Hetrombopag for patients with persistent primary immune thrombocytopenia: A post-hoc analysis of a multicenter, randomized phase Ⅲ Trial

Abstract Introduction: Hetrombopag has been approved for chronic primary immune thrombocytopenia (ITP) (duration >12 months). However, its efficacy and safety in persistent ITP (duration 3–12 months) remain unexplored. Based on existing clinical data, this post-hoc analysis aimed to evaluate the efficacy and safety of hetrombopag in persistent ITP patients and compare outcomes with chronic ITP and overall ITP patients. Methods: Data were derived from a registration phase Ⅲ trial (NCT03222843) which included a 10-week randomized, double-blind, placebo-controlled treatment period (initial dose: hetrombopag 2.5 mg as HETROM-2.5 group, hetrombopag 5.0 mg as HETROM-5.0 group, or placebo once daily), a 14-week open-label treatment period, a dose-tapering to withdrawal period and a 24-week single-arm extension period. The primary endpoint was the proportion of responders (a platelet count of ≥ 50×10⁹/L) after 8 weeks of treatments. Results: Of 395 who completed the double-blinded treatment, 80 patients had persistent ITP. The proportion of persistent ITP patients who achieved primary endpoint was 68.8% in the HETROM-2.5 group and 74.3% in the HETROM-5.0 group, significantly higher than 7.7% in the placebo group. Hetrombopag also demonstrated other therapeutic benefits over placebo including reduced bleeding risk and rescue therapy need. Similar efficacy results were observed both in the chronic ITP and the overall ITP population. Furthermore, hetrombopag manifested favorable safety profile in persistent ITP patients, and safety outcomes were comparable to those in chronic and overall ITP populations. Conclusions: Hetrombopag indicated favorable safe and efficacy performance in patients with persistent ITP, aligning with results observed in chronic ITP and overall ITP patients.

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Journal
Blood
Published
2025-11-03
DOI
https://doi.org/10.1182/blood-2025-4812
Primary Topic
Platelet Disorders and Treatments
Type
article
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article

Hetrombopag for patients with persistent primary immune thrombocytopenia: A post-hoc analysis of a multicenter, randomized phase Ⅲ Trial

Jie Jin, Jianmin Luo, Linhua Yang, Heng Mei et al.
Blood
Platelet Disorders and Treatments
article

Hetrombopag for patients with persistent primary immune thrombocytopenia: A post-hoc analysis of a multicenter, randomized phase Ⅲ Trial

Jie Jin, Jianmin Luo, Linhua Yang, Heng Mei, Yun Zeng, Xuliang Shen, Ruixiang Xia, Y. F. Wang, Yu Hu, Ruibin Huang, Guangxun Gao, Jianda Hu, Ting Niu, Xin Zhou, Ming Hou, Liping Wang, Li Liu, Cheng Peng, Yan Li, Xiaofan Liu, Hu Zhou, Ying Feng, Renchi Yang, Zhongxing Jiang, Yuansong Bai, Yazhou Yao, Depei Wu
article en

Abstract

Abstract Introduction: Hetrombopag has been approved for chronic primary immune thrombocytopenia (ITP) (duration >12 months). However, its efficacy and safety in persistent ITP (duration 3–12 months) remain unexplored. Based on existing clinical data, this post-hoc analysis aimed to evaluate the efficacy and safety of hetrombopag in persistent ITP patients and compare outcomes with chronic ITP and overall ITP patients. Methods: Data were derived from a registration phase Ⅲ trial (NCT03222843) which included a 10-week randomized, double-blind, placebo-controlled treatment period (initial dose: hetrombopag 2.5 mg as HETROM-2.5 group, hetrombopag 5.0 mg as HETROM-5.0 group, or placebo once daily), a 14-week open-label treatment period, a dose-tapering to withdrawal period and a 24-week single-arm extension period. The primary endpoint was the proportion of responders (a platelet count of ≥ 50×10⁹/L) after 8 weeks of treatments. Results: Of 395 who completed the double-blinded treatment, 80 patients had persistent ITP. The proportion of persistent ITP patients who achieved primary endpoint was 68.8% in the HETROM-2.5 group and 74.3% in the HETROM-5.0 group, significantly higher than 7.7% in the placebo group. Hetrombopag also demonstrated other therapeutic benefits over placebo including reduced bleeding risk and rescue therapy need. Similar efficacy results were observed both in the chronic ITP and the overall ITP population. Furthermore, hetrombopag manifested favorable safety profile in persistent ITP patients, and safety outcomes were comparable to those in chronic and overall ITP populations. Conclusions: Hetrombopag indicated favorable safe and efficacy performance in patients with persistent ITP, aligning with results observed in chronic ITP and overall ITP patients.

BloodVol. 146(Supplement 1)
Hebei Medical University (CN), Fujian Medical University (CN), Union Hospital (HK), Nanchang University (CN), Guangxi Medical University (CN), Shanxi Medical University (CN), Anhui Medical University (CN), Changzhi Medical College (CN), Chinese Academy of Medical Sciences & Peking Union Medical College (CN), Sichuan University (CN), Kunming Medical University (CN), Soochow University (CN), Xian Central Hospital (CN), West China Hospital of Sichuan University (CN), Second Affiliated Hospital of Guangzhou Medical University (CN), Second Hospital of Hebei Medical University (CN), First Affiliated Hospital of GuangXi Medical University (CN), Second Hospital of Shanxi Medical University (CN), First Affiliated Hospital of Anhui Medical University (CN), Henan Cancer Hospital (CN), Jiangsu Hengrui Medicine (China) (CN), Institute of Hematology & Blood Diseases Hospital (CN), Baoji City Central Hospital (CN), First Affiliated Hospital of Soochow University (CN), First Affiliated Hospital of Zhengzhou University (CN), Wuxi People's Hospital (CN), Xijing Hospital (CN), Qilu Hospital of Shandong University (CN), Union Hospital (CN), First Affiliated Hospital of Nanchang University (CN), First Affiliated Hospital Zhejiang University (CN), Huazhong University of Science and Technology (CN), Guangzhou Medical University (CN)
Openalex Percentile: Top 44%
Platelet Disorders and Treatments
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