Raras
Buscar doenças, sintomas, genes...
Doença de Von Willebrand, tipo 1
ORPHA:166078CID-10 · D68.0CID-11 · 3B12OMIM 193400DOENÇA RARA

A Doença de von Willebrand tipo 1 (DVW tipo 1) é uma forma da Doença de von Willebrand que se manifesta por um problema de sangramento. Isso acontece por causa de uma deficiência parcial (ou seja, uma quantidade menor) do fator de von Willebrand (FVW) no plasma do sangue. Importante ressaltar que, apesar de estar em menor quantidade, esse fator tem estrutura e função normais.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A Doença de von Willebrand tipo 1 (DVW tipo 1) é uma forma da Doença de von Willebrand que se manifesta por um problema de sangramento. Isso acontece por causa de uma deficiência parcial (ou seja, uma quantidade menor) do fator de von Willebrand (FVW) no plasma do sangue. Importante ressaltar que, apesar de estar em menor quantidade, esse fator tem estrutura e função normais.

Publicações científicas
56 artigos
Último publicado: 2025

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-5 / 10 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Europe
Início
All ages
🏥
SUS: Cobertura mínimaScore: 20%
1 medicamentos CEAFCID-10: D68.0
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🩸
Sangue
5 sintomas
❤️
Coração
2 sintomas
🦴
Ossos e articulações
2 sintomas
🫃
Digestivo
1 sintomas

+ 8 sintomas em outras categorias

Características mais comuns

67%prev.
Epistaxe
Frequência: 8/12
42%prev.
Suscetibilidade a hematomas
Frequência: 5/12
33%prev.
Sangramento prolongado após extração dentária
Frequência: 4/12
33%prev.
Sangramento persistente após trauma
Frequência: 4/12
33%prev.
Sangramento prolongado após cirurgia
Frequência: 4/12
Tempo de coagulação do sangue total prolongado
18sintomas
Frequente (5)
Sem dados (13)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 18 características clínicas mais associadas, ordenadas por frequência.

EpistaxeEpistaxis
Frequência: 8/1267%
Suscetibilidade a hematomasBruising susceptibility
Frequência: 5/1242%
Sangramento prolongado após extração dentáriaProlonged bleeding after dental extraction
Frequência: 4/1233%
Sangramento persistente após traumaPersistent bleeding after trauma
Frequência: 4/1233%
Sangramento prolongado após cirurgiaProlonged bleeding after surgery
Frequência: 4/1233%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2025
Total histórico56PubMed
Últimos 10 anos109publicações
Pico201614 papers
Linha do tempo
2025Hoje · 2026🧪 2004Primeiro ensaio clínico📈 2016Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal dominant.

VWFvon Willebrand factorDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sites of vascular injury by forming a molecular bridge between sub-endothelial collagen matrix and platelet-surface receptor complex GPIb-IX-V. Also acts as a chaperone for coagulation factor VIII, delivering it to the site of injury, stabilizing its heterodimeric structure and protecting it from premature clearance from plasma

LOCALIZAÇÃO

SecretedSecreted, extracellular space, extracellular matrix

VIAS BIOLÓGICAS (2)
Platelet Adhesion to exposed collagenEnhanced cleavage of VWF variant by ADAMTS13
MECANISMO DE DOENÇA

von Willebrand disease 1

A common hemorrhagic disorder due to defects in von Willebrand factor protein and resulting in impaired platelet aggregation. Von Willebrand disease type 1 is characterized by partial quantitative deficiency of circulating von Willebrand factor, that is otherwise structurally and functionally normal. Clinical manifestations are mucocutaneous bleeding, such as epistaxis and menorrhagia, and prolonged bleeding after surgery or trauma.

EXPRESSÃO TECIDUAL(Ubíquo)
Tecido adiposo
268.7 TPM
Adipose Visceral Omentum
236.8 TPM
Pulmão
211.2 TPM
Mama
188.8 TPM
Útero
129.2 TPM
OUTRAS DOENÇAS (7)
von Willebrand disease 3von Willebrand disease 2von Willebrand disease 1von Willebrand disease type 2N
HGNC:12726UniProt:P04275

Medicamentos aprovados (FDA)

1 medicamento encontrado nos registros da FDA americana.

