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Prolonged use of eltrombopag in patients with severe aplastic anemia in the real world

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单位： [1]Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, 300 Guangzhou Road, Nanjing 210029, Jiangsu, China [2]Department of Pediatrics, The Second Hospital of Anhui Medical University, 678 Furong Road, Hefei 230601, Anhui, China [3]Department of Hematology, The Second People’s Hospital of Lianyungang, 161 Xingfu Road, Lianyungang 230601, Jiangsu, China [4]Department of Hematology, Children’s Hospital of Soochow University, 92 Zhongnan Street, Suzhou 215003, Jiangsu, China [5]Department of Hematology, Zhejiang Province Hospital of Traditional Chinese Medicine, The First Affiliated Hospital of Zhejiang Chinese Medical University, 54 Post and Telegraph Road, Hangzhou 310006, Zhejiang, China [6]Department of Hematology, The First Bethune Hospital of Jilin University, 71 Xinmin Street, Changchun 130021, Jilin, China [7]Department of Hematology, Peking University People’s Hospital, Peking University Institute of Hematology, 11 Xizhimen South Street, Beijing 100044, China [8]Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, 1095 Jiefang Avenue, Wuhan 430030, Hubei, China [9]Department of Hematology, The Affiliated Jiangning Hospital of Nanjing Medical University, 169 Hushan Road, Nanjing 211100, Jiangsu, China

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关键词： Severe aplastic anemia Eltrombopag Immunosuppressive therapy Efficacy

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Eltrombopag (EPAG) can improve the efficacy of immunosuppressive therapy (IST) consisting of antithymocyte immunoglobulin (ATG) and cyclosporin in severe aplastic anemia (SAA) patients. This study explored whether patients with SAA could benefit from continuous usage of EPAG beyond 6 months.Seventy-four treatment-naive Chinese patients with SAA were administrated with rabbit ATG-based IST plus EPAG for 6 months. Patients not achieving complete remission (CR) at 6 months were treated with EPAG for another 6 months.At 1, 3, 6 and 12 months after IST, the cumulative response rates were 31%, 61%, 82% and 90%, and the cumulative CR rates were 0, 14%, 27% and 45%, respectively. The cumulative effect curve showed that 93% and 53% of all remission and CR occurred within 6 months, while 98% and 83% of all remission and CR occurred within 12 months. Thirty-seven percent of patients (11 of 30) with partial remission (PR) at 6 months continuously exposed to EPAG improved to CR within 3 (1-5) months of the extended median time. Six patients failing at 6 months continued to use EPAG. Three patients showed improved responses with an extended median time of 6 (1-6) months. The 2-year event-free survival (EFS) was better in those continuing with EPAG (89% vs. 49%, P = 0.006) for patients with PR or non-remission at 6 months.Continuous administration with EPAG could improve the hematologic response and EFS in patients without achieving CR at 6 months.This trial has been registered at the Chinese Clinical Trial Registry (ChiCTR2100045895).© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.

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出版当年[2022]版：

大类 | 3 区医学

小类 | 3 区医学：研究与实验

最新[2025]版：

大类 | 4 区医学

小类 | 4 区医学：研究与实验

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出版当年[2021]版：

Q2 MEDICINE, RESEARCH & EXPERIMENTAL

最新[2023]版：

Q2 MEDICINE, RESEARCH & EXPERIMENTAL

影响因子： 3.2 最新[2023版] 3.4 最新五年平均 5.057 出版当年[2021版] 4.037 出版当年五年平均 3.984 出版前一年[2020版] 4.6 出版后一年[2022版]

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第一作者单位： [1]Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, 300 Guangzhou Road, Nanjing 210029, Jiangsu, China

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Prolonged use of eltrombopag in patients with severe aplastic anemia in the real world

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