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

Yifan Han·Ning Lin·Jinghang Xu·Sikui Wang·Jinglan Jin·Jia Shang·Yongning Xin·Youwen Tan·Dazhi Zhang·Jinlin Hou·Ping An·Wen Xie·Yujuan Guan·Qingfang Xiong·Hong Wu·Huiguo Ding·Yu Chen·Rongkuan Li·Zhili Wen·Mingqin Lu·Jinhui Yang·Zhongyin Zhou·Yongjian Zhou·Zujiang Yu·Kecan Lin·Wei Wang·Yan Huang·Yuemin Nan·Xinhua Luo·Rongshu Shi·Yi Kang·Tao Han·Shiyan Chen·Kai Wang·Yunfeng Shan·Jiaping Li·Pingguo Liu·Zheng Lu·Jun Chen·Zhen Liu·Liaoyun Zhang·Li Yang·Jifang Sheng·Zhan Zeng·Yanyan Yu·Xiaoyuan Xu
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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 <&#x2009;50 &#xd7;&#x2009;109/L) received rhTPO (n =&#x2009;80) or placebo (n =&#x2009;40) once daily for up to 5 or 7&#x2009;days. The primary endpoint was the proportion of patients with sustained platelet counts &#x2265;&#x2009;50 &#xd7;&#x2009;109/L from 24 h before invasive procedure to 7&#x2009;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&#x2009;<&#x2009;0.0001). Preoperatively, platelet counts &#x2265;&#x2009;50 &#xd7;&#x2009;109/L were achieved in 92.5% and 20.0% of patients in the rhTPO and placebo groups, respectively (p&#x2009;<&#x2009;0.0001). Platelet transfusion was avoided in 92.5% of rhTPO-treated patients versus 25.0% of placebo-treated patients (p&#x2009;<&#x2009;0.0001). The median duration of platelet counts &#x2265;&#x2009;50 &#xd7;&#x2009;109/L was significantly longer with rhTPO than with placebo (21.0 vs. 3.0&#x2009;days, p =&#x2009;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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HumansThrombocytopeniaThrombopoietinFemalePlatelet TransfusionMaleMiddle AgedRecombinant ProteinsDouble-Blind MethodAdultAgedLiver DiseasesPlatelet CountChronic DiseaseTreatment Outcomechronic liver diseaseefficacyrecombinant human thrombopoietinsafetythrombocytopeniaJournal ArticleRandomized Controlled TrialMulticenter StudyClinical Trial, Phase III

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Yifan Han, Ning Lin, Jinghang Xu, Sikui Wang, Jinglan Jin, Jia Shang, Yongning Xin, Youwen Tan, Dazhi Zhang, Jinlin Hou, Ping An, Wen Xie, Yujuan Guan, Qingfang Xiong, Hong Wu, Huiguo Ding, Yu Chen, Rongkuan Li, Zhili Wen, Mingqin Lu, Jinhui Yang, Zhongyin Zhou, Yongjian Zhou, Zujiang Yu, Kecan Lin, Wei Wang, Yan Huang, Yuemin Nan, Xinhua Luo, Rongshu Shi, Yi Kang, Tao Han, Shiyan Chen, Kai Wang, Yunfeng Shan, Jiaping Li, Pingguo Liu, Zheng Lu, Jun Chen, Zhen Liu, Liaoyun Zhang, Li Yang, Jifang Sheng, Zhan Zeng, Yanyan Yu, Xiaoyuan Xu. 2026-07-28. Recombinant Human Thrombopoietin Reduces the Need for Platelet Transfusion in Patients With Chronic Liver Disease and Thrombocytopenia.. https://doi.org/10.1002/ajh.70444

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Adult subjectAged subjectClinical Trial, Phase III subjectDouble-Blind Method subjectFemale subjectHumans subjectJournal Article subjectJournal Article; Randomized Controlled Trial; Multicenter Study; Clinical Trial, Phase III resourceMale subjectMiddle Aged subjectMulticenter Study subjectPlatelet Count subjectPlatelet Transfusion subjectRandomized controlled trials studyefficacy subject

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