💊 ALTUVIIIO (ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC-VWF-XTEN FUSION PROTEIN-EHTL)
Ver no DailyMed/FDA

Variantes genéticas (ClinVar)

770 variantes patogênicas registradas no ClinVar.

🧬 VWF: GRCh38/hg38 12p13.33-11.1(chr12:64621-34650483)x3 ()
🧬 VWF: GRCh38/hg38 12p13.33-q13.12(chr12:82453-49847230)x3 ()
🧬 VWF: NM_000552.5(VWF):c.2673C>A (p.Tyr891Ter) ()
🧬 VWF: NM_000552.5(VWF):c.7096G>T (p.Glu2366Ter) ()
🧬 VWF: NM_000552.5(VWF):c.1110-24_1111del ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 311 variantes classificadas pelo ClinVar.

78
233
Patogênica (25.1%)
VUS (74.9%)
VARIANTES MAIS SIGNIFICATIVAS
VWF: NM_000552.5(VWF):c.7332G>A (p.Trp2444Ter) [Pathogenic]
VWF: NM_000552.5(VWF):c.1546_1556del (p.Tyr516fs) [Likely pathogenic]
VWF: NM_000552.5(VWF):c.5170+11G>C [Conflicting classifications of pathogenicity]
VWF: NM_000552.5(VWF):c.2494C>T (p.Gln832Ter) [Likely pathogenic]
VWF: NM_000552.5(VWF):c.3955del (p.Val1319fs) [Likely pathogenic]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
2Fase 23
·Pré-clínico2
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 5 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Doença de Von Willebrand, tipo 1

🗺️

Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

5 ensaios clínicos encontrados.

Distribuição por fase
Ver todos no ClinicalTrials.gov
🧪 Está conduzindo uma pesquisa?
Divulgue para pacientes e familiares que acompanham esta doença.
Divulgar pesquisa →

Publicações mais relevantes

Timeline de publicações
28 papers (10 anos)
#1

Genetic variants in VWF exon 26 and their implications for type 1 Von Willebrand disease in a Saudi Arabian population.

Open life sciences2025

Von Willebrand disease (VWD) is one of the most common bleeding disorders, stemming from irregularities in the Von Willebrand factor (VWF). Globally, type 1 VWD (VWD1) is the most prevalent form, characterized by decreased levels of VWF in the blood. Genotyping studies have found causative genetic variants in approximately 65% of VWD1 cases, revealing the presence of ethnic-specific VWF variants. This study is limited by its exclusive focus on exon 26, warranting further investigation of other exons and the inclusion of functional analyses. Moreover, these findings emphasize the importance of understanding genetic variability in population-specific contexts, supporting the necessity for future studies on additional exons and potential epigenetic interactions. Between 2018 and 2019, samples and relevant medical data were collected at King Fahad Hospital of the University in Al Khobar. Sanger sequencing of the exonic and flanking intronic regions of exon 26 was performed on 22 index cases clinically diagnosed with VWD1 and their first-degree relatives. Analysis revealed four exonic and 11 intronic variants in VWF exon 26 among the participants. None of the identified variants appeared to explain the VWD-related clinical features in these individuals. However, findings suggest a higher prevalence of specific VWF variants within the Saudi population compared to global databases. Further investigation, including sequencing of additional exons or next-generation sequencing, may help to uncover potential disease-causing variants in this cohort. Establishing a national sequencing project could also enhance our understanding of both common and rare variants in the genetic basis of complex diseases, such as VWD1.

#2

A bispecific nanobody for the treatment of von Willebrand disease type 1.

