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https://www.readbyqxmd.com/read/28651073/hemophilia-a-inhibitor-treatment-the-promise-of-engineered-t-cell-therapy
#1
REVIEW
Kalpana Parvathaneni, Maha Abdeladhim, Kathleen P Pratt, David W Scott
Hemophilia A is a bleeding disorder caused by mutations in the gene encoding factor VIII (FVIII), a cofactor protein that is essential for normal blood clotting. Approximately, 1 in 3 patients with severe hemophilia A produce neutralizing antibodies (inhibitors) that block its biologic function in the clotting cascade. Current efforts to eliminate inhibitors consist of repeated FVIII injections under what is termed an "ITI" protocol (Immune Tolerance Induction). However, this method is extremely costly and approximately 30% of patients undergoing ITI do not achieve peripheral tolerance...
June 9, 2017: Translational Research: the Journal of Laboratory and Clinical Medicine
https://www.readbyqxmd.com/read/28649180/procoagulatory-changes-induced-by-head-up-tilt-test-in-patients-with-syncope-observational-study
#2
Viktor Hamrefors, Artur Fedorowski, Karin Strandberg, Richard Sutton, Nazim Isma
BACKGROUND: Orthostatic hypercoagulability is proposed as a mechanism promoting cardiovascular and thromboembolic events after awakening and during prolonged orthostasis. We evaluated early changes in coagulation biomarkers induced by tilt testing among patients investigated for suspected syncope, aiming to test the hypothesis that orthostatic challenge evokes procoagulatory changes to a different degree according to diagnosis. METHODS: One-hundred-and-seventy-eight consecutive patients (age, 51 ± 21 years; 46% men) were analysed...
2017: Thrombosis Journal
https://www.readbyqxmd.com/read/28641762/laparoscopic-versus-robotic-surgery-for-hepatocellular-carcinoma-the-first-46-consecutive-cases
#3
Paolo Magistri, Giuseppe Tarantino, Cristiano Guidetti, Giacomo Assirati, Tiziana Olivieri, Roberto Ballarin, Andrea Coratti, Fabrizio Di Benedetto
BACKGROUND: Hepatocellular carcinoma has a growing incidence worldwide, and represents a leading cause of death in patients with cirrhosis. Nowadays, minimally invasive approaches are spreading in every field of surgery and in liver surgery as well. MATERIALS AND METHODS: We retrospectively reviewed demographics, clinical, and pathologic characteristics and short-term outcomes of patients who had undergone minimally invasive resections for hepatocellular carcinoma at our institution between June 2012 and May 2016...
May 8, 2017: Journal of Surgical Research
https://www.readbyqxmd.com/read/28641667/-research-progress-on-genetic-factors-and-hemophilia-a-clotting-factor-inhibitor-review
#4
Zheng-Bin Hu, Xin Sun, Li-Ya He
Hemophilia A (Hemophilia A, HA) is an X-linked recessive hereditary coagulation function disorder, the deficiency and dysfunction of blood coagulation were caused by the mutations of gene encoding clotting factor VIII. The treatment of hemophilia A still depends on the replacement therapy with blood coagulation factor. However, the repeated infusion of clotting factor will produce the neutralizing antibody against FVIII, then resulting in one of the serious complications. The reports on the incidence of inhibitor are different at home and abroad...
June 2017: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28641658/-effects-of-different-storage-time-and-temperature-on-coagulation-function-of-fresh-frozen-plasma-after-thawing
#5
Xiao-Yun Gao, Xin-Hua Wang
OBJECTIVE: To investigate the coagulation function changes of fresh frozen plasma storaged at different storage time and temperature after thawing. METHODS: Forty unit of fresh frozen plasma were collected, and thawed at 37 °C for 25 minutes. Each unit was divided into 2 halves: one was stored at (4±2)°C for 72 hours and the other one was stored at (25±2) °C for 72 hours. At the time point of 0, 4, 24, 48 and 72 h, thromboela-stogram TEG was measured for all samples...
June 2017: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28638302/extended-stability-and-sterility-of-antihemophilic-factor-human
#6
Sarah Shields, Abby Kim, Joshua Elder
OBJECTIVES: Antihemophilic factor human is a factor VIII product used to supplement those with hemophilia. Recent data show treatment benefit and cost saving opportunities if factor products are administered as a continuous infusion rather than conventional bolus dose. This method has not been widely used given the lack of evidence for safe and effective use beyond 3 hours from preparation. The objectives of this study were to determine the physical and chemical stability and sterility of antihemophilic factor human over a 7-day period...
