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https://www.readbyqxmd.com/read/29149425/correction-to-efficacy-and-safety-of-full-length-pegylated-recombinant-factor-viii-with-extended-half-life-in-previously-treated-patients-with-hemophilia-a-comparison-of-data-between-the-general-and-japanese-study-populations
#1
Keiji Nogami, Midori Shima, Katsuyuki Fukutake, Teruhisa Fujii, Masashi Taki, Tadashi Matsushita, Satoshi Higasa, Tetsuji Sato, Michio Sakai, Morio Arai, Haruhiko Uchikawa, Werner Engl, Brigitt Abbuehl, Barbara A Konkle
The authors would like to correct the error in Table 2 in the original publication of the article. The "Blood type" is not described in any part of "Results" and "Discussion" and had no impact on the conclusion hence the bottom of the table is removed.
November 17, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/29147724/glabrous-cmgl-encodes-a-hd-zip-iv-transcription-factor-playing-roles-in-multicellular-trichome-initiation-in-melon
#2
Huayu Zhu, Xiaofen Sun, Qi Zhang, Pengyao Song, Qianmei Hu, Xiaojing Zhang, Xiang Li, Jianbin Hu, Junsong Pan, Shouru Sun, Yiqun Weng, Luming Yang
Map-based cloning identified CmGL that encodes a HD-ZIP type IV transcription factor that controls multicellular trichome initiation in melon. Trichomes are small hairs covering the aerial parts of plants that originate from the epidermal cells, which can protect plants against the damage by insects and pathogens. The regulatory pathway of unicellular trichomes has been well studied in the model plant Arabidopsis. Little is known about the genetic control and regulation of trichome development in melon (Cucumis melo L...
November 17, 2017: TAG. Theoretical and Applied Genetics. Theoretische und Angewandte Genetik
https://www.readbyqxmd.com/read/29147591/upper-limb-deep-vein-thrombosis-in-patient-with-hemophilia-a-and-heterozygosity-for-prothrombin-g20210a-a-case-report-and-review-of-the-literature
#3
Fares Darawshy, Yosef Kalish, Issam Hendi, Ayman Abu Rmelieh, Tawfik Khoury
Deep vein thrombosis (DVT) is a rare disease in patients with hemophilia A. We report a case of 22-year-old male with severe hemophilia A who presented to the emergency room with 5-day history of right arm pain that was attributed initially to bleeding event. In the absence of external signs of bleeding or hematoma and normal hemoglobin level, we suspected an underlying DVT. Doppler ultrasonography of the right upper limb revealed thrombosis of the subclavian vein and this was confirmed by CT venography. The d-dimer level was normal and investigations for prothrombotic state revealed heterozygosity for prothrombin G20210A mutation...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/29146705/complement-c3-is-a-novel-modulator-of-the-anti-fviii-immune-response
#4
Julie Rayes, Mathieu Ing, Sandrine Delignat, Ivan Peyron, Laurent Gilardin, Carl-Wilhelm Vogel, David C Fritzinger, Véronique Frémeaux-Bacchi, Srinivas V Kaveri, Lubka T Roumenina, Sébastien Lacroix-Desmazes
Development of neutralizing antibodies against therapeutic factor VIII (FVIII) is the most serious complication of the treatment of hemophilia A. Increasing evidence shows the multifactorial origin of the anti-FVIII immune response, combining both genetic and environmental factors. While a role for the complement system on innate as well as adaptive immunity has been documented, the implication of complement activation on the onset of the anti-FVIII immune response is unknown. Here, using in vitro assays for FVIII endocytosis by human monocyte-derived dendritic cells and presentation to T cells, as well as animal models of in vivo complement depletion, we show a novel role for complement C3 in enhancing the immune response against therapeutic FVIII...
