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Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors.
Haemophilia
December 2024
Children's Hospital Los Angeles, Los Angeles, California, USA.
Introduction: Eptacog beta is a novel human recombinant FVIIa approved for use in the United States, European Union, United Kingdom and Mexico for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors (≥12 years). It is also indicated for perioperative care in the same patient population in Europe and the United Kingdom.
Aim: To assess the incidence of rebleeding and review treatment outcomes in subjects with haemophilia with inhibitors enrolled in the phase 3 PERSEPT 1 clinical trial.
Saliva of persons with hemophilia A triggers coagulation via extrinsic tenase complexes.
Blood
December 2024
Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
Article Synopsis
- Human saliva contains extracellular vesicles (EVs) that can activate blood coagulation through the presence of extrinsic tenase complexes, which include tissue factor and activated factor VII.
- Individuals with severe hemophilia A, who lack FVIII, have salivary EVs that can compensate for their condition by generating FXa, leading to infrequent oropharyngeal bleeding.
- In contrast, people with severe FVII deficiency lack these functional extrinsic tenase complexes in their saliva, resulting in more frequent oropharyngeal bleedings; however, adding recombinant FVIIa can restore coagulation potential in their saliva.
Article Synopsis
- - Infusion of von Willebrand factor (VWF) and factor VIII (FVIII) can lead to the development of anti-VWF antibodies, complicating management of bleeding in patients with type 3 von Willebrand disease.
- - A systematic review identified 15 cases where patient management varied, with treatments including continuous FVIII infusion, recombinant FVIIa, and other strategies.
- - Emicizumab, a bispecific monoclonal antibody, was used off-label in three cases and proved effective in managing a complex local case, highlighting its potential as a treatment option for patients with allo-immunisation.
Clinical, Laboratory, and Molecular Aspects of Factor VII Deficiency.
Semin Thromb Hemost
August 2024
Independent Scientist, London, United Kingdom.
Article Synopsis
- Factor VII deficiency is a blood condition that can cause various symptoms, from minor bleeding to serious issues like bleeding in the brain.
- The effectiveness of treatments like recombinant FVIIa is being improved with new methods that last longer and offer better care for patients.
- Researchers have studied many patients and found different changes in genes that affect FVII levels, helping us understand how this condition works better over the years.
Activated protein C resistance in the copresence of emicizumab and activated prothrombin complex concentrates.
Res Pract Thromb Haemost
May 2024
Department of Pediatrics, Nara Medical University, Kashihara, Nara, Japan.
Background: Venous thromboembolic events have been reported in persons with hemophilia A who received emicizumab and activated prothrombin complex concentrate (APCC) concomitantly, but the relevant mechanism(s) remains unclear. We speculated that activated protein C (APC) and antithrombin (AT) resistance might be associated with these adverse events.
Objectives: To investigate APC and AT resistance in factor (F)VIII-deficient (FVIIIdef) plasma in the presence of emicizumab and APCC.
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