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https://www.readbyqxmd.com/read/29758126/abrogating-fibrinolysis-does-not-improve-bleeding-or-rfviia-rfviii-treatment-in-a-non-mucosal-venous-injury-model-in-haemophilic-rodents
#1
Rikke Stagaard, Matthew J Flick, Barbara Bojko, Krzysztof Goryński, Paulina Z Goryńska, Carsten D Ley, Lisbeth H Olsen, Tom Knudsen
BACKGROUND: Fibrinolysis may exacerbate bleeding in haemophilia A (HA). Accordingly, antifibrinolytics have been used to help maintain haemostatic control. While antifibrinolytic drugs have been proven effective in the treatment of mucosal bleeds in the oral cavity, their efficacy in non-mucosal tissues remain an open question of significant clinical interest. OBJECTIVE: To determine whether inhibiting fibrinolysis improves the outcome in non-mucosal haemophilic tail vein transection (TVT) bleeding models, and to determine whether a standard ex vivo clotting/fibrinolysis assay can be used as a predictive surrogate for in vivo efficacy...
May 14, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29732708/pharmacokinetic-modelling-and-validation-of-the-half-life-extension-needed-to-reduce-the-burden-of-infusions-compared-with-standard-factor-viii
#2
C Hermans, J Mahlangu, J Booth, H Schütz, E Santagostino, G Young, H-Y Lee, K N Steinitz-Trost, V Blanchette, E Berntorp
INTRODUCTION: Currently, no universally accepted definition of extended half-life (EHL) recombinant FVIII (rFVIII) exists. Identifying the minimum half-life extension ratio required for a reduction in dosing frequency compared with standard rFVIII could enable a more practical approach to decisions around prophylaxis with EHL rFVIII. AIM: To identify the half-life extension ratio required to decrease rFVIII dosing frequency by at least 1 day while maintaining the proportion of patients with plasma rFVIII levels above 1 IU/dL and without increasing the total weekly dose...
May 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29731369/tailored-frequency-escalated-primary-prophylaxis-for-severe-haemophilia-a-results-of-the-16-year-canadian-hemophilia-prophylaxis-study-longitudinal-cohort
#3
Brian M Feldman, Georges E Rivard, Paul Babyn, John K M Wu, MacGregor Steele, Man-Chiu Poon, Robert T Card, Sara J Israels, Nicole Laferriere, Kulwant Gill, Anthony K Chan, Manuel Carcao, Robert J Klaassen, Stephanie Cloutier, Victoria E Price, Saunya Dover, Victor S Blanchette
BACKGROUND: Severe haemophilia A has high morbidity, and treatment, while effective, is very expensive. We report the 16-year follow-up of the Canadian Hemophilia Prophylaxis Study, which examined the effectiveness of tailored frequency-escalated primary prophylaxis with a focus on health outcomes within the domains of body structures and functions, and activities and participation (according to the WHO International Classification of Functioning, Disability and Health [WHO-ICF] framework) and a view to reducing consumption of costly clotting factor, which accounts for more than 90% of the cost of care of severe haemophilia...
May 3, 2018: Lancet Haematology
https://www.readbyqxmd.com/read/29726070/a-comparative-analysis-of-heterogeneity-in-commercially-available-recombinant-factor-viii-products
#4
D Baunsgaard, A D Nielsen, P F Nielsen, A Henriksen, A K Kristensen, H W Bagger, M Ezban
INTRODUCTION: Advances in analytical technologies enable investigation of possible correlations between molecular structure, aggregation and subvisible particle content. Regulatory agencies place increasing attention on potential risks associated with protein aggregates in the micron range in biological therapeutics. AIM: Assess the heterogeneity, high-molecular-weight protein (HMWP) species, subvisible particle content and posttranslational modifications in six commercially available recombinant FVIII (rFVIII) products...
