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https://www.readbyqxmd.com/read/29334169/impact-of-baseline-clinical-and-laboratory-features-on-the-risk-of-thrombosis-in-children-with-acute-lymphoblastic-leukemia-a-prospective-evaluation
#1
Uma H Athale, T Mizrahi, C Laverdière, T Nayiager, Y-L Delva, G Foster, L Thabane, M David, J-M Leclerc, A K C Chan
BACKGROUND: Children with acute lymphoblastic leukemia (ALL) have increased risk of thromboembolism (TE). However, the predictors of ALL-associated TE are as yet uncertain. OBJECTIVE: This exploratory, prospective cohort study evaluated the effects of clinical (age, gender, ALL risk group) and laboratory variables (hematological parameters, ABO blood group, inherited and acquired prothrombotic defects [PDs]) at diagnosis on the development of symptomatic TE (sTE) in children (aged 1 to ≤18) treated on the Dana-Farber Cancer Institute ALL 05-001 study...
January 15, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29330877/characterization-of-biologic-response-modifiers-in-the-supernatant-of-conventional-refrigerated-and-cryopreserved-platelets
#2
Lacey Johnson, Shereen Tan, Emily Jenkins, Ben Wood, Denese C Marks
BACKGROUND: Alternatives to room temperature storage of platelets (PLTs) are of interest to support blood banking logistics. The aim of this study was to compare the presence of biologic response modifiers (BRMs) in PLT concentrates stored under conventional room temperature conditions with refrigerated or cryopreserved PLTs. STUDY DESIGN AND METHODS: A three-arm pool-and-split study was carried out using buffy coat-derived PLTs stored in 30% plasma/70% SSP+. The three matched treatment arms were as follows: room temperature (20-24°C), cold (2-6°C), and cryopreserved (-80°C with DMSO)...
January 12, 2018: Transfusion
https://www.readbyqxmd.com/read/29314439/low-incidence-of-factor-viii-inhibitors-in-previously-untreated-patients-with-severe-haemophilia-a-treated-with-octanate%C3%A2-final-report-from-a-prospective-study
#3
A Klukowska, V Komrska, V Vdovin, A Pavlova, M Jansen, S Lowndes, L Belyanskaya, O Walter, P Laguna
INTRODUCTION: Octanate® is a human, plasma-derived, von Willebrand factor-stabilized coagulation factor VIII (FVIII) concentrate with demonstrated haemostatic efficacy in previously treated patients with haemophilia A. AIM: This prospective, open-label study aimed to assess the immunogenicity of octanate® in previously untreated patients (PUPs). METHODS: The study monitored development of FVIII inhibitors in 51 PUPs. Tolerability, viral safety, FVIII recovery and efficacy of octanate® for the prevention and treatment of bleeds and in surgical procedures were also assessed...
January 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29305412/desmopressin-in-moderate-hemophilia-a-patients-a-treatment-worth-considering
#4
Janneke I Loomans, Marieke J H A Kruip, Manuel Carcao, Shannon Jackson, Alice S van Velzen, Marjolein Peters, Elena Santagostino, Helen Platokouki, Erik Beckers, Jan Voorberg, Johanna G van der Bom, Karin Fijnvandraat
Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response. Therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though factor VIII levels may rise substantially in some of them. We aim to describe the response to desmopressin in moderate hemophilia A patients and to identify predictors...
January 5, 2018: Haematologica
https://www.readbyqxmd.com/read/29296914/severe-platelet-dysfunction-in-nhl-patients-receiving-ibrutinib-is-absent-in-patients-receiving-acalabrutinib
#5
Alexander P Bye, Amanda J Unsworth, Michael J Desborough, Catherine A T Hildyard, Niamh Appleby, David Bruce, Neline Kriek, Sophie H Nock, Tanya Sage, Craig E Hughes, Jonathan M Gibbins
The Bruton tyrosine kinase (Btk) inhibitor ibrutinib induces platelet dysfunction and causes increased risk of bleeding. Off-target inhibition of Tec is believed to contribute to platelet dysfunction and other side effects of ibrutinib. The second-generation Btk inhibitor acalabrutinib was developed with improved specificity for Btk over Tec. We investigated platelet function in patients with non-Hodgkin lymphoma (NHL) receiving ibrutinib or acalabrutinib by aggregometry and by measuring thrombus formation on collagen under arterial shear...
