Type 3 Von Willebrand International Registries Inhibitor Prospective Study

NCT02460458

NULLOBSERVATIONAL

Fondazione Angelo Bianchi Bonomi

265 participants

Started 2012-11-05 · Completed 2023-04-17

Status: Completed (as of 12-23-2025)

Conditions

Type 3 Von Willebrand's Disease

Interventions

DRUG: Von Willebrand Factor

Linked Papers (from SciSciNet-V2)

Total Papers116

Background

12

Result

0

Derived

0

Unknown

104

View on ClinicalTrials.gov

Total Citations Per Year

Brief Summary

International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand Factor (VWF) concentrates used to treat VWD patients.

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Background Papers24

Endothelial von Willebrand factor regulates angiogenesis

Endothelial von Willebrand factor regulates angiogenesis

Principles of care for the diagnosis and treatment of von Willebrand disease

Principles of care for the diagnosis and treatment of von Willebrand disease

Gene deletions correlate with the development of alloantibodies in von Willebrand disease.

Gene deletions correlate with the development of alloantibodies in von Willebrand disease.

Assembly and routing of von Willebrand factor variants: the requirements for disulfide-linked dimerization reside within the carboxy-terminal 151 amino acids.

Assembly and routing of von Willebrand factor variants: the requirements for disulfide-linked dimerization reside within the carboxy-terminal 151 amino acids.

Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN)

Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN)

Homozygous and heterozygous deletions of the von Willebrand factor gene in patients and carriers of severe von Willebrand disease.

Homozygous and heterozygous deletions of the von Willebrand factor gene in patients and carriers of severe von Willebrand disease.

VON WILLEBRAND FACTOR, ANGIODYSPLASIA AND ANGIOGENESIS

VON WILLEBRAND FACTOR, ANGIODYSPLASIA AND ANGIOGENESIS

Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor

Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor

Mutations of von Willebrand factor gene in families with von Willebrand disease in the Aland Islands.

Multiple substitutions in the von Willebrand factor gene that mimic the pseudogene sequence.

Multiple substitutions in the von Willebrand factor gene that mimic the pseudogene sequence.

Mutations of von Willebrand factor gene in families with von Willebrand disease in the Aland Islands.

Long‐acting somatostatin analogues provide significant beneficial effect in patients with refractory small bowel angiodysplasia: Results from a proof of concept open label mono‐centre trial

Long‐acting somatostatin analogues provide significant beneficial effect in patients with refractory small bowel angiodysplasia: Results from a proof of concept open label mono‐centre trial

Somatostatin Analogs in the Medical Management of Occult Bleeding of the Lower Digestive Tract

Somatostatin Analogs in the Medical Management of Occult Bleeding of the Lower Digestive Tract

Result Papers0

No result papers linked

Derived Papers0

No derived papers linked

Unknown Papers104

The structure and evolution of the human β-globin gene family

1,398 citations

Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor

1,083 citations

A risk score to predict need for treatment for uppergastrointestinal haemorrhage

1,045 citations

Recommendations for a nomenclature system for human gene mutations

ACG Clinical Guideline: Diagnosis and Management of Small Bowel Bleeding

A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM‐1 VWD)

Gene deletions causing human genetic disease: mechanisms of mutagenesis and the role of the local DNA sequence environment

The mutational spectrum of single base-pair substitutions causing human genetic disease: patterns and predictions

The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study

Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients

Biologic response to desmopressin in patients with severe type 1 and type 2 von Willebrand disease: results of a multicenter European study

Evaluation and management of postpartum hemorrhage: consensus from an international expert panel

Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD

Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study

v. Willebrand's Disease in Sweden.

von Willebrand's disease in Sweden.

