VWF

Chr 12ADAR

von Willebrand factor

Also known as: F8VWF, VWD

This gene encodes von Willebrand factor, a glycoprotein that promotes platelet adhesion at sites of vascular injury and serves as a chaperone for coagulation factor VIII. Mutations cause von Willebrand disease, the most common inherited bleeding disorder, with subtypes including type 1 (mild bleeding), type 2 variants (2A, 2B, 2M, 2N with specific functional defects), and type 3 (severe bleeding with absence of von Willebrand factor). The gene shows high tolerance to loss-of-function variants and inheritance can be autosomal dominant or autosomal recessive depending on the subtype.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

von Willebrand disease, type 1MIM #193400
AD
von Willebrand disease, type 3MIM #277480
AR
von Willebrand disease, types 2A, 2B, 2M, and 2NMIM #613554
ADAR
8
Active trials
627
Pubs (1 yr)
88
P/LP submissions
18%
P/LP missense
0.59
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryVWF
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Gene-Disease Validity (ClinGen)
hereditary von Willebrand disease · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
83 unique Pathogenic / Likely Pathogenic· 356 VUS of 600 total submissions
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Clinical Trials
8 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — VWF
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.59LOEUF
pLI 0.000
Z-score 5.54
OE 0.48 (0.390.59)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
0.99Z-score
OE missense 0.93 (0.890.97)
1491 obs / 1602.5 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.48 (0.390.59)
00.351.4
Missense OE0.93 (0.890.97)
00.61.4
Synonymous OE1.09
01.21.6
LoF obs/exp: 64 / 133.0Missense obs/exp: 1491 / 1602.5Syn Z: -1.88
DN
0.6744th %ile
GOF
0.6931th %ile
LOF
0.2677th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function, dominant-negative and loss-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to gain-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median · 1 literature citation
DNprediction above median · 1 literature citation
LOF1 literature citation · 51% of P/LP variants are LoF

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNVon Willebrand disease (VWD) is the most common inherited bleeding disorder and is mainly caused by dominant-negative mutations in the multimeric protein von Willebrand factor (VWF).PMID:32803740
GOFMoreover, force increases the effects of gain-of-function mutations found in von Willebrand disease (VWD) and platelet-type VWD by mechanically stabilizing bond formation and strength.PMID:25810255
LOFTherefore, to examine directly the requirement for VWF in TTP pathogenesis, we generated ADAMTS13-deficient mice on a TTP-susceptible genetic background that were also either haploinsufficient (Vwf+/-) or completely deficient (Vwf-/-) in VWF.PMID:18083848

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

600 submitted variants in ClinVar

Classification Summary

Pathogenic47
Likely Pathogenic36
VUS356
Likely Benign46
Benign55
Conflicting9
47
Pathogenic
36
Likely Pathogenic
356
VUS
46
Likely Benign
55
Benign
9
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
25
2
20
0
47
Likely Pathogenic
17
13
6
0
36
VUS
2
322
19
13
356
Likely Benign
0
4
16
26
46
Benign
0
0
54
1
55
Conflicting
9
Total4434111540549

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

VWF · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Cushing Syndrome

Cushing's Syndrome Before and After Treatment (CORRECT)

RECRUITING
NCT05521529University of AarhusStarted 2023-02-16
no intervention, as this is an observational study
Thrombotic Thrombocytopenic Purpura (TTP)

A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

NOT YET RECRUITING
NCT07353099TakedaStarted 2026-04-30
No Intervention
Congenital Heart Disease

Fontan Associated Liver Disease and the Evaluation of Biomarkers for Disease Severity Assessment

RECRUITING
NCT05213598National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Started 2022-09-07
Cerebral Aneurysm

Effect of Sirolimus on Molecular Alterations in Cerebral Aneurysms

RECRUITING
NCT04141020Phase PHASE2University of MiamiStarted 2021-10-27
Microsurgical clippingEndovascular treatmentSirolimus
Thrombotic Thrombocytopenic PurpuraCongenital Thrombotic Thrombocytopenic PurpuraFamilial Thrombotic Thrombocytopenic Purpura

Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)

RECRUITING
NCT01257269Insel Gruppe AG, University Hospital BernStarted 2006-10
Observation
Healthy Population

Italian Digital Primary Cardiovascular Prevention Study

ACTIVE NOT RECRUITING
NCT05339841Phase NACentro Cardiologico MonzinoStarted 2022-06-10
Mobile health application
Thrombotic Thrombocytopenic Purpura (TTP)

A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program

NOT YET RECRUITING
NCT07429942TakedaStarted 2026-04-07
No Intervention
Non-Alcoholic Fatty Liver DiseaseCardiovascular Diseases

EndoNAFLD: Relationship Between Fatty Liver Disease and Cardiovascular Diseases

RECRUITING
NCT06392828IMDEA FoodStarted 2024-04-15
Clinical Literature
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