Efficacy and safety of QL0911 in adult patients with chronic primary immune thrombocytopenia: A multicenter, randomized, double-blind, placebo-controlled, phase III trial

作者：Hu Zhou, Shouqing Han, Jie Jin, Ruibin Huang, Xinhong Guo, Xuliang Shen, Binghua Wang, Xin Wang, Hongxia Yao, Xin Du, Meijuan Huang, Xuehong Ran, Wei Wang, Tonghua Yang, Feng Zhang, Changcheng Zheng, Xuelan Zuo, Rong Fu, Da Gao, Zheng Ge, Ying Han, Yujie Li, Xiaoyan Kang, Yan Shi, Hou Ming · 发表于：Journal of Translational Internal Medicine · 年份：2023 · DOI：10.2478/jtim-2023-0106 · 被引用次数：13 · 研究领域：Platelet Disorders and Treatments、Biosimilars and Bioanalytical Methods、Clinical Laboratory Practices and Quality Control

Abstract Objective QL0911, a recombinant human thrombopoietin mimetic peptide-Fc fusion protein, is a romiplostim (Nplate ® ) biosimilar used to treat primary immune thrombocytopenia (ITP). This phase III study aimed to assess the efficacy and safety of QL0911 in adult patients with chronic primary ITP over a 24-week treatment period. Methods We conducted a double-blind, placebo-controlled, phase III study in patients diagnosed with primary ITP for at least 12 months who had received at least one first-line ITP treatment with no response or recurrence after treatment, or who relapsed after splenectomy at 44 sites in China. Patients were randomly allocated (2:1 ratio) to QL0911 or placebo injection subcutaneously once weekly at an initial dose of 1 μg/kg for 24 weeks. The doses were adjusted to maintain the target platelet counts from 50 × 109/L to 200 × 109/L. Patients and investigators were blinded to the assignment. The primary endpoints were the proportion of patients who achieved a durable platelet response at week 24 (platelet count, ≥ 50 × 109/L during 6 of the last 8 weeks of treatment) and safety. The study was registered at ClinicalTrials.gov (NCT05621330). Results Between October 2019 and December 2021, 216 patients were randomly assigned (QL0911,144; placebo,72). A durable platelet response was achieved by significantly more patients in the QL0911 group (61.8%, 95% CI: 53.3-69.8; P < 0.0001) than in the placebo group (0%). The mean duration of platelet responses...