November 16, 2017: Haematologica
https://www.readbyqxmd.com/read/29145340/open-repair-of-intraoperative-popliteal-artery-injury-during-total-knee-arthroplasty-in-a-patient-with-severe-hemophilia-a-a-case-report-and-literature-review
#5
Bin Feng, Ke Xiao, Jiang Shao, Yu Fan, Xisheng Weng
RATIONALE: Arterial injuries during total knee arthroplasty (TKA) though rare may occur. Hemophilia may further challenge the management of such injury. PATIENT CONCERNS: A 48-year-old male patient with severe hemophilia type A and stiff knee arthropathy underwent bilateral TKAs. Left popliteal artery injury was detected at the end of the left TKA. DIAGNOSES: Urgent angiography confirmed the diagnosis of the left popliteal artery transection...
November 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29144625/acquired-hemophilia-presenting-as-gross-hematuria-following-kidney-stone-a-case-report-and-review-of-the-literature
#6
Max Schmidt-Bowman, Lael Reinstatler, Eric P Raffin, Joseph E Yared, John D Seigne, Einar F Sverrisson
A rare condition in itself, acquired hemophilia A, seldom presents as isolated gross hematuria. It is a serious condition with a high mortality rate and thus clinical suspicion followed by prompt diagnosis is imperative (1). In fact, only 8 cases of such presentation of this condition have been reported thus far in the literature. Of these, none describe the initial presentation of hematuria with the inciting event of a kidney stone. We present a case of a 67-year-old man with signs and symptoms of nephrolithiasis accompanied by profuse hematuria, who was subsequently found to have developed expression of factor VIII inhibitor leading to acquired hemophilia A...
November 19, 2017: International Braz J Urol: Official Journal of the Brazilian Society of Urology
https://www.readbyqxmd.com/read/29144254/emicizumab-prophylaxis-overcomes-factor-viii-inhibitors-in-hemophilia-a
#7
Midori Shima
No abstract text is available yet for this article.
November 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/29139314/cost-utility-analysis-of-life-long-prophylaxis-with-recombinant-factor-viii-fc-vs-recombinant-factor-viii-for-the-management-of-severe-haemophilia-a-in-sweden
#8
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 haemophilia 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 haemophilia A from the perspective of the Swedish health system...
November 15, 2017: Journal of Medical Economics
https://www.readbyqxmd.com/read/29135477/thrombin-potential-and-traditional-coagulation-assay-are-they-useful-in-exploring-recurrent-pregnancy-loss-risk
#9
Ilaria Romagnuolo, Monica Attanasio, Mauro Cozzolino, Enrichetta Paladino, Giancarlo Castaman, Maria E Coccia, Cinzia Fatini
: An adequate hemostatic balance is mandatory to get successful pregnancy. Obstetric complications, such as recurrent pregnancy loss (RPL), might be due to an impairment of placental perfusion possibly related to an underlying prothrombotic status. In this study, we used the global coagulation assay, calibrated automated thrombography and traditional coagulation assay to search for a possible underlying hypercoagulable status in women with history of RPL compared with uneventful pregnancy women. Thrombin generation, Fibrinogen, factor VIII (FVIII), Plasminogen Activator Inhibitor-1 (PAI-1) and von Willebrand factor levels were analyzed in 92 not pregnant unexplained RPL and 64 uneventful pregnancy women...
November 10, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29127625/gene-therapy-for-hemophilia-progress-to-date
#10
John C Chapin, Paul Edward Monahan
Hemophilia is a congenital bleeding disorder that affects nearly half a million individuals worldwide. Joint bleeding and other co-morbidities are a significant source of debilitation for this population. Current therapies are effective but must be given lifelong at regular intervals, are costly, and are available to only about 25% of the hemophilia population living in resource-rich countries. Gene therapy for hemophilia has been in development for three decades and is now entering pivotal-stage clinical trials...
November 10, 2017: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
https://www.readbyqxmd.com/read/29115006/type-2n-von-willebrand-disease-characterization-and-diagnostic-difficulties
#11
A Casonato, E Galletta, L Sarolo, V Daidone
INTRODUCTION: An abnormal factor VIII (FVIII) binding capacity of von Willebrand factor (VWF) identifies type 2N von Willebrand disease (VWD). Type 2N VWD patients are identified by means of the VWF FVIII binding (VWF:FVIIIB) assay, and especially their VWF:FVIIIB/VWF:Ag ratio (VWF:FVIIIB ratio). AIM: We report on our 15-year experience of diagnosing type 2N VWD. METHODS: We have performed 2178 VWF:FVIIIB assays in bleeders and normal subjects...