May 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29681200/cost-analysis-of-plasma-derived-factor-viii-von-willebrand-factor-vs-recombinant-factor-viii-for-treatment-of-previously-untreated-patients-with-severe-hemophilia-a-in-the-united-states
#5
Ellis J Neufeld, Robert F Sidonio, Ken O'Day, M Chris Runken, Kellie Meyer, Jeffrey Spears
BACKGROUND: Inhibitor development to factor VIII (FVIII) hemophilia therapy results in increased complications and substantial economic costs. The SIPPET study, the first randomized controlled trial to compare the immunogenicity of plasma-derived FVIII (pdFVIII)/von Willebrand factor (VWF) and recombinant-DNA-derived FVIII (rFVIII), demonstrated higher inhibitor rates in previously untreated patients (PUPs) treated with rFVIII than in PUPs treated with pdFVIII/VWF. OBJECTIVE: To quantify the economic impact of treating PUPs with pdFVIII/VWF versus rFVIII...
April 21, 2018: Journal of Medical Economics
https://www.readbyqxmd.com/read/29665242/comparative-analysis-of-marketed-factor-viii-products-recombinant-products-are-not-alike-vis-a-vis-soluble-protein-aggregates-and-subvisible-particles
#6
J Anzengruber, C Lubich, T Prenninger, A Gringeri, F Scheiflinger, B M Reipert, M Malisauskas
BACKGROUND: Recombinant protein technologies have facilitated the development of novel FVIII therapeutics with improved production efficiency, potency, half-live, and low risk of viral transmission. The increasing number of recombinant Factor VIII (rFVIII) products and information on their efficacy, safety, and cost allow patients and healthcare professionals to adjust treatment to individual needs. Nonetheless, 20-32% of previously untreated patients with severe hemophilia A develop inhibitory antibodies to rFVIII following treatment...
April 17, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29611182/factor-f-viii-viia-enhances-global-haemostatic-function-in-the-co-presence-of-bypassing-agents-and-fviii-among-patients-with-haemophilia-a-with-inhibitor
#7
Keiji Nogami, Tomoko Matsumoto, Koji Yada, Kenichi Ogiwara, Shoko Furukawa, Yasuaki Shida, Masahiro Takeyama, Midori Shima
Bypassing therapy is essential for the haemostatic management of patients with haemophilia A with inhibitor (PWHA-inh), but the therapeutic effects are inconsistent. We previously reported that activated prothrombin complex concentrates (aPCC) activated factor (F)VIIIin vitro, and was mediated mainly by the activated FVII (FVIIa) contained in aPCC. We have extended those studies to assess global coagulation in whole blood from 18 PWHA-inh in the co-presence of aPCC and FVIII using Ca2+ -triggered rotational thromboelastometry...
April 2, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29461004/effects-of-pre-analytical-heat-treatment-in-factor-viii-fviii-inhibitor-assays-on-fviii-antibody-levels
#8
B Boylan, C H Miller
INTRODUCTION: The use of pre-analytical heat treatment (PHT) with the Nijmegen-Bethesda assay (NBA) for inhibitors to factor VIII (FVIII) can remove/destroy infused or endogenous FVIII from patient plasma samples, allowing testing of recently infused patients with haemophilia. Two PHT methods have been described as follows: heating to 56°C for 30 minutes and heating to 58°C for 90 minutes. Data examining the effects of PHT on anti-FVIII IgG4 , the antibodies known to correlate most closely with the presence of FVIII inhibitors, are limited...
February 20, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29444872/fviii-proteins-with-a-modified-immunodominant-t-cell-epitope-exhibit-reduced-immunogenicity-and-normal-fviii-activity
#9
Ruth A Ettinger, Joseph A Liberman, Devi Gunasekera, Komal Puranik, Eddie A James, Arthur R Thompson, Kathleen P Pratt
Factor VIII (FVIII)-neutralizing antibodies (inhibitors) are a serious complication in hemophilia A (HA). The peptide FVIII2194-2213 contains an immunodominant HLA-DRA*01-DRB1*01:01 (DRB1*01:01)-restricted epitope recognized by CD4+ T-effector cells from HA subjects. The aim of this study was to identify amino acid substitutions to deimmunize this epitope while retaining procoagulant function and expression levels comparable to those of wild-type (WT) FVIII proteins. The shortest DRB1*01:01-binding peptide was FVIII2194-2205 , and residues important for affinity were identified as F2196, M2199, A2201, and S2204...
