Emicizumab prophylaxis in current haemophilia A care in the Czech Republic—data from the Czech National Haemophilia Programme Registry [PDF]
Background: Nonfactor therapy with emicizumab has become an important part of the haemophilia A treatment landscape recently. Objectives: We aimed to analyze data on the transition from previous treatment regimens to emicizumab in routine clinical ...
Ester Zápotocká +9 more
doaj +2 more sources
Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis [PDF]
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 +3 more
doaj +2 more sources
Emicizumab Associated Rhabdomyolysis in Hemophilia A [PDF]
Emicizumab is increasingly the front-line treatment for patients with Hemophilia A with or without inhibitors. Rhabdomyolysis is a syndrome of muscle necrosis and release of intracellular muscle constituents into the circulation.
Joseph A. Wilson +5 more
doaj +3 more sources
Lifetime Cost-Utility Analysis of Emicizumab Prophylaxis in Severe Haemophilia A Without Inhibitors in China: A Markov Model Analysis. [PDF]
ABSTRACT Aim As emicizumab shares no sequence homology with factor VIII (FVIII), in patients with haemophilia A, haemostasis can be restored irrespective of the presence of FVIII inhibitors. Emicizumab, therefore, substantially improved previously available prophylactic options.
Bao H +6 more
europepmc +2 more sources
Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis. [PDF]
ABSTRACT Introduction Haemophilia A in paediatric patients presents a lifelong risk of spontaneous and trauma‐induced haemorrhage, leading to progressive joint damage, disability and impaired quality of life. Emicizumab, a bispecific monoclonal antibody administered subcutaneously, offers sustained haemostatic protection and has shown promising ...
Bolou K +6 more
europepmc +2 more sources
Aim: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is a gene therapy evaluated in the phase 3 GENEr8-1 trial that provides endogenous factor VIII (FVIII) production to prevent bleeding in people with severe hemophilia A and represents an alternative to ...
W Kuwabara +9 more
doaj +1 more source
Massive Intra-Abdominal Pseudotumor in a Patient With Hemophilia A Treated With Emicizumab: A Case Report. [PDF]
ABSTRACT Hemophilic pseudotumors, a rare complication of bleeding disorders, are more common in settings with limited access to clotting factor concentrates. Subcutaneous emicizumab, a relatively novel therapy for hemophilia A, was investigated for its ability to stabilize an inoperable pseudotumor in a patient with factor VIII deficiency in an under ...
Kilach CJ +9 more
europepmc +2 more sources
Integration of Efanesoctocog Alfa in Clinical Practice for Children, Adolescents, and Young Adults With Severe Haemophilia A. [PDF]
ABSTRACT Introduction Efanesoctocog alfa is a novel, “ultra‐extended half‐life” FVIII concentrate for bleed treatment and prevention in haemophilia A. Clinical trials excluded individuals with active or prior FVIII inhibitors, those on emicizumab, and previously untreated patients (PUPs).
Jiang D +3 more
europepmc +2 more sources
Recombinant factor VIII (rFVIII), rFVIIIFc and emicizumab are established treatment options in the management of hemophilia A. Each has its unique mode of action, which can influence thrombin generation kinetics and therefore also the kinetics of ...
Thibaud Sefiane +9 more
doaj +1 more source
The effect of emicizumab and bypassing agents in patients with hemophilia – An in vitro study
Background Emicizumab is a nonfactor replacement therapy for hemophilia A (HA) and is a bispecific monoclonal antibody mimicking factor VIII by binding both factors IXa and X.
Nina Haagenrud Schultz +3 more
doaj +1 more source

