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Current Treatment Patterns of Aplastic Anemia in China: A Prospective Cohort Registry Study

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单位: [1]Chinese Acad Med Sci, Inst Hematol, State Key Lab Expt Hematol, Ctr Pediat Blood Dis, Tianjin, Peoples R China [2]Chinese Acad Med Sci, Blood Dis Hosp, 288 Nanjing Rd, Tianjin 300020, Peoples R China [3]Peking Union Med Coll, 288 Nanjing Rd, Tianjin 300020, Peoples R China [4]Soochow Univ, Affiliated Childrens Hosp, Dept Hematol & Oncol, Suzhou, Peoples R China [5]Guangzhou Med Univ, Guangzhou Peoples Hosp 1, Dept Hematol, Guangzhou, Guangdong, Peoples R China [6]Shanghai Jiao Tong Univ, Sch Med, Dept Hematol & Oncol, Shanghai Childrens Med Ctr, Shanghai, Peoples R China [7]Chinese Acad Med Sci, Peking Union Med Coll Hosp, Dept Hematol, Beijing, Peoples R China [8]Huazhong Univ Sci & Technol, Tongji Med Coll, Tongji Hosp, Dept Hematol, Wuhan, Hubei, Peoples R China [9]Zhejiang Prov Hosp Tradit Chinese Med, Dept Hematol, Hangzhou, Zhejiang, Peoples R China [10]Soochow Univ, Affiliated Hosp 1, Dept Hematol, Suzhou, Peoples R China [11]Jilin Univ, Hosp 1, Dept Hematol, Changchun, Jilin, Peoples R China [12]Huazhong Univ Sci & Technol, Union Hosp, Dept Hematol, Tongji Med Coll, Wuhan, Hubei, Peoples R China [13]Sichuan Univ, West China Hosp, Dept Hematol, Chengdu, Sichuan, Peoples R China [14]Chinese Acad Chinese Med Sci, Xiyuan Hosp, Dept Hematol, Beijing, Peoples R China [15]Peking Univ, Peoples Hosp, Inst Hematol, Beijing Key Lab Hematopoiet Stem Cell Transplanta, Beijing, Peoples R China [16]Third Mil Med Univ, Xinqiao Hosp, Dept Hematol, Chongqing, Peoples R China [17]Jinan Mil Gen Hosp, Dept Hematol, Jinan, Shandong, Peoples R China [18]Fujian Med Univ, Fujian Prov Key Lab Hematol, Dept Hematol, Fujian Inst Hematol,Union Hosp, Fuzhou, Fujian, Peoples R China [19]Sun Yat Sen Univ, Sun Yat Sen Mem Hosp, Dept Pediat Hematol, Guangzhou, Guangdong, Peoples R China [20]Fourth Mil Med Univ, Xijing Hosp, Dept Hematol, Xian, Shaanxi, Peoples R China [21]Guangzhou Women & Childrens Med Ctr, Dept Pediat Hematol, Guangzhou, Guangdong, Peoples R China [22]Fourth Mil Med Univ, Tangdu Hosp, Dept Hematol, Xian, Shaanxi, Peoples R China [23]Zhejiang Univ, Affiliated Hosp 1, Sch Med, Dept Hematol, Hangzhou, Zhejiang, Peoples R China [24]Chinese Acad Med Sci, Inst Hematol, Dept Anemia Therapeut Ctr, 288 Nanjing Rd, Tianjin 300020, Peoples R China
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关键词: Aplastic anemia China Treatment Registry

摘要:
Aplastic anemia (AA) is a hematologic disease characterized by pancytopenia and hypocellular bone marrow, potentially leading to chronic anemia, hemorrhage, and infection. The China Aplastic Anemia Committee and British Committee for Standards in Haematology guidelines recommend hematopoietic stem-cell transplantation (HSCT) or immunosuppressive therapy (IST) comprising antithymocyte globulin (ATG) with cyclosporine (CsA) as initial treatment for AA patients. With limited epidemiological data on the clinical management of AA in Asia, a prospective cohort registry study involving 22 AA treatment centers in China was conducted to describe the disease characteristics of newly diagnosed AA patients and investigate real-world treatment patterns and patient outcomes. Of 340 AA patients, 72.9, 12.6, and 3.5% were receiving IST, traditional Chinese medicine, and HSCT, respectively, at baseline; only 22.2% of IST-treated patients received guideline-recommended ATG with CsA initially. Almost all patients received supportive care (95.6%) as blood transfusion (97.8%), antibiotics (63.7%), and/or hematopoietic growth factors (58.2%). Overall, 64.8% achieved a partial or complete response, and 0.9% experienced relapse. No new safety concerns were identified; serious adverse events were largely unrelated to the treatment regimen. These results demonstrate the need to identify and minimize treatment barriers to standardize and align AA management in China with treatment guideline recommendations and further improve patient outcomes.

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出版当年[2018]版:
大类 | 4 区 医学
小类 | 4 区 血液学
最新[2025]版:
大类 | 4 区 医学
小类 | 4 区 血液学
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出版当年[2017]版:
Q4 HEMATOLOGY
最新[2023]版:
Q3 HEMATOLOGY

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第一作者单位: [1]Chinese Acad Med Sci, Inst Hematol, State Key Lab Expt Hematol, Ctr Pediat Blood Dis, Tianjin, Peoples R China [2]Chinese Acad Med Sci, Blood Dis Hosp, 288 Nanjing Rd, Tianjin 300020, Peoples R China [3]Peking Union Med Coll, 288 Nanjing Rd, Tianjin 300020, Peoples R China
通讯作者:
通讯机构: [2]Chinese Acad Med Sci, Blood Dis Hosp, 288 Nanjing Rd, Tianjin 300020, Peoples R China [3]Peking Union Med Coll, 288 Nanjing Rd, Tianjin 300020, Peoples R China [24]Chinese Acad Med Sci, Inst Hematol, Dept Anemia Therapeut Ctr, 288 Nanjing Rd, Tianjin 300020, Peoples R China
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