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https://www.readbyqxmd.com/read/28544263/impact-of-a-product-specific-reference-standard-for-the-measurement-of-a-pegylated-rfviii-activity-the-swiss-multicentre-field-study
#1
O Bulla, A Poncet, L Alberio, L M Asmis, A Gähler, L Graf, M Nagler, J-D Studt, D A Tsakiris, P Fontana
INTRODUCTION: Measuring factor VIII (FVIII) activity can be challenging when it has been modified, such as when FVIII is pegylated to increase its circulating half-life. Use of a product-specific reference standard may help avoid this issue. AIM: Evaluate the impact of using a product-specific reference standard for measuring the FVIII activity of BAX 855 - a pegylated FVIII - in eight of Switzerland's main laboratories. METHODS: Factor VIII-deficient plasma, spiked with five different concentrations of BAX 855, plus a control FVIII sample, was sent to the participating laboratories...
May 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28543946/predicting-dose-sparing-benefit-and-bleeding-risk-of-pharmacokinetic-based-personalized-prophylactic-dosing-of-factor-viii-products
#2
M A Tegenge, H Yang, R A Forshee
BACKGROUND: Previously published factor VIII (FVIII) pharmacokinetic (PK)-based dosing approaches employ fixed infusion interval with a wide dose range that may lead to increased risk of bleeding, excessive doses or decreased health-related quality of life. AIM: The objectives of the study includes (i) personalizing infusion interval in lieu of fixed infusion, (ii) constraining dose within the range of 10-50 IU/kg and (iii) characterizing bleeding risk of PK-based dosing in comparison with empiric standard doses...
May 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28532239/status-and-trend-analysis-of-prophylactic-usage-of-recombinant-factor-viii-in-chinese-pediatric-patients-with-hemophilia-a-recare-a-retrospective-phase-iv-non-interventional-study
#3
Changgang Li, Xinsheng Zhang, Yongqiang Zhao, Runhui Wu, Qun Hu, Vicky Xu, Jing Sun, Renchi Yang, Xiaojing Li, Rongfu Zhou, Shinmei Lian, Jian Gu, Junde Wu, Qingsong Hou
BACKGROUND: No study has reported the status and chronological trend of prophylactic recombinant factor VIII (rFVIII) use in Chinese pediatric patients with hemophilia A (HA). OBJECTIVE: We aimed to analyze the status and trend of rFVIII-contained prophylaxis in Chinese pediatric patients with HA. METHODS: ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained regular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers across China...
May 22, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28523993/acquired-hemophilia-as-initial-presentation-in-a-patient-with-systemic-lupus-erythematosus
#4
Zohre Khodamoradi, Mohammad Ali Nazarinia, Somaye Bazdar
BACKGROUND: Acquired hemophilia is a rare bleeding disease but may be associated with some autoimmune diseases. Acquired hemophilia may be the result of autoantibodies against factor VIII. CASE PRESENTATION: In this study, we describe a 55 year old patient who developed hematoma and hematuria due to acquired hemophilia secondary to systemic lupus erythematosus(SLE). Then, she developed arthritis and thrombosis after some evaluation. Laboratory data showed prolonged aPTT, normal PT and platelet, low FVIII, high FVIII inhibitor, ANA, anti-ds-DNA and anti-cardiolipin...
May 19, 2017: Current Rheumatology Reviews
https://www.readbyqxmd.com/read/28507083/frequency-and-epitope-specificity-of-anti-factor-viii-c1-domain-antibodies-in-acquired-and-congenital-hemophilia-a
#5
Joerg Kahle, Aleksander Orlowski, Diana Stichel, John F Healey, Ernest T Parker, Marc Jaquemin, Manuela Krause, Andreas Tiede, Dirk Schwabe, Pete Lollar, Christoph Königs
Several studies showed that neutralizing anti-factor VIII (fVIII) antibodies (inhibitors) in patients with acquired hemophilia A (AHA) and congenital hemophilia A (HA) are primarily directed to the A2 and C2 domains. In this study the frequency and epitope specificity of anti-C1 antibodies were analyzed in acquired and congenital hemophilia inhibitor patients (n=178). The domain specificity of antibodies was studied by homologue-scanning mutagenesis (HSM) with single human domain human/porcine fVIII proteins and antibody binding to human A2, C1 and C2 domains presented as human serum albumin (HSA) fusion proteins...
