11,439 results match your criteria Factor IX


Coagulation in liver surgery: an observational haemostatic profile and thromboelastography study.

ANZ J Surg 2020 May 26. Epub 2020 May 26.

Department of Surgical Oncology, Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia.

Background: International normalized ratio (INR) is used as a marker of the haemostatic status following liver resection. However, the impact of liver resection on haemostasis is complex and beyond what can be measured by INR. This study aimed to prospectively assess haemostatic profile following liver resection and determine if INR measurement can safely guide post-operative thromboprophylaxis. Read More

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http://dx.doi.org/10.1111/ans.15912DOI Listing

Identification of Novel Carbonic Anhydrase IX Inhibitors Using High-Throughput Screening of Pooled Compound Libraries by DNA-Linked Inhibitor Antibody Assay (DIANA).

SLAS Discov 2020 May 26:2472555220918836. Epub 2020 May 26.

Institute of Organic Chemistry and Biochemistry of the Czech Academy of Sciences, Prague, Czech Republic.

The DNA-linked inhibitor antibody assay (DIANA) has been recently validated for ultrasensitive enzyme detection and for quantitative evaluation of enzyme inhibitor potency. Here we present its adaptation for high-throughput screening of human carbonic anhydrase IX (CAIX), a promising drug and diagnostic target. We tested DIANA's performance by screening a unique compound collection of 2816 compounds consisting of lead-like small molecules synthesized at the Institute of Organic Chemistry and Biochemistry (IOCB) Prague ("IOCB library"). Read More

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http://dx.doi.org/10.1177/2472555220918836DOI Listing

Factor Analysis of the Administrator-Research Campus Climate Collaborative (ARC3) Survey.

Health Educ Behav 2020 Jun;47(1_suppl):54S-69S

Texas Woman's University, Denton, TX, USA.

Sexual assault, dating violence, stalking, and sexual harassment present serious challenges to the health and well-being of college students across the United States. An estimated one in five female college students is sexually assaulted during college. Under the federal protection of Title IX, institutions of higher education are responsible for addressing sexual harassment and sexual misconduct as forms of discrimination, thus campus climate surveys are increasingly being conducted in campus settings to examine student beliefs and experiences about sexual assault, dating violence, stalking, sexual harassment, and related campus prevention and response efforts. Read More

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http://dx.doi.org/10.1177/1090198120911613DOI Listing

Pharmacokinetics for haemophilia treaters: Meaning of PK parameters, interpretation pitfalls, and use in the clinic.

Thromb Res 2020 May 11;192:52-60. Epub 2020 May 11.

Unité d'Hémostase Clinique, Hôpital Cardiologique Louis Pradel, Université Lyon 1, Lyon, France.

Replacement therapy with concentrates of factor VIII or IX remains the gold standard for severe haemophilia management. The recent development of clotting factor products with extended half-life, widely available on the market since 2 years, facilitates adherence, improves considerably the patients' quality of life, and simplifies the management of breakthrough bleedings or surgery. These molecules have also brought to the limelight the concepts of optimization and personalization of anti-haemophilic prophylaxis. Read More

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http://dx.doi.org/10.1016/j.thromres.2020.05.005DOI Listing

ZJ01, a Small Molecule Inhibitor of the Kelch-Like ECH-Associated Protein 1-Nuclear Factor Erythroid 2-Related Factor 2 (Keap1-Nrf2) Protein-Protein Interaction, Reduces Hyperoxic Acute Lung Injury in a Mouse Model.

Med Sci Monit 2020 May 21;26:e920467. Epub 2020 May 21.

Department of Cardiovascular Surgery, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China (mainland).

BACKGROUND Hyperoxic acute lung injury (ALI) is a complication of ventilation in patients with respiratory failure. Nuclear factor erythroid-2-related factor 2 (Nrf2) has an important role in ALI. Kelch-like ECH-associated protein 1 (Keap1) binds to Nrf2. Read More

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http://dx.doi.org/10.12659/MSM.920467DOI Listing

The rapid Bethesda assay is equivalent to the standard Bethesda assay for detection of factor IX inhibitors in patients with severe haemophilia B.

Haemophilia 2020 May 18. Epub 2020 May 18.

Quest Diagnostics, Secaucus, NJ, USA.

Introduction: The time-dependent nature of factor VIII (FVIII) inhibitors is well described, and the standard FVIII Bethesda assay used to measure inhibitors incorporates a 2-hour incubation. Despite case reports and reviews describing the immediate-acting nature of factor IX (FIX) inhibitors, many coagulation laboratories continue to use a traditional prolonged incubation for FIX Bethesda assays. To our knowledge, a comprehensive evaluation of the FIX Bethesda assay without incubation has not been reported. Read More

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http://dx.doi.org/10.1111/hae.14028DOI Listing

Fibroblast Growth Factor 23 and Blood Pressure in Older Adults: The Health, Aging, and Body Composition Study.

Hypertension 2020 May 18:HYPERTENSIONAHA12014703. Epub 2020 May 18.