November 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29108420/inhibitor-risk-stratification-and-individualized-treatment-in-patients-with-nonsevere-hemophilia-a-a-single-institution-practice-audit
#12
Haowei Linda Sun, Stella Chan, Paul Yenson, Shannon Jackson
Inhibitor risk in nonsevere hemophilia A increases with cumulative factor VIII (FVIII) exposure days and high-risk mutations. A standardized approach to minimize inhibitor risk is warranted. Following establishment of a systematic approach to reduce inhibitor risk in nonsevere hemophilia, we evaluated the uptake of these strategies into clinical practice. All adult males with nonsevere hemophilia A followed by British Columbia Adult Hemophilia Program from 2004 to 2016 were included in this retrospective audit...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/29106782/expression-and-assembly-of-largest-foreign-protein-in-chloroplasts-oral-delivery-of-human-fviii-made-in-lettuce-chloroplasts-robustly-suppresses-inhibitor-formation-in-hemophilia-a-mice
#13
Kwang-Chul Kwon, Alexandra Sherman, Wan-Jung Chang, Aditya Kamesh, Moanaro Biswas, Roland W Herzog, Henry Daniell
Inhibitor formation is a serious complication of factor VIII (FVIII) replacement therapy for the X-linked bleeding disorder hemophilia A and occurs in 20-30% of patients. No prophylactic tolerance protocol currently exists. Although we reported oral tolerance induction using FVIII domains expressed in tobacco chloroplasts, significant challenges in clinical advancement include expression of the full-length CTB-FVIII sequence to cover the entire patient population, regardless of individual CD4(+) T cell epitope responses...
November 6, 2017: Plant Biotechnology Journal
https://www.readbyqxmd.com/read/29106354/characteristics-of-thawed-pooled-cryoprecipitate-stored-at-refrigerated-temperature-for-24-hours
#14
Esther P Soundar, Meredith Reyes, Laura Korte, Arthur Bracey
BACKGROUND: The need for thawed cryoprecipitate is growing. However, according to current guidelines, the shelf-life of pooled thawed cryoprecipitate at room temperature is limited because of possible bacterial contamination and loss of clotting factor activity. Here we assessed microbial growth and retention of clotting activity in cryoprecipitate stored at 4 °C after thawing to see whether its shelf life could be safely extended. MATERIALS AND METHODS: Pooled thawed cryoprecipitate units (n=10) were maintained at room temperature for 6 hours and then placed at 1-6 °C for 18 hours after thawing...
October 30, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/29104003/assessment-of-the-procoagulant-potential-after-laparoscopic-sleeve-gastrectomy-a-potential-role-for-extended-thromboprophylaxis
#15
Amihai Rottenstreich, Ram Elazary, Jonathan B Yuval, Galia Spectre, Geffen Kleinstern, Yosef Kalish
BACKGROUND: Bariatric surgery is associated with increased thromboembolic risk, which may extend well beyond hospital stay. The hemostatic mechanisms implicated in this risk are not well established. OBJECTIVES: We aimed to determine the dynamics of hemostatic changes and procoagulant potential among patients undergoing laparoscopic sleeve gastrectomy, during both the early and late postoperative periods. SETTING: A university hospital. METHODS: Patients who underwent laparoscopic sleeve gastrectomy were recruited consecutively to this study...
September 28, 2017: Surgery for Obesity and related Diseases: Official Journal of the American Society for Bariatric Surgery
https://www.readbyqxmd.com/read/29102549/lipidic-nanoparticles-comprising-of-phosphatidylinositol-mitigate-immunogenicity-and-improve-efficacy-of-recombinant-human-acid-alpha-glucosidase-in-a-murine-model-of-pompe-disease
#16
Jennifer L Schneider, Robert K Dingman, Sathy V Balu-Iyer
Enzyme replacement therapy with recombinant human acid α-glucosidase (rhGAA) is complicated by the formation of anti-rhGAA antibodies, a short circulating half-life, instability in the plasma, and limited uptake into target tissue. Previously, we have demonstrated that phosphatidylinositol (PI) containing liposomes can reduce the immunogenicity and extend plasma survival of Factor VIII (FVIII) in a mouse model of Hemophilia A. In this manuscript we investigate the ability of PI liposomes to be used as a delivery vehicle to overcome the issues that complicate therapy with rhGAA...
