Evaluating the Safety and Efficacy of Concizumab in Hemophilia A/B Patients: A Systematic Review.

IF 2.3 4区 医学 Q2 HEMATOLOGY
Erum Siddiqui, Maliha Khalid, Muhammad Saad Khan, Kanza Farhan, Muhammad Mohsin Khan, Aminath Waafira
{"title":"Evaluating the Safety and Efficacy of Concizumab in Hemophilia A/B Patients: A Systematic Review.","authors":"Erum Siddiqui, Maliha Khalid, Muhammad Saad Khan, Kanza Farhan, Muhammad Mohsin Khan, Aminath Waafira","doi":"10.1177/10760296251342968","DOIUrl":null,"url":null,"abstract":"<p><p>BackgroundHemophilia A and B are X-linked recessive bleeding disorders caused by deficiencies of coagulation factors VIII and IX, respectively. These conditions lead to spontaneous bleeding, joint damage, inhibitor development, and the burden of frequent intravenous infusions. Concizumab, a monoclonal antibody targeting tissue factor pathway inhibitor (TFPI), is a novel non-factor therapy that enhances thrombin generation. This systematic review evaluates the efficacy and safety of concizumab prophylaxis in patients with hemophilia A and B.MethodsThis systematic review was conducted in accordance with PRISMA guidelines. Randomized controlled trials (RCTs) assessing the use of concizumab in hemophilia A or B were identified through a comprehensive search of electronic databases. Outcomes of interest included annualized bleeding rate (ABR), thrombin generation, bleeding episodes, immunogenicity, and adverse events. The Cochrane Risk of Bias Tool 2.0 was used for quality assessment.ResultsFive studies were included. Concizumab prophylaxis was associated with a notable reduction in ABR, with reported decreases from 9.4 to 1.3 episodes/year and from 19.6 to 2.9 episodes/year in hemophilia A, and from 14.9 to 1.6 episodes/year in hemophilia B. Thrombin generation increased in a dose-dependent manner and stabilized by week 24. Across all studies, bleeding episodes were significantly reduced. Adverse events were primarily mild to moderate. No thromboembolic events were reported.ConclusionConcizumab appears to be an effective and safe prophylactic treatment for patients with hemophilia A and B, demonstrating consistent reductions in bleeding rates and enhanced thrombin generation. Further long-term studies are warranted to establish its sustained safety and efficacy.</p>","PeriodicalId":10335,"journal":{"name":"Clinical and Applied Thrombosis/Hemostasis","volume":"31 ","pages":"10760296251342968"},"PeriodicalIF":2.3000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12078972/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Clinical and Applied Thrombosis/Hemostasis","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/10760296251342968","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/5/14 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

BackgroundHemophilia A and B are X-linked recessive bleeding disorders caused by deficiencies of coagulation factors VIII and IX, respectively. These conditions lead to spontaneous bleeding, joint damage, inhibitor development, and the burden of frequent intravenous infusions. Concizumab, a monoclonal antibody targeting tissue factor pathway inhibitor (TFPI), is a novel non-factor therapy that enhances thrombin generation. This systematic review evaluates the efficacy and safety of concizumab prophylaxis in patients with hemophilia A and B.MethodsThis systematic review was conducted in accordance with PRISMA guidelines. Randomized controlled trials (RCTs) assessing the use of concizumab in hemophilia A or B were identified through a comprehensive search of electronic databases. Outcomes of interest included annualized bleeding rate (ABR), thrombin generation, bleeding episodes, immunogenicity, and adverse events. The Cochrane Risk of Bias Tool 2.0 was used for quality assessment.ResultsFive studies were included. Concizumab prophylaxis was associated with a notable reduction in ABR, with reported decreases from 9.4 to 1.3 episodes/year and from 19.6 to 2.9 episodes/year in hemophilia A, and from 14.9 to 1.6 episodes/year in hemophilia B. Thrombin generation increased in a dose-dependent manner and stabilized by week 24. Across all studies, bleeding episodes were significantly reduced. Adverse events were primarily mild to moderate. No thromboembolic events were reported.ConclusionConcizumab appears to be an effective and safe prophylactic treatment for patients with hemophilia A and B, demonstrating consistent reductions in bleeding rates and enhanced thrombin generation. Further long-term studies are warranted to establish its sustained safety and efficacy.

评价Concizumab在血友病A/B患者中的安全性和有效性:一项系统综述。
血友病A和B分别是由凝血因子VIII和IX缺乏引起的x连锁隐性出血性疾病。这些情况会导致自发性出血、关节损伤、抑制剂的产生和频繁静脉输液的负担。Concizumab是一种靶向组织因子途径抑制剂(TFPI)的单克隆抗体,是一种增强凝血酶生成的新型非因子疗法。本系统评价评估了concizumab预防A和b血友病患者的有效性和安全性。方法本系统评价按照PRISMA指南进行。通过对电子数据库的全面检索,确定了评估康珠单抗在血友病A或B中使用的随机对照试验(rct)。研究结果包括年化出血率(ABR)、凝血酶生成、出血发作、免疫原性和不良事件。采用Cochrane风险偏倚工具2.0进行质量评价。结果共纳入5项研究。康珠单抗预防与ABR的显著降低相关,据报道血友病a从9.4次减少到1.3次/年,血友病a从19.6次减少到2.9次/年,血友病b从14.9次减少到1.6次/年,凝血酶生成以剂量依赖性方式增加并在第24周稳定下来。在所有研究中,出血发作明显减少。不良事件主要为轻度至中度。无血栓栓塞事件报道。结论conzumab似乎是一种有效和安全的预防性治疗血友病A和B患者,显示出血率持续降低和凝血酶生成增强。需要进一步的长期研究来确定其持续的安全性和有效性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
CiteScore
4.40
自引率
3.40%
发文量
150
审稿时长
2 months
期刊介绍: CATH is a peer-reviewed bi-monthly journal that addresses the practical clinical and laboratory issues involved in managing bleeding and clotting disorders, especially those related to thrombosis, hemostasis, and vascular disorders. CATH covers clinical trials, studies on etiology, pathophysiology, diagnosis and treatment of thrombohemorrhagic disorders.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信