University of Alabama at Birmingham (O.M.G.).

FGF-23 (fibroblast growth factor 23) regulates phosphorus and vitamin D. Elevated FGF-23 is associated with incident hypertension in young- and middle-aged adults, but there is limited data in older adults. Serum FGF-23 was measured using an intact ELISA assay in 2496 participants of the Healthy Aging and Body Composition Study. Read More

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http://dx.doi.org/10.1161/HYPERTENSIONAHA.120.14703DOI Listing

Metabolic reprogramming and angiogenesis in primary cutaneous Merkel cell carcinoma: expression of hypoxia inducible factor-1α and its central downstream factors.

J Eur Acad Dermatol Venereol 2020 May 12. Epub 2020 May 12.

Dermatopathology, Bodensee, Friedrichshafen, Germany.

Background: Metabolic reprogramming and altered gene expression mediated by hypoxia inducible-factors play crucial roles during tumour growth and progression. Nevertheless, studies analysing the expression of hypoxia inducible factor-1α, and its downstream targets in Merkel cell carcinoma (MCC) are lacking but are warranted to shed more light on MCC pathogenesis and to potentially provide new therapeutic options.

Objectives: To analyse the immunohistochemical expression of hypoxia-inducible factor-1α (HIF-1α), vascular endothelial growth factor-A (referred to as VEGF throughout the manuscript), VEGF receptor-2 (VEGFR-2), VEGF receptor-3 (VEGFR-3), glucose transporter-1 (Glut-1), monocarboxylate transporter 4 (MCT4), and carbonic anhydrase IX (CAIX) in primary cutaneous MCC. Read More

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http://dx.doi.org/10.1111/jdv.16600DOI Listing

NFI transcription factors provide chromatin access to maintain stem cell identity while preventing unintended lineage fate choices.

Nat Cell Biol 2020 May 11. Epub 2020 May 11.

Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Development, Howard Hughes Medical Institute, The Rockefeller University, New York, NY, USA.

Tissue homeostasis and regeneration rely on resident stem cells (SCs), whose behaviour is regulated through niche-dependent crosstalk. The mechanisms underlying SC identity are still unfolding. Here, using spatiotemporal gene ablation in murine hair follicles, we uncover a critical role for the transcription factors (TFs) nuclear factor IB (NFIB) and IX (NFIX) in maintaining SC identity. Read More

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http://dx.doi.org/10.1038/s41556-020-0513-0DOI Listing

Whole Exome Sequencing with Comprehensive Gene Set Analysis Identified a Biparental-Origin Homozygous c.509G>A Mutation in Gene Clustered in Two Taiwanese Families Exhibiting Fetal Skeletal Dysplasia during Prenatal Ultrasound.

Diagnostics (Basel) 2020 May 7;10(5). Epub 2020 May 7.

Department of Genomic Medicine and Center for Medical Genetics, Changhua Christian Hospital, Changhua 50046, Taiwan.

Skeletal dysplasia (SD) is a complex group of bone and cartilage disorders often detectable by fetal ultrasound, but the definitive diagnosis remains challenging because the phenotypes are highly variable and often overlap among different disorders. The molecular mechanisms underlying this condition are also diverse. Hundreds of genes are involved in the pathogenesis of SD, but most of them are yet to be elucidated, rendering genotyping almost infeasible except those most common such as fibroblast growth factor receptor 3 (), collagen type I alpha 1 chain (), collagen type I alpha 2 chain (), diastrophic dysplasia sulfate transporter (), and SRY-box 9 (). Read More

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http://dx.doi.org/10.3390/diagnostics10050286DOI Listing

Surface engineering for efficient capture of circulating tumor cells in renal cell carcinoma: From nanoscale analysis to clinical application.

Biosens Bioelectron 2020 Aug 1;162:112250. Epub 2020 May 1.

Pharmaceutical Sciences Division, School of Pharmacy, University of Wisconsin, Madison, Madison, WI, 53705, USA; Yonsei Frontier Lab and Department of Pharmacy, Yonsei University, Seoul, 03722, South Korea. Electronic address:

Sensitive detection of circulating tumor cells (CTCs) from patients' peripheral blood facilitates on-demand monitoring of tumor progression. However, clinically significant capture of renal cell carcinoma CTCs (RCC-CTCs) remains elusive due to their heterogenous surface receptor expression. Herein, a novel capture platform is developed to detect RCC-CTCs through integration of dendrimer-mediated multivalent binding, a mixture of antibodies, and biomimetic cell rolling. Read More

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http://dx.doi.org/10.1016/j.bios.2020.112250DOI Listing

Implantable venous access devices in children with severe hemophilia: a tertiary pediatric institutional experience.

Pediatr Radiol 2020 May 11. Epub 2020 May 11.

Department of Radiology, The Children's Hospital of Philadelphia, 34th Street and Civic Center Boulevard, Philadelphia, PA, 19104, USA.

