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https://www.readbyqxmd.com/read/28345299/a-prospective-study-of-health-related-quality-of-life-of-boys-with-severe-haemophilia-a-in-china-comparing-on-demand-to-prophylaxis-treatment
#1
R Wu, J Sun, J Xiao, Y Liu, F Xue, H Wang, L Tang, Y Zhao, K Li, R Yang, Y Hu, K-H Luke, M-C Poon, V S Blanchette, K Usuba, N L Young
INTRODUCTION: Treatment for boys with haemophilia in China is rapidly improving; however, comprehensive outcomes have not been examined prospectively. AIM: The aim of this study was to evaluate the effect of short-term full-dose prophylaxis compared to on-demand treatment, on the Health-Related Quality of Life (HR-QoL) of boys with severe haemophilia A (HA) in China. METHODS: Boys with severe HA (FVIII<1%) completed 3 months of on-demand treatment and 3 months of full-dose prophylaxis (25 FVIII IU per kg 3x per week)...
March 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28340620/stable-high-level-expression-of-factor-viii-in-chinese-hamster-ovary-cells-in-improved-elongation-factor-1-alpha-based-system
#2
Nadezhda A Orlova, Sergey V Kovnir, Alexandre G Gabibov, Ivan I Vorobiev
BACKGROUND: Recombinant factor VIII (FVIII), used for haemophilia A therapy, is one of the most challenging among the therapeutic proteins produced in heterologous expression systems. Deletion variant of FVIII, in which the entire domain B is replaced by a short linker peptide, was approved for medical use. Efficacy and safety of this FVIII deletion variant are similar to full-length FVIII preparations while the level of production in CHO cells is substantially higher. Typical levels of productivity for CHO cell lines producing deletion variant FVIII-BDD SQ, described elsewhere, are 0...
March 24, 2017: BMC Biotechnology
https://www.readbyqxmd.com/read/28335488/italian-registry-of-congenital-bleeding-disorders
#3
Adele Giampaolo, Francesca Abbonizio, Romano Arcieri, Hamisa Jane Hassan
In Italy, the surveillance of people with bleeding disorders is based on the National Registry of Congenital Coagulopathies (NRCC) managed by the Italian National Institute of Health (Istituto Superiore di Sanità). The NRCC collects epidemiological and therapeutic data from the 54 Hemophilia Treatment Centers, members of the Italian Association of Hemophilia Centres (AICE). The number of people identified with bleeding disorders has increased over the years, with the number rising from approx. 7000 in 2000 to over 11,000 in 2015...
March 19, 2017: Journal of Clinical Medicine
https://www.readbyqxmd.com/read/28327546/missense-mutations-near-the-n-glycosylation-site-of-the-a2-domain-lead-to-various-intracellular-trafficking-defects-in-coagulation-factor-viii
#4
Wei Wei, Chunlei Zheng, Min Zhu, Xiaofan Zhu, Renchi Yang, Saurav Misra, Bin Zhang
Missense mutation is the most common mutation type in hemophilia. However, the majority of missense mutations remain uncharacterized. Here we characterize how hemophilia mutations near the unused N-glycosylation site of the A2 domain (N582) of FVIII affect protein conformation and intracellular trafficking. N582 is located in the middle of a short 310-helical turn (D580-S584), in which most amino acids have multiple hemophilia mutations. All 14 missense mutations found in this 310-helix reduced secretion levels of the A2 domain and full-length FVIII...
March 22, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28306626/pregnancy-and-delivery-in-a-woman-with-severe-haemophilia-a
#5
Jelena N Bodrozic, Predrag S Miljic, Danijela R Lekovic, Milos A Petronijevic, Darko A Antic, Mirjana M Mitrovic, Svetlana M Petronijevic-Vrzic, Irena S Djunic
Severe form of haemophilia in women is an extremely rare condition. Owing to the rarity of the disease there are no precise recommendations concerning the optimal management of pregnancy and delivery in these patients. We are reporting the clinical course and management of a 30-year-old woman with a severe form of haemophilia A (factor VIII <1 IU/dl) during her first pregnancy and delivery. Antepartum, she was treated on demand by FVIII concentrate and she delivered at 37 weeks of gestation by cesarean section...
