先天性纤维蛋白原缺乏:并不罕见。

IF 2.7 4区 医学 Q2 HEMATOLOGY
Alexander Couzens, Marguerite Neerman-Arbez
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引用次数: 0

摘要

先天性纤维蛋白原缺乏(CFDs),传统上被认为是罕见的单基因疾病,现在被认为比以前认为的更为普遍和遗传复杂。事实上,CFD患者所表现的症状,如出血和血栓形成,可能是由几个基因的变异引起的,而不是仅仅由三种纤维蛋白原基因FGB、FGA和FGG中的一种基因的变异所驱动。这篇综述强调了在了解CFD的遗传原因及其可变性方面的最新进展,这得益于下一代测序数据的日益使用和可用性。使用gnomAD v4.1.0。基于FGB、FGA和FGG中预测的有害变异的频率,我们提供了最新的全球差价合约患病率估计。隐性遗传纤维蛋白原缺陷(纯合子基因型)可能存在于每百万人中约29人,而显性遗传缺陷(杂合子基因型)可能存在于每百万人中多达15,000人。这些增加的估计可归因于在新版本的gnomAD中包含了更广泛、更多样化的遗传数据集,从而捕获了更大范围的罕见变体和纯合子病例。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Congenital Fibrinogen Deficiencies: Not So Rare.

Congenital fibrinogen deficiencies (CFDs), traditionally considered rare monogenic disorders, are now recognized as more prevalent and genetically complex than previously thought. Indeed, the symptoms manifested in CFD patients, such as bleeding and thrombosis, are likely to result from variation in several genes rather than solely driven by variants in one of the three fibrinogen genes, FGB, FGA, and FGG. This review highlights recent advances in understanding the genetic causes of CFD and their variability, facilitated by the growing use and availability of next-generation sequencing data. Using gnomAD v4.1.0. data, which includes more than 800,000 individuals, we provide updated global prevalence estimates for CFDs based on frequencies of predicted deleterious variants in FGB, FGA, and FGG. Recessively inherited fibrinogen deficiencies (homozygous genotypes) could be present in around 29 individuals per million, while dominantly inherited deficiencies (heterozygous genotypes) may be present in up to 15,000 per million. These increased estimates can be attributed to the inclusion of broader, more diverse genetic datasets in the new version of gnomAD, thus capturing a greater range of rare variants and homozygous cases.

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来源期刊
Hamostaseologie
Hamostaseologie HEMATOLOGY-
CiteScore
5.50
自引率
6.20%
发文量
62
审稿时长
6-12 weeks
期刊介绍: Hämostaseologie is an interdisciplinary specialist journal on the complex topics of haemorrhages and thromboembolism and is aimed not only at haematologists, but also at a wide range of specialists from clinic and practice. The readership consequently includes both specialists for internal medicine as well as for surgical diseases.
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