Background: Clotting factor replacement forms the pillar of treatment for children with hemophilia. Most children can be treated using peripheral venipuncture, but very young children and children with poor venous access might require a central venous catheter. Short-term and long-term complications of implantable venous access device placement (also known as port placement) can result in important morbidity and mortality in children with hemophilia. Read More

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http://dx.doi.org/10.1007/s00247-020-04668-3DOI Listing

Association between NR1I2 polymorphisms and susceptibility to anti-tuberculosis drug-induced hepatotoxicity in an Eastern Chinese Han population: A case-control study.

Infect Genet Evol 2020 May 7;83:104349. Epub 2020 May 7.

Department of Epidemiology and Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China. Electronic address:

Objective: Anti-tuberculosis drug-induced hepatotoxicity (ATDH) is a serious adverse drug reaction, and its pathogenic mechanism is still largely unknown. Pregnane X receptor (PXR, encoded by the NR1I2 gene) is a ligand-dependent transcription factor, and rifampicin is a human PXR-specific activator. Rifampicin and isoniazid co-therapy targets porphyrin biosynthesis via PXR and results in hepatic protoporphyrin IX accumulation and subsequent liver injury. Read More

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http://dx.doi.org/10.1016/j.meegid.2020.104349DOI Listing

Identification of novel glycosylation events on human serum-derived factor IX.

Glycoconj J 2020 May 6. Epub 2020 May 6.

School of Chemistry and Molecular Biosciences, The University of Queensland, St Lucia, QLD, 4072, Australia.

Human Factor IX is a highly post-translationally modified protein that is an important clotting factor in the blood coagulation cascade. Functional deficiencies in Factor IX result in the bleeding disorder haemophilia B, which is treated with plasma-derived or recombinant Factor IX concentrates. Here, we investigated the post-translational modifications of human serum-derived Factor IX and report previously undescribed O-linked monosaccharide compositions at serine 141 and a novel site of glycosylation. Read More

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http://dx.doi.org/10.1007/s10719-020-09922-2DOI Listing

Heme oxygenase-1 alleviated non-alcoholic fatty liver disease via suppressing ROS-dependent endoplasmic reticulum stress.

Life Sci 2020 May 3;253:117678. Epub 2020 May 3.

Department of Traditional and Western Medical Hepatology, Third Hospital of Hebei Medical University, 050051 Shijiazhuang, China; Hebei Provincial Key Laboratory of Liver Fibrosis in Chronic Liver Diseases, China. Electronic address:

Aims: The endoplasmic reticulum (ER) stress response plays a crucial role in the development of nonalcoholic steatohepatitis (NASH). Heme oxygenase-1 (HO-1) exerts beneficial effects against oxidative injury in NASH. This study is aimed to clarify whether HO-1 is an effective therapeutic strategy for NASH via regulation of ER stress. Read More

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http://dx.doi.org/10.1016/j.lfs.2020.117678DOI Listing

HDAC inhibitor protects chronic cerebral hypoperfusion and oxygen-glucose deprivation injuries via H3K14 and H4K5 acetylation-mediated BDNF expression.

J Cell Mol Med 2020 May 6. Epub 2020 May 6.

Taipei Neuroscience Institute, Taipei Medical University, Taipei, Taiwan.

Vascular dementia (VaD) is the second most common cause of dementia, but the treatment is still lacking. Although many studies have reported that histone deacetylase inhibitors (HDACis) confer protective effects against ischemic and hypoxic injuries, their role in VaD is still uncertain. Previous studies shown, one HDACi protected against cognitive decline in animals with chronic cerebral hypoperfusion (CCH). Read More

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http://dx.doi.org/10.1111/jcmm.15358DOI Listing

Long-Term Safety and Efficacy of Nonacog Beta Pegol (N9-GP) Administered for at Least 5 Years in Previously Treated Children with Hemophilia B.

Thromb Haemost 2020 May 5;120(5):737-746. Epub 2020 May 5.

Foundation IRCCS Cá Granda, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Maggiore Hospital Policlinico, Milan, Italy.

Long-term safety and efficacy data of extended half-life factor IX (FIX) prophylaxis in children with hemophilia B (HB) are sparse. paradigm 5 is a multinational, open-label, single-arm, phase III trial assessing once-weekly (40 IU/kg) prophylactic nonacog beta pegol (N9-GP) in previously treated patients (PTPs) aged ≤ 12 years with HB (FIX activity ≤ 2%). Primary endpoint: incidence of anti-FIX inhibitory antibodies (≥ 0. Read More

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http://dx.doi.org/10.1055/s-0040-1709521DOI Listing

The Patient Experience of Gene Therapy for Hemophilia: Qualitative Interviews with Trial Patients.

Patient Prefer Adherence 2020 22;14:767-770. Epub 2020 Apr 22.

Department for Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Klinikum im Friedrichshain, Berlin D - 10249, Germany.

Background: The phase ½ hemophilia B clinical trial (AMT-060) demonstrated stable endogenous FIX levels after 3.5 years (mean FIX activity between 5.1% and 7. Read More

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http://dx.doi.org/10.2147/PPA.S239810DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184858PMC

Registries and databases-A European perspective.

