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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.
Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.
Haemophilia
November 2024
Hematology Laboratory, Reference Center for Inherited Platelet Disorders, Aix-Marseille Univ, APHM, INSERM, INRAe, C2VN, Marseille, France.
Background: Platelet transfusion is considered the standard treatment for preventing or controlling severe haemorrhage in Glanzmann thrombasthenia (GT). However, platelet transfusion can have detrimental effects, including the production of anti-GPIIb/IIIa isoantibodies or anti-HLA antibodies (Ab) and platelet transfusion refractoriness. Recombinant activated factor VII (rFVIIa) has been proposed as an alternative treatment to platelet transfusion.
View Article and Find Full Text PDFA Serendipitous Finding of Coagulation Factor VII Deficiency in Two Asymptomatic Patients.
Cureus
August 2024
Pediatrics and Child Health, Mohammed VIth University Hospital, Mohammed 1st University Faculty of Medicine and Pharmacy of Oujda, Oujda, MAR.
Article Synopsis
- - Congenital Factor VII deficiency is a rare disorder affecting blood clotting, with a prevalence of about 1 in 500,000 and is diagnosed through prolonged prothrombin time (PT).
- - It plays a vital role in the coagulation process by activating other factors, and management options include fresh frozen plasma, prothrombin complex concentrates, and the more preferred recombinant activated FVII due to its efficacy and safety.
- - The text presents two pediatric cases—one boy with a PT of 55% and FVII levels at 25.1%, and another with a PT at 24% and FVII levels at 4.6%—emphasizing the need for individualized diagnosis and treatment
Global Hemostatic Methods to Tailor Treatment With Bypassing Agents in Hemophilia A With Inhibitors- A Single-Center, Pilot Study.
Clin Appl Thromb Hemost
July 2024
Department of Molecular Medicine and Surgery, Karolinska Institutet, Stockholm, Sweden.
For patients with hemophilia A and high-titer inhibitors treated with bypassing agents there are no reliable methods to assess treatment effect. We investigated the utility of global hemostatic methods in assessing treatment with bypassing agents (rFVIIa or activated prothrombin complex [aPCC]). All patients with hemophilia A and inhibitors followed at the Coagulation Unit or the Pediatric Coagulation Unit at Karolinska University Hospital aged 6 years and above were eligible for this noninterventional study.
View Article and Find Full Text PDFDiagnosis and management of factor XI alloinhibitors in patients with congenital factor XI deficiency-A large single-centre experience.
Haemophilia
September 2024
Haemophilia Centre and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, UK.
Introduction: Factor (F) XI deficiency is an inherited bleeding disorder with increased prevalence in Ashkenazi Jews where it is mainly caused by two variants, p.Glu135* (type II, leading to a null allele) and p.Phe301Leu (type III, missense variant).
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