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Journals Journal of Thrombosis and Haem...

Journal of Thrombosis and Haemostasis : JTH

https://read.qxmd.com/read/38614387/three-year-outcomes-of-valoctocogene-roxaparvovec-gene-therapy-for-hemophilia-a
#1
JOURNAL ARTICLE
Bella Madan, Margareth C Ozelo, Priyanka Raheja, Emily Symington, Doris V Quon, Andrew D Leavitt, Steven W Pipe, Gillian Lowe, Gili Kenet, Mark T Reding, Jane Mason, Michael Wang, Annette von Drygalski, Robert Klamroth, Susan Shapiro, Hervé Chambost, Amy L Dunn, Johannes Oldenburg, Sheng-Chieh Chou, Flora Peyvandi, Carolyn M Millar, Dane Osmond, Hua Yu, Ebony Dashiell-Aje, Tara M Robinson, Johnny Mahlangu
BACKGROUND: Valoctocogene roxaparvovec transfers a human factor VIII (FVIII) coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection. OBJECTIVE: Present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial. METHODS: GENEr8-1 enrolled 134 adult males with severe hemophilia A who were receiving FVIII prophylaxis. Efficacy endpoints included annualized bleeding rate (ABR), annualized FVIII utilization (AFU), FVIII activity (chromogenic substrate assay; imputed as 1 IU/dL at baseline and 0 IU/dL after discontinuation), and the Haemophilia-Specific Quality of Life Questionnaire for Adults (Haemo-QOL-A)...
April 11, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38608731/the-proteasome-inhibitor-carfilzomib-exerts-anti-inflammatory-and-anti-thrombotic-effects-on-the-endothelium
#2
JOURNAL ARTICLE
Ahmed Hjazi, Celia Gonzalez Maroto, Michael Appiah, Maria Elena Rodriguez-Gutierrez, Ana Ignat, Golzar Mobayen, Theresa Page, Thomas A J McKinnon
BACKGROUND: Carfilzomib (CFZ) is a second-generation proteasome inhibitor used to treat multiple myeloma. Potent inhibition of the proteasome results in chronic proteotoxic endoplasmic reticulum (ER) stress, leading to apoptosis. While CFZ has improved survival rates in multiple myeloma, it is associated with an increased risk of cardiovascular adverse effects. While this has been putatively linked to cardiotoxicity, CFZ could potentially also exhibit adverse effects on the endothelium...
April 10, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38583717/multicenter-evaluation-of-the-haemostatic-activity-of-emicizumab-in-patients-with-severe-haemophilia-a
#3
JOURNAL ARTICLE
Laurie Josset, Alexandre Leuci, Maissaa Janbain, Anaëlle De-Wreede, Stephanie Desage, Anne Lienhart, Valérie Bin, Dorothée Lambert, Xavier Delavenne, Yesim Dargaud
BACKGROUND: Emicizumab has been approved for the prophylaxis of patients with hemophilia A with or without inhibitors. However, spontaneous and trauma-induced breakthrough bleeds have been reported in patients on emicizumab prophylaxis and no laboratory assay is validated to evaluate the hemostatic activity of emicizumab . OBJECTIVES: The thrombin generation assay (TGA) could be a surrogate marker of the hemostatic efficacy of emicizumab. The correlation between TGA and the methods used to measure emicizumab blood concentration was evaluated in this study...
April 5, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38582384/unmet-needs-and-barriers-in-venous-thromboembolism-education-and-awareness-among-people-living-with-cancer-a-global-survey
#4
JOURNAL ARTICLE
Nicola Potere, Isabelle Mahé, Pantep Angchaisuksiri, Gabriela Cesarman-Maus, Chee Wee Tan, Anila Rashid, Farjah H AlGahtani, Egidio Imbalzano, Nick van Es, Avi Leader, Edeghonghon Olayemi, Ettore Porreca, Fionnuala Ní Áinle, Helen C Okoye, Matteo Candeloro, Didier Mayeur, Luca Valerio, R Cary Clark, Lana A Castellucci, Stefano Barco, Marcello Di Nisio
BACKGROUND: Venous thromboembolism (VTE) is a major preventable cause of morbidity, disability, and mortality in subjects with cancer. A global appraisal of cancer-associated VTE education and awareness is not available. OBJECTIVES: To evaluate VTE-related education, awareness, and unmet needs from the perspective of people living with cancer using a quantitative and qualitative approach. PATIENTS/METHODS: This cross-sectional study used data from an online-based survey covering multidimensional domains of cancer-associated VTE...
