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Von Willebrand Disease

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1. Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions. (PubMed)

Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions. Minor oral surgery or dental extractions (oral or dental procedures) are widely performed and can be complicated by hazardous oral bleeding, especially in people with an inherited bleeding disorder such as haemophilia or Von Willebrand disease (VWD). The amount and severity of singular bleedings depend on disease-related factors

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2019 Cochrane

3. Von Willebrand disease

Von Willebrand disease Von Willebrand disease - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Von Willebrand disease Last reviewed: February 2019 Last updated: November 2018 Summary Autosomal inheritance with variable penetrance and phenotypic expression. Usually presents with mucocutaneous bleeding. Menorrhagia and postnatal haemorrhage common in affected females. Joint bleeding rare and seen only in patients (...) with more severe disease. Most patients have type 1 von Willebrand disease; more severe symptoms are seen in types 2 and 3. Clinical bleeding scores may be helpful in identifying patients with disease. Definition Von Willebrand disease (VWD), the most common inherited bleeding disorder, is due to either a quantitative or qualitative abnormality of von Willebrand factor (VWF). VWF provides the critical link between platelets and exposed vascular subendothelium, and also binds and stabilises coagulation

2018 BMJ Best Practice

4. Von Willebrand disease

Von Willebrand disease Von Willebrand disease - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Von Willebrand disease Last reviewed: February 2019 Last updated: November 2018 Summary Autosomal inheritance with variable penetrance and phenotypic expression. Usually presents with mucocutaneous bleeding. Menorrhagia and postnatal haemorrhage common in affected females. Joint bleeding rare and seen only in patients (...) with more severe disease. Most patients have type 1 von Willebrand disease; more severe symptoms are seen in types 2 and 3. Clinical bleeding scores may be helpful in identifying patients with disease. Definition Von Willebrand disease (VWD), the most common inherited bleeding disorder, is due to either a quantitative or qualitative abnormality of von Willebrand factor (VWF). VWF provides the critical link between platelets and exposed vascular subendothelium, and also binds and stabilises coagulation

2018 BMJ Best Practice

5. Blood volume–based von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease (PubMed)

Blood volume–based von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease Publisher's Note: This article has a companion Point by Kouides. Publisher's Note: Join in the discussion of these articles at Blood Advances Community Conversations.

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2017 Blood advances

6. Evaluation of the Utility of von Willebrand Factor Propeptide in the Differential Diagnosis of von Willebrand Disease and Acquired von Willebrand Syndrome. (PubMed)

Evaluation of the Utility of von Willebrand Factor Propeptide in the Differential Diagnosis of von Willebrand Disease and Acquired von Willebrand Syndrome. An increased von Willebrand factor propeptide (VWFpp) to VWF antigen (VWF:Ag) ratio (VWFpp/VWF:Ag) indicates an enhanced clearance of VWF. This finding has been described in von Willebrand disease (VWD) and in acquired von Willebrand syndrome (AVWS). A distinction between these two diseases, one congenital and the other acquired

2018 Seminars In Thrombosis And Hemostasis

7. Differences in von Willebrand factor function in type 2A von Willebrand disease and left ventricular assist device‐induced acquired von Willebrand syndrome (PubMed)

Differences in von Willebrand factor function in type 2A von Willebrand disease and left ventricular assist device‐induced acquired von Willebrand syndrome Patients with von Willebrand disease (VWD) type 2A or acquired von Willebrand syndrome (aVWS) as a consequence of implantation of left ventricular assist devices (LVAD) are both characterized by a loss of von Willebrand factor (VWF) function. Loss of VWF function is however more severe in VWD type 2A than in LVAD patients.To compare VWF

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2018 Research and Practice in Thrombosis and Haemostasis

8. Phase 3 study of recombinant von Willebrand factor in patients with severe von Willebrand disease who are undergoing elective surgery: comment. (PubMed)

Phase 3 study of recombinant von Willebrand factor in patients with severe von Willebrand disease who are undergoing elective surgery: comment. We were interested to read the recently published paper in the Journal of Thrombosis and Haemostasis by Peyvandi et al. [1]. The phase 3 study evaluates the efficacy and safety of recombinant von Willebrand factor (rVWF) in patients with severe von Willebrand disease (VWD) undergoing elective surgery. The paper provides supporting evidence for the use

