机构：[1]Institute of Hematology, Union Hospital, Tongji Medical College, HuazhongUniversity of Science and Technology, Wuhan, Hubei 430022, China华中科技大学同济医学院附属协和医院[2]Thrombosisand Hemostasis Center, State Key Laboratory of Experimental Hematology,National Clinical Research Center for Hematological Disorders, Instituteof Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciencesand Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin300020, China[3]Department of Hematology, Institute of Hematology, WestChina Hospital, Sichuan University, Chengdu 610041, Sichuan, China[4]Departmentof Hematology, Affiliated Cancer Hospital of Zhengzhou University,Zhengzhou, China河南省肿瘤医院[5]Department of Hematopathology, The Second AffiliatedHospital of Guangzhou Medical University, Guangzhou, China[6]The BloodInternal Medicine, The First Affiliated Hospital of Air Force Medical University,Xi’an, China[7]Hematology Department, The First Affiliated Hospital of GuangxiMedical University, Nanning, China[8]Hematology Department, The FirstAffiliated Hospital of Nanchang University, Nanchang, China[9]Departmentof Hematology, The Second Hospital of Shanxi Medical University, Taiyuan,China[10]Fujian Medical University Union Hospital, Fuzhou, China[11]Departmentof Hematology, Qilu Hospital, Shandong University, Jinan, China[12]Hematology Department, Baoji Central Hospital, Baoji, China[13]Departmentof Hematopathology, The Second Affiliated Hospital of Air Force MedicalUniversity, Xi’an, China[14]Department of Hematopathology, Shaanxi ProvincialPeople’s Hospital, Xi’an, China[15]Hematology Department, The First AffiliatedHospital of Soochow University, Suzhou, China[16]Hematology Department,Lanzhou University Second Hospital, Lanzhou, China[17]HematologyDepartment, The First Affiliated Hospital of USTC, Hefei, China[18]Departmentof Hematology, Heping Hospital Affiliated To Changzhi Medical College,Changzhi, China[19]Department of Hematology, Shanghai Municipal Hospitalof Traditional ChineseMedicine, Shanghai, China[20]The Third Xiangya Hospitalof Central South University, Changsha, China[21]Department of Hematology,The First Affiliated Hospital, Zhejiang University College of Medicine, Hangzhou,China浙江大学医学院附属第一医院[22]Department of Hematology, The Second Hospital of HebeiMedical University, Shijiazhuang, China[23]Department of Hematology, FirstAffiliated Hospital of Kunming Medical University, KunMing, China[24]The FirstHospital of Jilin University, Changchun, China[25]Department of Hematology,Peking University People’s Hospital, Beijing, China[26]Hematology Department,Wuxi People’s Hospital, Wuxi, China[27]Hematology Department, The SecondAffiliated Hospital of Nanchang University, Nanchang, China[28]HematologyDepartment, The First Affiliated Hospital of Anhui Medical University, Hefei,China[29]Hematology Department, The First People’s Hospital of Changzhou,Changzhou, China[30]Department of Hematology, The First Affiliated Hospitalof Zhengzhou University, Zhengzhou, China[31]Department of Hematology,The Second Affiliated Hospital of Military Medical University PLA, Chongqing,China[32]Hematology and Oncology, China–Japan Union Hospital of JilinUniversity, Changchun, China[33]Hematology Department, The First Hospitalof China Medical University, Shenyang, China[34]Clinical Research & Development,Jiangsu Hengrui Medicine Co . Ltd, Shanghai, China
Hetrombopag, a novel thrombopoietin receptor agonist, has been found in phase I studies to increase platelet counts and reduce bleeding risks in adults with immune thrombocytopenia (ITP). This phase III study aimed to evaluate the efficacy and safety of hetrombopag in ITP patients.
Patients who had not responded to or had relapsed after previous treatment were treated with an initial dosage of once-daily 2.5 or 5 mg hetrombopag (defined as the HETROM-2.5 or HETROM-5 group) or with matching placebo in a randomized, double-blind, 10-week treatment period. Patients who received placebo and completed 10 weeks of treatment switched to receive eltrombopag, and patients treated with hetrombopag in the double-blind period continued hetrombopag during the following open-label 14-week treatment. The primary endpoint was the proportion of responders (defined as those achieving a platelet count of ≥ 50 × 109/L) after 8 weeks of treatment.
The primary endpoint was achieved by significantly more patients in the HETROM-2.5 (58.9%; odds ratio [OR] 25.97, 95% confidence interval [CI] 9.83-68.63; p < 0.0001) and HETROM-5 (64.3%; OR 32.81, 95% CI 12.39-86.87; p < 0.0001) group than in the Placebo group (5.9%). Hetrombopag was also superior to placebo in achieving a platelet response and in reducing the bleeding risk and use of rescue therapy throughout 8 weeks of treatment. The durable platelet response to hetrombopag was maintained throughout 24 weeks. The most common adverse events were upper respiratory tract infection (42.2%), urinary tract infection (17.1%), immune thrombocytopenic purpura (17.1%) and hematuria (15%) with 24-week hetrombopag treatment.
In ITP patients, hetrombopag is efficacious and well tolerated with a manageable safety profile. Trial registration Clinical trials.gov NCT03222843 , registered July 19, 2017, retrospectively registered.
第一作者机构：[1]Institute of Hematology, Union Hospital, Tongji Medical College, HuazhongUniversity of Science and Technology, Wuhan, Hubei 430022, China
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推荐引用方式(GB/T 7714)：
Heng Mei,Xiaofan Liu,Yan Li,et al.A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia.[J].JOURNAL OF HEMATOLOGY & ONCOLOGY.2021,14(1):doi:10.1186/s13045-021-01047-9.
APA：
Heng Mei,Xiaofan Liu,Yan Li,Hu Zhou,Ying Feng...&Yu Hu.(2021).A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia..JOURNAL OF HEMATOLOGY & ONCOLOGY,14,(1)
MLA：
Heng Mei,et al."A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia.".JOURNAL OF HEMATOLOGY & ONCOLOGY 14..1(2021)
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