Results 41 to 50 of about 1,126 (138)

Current State of Moderate Congenital Haemophilia A and the Need for Preventive Treatment

Haemophilia, EarlyView.
ABSTRACT Introduction The severity of haemophilia A is classified by the degree of factor VIII (FVIII) deficiency, rather than by clinical manifestations. However, FVIII activity alone does not necessarily accurately reflect clinical severity such as bleeding tendency, and patients with mild‐to‐moderate haemophilia A can experience significant disease ...
Masahiro Takeyama, Atsuki Yamashita, Tomoko Yamaguchi, Takahiro Ito, Yui Sugawara, Akiko Ioka, Tazuko Tokugawa   +6 more
wiley   +1 more source

Application of systems biology to identify pharmacological mechanisms of thrombotic microangiopathy evoked by combined activated prothrombin complex concentrate and emicizumab

Scientific Reports, 2023
Emicizumab is a bispecific monoclonal antibody that substitutes for the function of missing or deficient factor VIII (FVIII) in people with hemophilia A (PwHA). Long-term safety and efficacy of emicizumab have been demonstrated in several clinical trials.
Raquel Valls, Jonathan Wagg, Ido Paz-Priel, Gabriel Man, Laura Artigas, Georges Jaccard, Mireia Coma, Christophe Schmitt   +7 more
doaj   +1 more source

Emicizumab for acquired haemophilia A: A case series.

, 2023
BACKGROUND: Emicizumab is approved to prevent bleeding in patients with congenital haemophilia A with or without inhibitors. However, no randomized trials addressed the efficacy of emicizumab in acquired haemophilia A (AHA).
Kathelijne Peerlinck, Vandesande, Johan, Van Laer, Christine, Jacquemin, Marc, Hermans, Cédric, Matthias M. Engelen, Verhamme, Peter, Vanassche, Thomas, Johan De Bent, Engelen, Matthias M., Veerle Labarque, Marc Jacquemin, Cedric Hermans, Johan Vandesande, Peerlinck, Kathelijne, Christine Van Laer, De Bent, Johan, Labarque, Veerle, Peter Verhamme, Engelen, Matthias M, Thomas Vanassche   +20 more
core   +1 more source

The XX Factor in Hemophilia: Diagnostic, Therapeutic, and Research Gaps for Women and Girls

Haemophilia, EarlyView.
ABSTRACT Introduction Hemophilia has traditionally been viewed as an X‐linked disorder affecting men and boys, with women and girls labeled as “carriers”, presumed to be clinically unaffected. This paradigm has contributed to under‐recognition, delayed diagnosis, and undertreatment of females with hemophilia‐associated genotypes despite an increasingly
Kelsey Uminski, Ellen Cusano
wiley   +1 more source

From Bleeding Control to Health: Implementing Multidimensional and Multidisciplinary Hemophilia Care Into the Era of Therapeutic Innovation

Haemophilia, EarlyView.
ABSTRACT Introduction Over the past decade, hemophilia management has been transformed by modern prophylactic strategies, achieving dramatic reductions in annualized bleeding rates (ABR), often approaching zero. However, the conceptual evolution of hemophilia care toward multidimensional health assessment and comprehensive multidisciplinary management ...
Nicolas Giraud, Yesim Dargaud
wiley   +1 more source

A hemophilia A mouse model for the in vivo assessment of emicizumab function

, 2020
International audienceThe bispecific antibody emicizumab is increasingly used for hemophilia A treatment. However, its specificity for human factors IX and X (FIX and FX) has limited its in vivo functional analysis to primate models of acquired ...
Kawecki, Charlotte, Peyron, Ivan, Kauskot, Alexandre, Lenting, Peter, J., Ferrière, Stephen, Muczynski, Vincent, Christophe, Olivier, D., Nathwani, Amit, Casari, Caterina, Denis, Cecile   +9 more
core   +1 more source

Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis

BMJ Open
Objectives Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how ...
Randall Curtis, Thomas Majda, Janet S. Lee, Stacey L. Kowal   +3 more
doaj   +1 more source

Optimization and evaluation of a two-stage chromogenic assay procedure for measurement of emicizumab plasma levels.

PLoS ONE, 2022
Emicizumab mimics the hemostatic activity of activated factor VIII (FVIIIa) within the tenase complex. Despite functional similarities between FVIIIa and emicizumab, conventional laboratory methods designed for monitoring of FVIII activity are ...
Nasim Shahidi Hamedani, Johannes Oldenburg, Bernd Pötzsch, Jens Müller   +3 more
doaj   +1 more source

Population‐Level Real‐World Healthcare Costs in Men and Women With Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Even with modern prophylaxis options, people living with haemophilia have significantly lower quality of life and joint health outcomes than those without. Aim Our goal was to determine if real‐world health‐system costs are higher for people living with haemophilia A compared to those without and determine how joint‐health state ...
Sam Hirniak, Samantha S. M. Drover, Andrea N. Edginton, Dagmar M. Hajducek, Alfonso Iorio, Mhd. Wasem Alsabbagh, William W. L. Wong   +6 more
wiley   +1 more source

Real-World Data on Bleeding Patterns of Hemophilia A Patients Treated with Emicizumab

, 2021
Emicizumab (Hemlibra™) is approved for prophylaxis of hemophilia A (HA) patients. The HAVEN studies addressed bleeding reduction in emicizumab-treated patients, but real-world data on bleeding patterns during emicizumab therapy are lacking.
Ivan Budnik, Einat Avishai, Mudi Misgav, Sarina Levy-Mendelovich, Tami Brutman-Barazani, Gili Kenet, Tamara Levy, Tami Livnat, Assaf A. Barg   +8 more
core   +1 more source