Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context

Summary: Background: Recombinant human thrombopoietin (rhTPO) at a fixed dose of 300 U/kg/day for 2 weeks has demonstrated good efficacy and safety in adults with immune thrombocytopenia (ITP). This trial aimed to develop a flexible and personalized rhTPO regimen that ensures efficacy and safety be...

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Main Authors: Yunfei Chen, Ting Sun, Da Gao, Wei Wang, Zeping Zhou, Guangxun Gao, Yi Wang, Hu Zhou, Yanping Song, Yinghui Lai, Zhenyu Yan, Jinsong Yan, Jie Bai, Lei Zhang
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Language:English
Published: Elsevier 2025-09-01
Series:EClinicalMedicine
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Online Access:http://www.sciencedirect.com/science/article/pii/S2589537025003918
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author Yunfei Chen
Ting Sun
Da Gao
Wei Wang
Zeping Zhou
Guangxun Gao
Yi Wang
Hu Zhou
Yanping Song
Yinghui Lai
Zhenyu Yan
Jinsong Yan
Jie Bai
Lei Zhang
author_facet Yunfei Chen
Ting Sun
Da Gao
Wei Wang
Zeping Zhou
Guangxun Gao
Yi Wang
Hu Zhou
Yanping Song
Yinghui Lai
Zhenyu Yan
Jinsong Yan
Jie Bai
Lei Zhang
author_sort Yunfei Chen
collection DOAJ
description Summary: Background: Recombinant human thrombopoietin (rhTPO) at a fixed dose of 300 U/kg/day for 2 weeks has demonstrated good efficacy and safety in adults with immune thrombocytopenia (ITP). This trial aimed to develop a flexible and personalized rhTPO regimen that ensures efficacy and safety beyond previous fixed dose, with eltrombopag as an active comparator. Methods: The TE-ITP trial was conducted in 12 centers across China. Adult ITP patients with platelet count <30 × 109/L were randomised (2:1) to receive rhTPO or eltrombopag. The initial dose in patients with baseline platelet count of 20–30 × 109/L versus <20 × 109/L was 300 versus 600 U/kg/day for rhTPO and 25 versus 50 mg/day for eltrombopag, respectively. Dosage was adjusted weekly according to platelet count, with maximum of 600 U/kg/day for rhTPO and 75 mg/day for eltrombopag. The primary endpoint was the time to first platelet count ≥50 × 109/L. The trial is registered on ClinicalTrials.gov (NCT05583838). Findings: Between November 22, 2022 and January 16, 2024, the trial enrolled 157 patients (median age: 52 years; 104 women): 105 and 52 in the rhTPO and eltrombopag groups, respectively. Baseline platelet count was <20 × 109/L in 57.1% (60/105) and 57.7% (30/52) in the rhTPO and eltrombopag groups, respectively. The median time to the first platelet count ≥50 × 109/L was 7 days (95% CI 6.0–7.0) in the rhTPO group versus 15 days (95% CI 9.0–25.0) in the eltrombopag group (p < 0.001). The risk of bleeding was lower in the rhTPO group (OR 0.523, 95% CI 0.360–0.758; p < 0.001). Adverse events occurred in 45.7% (48/105) and 60.8% (31/52) in the rhTPO and eltrombopag groups, respectively. Interpretation: The optimised rhTPO regimen, with individualized dosing based on platelet response, showed faster platelet elevation and lower bleeding risk than eltrombopag. Funding: This trial was supported by grants from the CAMS Innovation Fund for Medical Sciences (CIFMS) (2023-I2M-2-007), Noncommunicable Chronic Diseases-National Science and Technology Major Project (2023ZD0500803), National Natural Science Foundation of China (82430010), Tianjin Municipal Science and Technology Commission Grant (24ZXZSSS00230).