April 4, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38580096/the-effect-of-administering-preprocedural-vitamin-k-on-the-international-normalized-ratio-in-patients-anticoagulated-with-acenocoumarol-vitkace-study
#5
JOURNAL ARTICLE
Stella Bijkerk, Harm J Lourens, Wout G N Mares, Corine A van Kampen, Maurits J van der Veen, Henk J Adriaansen, Erik D Ponfoort, Barbara Festen, G Wouter Westendorp, Jörgen M P Rovers, Gerie M C Groot, Hans-Peter R Bootsma, Linde M Amelung, Sander Bins, Gerjo Velders, Remy H H Bemelmans
BACKGROUND: The effect of the vitamin K antagonist (VKA) acenocoumarol on coagulation needs to be reversed when patients undergo an invasive procedure with considerable bleeding risk. A strategy to achieve this is administering oral vitamin K before a procedure while continuing acenocoumarol. OBJECTIVES: To assess the effect on periprocedural International Normalized Ratio (INR)-values and safety using oral vitamin K as anticoagulant reversal method. METHODS: In this prospective cohort study, consecutive patients using acenocoumarol undergoing elective procedures between 2019 and 2022 were included...
April 3, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38574863/communication-from-the-isth-ssc-subcommittee-on-hemostasis-and-malignancy-a-meta-analysis-to-assess-the-risk-of-bleeding-and-thrombosis-following-car-t-cell-therapy
#6
JOURNAL ARTICLE
Poorva Bindal, Rushad Patell, Thita Chiasakul, Mandy N Lauw, Amica Ko, Tzu-Fei Wang, Jeffrey I Zwicker
BACKGROUND: Chimeric antigen receptor T cell (CAR T-cell) therapy is increasingly utilized for treatment of hematologic malignancies. Hematologic toxicities including thrombosis and bleeding complications have been reported. Accurate estimates for thrombotic and bleeding outcomes are lacking. METHODS: We searched MEDLINE, EMBASE, and Cochrane CENTRAL up to February 2022 for studies reporting thrombotic or bleeding outcomes in patients receiving CAR T-cell therapy...
April 2, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38574862/biomarkers-of-bleeding-and-venous-thromboembolism-in-patients-with-acute-leukemia
#7
JOURNAL ARTICLE
Yohei Hisada, Sierra J Archibald, Karan Bansal, Yanjun Chen, Chen Dai, Sindhu Dwarampudi, Nora Balas, Lindsey Hageman, Nigel S Key, Smita Bhatia, Ravi Bhatia, Nigel Mackman, Radhika Gangaraju
BACKGROUND: Coagulopathy and associated bleeding and deep vein thrombosis (DVT) are major causes of morbidity and mortality in patients with acute leukemia. The underlying mechanisms of these complications have not been fully elucidated. OBJECTIVES: To evaluate the associations between biomarker levels and bleeding and DVT in acute leukemia patients. PATIENTS/METHOD: We examined plasma levels of activators, inhibitors and biomarkers of the coagulation and fibrinolytic pathways in patients ≥18 years with newly diagnosed acute leukemia compared to normal controls...
April 2, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38574861/transcriptional-and-functional-profiling-identifies-inflammation-and-endothelial-to-mesenchymal-transition-as-potential-drivers-for-phenotypic-heterogeneity-within-a-cohort-of-endothelial-colony-forming-cells
#8
JOURNAL ARTICLE
Sebastiaan N J Laan, Suzan de Boer, Richard J Dirven, Iris van Moort, Thomas B Kuipers, Hailiang Mei, Ruben Bierings, Jeroen Eikenboom
BACKGROUND: Endothelial colony forming cells (ECFCs) derived from patients can be used to investigate pathogenic mechanisms of vascular diseases like Von Willebrand Disease. Considerable phenotypic heterogeneity has been observed between ECFC clones derived from healthy donors. This heterogeneity needs to be well understood in order to use ECFCs as endothelial models for disease. Therefore, we aim to determine phenotypic and gene expression differences between control ECFCs. METHODS: A total of 34 ECFC clones derived from 16 healthy controls were analyzed...
April 2, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38554936/mitd1-deficiency-leads-to-poor-survival-via-tissue-factor-mediated-coagulation-in-bladder-cancer
#9
JOURNAL ARTICLE
Yuanbin Chen, Wei Jiao, Yonghua Wang, Zhijuan Liang, Liping Wang, Dan Li, Ye Liang, Haitao Niu
BACKGROUND: Patients with cancer are at an increased risk of developing a hypercoagulative phenotype and venous thromboembolism. However, no clinical trial has yet confirmed that anticoagulant therapy improves cancer prognosis, and the mechanism underlying hypercoagulation in patients with bladder cancer is not well understood. OBJECTIVES: We hypothesized that the prognostic genes affect tumor progression via tumor-mediated coagulation. METHODS: We detected the most significant prognostic genes of bladder cancer with The Cancer Genome Atlas dataset, and validated them in two Gene Expression Omnibus and one ArrayExpress datasets...
