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A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy.

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资源类型：
Pubmed体系：
作者：
Guangxin Peng[1] ; Guangsheng He[2] ; Hong Chang[3] ; Sujun Gao[4] ; Xinjian Liu[5] ; Tong Chen[6] ; Pei Li[6] ; Bing Han[7] ; Miao Miao[8] ; Zheng Ge[9] ; Xiaoyan Ge[10] ; Fei Li[11] ; Yingmei Li[12] ; Shunqing Wang[13] ; Yi Wang[14] ; Yaqi Shen[15] ; Tao Zhang[15] ; Jianjun Zou[15] ; Fengkui Zhang[16,*1] ;
机构： [1]Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China [2]Department of Hematology, Jiangsu Province Hospital, Nanjing, China [3]Department of Hematology, Institute of Hematology, West China Hospital, Sichuan University, Chengdu, China [4]Department of Hematology, The First Hospital of Jilin University, Changchun, China [5]Department of Hematology, Henan Cancer Hospital, Zhengzhou, China [6]Department of Hematology, Huashan Hospital Affiliated to Fudan University, Shanghai, China [7]Department of Hematology, Peking Union Medical College Hospital, Beijing, China [8]Department of Hematology, The First Affiliated Hospital of Suzhou University, Suzhou, China [9]Department of Hematology, Zhongda Hospital Affiliated to Southeast University, Nanjing, China [10]Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan, China [11]Department of Hematology, The First Affiliated Hospital of Nanchang University, Nanchang, China [12]Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China [13]Department of Hematology, Guangzhou First People's Hospital, Guangzhou, China [14]Department of Hematology, Shaanxi Provincial People's Hospital, Xi'an, China [15]Jiangsu Hengrui Pharmaceuticals Co., Ltd,Shanghai, China [16]Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, No.288 Nanjing Road, Heping District, 300020 Tianjin, China
出处：
DOI： 10.1177/20406207221085197
ISSN：

关键词： clinical trial hematologic response hetrombopag severe aplastic anemia thrombopoietin receptor

摘要：
In this single-arm phase II study (NCT03557099), we evaluated the efficacy and safety of hetrombopag, a small molecule thrombopoietin (TPO) receptor agonist, in patients with severe aplastic anemia (SAA) who were refractory to standard first-line immunosuppressive therapy (IST).SAA patients who were refractory to standard first-line IST were given hetrombopag orally at an initial dose of 7.5 mg once daily to a maximum of 15 mg once daily, for a total of 52 weeks. The primary endpoint was proportion of patients achieving hematologic responses in ⩾1 lineage at week 18.A total of 55 eligible patients were enrolled and received hetrombopag treatment. This study met its primary endpoint, with 23 [41.8%, 95% confidence interval (CI) = 28.7-55.9] patients achieving hematologic response in ⩾1 lineage at week 18 after initiation of hetrombopag treatment. Twenty-four (43.6%, 95% CI = 30.3-57.7) and 27 (49.1%, 95% CI = 35.4-62.9) of the 55 patients responded in ⩾1 lineage at weeks 24 and 52, respectively. Median time to initial hematologic response was 7.9 weeks (range = 2.0-32.1). The responses were durable, with a 12-month relapse-free survival rate of 82.2% (95% CI = 62.2-92.2). Adverse events occurred in 54 (98.2%) patients, and 28 (50.9%) patients had treatment-related adverse events. Seventeen (30.9%) patients had adverse events of grade ⩾3. Serious adverse events occurred in 15 (27.3%) patients and three deaths (5.5%) were reported.Hetrombopag showed encouraging efficacy with durable hematologic responses in patients with SAA who were refractory to IST. Hetrombopag was well tolerant and safe for long-term use.NCT03557099.© The Author(s), 2022.

基金：
The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This study was sponsored by Jiangsu Hengrui Pharmaceuticals Co., Ltd. The authors and sponsor collaborated in data collection, analysis, and interpretation,and guaranteed the authenticity and integrity of the data. All authors had full access to the data in this study and had final responsibility for the decision to submit for publication.
语种：
外文
PubmedID：
中科院(CAS)分区：
出版当年[2022]版：
大类 | 2 区 医学
小类 | 2 区 血液学
最新[2023]版：
大类 | 3 区 医学
小类 | 3 区 血液学
第一作者：
第一作者机构： [1]Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
通讯作者：
通讯机构： [16]Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, No.288 Nanjing Road, Heping District, 300020 Tianjin, China [*1]Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, No. 288 Nanjing Road, Heping District, 300020 Tianjin, China.
推荐引用方式(GB/T 7714)：
Guangxin Peng,Guangsheng He,Hong Chang,et al.A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy.[J].Therapeutic advances in hematology.2022,13:20406207221085197.doi:10.1177/20406207221085197.
APA：
Guangxin Peng,Guangsheng He,Hong Chang,Sujun Gao,Xinjian Liu...&Fengkui Zhang.(2022).A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy..Therapeutic advances in hematology,13,
MLA：
Guangxin Peng,et al."A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy.".Therapeutic advances in hematology 13.(2022):20406207221085197

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