December 12, 2017: Blood Advances
https://www.readbyqxmd.com/read/29296836/long-term-safety-and-efficacy-of-emicizumab-in-a-phase-1-2-study-in-patients-with-hemophilia-a-with-or-without-inhibitors
#6
Midori Shima, Hideji Hanabusa, Masashi Taki, Tadashi Matsushita, Tetsuji Sato, Katsuyuki Fukutake, Ryu Kasai, Koichiro Yoneyama, Hiroki Yoshida, Keiji Nogami
Emicizumab (ACE910), a recombinant humanized bispecific monoclonal antibody, provides factor VIII (FVIII) cofactor bridging function to restore hemostasis in people with hemophilia A. In a phase 1 trial involving 18 Japanese patients with severe hemophilia A, once-weekly subcutaneous administration of emicizumab 0.3, 1, or 3 mg/kg (cohorts 1, 2, and 3, respectively) was well tolerated and substantially reduced annualized bleeding rates (ABRs) in the presence or absence of FVIII inhibitors. The current study represents an open-label, long-term extension of the previously reported 12-week phase 1 study, in which 16 of 18 patients continued to receive emicizumab for up to 33...
October 10, 2017: Blood Advances
https://www.readbyqxmd.com/read/29296830/cd4-t-cells-specific-for-factor-viii-are-present-at-high-frequency-in-healthy-donors-and-comprise-na%C3%A3-ve-and-memory-cells
#7
Sylvain Meunier, Catherine Menier, Elodie Marcon, Sébastien Lacroix-Desmazes, Bernard Maillère
We investigated the frequency and subset origin of circulating factor VIII (FVIII)-specific CD4 T cells in healthy donors. Total CD4 T cells and purified CD4 T-cell subsets were stimulated with FVIII-loaded autologous dendritic cells and challenged for specificity in interferon-γ enzyme-linked immunospots. The number of specific T-cell lines allowed estimation of the frequency of T cells circulating in the blood of the donors. All the 16 healthy donors generated strong in vitro T-cell responses, leading to the generation of 154 FVIII-specific T-cell lines...
September 26, 2017: Blood Advances
https://www.readbyqxmd.com/read/29288565/high-and-long-term-von-willebrand-factor-expression-after-sleeping-beauty-transposon-mediated-gene-therapy-in-a-mouse-model-of-severe-von-willebrand-disease
#8
Irina Portier, Karen Vanhoorelbeke, Sebastien Verhenne, Inge Pareyn, Nele Vandeputte, Hans Deckmyn, Daniel S Goldenberg, Himanshu B Samal, Manvendra Singh, Zoltán Ivics, Zsuzsanna Izsvák, Simon F De Meyer
BACKGROUND: Type 3 von Willebrand disease (VWD) is characterized by complete absence of von Willebrand factor (VWF). Current therapy is limited to treatment with exogenous VWF/FVIII products, which only provide a short-term solution. Gene therapy offers the potential for a long-term treatment for VWD. OBJECTIVES: To develop an integrative Sleeping Beauty (SB) transposon-mediated VWF gene transfer approach in a preclinical mouse model of severe VWD. METHODS: We established a robust platform for sustained transgene murine (m)VWF expression in the liver of Vwf-/- mice by combining a liver-specific promoter with a sandwich transposon design and the SB100X transposase via hydrodynamic gene delivery...
December 29, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29285874/early-cellular-interactions-and-immune-transcriptome-profiles-in-human-factor-viii-exposed-hemophilia-a-mice
#9
J D Lai, D Cartier, R B Hartholt, L L Swystun, A S van Velzen, J M M den Haan, C Hough, J Voorberg, D Lillicrap
BACKGROUND: Developing factor VIII (FVIII) inhibitory antibodies is the most serious complication in hemophilia A treatment, representing a significant health and economic burden. A better understanding of the early events in an immune response leading to this outcome may provide insight into inhibitor development. OBJECTIVE: To identify early mediators of FVIII immunity and to detail immune expression profiles in the spleen and liver. METHODS: C57Bl/6 F8 E16 knockout mice were infused with 5-20 μg (2000-8000 IU/kg) of recombinant FVIII...
