Results 11 to 20 of about 13,061,190 (344)

A critical appraisal of one‐stage and chromogenic assays of factor VIII activity

Journal of Thrombosis and Haemostasis, 2016
F. Peyvandi, J. Oldenburg, K. Friedman
exaly   +2 more sources

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

New England Journal of Medicine, 2021
BACKGROUND The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.
Lindsey A. George, P. Monahan, M. Eyster, S. Sullivan, M. Ragni, S. Croteau, J. Rasko, M. Recht, Benjamin J. Samelson-Jones, A. Macdougall, Kristen Jaworski, R. Noble, M. Curran, K. Kuranda, F. Mingozzi, Tiffany Chang, K. Reape, X. Anguela, K. High   +18 more
semanticscholar   +1 more source

Acquired Hemophilia A: Bleeding Pattern and Hemostatic Therapeutic Strategies

Medicina, 2023
Acquired Hemophilia A (AHA) is a rare autoimmune disorder characterized by the onset of a sudden and unexpected bleeding episode in a patient with no personal or family history of bleeding diathesis, and with a typical laboratory feature, i.e., a ...
Renato Marino
doaj   +1 more source

Purified factor VIII [PDF]

BMJ, 1995
Patients with haemophilia A lack only factor VIII. Why give them anything else as replacement treatment? Previously the answer to this question was determined largely by the practical difficulty in separating factor VIII from fibrinogen, fibronectin, and immunoglobulins, so that concentrates contained 2000 U/mg respectively, but concentrates prepared ...
E G, Tuddenham, M, Laffan
openaire   +2 more sources

BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.

New England Journal of Medicine, 2020
BACKGROUND Factor VIII replacement products have improved the care of patients with hemophilia A, but the short half-life of these products affects the patients' quality of life. The half-life of recombinant factor VIII ranges from 15 to 19 hours because
B. Konkle, A. Shapiro, D. Quon, J. Staber, R. Kulkarni, M. Ragni, E. S. Chhabra, S. Poloskey, K. Rice, S. Katragadda, J. Fruebis, C. Benson   +11 more
semanticscholar   +1 more source

Recombinant porcine factor VIII corrects thrombin generation in vitro in plasma from patients with congenital hemophilia A and inhibitors

Research and Practice in Thrombosis and Haemostasis, 2022
Background Neutralizing factor VIII (FVIII) antibodies are a major complication in hemophilia A. Antihemophilic factor VIII (recombinant), porcine sequence (rpFVIII; susoctocog alfa; Baxalta US Inc., a Takeda company) has low cross‐reactivity to anti ...
Claude Négrier, Johannes Oldenburg, Gili Kenet, Shannon L. Meeks, Jean‐Claude Bordet, Jens Müller, Sandra Le Quellec, Peter L. Turecek, Nikola Tripkovic, Yesim Dargaud   +9 more
doaj   +1 more source

A Treg-Selective IL-2 Mutein Prevents the Formation of Factor VIII Inhibitors in Hemophilia Mice Treated With Factor VIII Gene Therapy

Frontiers in Immunology, 2020
Hemophilia A is a genetic disorder that results in the deficiency of functional factor VIII protein, which plays a key role in blood coagulation. Currently, the majority of hemophilia A patients are treated with repeated infusions of factor VIII protein.
Alex C. Chen, Xiaohe Cai, Chong Li, Liliane Khoryati, Marc A. Gavin, Carol H. Miao   +5 more
doaj   +1 more source

The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia

Pilot and Feasibility Studies, 2018
Background The interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There is a substantial need to improve capacity to collect and interpret relevant PRO data to support ...
MW. Skinner, C. Chai-Adisaksopha, R. Curtis, N. Frick, M. Nichol, D. Noone, B. O’Mahony, D. Page, J. S. Stonebraker, A. Iorio   +9 more
doaj   +1 more source

Prevention of the anti-factor VIII memory B-cell response by inhibition of Bruton tyrosine kinase in experimental hemophilia A

Haematologica, 2019
Hemophilia A is a rare hemorrhagic disorder caused by the lack of functional pro-coagulant factor VIII. Factor VIII replacement therapy in patients with severe hemophilia A results in the development of inhibitory anti-factor VIII IgG in up to 30% of ...
Sandrine Delignat, Jules Russick, Bagirath Gangadharan, Julie Rayes, Mathieu Ing, Jan Voorberg, Srinivas V. Kaveri, Sébastien Lacroix-Desmazes   +7 more
doaj   +1 more source

CLINICAL EFFICACY OF RECOMBINANT FACTOR VIII FC FUSION PROTEIN IN HAEMOPHILIA A PATIENT RECEIVING ON DEMAND TREATMENT ONLY

Pakistan Armed Forces Medical Journal, 2021
Objective: To evaluate the efficacy of recombinant factor VIII FC fusion protein in haemophilia A patient receiving on demand treatment only. Study Design: Comparative cross sectional study.
Saima Zahir, Tahira Zafar, Altaf Hussain, Hamid Saeed Malik, Pervez Ahmed, Saleem Ahmed Khan   +5 more
doaj   +1 more source