2019 Journal of Thrombosis and Haemostasis

9. Evolution of replacement therapy for von Willebrand disease: From plasma fraction to recombinant von Willebrand factor. (PubMed)

Evolution of replacement therapy for von Willebrand disease: From plasma fraction to recombinant von Willebrand factor. The diagnosis and treatment of von Willebrand disease (VWD) are challenging, in part because patients exhibit a wide range of bleeding patterns and manifestations (e.g. epistaxis, gingival bleeding, heavy menstrual bleeding, gastrointestinal bleeds, postoperative bleeding, hemarthroses) and in part because many tests are required to make an accurate diagnosis. Factor (...) replacement therapies for VWD are the mainstay of treatment for patients who do not respond to desmopressin. They have gradually evolved from crude preparations of plasma proteins to plasma-derived concentrates containing both von Willebrand factor (VWF) and factor VIII (FVIII). However, varying amounts and quality of VWF and varying content of FVIII have contributed to the lack of a standardized approach to replacement therapy. More recently, the treatment of VWD has undergone a slow yet significant

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2019 Blood reviews

10. A Mechanistic Model to Quantify von Willebrand Factor Release, Survival and Proteolysis in Patients with von Willebrand Disease. (PubMed)

A Mechanistic Model to Quantify von Willebrand Factor Release, Survival and Proteolysis in Patients with von Willebrand Disease. A reduced von Willebrand factor (VWF) synthesis or survival, or its increased proteolysis, alone or in combination, contributes to the development of von Willebrand disease (VWD).We describe a new, simple mechanistic model for exploring how VWF behaves in well-defined forms of VWD after its 1-desamino-8-D-arginine vasopressin (DDAVP)-induced release from endothelial

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2018 Thrombosis and haemostasis

11. Phase 3 Study of Recombinant von Willebrand Factor in Patients With Severe von Willebrand Disease Who Are Undergoing Elective Surgery. (PubMed)

Phase 3 Study of Recombinant von Willebrand Factor in Patients With Severe von Willebrand Disease Who Are Undergoing Elective Surgery. Essentials Recombinant von Willebrand factor (rVWF) is effective in von Willebrand disease (VWD). A phase 3 study of rVWF, with/without recombinant factor VIII (rFVIII) before surgery in VWD. Overall rVWF's efficacy was rated excellent/good; rVWF was administered alone in most patients. rVWF was well-tolerated and hemostasis was achieved in patients with severe (...) VWD undergoing surgery. SUMMARY: Background Recombinant von Willebrand factor (rVWF) has demonstrated efficacy for on-demand treatment of bleeding in severe von Willebrand disease (VWD), warranting evaluation in the surgical setting. Objectives This study (NCT02283268) evaluated the hemostatic efficacy/safety profile of rVWF, with/without recombinant factor VIII (rFVIII), in patients with severe VWD undergoing surgery. Patients/Methods Patients received rVWF 40-60 IU kg-1 , VWF ristocetin cofactor

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2018 Journal of Thrombosis and Haemostasis

12. A comparative analysis of different automated von Willebrand factor Glycoprotein Ib-binding activity assays in well typed von Willebrand Disease patients. (PubMed)

A comparative analysis of different automated von Willebrand factor Glycoprotein Ib-binding activity assays in well typed von Willebrand Disease patients. Essentials Von Willebrand ristocetin cofactor activity (VWF:RCo) is not a completely reliable assay. Three automated VWF activity assays were compared within a von Willebrand disease (VWD) cohort. Raw values for all three assays were virtually the same. An overall problem within type 2A/IIE VWD using VWF:GPIb-binding activity/VWF:Ag (...) was observed.Background von Willebrand disease (VWD) is an inherited bleeding disorder caused by quantitative (type 1 and 3) or qualitative (type 2) von Willebrand factor (VWF) defect. VWD diagnosis and classification require numerous laboratory tests. VWF: glycoprotein Ib (GPIb)-binding activity assays are used to distinguish type 1 from type 2 VWD. Objectives Three different automated VWF:GPIb-binding activity assays were compared. Patients and methods BC-VWF:RCo (Siemens Healthcare Diagnostics), HemosIL® VWF:RCo

