Hetrombopag for the treatment of chemotherapy-induced thrombocytopenia in advanced solid tumors: A multicenter, randomized, double-blind, placebo-controlled phase III trial

作者：Shukui Qin, Yanqiao Zhang, Hongli Li, Yong Mao, Wenhui Yang, Zhong Xie, Yanyan Liu, Lixin Wan, Yanjun Zhang, Xizhi Zhang, Xinghua Han, Lizhu Lin, Jun Yao, Yong Li, Yun Wu, Lin Liu, Mingjun Zhang, Guixiang Weng, Junyan Yu, Si Tao, Li Zhang, Zhenyang Liu, Guoping Zhang, Yong Li, Jin Xia, Xiaoli Liao, Qingshan Li, Jin Lü, Tienan Yi, Na Li, Zhitu Zhu, Xiaoyan Lin, Kaijian Lei, Jie Ma, Donglu Zhao, Junye Xiong, Runzi Li, Xiang Lin, Qian Tang, Jun Ma · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-1256 · 被引用次数：2 · 研究领域：Platelet Disorders and Treatments、Inflammatory Biomarkers in Disease Prognosis、Angiogenesis and VEGF in Cancer

Abstract Introduction: Chemotherapy-induced thrombocytopenia (CIT) is a common complication resulting from the myelosuppressive effects of cytotoxic regimens. It frequently necessitates dose reductions, treatment delays, or platelet transfusions, which could compromise the efficacy of anticancer therapy. However, no widely approved therapies are currently available to address this clinical gap. This phase III trial was designed to evaluate the efficacy and safety of hetrombopag, a potent oral thrombopoietin receptor agonist, in patients with CIT. Methods: This randomized, double-blind, placebo-controlled phase III study (ClinicalTrials.gov: NCT05864014) enrolled patients with malignancies experiencing ≥7-day chemotherapy delay due to thrombocytopenia (platelet count <75×10⁹/L) following platinum-based combination regimens. Eligible patients were randomly assigned 1:1:1 to Arm I (hetrombopag), Arm II (hetrombopag administered only prior to the initiation of the first chemotherapy cycle [C1], with subsequent switch to placebo) or the Control Arm (placebo). Stratification factors included baseline platelet count (≥50 vs <50×10⁹/L) and immune checkpoint inhibitor use. The starting dose of investigational medicinal product was 7.5 mg/day which could be titrated to a maximum of 15 mg/day. The primary endpoint was the proportion of treatment responders, defined as patients who: (1) achieved platelet recovery (≥100×10⁹/L) within 14 days; (2) completed C1 and maintained ...