May 15, 2017: Blood
https://www.readbyqxmd.com/read/28492697/cd32-inhibition-and-high-dose-of-rhfviii-suppress-murine-fviii-specific-recall-response-by-distinct-mechanisms-in-vitro
#6
Nadine Vollack, Julia Friese, Sabine Bergmann, Andreas Tiede, Sonja Werwitzke
Development of neutralising antibodies (inhibitors) against factor VIII (FVIII) is a frequent and severe complication of replacement therapy in haemophilia A. Previous data from haemophilia A mouse model demonstrates that both CD32 inhibition and high doses of rhFVIII prevent the differentiation of FVIII-specific memory B cells (MBCs) into antibody secreting cells (ASCs). Here, cellular targets responsible for the suppression of ASC formation by means of CD32 inhibition and high dose of rhFVIII were analysed...
May 11, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28486277/evaluation-of-recombinant-factor-viii-fc-eloctate-activity-by-thromboelastometry-in-a-multicenter-phase-3-clinical-trial-and-correlation-with-bleeding-phenotype
#7
Frank Driessler, Maricel G Miguelino, Glenn F Pierce, Robert T Peters, Jurg M Sommer
: The aim of this study was to compare the hemostatic efficacy of recombinant factor VIII Fc (rFVIIIFc) (Eloctate) and Advate by ex-vivo rotation thromboelastometry (ROTEM) of whole blood and to explore potential ROTEM parameters that may be more predictive of a patient's bleeding tendency than plasma FVIII activity. Thirteen clinical sites were selected to perform ROTEM on freshly collected blood samples from 44 patients in the phase 3 study for rFVIIIFc, including 16 patients undergoing sequential pharmacokinetic assessment of Advate and rFVIIIFc...
May 8, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28481865/effect-of-hemodilution-in-vitro-with-hydroxyethyl-starch-on-hemostasis
#8
Shanliang Jin, Guifang Yu, Ruijiao Hou, Boxiong Shen, Hong Jiang
BACKGROUND Hydroxyethyl starch (HES) solutions are used for volume expansion during surgery. We aimed to investigate how 6%HES 130/0.4 affects hemostasis. MATERIAL AND METHODS Blood samples were collected from 12 healthy adult volunteers, diluting with 6%HES 130/0.4 (HES group) or Ringer lactate solution (RL control group). The hemodilution ratio (HR) of citrated blood volume to plasma substitute volume was 10: 0 (undiluted), 10: 2, 10: 4, and 10: 6. Clotting factors activity was measured. Thrombin generation was monitored...
May 8, 2017: Medical Science Monitor: International Medical Journal of Experimental and Clinical Research
https://www.readbyqxmd.com/read/28481034/impact-of-different-storage-times-at-room-temperature-of-unspun-citrated-blood-samples-on-routine-coagulation-tests-results-results-of-a-bicenter-study-and-review-of-the-literature
#9
P Toulon, S Metge, M Hangard, S Zwahlen, S Piaulenne, V Besson
INTRODUCTION: A maximum delay between blood collection and coagulation testing of 4 hours is recommended by most guidelines. As information on optimal storage times is limited, we investigated the potential effect of different storage times of unspun tubes, that is, ≤2, 4, 6, and 8 hours, on routine coagulation test results. METHODS: Four evacuated polymer tubes containing 0.109 mol/L tri-Na citrate were drawn from 144 patients, including 39 patients on vitamin K-antagonists...
May 8, 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28478688/gene-delivery-of-activated-factor-vii-using-alternative-aav-serotype-improves-hemostasis-in-hemophiliac-mice-with-fviii-inhibitors-and-aav-neutralizing-antibodies
#10
Junjiang Sun, Baolai Hua, R Jude Samulski, Chengwen Li
While therapeutic expression of coagulation factors from adeno-associated virus (AAV) vectors has been successfully achieved in patients with hemophilia, neutralizing antibodies to the vector and inhibitory antibodies to the transgene severely limit efficacy. Indeed, approximately 40% of mice transduced with human Factor VIII using the AAV8 serotype developed inhibitory antibodies to Factor VIII (FVIII inhibitor), as well as extremely high titers (≥ 1:500) of neutralizing antibodies to AAV8. To correct hemophilia in these mice we used AAV9, a serotype with low in vitro cross-reactivity (≤ 1:5) to anti-AAV8, to deliver mouse activated Factor VII (mFVIIa)...
