Avatrombopag for adult chronic primary immune thrombocytopenia: a randomized phase 3 trial in China

作者：Heng Mei, Hu Zhou, Ming Hou, Jing Sun, Lei Zhang, Jianmin Luo, Zhongxing Jiang, Ye Xu, Yajing Xu, Jun Lü, Hui Wang, Ai‐Min Hui, Yongchun Zhou, Yu Hu · 发表于：Research and Practice in Thrombosis and Haemostasis · 年份：2023 · DOI：10.1016/j.rpth.2023.102158 · 被引用次数：18 · 研究领域：Platelet Disorders and Treatments、Heparin-Induced Thrombocytopenia and Thrombosis、Blood groups and transfusion

Background Immune thrombocytopenia (ITP) is an autoimmune disorder with decreased platelet counts and increased bleeding risk. Objectives To evaluate the efficacy and safety of avatrombopag, a second-generation oral thrombopoietin receptor agonist, for the treatment of Chinese patients with chronic primary ITP. Methods This multicenter, randomized, double-blind, placebo-controlled phase 3 study (CTR20210431) consisted of a 6-week double-blind core treatment phase followed by a 20-week, open-label extension phase. Chinese adults with chronic primary ITP for at least 12 months and a platelet count <30 × 10 9 /L were randomized (2:1) to receive avatrombopag (initial dose of 20 mg/day) or matched placebo. The primary endpoint was the proportion of subjects with a platelet count ≥50 × 10 9 /L at week 6 of the core treatment phase in absence of rescue therapy. Results In total, 74 patients were randomized (avatrombopag: N = 48; placebo: N = 26) between March 5, 2021, and August 6, 2021; all of whom entered the extension phase (72 received avatrombopag up to 26 weeks). At week 6 of the core study, the platelet response (≥50 x 10 9 /L) rate was significantly higher in the avatrombopag group (77.1%; 95% CI, 62.7, 88.0) vs placebo (7.7%; 95% CI, 1.0, 25.1); the treatment difference was 69.4% (95% CI, 56.2, 86.3; P < .0001). During the 6-week core study, treatment-emergent adverse events were reported in 41 (85.4%) and 20 (76.9%) patients in the avatrombopag and placebo groups, respecti...