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.
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.