May 2017: Journal of Pediatric Pharmacology and Therapeutics: JPPT: the Official Journal of PPAG
https://www.readbyqxmd.com/read/28636264/desmopressin-in-haemophilia-the-need-for-a-standardised-clinical-response-and-individualised-test-regimen
#7
S C M Stoof, L M Schütte, F W G Leebeek, M H Cnossen, M J H A Kruip
INTRODUCTION: Due to interindividual variation in desmopressin response, non-severe haemophilia A patients require desmopressin testing prior to therapeutic treatment. However, adequate response or frequency of blood sampling is not standardised in international guidelines. Consequently, various definitions and blood sampling protocols are currently applied. Interestingly, sustainability of desmopressin response is not incorporated into these definitions. AIM: To study desmopressin response rates in a cohort of non-severe haemophilia A patients using currently accepted desmopressin response definitions...
June 21, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28633535/changes-in-the-level-of-cardiac-troponine-and-disorders-in-pulmonary-gas-exchange-as-predictors-of-short-and-long-term-outcomes-of-patients-with-aneurysm-subarachnoid-haemorrhage
#8
Małgorzata Burzyńska, Agnieszka Uryga, Magdalena Kasprowicz, Jarosław Kędziora, Ewa Szewczyk, Jowita Woźniak, Włodzimierz Jarmundowicz, Andrzej Kübler
SUBJECT: Cardiopulmonary abnormalities are common after aneurysmal subarachnoid haemorrhage (aSAH). However, the relationship between short- and long-term outcome is poorly understood. In this paper, we present how cardiac troponine elevations (cTnI) and pulmonary disorders are associated with short- and long-term outcomes assessed by the Glasgow Outcome Scale (GOS) and Extended Glasgow Outcome Scale (GOSE). METHODS: A total of 104 patients diagnosed with aSAH were analysed in the study...
June 21, 2017: British Journal of Neurosurgery
https://www.readbyqxmd.com/read/28625333/liver-metastases-from-gastric-carcinoma-a-case-report-and-review-of-the-literature
#9
Beata Polkowska-Pruszyńska, Karol Rawicz-Pruszyński, Bogumiła Ciseł, Robert Sitarz, Grażyna Polkowska, Witold Krupski, Wojciech P Polkowski
Gastric carcinoma (GC) is the fifth most common malignancy worldwide but the third leading cause of cancer death, and surgery remains the only curative treatment option. Prognosis of patients with liver metastases from gastric carcinoma (LMGC) is poor, and the optimal treatment of metastatic gastric cancer remains a matter of debate. In 2002, a 53-year-old male patient with GC and synchronous oligometastatic lesion in liver VIII segment underwent a total gastrectomy combined with metastasectomy. The pathologic diagnosis was stage IV gastric adenocarcinoma (pT3N2M1), which was treated with adjuvant chemotherapy (cisplatin, epirubicin, leucovorin, and 5-fluorouracil)...
May 2017: Current Problems in Cancer
https://www.readbyqxmd.com/read/28602126/clinical-and-prognostic-significance-of-lupus-anticoagulant-measurement-in-patients-with-lung-cancer
#10
Xianming Fei, Huan Wang, Lei Jiang, Tongwei Zhao, Maoliang Cheng, Wufeng Yuan
Lupus anticoagulants is related to both recurrent thrombosis and cancer. Thrombotic complications occur more frequently in patients with lung cancer. The aim of this study is to investigate the association of lupus anticoagulants with hypercoagulability and thrombotic complications, as well as prognostic significance of lupus anticoagulants for patients with lung cancer. The study comprised 205 patients with non-small cell lung cancer. Plasma normalized LAC ratio, D-dimer, fibrinogen, activities of antithrombin, and FVIII before treatment were analyzed by coagulation analyzer, and routine hematologic and biochemical parameters were also evaluated...