Blood2025 Nov 20

von Willebrand disease (VWD) type 1 is a bleeding disorder characterized by a quantitative deficiency of functional von Willebrand factor (VWF). We designed a novel bispecific nanobody, named KB-V13A12, that aims to increase endogenous VWF levels by bridging it to albumin. KB-V13A12 comprises 2 single-domain antibodies, 1 targeting VWF and 1 targeting albumin. VWF bound efficiently to the albumin/KB-V13A12 complex (2.0 ± 0.4 nM) in immunosorbent assays, and binding was stable at pH 5.6 and 7.4. VWF ristocetin activity and factor VIII binding remained unaffected in the presence of a 100- to 200-fold molar excess of KB-V13A12/albumin. Humanized VWD type 1 mice were used for in vivo analysis. A single subcutaneous dose of KB-V13A12 (5 mg/kg) was associated with a nanobody half-life of 3.0 ± 0.7 days, and dose-dependently increased VWF in VWD type 1 mice 1.4- to 2.1-fold for up to 14 days. Factor VIII activity was also increased during this period. The VWF propeptide/VWF antigen ratio (a marker for VWF clearance) was significantly reduced in the presence of KB-V13A12, suggesting that delayed clearance contributes to increased VWF levels. Clearance experiments in wild-type mice using recombinant VWF preincubated with KB-V13A12 indeed confirmed a prolonged survival, while this prolongation was absent in FcRn-deficient mice. Finally, treatment with KB-V13A12 resulted in a significantly improved bleeding tendency in VWD type 1 mice when using the saphenous vein puncture model. In conclusion, KB-V13A12 is a bispecific nanobody that efficiently increases functional levels of endogenous VWF, and could be a therapeutic option to treat VWD type 1.

#3

Impact of tranexamic acid on postpartum hemorrhage in type 1 von Willebrand disease treated with recombinant VWF.

Blood advances2025 Dec 09

Postpartum hemorrhage (PPH) affects up to 44% of women with von Willebrand disease (VWD) despite von Willebrand factor (VWF) replacement. Because tranexamic acid (TXA) reduced PPH-related deaths in the WOMAN trial, we assessed whether TXA combined with rVWF vs rVWF alone prevents PPH in VWD. VWD-Woman, a phase 3, open-label, randomized pilot trial, enrolled pregnant women with VWD, aged ≥18 years (von Willebrand Factor Ristocetin Cofactor activity [VWF:RCo] of <0.50 IU/mL, bleeding history). Participants received IV rVWF 80 IU/kg at delivery and postpartum days 1 and 2, with or without TXA (1 g within 3 hours of delivery). The primary outcome was quantitative blood loss (QBL) at delivery. Secondary outcomes included 21-day pictorial blood assessment chart (PBAC) scores, hemoglobin changes, transfusions, hysterectomy, and safety. Of 103 screened, 40 were eligible, and 20 enrolled (10 per group), 100% were iron-deficient. Mean QBL was similar (TXA + rVWF: 727.0 mL; 95% CI, 434.5-1019.5] vs rVWF: 539.7 mL [95% CI, 132.8-946.6]; P = 0.41), as were rates of PPH (30% in both groups). No differences were observed in hemoglobin change (-1.90 g/dL vs -1.42 g/dL, P = 0.49) or 21-day PBAC score (467.1 vs 344.8, P = 0.32). Stratified analyses showed no differences by age, body mass index, VWF activity, or delivery type. No serious adverse events or thrombosis occurred. TXA plus rVWF is feasible and safe in type 1 VWD, but in this small pilot study, was not associated with a reduction in PPH compared with rVWF alone. Iron deficiency is prevalent. Further studies are needed to improve PPH prevention in VWD. This trial was registered at www.ClinicalTrials.gov as #NCT04344860.

#4

A novel cause of type 1 von Willebrand disease: impaired exocytosis of Weibel-Palade bodies due to biallelic MADD variants.

