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艾曲波帕治疗再生障碍性贫血患者的安全性和有效性:系统评价和随机对照试验的荟萃分析。

Safety and efficacy of eltrombopag in patients with aplastic anemia: a systematic review and meta-analysis of randomized controlled trials.

机构信息

Department of Hematopathology, Second Affiliated Hospital of Air Force Medical University, Xi'an, People's Republic of China.

Department of Cardiology, Second Affiliated Hospital of Air Force Medical University, Xi'an, People's Republic of China.

出版信息

Hematology. 2024 Dec;29(1):2335419. doi: 10.1080/16078454.2024.2335419. Epub 2024 Mar 30.

DOI:10.1080/16078454.2024.2335419
PMID:38553907
Abstract

OBJECTIVE

This article conducts a systematic review of eltrombopag combined with immunosuppressive therapy for the treatment of aplastic anemia (AA), to demonstrate the effectiveness and safety of eltrombopag.

METHODS

PubMed, Cochrane Library, Embase, OVID, Web of Science, China National Knowledge Infrastructure, and Wanfang databases were searched. Studies that met the inclusion criteria were collected, ranging from the establishment of the database to August 2023. Two reviewers performed meta-analyses using the Cochrane systematic review method and RevMan 5.3 software.

RESULTS

This meta-analysis enrolled 5 studies with a total of 542 AA patients, including 274 in the experimental group and 268 in the control group. Meta-analyses were performed for efficacy and adverse reactions. The endpoint of effects included 6-month complete response (CR), 6-month partial response (PR), and 6-month overall response (OR). Eltrombopag combined with immunotherapy showed significant improvements in 6-month CR (OR: 2.20; 95% CI;1.54-3.12;  < 0.0001) and 6-month OR (OR = 3.66, 95% CI 2.39-5.61,  < 0.001)compared to immunosuppressive therapy for AA patients. In terms of safety, eltrombopag combined with immunosuppressive therapy showed significantly increased pigment deposition and abnormal liver function compared to immunosuppressive therapy alone.

CONCLUSION

Compared to immunosuppressive therapy alone, eltrombopag combined with immunosuppressive therapy showed significant improvements in 6-month CR and 6-month OR. However, it also resulted in increased pigment deposition and abnormal liver function in terms of safety.

摘要

目的

本文对血小板生成素受体激动剂(TPO-RA)联合免疫抑制治疗再生障碍性贫血(AA)进行系统评价,以明确 TPO-RA 治疗 AA 的有效性和安全性。

方法

计算机检索 PubMed、Cochrane Library、Embase、OVID、Web of Science、中国知网(CNKI)、万方数据库,搜集 TPO-RA 联合免疫抑制治疗 AA 的随机对照试验(RCT),检索时限均从建库至 2023 年 8 月。由 2 位研究者独立筛选文献、提取资料并评价纳入研究的偏倚风险后,采用 Cochrane 系统评价方法和 RevMan 5.3 软件进行 Meta 分析。

结果

共纳入 5 项 RCT,包含 542 例 AA 患者,试验组 274 例,对照组 268 例。Meta 分析结果显示:①疗效方面,TPO-RA 联合免疫抑制治疗 AA 患者的 6 个月完全缓解(CR)率[OR=2.20,95%CI(1.54,3.12),P<0.0001]和 6 个月总反应率(OR=3.66,95%CI(2.39,5.61),P<0.001)均显著高于免疫抑制治疗。②安全性方面,TPO-RA 联合免疫抑制治疗较免疫抑制治疗更易发生色素沉着[OR=3.54,95%CI(2.06,6.08),P<0.0001]和肝功能异常[OR=3.22,95%CI(1.69,6.15),P=0.0006]。

结论

与免疫抑制治疗相比,TPO-RA 联合免疫抑制治疗可显著提高 AA 患者的 6 个月 CR 率和 6 个月 OR 率,但安全性方面更易发生色素沉着和肝功能异常。

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引用本文的文献

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Adding eltrombopag to intensive immunosuppressive therapy for severe aplastic anaemia may help adult patients achieve outcomes similar to paediatric patients.在重度再生障碍性贫血的强化免疫抑制治疗中添加艾曲泊帕可能有助于成年患者获得与儿科患者相似的治疗效果。
Leukemia. 2025 Jan;39(1):261-264. doi: 10.1038/s41375-024-02450-0. Epub 2024 Nov 21.
2
Whole Exome Sequencing of Adult Indians with Apparently Acquired Aplastic Anaemia: Initial Experience at Tertiary Care Hospital.成年印度人明显获得性再生障碍性贫血的全外显子组测序:三级护理医院的初步经验。
Diseases. 2024 Sep 23;12(9):225. doi: 10.3390/diseases12090225.