November 1, 2017: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/29098718/outgrowing-the-laboratory-diagnosis-of-type-1-von-willebrand-disease-a-two-decade-study
#17
Mouhamed Yazan Abou-Ismail, Gbolahan O Ogunbayo, Michelle Secic, Peter A Kouides
Von Willebrand Factor (VWF) levels are known to increase with age in the general population, but that effect is unclear in von Willebrand's disease (VWD) patients. Thus, it is important to assess the trends of VWF levels with age, and the extent and rate of their normalization in patients with VWD. In a retrospective cohort study, we reviewed the medical records of 126 patients between 1996 and 2016 who met the NHLBI diagnostic criteria for Type 1 VWD or "Low VWF" (LVWF). We followed all their historically documented VWF antigen (VWF:Ag), VWF activity (VWF:RCo), and Factor VIII (FVIII) levels longitudinally over time, correlating data with clinical setting at time of testing...
November 3, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/29093073/midlife-systemic-inflammatory-markers-are-associated-with-late-life-brain-volume-the-aric-study
#18
Keenan A Walker, Ron C Hoogeveen, Aaron R Folsom, Christie M Ballantyne, David S Knopman, B Gwen Windham, Clifford R Jack, Rebecca F Gottesman
OBJECTIVE: To clarify the temporal relationship between systemic inflammation and neurodegeneration, we examined whether a higher level of circulating inflammatory markers during midlife was associated with smaller brain volumes in late life using a large biracial prospective cohort study. METHODS: Plasma levels of systemic inflammatory markers (fibrinogen, albumin, white blood cell count, von Willebrand factor, and Factor VIII) were assessed at baseline in 1,633 participants (mean age 53 [5] years, 60% female, 27% African American) enrolled in the Atherosclerosis Risk in Communities Study...
November 1, 2017: Neurology
https://www.readbyqxmd.com/read/29082639/improved-joint-health-in-subjects-with-severe-haemophilia-a-treated-prophylactically-with-recombinant-factor-viii-fc-fusion-protein
#19
J Oldenburg, R Kulkarni, A Srivastava, J N Mahlangu, V S Blanchette, E Tsao, B Winding, J Dumont, N Jain
INTRODUCTION: Joint arthropathy is the long-term consequence of joint bleeding in people with severe haemophilia. AIM: This study assessed change in joint health over time in subjects receiving recombinant factor VIII Fc fusion protein (rFVIIIFc) prophylaxis. METHODS: ALONG is the phase 3 pivotal study in which the benefit of rFVIIIFc as a prophylactic treatment for bleeding control was shown in previously treated severe haemophilia patients ≥12 years of age (arm 1: 25-65 IU/kg every 3-5 days, arm 2: 65 IU/kg weekly and arm 3: episodic)...
October 30, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29080391/timing-and-severity-of-inhibitor-development-in-recombinant-versus-plasma-derived-factor-viii-concentrates-a-sippet-analysis
#20
F Peyvandi, A Cannavò, I Garagiola, R Palla, P M Mannucci, F R Rosendaal
BACKGROUND: The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe haemophilia A. Recently a randomized trial, the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) demonstrated a two-fold higher risk of inhibitors development in children treated with recombinant FVIII (rFVIII) products than with plasma-derived FVIII (pdFVIII) during the first 50 exposure days (EDs). OBJECTIVE/METHODS: In this post-hoc SIPPET analysis we evaluated the rate of inhibitor incidence over time by every 5 EDs (from 0 to 50 EDs) in patients treated with different classes of FVIII product, made possible by a frequent testing regime...
October 28, 2017: Journal of Thrombosis and Haemostasis: JTH
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