Blood2025 Oct 23

The regulated secretion of von Willebrand factor (VWF) from Weibel-Palade bodies (WPBs) in endothelial cells is fundamental to hemostasis. This process relies on recruiting Rab GTPases and their effectors to the WPB membrane, with the guanine nucleotide exchange factor MAPK-activating death domain (MADD) playing a central role. Biallelic variants in MADD lead to a pleiotropic neurological and developmental disorder that can include bleeding abnormalities. This study investigates the impact of pathogenic MADD variants on VWF secretion using patient-derived endothelial cells. We isolated endothelial colony-forming cells (ECFCs) from 3 pediatric patients with biallelic MADD variants and unaffected heterozygous family members. All patients exhibited low VWF plasma levels (22-30 IU/dL). Proteomic analysis of patient-derived ECFCs revealed an absence of MADD peptides, reduced VWF, and downregulation of proteins involved in the exocytotic machinery, including Rab3D and the Rab3/27 effector Slp4-a. Functional assays demonstrated diminished Rab27A and Rab3D activity and their failure to localize to WPBs in patient cells. Biochemical and live-imaging studies showed that histamine-induced VWF and VWF propeptide secretion were significantly reduced in patient cells due to delayed and reduced degranulation of WPBs. Our findings demonstrate the critical role of MADD in maintaining the secretion competence of WPBs and the magnitude of VWF secretion by regulating the recruitment of the endothelial exocytotic machinery. This study highlights the in vivo significance of WPB exocytosis in maintaining plasma VWF levels and establishes MADD as the first causal gene for quantitative von Willebrand disease in patients without pathogenic VWF variants.

#5

MADDlessness may cause von Willebrand disease type 1.

Blood2025 Oct 23

Publicações recentes

Ver todas no PubMed

📚 EuropePMC30 artigos no totalmostrando 109

2025

Genetic variants in VWF exon 26 and their implications for type 1 Von Willebrand disease in a Saudi Arabian population.

Open life sciences
2025

MADDlessness may cause von Willebrand disease type 1.

Blood
2025

Cavitary Lung Abscess Secondary to a Tracheal Bronchus: A Pediatric Patient With Noonan Syndrome.

Cureus
2025

A bispecific nanobody for the treatment of von Willebrand disease type 1.

Blood
2025

Impact of tranexamic acid on postpartum hemorrhage in type 1 von Willebrand disease treated with recombinant VWF.

Blood advances
2025

Acute Coronary Syndrome in a Young Woman With Protein S Deficiency and Von Willebrand Disease Type 1: A Case of Mixed Hemostatic Disorders.

Cureus
2025

A novel cause of type 1 von Willebrand disease: impaired exocytosis of Weibel-Palade bodies due to biallelic MADD variants.

Blood
2025

Heavy Menstrual Bleeding and Hormonal Therapy in Women with Type 1 von Willebrand Disease Enrolled on the Zimmerman Program.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2025

von Willebrand disease-specific defects and proteomic signatures in endothelial colony-forming cells.

Journal of thrombosis and haemostasis : JTH
2025

A fully humanized von Willebrand disease type 1 mouse model as unique platform to investigate novel therapeutic options.

Haematologica
2024

Tropical Myositis: A Not-So-Tropical Diagnosis in a Febrile Type 1 Diabetic Patient.

WMJ : official publication of the State Medical Society of Wisconsin
2024

Low von Willebrand factor-unraveling an enigma wrapped in a conundrum.

Journal of thrombosis and haemostasis : JTH
2024

Unravelling the spectrum of von Willebrand factor variants in quantitative von Willebrand disease: results from a German cohort study.

Journal of thrombosis and haemostasis : JTH
2024

Type 1 VWD classification revisited: novel insights from combined analysis of the LoVIC and WiN studies.

Blood
2024

The common VWF variant p.Y1584C: detailed pathogenic examination of an enigmatic sequence change.

Journal of thrombosis and haemostasis : JTH
2024

Von Willebrand Factor (VWF) multiplex activity assay differentiation of type 1 von Willebrand Disease (VWD) and variant VWD.