2018 Journal of Thrombosis and Haemostasis

13. Comorbidities associated with higher von Willebrand factor (VWF) levels may explain the age-related increase of VWF in von Willebrand disease. (PubMed)

Comorbidities associated with higher von Willebrand factor (VWF) levels may explain the age-related increase of VWF in von Willebrand disease. Some comorbidities, such as hypertension, are associated with higher von Willebrand factor (VWF) levels in the general population. No studies have been conducted to assess this association in patients with von Willebrand disease (VWD). Therefore, we studied this association in patients with type 1 (n = 333) and type 2 (n = 203) VWD from the 'WiN" study

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2018 British journal of haematology

14. Vonicog alfa for severe von Willebrand disease

Vonicog alfa for severe von Willebrand disease Vonicog alfa for severe von Willebrand disease Vonicog alfa for severe von Willebrand disease NIHR HSRIC Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Vonicog alfa for severe von Willebrand disease. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors (...) ' conclusions People with von Willebrand disease either have a deficiency of a blood protein called von Willebrand factor, or this protein doesn't work properly. This means that their blood cannot clot properly and they are prone to prolonged or excessive bleeding. There are three different types of von Willebrand disease – types 1, 2, and 3. Type 3 is rare and people with this type have very low levels of von Willebrand factor in their blood, or none at all. Vonicog alfa is a new drug that is given

2016 Health Technology Assessment (HTA) Database.

15. MASAC Recommendations Regarding the Treatment of von Willebrand Disease

MASAC Recommendations Regarding the Treatment of von Willebrand Disease MASAC Recommendations Regarding the Treatment of von Willebrand Disease | National Hemophilia Foundation Secondary menu Search form Search this site Search Main menu » » » » MASAC Recommendations Regarding The Treatment of Von Willebrand Disease PrintFriendly MASAC Recommendations Regarding the Treatment of von Willebrand Disease Date: November 3, 2016 ID: 244 Revisions: 186; 173; 196 Attachment Size 196.75 KB von (...) of hemostasis. Prog Hemost Thromb 1986; 8: 19-45. Mannucci PM, Canciani MT, Rota L, Donovan BS. Response of factor VIII/von Willebrand factor to DDAVP in healthy subjects and patients with haemophilia A and von Willebrand disease. Br J Haematol 1981; 47: 283-93. Mannucci PM, Lusher JM. Desmopressin and thrombosis. Lancet 1989; 2: 675-6. Nilsson IM, Hethagen S. Current status of DDAVP formulations and their use. In Lusher JM, Kessler CM, eds. Hemophilia and von Willebrand disease in the 1990’s. International

2016 National Hemophilia Foundation

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

Evaluation of a microfluidic flow assay to screen for von Willebrand disease and low von Willebrand factor levels. Essentials von Willebrand factor (VWF) function is shear stress dependent. Platelet accumulation in a microfluidic assay correlates with VWF levels. The microfluidic assay discriminates type 1 von Willebrand disease from healthy controls. The microfluidic flow assay detects responses to therapeutic intervention (DDAVP).Background von Willebrand disease (VWD) is a mucocutaneous (...) bleeding disorder with a reported prevalence of 1 in 10 000. von Willebrand factor (VWF) function and platelet adhesion are regulated by hemodynamic forces that are not integrated into most current clinical assays. Objective We evaluated whether a custom microfluidic flow assay (MFA) can screen for deficiencies in VWF in patients presenting with mucocutaneous bleeding. Methods Whole blood from individuals with mucocutaneous bleeding was assayed in a custom MFA. Results Thirty-two patients with type 1

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2017 Journal of Thrombosis and Haemostasis