January 1, 2017: Technology in Cancer Research & Treatment
https://www.readbyqxmd.com/read/28601434/an-open-label-single-dose-pharmacokinetic-study-of-factor-viii-activity-after-administration-of-moroctocog-alfa-af-cc-in-male-chinese-patients-with-hemophilia-a
#11
Hongzhong Liu, Runhui Wu, Pei Hu, Feifei Sun, Lihong Xu, Yali Liang, Sunil Nepal, Peng Roger Qu, Francois Huard, Joan M Korth-Bradley
PURPOSE: Hemophilia A represents up to 80% of all hemophilia cases in China. In patients with this condition, bleeding can be prevented and controlled by administering clotting factor VIII (FVIII). Since their initial availability, recombinant FVIII products have undergone several iterations to enhance their safety. Moroctocog alfa albumin-free cell culture (AF-CC) is among the third generation of recombinant FVIII products and received regulatory approval in China in August 2012. The present study characterizes the single-dose pharmacokinetic parameters of FVIII activity (FVIII:C) after administration of moroctocog alfa (AF-CC) in male Chinese patients with hemophilia A...
June 7, 2017: Clinical Therapeutics
https://www.readbyqxmd.com/read/28601327/milk-fat-globule-epidermal-growth-factor-factor-viii-attenuates-sepsis-induced-acute-kidney-injury
#12
Cindy Cen, Monowar Aziz, Weng-Lang Yang, Mian Zhou, Jeffrey M Nicastro, Gene F Coppa, Ping Wang
BACKGROUND: Acute kidney injury (AKI) is most commonly caused by sepsis in critically ill patients, and it is associated with high morbidity and mortality. The pathophysiology of sepsis-induced AKI is generally accepted to include direct inflammatory injury, endothelial cell dysfunction, and apoptosis. Milk fat globule-epidermal growth factor-factor VIII (MFG-E8) is a secretory glycoprotein with a known role in the enhancement of apoptotic cell clearance and regulation of inflammation...
June 1, 2017: Journal of Surgical Research
https://www.readbyqxmd.com/read/28599169/modifiers-of-clinical-phenotype-in-severe-congenital-hemophilia
#13
Massimo Franchini, Pier Mannuccio Mannucci
Patients with inherited hemophilia A and B usually exhibit a bleeding tendency of a severity proportional to the degree of plasmatic deficiency of the coagulant activity of factor VIII (FVIII:C) and factor IX (FIX:C). Although patients with severe hemophilia (i.e., with FVIII:C and FIX:C levels <1IU/dL) are generally those with the most severe bleeding phenotype, it is common experience that a variable proportion of them experiences a milder bleeding tendency. In this review, we summarize the current knowledge on the possible mechanisms at the basis of the phenotypic heterogeneity of severe hemophilia, focusing in particular on the role of FVIII/FIX gene mutations and thrombophilic polymorphisms...
June 1, 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28597369/first-report-of-real-time-monitoring-of-coagulation-function-potential-and-igg-subtype-of-anti-fviii-autoantibodies-in-a-child-with-acquired-hemophilia-a-associated-with-streptococcal-infection-and-amoxicillin
#14
Masahiro Takeyama, Keiji Nogami, Takahiro Kajimoto, Kenichi Ogiwara, Tomoko Matsumoto, Midori Shima
We describe an 8-year-old boy with acquired hemophilia A (AHA) associated with streptococcal infection and amoxicillin. Laboratory data revealed low factor VIII activity (FVIII:C, 1.5 IU/dl), and FVIII inhibitor (15.9 BU/ml). Comprehensive coagulation function assays, including rotation thromboelastometry (ROTEM(®)), revealed a markedly prolonged clotting time. Thrombin and plasmin generation (TG/PG) appeared to be moderately impaired. The inhibitor epitope of his anti-FVIII autoantibody recognized light and heavy chains...
June 8, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28596657/increased-level-of-factor-viii-and-physiological-inhibitors-of-coagulation-in-patients-with-sickle-cell-disease
#15
Mohamed Chekkal, Mohamed Chakib Arslane Rahal, Khedidja Moulasserdoun, Fatima Seghier
Sickle cell disease (SCD) is a hemoglobinopathy characterized by hemolysis, oxidative stress, and vaso-occlusive crises. Thromboembolism also remains a serious complication and probably underestimated in the SCD. Our objective was to seek the existence of hemostasis abnormalities that predispose to thrombosis such as elevation of FVIII and Physiological inhibitors of coagulation deficiency. We studied 81 patients with SCD, including 32 homozygous S/S, 20 double heterozygous S/β thalassemia and 29 heterozygous S/A...