Von Willebrand factor and angiogenesis: basic and applied issues

Family studies and prenatal diagnosis in severe von Willebrand disease by polymerase chain reaction amplification of a variable number tandem repeat region of the von Willebrand factor gene

Abnormal von Willebrand factor in bleeding angiodysplasias of the digestive tract

Evaluation of the PFA-100® System in the Diagnosis and Therapeutic Monitoring of Patients with von Willebrand Disease

Management of Gastrointestinal Angiodysplastic Lesions (GIADs): A Systematic Review and Meta-Analysis

Guidelines for the diagnosis and management of von Willebrand disease in Italy

Von Willebrand Factor Multimers during Transcatheter Aortic-Valve Replacement

The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease

Different bleeding risk in type 2A and 2M von Willebrand disease: a 2‐year prospective study in 107 patients

Efficacy and Safety of the Factor VIII/von Willebrand Factor Concentrate, Haemate-P/Humate-P: Ristocetin Cofactor Unit Dosing in Patients with von Willebrand Disease

Congenital von Willebrand disease type 3: clinical manifestations, pathophysiology and molecular biology

von Willebrand factor/factor VIII concentrate (Haemate® P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery

Molecular defects in type 3 von Willebrand disease: updated results from 40 multiethnic patients

Long-term prophylaxis in von Willebrand disease

Treatment of severe von Willebrand disease with a high‐purity von Willebrand factor concentrate (Wilfactin®): a prospective study of 50 patients

Posttransfusion anaphylactic reactions in a patient with severe von Willebrand disease: role of complement and alloantibodies to von Willebrand factor.

Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate‐P^®)

Pharmacokinetics, efficacy and safety of Humate‐P^® in von Willebrand disease

Genetic heterogeneity of severe von Willebrand disease type III in the German population

DDAVP shortens the prolonged bleeding times of patients with severe von Willebrand disease treated with cryoprecipitate. Evidence for a mechanism of action independent of released von Willebrand factor

Atherosclerosis and von Willebrand factor

Characterization of the Genetic Defects in Recessive Type 1 and Type 3 von Willebrand Disease Patients of Italian Origin

Characterization of the von Willebrand Factor Gene (VWF) in von Willebrand Disease Type III Patients from 24 Families of Swedish and Finnish Origin

Octreotide in the treatment of gastrointestinal bleeding caused by angiodysplasia in two patients with von Willebrand's disease

Italian Registry of Haemophilia and Allied Disorders. Objectives, methodology and data analysis

Use of Recombinant Factor VIla (NovoSeven®) in the Treatment of Two Patients with Type III von Willebrand’s Disease and an inhibitor against von Willebrand Factor

Pharmacokinetic studies on Wilfactin®, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods

Clinical use of Haemate(R) P in inherited von Willebrand's disease: a cohort study on 100 Italian patients

Detection of Heterozygotes for Recessive von Willebrand's Disease by the Assay of Antihemophilic-Factor-like Antigen

Molecular Characterization of a Multiethnic Group of 21 Patients with Type 3 von Willebrand Disease

Severe type III von Willebrand's disease caused by deletion of exon 42 of the von Willebrand factor gene: family studies that identify carriers of the condition and a compound heterozygous individual

Antibodies to von Willebrand factor in von Willebrand disease

A novel von Willebrand disease–causing mutation (Arg273Trp) in the von Willebrand factor propeptide that results in defective multimerization and secretion

Nonsense mutations of the von Willebrand factor gene in patients with von Willebrand disease type III and type I.

Gastrointestinal angiodysplasia and bleeding in von Willebrand disease

Successful treatment of urgent bleeding in von Willebrand disease with factor VIII/VWF concentrate (Humate‐P^®): use of the ristocetin cofactor assay (VWF:RCo) to measure potency and to guide therapy

Efficacy and safety of factor VIII/von Willebrand's factor concentrate (Haemate-P) in preventing bleeding during surgery or invasive procedures in patients with von Willebrand disease.

Circulatory support devices: fundamental aspects and clinical management of bleeding and thrombosis

Impact of plasma von Willebrand factor levels in the diagnosis of type 1 von Willebrand disease: results from a multicenter European study (MCMDM‐1VWD)

Clinical efficacy of highly purified, doubly virus‐inactivated factor VIII/von Willebrand factor concentrate (Fanhdi^®) in the treatment of von Willebrand disease: a retrospective clinical study

Mutations in Severe, Type III von Willebrand’s Disease in the Dutch Population: Candidate Missense and Nonsense Mutations Associated with Reduced Levels of von Willebrand Factor Messenger RNA

Developments in the diagnostic procedures for von Willebrand disease

Management of inherited von Willebrand disease in 2007

The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease

Gene conversions are a common cause of von Willebrand disease

Factor VIII: C increases after desmopressin in a subgroup of patients with autosomal recessive severe von Willebrand disease

