von Willebrand's disease

Holmberg, Lars; Nilsson, Inga Marie

doi:10.1111/j.1600-0609.1992.tb00584.x

Cited by 43 publications

(7 citation statements)

References 128 publications

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“…von Willebrand disease (originally termed ‘hereditary pseudo‐haemophilia’) is caused by a deficiency in, or a dysfunction of, VWF (28, 29). The inheritance modus is mainly autosomal dominant, and only occasionally autosomal recessive, affecting males and females with almost the same frequency.…”

Section: Disease Overviewmentioning

confidence: 99%

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Berntorp

Archey

Auerswald

et al. 2008

European J of Haematology

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Patients with von Willebrand disease (VWD) and haemophilia A (HA) lack, to varying degrees, the von Willebrand factor (VWF) and coagulation factor VIII (FVIII) that are critical for normal haemostasis. These conditions in turn make patients prone to uncontrolled bleeding. Historically, patients with severe forms of VWD or HA were crippled before adulthood and their life expectancy was significantly reduced. Over the past decades, specific coagulation factor replacement therapies including Haemate Ò P, have been developed to help patients achieve and maintain normal haemostasis. Haemate Ò P is a human, plasma-derived VWF ⁄ FVIII medicinal product, which was first licensed in Germany in 1981 for the treatment of HA-associated bleeding. It has since then come to be accepted as the gold standard for both the treatment and prophylaxis of bleeding in VWD, especially in cases where desmopressin [1-deamino-8-D-arginine vasopressin (DDAVP)] has been ineffective. Haemate Ò P was the first effectively virus-inactivated (pasteurisation: 60°C for 10 h in aqueous solution) FVIII product, whereby the risk of potentially threatening infective complications of plasma-derived products was reduced. Haemate Ò P was also shown to have a VWF multimer profile remarkably close to that of normal plasma. This bibliographic review presents previously unpublished clinical data of Haemate Ò P, based upon internal clinical study reports of the proprietor, CSL Behring, in addition to data already presented in other publications. The data demonstrate a predictable and well-characterised pharmacokinetic profile, and a proven record of short-and long-term safety, while effectively correcting the haemostatic defects in VWD and HA. Recently available data have also shown Haemate Ò P to be of haemostatic value in exceptional clinical circumstances including surgical interventions. By virtue of its plasma-derived combination of VWF and FVIII, in addition to its high VWF:FVIII content ratio (2.4:1), Haemate Ò P is also associated with successful immune tolerance induction in those patients developing inhibitor antibodies. Although the theoretical risk of thromboembolic complications does exist while receiving Haemate Ò P, as it does with any FVIII replacement therapy, the incidence of such complications has remained notably low. Given the robust data that have accumulated for the use of Haemate Ò P, dosing recommendations are also described in this review; the recommendations are tailored to patient-specific contexts including baseline VWF and FVIII levels in plasma and the type of surgical intervention being undertaken. A wide variety of studies have also provided data on paediatric and geriatric populations, all of which have suggested that Haemate Ò P can be safely and effectively used in a wide variety of clinical circumstances. Effective haemostasis requires complex bodily processes such as vasoconstriction and blood clotting so as to ensure the integrity of the blood vessel system and to prevent excessive blood loss following injury. B...

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Section: Disease Overviewmentioning

confidence: 99%

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Berntorp

Archey

Auerswald

et al. 2008

European J of Haematology

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“…von Willebrand factor (VWF) is a large multimeric adhesive sialoglycoprotein that mediates platelet adhesion to sub‐endothelium structures and acts as a factor VIII (FVIII) carrier molecule, thus stabilizing the procoagulant activity of FVIII in the circulation . The protein is synthesized by endothelial cells and megakaryocytes as a polypeptide and is composed of identical monomers that assemble into a series of multimers. The multimer organization is critical for the function of VWF .…”

Section: Introductionmentioning

confidence: 99%

“…von Willebrand disease (VWD) is the most common congenital bleeding disorder, with a worldwide prevalence of 1% . The current classification of VWD variants by the International Society on Thrombosis and Haemostasis (ISTH) recognizes six different types, reviewed with updates elsewhere .…”

Section: Introductionmentioning

confidence: 99%

Preclinical evaluation of a semi‐automated and rapid commercial electrophoresis assay for von Willebrand factor multimers

Pikta

Zemtsovskaja

Bautista

et al. 2018

Clinical Laboratory Analysis

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“…von Willebrand's disease (vWD) can be subdivided into several forms depending on the nature of the vWF deficiency. Briefly, type I is characterized by a moderate reduction of plasma and sometimes platelet vWF concentration, type I1 by a qualitative abnormality of the protein, and type 111 by very low or undetectable vWF levels (2).…”

Section: Introductionmentioning

confidence: 99%

An Arg⁵⁴⁵→ Cys⁵⁴⁵ substitution mutation of the von Willebrand factor in type IIB von Willebrand's disease

Donnér

Andersson

Kristoffersson

et al. 1991

European J of Haematology

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Type IIB is a special variant of von Willebrand's disease, characterized by an abnormal von Willebrand factor which shows an increased interaction with platelets. This interaction sometimes causes platelet aggregation and thrombocytopenia in vivo. It involves the glycoprotein‐Ib (GPIb) receptor on platelets and corresponding GPIb‐binding sites in the von Willebrand factor. We here demonstrate a C ± T mutation at codon 1308 of the von Willebrand factor gene in 2 related patients with IIB von Willebrand's disease. The transition gives rise to a substitution of arginine by cysteine at position 545 of the mature von Willebrand factor subunit. This position is close to the GPIb‐ as well as the collagen‐ and heparin‐binding domains of the von Willebrand factor. The mutation may change the conformation of the molecule in this region and activate the GPIb‐binding domain, which is normally not exposed in the von Willebrand factor of circulating blood.

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von Willebrand's disease

Cited by 43 publications

References 128 publications

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Preclinical evaluation of a semi‐automated and rapid commercial electrophoresis assay for von Willebrand factor multimers

An Arg⁵⁴⁵→ Cys⁵⁴⁵ substitution mutation of the von Willebrand factor in type IIB von Willebrand's disease

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von Willebrand's disease

Cited by 43 publications

References 128 publications

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate®P/ Humate®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate®P/ Humate®‐P: History and clinical performance

Preclinical evaluation of a semi‐automated and rapid commercial electrophoresis assay for von Willebrand factor multimers

An Arg545→ Cys545 substitution mutation of the von Willebrand factor in type IIB von Willebrand's disease

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A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

An Arg⁵⁴⁵→ Cys⁵⁴⁵ substitution mutation of the von Willebrand factor in type IIB von Willebrand's disease