March 16, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28306186/sippet-methodology-analysis-and-generalizability
#6
REVIEW
F Peyvandi, P M Mannucci, R Palla, F R Rosendaal
The development of anti-FVIII neutralizing alloantibodies (inhibitors), occurring in about one-third of previously untreated patients (PUPs) with severe haemophilia A, depends on various genetic and environmental risk factors. Several previous studies have reported on the immunogenicity of FVIII concentrates, and due to differences in study design, study period, inhibitor testing frequency and follow-up duration the results were inconclusive. The first randomized trial on this unresolved question (SIPPET) included 251 previously untreated or minimally treated patients with severe haemophilia A treated with either a single plasma-derived FVIII (pdFVIII) containing VWF or a recombinant FVIII (rFVIII)...
March 17, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28299631/clinical-characteristics-and-outcomes-of-acquired-hemophilia-a-experience-at-a-single-center-in-japan
#7
Yoshiyuki Ogawa, Kunio Yanagisawa, Hideki Uchiumi, Takuma Ishizaki, Takeki Mitsui, Fumito Gouda, Masahiro Ieko, Akitada Ichinose, Yoshihisa Nojima, Hiroshi Handa
Acquired hemophilia A (AHA), which is caused by autoantibodies against coagulation factor VIII (FVIII) is a rare, life-threatening bleeding disorder, the incidence of which appears to be increasing in Japan as the population ages. However, the clinical characteristics, treatment, and outcomes of AHA remain difficult to establish due to the rarity of this disease. We retrospectively analyzed data from 25 patients (median age 73 years; range 24-92 years; male n = 15) diagnosed with AHA between 1999 and 2015 at Gunma University Hospital...
March 15, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28296129/comparing-thrombin-generation-in-patients-with-hemophilia-a-and-patients-on-vitamin-k-antagonists
#8
M L Y de Koning, K Fischer, B de Laat, A Huisman, M Ninivaggi, R E G Schutgens
BACKGROUND: It is unknown whether patients with hemophilia A with atrial fibrillation require treatment with vitamin K antagonists (VKA) to the same extent as the normal population. OBJECTIVE: To compare hemostatic potential in hemophilia patients and patients on VKA using thrombin generation (TG). METHODS: In this cross-sectional study, TG, initiated with 1pM tissue factor, was measured in 133 patients with severe (FVIII <1%, n=15) and non-severe (FVIII 1-50%, n=118) hemophilia A, 97 patients on VKA with an international normalized ratio (INR) ≥1...
March 15, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28279252/calibration-of-the-ph-eur-human-coagulation-factor-viii-concentrate-brp-batch-5
#9
S Raut, A Costanzo, A Daas, K H Buchheit
The European Pharmacopoeia Biological Reference Preparation (Ph. Eur. BRP) for Factor VIII Concentrate batch 5 was established through a collaborative study involving 14 laboratories organised by the European Directorate for the Quality of Medicines & HealthCare (EDQM, Council of Europe) to be used as working standard for potency determination of human coagulation Factor VIII (FVIII) preparations. The potency of the BRP batch 5 was assigned with reference to the WHO 8(th) International Standard (IS) for FVIII Concentrate and the BRP batch 4...
2017: Pharmeuropa Bio & Scientific Notes
https://www.readbyqxmd.com/read/28277160/production-of-recombinant-coagulation-factors-are-humans-the-best-host-cells
#10
Kamilla Swiech, Virgínia Picanço-Castro, Dimas Tadeu Covas
The main treatment option for Hemophilia A/B patients involves the administration of recombinant coagulation factors on-demand or in a prophylactic approach. Despite the safety and efficacy of this replacement therapy, the development of antibodies against the coagulation factor infused, which neutralize the procoagulant activity, is a severe complication. The production of recombinant coagulation factors in human cell lines is an efficient approach to avoid such complication. Human cell lines can produce recombinant proteins with post translation modifications more similar to their natural counterpart, reducing potential immunogenic reactions...
