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Clinical and laboratory variability in a cohort of patients diagnosed with type 1 VWD in the United States.

Abstract：

:von Willebrand disease (VWD) is the most common inherited bleeding disorder, and type 1 VWD is the most common VWD variant. Despite its frequency, diagnosis of type 1 VWD remains the subject of debate. In order to study the spectrum of type 1 VWD in the United States, the Zimmerman Program enrolled 482 subjects with a previous diagnosis of type 1 VWD without stringent laboratory diagnostic criteria. von Willebrand factor (VWF) laboratory testing and full-length VWF gene sequencing was performed for all index cases and healthy control subjects in a central laboratory. Bleeding phenotype was characterized using the International Society on Thrombosis and Haemostasis bleeding assessment tool. At study entry, 64% of subjects had VWF antigen (VWF:Ag) or VWF ristocetin cofactor activity below the lower limit of normal, whereas 36% had normal VWF levels. VWF sequence variations were most frequent in subjects with VWF:Ag <30 IU/dL (82%), whereas subjects with type 1 VWD and VWF:Ag ≥30 IU/dL had an intermediate frequency of variants (44%). Subjects whose VWF testing was normal at study entry had a similar rate of sequence variations as the healthy controls (14%). All subjects with severe type 1 VWD and VWF:Ag ≤5 IU/dL had an abnormal bleeding score (BS), but otherwise BS did not correlate with VWF:Ag. Subjects with a historical diagnosis of type 1 VWD had similar rates of abnormal BS compared with subjects with low VWF levels at study entry. Type 1 VWD in the United States is highly variable, and bleeding symptoms are frequent in this population.

journal_name

Blood

journal_title

Blood

authors

Flood VH,Christopherson PA,Gill JC,Friedman KD,Haberichter SL,Bellissimo DB,Udani RA,Dasgupta M,Hoffmann RG,Ragni MV,Shapiro AD,Lusher JM,Lentz SR,Abshire TC,Leissinger C,Hoots WK,Manco-Johnson MJ,Gruppo RA,Boggio LN

doi

10.1182/blood-2015-10-673681

subject

Has Abstract

pub_date

2016-05-19 00:00:00

pages

2481-8

issue

20

eissn

0006-4971

issn

1528-0020

pii

blood-2015-10-673681

journal_volume

127

pub_type

杂志文章,多中心研究

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