May 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28475272/recombinant-factor-viia-addition-to-haemophilic-blood-perfused-over-collagen-tissue-factor-can-sufficiently-bypass-the-factor-ixa-viiia-defect-to-rescue-fibrin-generation
#11
R Li, K A Panckeri, P F Fogarty, A Cuker, S L Diamond
INTRODUCTION: Factor VIII (FVIII) or factor IX (FIX)-deficient haemophilic patients display deficits in platelet and fibrin deposition under flow detectable in microfluidics. Compared to fibrin generation, decreased platelet deposition in haemophilic blood flow is more easily rescued with recombinant factor VIIa (rFVIIa), whereas rFVIIa requires FXIIa participation to generate fibrin when tissue factor (TF) is absent. AIMS: Perfusion of haemophilic whole blood (WB) over collagen/TF surfaces was used to determine whether rFVIIa/TF was sufficient to bypass poor FIXa/FVIIIa function in blood from patients with haemophilia A and B...
May 5, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28470862/first-report-on-the-safety-and-efficacy-of-an-extended-half-life-glycopegylated-recombinant-fviii-for-major-surgery-in-severe-haemophilia-a
#12
K Hampton, P Chowdary, S Dunkley, S Ehrenforth, L Jacobsen, A Neff, E Santagostino, J Sathar, H Takedani, C M Takemoto, C Négrier
BACKGROUND: N8-GP (turoctocog alfa pegol) is an extended half-life glycoPEGylated recombinant factor VIII (FVIII) product developed for the prevention and treatment of bleeds in haemophilia A patients. AIM: This is a planned interim analysis of pathfinder™3, an international, open-label, Phase 3 trial evaluating the efficacy and safety (including immunogenicity) of N8-GP administered before, during and after major surgery in severe haemophilia A patients aged ≥12 years...
May 4, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28470674/acquired-hemophilia-a-updated-review-of-evidence-and-treatment-guidance
#13
REVIEW
Rebecca Kruse-Jarres, Christine L Kempton, Francesco Baudo, Peter W Collins, Paul Knoebl, Cindy A Leissinger, Andreas Tiede, Craig M Kessler
Acquired hemophilia A (AHA) is a rare disease resulting from autoantibodies (inhibitors) against endogenous factor VIII (FVIII) that leads to bleeding, which is often spontaneous and severe. AHA tends to occur in elderly patients with comorbidities and is associated with high mortality risk from underlying comorbidities, bleeding, or treatment complications. Treatment, which consists of hemostatic management and eradication of the inhibitors, can be challenging to manage. Few data are available to guide the management of AHA-related bleeding and eradication of the disease-causing antibodies...
May 3, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28452855/novel-mutations-resulting-in-a-moderate-to-severe-phenotypic-manifestation-of-hemophilia-a-in-a-female
#14
Luani Barge, Amy J Holmes, James Slade, Nalini Pati
Hemophilia A is an X-linked, recessive disorder resulting from mutations in the f8 gene. Here we report the rare case of a female compound heterozygote with mild factor VIII deficiency (fVIII:C 9%) and moderate phenotype. On investigation she was confirmed to have normal Von Willebrand factor studies with a 46XY genotype. Further genetic testing revealed 3 mutations in the f8 gene: 1 novel missense mutation (c.6142T>G), 1 novel in-frame deletion (c.1281_1292del), and another missense mutation of unclear significance (c...
April 27, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28452151/pk-guided-personalized-prophylaxis-with-nuwiq-%C3%A2-human-cl-rhfviii-in-adults-with-severe-haemophilia-a
#15
T Lissitchkov, L Rusen, P Georgiev, J Windyga, R Klamroth, L Gercheva, L Nemes, A Tiede, J Bichler, S Knaub, L Belyanskaya, O Walter, K J Pasi
INTRODUCTION: Nuwiq(®) (human-cl rhFVIII) is a 4(th) generation recombinant human FVIII, without chemical modification or protein fusion, produced in a human cell-line. AIMS/METHODS: This study (NuPreviq) was a prospective, open-label, multicentre, phase IIIb study of the efficacy and safety of personalized prophylaxis with Nuwiq(®) in 66 previously treated adults with severe haemophilia A. NuPreviq had three phases: (i) a 72-h pharmacokinetic (PK) phase; (ii) a 1-3 month standard prophylaxis phase; and (iii) a 6-month personalized prophylaxis phase...