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spelling doaj-art-2f90acda07b84dc7b23d828658ae93c22025-08-22T04:57:17ZengElsevierEClinicalMedicine2589-53702025-09-018710345910.1016/j.eclinm.2025.103459Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in contextYunfei Chen0Ting Sun1Da Gao2Wei Wang3Zeping Zhou4Guangxun Gao5Yi Wang6Hu Zhou7Yanping Song8Yinghui Lai9Zhenyu Yan10Jinsong Yan11Jie Bai12Lei Zhang13State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College, Tianjin, ChinaState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College, Tianjin, ChinaDepartment of Hematology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, ChinaDepartment of Hematology, The Second Affiliated Hospital of Harbin Medical University, Harbin, ChinaDepartment of Hematology, The Second Affiliated Hospital of Kunming Medical University, Kunming, ChinaDepartment of Hematology, The First Affiliated Hospital of Airforce Medical University, Xi'an, ChinaDepartment of Hematology, Shaanxi Provincial People's Hospital, Xi'an, ChinaDepartment of Hematology, Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Zhengzhou, ChinaInstitute of Hematology, Central Hospital of Xi'an, Xian, ChinaDepartment of Hematology, The Second Affiliated Hospital of Guangxi Medical University, Nanning, ChinaDepartment of Hematology, North China University of Science and Technology Affiliated Hospital, Tangshan, ChinaDepartment of Hematology, The Second Hospital of Dalian Medical University, Dalian, ChinaDepartment of Hematology, The Second Hospital of Tianjin Medical University, Tianjin, ChinaState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College, Tianjin, China; Tianjin Institutes of Health Science, Tianjin, China; School of Population Medicine and Public Health, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China; Corresponding author. State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College, Tianjin 300020, China.Summary: Background: Recombinant human thrombopoietin (rhTPO) at a fixed dose of 300 U/kg/day for 2 weeks has demonstrated good efficacy and safety in adults with immune thrombocytopenia (ITP). This trial aimed to develop a flexible and personalized rhTPO regimen that ensures efficacy and safety beyond previous fixed dose, with eltrombopag as an active comparator. Methods: The TE-ITP trial was conducted in 12 centers across China. Adult ITP patients with platelet count <30 × 109/L were randomised (2:1) to receive rhTPO or eltrombopag. The initial dose in patients with baseline platelet count of 20–30 × 109/L versus <20 × 109/L was 300 versus 600 U/kg/day for rhTPO and 25 versus 50 mg/day for eltrombopag, respectively. Dosage was adjusted weekly according to platelet count, with maximum of 600 U/kg/day for rhTPO and 75 mg/day for eltrombopag. The primary endpoint was the time to first platelet count ≥50 × 109/L. The trial is registered on ClinicalTrials.gov (NCT05583838). Findings: Between November 22, 2022 and January 16, 2024, the trial enrolled 157 patients (median age: 52 years; 104 women): 105 and 52 in the rhTPO and eltrombopag groups, respectively. Baseline platelet count was <20 × 109/L in 57.1% (60/105) and 57.7% (30/52) in the rhTPO and eltrombopag groups, respectively. The median time to the first platelet count ≥50 × 109/L was 7 days (95% CI 6.0–7.0) in the rhTPO group versus 15 days (95% CI 9.0–25.0) in the eltrombopag group (p < 0.001). The risk of bleeding was lower in the rhTPO group (OR 0.523, 95% CI 0.360–0.758; p < 0.001). Adverse events occurred in 45.7% (48/105) and 60.8% (31/52) in the rhTPO and eltrombopag groups, respectively. Interpretation: The optimised rhTPO regimen, with individualized dosing based on platelet response, showed faster platelet elevation and lower bleeding risk than eltrombopag. Funding: This trial was supported by grants from the CAMS Innovation Fund for Medical Sciences (CIFMS) (2023-I2M-2-007), Noncommunicable Chronic Diseases-National Science and Technology Major Project (2023ZD0500803), National Natural Science Foundation of China (82430010), Tianjin Municipal Science and Technology Commission Grant (24ZXZSSS00230).http://www.sciencedirect.com/science/article/pii/S2589537025003918Immune thrombocytopeniaRecombinant human thrombopoietinEltrombopagIndividualized dosingPlatelet count
spellingShingle Yunfei Chen
Ting Sun
Da Gao
Wei Wang
Zeping Zhou
Guangxun Gao
Yi Wang
Hu Zhou
Yanping Song
Yinghui Lai
Zhenyu Yan
Jinsong Yan
Jie Bai
Lei Zhang
Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
EClinicalMedicine
Immune thrombocytopenia
Recombinant human thrombopoietin
Eltrombopag
Individualized dosing
Platelet count
title Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
title_full Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
title_fullStr Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
title_full_unstemmed Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
title_short Dose-optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study)Research in context
title_sort dose optimised recombinant human thrombopoietin versus eltrombopag in patients with immune thrombocytopenia a multicenter randomised controlled trial the te itp study research in context
topic Immune thrombocytopenia
Recombinant human thrombopoietin
Eltrombopag
Individualized dosing
Platelet count
url http://www.sciencedirect.com/science/article/pii/S2589537025003918
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