February 23, 2017: Bioengineered
https://www.readbyqxmd.com/read/28264199/factor-activity-assays-for-monitoring-extended-half-life-fviii-and-factor-ix-replacement-therapies
#11
Steve Kitchen, Stefan Tiefenbacher, Robert Gosselin
The advent of modified factor VIII (FVIII) and factor IX (FIX) molecules with extended half-lives (EHLs) compared with native FVIII and FIX represents a major advance in the field of hemophilia care, with the potential to reduce the frequency of prophylactic injections and/or to increase the trough level prior to subsequent injections. Monitoring treatment through laboratory assays will be an important part of ensuring patient safety, including any tailoring of prophylaxis. Several approaches have been used to extend half-lives, including PEGylation, and fusion to albumin or immunoglobulin...
April 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/28263027/coagulation-factors-anticoagulant-proteins-and-plasminogen-in-mexican-older-adults
#12
E Hernández Zamora, L O González-Espinosa, C Zavala-Hernández, E Rosales-Cruz, E Reyes-Maldonado
INTRODUCTION: Hemostasis protects upon the occurrence of vascular endothelial damage, with involving of different factors. The interaction of these factors in older adults is poorly known, and has been associated with different disorders. Therefore, we determined the activity of coagulation factors (CF), anticoagulant proteins (AP), and plasminogen (Plg), as well as the frequency of deficiencies of these proteins in a population of healthy Mexican older adults (OA). METHODS: CF (I, II, V, VII, VIII, IX, X, and XI y XII), AP [protein C (PC), protein S (PS), and antithrombin (AT)], and Plg were determined from 244 plasma samples of OA using commercial kits in a coagulometer ACL Elite Pro...
March 6, 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28262242/an-extra-x-does-not-prevent-acquired-hemophilia-pregnancy-associated-acquired-hemophilia-a
#13
Assaf A Barg, Tami Livnat, Gili Kenet
Acquired hemophilia A (AHA) is a severe bleeding disorder caused by autoantibodies against clotting factor VIII (FVIII). With an estimated annual incidence of 1.3 to 1.5 per million, AHA is a rare disease. An extremely rare form of AHA has been described among women in the peripartum period, and may present with peripartum hemorrhage. Notably, although hemorrhagic symptoms commonly present 1-4 months around delivery, they may occur up to 1 year after parturition. When caring for a mother with AHA it is important to note that Factor VIII inhibitor may be transferred via the placenta from the mother to the fetus...
March 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28246607/tissue-factor-pathway-inhibitor-1-is-a-valuable-marker-for-the-prediction-of-deep-venous-thrombosis-and-tumor-metastasis-in-patients-with-lung-cancer
#14
Xianming Fei, Huan Wang, Wufeng Yuan, Mingyi Wo, Lei Jiang
Activation of blood coagulation contributes to cancer progression. Tissue factor pathway inhibitor-1 (TFPI-1) is the main inhibitor of extrinsic coagulation pathway. The aim of this study is to assess the predicting significance of TFPI-1 for thrombotic complication and metastasis in lung cancer patients. Total of 188 non-small cell lung cancer (NSCLC) patients were included in this study. Plasma TFPI-1, D-dimer (D-D), antithrombin (AT), Fibrinogen (Fbg), and coagulating factor VIII activity (FVIII:C) were measured...
2017: BioMed Research International
https://www.readbyqxmd.com/read/28244200/population-pharmacokinetics-of-recombinant-coagulation-factor-viii-singlechain-in-patients-with-severe-hemophilia-a
#15
Y Zhang, J Roberts, M Tortorici, A Veldman, K St Ledger, A Feussner, J Sidhu
BACKGROUND: rVIII-SingleChain is a unique recombinant factor VIII (FVIII) molecule. OBJECTIVES: To (i) characterize the population pharmacokinetics (PK) of rVIII-SingleChain in severe hemophilia A patients, (ii) identify correlates of variability in rVIII-SingleChain PK, and (iii) simulate various dosing scenarios of rVIII-SingleChain. PATIENTS/METHODS: A population PK model was developed based on FVIII activity levels of 130 severe hemophilia A patients (n = 91 for ≥ 12 to 65 years; n = 39 for 1 to < 12 years) who had participated in a single-dose PK investigation with rVIII-SingleChain 50 IU kg(-1) ...