March 28, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38554935/diagnosis-of-thrombotic-thrombocytopenic-purpura-easy-to-use-fiber-optic-surface-plasmon-resonance-immunoassays-for-automated-adamts13-antigen-and-conformation-evaluation
#10
JOURNAL ARTICLE
Quintijn Bonnez, Charlotte Dekimpe, Tim Bekaert, Edwige Tellier, Gilles Kaplanski, Bérangère S Joly, Agnès Veyradier, Paul Coppo, Jeroen Lammertyn, Claudia Tersteeg, Simon F De Meyer, Karen Vanhoorelbeke
BACKGROUND: Laboratory diagnosis of immune-mediated thrombotic thrombocytopenic purpura (iTTP) remains challenging when ADAMTS13 activity ranges between 10-20%. To prevent misdiagnosis, open ADAMTS13 conformation gained clinical attention as a novel biomarker especially to diagnose acute iTTP in patient with diagnostic undecisive ADAMTS13 activity. Plasma ADAMTS13 conformation analysis corrects for ADAMTS13 antigen with both parameters being characterized in enzyme-linked immunosorbent assay (ELISA)-based reference assays requiring expert technicians...
March 28, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38554934/disparities-in-access-and-timing-of-interventional-therapies-for-pulmonary-embolism-across-the-united-states
#11
JOURNAL ARTICLE
Barret Rush, Jennifer Ziegler, Stephanie Dyck, Surinder Dhaliwal, Owen Mooney, Sylvain Lother, Leo Anthony Celi, Asher Mendelson
BACKGROUND: Interventional therapies (IT) are an emerging treatment modality for pulmonary embolism, however the degree of racial, sex-based and sociodemographic disparities in access and timing is unknown. METHODS: A retrospective cohort study utilizing the Nationwide Inpatient Sample from 2016-2020 included adult patients with PE. The use of IT (mechanical thrombectomy and catheter-directed thrombolysis) was identified via ICD10 code. Early IT was defined as procedure within the first 2 days after admission...
March 28, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38554933/hormonal-contraceptive-use-after-a-first-venous-thrombotic-event-and-the-risk-of-recurrence-in-premenopausal-women
#12
JOURNAL ARTICLE
Judith P L Verlaan, Bernadine H Stegeman, Jasmijn F Timp, Luuk J J Scheres, Linda E Flinterman, Frans M Helmerhorst, Frits R Rosendaal, Suzanne C Cannegieter, Astrid van Hylckama Vlieg
BACKGROUND: Extensive evidence is available on hormonal contraceptive (HC) use and the risk of a first venous thromboembolism (VTE) event. Despite recommendations to discontinue combined hormonal contraceptive (CHC) use, some women continue or start after a first VTE. We aimed to evaluate the VTE recurrence risk of HC use in premenopausal women. METHODS: Premenopausal women with a first VTE, included in the MEGA study between 1999 and 2004, were followed for a recurrence until 2010...
March 28, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38537781/setd2-promotes-megakaryocyte-polyploidization-and-platelet-generation-through-methylation-of-%C3%AE-tubulin
#13
JOURNAL ARTICLE
Lei Chen, Jingkun Liu, Kunying Chen, Yanxun Su, Yihe Chen, Ying Lei, Jia Si, Jie Zhang, Zhaojun Zhang, Weiguo Zou, Xiaohui Zhang, Matthew T Rondina, Qian-Fei Wang, Yueying Li
BACKGROUND: Megakaryocytes (MKs) are polyploid cells responsible for producing ∼1011 platelets daily in humans. Unraveling the mechanisms regulating megakaryopoiesis holds the promise for the production of clinical-grade platelets from stem cells, overcoming significant current limitations in platelet transfusion medicine. Previous work identified that loss of the epigenetic regulator SETD2 was associated with an increased platelet count in mice. However, the role of SETD2 in megakaryopoiesis remains unknown...
March 25, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38537780/extending-venous-thromboembolism-secondary-prevention-with-apixaban-in-cancer-patients-the-eve-trial
#14
JOURNAL ARTICLE
Robert D McBane, Charles L Loprinzi, Tyler Zemla, Alfonso Tafur, Kristen Sanfilippo, Jane Jijun Liu, David A Garcia, James Heun, Krishna Gundabolu, Adedayo A Onitilo, Usha Perepu, Monic R Drescher, Stanislav Henkin, Damon Houghton, Aneel Ashrani, Henny Billett, Shaylene A McCue, Minji K Lee, Jennifer G Le-Rademacher, Waldemar E Wysokinski
PURPOSE: Cancer-associated venous thromboembolism (VTE) management guideline recommendations include continued therapeutic anticoagulation while active cancer persists. The Federal Drug Administration label for apixaban for secondary VTE prevention includes a dose reduction to 2.5 mg twice daily after 6 months of treatment. The study purpose was to determine whether this dose reduction is advisable for cancer-associated VTE. PATIENTS AND METHODS: A randomized, double-blind trial compared apixaban 2...
