Emicizumab prophylaxis in people with hemophilia A and inhibitors: a systematic review and meta-analysis.

IF 1.3 4区 医学 Q2 MEDICINE, GENERAL & INTERNAL
Sao Paulo Medical Journal Pub Date : 2024-05-10 eCollection Date: 2024-01-01 DOI:10.1590/1516-3180.2023.0102.R1.20022024
Tiago Paiva Prudente, Ricardo Mesquita Camelo, Rafael Alves Guimarães, Maria do Rosário Ferraz Roberti
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引用次数: 0

Abstract

Background: Until recently, the treatment of people with hemophilia A and inhibitors (PwHAi) was based on the use of bypassing agents (BPA). However, the advent of emicizumab as prophylaxis has demonstrated promising results.

Objectives: We aimed to compare the bleeding endpoints between PwHAi on BPA and those on emicizumab prophylaxis.

Design and setting: Systematic review of interventions and meta-analysis conducted at the Universidade Federal de Goiás, Goiânia, Goiás, Brazil.

Methods: The CENTRAL, MEDLINE, Scopus, and LILACS databases were searched on February 21, 2023. Two authors conducted the literature search, publication selection, and data extraction. The selected publications evaluated the bleeding endpoints between PwHAi on emicizumab prophylaxis and those on BPA prophylaxis. The risk of bias was evaluated according to the Joanna Briggs Institute criteria. A meta-analysis was performed to determine the annualized bleeding rate (ABR) for treated bleeds.

Results: Five publications (56 PwHAi) were selected from the 543 retrieved records. Overall, bleeding endpoints were lower during emicizumab prophylaxis than during BPA prophylaxis. All the publications had at least one risk of bias. The only common parameter for the meta-analysis was the ABR for treated bleeds. During emicizumab prophylaxis, the ABR for treated bleeds was lower than during BPA prophylaxis (standard mean difference: -1.58; 95% confidence interval -2.50, -0.66, P = 0.0008; I2 = 68.4%, P = 0.0031).

Conclusion: Emicizumab was superior to BPA in bleeding prophylaxis in PwHAi. However, both the small population size and potential risk of bias should be considered when evaluating these results.

Systematic review registration: CRD42021278726, https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=278726.

血友病 A 和抑制剂患者的埃米珠单抗预防治疗:系统综述和荟萃分析。
背景:直到最近,A 型血友病和抑制剂患者(PwHAi)的治疗仍以使用旁路药物(BPA)为基础。然而,作为预防用药的埃米珠单抗的问世取得了可喜的成果:我们旨在比较使用 BPA 和使用埃米珠单抗预防性治疗的 PwHAi 的出血终点:在巴西戈亚斯州戈亚尼亚市的戈亚斯联邦大学对干预措施进行了系统回顾和荟萃分析:于 2023 年 2 月 21 日检索了 CENTRAL、MEDLINE、Scopus 和 LILACS 数据库。两位作者进行了文献检索、出版物选择和数据提取。所选出版物评估了接受埃米珠单抗预防治疗和接受 BPA 预防治疗的 PwHAi 之间的出血终点。根据乔安娜-布里格斯研究所(Joanna Briggs Institute)的标准对偏倚风险进行了评估。进行了一项荟萃分析,以确定治疗出血的年化出血率(ABR):从检索到的 543 条记录中筛选出 5 篇文献(56 名艾滋病患者)。总体而言,埃米珠单抗预防治疗期间的出血终点低于双酚A预防治疗期间。所有出版物至少存在一个偏倚风险。荟萃分析的唯一共同参数是治疗出血的 ABR。在埃米珠单抗预防治疗期间,治疗出血的 ABR 低于 BPA 预防治疗期间(标准平均差:-1.58;95% 置信区间:-1.58):-1.58;95%置信区间-2.50,-0.66,P = 0.0008;I2 = 68.4%,P = 0.0031):埃米珠单抗在预防HAI患者出血方面优于BPA。然而,在评估这些结果时应考虑到小规模人群和潜在的偏倚风险:CRD42021278726,https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=278726。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Sao Paulo Medical Journal
Sao Paulo Medical Journal 医学-医学:内科
CiteScore
2.20
自引率
7.10%
发文量
210
审稿时长
6-12 weeks
期刊介绍: Published bimonthly by the Associação Paulista de Medicina, the journal accepts articles in the fields of clinical health science (internal medicine, gynecology and obstetrics, mental health, surgery, pediatrics and public health). Articles will be accepted in the form of original articles (clinical trials, cohort, case-control, prevalence, incidence, accuracy and cost-effectiveness studies and systematic reviews with or without meta-analysis), narrative reviews of the literature, case reports, short communications and letters to the editor. Papers with a commercial objective will not be accepted.
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