February 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28229158/detection-of-procoagulant-imbalance-modified-endogenous-thrombin-potential-with-results-expressed-as-ratio-of-values-with-to-without-thrombomodulin
#16
Armando Tripodi
Each individual possesses his/her own endogenous-thrombin-potential (ETP) (i. e. the ability to generate thrombin) which depends on the relative strength of the pro- and anticoagulant drivers operating in plasma. This ability depends in turn on the clinical conditions in which the balance between the two drivers is variably affected. One of the major determinants of this balance is the factor (F)VIII-protein C(PC) axis and its effect can be conveniently explored by the thrombin generation procedures with results expressed as ETP ratio with/without thrombomodulin (TM) (ETP-TM ratio)...
February 23, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28226363/coagulation-profile-in-patients-with-different-etiologies-for-cushing-syndrome-a-prospective-observational-study
#17
Amit Tirosh, Maya Lodish, Charalampos Lyssikatos, Elena Belyavskaya, Richard A Feelders, Constantine A Stratakis
Previous studies reported a higher prevalence of venous-thromboembolic events among patients with Cushing disease (CD) compared to those with ACTH-independent Cushing syndrome (CS) from adrenal sources. The objective of the current study was to evaluate the coagulation profile of patients with CS from different etiologies. A prospective observational study was conducted at a clinical research center. The study included adult patients admitted for evaluation of suspected CS (n=85), that were divided into 3 groups: CD (n=22), ACTH-independent CS from an adrenal tumor/hyperplasia (adrenal CS, n=21), and a control group consisting of subjects with negative screening for CS (rule-out CS, n=42)...
February 22, 2017: Hormone and Metabolic Research, Hormon- und Stoffwechselforschung, Hormones et Métabolisme
https://www.readbyqxmd.com/read/28220631/treatment-of-bleeding-episodes-with-recombinant-factor-viii-fc-fusion-protein-in-a-long-study-subjects-with-severe-haemophilia-a
#18
A D Shapiro, J N Mahlangu, D Perry, J Pasi, D V Quon, P Chowdary, E Tsao, S Li, A Innes, G F Pierce, G A Allen
INTRODUCTION: The Phase 3 A-LONG study demonstrated the safety and efficacy of rFVIIIFc for the control and prevention of bleeding episodes in severe haemophilia A. AIM: To describe the treatment of bleeding episodes with rFVIIIFc in the A-LONG study. METHODS: A-LONG subjects (<1 IU dL(-1) endogenous FVIII) were treated with individualized prophylaxis (Arm 1), weekly prophylaxis (Arm 2) or episodic treatment (Arm 3). Information recorded for each bleeding episode included type, location and dose to treat the episode...
February 20, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28220555/population-pharmacokinetic-characterization-of-bay-81-8973-a-full-length-recombinant-factor-viii-lessons-learned-importance-of-including-samples-with-factor-viii-levels-below-the-quantitation-limit
#19
D Garmann, S McLeay, A Shah, P Vis, M Maas Enriquez, B A Ploeger
INTRODUCTION: The pharmacokinetics (PK), safety and efficacy of BAY 81-8973, a full-length, unmodified, recombinant human factor VIII (FVIII), were evaluated in the LEOPOLD trials. AIM: The aim of this study was to develop a population PK model based on pooled data from the LEOPOLD trials and to investigate the importance of including samples with FVIII levels below the limit of quantitation (BLQ) to estimate half-life. METHODS: The analysis included 1535 PK observations (measured by the chromogenic assay) from 183 male patients with haemophilia A aged 1-61 years from the 3 LEOPOLD trials...
February 20, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28212975/budget-impact-analysis-of-prolonged-half-life-recombinant-fviii-therapy-for-hemophilia-in-the-united-states
#20
Suzanne McMullen, Brieana Buckley, Eric Hall, Jon Kendter, Karissa Johnston
BACKGROUND: Hemophilia A is a factor VIII deficiency, associated with spontaneous, recurrent bleeding episodes. This may lead to comorbidities such as arthropathy and joint replacement, which contribute to morbidity and increased health care expenditure. Recombinant factor VIII Fc fusion protein (rFVIIIFc), a prolonged half-life factor therapy, requires fewer infusions, resulting in reduced treatment burden. OBJECTIVE: Use a budget impact analysis to assess the potential economic impact of introducing rFVIIIFc to a formulary from the perspective of a private payer in the United States...
January 2017: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
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