依库珠单抗预防血友病 A 伴抑制物患者出血：系统评价和荟萃分析。
Emicizumab prophylaxis in people with hemophilia A and inhibitors: a systematic review and meta-analysis.
机构信息
Medical student, Faculty of Medicine, Universidade Federal de Goiás (UFG), Goiânia (GO), Brazil.
MD, PhD. Physician, Postdoctoral associate, Faculty of Medicine, Universidade Federal de Minas Gerais (UFMG), Belo Horizonte (MG), Brazil.
出版信息
Sao Paulo Med J. 2024 May 10;142(5):e2023102. doi: 10.1590/1516-3180.2023.0102.R1.20022024. eCollection 2024.
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.
背景
直到最近，接受凝血因子八抑制物（inhibitor）治疗的甲型血友病患者（PwHAi）的治疗方案仍基于旁路制剂（BPA）的使用。然而，emicizumab 作为预防性治疗药物的出现带来了令人鼓舞的结果。
目的
我们旨在比较接受 BPA 治疗和接受 emicizumab 预防性治疗的 PwHAi 的出血终点。
设计和设置
在巴西戈亚斯联邦大学（Universidade Federal de Goiás）进行的干预措施的系统评价和荟萃分析。
方法
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。入选的出版物评估了接受 emicizumab 预防性治疗和 BPA 预防性治疗的 PwHAi 的出血终点。根据 Joanna Briggs 研究所的标准评估了偏倚风险。进行荟萃分析以确定治疗性出血的年化出血率（ABR）。
结果
从检索到的 543 条记录中筛选出 5 篇文献（56 例 PwHAi）。总体而言，emicizumab 预防性治疗时的出血终点低于 BPA 预防性治疗时。所有出版物均存在至少一个偏倚风险。荟萃分析唯一的共同参数是治疗性出血的 ABR。在接受 emicizumab 预防性治疗时，治疗性出血的 ABR 低于接受 BPA 预防性治疗时（标准均数差：-1.58；95%置信区间：-2.50，-0.66，P = 0.0008；I2 = 68.4%，P = 0.0031）。
结论
emicizumab 在预防 PwHAi 的出血方面优于 BPA。然而，在评估这些结果时，应考虑到研究人群规模较小和潜在的偏倚风险。
系统评价注册
CRD42021278726，https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=278726。
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