Highly purified VWF/FVIII concentrates in the treatment and prophylaxis of von Willebrand disease: the PRO.WILL Study

A relatively high frequency of severe (type III) von Willebrand's disease in Israel

Pharmacokinetics of von Willebrand factor and factor VIIIC in patients with severe von Willebrand disease (type 3 VWD): estimation of the rate of factor VIIIC synthesis

Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy

Prophylaxis escalation in severe von Willebrand disease: a prospective study from the von Willebrand Disease Prophylaxis Network

Effects of the mutant von Willebrand factor gene in von Willebrand disease

Efficacy and safety of highly purified, doubly virus‐inactivated VWF/FVIII concentrates in inherited von Willebrand’s disease: results of an Italian cohort study on 120 patients characterized by bleeding severity score

Identification of a new nonsense mutation in the von Willebrand factor gene in patients with von Willebrand disease type III

Characterisation of a novel high-purity, double virus inactivated von Willebrand Factor and Factor VIII concentrate (Wilate®)

A common 253‐kb deletion involving VWF and TMEM16B in German and Italian patients with severe von Willebrand disease type 3

A single cytosine deletion in exon 18 of the von Willebrand factor gene is the most common mutation in Swedish vWD type III patients

Heterogeneous phenotypes of platelet and plasma von Willebrand factor in obligatory heterozygotes for severe von Willebrand disease

Genetic defects in von Willebrand disease type 3 in Indian and Greek patients

An Alu‐mediated novel large deletion is the most frequent cause of type 3 von Willebrand disease in Hungary

Fanhdi^®, efficacy and safety in von Willebrand’s disease: prospective international study results

Comparison of the pharmacokinetics of two von Willebrand factor concentrates [Biostate and AHF (High Purity)] in people with von Willebrand disorder. A randomised cross-over, multi-centre study.

A common frameshift mutation in von Willebrand factor does not alter mRNA stability but interferes with normal propeptide processing

Characterization of Partial Gene Deletions in Type III von Willebrand Disease with Alloantibody Inhibitors

Thalidomide for treatment of gastrointestinal bleedings due to angiodysplasia: a case report in acquired von Willebrand syndrome and review of the literature

Expression studies of missense mutations p.D141Y, p.C275S located in the propeptide of von Willebrand factor in patients with type 3 von Willebrand disease

Clinical and laboratory diagnosis of VWD

A stop codon in a patient with severe type III von Willebrand disease [letter]

Homozygous C2362F von Willebrand factor induces intracellular retention of mutant von Willebrand factor resulting in autosomal recessive severe von Willebrand disease

Intracranial hemorrhage in a term newborn with severe von Willebrand disease type 3 associated with sinus venous thrombosis.

Circulating and progenitor endothelial cells are abnormal in patients with different types of von Willebrand disease and correlate with markers of angiogenesis

Efficacy and safety of a high purity, double virus inactivated factor VIII/von Willebrand factor concentrate (Biostate^®) in patients with von Willebrand disorder requiring invasive or surgical procedures

A novel Alu-mediated 61-kb deletion of the von Willebrand factor (VWF) gene whose breakpoints co-locate with putative matrix attachment regions

A Novel Mutation Glyl672→Arg in Type 2A and a Homozygous Mutation in Type 2B von Willebrand Disease

Instability of repeats of the von Willebrand factor gene variable number tandem repeat sequence in intron 40

Q1311X: a novel nonsense mutation of putative ancient origin in the von Willebrand factor gene. SHORT REPORT

Current and emerging approaches for assessing von Willebrand disease in 2016

A G+3-to-T Donor Splice Site Mutation Leads to Skipping of Exon 50 in Von Willebrand Factor mRNA

Clinical and molecular markers of inherited von Willebrand disease type 3: are deletions of theVWF gene associated with alloantibodies to VWF?

Heightened proteolysis of the von Willebrand factor subunit in patients with von Willebrand disease hemizygous or homozygous for the C2362F mutation

Duodenal and gastric Dieulafoy's lesions in a patient with type 2A von Willebrand's disease

Compound heterozygosity for two novel mutations (1203insG/Y1456X) in the von Willebrand factor gene causing type 3 von Willebrand disease