December 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29285859/determinants-of-impaired-renal-and-vascular-function-are-associated-with-higher-levels-of-procoagulant-factors-in-the-general-population
#10
Ilona A Dekkers, Renée de Mutsert, Aiko P J de Vries, Frits R Rosendaal, Suzanne C Cannegieter, J Wouter Jukema, S le Cessie, Ton J Rabelink, Hildo J Lamb, Willem M Lijfering
BACKGROUND: Impaired renal and vascular function have been associated with venous thrombosis, but the mechanism is unclear. OBJECTIVES: We investigated whether estimated glomerular filtration rate (eGFR), urinary albumin-creatinine ratio (UACR), and pulse wave velocity (PWV) are associated with a procoagulant state. METHODS: In this cross-sectional analysis of the NEO Study, eGFR, UACR, fibrinogen, and coagulation factors (F)VIII, FIX, and FXI were determined in all participants (n=6536), and PWV was assessed in a random subset (n=2433)...
December 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29274194/international-comparative-field-study-evaluating-the-assay-performance-of-afstyla-in-plasma-samples-at-clinical-hemostasis-laboratories
#11
Katie St Ledger, Annette Feussner, Uwe Kalina, Carsten Horn, Hubert J Metzner, Debra Bensen-Kennedy, Nicole Blackman, Alex Veldman, Anthony Stowers, Kenneth D Friedman
INTRODUCTION: AFSTYLA (antihemophilic factor [recombinant] single chain) is a novel B-domain truncated recombinant Factor VIII (rFVIII). For AFSTYLA, an approximate 50% discrepancy was observed between results of the one-stage (OS) and chromogenic substrate (ChS) FVIII activity AFSTYLA assays. An investigation was undertaken to test whether there is a linear relationship between ChS and OS assay results that would allow reliable clinical interpretation of results independent of the assay method used...
December 23, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29258406/a-contemporary-look-at-fviii-inhibitor-development-still-a-great-influence-on-the-evolution-of-hemophilia-therapies
#12
Elena Santagostino, Guy Young, Manuel Carcao, Pier Mannuccio Mannucci, Susan Halimeh, Steve Austin
The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia...
December 19, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29243367/vaccinations-are-not-associated-with-inhibitor-development-in-boys-with-severe-haemophilia-a
#13
H Platokouki, K Fischer, S C Gouw, A Rafowicz, M Carcao, G Kenet, R Liesner, K Kurnik, G E Rivard, H M van den Berg
BACKGROUND: Inhibitor development in previously untreated patients (PUPs) with severe haemophilia A is a multifactorial event. It is unknown whether paediatric vaccinations given in close proximity to factor VIII (FVIII) are associated with inhibitor development. OBJECTIVE: To assess whether paediatric vaccinations in close proximity to FVIII within the first 75 exposure days (EDs) are associated with inhibitor development in PUPs with severe haemophilia A. METHODS: We included 375 PUPs with severe haemophilia A (<0...
December 15, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29240027/congenital-hemophilia-a-presenting-with-hashimoto-s-encephalopathy-and-myocarditis-the-first-reported-case
#14
Ayse B Ergul, Yasemin Altuner Torun, Umut Altug, Fatma T Mutlu, Serkan F Celik, Ahmet S Guven
Here, we report a case of hemophilia A with myocarditis, encephalopathy, and spontaneous intramedullary hemorrhage. A 14-month-old male infant presented with loss of consciousness, generalized tonic-clonic convulsions, and cardiac failure. The neurological examination was normal. Myocarditis was diagnosed. After administration of fresh frozen plasma, the aPTT did not return to normal. The factor VIII (FVIII) level was 10.2% the normal level, and the patient was diagnosed with hemophilia A. The cerebrospinal fluid (CSF) evaluation was unremarkable, with the exception of elevated CSF protein levels...