Haemophilia : the official journal of the World Federation of Hemophilia
2023

Efficacy of adjusted weight-based dosing of desmopressin (1-deamino-8-d-arginine vasopressin in type 1 von Willebrand disease.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2023

First case report of hematidrosis complicated with type 1 von Willebrand disease.

Pediatrics international : official journal of the Japan Pediatric Society
2023

Associations of multiple genetic variations with plasma levels of Von Willebrand Factor and clinical phenotype in Iranian patients with Von Willebrand disease type 1.

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis
2023

Phenotypic and genotypic (exon 28) characterization of patients diagnosed with von Willebrand disease type 1 in Eastern Saudi Arabia.

Journal of medicine and life
2023

Coexpression of factor VIII and factor von Willebrand variants in a woman with heavy menstrual bleeding.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2023

Genetic determinants of enhanced von Willebrand factor clearance from plasma.

Journal of thrombosis and haemostasis : JTH
2022

Diagnostic pitfalls and conundrums in type 1 von Willebrand disease.

Hematology. American Society of Hematology. Education Program
2023

Type 1 von Willebrand Disease in a Pediatric Patient Caused by a Novel Heterozygous Deletion of Exons 1 to 6 of the von Willebrand Factor Gene: A Case Report.

Laboratory medicine
2023

A decreased and less sustained response to surrogates of haemostatic stress correlates with bleeding in type 1 von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2023

Analysis of Doberman Pinscher and Toy Poodle samples with targeted next-generation sequencing.

Gene
2022

Influence of Von Willebrand Disease (VWD) and pregnancy on the expression of angiogenic factors in the porcine female reproductive tract.

Reproductive biology
2022

How Do Laboratories Perform von Willebrand Disease Diagnostics and Classification of von Willebrand Disease Patients? Results from External Quality Data and an International Survey.

Seminars in thrombosis and hemostasis
2023

A transcriptome analysis of basal and stimulated VWF release from endothelial cells derived from patients with type 1 VWD.

Blood advances
2022

Longitudinal bleeding assessment in von Willebrand disease utilizing an interim bleeding score.

Journal of thrombosis and haemostasis : JTH
2022

Quantification of the relationship between desmopressin concentration and Von Willebrand factor in Von Willebrand disease type 1: A pharmacodynamic study.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Emicizumab enhances thrombus formation in vitro under high shear flow conditions in whole blood from patients with type 1 and type 3 von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Disruption of the glomerular basement membrane associated with nutcracker syndrome and double inferior vena cava in Noonan syndrome: a case report.

BMC nephrology
2022

Parathyroid Adenoma in a Young Girl with Type 3 von Willebrand Disease: Comment on "Cancers in Patients with von Willebrand Disease".

Seminars in thrombosis and hemostasis
2022

The dominant p.Thr274Pro mutation in the von Willebrand factor propeptide causes the von Willebrand disease type 1 phenotype in two unrelated patients.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Social participation is reduced in type 3 Von Willebrand disease patients and in patients with a severe bleeding phenotype.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Impact of obesity on factor VIII and von Willebrand factor levels in patients with Type 1 von Willebrand disease and low von Willebrand factor: An analysis of the ATHNdataset.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Postpartum Hemorrhage in Patients with Type 1 von Willebrand Disease: A Systematic Review.

Seminars in thrombosis and hemostasis
2022

von Willebrand factor levels in the diagnosis of von Willebrand disease: a systematic review and meta-analysis.

Blood advances
2021

von Willebrand factor antigen levels are associated with burden of rare nonsynonymous variants in the VWF gene.

Blood
2021

The effect of age at diagnosis of type 1 von Willebrand disease on diagnostic laboratory values: A paediatric perspective.

Haemophilia : the official journal of the World Federation of Hemophilia
2021

Endothelial Function in Patients With Von Willebrand Disease.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2021

Criteria for low von Willebrand factor diagnosis and risk score to predict future bleeding.

Journal of thrombosis and haemostasis : JTH
2021

Increasing levels of von Willebrand factor and factor VIII with age in patients affected by von Willebrand disease.

