Tiago Paiva Prudente, Ricardo Mesquita Camelo, Rafael Alves Guimarães, Maria do Rosário Ferraz Roberti
{"title":"Emicizumab prophylaxis in people with hemophilia A and inhibitors: a systematic review and meta-analysis.","authors":"Tiago Paiva Prudente, Ricardo Mesquita Camelo, Rafael Alves Guimarães, Maria do Rosário Ferraz Roberti","doi":"10.1590/1516-3180.2023.0102.R1.20022024","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>We aimed to compare the bleeding endpoints between PwHAi on BPA and those on emicizumab prophylaxis.</p><p><strong>Design and setting: </strong>Systematic review of interventions and meta-analysis conducted at the Universidade Federal de Goiás, Goiânia, Goiás, Brazil.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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).</p><p><strong>Conclusion: </strong>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.</p><p><strong>Systematic review registration: </strong>CRD42021278726, https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=278726.</p>","PeriodicalId":49574,"journal":{"name":"Sao Paulo Medical Journal","volume":"142 5","pages":"e2023102"},"PeriodicalIF":1.3000,"publicationDate":"2024-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11087007/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Sao Paulo Medical Journal","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1590/1516-3180.2023.0102.R1.20022024","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
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.
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.