June 2017: Indian Journal of Hematology & Blood Transfusion
https://www.readbyqxmd.com/read/28591493/-perception-of-quality-of-life-related-with-health-and-depression-in-patients-with-hemophilia
#16
Maricela Osorio-Guzmán, Graciela Gutiérrez-González, Georgina Eugenia Bazán-Riverón, Nora Nancy Núñez-Villegas, Gabriela Jazmín Fernández-Castillo
BACKGROUND: Hemophilia is a hemorrhagic disease characterized by the deficiency of either coagulation factor VIII or IX. It impacts negatively in people's quality of life and it causes side effects, such as depression. The objective was to describe and analyze the health-related quality of life (HRQoL) and depression levels in a group of 50 pediatric patients with hemophilia (PPwH) and their parents. The specific objectives were: to analyze the correlation between HRQoL levels reported by patients and their parents, and to analyze the correlation between HRQoL levels and depression in PPwH...
July 2017: Revista Médica del Instituto Mexicano del Seguro Social
https://www.readbyqxmd.com/read/28585251/tfe3-rearranged-hepatic-epithelioid-hemangioendothelioma-a-case-report-with-immunohistochemical-and-molecular-study
#17
Fang-Ying Kuo, Hsuan-Ying Huang, Chao-Long Chen, Hock-Liew Eng, Chao-Cheng Huang
A recurrent YAP1-TFE3 gene fusion has been identified in WWTR1-CAMTA1-negative epithelioid hemangioendotheliomas arising in soft tissue, bone, and lung, but not in liver. We present the first case of TFE3-rearranged hepatic epithelioid hemangioendothelioma in a 39-year-old Taiwanese woman. Computed tomography scan revealed multifocal, ill-defined nodules involving both hepatic lobes. She then underwent deceased donor liver transplantation. Histologically, the tumors in the liver explant showed a biphasic growth pattern...
June 6, 2017: APMIS: Acta Pathologica, Microbiologica, et Immunologica Scandinavica
https://www.readbyqxmd.com/read/28584903/incidence-and-outcome-of-retained-port-a-cath-fragments-during-removal
#18
Olugbenga Michael Aworanti, Niall Linnane, Farhan Tareen, Alan Mortell
PURPOSE: Port-A-Cath devices are frequently used for long-term venous access. We postulate that long-term use predisposes them to getting stuck and retained in a central vein at the time of removal. We aim to report the incidence and outcome of this complication. METHODS: Between January 2006 and July 2016, a retrospective review of all Port-A-Cath removals that were performed at our centre was conducted. At the time of removal, catheters that could not be removed from the vein were considered retained...
June 5, 2017: Pediatric Surgery International
https://www.readbyqxmd.com/read/28583468/-role-of-quality-control-for-improvement-of-blood-components-in-the-regional-blood-centre-of-ouagadougou-in-burkina-faso-2014
#19
K Nebie, S Sawadogo, E Kafando, E M Bationo, H Dahourou, S Ouattara, K Kienou, S Nana, L Kaba, C Fretz, E L Murphy
OBJECTIVES: In Burkina Faso, blood components must comply with national standards. Then, all Transfusion services must implement a quality control process to ensure compliance. Our study aims to establish the main characteristics of blood components of the regional transfusion center of Ouagadougou, and evaluate the capability of this center to improve its manufacturing process. METHODS: We conducted from marsh to December 2014 a pre-post study, assessing blood components' characteristics before and after the implementation of a six months' improvement plan...
June 2, 2017: Transfusion Clinique et Biologique: Journal de la Société Française de Transfusion Sanguine
https://www.readbyqxmd.com/read/28581694/abnormal-von-willebrand-factor-secretion-factor-viii-stabilization-and-thrombus-dynamics-in-type-2n-von-willebrand-disease-mice
#20
Laura L Swystun, Ilinca Georgescu, Jeff Mewburn, Meghan Deforest, Kate Nesbitt, Kassandra Hebert, Courtney Dwyer, Christine Brown, Colleen Notley, David Lillicrap
BACKGROUND: von Willebrand factor (VWF) and factor VIII (FVIII) circulate as a non-covalent complex with VWF serving as the carrier for FVIII. VWF indirectly influences secondary hemostasis by stabilizing FVIII and transporting it to the site of primary hemostasis. Type 2N von Willebrand disease involves impaired binding of VWF to FVIII, resulting in decreased plasma levels of FVIII. OBJECTIVES: In these studies, we characterize the impact of three type 2N VWD variants (R763A, R854Q, R816W) on VWF secretion, FVIII stabilization, and thrombus formation in a murine model...
June 5, 2017: Journal of Thrombosis and Haemostasis: JTH
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