FDA Approves Baxalta's von Willebrand Disease Drug

Vonvendi is the first FDA-approved recombinant von Willebrand factor for the on-demand treatment and control of bleeding episodes
Source: PharmaManufacturing.com - Category: Pharmaceuticals Source Type: news

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Authors: Rodriguez-Merchan EC Abstract Introduction: The purpose of this article is to review which data about hemophilia are currently provided by the Cochrane database of systematic reviews (CDBSR). Methodological consideration: All statements about hemophilia in the Cochrane Collaboration are based on evidence generated in randomized controlled clinical trials. Areas covered: There is a high degree of evidence that prophylaxis preserves joint function in children with hemophilia compared to on-demand treatment. Also, that recombinant factor VII activated (rFVIIa) and activated prothrombin complex concentrates (A...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Acetaminophen (paracetamol, APAP) overdose is a leading cause of drug-induced acute liver injury and acute liver failure (ALF) in the western world.[1] Accumulating evidence from experimental and clinical studies suggests that the hemostatic system contributes to the progression of acute liver injury after APAP overdose.[2 –4] Although not associated with clinically significant bleeding[5], substantial alterations in the hemostatic system are evident in patients with AAP-induced liver failure, including a reduced platelet count.
Source: Journal of Hepatology - Category: Gastroenterology Authors: Source Type: research
We present a case of a 68-year-old Hispanic female with a history of factor VII deficiency who presented with shortness of breath, chest pain, and palpitations and was found to have pulmonary embolism. Our patient did not have any of the above-mentioned thrombotic risk factors. Our case and review of the literature show that factor VII deficiency does not provide protection against thrombosis. PMID: 31590173 [PubMed - as supplied by publisher]
Source: Acta Haematologica - Category: Hematology Authors: Tags: Acta Haematol Source Type: research
Abstract Platelet aggregate formation is a multistep process involving receptor-mediated, as well as biomechanical signaling cascades, which are highly dependent on actin dynamics. We have previously shown that actin depolymerizing factor (ADF)/n-cofilin and Twinfilin 2a, members of the ADF homology (ADF-H) protein family, have distinct roles in platelet formation and function. Coactosin-like 1 (Cotl1) is another ADF-H protein that binds actin and was additionally shown to enhance biosynthesis of pro-inflammatory leukotrienes (LTs) in granulocytes. Here, we generated mice lacking Cotl1 in the megakaryocyte lineage...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
We present the case of a 61-year-old woman who was misdiagnosed as immune thrombocytopenic purpura during her three pregnancies resulting in a delayed diagnosis of VWD type 2B. This genetically confirmed diagnosis resulted in testing and the establishment of the diagnosis in her otherwise asymptomatic adult son as well. VWD may not be diagnosed till beyond mid adulthood in women with thrombocytopenia previously attributed to pregnancy and should be considered as a differential in female patients developing thrombocytopenia less than 100 × 103/μl with an increased bleeding assessment tool score.
Source: Blood Coagulation and Fibrinolysis - Category: Hematology Tags: CASE REPORTS Source Type: research
Conclusions: Acquired von Willebrand syndrome is a common but under recognized disorder in pediatric extracorporeal membrane oxygenation patients. The clinical implications of this disorder on bleeding and its potential treatments are still unclear.
Source: Pediatric Critical Care Medicine - Category: Pediatrics Tags: Extracorporeal Support Source Type: research
Thrombosis is the major cause of morbidity and mortality in patients with polycythemia vera (PV). Aspirin, and phlebotomy with or without cytoreductive therapy are key components of PV management to reduce thrombotic risk. However, patients with PV are also at risk for hemorrhagic complications, especially in the context of thrombocytosis and acquired Von Willebrand factor deficiency. The net benefit of antithrombotic therapy for primary and secondary thromboprophylaxis is uncertain. A systematic review conducted in 2013 identified 2 randomized control trials (RCTs) and concluded that low-dose aspirin does not increase the...
Source: Thrombosis Research - Category: Hematology Authors: Source Type: research
The etiology of bleeding during cardiac surgery is multifactorial and includes both surgical and hematologic causes.1 When evaluating a patient with a known bleeding disorder for cardiac surgery, it is important to understand the mechanism that prevents the formation of adequate clotting. In the case of von Willebrand disease (vWD), there is either a quantitative or qualitative deficiency of a multimeric glycoprotein called von Willebrand factor (vWF). This factor is essential for hemostasis after a vascular injury and supports the interaction between platelets and platelet-vessel surfaces.
Source: Journal of Cardiothoracic and Vascular Anesthesia - Category: Anesthesiology Authors: Tags: Review Article Source Type: research
CONCLUSION: This population PK model derived from real world data adequately describes FVIII levels following perioperative administration of the FVIII/VWF plasma-derived concentrate (Haemate® P/Humate P®) and will help to facilitate future dosing in VWD patients. PMID: 31557387 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
CONCLUSIONS: Our results provide HLAcII peptidomic level explanations for several important clinical observations/issues including the differential immunogenicity of distinct FVIIIs and the role of HLAcII genetics in inhibitor development. PMID: 31556206 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
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