Thaís Nóbrega, Paula Villaça, Erica Okazaki, Cynthia Rothschild, Bianca Stefanello, Tânia Rocha, Vanderson Rocha, Fernanda A Orsi
{"title":"与先天性纤维蛋白原疾病相关的基因突变:全球分布和临床结果","authors":"Thaís Nóbrega, Paula Villaça, Erica Okazaki, Cynthia Rothschild, Bianca Stefanello, Tânia Rocha, Vanderson Rocha, Fernanda A Orsi","doi":"10.1007/s11239-025-03134-x","DOIUrl":null,"url":null,"abstract":"<p><p>Congenital fibrinogen disorders (CFD) are characterized by heterogeneous manifestations, from asymptomatic to severe bleeding or thrombosis, associated with genetic mutations in FGA, FGB, or FGG genes. As a result, diagnosis is challenging, particularly in low- and middle-income countries, where evidence is scarce. The aim of this review is to describe the distribution of CFD-associated genetic mutations across different regions of the world and their corresponding phenotypes. Data from MEDLINE and the French Group for the Study of Hemostasis and Thrombosis databases were qualitatively organized based on the United Nations regional classification. A total of 132 studies on CFD were selected from MEDLINE and GFHT fibrinogen database, comprising over 1000 mutations descriptions and approximately 340 unique mutations. FGA mutations are most associated with dys- or afibrinogenemia, while FGB mutations are associated with hypo- or afibrinogenemia and FGG with dys- or hypofibrinogenemia Across countries, the most common mutations in afibrinogenemia and hypofibrinogenemia were intronic variant sequence in FGA, p. Arg47stop in FGB, and mutations in exon 8 of FGG. Dysfibrinogenemia was associated with mutations in exon 2 of FGA, typically resulting in asymptomatic individuals and with mutations in exon 8 of FGG, which are associated with thrombosis. The majority of mutations related to CFD and their associated phenotypes have been reported in Western Europe, North America and East Asia. Evidence from Latin America, Southeast Asia, and Africa remains limited, with Brazil having only one study that evaluated CFD mutations. Data on CFD phenotypes and associated genetic mutations from low and middle income countries are necessary to ensure equity in the management of these rare diseases.t.</p>","PeriodicalId":17546,"journal":{"name":"Journal of Thrombosis and Thrombolysis","volume":" ","pages":""},"PeriodicalIF":2.2000,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Genetic mutations associated with congenital fibrinogen disorders: global distribution and clinical outcomes.\",\"authors\":\"Thaís Nóbrega, Paula Villaça, Erica Okazaki, Cynthia Rothschild, Bianca Stefanello, Tânia Rocha, Vanderson Rocha, Fernanda A Orsi\",\"doi\":\"10.1007/s11239-025-03134-x\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Congenital fibrinogen disorders (CFD) are characterized by heterogeneous manifestations, from asymptomatic to severe bleeding or thrombosis, associated with genetic mutations in FGA, FGB, or FGG genes. As a result, diagnosis is challenging, particularly in low- and middle-income countries, where evidence is scarce. The aim of this review is to describe the distribution of CFD-associated genetic mutations across different regions of the world and their corresponding phenotypes. Data from MEDLINE and the French Group for the Study of Hemostasis and Thrombosis databases were qualitatively organized based on the United Nations regional classification. A total of 132 studies on CFD were selected from MEDLINE and GFHT fibrinogen database, comprising over 1000 mutations descriptions and approximately 340 unique mutations. FGA mutations are most associated with dys- or afibrinogenemia, while FGB mutations are associated with hypo- or afibrinogenemia and FGG with dys- or hypofibrinogenemia Across countries, the most common mutations in afibrinogenemia and hypofibrinogenemia were intronic variant sequence in FGA, p. Arg47stop in FGB, and mutations in exon 8 of FGG. Dysfibrinogenemia was associated with mutations in exon 2 of FGA, typically resulting in asymptomatic individuals and with mutations in exon 8 of FGG, which are associated with thrombosis. The majority of mutations related to CFD and their associated phenotypes have been reported in Western Europe, North America and East Asia. Evidence from Latin America, Southeast Asia, and Africa remains limited, with Brazil having only one study that evaluated CFD mutations. Data on CFD phenotypes and associated genetic mutations from low and middle income countries are necessary to ensure equity in the management of these rare diseases.t.</p>\",\"PeriodicalId\":17546,\"journal\":{\"name\":\"Journal of Thrombosis and Thrombolysis\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.2000,\"publicationDate\":\"2025-08-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Thrombosis and Thrombolysis\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s11239-025-03134-x\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CARDIAC & CARDIOVASCULAR SYSTEMS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Thrombosis and Thrombolysis","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11239-025-03134-x","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
Genetic mutations associated with congenital fibrinogen disorders: global distribution and clinical outcomes.
Congenital fibrinogen disorders (CFD) are characterized by heterogeneous manifestations, from asymptomatic to severe bleeding or thrombosis, associated with genetic mutations in FGA, FGB, or FGG genes. As a result, diagnosis is challenging, particularly in low- and middle-income countries, where evidence is scarce. The aim of this review is to describe the distribution of CFD-associated genetic mutations across different regions of the world and their corresponding phenotypes. Data from MEDLINE and the French Group for the Study of Hemostasis and Thrombosis databases were qualitatively organized based on the United Nations regional classification. A total of 132 studies on CFD were selected from MEDLINE and GFHT fibrinogen database, comprising over 1000 mutations descriptions and approximately 340 unique mutations. FGA mutations are most associated with dys- or afibrinogenemia, while FGB mutations are associated with hypo- or afibrinogenemia and FGG with dys- or hypofibrinogenemia Across countries, the most common mutations in afibrinogenemia and hypofibrinogenemia were intronic variant sequence in FGA, p. Arg47stop in FGB, and mutations in exon 8 of FGG. Dysfibrinogenemia was associated with mutations in exon 2 of FGA, typically resulting in asymptomatic individuals and with mutations in exon 8 of FGG, which are associated with thrombosis. The majority of mutations related to CFD and their associated phenotypes have been reported in Western Europe, North America and East Asia. Evidence from Latin America, Southeast Asia, and Africa remains limited, with Brazil having only one study that evaluated CFD mutations. Data on CFD phenotypes and associated genetic mutations from low and middle income countries are necessary to ensure equity in the management of these rare diseases.t.
期刊介绍:
The Journal of Thrombosis and Thrombolysis is a long-awaited resource for contemporary cardiologists, hematologists, vascular medicine specialists and clinician-scientists actively involved in treatment decisions and clinical investigation of thrombotic disorders involving the cardiovascular and cerebrovascular systems. The principal focus of the Journal centers on the pathobiology of thrombosis and vascular disorders and the use of anticoagulants, platelet antagonists, cell-based therapies and interventions in scientific investigation, clinical-translational research and patient care.
The Journal will publish original work which emphasizes the interface between fundamental scientific principles and clinical investigation, stimulating an interdisciplinary and scholarly dialogue in thrombosis and vascular science. Published works will also define platforms for translational research, drug development, clinical trials and patient-directed applications. The Journal of Thrombosis and Thrombolysis'' integrated format will expand the reader''s knowledge base and provide important insights for both the investigation and direct clinical application of the most rapidly growing fields in medicine-thrombosis and vascular science.