17. High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease. (PubMed)

High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease. Essentials von Willebrand disease (VWD) is the most common inherited bleeding disorder. Gene therapy for VWD offers long-term therapy for VWD patients. Transposons efficiently integrate the large von Willebrand factor (VWF) cDNA in mice. Liver-directed transposons support sustained VWF expression with suboptimal multimerization.Background (...) Type 3 von Willebrand disease (VWD) is characterized by complete absence of von Willebrand factor (VWF). Current therapy is limited to treatment with exogenous VWF/FVIII products, which only provide a short-term solution. Gene therapy offers the potential for a long-term treatment for VWD. Objectives To develop an integrative Sleeping Beauty (SB) transposon-mediated VWF gene transfer approach in a preclinical mouse model of severe VWD. Methods We established a robust platform for sustained

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2017 Journal of Thrombosis and Haemostasis

18. Abnormal von Willebrand factor secretion, factor VIII stabilization, and thrombus dynamics in type 2N von Willebrand disease mice. (PubMed)

Abnormal von Willebrand factor secretion, factor VIII stabilization, and thrombus dynamics in type 2N von Willebrand disease mice. Essentials Type 2N von Willebrand disease involves impaired von Willebrand factor to factor VIII binding. Type 2N von Willebrand disease mutations exhibit qualitative and mild quantitative deficiencies. Type 2N von Willebrand disease mice exhibit unstable venous hemostatic thrombi. The factor VIII-binding ability of von Willebrand factor regulates arteriole (...) thrombosis dynamics.Background von Willebrand factor (VWF) and factor VIII (FVIII) circulate as a non-covalent complex, with VWF serving as the carrier for FVIII. VWF indirectly influences secondary hemostasis by stabilizing FVIII and transporting it to the site of primary hemostasis. Type 2N von Willebrand disease involves impaired binding of VWF to FVIII, resulting in decreased plasma levels of FVIII. Objectives In these studies, we characterize the impact of three type 2N VWD variants (R763A, R854Q

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2017 Journal of Thrombosis and Haemostasis

19. Discrepant platelet and plasma von Willebrand factor in von Willebrand disease patients with p.Pro2808Leufs*24. (PubMed)

Discrepant platelet and plasma von Willebrand factor in von Willebrand disease patients with p.Pro2808Leufs*24. Essentials von Willebrand factor (VWF) is synthesized in endothelial cells and platelet precursors. Type 3 patients with Pro2808Leufs*24 have lower bleeding scores than other type 3s. The Pro2808Leufs*24 variant was examined in patient platelets and endothelial cells. Type 3s with this variant contain releaseable VWF, possibly reducing bleeding.Background A novel variant (...) , p.Pro2808Leufs*24, in the von Willebrand factor (VWF) gene was previously identified in the Canadian von Willebrand disease (VWD) patient population. Clinical observations of type 3 VWD patients with this variant indicate a milder bleeding phenotype compared with other type 3 patients. Objective To assess the effect of the Pro2808Leufs*24 variant on the molecular pathogenesis of VWD and correlate this with the phenotype observed in patients. Patients/Methods Phenotypic data from individuals in the Canadian

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2017 Journal of Thrombosis and Haemostasis

20. Increase of von Willebrand factor with aging in type 1 von Willebrand disease: fact or fiction? (PubMed)

Increase of von Willebrand factor with aging in type 1 von Willebrand disease: fact or fiction? 28751564 2018 10 18 2018 11 13 1592-8721 102 11 2017 11 Haematologica Haematologica Increase of von Willebrand factor with aging in type 1 von Willebrand disease: fact or fiction? e431-e433 10.3324/haematol.2017.168013 Borghi Mariachiara M Department of Medicine, Section of Internal and Cardiovascular Medicine, University of Perugia, Italy. Guglielmini Giuseppe G Department of Medicine, Section (...) and Cardiovascular Medicine, University of Perugia, Italy. Gresele Paolo P Department of Medicine, Section of Internal and Cardiovascular Medicine, University of Perugia, Italy paolo.gresele@unipg.it. eng Letter Research Support, Non-U.S. Gov't 2017 07 27 Italy Haematologica 0417435 0390-6078 0 von Willebrand Factor 9001-27-8 Factor VIII IM Age Factors Factor VIII metabolism Female Follow-Up Studies Humans Male Platelet Aggregation von Willebrand Disease, Type 1 blood diagnosis von Willebrand Factor metabolism

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2017 Haematologica

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