New therapies for von Willebrand disease.

New therapies for von Willebrand disease. Hematology Am Soc Hematol Educ Program. 2019 Dec 06;2019(1):590-595 Authors: Mannucci PM Abstract The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C), due to the loss of binding and stabilization by VWF of this intrinsic coagulation factor in flowing blood. The traditional therapeutic weapons (the synthetic derivative of the antidiuretic hormone desmopressin and plasma-derived VWF/FVIII concentrates) are able to transiently correct both the defects. With the goal of tackling the primary deficiency in the disease, that is, VWF, but at the same time exploiting the normal capacity of patients to produce FVIII, the novel approach of replacing only VWF was implemented in the last 10 years. Following the manufacturing of a concentrate fractionated from human plasma and of one obtained by recombinant DNA technology, clinical studies have shown that VWF-only products correct not only the primary VWF deficiency but also the secondary FVIII:C deficiency. The demonstrated efficacy of these products in various clinical situations and, ultimately, in such a hemostasis-challenging context as surgery testifies to the effectiveness and safety of this approach. It remains to be seen whether VWF-only products are efficacious and safe in...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research

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This article comprehensively reviews the efficacy, safety, pharmacokinetics, and pharmacodynamics of vonicog alfa. These points will be discussed using institutional experiential data from the University of Virginia (UVA) Health System in relation to the clinical studies. The goal of this review article is to offer insights to clinical directions, discuss operational challenges, and offer guidance for future studies and formulary decisions.
Source: Journal of Thrombosis and Thrombolysis - Category: Hematology Source Type: research
Semin Thromb Hemost DOI: 10.1055/s-0039-3400273Cardiopulmonary bypass and extracorporeal membrane oxygenation (ECMO) cause hemostatic derangements that can predispose patients to both bleeding and thrombotic complications. Often, patients present for urgent surgery while taking medications including antiplatelet agents, vitamin K antagonists, and direct oral anticoagulants, which must be recognized, monitored, and managed. During extracorporeal circulation, appropriate anticoagulation, most commonly with heparin, is required to maintain blood flow and avoid thrombotic complications. However, anticoagulation and other effec...
Source: Seminars in Thrombosis and Hemostasis - Category: Hematology Authors: Tags: Review Article Source Type: research
Authors: Itzhar-Baikian N, Boisseau P, Joly B, Veyradier A Abstract Introduction: Von Willebrand disease (VWD) is the most common inherited bleeding disorder, characterized by a quantitative or qualitative defect of von Willebrand factor (VWF), a multimeric glycoprotein crucial for primary hemostasis and coagulation. VWD pathophysiology is heterogeneous as it includes several types and subtypes which therapeutic management is different. The mainstays of VWD treatment are desmopressin and replacement therapy based on both plasma-derived concentrates and a recently developed recombinant VWF. VWD definitive diagnosis ...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
The goal of treating Von Willebrand Disease (VWD) is to replace deficient or dysfunctional Von Willebrand Factor (VWF) protein. However, the choice of treatment has to be considered carefully in view of patient factors and the unique properties of replacement products. Tailoring a treatment plan to an individual patient ’s bleeding challenge is an intricate process. This review describes personalization of treatment selection for desmopressin (DDAVP), VWF replacement concentrates, including the newly available recombinant VWF (rVWF) and prophylaxis as a treatment approach in VWD.
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
This article reviews these recent advances, as well as currently available treatment options, including desmopressin, antifibrinolytics, and both plasma-based and recombinant von Willebrand factor products. The diagnosis of VWD remains complex and this article provides an understanding of the various subtypes and phenotypes of VWD, as well as the appropriateness and interpretation of the many available laboratory tests.
Source: Hematology/Oncology Clinics of North America - Category: Cancer & Oncology Authors: Source Type: research
Introduction:Platelet storage pool deficiency (PSPD) is a disorder of platelet dysfunction with a varying degree of bleeding severity that can range from no symptoms to severe bleeding with hemostatic challenges. The true incidence of the disorder is unknown; however, some believe that it could be as common as von Willebrand disease (vWD). The current standard for diagnosis of PSPD is platelet electron microscopy (EM). However, there is a lack of standardization of techniques and reference ranges. In addition, some experts debate the clinical significance of the diagnosis based on the abnormal platelet EM finding alone. Av...
Source: Blood - Category: Hematology Authors: Tags: 311. Disorders of Platelet Number or Function: Poster I Source Type: research
Recent international consensus guidelines recommend that patients with significant bleeding phenotypes and plasma VWF levels in the 30-50 IU/dL range should be classified as Low von Willebrand factor (Low VWF). Critically however, evidence-based guidelines regarding optimal clinical management strategies for this cohort have not been defined. In particular, data regarding the need for hemostatic cover for procedures in Low VWF are limited. To address this deficit, as part of the Low Von Willebrand in Ireland Cohort (LoVIC) study we conducted a systematic retrospective review of all procedures in a large cohort of well char...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster I Source Type: research
IntroductionType 1 von Willebrand disease (VWD 1) accounts for ~75% of VWD. The National Heart, Lung, and Blood (NHLBI) VWD guidelines recommend VWF:Ag and VWF:RCo cut-off levels of 1 year (n=36) after initial lab testing at our institution. Median age of patients at time of most recent measurement was 42.5 (5-87) years compared to 35.5 (0-75) years at first measurement. VWF:Ag was significantly higher in the most recent measurement compared to the first measurement (p
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster I Source Type: research
We report a patient with type 3 vWD who inherited two different mutations, one from each parent, resulting in compound heterozygosity.Case:Our patient, now a 2 year old female, initially presented with prolonged bleeding lasting approximately 5 hours at the injection site of her 2 month immunizations. Labs on initial presentation showed a normal WBC count, hemoglobin, hematocrit, and platelet count, with normal levels of Factor IX, XI, and XII activity. PTT was prolonged at 59 (reference range 23.3-35.7) with a normal INR. Von Willebrand panel showed markedly decreased Factor VIII (2%), vWF antigen (6%), and vWF activity (...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis Source Type: research
Background: Von Willebrand disease (vWD) is the most common inherited bleeding disorder with a reported prevalence of 1% in epidemiological studies and symptomatic prevalence of 1 in 10,000.Pregnancy in vWD is associated with increased bleeding risk particularly postpartum hemorrhage. Treatment options include desmopressin acetate (DDAVP), plasma derived factor concentrates and antifibrinolytic agents. Human Recombinant von Willebrand factor (vWF) (Vonvendi®) has been approved in the United States for on demand treatment and perioperative management of adults with vWD. It has been shown to maintain sustained levels of ...
Source: Blood - Category: Hematology Authors: Tags: 401. Basic Science and Clinical Practice in Blood Transfusion Source Type: research
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