LIFE-ACTIVE: observational study evaluating the physical activity in a subset of damoctocog alfa pegol treated Hemophilia A patients who are enrolled in the HEM-POWR study
Disclosure of conflict of interest Emilio Musi, Bayer employee. Martin Sanabria, Bayer employee. María Teresa Alvarez Román, Receipt of grants/research support: Shire/Takeda. Company sponsored speaker’s bureau: Amgen, Bayer, CSL Behring, Novartis, Novo Nordisk, Roche,...
Baseline Patient Characteristics in ReITIrate – A Prospective Study of Rescue ITI with Recombinant Factor VIIIFc (rFVIIIFc) in Patients who Have Failed Previous ITI Attempts
Background Development of neutralising anti-factor VIII (FVIII) alloantibodies (inhibitors) is the most serious complication in patients with haemophilia A, affecting approximately 25–30% of patients with severe haemophilia A.1,2 Immune tolerance induction (ITI),...
MOTIVATE: MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A – An international observational study
Background Immune tolerance induction (ITI) is the only clinically proven strategy for eradication of anti-factor VIII (FVIII) alloantibodies (inhibitors) and is recommended as the primary treatment option in European and US guidelines1-3 The bispecific FIX/FX...
Observational immune tolerance induction study (ObsITI): Immune tolerance induction with a single factor VIII/von Willebrand factor concentrate for treatment of haemophilia A patients with inhibitors
Introduction Neutralising antibodies against exogenous factor VIII (FVIII) remain the most significant treatment complication in approximately one-third of previously untreated patients (PUPs)1,2 Development of a FVIII inhibitor reduces the effectiveness of bleeding...
Structural Evidence of an Anti-Factor C1 Domain Antibody Bound to B Domain-Deleted Factor VIII
Abstract The most common epitopes for neutralizing anti-factor VIII (fVIII) antibodies (inhibitors) are localized to the A2 and C2 domains, which are shared amongst both acquired hemophilia A and congenital hemophilia A patients. These inhibitors commonly interfere...
Transplantation of fetal liver cells into newborn hemophilic mice for the treatment of hemophilia A without inhibitors formation
BACKGROUND Hemophilia A cell therapy approaches in paediatric individuals require suitable FVIII-producing cell populations presenting stable engraftment potential. We previously showed that adult-derived FVIII-producing cells, i.e. liver sinusoidal endothelial cells...
Current view on the safety of PEGylated biologics in hemophilia treatment
Disclosure of conflict of interest The author is an employee of Bayer. Acknowledgements Medical writing support was provided by Shakirah Chowdhury of Darwin Healthcare.Background The addition of polyethylene glycol (PEG) to molecules (PEGylation) is a valuable...
Type 1 VWD caused by a novel dominant p.Thr274Pro mutation localized in VWF propeptide
Introduction von Willebrand factor (VWF) is a large multimeric glycoprotein that is essential for platelet-dependent primary haemostasis1. VWF propeptide (VWFpp) has an important role in the intracellular processing of VWF. Indeed, it takes part in the multimerization...
Evaluation of the von Willebrand factor (VWF) inhibitor in a large cohort of European and Iranian patients previously diagnosed with type 3 von Willebrand disease (VWD) enrolled into the 3WINTER-IPS Project
Background Type 3 VWD (VWD3) is inherited with an autosomal recessive pattern and is the least common form of VWD in developed countries (affecting 1 in 1 million people). VWD3 is characterized by virtually undetectable levels (<5 IU/dL) of VWF in plasma and very...
Evaluation of platelet-dependent VWF activity on a heterogeneous group of von Willebrand disease patients using four different methods: two VWF:GPIbM and two VWF:RCo assays
INTRODUCTION In the last few years alternative assays to evaluate platelet-dependent VWF activity have emerged as alternative to VWF:RCo assay. This test suffers of high inter-assay variation [1] and has some limitations as the demonstrated artefact with specific...
The presence of inhibitors against von Willebrand factor and factor VIII in the same patient: a case report
BACKGROUND/AIMS: The development of an inhibitor against von Willebrand factor (VWF) is a rare but serious treatment complication of von Willebrand disease (VWD). It has been reported that some VWF antibodies additionally inhibit factor VIII coagulation activity...