Authors:
Rolf C R Ljung

Haemophilia 2020 Apr;26 Suppl 3:26-28

Department of Clinical Sciences - Paediatrics, Lund University, Lund and Malmö, Sweden.

Registries will enable cohort studies to be performed, which are usually considered to be the best quality of observational studies. The quality of data of registries can be increased if is it possible to merge results ('crosstalk') between registries. A prerequisite for that is an agreed uniform core set of data to be collected and uniform definitions on the items to be collected. Read More

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http://dx.doi.org/10.1111/hae.13920DOI Listing

Gene therapy for haemophilia.

Cochrane Database Syst Rev 2020 04 28;4:CD010822. Epub 2020 Apr 28.

Hematology Department, St Jude Children's Research Hospital, Memphis, Tennessee, USA.

Background: Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy for haemophilia is a curative treatment modality currently under investigation. Read More

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http://dx.doi.org/10.1002/14651858.CD010822.pub4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7192367PMC

Real-World Utilisation and Bleed Rates in Patients with Haemophilia B Who Switched to Recombinant Factor IX Fusion Protein (rIX-FP): A Retrospective International Analysis.

Adv Ther 2020 Jun 24;37(6):2988-2998. Epub 2020 Apr 24.

Hemophilia and Thrombosis Center, University Hospital of Bari, Bari, Italy.

Introduction: Despite the well-documented benefits of prophylaxis, treatment burden is still a barrier to adherence in patients with haemophilia. An extended half-life fusion protein linking recombinant FIX (rFIX) with human albumin (rIX-FP) has been developed for the treatment of patients with haemophilia B and is indicated for dosing up to every 14 days. This analysis evaluated real-world outcomes in patients switching to rIX-FP from the previous FIX product in Italy, Belgium and the UK. Read More

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http://dx.doi.org/10.1007/s12325-020-01300-6DOI Listing

Enhancing the antitumor activity of an engineered TRAIL-coated oncolytic adenovirus for treating acute myeloid leukemia.

Signal Transduct Target Ther 2020 Apr 24;5(1):40. Epub 2020 Apr 24.

National Engineering Laboratory for AIDS Vaccine, School of Life Sciences, Jilin University, Changchun, 130012, China.

The use of oncolytic viruses has emerged as a promising therapeutic approach due to the features of these viruses, which selectively replicate and destroy tumor cells while sparing normal cells. Although numerous oncolytic viruses have been developed for testing in solid tumors, only a few have been reported to target acute myeloid leukemia (AML) and overall patient survival has remained low. We previously developed the oncolytic adenovirus rAd5pz-zTRAIL-RFP-SΔ24E1a (A4), which carries the viral capsid protein IX linked to tumor necrosis factor-related apoptosis-inducing ligand (TRAIL) and results in increased infection of cancer cells and improved tumor targeting. Read More

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http://dx.doi.org/10.1038/s41392-020-0135-9DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7181830PMC

Human Serum Albumin Facilitates Heme-Iron Utilization by Fungi.

mBio 2020 Apr 21;11(2). Epub 2020 Apr 21.

Department of Molecular Microbiology, B. Rappaport Faculty of Medicine, Technion-I.I.T. and the Rappaport Institute for Research in the Medical Sciences, Haifa, Israel

A large portion of biological iron is found in the form of an iron-protoporphyrin IX complex, or heme. In the human host environment, which is exceptionally poor in free iron, heme iron, particularly from hemoglobin, constitutes a major source of iron for invading microbial pathogens. Several fungi were shown to utilize free heme, and , a major opportunistic pathogen, is able both to capture free heme and to extract heme from hemoglobin using a network of extracellular hemophores. Read More

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http://dx.doi.org/10.1128/mBio.00607-20DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7175094PMC

Measurements of eftrenonacog alfa by 19 different combinations reagents/instrument: A single-centre study.

Haemophilia 2020 May 20;26(3):543-552. Epub 2020 Apr 20.

Service d'Hématologie Biologique, CHU Clermont-Ferrand, Clermont-Ferrand, France.

Introduction: Recombinant factor IX Fc fusion protein (rFIXFc) is an extended half-life concentrate for the treatment of haemophilia B (HB). rFIXFc activity monitoring is crucial in several clinical situations. However, differences were observed between one-stage clotting (OSC) and chromogenic assays, but not for all factor IX (FIX) concentrations. Read More

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http://dx.doi.org/10.1111/hae.14003DOI Listing

Treatment with radiosynoviorthesis in hemophilic patients with and without inhibitor.

BMC Pediatr 2020 Apr 20;20(1):173. Epub 2020 Apr 20.

Medical Research Unit in Clinical Epidemiology, UMAE Hospital de Pediatría "Dr. Silvestre Frenk Freund", Centro Médico Nacional Siglo XXI, Instituto Mexicano del Seguro Social, Mexico City, Mexico.