April 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28447409/factor-viii-inhibitors-advances-in-basic-and-translational-science
#16
REVIEW
J D Lai, D Lillicrap
In the treatment of hemophilia A, the 20%-30% risk of developing of anti-factor VIII (FVIII) antibodies, or inhibitors, is the dominant concern among healthcare providers. Immune tolerance induction remains the only effective method of eradicating inhibitors in approximately 75% of patients, but is accompanied by significant emotional and economical burden. While certain risk factors, such as the type of FVIII mutation, offer some insight, there remains no strategy to confidently predict the development of an inhibitor...
May 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28440011/intensity-of-factor-viii-treatment-and-the-development-of-inhibitors-in-nonsevere-hemophilia-a-patients-results-of-the-insight-case-control-study
#17
A S van Velzen, C L Eckhardt, M Peters, F W G Leebeek, C Escuriola-Ettingshausen, C Hermans, R Keenan, J Astermark, C Male, K Peerlinck, S le Cessie, J G van der Bom, K Fijnvandraat
BACKGROUND: Inhibitor development is a major complication of treatment with factor VIII concentrates in hemophilia. Findings from studies among severe hemophilia A patients suggest that intensive treatment episodes increase the risk to develop inhibitors. OBJECTIVES: We set out to assess whether intensive treatment is also associated with an increased risk of inhibitor development among nonsevere hemophilia A patients. PATIENTS/METHODS: We performed a nested case-control study...
April 25, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28436749/differential-diagnosis-of-neonatal-alloimmune-thrombocytopenia-type-2b-von-willebrand-disease
#18
Mathilde Penel-Page, Sandrine Meunier, Mathilde Fretigny, Sandra Le Quellec, Pierre Boisseau, Christine Vinciguerra, Catherine Ternisien, Lucia Rugeri
At birth, severe thrombocytopenia without context of infection should mainly suggest neonatal alloimmune thrombocytopenia (NAIT), especially in case of a platelet count below 20 GL(-1). We report two cases of severe neonatal thrombocytopenia, first suspected as being NAIT. Both had a platelet count below 20 GL(-1) with platelet clumps. The absence of alloantibodies and failure of platelet transfusion and intravenous immunoglobulins to improve the platelet count led to question the diagnosis and to evoke inherited bleeding disorders...
April 24, 2017: Platelets
https://www.readbyqxmd.com/read/28436106/therapeutic-plasma-exchange-as-part-of-multimodal-treatment-of-acquired-hemophilia-in-a-patient-with-concurrent-acute-intracerebral-bleed-and-pulmonary-embolism
#19
Geoffrey D Wool, David Chapel, Angela Treml, Jonathan L Miller
BACKGROUND: Autoantibodies against Factor VIII (FVIII) define the rare but life-threatening bleeding disorder acquired hemophilia A (AHA). Correction of FVIII deficiency and eradication of the factor inhibitor are the ultimate therapeutic goals in this disorder. Bypassing agents such as recombinant factor VIIa (rFVIIa) or FVIII inhibitor bypassing agent are often used to control coagulopathy before the inhibitor is eradicated. Bypassing agents carry a risk of thrombosis, however. CASE REPORT: We report a patient with newly diagnosed AHA and thalamic bleed who additionally had active atrial fibrillation and developed a segmental pulmonary embolism, limiting tolerable rFVIIa dosage...
April 24, 2017: Transfusion
https://www.readbyqxmd.com/read/28432221/biological-considerations-of-plasma-derived-and-recombinant-factor-viii-immunogenicity
#20
Jesse Lai, Christine Hough, Julie Tarrant, David Lillicrap
In hemophilia A, the most severe complication of factor VIII (FVIII) replacement therapy involves the formation of FVIII neutralizing antibodies, also known as inhibitors, in 25-30% of patients. This adverse event is associated with a significant increase in morbidity and economic burden, thus highlighting the need to identify methods to limit FVIII immunogenicity. Inhibitor development is regulated by a complex balance of genetic factors, such as FVIII genotype, and environmental variables, such as coexistent inflammation...
April 21, 2017: Blood
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