Dose-optimised recombinant human thrombopoietin <i>versus</i> eltrombopag in patients with immune thrombocytopenia: a multicenter, randomised controlled trial (The TE-ITP Study).

Chen, Yunfei; Sun, Ting; Gao, Da; Wang, Wei; Zhou, Zeping; Gao, Guangxun; Wang, Yi; Zhou, Hu et al. · EClinicalMedicine · 2025

rct · Level II

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Abstract

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<sup>9</sup>/L were randomised (2:1) to receive rhTPO or eltrombopag. The initial dose in patients with baseline platelet count of 20-30 × 10<sup>9</sup>/L <i>versus</i> <20 × 10<sup>9</sup>/L was 300 <i>versus</i> 600 U/kg/day for rhTPO and 25 <i>versus</i> 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<sup>9</sup>/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<sup>9</sup>/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<sup>9</sup>/L was 7 days (95% CI 6.0-7.0) in the rhTPO group <i>versus</i> 15 days (95% CI 9.0-25.0) in the eltrombopag group (<i>p</i> < 0.001). The risk of bleeding was lower in the rhTPO group (OR 0.523, 95% CI 0.360-0.758; <i>p</i> < 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).