Journal of thrombosis and haemostasis : JTH
2020

How I treat von Willebrand disease.

Thrombosis research
2020

Dramatic Response to Pramipexole in Delayed-Onset Parkinsonism from Osmotic Demyelinating Syndrome.

Tremor and other hyperkinetic movements (New York, N.Y.)
2020

Detection of acquired von Willebrand syndrome after ventricular assist device by total thrombus-formation analysis system.

ESC heart failure
2020

Low VWF: insights into pathogenesis, diagnosis, and clinical management.

Blood advances
2020

Characterization of large in-frame von Willebrand factor deletions highlights differing pathogenic mechanisms.

Blood advances
2020

Use of Tranexamic Acid for the Treatment of Mittelschmerz in a Patient with Type 1 von Willebrand Disease and Recurrent Hemorrhagic Cysts.

Journal of obstetrics and gynaecology Canada : JOGC = Journal d'obstetrique et gynecologie du Canada : JOGC
2020

Bleeding symptoms in patients diagnosed as type 3 von Willebrand disease: Results from 3WINTERS-IPS, an international and collaborative cross-sectional study.

Journal of thrombosis and haemostasis : JTH
2020

Clinical significance of slightly reduced von Willebrand factor activity.

Polish archives of internal medicine
2019

von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD.

Blood advances
2019

Expression of angiogenic factors in the uteroplacental unit is altered at time of placentation in a porcine model of von Willebrand disease type 1.

Reproductive biology
2020

Higher rates of bleeding and use of treatment products among young boys compared to girls with von Willebrand disease.

American journal of hematology
2019

Severe postpartum haemorrhage as first presenting symptom of an inherited bleeding disorder.

Haemophilia : the official journal of the World Federation of Hemophilia
2019

Characterization of a Porcine Model for Von Willebrand Disease Type 1 and 3 Regarding Expression of Angiogenic Mediators in the Nonpregnant Female Reproductive Tract.

Comparative medicine
2019

Bleeding Symptoms and von Willebrand Factor Levels: 30-Year Experience in a Tertiary Care Center.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2019

Case of Hemorrhagic Moyamoya Disease Associated with Von Willebrand Disease.

World neurosurgery
2019

The role of genetics in the pathogenesis and diagnosis of type 1 Von Willebrand disease.

Current opinion in hematology
2019

BMI is an important determinant of VWF and FVIII levels and bleeding phenotype in patients with von Willebrand disease.

American journal of hematology
2019

Whole blood ristocetin-induced platelet impedance aggregometry does not reflect clinical severity in patients with type 1 von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2019

Quantitative analysis of desmopressin (DDAVP) response in adult patients with type 1 von Willebrand disease.

International journal of laboratory hematology
2019

How I treat low von Willebrand factor levels.

Blood
2019

Diagnostic challenges of inherited mild bleeding disorders: a bait for poorly explored clinical and basic research.

Journal of thrombosis and haemostasis : JTH
2018

Genetic Variation in the Syntaxin-Binding Protein STXBP5 in Type 1 von Willebrand Disease Patients.

Thrombosis and haemostasis
2018

A case of inherited type 1 and type 2A von Willebrand disease confirmed by diagnostic exome sequencing.

Pediatric blood &amp; cancer
2018

Anesthetic Management of a Patient With Type 1 von Willebrand Disease and Uterine Placental Abruption: A Case Report.

AANA journal
2018

Inheritance of von Willebrand disease Vicenza in a Japanese family.

Haemophilia : the official journal of the World Federation of Hemophilia
2018

Genetic variation in the C-type lectin receptor CLEC4M in type 1 von Willebrand Disease patients.

PloS one
2018

Genetic Variation in the von Willebrand Factor Gene in Swedish von Willebrand Disease Patients.

TH open : companion journal to thrombosis and haemostasis
2018

von Willebrand disease type 1 in Doberman Pinscher dogs: genotyping and prevalence of the mutation in the Buenos Aires region, Argentina.