March 25, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38521192/mathematical-models-of-coagulation-are-we-there-yet
#15
JOURNAL ARTICLE
Matt J Owen, Joy R Wright, Edward G D Tuddenham, John R King, Alison H Goodall, Joanne L Dunster
BACKGROUND: Mathematical models of coagulation have been developed to mirror thrombin generation in plasma, with the aim of investigating how variation in coagulation factor levels regulate haemostasis. However, current models vary in the reactions they capture, in reaction rates used, and their validation is restricted by a lack of large coherent datasets, resulting in questioning of their utility. OBJECTIVES AND METHODS: To address this debate, we systematically assessed current models against a large dataset, using plasma coagulation factor levels from 348 individuals with normal haemostasis as inputs, and compared model predictions with measured thrombin generation...
March 21, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38518897/recent-advances-in-vascular-thiol-isomerases-and-redox-systems-in-platelet-function-and-thrombosis
#16
REVIEW
David W Essex, Lu Wang
There have been substantial advances in vascular protein disulfide isomerases (PDIs) in platelet function and thrombosis in recent years. There are four known prothrombotic thiol isomerases, PDI, ERp57, ERp72, ERp46 and one antithrombotic PDI, transmembrane protein 1 (TMX1). A sixth PDI, ERp5, may exhibit either pro- or anti-thrombotic properties in platelets. Studies on ERp46 in platelet function and thrombosis provide insight into the mechanisms by which these enzymes function. ERp46-catalyzed disulfide cleavage in the αIIbβ3 platelet integrin occurs prior to PDI-catalyzed events to maximally support platelet aggregation...
March 20, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38518896/standardisation-of-definition-and-management-for-bleeding-disorder-of-unknown-cause-communication-from-the-ssc-of-the-isth
#17
Ross I Baker, Philip Choi, Nicola Curry, Johanna Gebhart, Keith Gomez, Yvonne Henskens, Floor Heubel-Moenen, Paula James, Rezan Abdul Kadir, Peter Kouides, Michelle Lavin, Marie Lordkipanidze, Gillian Lowe, Andrew Mumford, Nicola Mutch, Michael Nagler, Maha Othman, Ingrid Pabinger, Robert Sidonio, Will Thomas, James S O'Donnell
In many patients referred with significant bleeding phenotype, laboratory testing fails to define any hemostatic abnormalities. Clinical practise with respect to diagnosis and management of this patient cohort poses significant clinical challenges. We recommend that bleeding history in these patients should be objectively assessed using the ISTH bleeding assessment tool (BAT). Patients with increased BAT scores should progress to hemostasis laboratory testing. To diagnose BDUC, normal complete blood count, prothrombin time, activated partial thromboplastin time, thrombin time, von Willebrand factor antigen, von Willebrand factor function; coagulation factors VIII, IX and XI and platelet light transmission aggregometry (LTA) should be the minimum laboratory assessment...
March 20, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38518895/l-arginine-supplementation-protects-against-thrombosis-and-renal-dysfunction-in-mice-treated-with-the-cox-2-inhibitor-parecoxib
#18
LETTER
Ricky Vaja, Maria Lopes-Pires, Fisnik Shala, Neringa Cypaite, Maria Vinokurova, Plinio Ferreira, Jane A Mitchell, Nicholas S Kirkby
No abstract text is available yet for this article.
March 20, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38521666/will-the-real-authors-please-stand-up
#19
EDITORIAL
Ton Lisman, Suzanne C Cannegieter
No abstract text is available yet for this article.
March 18, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38508397/a-mouse-model-of-the-protease-activated-receptor-4-par4-pro310leu-variant-has-reduced-platelet-reactivity
#20
JOURNAL ARTICLE
Xu Han, Elizabeth A Knauss, Maria de la Fuente, Wei Li, Ronald A Conlon, David F LePage, Weihong Jiang, Stephanie A Renna, Steven E McKenzie, Marvin T Nieman
BACKGROUND: Protease activated receptor 4 (PAR4) mediates thrombin signaling on platelets and other cells. Our recent structural studies demonstrated a single nucleotide polymorphism in extracellular loop 3 (ECL3), PAR4-P310L (rs2227376) leads to a hypo-reactive receptor. OBJECTIVES: The goal of this study was to determine how the hypo-reactive PAR4 variant in ECL3 impacts platelet function in vivo using a novel knock-in mouse model (PAR4-322L). METHODS: A point mutation was introduced into the PAR4 gene, F2rl3, via CRISPR/Cas9 to create PAR4-P322L, the mouse homolog to human PAR4-P310L...
March 18, 2024: Journal of Thrombosis and Haemostasis: JTH
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