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Research Article: Pharmacokinetic-Pharmacodynamic Comparison of Recombinant and Plasma-Derived von Willebrand Factor in Patients with von Willebrand Disease Type

Date Published: 2023-06-13

Abstract:
Von Willebrand disease (VWD) is an inherited bleeding disorder characterized by qualitative or quantitative defects in von Willebrand factor (VWF), a large multimeric glycoprotein that mediates platelet adhesion and stabilizes factor VIII (FVIII) in the circulation.– Recombinant VWF (rVWF, vonicog alfa, Vonvendi® [USA, Japan, Australia] / VEYVONDI™ [EU, UK, Switzerland], Takeda Pharmaceuticals USA, Lexington, MA, USA) has demonstrated efficacy with a favorable safety profile when used for on-demand treatment of hemorrhage and the prevention and treatment of surgical bleeding in adults with VWD.– The efficacy and safety of prophylaxis with rVWF in patients with severe VWD has also been assessed in a phase , open-label, nonrandomized trial. In that study, rVWF reduced the rate of treated, spontaneous bleeding events in patients previously receiving on-demand VWF therapy by .%. Several human plasma-derived VWF (pdVWF)/FVIII concentrates have been approved for the treatment of bleeding episodes in patients with VWD, and their efficacy, safety, and pharmacokinetic (PK) data are published.– Of these, Haemate P®/Humate-P® (pdFVIII/VWF complex [Human]; CSL Behring GmbH, Marburg, Germany: VWF: ristocetin cofactor [RCo]/ factor VIII activity [FVIII:C] .:) is a widely used pdVWF/FVIII concentrate for the treatment of VWD that is effective in the prevention and control of bleeding in patients with VWD.–

Introduction:
Von Willebrand disease (VWD) is an inherited bleeding disorder characterized by qualitative or quantitative defects in von Willebrand factor (VWF), a large multimeric glycoprotein that mediates platelet adhesion and stabilizes factor VIII (FVIII) in the circulation. – Recombinant VWF (rVWF, vonicog alfa, Vonvendi ® [USA, Japan, Australia] / VEYVONDI™ [EU, UK, Switzerland], Takeda Pharmaceuticals USA, Lexington, MA, USA) has demonstrated efficacy with a favorable safety profile when used for on-demand treatment of…

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