February 27, 2018: Blood Advances
https://www.readbyqxmd.com/read/29405496/clinical-use-of-recombinant-factor-viii-fc-and-recombinant-factor-ix-fc-in-patients-with-haemophilia-a-and-b
#10
C Wang, G Young
INTRODUCTION: Although clinical trials have demonstrated extended half-life (EHL) VIII and IX fusion proteins to be safe and efficacious in patients with haemophilia A and B, studies on real-world clinical application have not been performed. AIM: To retrospectively examine the real-world experience of rFVIII Fc and rFIX Fc in patients. METHODS: A retrospective review of existing medical records of patients with haemophilia A or haemophilia B who had been prescribed rFVIII Fc or rFIX Fc was conducted from the Children's Hospital Los Angeles Haemostasis and Thrombosis Centre database...
February 5, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29384900/a-case-report-of-acute-inferior-myocardial-infarction-in-a-patient-with-severe-hemophilia-a-after-recombinant-factor-viii-infusion
#11
Silva Zupančić-Šalek, Marijo Vodanović, Dražen Pulanić, Boško Skorić, Irina Matytsina, Jolanta Klovaite
RATIONALE: The extent of protective effects of hemophilia against thrombotic events such as myocardial infarction (MI) and other acute coronary syndromes remains to be determined, as major risk factors for cardiovascular disease exist despite factor VIII (FVIII) deficiency. We present a case report of a 41-year-old male with severe hemophilia A and several cardiovascular risk factors. PATIENT CONCERNS: This morbidly obese patient developed chest pressure, followed by chest pain and difficulty in breathing shortly after receiving on-demand treatment with intravenous recombinant FVIII (rFVIII) (turoctocog alfa) dosed per body weight...
December 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29369801/prediction-of-human-pharmacokinetics-of-activated-recombinant-factor-vii-and-b-domain-truncated-factor-viii-from-animal-population-pharmacokinetic-models-of-haemophilia
#12
Malte Selch Larsen, Rasmus Vestergaard Juul, Andreas Velsing Groth, Ulrika S H Simonsson, Annemarie T Kristensen, Tom Knudsen, Henrik Agersø, Mads Kreilgaard
Various experimental animal models are used in haemophilia research, however, little is known about how well the different species predict pharmacokinetic (PK) profiles in haemophilia patients. The aim of the current study was to describe the plasma concentration-time profile of recombinant activated factor VII (rFVIIa) and recombinant factor VIII (rFVIII) in several experimental animal models using population PK modelling, and apply a simulation-based approach to evaluate how well the developed animal population PK models predict human PK...
March 30, 2018: European Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/29274194/international-comparative-field-study-evaluating-the-assay-performance-of-afstyla-in-plasma-samples-at-clinical-hemostasis-laboratories
#13
K St Ledger, A Feussner, U Kalina, C Horn, H J Metzner, D Bensen-Kennedy, N Blackman, A Veldman, A Stowers, K D Friedman
Essentials AFSTYLA exhibits ≈50% underestimation in activity when the one-stage (OS) assay is utilized. A field study compared the performance of AFSTYLA with Advate in factor VIII activity assays. AFSTYLA activity can be monitored with both the chromogenic substrate and the OS assay. The consistent OS underestimation allows for a conversion factor to be applied to OS results. SUMMARY: Introduction AFSTYLA (antihemophilic factor [recombinant] single chain) is a novel B-domain truncated recombinant factor VIII (rFVIII)...
March 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29139314/cost-utility-analysis-of-life-long-prophylaxis-with-recombinant-factor-viiifc-vs-recombinant-factor-viii-for-the-management-of-severe-hemophilia-a-in-sweden
#14
Nathaniel Henry, Jelena Jovanović, Max Schlueter, Persefoni Kritikou, Koo Wilson, Karl-Johan Myrén
AIMS: Prophylaxis with recombinant factor VIII (rFVIII) is the standard of care for severe hemophilia A in Sweden. The need for frequent injections with existing rFVIII products may, however, result in poor adherence to prophylaxis, leading to increased bleeding and long-term joint damage. Recombinant FVIIIFc (rFVIIIFc) is an extended half-life fusion protein which can offer prolonged protection and reduced dosing frequency. The objective of this study was to evaluate the cost-utility of prophylaxis with rFVIIIFc in severe hemophilia A from the perspective of the Swedish health system...
