Chen Yunfei, Sun Ting, Gao Da, Wang Wei, Zhou Zeping, Gao Guangxun, Wang Yi, Zhou Hu, Song Yanping, Lai Yinghui, Yan Zhenyu, Yan Jinsong, Bai Jie, Zhang Lei
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 & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Department of Hematology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
EClinicalMedicine. 2025 Aug 21;87:103459. doi: 10.1016/j.eclinm.2025.103459. eCollection 2025 Sep.
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.
The TE-ITP trial was conducted in 12 centers across China. Adult ITP patients with platelet count <30 × 10/L were randomised (2:1) to receive rhTPO or eltrombopag. The initial dose in patients with baseline platelet count of 20-30 × 10/L <20 × 10/L was 300 600 U/kg/day for rhTPO and 25 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 × 10/L. The trial is registered on ClinicalTrials.gov (NCT05583838).
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 × 10/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 × 10/L was 7 days (95% CI 6.0-7.0) in the rhTPO group 15 days (95% CI 9.0-25.0) in the eltrombopag group ( < 0.001). The risk of bleeding was lower in the rhTPO group (OR 0.523, 95% CI 0.360-0.758; < 0.001). Adverse events occurred in 45.7% (48/105) and 60.8% (31/52) in the rhTPO and eltrombopag groups, respectively.
The optimised rhTPO regimen, with individualized dosing based on platelet response, showed faster platelet elevation and lower bleeding risk than eltrombopag.
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).
重组人血小板生成素（rhTPO）以300 U/kg/天的固定剂量给药2周，已在成人免疫性血小板减少症（ITP）患者中显示出良好的疗效和安全性。本试验旨在制定一种灵活且个性化的rhTPO治疗方案，确保其疗效和安全性优于既往固定剂量方案，并以艾曲泊帕作为活性对照药。
TE-ITP试验在中国的12个中心开展。血小板计数<30×10⁹/L的成人ITP患者被随机分组（2:1），分别接受rhTPO或艾曲泊帕治疗。基线血小板计数为20 - 30×10⁹/L和<20×10⁹/L的患者，rhTPO的初始剂量分别为300和600 U/kg/天，艾曲泊帕的初始剂量分别为25和50 mg/天。根据血小板计数每周调整剂量，rhTPO最大剂量为600 U/kg/天，艾曲泊帕最大剂量为75 mg/天。主要终点为首次血小板计数≥50×10⁹/L的时间。该试验已在ClinicalTrials.gov注册（NCT05583838）。
在2022年11月22日至2024年1月16日期间，该试验共纳入157例患者（中位年龄：52岁；女性104例）：rhTPO组105例，艾曲泊帕组52例。rhTPO组和艾曲泊帕组基线血小板计数<20×10⁹/L的患者分别占57.1%（60/105）和57.7%（30/52）。rhTPO组首次血小板计数≥50×10⁹/L的中位时间为7天（95%CI 6.0 - 7.0），艾曲泊帕组为15天（95%CI 9.0 - 25.0）（P<0.001）。rhTPO组出血风险更低（OR 0.523，95%CI 0.360 - 0.758；P<0.001）。rhTPO组和艾曲泊帕组不良事件发生率分别为45.7%（48/105）和60.8%（31/52）。
基于血小板反应进行个体化给药的优化rhTPO方案，与艾曲泊帕相比，血小板提升更快，出血风险更低。
本试验由中国医学科学院医学创新基金（CIFMS）（2023-I2M-2-007）、国家重大慢性非传染性疾病防控科技重大专项（2023ZD0500803）、国家自然科学基金（82430010）、天津市科学技术委员会资助项目（24ZXZSSS00230）资助。