Journal of veterinary diagnostic investigation : official publication of the American Association of Veterinary Laboratory Diagnosticians, Inc
2017

Advances in the diagnosis and treatment of Von Willebrand disease.

Hematology. American Society of Hematology. Education Program
2018

Type 1 von Willebrand disease due to a vicinal cysteine loss (p.C524Y) disclosed after a thrombotic episode.

Thrombosis research
2018

Bleeding score in Type 1 von Willebrand disease patients using the ISTH-BAT questionnaire.

International journal of laboratory hematology
2018

Outgrowing the laboratory diagnosis of type 1 von Willebrand disease: A two decade study.

American journal of hematology
2018

Evaluation of a microfluidic flow assay to screen for von Willebrand disease and low von Willebrand factor levels.

Journal of thrombosis and haemostasis : JTH
2018

Discarding Occam's Razor: Atypical Bruising in Severe Hemophilia A.

Clinical pediatrics
2018

The Association of Aging With Von Willebrand Factor Levels and Bleeding Risk in Type 1 Von Willebrand Disease.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2017

Revascularization Operation for Moyamoya Disease with Concurrent von Willebrand Disease.

World neurosurgery
2017

Decreased severity of Shiga toxin-producing Escherichia coli haemolytic uraemic syndrome (STEC-HUS) in a child with type 1 von Willebrand disease.

BMJ case reports
2017

Increase of von Willebrand factor with aging in type 1 von Willebrand disease: fact or fiction?

Haematologica
2017

Whole blood ristocetin-activated platelet impedance aggregometry (Multiplate) for the rapid detection of Von Willebrand disease.

Thrombosis and haemostasis
2017

A 24-year-old woman with heavy menstrual bleeding.

CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienne
2017

Controversies in the diagnosis of Type 1 von Willebrand disease.

International journal of laboratory hematology
2016

Droplet-Shaped Deep Intraretinal Hemorrhage as Initial Presentation of Von Willebrand Disease Type 1.

Ophthalmic surgery, lasers &amp; imaging retina
2016

Intron retention resulting from a silent mutation in the VWF gene that structurally influences the 5' splice site.

Blood
2016

von Willebrand disease type 1 mutation p.Arg1379Cys and the variant p.Ala1377Val synergistically determine a 2M phenotype in four Italian patients.

Haemophilia : the official journal of the World Federation of Hemophilia
2016

A prospective study of von Willebrand factor levels and bleeding in pregnant women with type 1 von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2016

Diagnostic Value of Measuring Platelet Von Willebrand Factor in Von Willebrand Disease.

PloS one
2017

Functional characterisation of the type 1 von Willebrand disease candidate VWF gene variants: p.M771I, p.L881R and p.P1413L.

Blood transfusion = Trasfusione del sangue
2017

Diagnostic Differentiation of von Willebrand Disease Types 1 and 2 by von Willebrand Factor Multimer Analysis and DDAVP Challenge Test.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2017

Haemostatic patterns and bleeding scores of a genetically characterised Italian family with combined haemophilia A and type 1 von Willebrand disease.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2016

Characterization of aberrant splicing of von Willebrand factor in von Willebrand disease: an underrecognized mechanism.

Blood
2016

Identification of von Willebrand disease type 1 in a patient with Ehlers-Danlos syndrome classic type.

Haemophilia : the official journal of the World Federation of Hemophilia
2016

Prophylaxis in von Willebrand Disease: Coming of Age?

Seminars in thrombosis and hemostasis
2017

Gingival bleeding and mild type 1 von Willebrand disease.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2016

Spontaneous recurrent hematuria and hematospermia: Unique manifestations of von Willebrand disease type I. Case report.

Archivio italiano di urologia, andrologia : organo ufficiale [di] Societa italiana di ecografia urologica e nefrologica
2016

Assessing the clinical severity of type 1 von Willebrand disease patients with a microchip flow-chamber system.

