Djambas Khayat, Claudia Iosava, Genadi Romashevskaya, Irina Stasyshyn, Oleksandra Lopez, Marta Julia Pompa, Maria Teresa Rogosch, Tobias Seifert, Wilfried
Published in
Journal of Blood Medicine
Background Plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII; VONCENTO®, CSL Behring) is a high-concentration, low-volume, high-purity concentrate, with a high level of VWF high-molecular-weight multimers and a VWF/FVIII ratio of ~2.4:1. Methods This study (NCT01229007) investigated the pharmacokinetics (PK), efficacy and safety of pdVW...
Ronayne, Estelle K. Peters, Shaun C. Gish, Joseph S. Wilson, Celena Spencer, H. Trent Doering, Christopher B. Lollar, Pete Spiegel, P. Clint Jr Childers, Kenneth C.
Published in
Frontiers in Immunology
Factor VIII (fVIII) is a procoagulant protein that binds to activated factor IX (fIXa) on platelet surfaces to form the intrinsic tenase complex. Due to the high immunogenicity of fVIII, generation of antibody inhibitors is a common occurrence in patients during hemophilia A treatment and spontaneously occurs in acquired hemophilia A patients. Non-...
McGill, Joseph R. Simhadri, Vijaya L. Sauna, Zuben E.
Published in
Frontiers in Medicine
In hemophilia A (HA) patients, F8 gene-defects as genetic risk-factors for developing inhibitors to Factor VIII have been extensively studied. Here we provide estimates of inhibitor-risk associated with the patient's Human Leukocyte Antigen (HLA). We used next generation sequencing for high-resolution HLA Class II typing of 997 HA patients. Using i...
Russell, Athena L. Prince, Chengyu Lundgren, Taran S. Knight, Kristopher A. Denning, Gabriela Alexander, Jordan S. Zoine, Jaquelyn T. Spencer, H. Trent Chandrakasan, Shanmuganathan Doering, Christopher B.
...
Published in
Molecular Therapy. Methods & Clinical Development
Doering and colleagues develop a non-genotoxic conditioning regimen that permits successful long-term engraftment of hematopoietic stem and progenitor cells genetically modified with a high-expression factor VIII variant in hemophilia A mice. Durable factor VIII expression and hemostatic correction are achieved through non-genotoxic hematopoietic s...
Rohaert, Celine Labarque, Veerle
Published in
Haemophilia : the official journal of the World Federation of Hemophilia
Rohaert, Celine; Labarque, Veerle; 41869;
status: Published online
Escuriola-Ettingshausen, Carmen Auerswald, Günter Königs, Christoph Kurnik, Karin Scholz, Ute Klamroth, Robert Oldenburg, Johannes
Published in
Haemophilia : the official journal of the World Federation of Hemophilia
Standard treatment of haemophilia A is based on replacing the missing coagulation factor VIII (FVIII) to treat and prevent bleeding episodes. The most challenging complication of FVIII therapy is the development of neutralizing antibodies (inhibitors) that can render treatment ineffective. Eradication of the inhibitor through immune tolerance induc...
Oldenburg, Johannes Tran, Huyen Peyvandi, Flora Núñez, Ramiro Trask, Peter Chebon, Sammy Mahlangu, Johnny N Lehle, Michaela Jiménez-Yuste, Victor von Mackensen, Sylvia
...
Published in
Haemophilia : the official journal of the World Federation of Hemophilia
Real-world data on health-related outcomes in persons with haemophilia A (PwHA) can provide useful information for improving patient care. The global, non-interventional study (NIS; NCT02476942) prospectively collected high-quality data in PwHA, including those without factor VIII (FVIII) inhibitors treated according to local routine clinical pract...
Lelas, Antonela Greinix, Hildegard Theresia Wolff, Daniel Eissner, Günther Pavletic, Steven Zivko Pulanic, Drazen
Published in
Frontiers in Immunology
Chronic graft-versus-host disease (cGvHD) is an immune mediated late complication of allogeneic hematopoietic stem cell transplantation (alloHSCT). Discovery of adequate biomarkers could identify high-risk patients and provide an effective pre-emptive intervention or early modification of therapeutic strategy, thus reducing prevalence and severity ...
Poyant, Janelle O Gleason, Augustus M
Published in
Journal of pharmacy practice
Argatroban, a synthetic, parenteral, nonheparin anticoagulant, is a direct thrombin inhibitor indicated for the prophylaxis or treatment of venous thromboembolism (VTE) in patients with heparin-induced thrombocytopenia with thrombosis (HITT) and for use during percutaneous coronary intervention (PCI) in patients who have or are at risk for developi...
