOI
https://doi.org/10.1002/ajh.70444
Primary Topic
Platelet Disorders and Treatments
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article
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article

Recombinant Human Thrombopoietin Reduces the Need for Platelet Transfusion in Patients With Chronic Liver Disease and Thrombocytopenia

Zhan Zeng, Yanyan Yu, 林科灿, Jinglan Jin et al.
American Journal of Hematology
Platelet Disorders and Treatments
article

Recombinant Human Thrombopoietin Reduces the Need for Platelet Transfusion in Patients With Chronic Liver Disease and Thrombocytopenia

Zhan Zeng, Yanyan Yu, 林科灿, Jinglan Jin, Yongning Xin, Mingqin Lu, Jiaping Li, Xiaoyuan Xu, Zhou Zhongyin, Huiguo Ding, Wen Xie, Jinlin Hou, Rongkuan Li, Rongshu Shi, Yongjian Zhou, Ning Lin, Tao Han, Pingguo Liu, Liaoyun Zhang, Yuemin Nan, Si Chen, Kai Wang, Jun Chen, Dazhi Zhang, Ping An, Youwen Tan, Yi Kang, Sikui Wang, Xinhua Luo, Yifan Han, Qingfang Xiong, Yunfeng Shan, Zhen Liu, Zujiang Yu, Jifang Sheng, Yan Huang, Zhili Wen, Jia Shang, Yu Chen, Jinghang Xu, Hong Wu, Zheng Lu, Jinhui Yang, Wei Wang, Li Yang, Yujuan Guan
article en

Abstract

ABSTRACT Chronic liver disease (CLD)‐related thrombocytopenia can limit the feasibility of invasive procedures. Recombinant human thrombopoietin (rhTPO) has demonstrated a favorable safety profile without hepatotoxicity. We evaluated the efficacy and safety of rhTPO in patients with CLD‐related thrombocytopenia who were undergoing elective invasive procedures. In this multicenter, randomized (2:1), double‐blind, placebo‐controlled phase III trial, 120 adult Chinese patients with CLD‐related thrombocytopenia (platelet count < 50 × 10 9 /L) received rhTPO ( n = 80) or placebo ( n = 40) once daily for up to 5 or 7 days. The primary endpoint was the proportion of patients with sustained platelet counts ≥ 50 × 10 9 /L from 24 h before invasive procedure to 7 days post‐procedure, without requiring emergency bleeding management. The primary endpoint was achieved by 85.0% of patients in the rhTPO group versus 12.5% in the placebo group ( p < 0.0001). Preoperatively, platelet counts ≥ 50 × 10 9 /L were achieved in 92.5% and 20.0% of patients in the rhTPO and placebo groups, respectively ( p < 0.0001). Platelet transfusion was avoided in 92.5% of rhTPO‐treated patients versus 25.0% of placebo‐treated patients ( p < 0.0001). The median duration of platelet counts ≥ 50 × 10 9 /L was significantly longer with rhTPO than with placebo (21.0 vs. 3.0 days, p = 0.0007). Treatment‐related treatment‐emergent adverse events (TEAEs) occurred in 12.5% of patients in both the rhTPO and placebo groups. No treatment‐related serious adverse events were reported. Overall, rhTPO was effective and well tolerated in patients with CLD‐related thrombocytopenia and may represent a viable therapeutic option for those undergoing elective invasive procedures. Trial Registration: www.chinadrugtrials.org.cn : number CTR20230919.

American Journal of Hematology
Qingdao University (CN), Hebei Medical University (CN), Jiangsu University (CN), Fujian Medical University (CN), Central South University (CN), Nanchang University (CN), Sun Yat-sen University (CN), Shanxi Medical University (CN), Zunyi Medical University (CN), Capital Medical University (CN), Dalian Medical University (CN), Jilin University (CN), Peking University (CN), Sichuan University (CN), Fudan University (CN), Kunming Medical University (CN), Wenzhou Medical University (CN), First Affiliated Hospital of Wenzhou Medical University (CN), Wuhan University (CN), Third Hospital of Hebei Medical University (CN), West China Hospital of Sichuan University (CN), Liuzhou General Hospital (CN), Second Affiliated Hospital of Chongqing Medical University (CN), Second Affiliated Hospital of Dalian Medical University (CN), Central People's Hospital of Zhanjiang (CN), Second Affiliated Hospital of Nanchang University (CN), Affiliated Hospital of Guizhou Medical University (CN), Tianjin People's Hospital (CN), Mengchao Hepatobiliary Hospital (CN), Guangzhou First People's Hospital (CN), Qingdao Municipal Hospital (CN), Zhongshan Hospital of Xiamen University (CN), First Hospital of Jilin University (CN), First Hospital of Shanxi Medical University (CN), Zhongshan Hospital (CN), The First Affiliated Hospital, Sun Yat-sen University (CN), Peking University First Hospital (CN), Henan Provincial People's Hospital (CN), Second Affiliated Hospital of Fujian Medical University (CN), Renmin Hospital of Wuhan University (CN), Zhenjiang City Fourth People's Hospital (CN), Beijing YouAn Hospital (CN), First Affiliated Hospital of Bengbu Medical College (CN), Beijing Ditan Hospital (CN), Nanjing Second Hospital (CN), Guizhou Provincial People's Hospital (CN), Guangzhou Eighth People's Hospital (CN), First Affiliated Hospital of Zhengzhou University (CN), The Sixth People's Hospital of Shenyang (CN), Xiangya Hospital Central South University (CN), Liaocheng People's Hospital (CN), Affiliated Hospital of Zunyi Medical College (CN), Shenzhen Third People’s Hospital (CN), First Affiliated Hospital Zhejiang University (CN), Southern Medical University (CN), Zhejiang University (CN), Chongqing Medical University (CN), Guangzhou Medical University (CN)
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Openalex Percentile: Top 9%
Platelet Disorders and Treatments
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