December 12, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29232255/lack-of-grading-agreement-among-international-hemostasis-external-quality-assessment-programs
#15
John D Olson, Ian Jennings, Piet Meijer, Chantal Bon, Roslyn Bonar, Emmanuel J Favaloro, Russell A Higgins, Michael Keeney, Joy Mammen, Richard A Marlar, Roland Meley, Sukesh C Nair, William L Nichols, Anne Raby, Joan C Reverter, Alok Srivastava, Isobel Walker
: Laboratory quality programs rely on internal quality control and external quality assessment (EQA). EQA programs provide unknown specimens for the laboratory to test. The laboratory's result is compared with other (peer) laboratories performing the same test. EQA programs assign target values using a variety of methods statistical tools and performance assessment of 'pass' or 'fail' is made. EQA provider members of the international organization, external quality assurance in thrombosis and hemostasis, took part in a study to compare outcome of performance analysis using the same data set of laboratory results...
December 11, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29215814/safety-and-efficacy-of-b-domain-deleted-third-generation-recombinant-factor-viii-greengene-f%C3%A2-in-korean-patients-with-hemophilia-a-data-from-a-post-marketing-surveillance-study
#16
Soon Ki Kim, Ki Young Yoo, Kun Soo Lee, Taiju Hwang, Yong Mook Choi, Eun Jin Choi, Sang Kyu Park
BACKGROUND: New B-domain deleted third generation recombinant factor VIII (FVIII; GreenGene F™, beroctocog alfa) was launched in 2010. We determined safety and efficacy of GreenGene F™ during routine clinical practice in patients with hemophilia A over a period of 12 months. METHODS: From July 2010 to July 2014, a total of 136 hemophilia A patients were enrolled in a post-marketing surveillance (PMS) study. Among them, 134 patients were assessed for drug safety and 114 patients were analyzed for drug efficacy...
January 1, 2018: Journal of Korean Medical Science
https://www.readbyqxmd.com/read/29212111/pathophysiological-mechanisms-of-endogenous-fviii-release-following-strenuous-exercise-in-non-severe-haemophilia-a-review
#17
C L Venema, R E G Schutgens, K Fischer
No abstract text is available yet for this article.
December 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29202211/acute-haemarthrosis-in-the-haemophilia-a-rat-generates-a-local-and-systemic-proinflammatory-response
#18
Karin M Lövgren, Kristine R Christensen, Wiktor Majewski, Olga Østrup, Søren Skov, Bo Wiinberg
Background Replacement therapy with coagulation factor VIII (FVIII) concurrent with bleeds (on-demand) in haemophilia A (HA) patients has been hypothesized to increase the risk for antidrug antibodies (inhibitors). A danger signal environment, characterized by tissue damage and inflammation at the site of a bleed, is thought to contribute to the anti-FVIII response. The nature of this inflammatory reaction is, however, not fully known, and new insights will be valuable for both managing inhibitors and understanding arthropathy development...
November 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29197156/identification-of-aggregates-in-therapeutic-formulations-of-recombinant-full-length-factor-viii-products-by-sedimentation-velocity-analytical-ultracentrifugation
#19
J F Healey, E T Parker, P Lollar
BACKGROUND: The development of inhibitory anti-factor VIII (fVIII) antibodies is the most serious complication in the management of patients with hemophilia A. Studies have suggested that recombinant full-length fVIII is more immunogenic than plasma-derived fVIII and that among recombinant fVIII products, Kogenate is more immunogenic than Advate. Aggregates in biopharmaceutical products are considered a risk factor for the development of anti-drug antibodies. OBJECTIVE: To evaluate recombinant full-length fVIII products for the presence of aggregates...
December 2, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29194866/break-through-bleeding-in-relation-to-pharmacokinetics-of-factor-viii-in-paediatric-patients-with-severe-haemophilia-a
#20
X Cheng, P Li, Z Chen, N Zhang, Y Zhen, L Zhao, X Wang, R Wu
INTRODUCTION: As the pharmacokinetics (PK) of factor VIII (FVIII) is individualized in children with haemophilia A (HA), PK parameters may be indicators of patients' bleeding phenotype and instruction for their personalized replacement program. AIM: The aim of this study was to investigate the possible relationship between PK/FVIII level and bleeding frequency in Chinese paediatric patients with severe (HA). METHODS: A total of 24 patients were enrolled in Beijing Children's Hospital from February to October 2015, all of whom were given 50 IU/kg of FVIII concentrates after a 72-hours washout period...
December 1, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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