Background: Spontaneous bleedings occurring into joints (hemarthrosis) are the most common manifestations of hemophilia and causes severe joint damage ultimately resulting in joint disfunction known as hemophilic arthropathy. Among available therapeutic options for reducing recurrent hemarthrosis-associated damage, radiosynoviorthesis (RS) has proven effective in improving joint function.

Aim: To assess the impact of RS with Yttrium(90) citrate (C-Y(90)) on frequency of hemarthroses and joint function in a group of pediatric patients. Read More

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http://dx.doi.org/10.1186/s12887-020-02071-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7168877PMC

Performance of FIX Extended Half-life product measurements in External Quality Control Assessment programmes.

J Thromb Haemost 2020 Apr 20. Epub 2020 Apr 20.

Dept. Haematology, Erasmus University Medical Centre Rotterdam, Rotterdam, the Netherlands.

Background: Patients with haemophilia B are increasingly treated with extended half-life (EHL) Factor IX (FIX) concentrates. For the laboratory, introduction of these EHL concentrates presents a major challenge. To understand the variation in FIX activity levels, all available diagnostic assays need to be directly compared. Read More

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http://dx.doi.org/10.1111/jth.14847DOI Listing

Relation of Plasma Renin Activity to Subclinical Peripheral and Coronary Artery Disease (from the Multiethnic Study of Atherosclerosis).

Am J Cardiol 2020 Apr 2. Epub 2020 Apr 2.

Division of Preventive Medicine, Department of Family Medicine and Public Health, University of California San Diego, La Jolla, California; Department of Surgery and Abdelmalek, Department of Medicine at the VA, Veterans' Affairs Hospital, San Diego, California; Division of Nephrology-Hypertension, Department of Medicine, University of California San Diego, La Jolla, California.

Experimental studies support a link between activation of the renin-angiotensin-aldosterone system and cardiovascular disease (CVD). The relationship with subclinical atherosclerosis is uncertain. Among 1,699 individuals without prevalent CVD from the Multiethnic Study of Atherosclerosis, we measured plasma renin activity (PRA) and aldosterone. Read More

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http://dx.doi.org/10.1016/j.amjcard.2020.03.022DOI Listing

[Effect of Fei-Liu-Ping ointment combined with cyclophosphamide on lung cancer cell proliferation and acidic microenvironment].

Authors:
L Geng J Lv J Fan

Beijing Da Xue Xue Bao Yi Xue Ban 2020 Apr;52(2):247-253

College of Management, Henan University of Chinese Medicine, Zhengzhou 450008, China.

Objective: To observe the effects of Fei-Liu-Ping ointment and chemotherapy on mice with lung cancer, and to explore the inherent mechanism of action from the point of acidic microenvironment and apoptosis.

Methods: First of all, the Lewis lung cancer transplanted mouse model was established. Therefore, they were treated by Fei-Liu-Ping ointment, cyclophosphamide, Fei-Liu-Ping ointment + cyclophosphamide and the saline as control. Read More

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Hemophilia C: A Case Report With Updates on Diagnosis and Management of a Rare Bleeding Disorder.

J Hematol 2019 Sep 30;8(3):144-147. Epub 2019 Sep 30.

Division of Hematology Oncology, Department of Medicine, Allegheny General Hospital, Pittsburgh, PA 15212, USA.

Hemophilia C or factor XI deficiency is a rare clotting disorder with prevalence of only 1 per 1 million. A 24-year-old male with multiple abdominal surgeries complicated by wound infections and poor healing was admitted to plastic surgery service for an elective abdominoplasty. Hematology was consulted for increased intraoperative and postoperative bleeding. Read More

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http://dx.doi.org/10.14740/jh522DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7153668PMC
September 2019

Coagulation Parameters in Adult Patients With Type-1 Gaucher Disease.

J Hematol 2019 Sep 30;8(3):121-124. Epub 2019 Sep 30.

Internal Medicine Department, Tours University Hospital, University of Tours, UMR INSERM 1253, 37044 Tours, France.

Background: Gaucher disease is a rare inborn error of lysosomal metabolism, characterized by lysosomal storage of the β-glucosylceramide. Bleedings observed in type-1 Gaucher disease (GD1) are commonly attributed to a low platelet count, but they can also occur when the platelet count is normal or slightly low. Abnormal platelet function has been described and deficiencies in coagulation factors too, such as factors II, V, VII, VIII, IX, X, XI, XII, and von Willebrand factor. Read More

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http://dx.doi.org/10.14740/jh543DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7153659PMC
September 2019

Nuclear factor IX promotes glioblastoma development through transcriptional activation of Ezrin.

Oncogenesis 2020 Apr 14;9(4):39. Epub 2020 Apr 14.

Department of Neurosurgery, Shenzhen Hospital, Southern Medical University, Shenzhen, Guangdong, China.