April 2018: Journal of Medical Economics
https://www.readbyqxmd.com/read/29080391/timing-and-severity-of-inhibitor-development-in-recombinant-versus-plasma-derived-factor-viii-concentrates-a-sippet-analysis
#15
F Peyvandi, A Cannavò, I Garagiola, R Palla, P M Mannucci, F R Rosendaal
Essentials Recombinant factor VIII (rFVIII) was contrasted with plasma-derived FVIII (pdFVIII). In previously untreated patients with hemophilia A, rFVIII led to more inhibitors than pdFVIII. Inhibitors with rFVIII developed earlier, and the peak rate was higher than with pdFVIII. Inhibitors with rFVIII were more severe (higher titre) than with pdFVIII. SUMMARY: Background The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe hemophilia A...
January 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29080389/sippet-insights-into-factor-viii-immunogenicity
#16
P G Fallon, M Lavin, J S O'Donnell
No abstract text is available yet for this article.
January 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29050497/simoctocog-alfa-for-the-treatment-of-hemophilia-a
#17
REVIEW
Massimo Morfini
Hemophilia A is the most frequent inherited bleeding disorder and most challenging coagulation disorder. To combat this, a number of new improved rFVIII/IX concentrates have recently been approved. Some of them are derived from protein fusion biotechnology or pegylation to extend their half-life (HL). However, prophylaxis has become a standard of care to prevent arthropathy in hemophiliacs though the need of frequent venipunctures is a major obstacle to primary prophylaxis. The new Extended Half-Life (EHL) rFIX concentrates allow increased intervals, while the improved HL of new rFVIII was moderate...
December 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28921438/purification-methods-for-recombinant-factor-viii-expressed-in-human-liver-sk-hep-cells
#18
Vladimir Granovski, Mario Soares Abreu-Neto, Dimas Tadeu Covas
Coagulation factor VIII is one of the largest proteins attempted to be expressed in recombinant form. A very complex and labile protein which has a very short half-live and need a fast and efficient purification chain. Here, we describe a simple purification sequence using multimodal Capto MMC, affinity FVIII select and ion exchange SP-Fastflow chromatography steps without subjecting the target molecule to mechanical and temperature stress, separating impurities from rFVIII using net charge, hydrophobicity, and affinity of the molecules...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28921428/production-of-recombinant-factor-viii-in-human-cell-lines
#19
Daianne Maciely Carvalho Fantacini, Virgínia Picanço-Castro
Human cell lines can produce recombinant proteins much more similar to their natural counterpart, compared to other mammalian cell lines, reducing potential immunogenic reactions. Recombinant proteins produced in nonhuman cells can have in its structure glycan epitopes, such as Galα1,3-Gal (alpha-Gal) and N-glycolylneuraminic acid (Neu5Gc) residues, that are antigenic to humans and can potentially affect the efficacy of the recombinant product. Therefore, the production of recombinant factor VIII (rFVIII) in human cell lines is a new approach to avoid nonhuman glycosylation...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28836341/effect-of-late-prophylaxis-in-hemophilia-on-joint-status-a-randomized-trial
#20
M J Manco-Johnson, B Lundin, S Funk, C Peterfy, D Raunig, M Werk, C L Kempton, M T Reding, S Goranov, L Gercheva, L Rusen, V Uscatescu, M Pierdominici, S Engelen, J Pocoski, D Walker, W Hong
Essentials High-quality data are lacking on use of prophylaxis in adults with hemophilia and arthropathy. SPINART was a 3-year randomized clinical trial of late/tertiary prophylaxis vs on-demand therapy. Prophylaxis improved function, quality of life, activity and pain but not joint structure by MRI. Prophylaxis improves function but must start before joint bleeding onset to prevent arthropathy. SUMMARY: Background Limited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre-existing joint disease...
August 24, 2017: Journal of Thrombosis and Haemostasis: JTH
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