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与先天性纤维蛋白原异常相关的基因突变:全球分布及临床结局

Genetic mutations associated with congenital fibrinogen disorders: global distribution and clinical outcomes.

作者信息

Nóbrega Thaís, Villaça Paula, Okazaki Erica, Rothschild Cynthia, Stefanello Bianca, Rocha Tânia, Rocha Vanderson, A Orsi Fernanda

机构信息

Hospital University of São Paulo, São Paulo, Brazil.

Department of Pathology, School of Medical Sciences, University of Campinas, Campinas, Brazil.

出版信息

J Thromb Thrombolysis. 2025 Aug 29. doi: 10.1007/s11239-025-03134-x.

Abstract

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.

摘要

先天性纤维蛋白原异常(CFD)的表现具有异质性,从无症状到严重出血或血栓形成,与FGA、FGB或FGG基因的基因突变有关。因此,诊断具有挑战性,尤其是在证据稀少的低收入和中等收入国家。本综述的目的是描述与CFD相关的基因突变在世界不同地区的分布及其相应的表型。来自MEDLINE和法国止血与血栓形成研究小组数据库的数据根据联合国区域分类进行了定性整理。从MEDLINE和GFHT纤维蛋白原数据库中总共选取了132项关于CFD的研究,包括1000多个突变描述和约340个独特突变。FGA突变最常与低纤维蛋白原血症或无纤维蛋白原血症相关,而FGB突变与低纤维蛋白原血症或无纤维蛋白原血症相关,FGG突变与低纤维蛋白原血症或异常纤维蛋白原血症相关。在各个国家中,无纤维蛋白原血症和低纤维蛋白原血症最常见的突变分别是FGA的内含子变异序列、FGB的p.Arg47stop以及FGG第8外显子的突变。异常纤维蛋白原血症与FGA第2外显子的突变有关,通常导致无症状个体,也与FGG第8外显子的突变有关,后者与血栓形成有关。与CFD及其相关表型相关的大多数突变已在西欧、北美和东亚报道。拉丁美洲、东南亚和非洲的证据仍然有限,巴西仅有一项评估CFD突变的研究。低收入和中等收入国家关于CFD表型和相关基因突变的数据对于确保这些罕见疾病管理的公平性至关重要。

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