Enhanced migration is pivotal for the malignant development of glioblastoma (GBM), but the underlying molecular mechanism that modulates the migration of the GBM cells remains obscure. Here we show that nuclear factor IX (NFIX) is significantly upregulated in human GBM lesions compared with normal or low-grade gliomas. NFIX deficiency impairs the migration of GBM cells and inhibits the tumor growth in the hippocampus of immunodeficient nude mice. Read More

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http://dx.doi.org/10.1038/s41389-020-0223-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7156762PMC

Development and evaluation of the population pharmacokinetic models for FVIII and FIX concentrates of the WAPPS-Hemo project.

Haemophilia 2020 May 13;26(3):384-400. Epub 2020 Apr 13.

School of Pharmacy, University of Waterloo, Waterloo, ON, Canada.

Background: The Web-Accessible Population Pharmacokinetic Service (WAPPS) project generates individually predicted pharmacokinetic (PK) profiles and tailored prophylactic treatment regimens for haemophilic patients, which rely on a set of population PK (PopPK) models providing concentrate-specific priors for the Bayesian forecasting methodology.

Aim: To describe the predictive performance of the WAPPS PopPK models in use on the WAPPS-Hemo platform.

Methods: Data for modelling include dense PK data obtained from industry sponsored and independent PK studies, and dense and sparse data accumulated through WAPPS-Hemo. Read More

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http://dx.doi.org/10.1111/hae.13977DOI Listing

Development of an Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release.

Mol Ther Methods Clin Dev 2020 Jun 17;17:581-588. Epub 2020 Mar 17.

Baxalta Innovations GmbH, a member of the Takeda group of companies, Uferstraße. 15, A-2304 Orth an der Donau, Austria.

Gene therapy product release requires reliable and consistent demonstration of biopotency. In hemophilia B vectors, this is usually determined by measuring the plasma levels of the expressed human factor IX (FIX) transgene product in FIX knockout mice. To circumvent this laborious assay, we developed an method in which the HepG2 human liver cell line was infected with the vector, and the resulting FIX activity was determined in the conditioned medium using a chromogenic assay. Read More

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http://dx.doi.org/10.1016/j.omtm.2020.03.013DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7139127PMC

Factor VIII and IX assays for post-infusion monitoring in hemophilia patients: Guidelines from the French BIMHO group (GFHT).

Eur J Haematol 2020 Apr 10. Epub 2020 Apr 10.

Service d'Hématologie Hémostase, Hospices Civils de Lyon, Bron, France.

Replacement therapy with plasma-derived or recombinant FVIII and FIX (pdFVIII/pdFIX or rFVIII/rFIX) concentrates is the standard of treatment in patients with haemophilia A and B, respectively. Measurement of factor VIII (FVIII:C) or factor IX (FIX:C) levels can be done by one-stage clotting assay (OSA) or chromogenic substrate assay (CSA). The French study group on the Biology of Hemorrhagic Diseases (a collaborative group of the GFHT and MHEMO network) presents a literature review and proposals for the monitoring of FVIII:C and FIX:C levels in treated haemophilia A and B patients, respectively. Read More

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http://dx.doi.org/10.1111/ejh.13423DOI Listing

Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B.

Blood Coagul Fibrinolysis 2020 Apr;31(3):186-192

Pfizer Inc, New York, New York, USA.

: Standard-of-care treatment for haemophilia A or B is to maintain adequate coagulation factor levels through clotting factor administration. The current study aimed to evaluate annualised bleeding rates (ABR) and treatment adherence for haemophilia A or B patients receiving standard half-life (SHL) vs. extended half-life (EHL) factor replacement products. Read More

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http://dx.doi.org/10.1097/MBC.0000000000000885DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7195855PMC

Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.

PLoS Genet 2020 Apr 8;16(4):e1008690. Epub 2020 Apr 8.

Institute of Virology, Hannover Medical School, Hannover, Germany.

Loss-of-function mutations in the human coagulation factor 9 (F9) gene lead to hemophilia B. Here, we dissected the consequences and the pathomechanism of a non-coding mutation (c.2545A>G) in the F9 3' untranslated region. Read More

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http://dx.doi.org/10.1371/journal.pgen.1008690DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7141619PMC

Computational discovery of plant-based inhibitors against human carbonic anhydrase IX and molecular dynamics simulation.

J Biomol Struct Dyn 2020 Apr 29:1-17. Epub 2020 Apr 29.

Genetic Engineering and Biotechnology, University of Rajshahi, Rajshahi, Bangladesh.

Carbonic anhydrase IX (hCAIX) is a membrane-spanning metalloenzyme, encoded by gene, which can lead to various carcinomas if upregulated. Due to its overexpression in many cancer tissues, hCAIX has become a promising target for developing anticancer therapeutics. Furthermore, several classes of small-molecules have shown to inhibit the hCAIX expression. Read More

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http://dx.doi.org/10.1080/07391102.2020.1753579DOI Listing

The Liver That Cured Christmas: Case Report of Orthotopic Liver Transplant in a Patient with Hemophilia B.

Case Rep Transplant 2020 18;2020:7873803. Epub 2020 Mar 18.

Department of Oncology, Houston Methodist Hospital, USA.

