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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.

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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