Department of Medicine, University of Washington, Seattle, WA, USA.
Blood. 2013 Jul 25;122(4):590-7. doi: 10.1182/blood-2013-02-485094. Epub 2013 May 20.
Several rare European von Willebrand disease missense variants of VWF (including p.Arg2185Gln and p.His817Gln) were recently reported to be common in apparently healthy African Americans (AAs). Using data from the NHLBI Exome Sequencing Project, we assessed the association of these and other VWF coding variants with von Willebrand factor (VWF) and factor VIII (FVIII) levels in 4468 AAs. Of 30 nonsynonymous VWF variants, 6 were significantly and independently associated (P < .001) with levels of VWF and/or FVIII. Each additional copy of the common VWF variants encoding p.Thr789Ala or p.Asp1472His was associated with 6 to 8 IU/dL higher VWF levels. The VWF variant encoding p.Arg2185Gln was associated with 7 to 13 IU/dL lower VWF and FVIII levels. The type 2N-related VWF variant encoding p.His817Gln was associated with 17 IU/dL lower FVIII level but normal VWF level. A novel, rare missense VWF variant that predicts disruption of an O-glycosylation site (p.Ser1486Leu) and a rare variant encoding p.Arg2287Trp were each associated with 30 to 40 IU/dL lower VWF level (P < .001). In summary, several common and rare VWF missense variants contribute to phenotypic differences in VWF and FVIII among AAs.
几种罕见的欧洲血管性血友病因子(VWF)错义变异体(包括 p.Arg2185Gln 和 p.His817Gln)最近在表现健康的非裔美国人(AA)中被报道为常见。利用 NHLBI 外显子组测序计划的数据,我们评估了这些和其他 VWF 编码变异与 4468 名 AA 中 VWF 和/或 FVIII 水平的关联。在 30 种非同义 VWF 变异体中,有 6 种与 VWF 和/或 FVIII 水平呈显著且独立相关(P <.001)。每个常见 VWF 变异体编码 p.Thr789Ala 或 p.Asp1472His 的额外拷贝与 VWF 水平升高 6 至 8 IU/dL 相关。编码 p.Arg2185Gln 的 VWF 变异体与 VWF 和 FVIII 水平降低 7 至 13 IU/dL 相关。编码 p.His817Gln 的与 2N 型相关的 VWF 变异体与 FVIII 水平降低 17 IU/dL 相关,但 VWF 水平正常。一种新型的罕见错义 VWF 变异体,预测破坏一个 O-糖基化位点(p.Ser1486Leu),以及一种罕见的变异体编码 p.Arg2287Trp,与 VWF 水平降低 30 至 40 IU/dL 相关(P <.001)。总之,几种常见和罕见的 VWF 错义变异体导致了 AA 中 VWF 和 FVIII 表型差异。