Herein, we discuss a case of a 39-year-old male with hemophilia B, who developed end-stage liver disease secondary to nonalcoholic steatohepatitis, that underwent orthotopic liver transplantation (OLT) as a curative means for his liver disease and coagulation disorder. Existing case reports have demonstrated favorable outcomes in patients outside of the United States who received continuous infusions of recombinant factor IX replacement in the perioperative setting after liver transplant. Given limitations in the stability of the recombinant factor IX products in the United States, a dosing strategy was comprised of once daily bolus dosing to achieve satisfactory factor IX levels. Read More

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http://dx.doi.org/10.1155/2020/7873803DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7104118PMC

Effects of emicizumab on APTT, one-stage and chromogenic assays of factor VIII in artificially spiked plasma and in samples from haemophilia A patients with inhibitors.

Haemophilia 2020 May 6;26(3):536-542. Epub 2020 Apr 6.

Department of Coagulation, Sheffield Haemophilia and Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, UK.

Introduction: Emicizumab (Hemlibra, Roche-Chugai) is a recombinant humanized bispecific IgG4 antibody which mimics some of the actions of activated factor VIII (FVIIIa) by binding to factor X (FX) and activated factor IX (FIXa) to activate FX.

Aim: To evaluate the effect of emicizumab on the APTT, standard one-stage APTT-based FVIII activity assay (sOSA) using plasma calibrators, modified OSA (mOSA) using r Diagnostics emicizumab specific calibrator and chromogenic FVIII assays. Tests were performed on plasma artificially spiked with emicizumab and from four severe haemophilia A (SHA) patients treated with emicizumab. Read More

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http://dx.doi.org/10.1111/hae.13990DOI Listing

The Australian experience with switching to extended half-life factor VIII and IX concentrates: On behalf of the Australian Haemophilia Centre Directors' Organisation.

Haemophilia 2020 May 3;26(3):529-535. Epub 2020 Apr 3.

Australian Haemophilia Centre Directors Organisation (AHCDO), Melbourne, Vic., Australia.

Introduction: Extended half-life (EHL) factor (F) VIII and FIX concentrates became available to selected haemophilia A (HA) and haemophilia B (HB) patients in Australia in March 2018.

Aim: To determine factor utilization and bleeding outcomes during the first 6 months of prophylaxis with EHL concentrates, and compare it to the last 6 months of prophylaxis with standard half-life (SHL) concentrates.

Methods: A national, retrospective study was performed using data extracted from the Australian Bleeding Disorders Registry (ABDR). Read More

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http://dx.doi.org/10.1111/hae.13970DOI Listing

Novel Insights and New Developments Regarding Coagulation Revealed by Studies of the Anti-Factor IXa (Activated Factor IX)/Factor X Bispecific Antibody, Emicizumab.

Arterioscler Thromb Vasc Biol 2020 May 2;40(5):1148-1154. Epub 2020 Apr 2.

From the Department of Pediatrics (K.Y., K.N.), Nara Medical University, Kashihara, Japan.

Emicizumab is a humanized anti-FIXa/FX (factor IXa/X) bispecific monoclonal antibody that mimics FVIIIa (activated factor VIII) cofactor function. The hemostatic efficacy of emicizumab has been confirmed in clinical studies of patients with hemophilia A, irrespective of the presence of FVIII inhibitors. Emicizumab differs in some properties from FVIIIa molecule. Read More

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http://dx.doi.org/10.1161/ATVBAHA.120.312919DOI Listing

Two-Step Size-Exclusion Nanofiltration of Prothrombin Complex Concentrate Using Nanocellulose-Based Filter Paper.

Biomedicines 2020 Mar 26;8(4). Epub 2020 Mar 26.

Nanotechnology and Functional Materials, Department of Materials Science and Engineering, Uppsala University, Box 534, 751 21 Uppsala, Sweden.

Coagulation Factor IX-rich protrhombin complex concentrate (FIX-PCC) is a therapeutic biologic product that consists of a mixture of several human plasma-derived proteins, useful for treating hemophilia B. Due to its complex composition, FIX-PCC is very challenging to bioprocess through virus removing nanofilters in order to ensure its biosafety. This article describes a two-step filtration process of FIX-PCC using a nanocellulose-based filter paper with tailored porosity. Read More

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http://dx.doi.org/10.3390/biomedicines8040069DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7235758PMC

Formation of novel N-acetylcysteine-hemin adducts abrogates hemin-induced cytotoxicity and suppresses the NRF2-driven stress response in human pro-erythroid K562 cells.

Eur J Pharmacol 2020 Mar 25:173077. Epub 2020 Mar 25.

Laboratory of Pharmacology Department of Pharmaceutical Sciences, School of Health, Aristotle University of Thessaloniki (A.U.Th), Thessaloniki, 54124, Greece. Electronic address:

Heme (iron protoporphyrin IX), as the prosthetic group in hemoproteins, regulates vital cellular functions in human tissues. However, free heme released during hemolysis events promotes severe complications to millions of people worldwide. Over the years, thiols like glutathione (GSH) were known to antagonize heme toxicity. Read More

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http://dx.doi.org/10.1016/j.ejphar.2020.173077DOI Listing
March 2020
2.532 Impact Factor

The Hypoxic Response Expression as a Survival Biomarkers in Treatment-Naive Advanced Breast Cancer.