Journal of thrombosis and haemostasis : JTH
2016

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

Blood
2016

Baseline factor VIII plasma levels and age at first bleeding in patients with severe forms of von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2016

Validation Study of the Composite Score to Identify Von Willebrand Disease in Children.

Journal of pediatric hematology/oncology
2015

Severe, recessive type 1 is a discrete form of von Willebrand disease: the lesson learned from the c.1534-3C>A von Willebrand factor mutation.

Thrombosis research
2016

Side effects of desmopressin in patients with bleeding disorders.

Haemophilia : the official journal of the World Federation of Hemophilia
2015

CLEC4M and STXBP5 gene variations contribute to von Willebrand factor level variation in von Willebrand disease.

Journal of thrombosis and haemostasis : JTH
2015

Factor X deficiency associated with compound heterozygosity involving a novel missense mutation at codon 38 from Val (GTC) to Leu (CTC) in exon 2.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2015

Changes in von Willebrand factor level and von Willebrand activity with age in type 1 von Willebrand disease.

Haemophilia : the official journal of the World Federation of Hemophilia
2015

von Willebrand factor arginine 1205 substitution results in accelerated macrophage-dependent clearance in vivo.

Journal of thrombosis and haemostasis : JTH
2014

Spectrum of Von Willebrand's disease in Punjab: clinical features and types.

Journal of Ayub Medical College, Abbottabad : JAMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

Ainda não temos associações cadastradas para Doença de Von Willebrand, tipo 1.

É de uma associação que acompanha esta doença? Fale com a gente →

Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

Ainda não existe comunidade no Raras para Doença de Von Willebrand, tipo 1

Pacientes, familiares e cuidadores se organizam em comunidades pra compartilhar experiências, fazer perguntas e se apoiar. Você pode ser o primeiro.

Tire suas dúvidas

Perguntas, dicas e experiências compartilhadas aqui na página

Participe da discussão

Faça login para postar dúvidas, compartilhar experiências e interagir com especialistas.

Fazer login

Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Genetic variants in VWF exon 26 and their implications for type 1 Von Willebrand disease in a Saudi Arabian population.
    Open life sciences· 2025· PMID 41322438mais citado
  2. A bispecific nanobody for the treatment of von Willebrand disease type 1.
    Blood· 2025· PMID 40906928mais citado
  3. Impact of tranexamic acid on postpartum hemorrhage in type 1 von Willebrand disease treated with recombinant VWF.
    Blood advances· 2025· PMID 40902088mais citado
  4. A novel cause of type 1 von Willebrand disease: impaired exocytosis of Weibel-Palade bodies due to biallelic MADD variants.
    Blood· 2025· PMID 40668965mais citado
  5. MADDlessness may cause von Willebrand disease type 1.
    Blood· 2025· PMID 41129183mais citado
  6. Cavitary Lung Abscess Secondary to a Tracheal Bronchus: A Pediatric Patient With Noonan Syndrome.
    Cureus· 2025· PMID 41018327recente
  7. Acute Coronary Syndrome in a Young Woman With Protein S Deficiency and Von Willebrand Disease Type 1: A Case of Mixed Hemostatic Disorders.
    Cureus· 2025· PMID 40799883recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:166078(Orphanet)
  2. OMIM OMIM:193400(OMIM)
  3. MONDO:0008668(MONDO)
  4. GARD:17019(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q26492788(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Doença de Von Willebrand, tipo 1
Compêndio · Raras BR

Doença de Von Willebrand, tipo 1

ORPHA:166078 · MONDO:0008668
🇧🇷 Brasil SUS
CEAF
1AFator VIII de von Willebrand
Geral
Prevalência
1-5 / 10 000
Herança
Autosomal dominant
CID-10
D68.0 · Doença de Von Willebrand
CID-11
Início
All ages
Prevalência
0.0 (Europe)
MedGen
UMLS
C1264039
EuropePMC
Wikidata
Papers 10a
DiscussaoAtiva

Nenhuma novidade ainda. O agente esta monitorando.

0membros
0novidades