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促血小板生成素治疗再生障碍性贫血的疗效和安全性:系统评价和荟萃分析。

Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis.

机构信息

Fujian Provincial Key Laboratory on Hematology, Department of Hematology, Fujian Institute of Hematology, Fujian Medical University Union Hospital, No. 29 Xinquan Street, Gulou District, Fuzhou, 350001, China.

Union Clinical Medical Colleges, Fujian Medical University, No. 1 Xuefubei Street, Fuzhou, 350108, Minhou County, China.

出版信息

Clin Drug Investig. 2019 Feb;39(2):141-156. doi: 10.1007/s40261-018-0725-2.

DOI:10.1007/s40261-018-0725-2
PMID:30406906
Abstract

BACKGROUND AND OBJECTIVES

Eltrombopag seems to be effective in treating patients with aplastic anemia in several clinical trials. This paper aims to perform the first meta-analysis analyzing the efficacy and safety of eltrombopag for aplastic anemia.

METHODS

Literatures were retrieved from PubMed, EMBASE, OVID, Web of Science, Cochrane, Wanfang, http://clinicaltrials.gov and World Health Organization International Clinical Trials Registry Platform search portal from establishment to July 2018. Using Stata statistical software version 12.0, subgroup analyses and sensitivity analyses were conducted.

RESULTS

The overall hematologic response rate is 88% (95% CI 83-94%) for patients treated with eltrombopag plus immunosuppressive therapy, and 47% (95% CI 38-56%) for patients with refractory aplastic anemia using eltrombopag alone. Karyotype abnormality rates include an overall rate of 10% (95% CI 7-14%), a subtotal rate of 8% (95% CI 3-13%) for patients who are treated with eltrombopag plus immunosuppressive therapy without using antithymocyte globulin before, and a subtotal rate of 17% (95% CI 10-24%) for patients with refractory aplastic anemia treated with eltrombopag alone.

CONCLUSIONS

With different treatments and in different conditions eltrombopag showed a distinctive effect for aplastic anemia. However, clone evolution and adverse events were associated with treatment.

摘要

背景和目的

依鲁替尼在几项临床试验中似乎对治疗再生障碍性贫血患者有效。本文旨在进行首次荟萃分析,以分析依鲁替尼治疗再生障碍性贫血的疗效和安全性。

方法

从建立到 2018 年 7 月,从 PubMed、EMBASE、OVID、Web of Science、Cochrane、万方、http://clinicaltrials.gov 和世界卫生组织国际临床试验注册平台搜索门户检索文献。使用 Stata 统计软件版本 12.0 进行亚组分析和敏感性分析。

结果

接受依鲁替尼联合免疫抑制治疗的患者总体血液学反应率为 88%(95%CI 83-94%),单独使用依鲁替尼治疗难治性再生障碍性贫血的患者为 47%(95%CI 38-56%)。染色体异常率包括接受依鲁替尼联合免疫抑制治疗且未在使用抗胸腺细胞球蛋白之前的患者的总体发生率为 10%(95%CI 7-14%),亚组发生率为 8%(95%CI 3-13%),单独使用依鲁替尼治疗难治性再生障碍性贫血的患者的亚组发生率为 17%(95%CI 10-24%)。

结论

在不同的治疗和不同的情况下,依鲁替尼对再生障碍性贫血具有独特的疗效。然而,克隆进化和不良反应与治疗有关。

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本文引用的文献

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Eltrombopag promotes DNA repair in human hematopoietic stem and progenitor cells.依鲁替尼促进人造血干细胞和祖细胞的 DNA 修复。
Exp Hematol. 2019 May;73:1-6.e6. doi: 10.1016/j.exphem.2019.03.002. Epub 2019 Apr 13.
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Clinical Implications of Clonal Hematopoiesis.**标题**:**克隆性造血的临床意义**。
Mayo Clin Proc. 2018 Aug;93(8):1122-1130. doi: 10.1016/j.mayocp.2018.04.002. Epub 2018 Jul 4.
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Hepatic thrombopoietin is required for bone marrow hematopoietic stem cell maintenance.肝脏血小板生成素是维持骨髓造血干细胞所必需的。
Studying the pathogenicity of 26 variants characterized in the first molecular analyses of Egyptian aplastic anemia patients.
研究在埃及再生障碍性贫血患者的首次分子分析中鉴定出的26种变异的致病性。
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Efficacy of Eltrombopag with Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone on Severe Aplastic Anaemia: A Systematic Review and Meta-analysis.Eltrombopag 联合免疫抑制疗法与单纯免疫抑制疗法治疗重型再生障碍性贫血的疗效:系统评价和荟萃分析。
Clin Drug Investig. 2023 May;43(5):315-324. doi: 10.1007/s40261-023-01266-7. Epub 2023 May 11.
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Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan.在日本,免疫抑制治疗初治的再生障碍性贫血患者中,联合用艾曲波帕与兔抗胸腺细胞球蛋白/环孢素 A。
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Eltrombopag is effective in patients with relapse/refractory aplastic anemia-report from a single center in China.依鲁替尼治疗复发/难治性再生障碍性贫血的疗效分析:来自中国单中心的报告。
Ann Hematol. 2020 Dec;99(12):2755-2761. doi: 10.1007/s00277-020-04266-1. Epub 2020 Sep 17.
Science. 2018 Apr 6;360(6384):106-110. doi: 10.1126/science.aap8861.
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Haematologica. 2016 Dec;101(12):1479-1488. doi: 10.3324/haematol.2016.146746. Epub 2016 Aug 11.