Asian Pac J Cancer Prev 2020 Mar 1;21(3):629-637. Epub 2020 Mar 1.

Department of Surgery, Faculty of Medicine, Hasanuddin University, Makassar, Indonesia.

Objective: Hypoxia-associated biomarkers profiling may provide information for prognosis, staging, and subsequent therapy. We aim to evaluate whether the quantitative gene and protein expression of hypoxic response tumor markers - carbonic anhydrase IX (CAIX) and hypoxia- inducible factor 1 alpha (HIF1A) - may have a role in predicting survival in advanced breast cancer of Indonesian population.

Methods: Tumor tissues and peripheral blood samples were collected from treatment - naïve locally advanced (LABC) or metastatic breast cancer patients (MBC) at Wahidin Sudirohusodo General Hospital (Makassar, South Sulawesi) and its referral network hospitals from July 2017 to March 2019. Read More

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http://dx.doi.org/10.31557/APJCP.2020.21.3.629DOI Listing
March 2020
1.500 Impact Factor

Real-world assay variability between laboratories in monitoring of recombinant factor IX Fc fusion protein activity in plasma samples.

Int J Lab Hematol 2020 Jun 23;42(3):350-358. Epub 2020 Mar 23.

Swedish Orphan Biovitrum AB (Sobi), Stockholm, Sweden.

Introduction: Monitoring of factor IX (FIX) replacement therapy in haemophilia B relies on accurate coagulation assays. However, considerable interlaboratory variability has been reported for one-stage clotting (OSC) assays. This study aimed to evaluate the real-world, interlaboratory variability of routine FIX activity assays used in clinical haemostasis laboratories for the measurement of recombinant FIX Fc fusion protein (rFIXFc) activity. Read More

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http://dx.doi.org/10.1111/ijlh.13189DOI Listing

Physiological Roles of the von Willebrand Factor-Factor VIII Interaction.

Subcell Biochem 2020 ;94:437-464

Center for Thrombosis and Hemostasis (CTH), University Medical Center Mainz, Langenbeckstrasse 1, Building 708, 55131, Mainz, Germany.

Von Willebrand factor (VWF) and coagulation factor VIII (FVIII) circulate as a complex in plasma and have a major role in the hemostatic system. VWF has a dual role in hemostasis. It promotes platelet adhesion by anchoring the platelets to the subendothelial matrix of damaged vessels and it protects FVIII from proteolytic degradation. Read More

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http://dx.doi.org/10.1007/978-3-030-41769-7_18DOI Listing

Long-Term Safety and Efficacy of Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Previously Treated Pediatric Patients with Hemophilia B: Results from a Phase 3b Extension Study.

Thromb Haemost 2020 Apr 17;120(4):599-606. Epub 2020 Mar 17.

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.

Introduction:  A phase 3b extension study evaluated the long-term safety and efficacy of a recombinant fusion protein-linking coagulation factor IX (FIX) with albumin (rIX-FP) for the routine prophylaxis and on-demand treatment of bleeding in pediatric hemophilia B patients.

Methods:  Previously treated patients aged <12 years with moderate to severe hemophilia B enrolled in a 3-year extension study following a phase 3 pivotal study in which they received weekly rIX-FP prophylaxis. In the extension study, they could maintain or extend their prophylaxis interval to every 10 or 14 days if they were well controlled on the 7-day regimen. Read More

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http://dx.doi.org/10.1055/s-0040-1705116DOI Listing
April 2020
4.984 Impact Factor

Enantioanalysis of glutamine-a key factor in establishing the metabolomics process in gastric cancer.

Anal Bioanal Chem 2020 May 18;412(13):3199-3207. Epub 2020 Mar 18.

National Institute for Research and Development of Isotopic and Molecular Technologies, Donat Street, No. 67-103, 400293, Cluj-Napoca, Romania.

Gastric cancer is the second leading cause of death in the world. Early detection will facilitate early treatment and full recovery of the patients. Metabolomics facilitated the detection of few amino acids able to be used as biomarkers. Read More

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http://dx.doi.org/10.1007/s00216-020-02575-yDOI Listing

Extended half-life rFIX in major surgery-How to improve clinical practice: An intraindividual comparison.

Clin Case Rep 2020 Mar 12;8(3):531-534. Epub 2020 Feb 12.

Haematology Unit Regional Center for Hemorrhagic and Thrombotic Diseases City of Health and Science University Hospital of Molinette Turin Italy.

Practical, safe, and effective hemostatic approach to orthopedic surgery using Extended Half-Life factor IX in hemophilia B. By intraindividual comparison, we found a lower FIX consumption, number of infusions, and cost compared to plasma-derived FIX. Read More

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http://dx.doi.org/10.1002/ccr3.2450DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7069879PMC