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Factor IX Deficiency

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1. Recombinant Human Coagulation Factor IX-FC Fusion Protein (rFIXFc) (ALPROLIX) - hemophilia B (congenital factor IX deficiency or Christmas disease)

Recombinant Human Coagulation Factor IX-FC Fusion Protein (rFIXFc) (ALPROLIX) - hemophilia B (congenital factor IX deficiency or Christmas disease) Search Page - Drug and Health Product Register Language selection Search and menus Search Search website Search Topics menu You are here: Summary Basis of Decision - - Health Canada Expand all Summary Basis of Decision (SBD) for Contact: Summary Basis of Decision (SBD) documents provide information related to the original authorization of a product

2018 Health Canada - Drug and Health Product Register

2. Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use

Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use CasesBlog - Medical and Health Blog: Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use Health News Updated Daily by Internist and Allergist at Cleveland Clinic Florida Pages Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX (...) Variant: the researchers infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX–R338L) transgene in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value. They found sustained therapeutic expression of factor IX coagulant activity after gene transfer in the 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant

2018 CasesBlog - Medical and Health Blog

3. Emicizumab, the bispecific antibody to factors IX/IXa and X/Xa, potentiates coagulation function in factor XI-deficient plasma in vitro. (PubMed)

Emicizumab, the bispecific antibody to factors IX/IXa and X/Xa, potentiates coagulation function in factor XI-deficient plasma in vitro. Essentials Emicizumab mimics factor (F)VIIIa cofactor function, augments the intrinsic tenase activity. We assessed the emicizumab-driven hemostatic function in FXI-deficient plasmas. Emicizumab improved the coagulation potentials in severe FXI-deficient plasma. Emicizumab may provide a possibility for clinical application in patients with FXI deficiency (...) . SUMMARY: Background Patients with factor (F)XI deficiency commonly present with markedly prolonged activated partial thromboplastin times (APTT), although bleeding phenotypes are heterogeneous. Emicizumab, a bispecific monoclonal antibody to FIX/FIXa and FX/FXa, mimics FVIIIa cofactor function on phospholipid (PL) surfaces. Antibody reactions were designed, therefore, to augment mechanisms during the propagation phase of blood coagulation. Aim To assess emicizumab-driven hemostatic function in FXI

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2018 Journal of Thrombosis and Haemostasis

4. Factor XI promotes hemostasis in factor IX deficient mice. (PubMed)

Factor XI promotes hemostasis in factor IX deficient mice. Essentials Mice lacking factor IX (FIX) or factor XI (FXI) were tested in a saphenous vein bleeding model. FIX-deficient mice displayed a hemostatic defect and FXI-deficient mice were similar to wild type mice. Infusion of FXI or over-expression of FXI in FIX-deficient mice improved hemostasis. FXI may affect the phenotype of FIX-deficiency (hemophilia B).Background In humans, deficiency of coagulation factor XI may be associated (...) with a bleeding disorder, but, until recently, FXI-deficient mice did not appear to have a hemostatic abnormality. A recent study, however, indicated that FXI-deficient mice show a moderate hemostatic defect in a saphenous vein bleeding (SVB) model. Objectives To study the effect of FXI on bleeding in mice with normal levels of the FXI substrate FIX and in mice lacking FIX (a murine model of hemophilia B). Methods Wild-type mice and mice lacking either FIX (F9- ) or FXI (F11-/- ) were tested in the SVB model

2018 Journal of Thrombosis and Haemostasis

5. Factor IX Deficiency

Factor IX Deficiency Factor IX Deficiency Toggle navigation Brain Head & Neck Chest Endocrine Abdomen Musculoskeletal Skin Infectious Disease Hematology & Oncology Cohorts Diagnostics Emergency Findings Procedures Prevention & Management Pharmacy Resuscitation Trauma Emergency Procedures Ultrasound Cardiovascular Emergencies Lung Emergencies Infectious Disease Pediatrics Neurologic Emergencies Skin Exposure Miscellaneous Abuse Cancer Administration 4 Factor IX Deficiency Factor IX Deficiency (...) Aka: Factor IX Deficiency , Christmas Disease , Hemophilia B , Plasma thromboplastin component deficiency From Related Chapters II. Pathophysiology Inherited sex linked trait Clinically indistinguishable from III. Symptoms Chronic history of since childhood Spontaneous bleeding Excessive follows: Dental procedures Surgery IV. Signs Joint deformities Muscle contractures V. Labs (PTT) prolonged Corrects with Factor IX supplementation Corrects with Normal serum VI. Management: Recombinant Factor IX

2018 FP Notebook

6. Factor IX from prothrombin complex concentrate augments low dose tissue factor-triggered thrombin generation in vitro. (PubMed)

Factor IX from prothrombin complex concentrate augments low dose tissue factor-triggered thrombin generation in vitro. Prothrombin complex concentrate (PCC) is increasingly used to correct acquired coagulopathy in trauma and surgery. Dosing of PCC is guided by the prothrombin time, which only reflects the onset of thrombin generation, but does not account for variations in intrinsic pathway coagulation factors, including factor IX (FIX). We hypothesised that FIX contained in PCC could strongly (...) , or both, to FIX-deficient plasma increased peak thrombin generation, and prolonged the inhibitory phase of the endogenous thrombin potential.FIX derived from PCC strongly enhances tissue factor-triggered thrombin generation in the presence of elevated FVIII activity. Haemodilution further enhances procoagulant effects of FIX and FVIII by slowing down inhibition of procoagulant enzymes. Dosing of PCC per prothrombin time may underestimate PCC's procoagulant potential because it does not account

2018 British Journal of Anaesthesia

7. Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX (PubMed)

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient mouse model. We show that transplanted, cryopreserved, cadaveric human hepatocytes remain functional for more than a year and secrete FIX at therapeutic

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2018 Cell reports

8. Population pharmacokinetics of factor IX in hemophilia B patients undergoing surgery. (PubMed)

: Background Hemophilia B is a bleeding disorder characterized by a deficiency of coagulation factor IX (FIX). In the perioperative setting, patients receive FIX concentrates to ensure hemostasis. Although FIX is usually dosed according to bodyweight, under- and overdosing occurs frequently during surgery. Aim The objective was to quantify and explain the interpatient variability of perioperatively administered plasma-derived (pd) and recombinant (r) FIX concentrates. Methods Data were collected from 118 (...) Population pharmacokinetics of factor IX in hemophilia B patients undergoing surgery. Essentials Factor IX (FIX) dosing using body weight frequently results in under and overdosing during surgery. We aimed to establish a population pharmacokinetic (PK) model describing the perioperative FIX levels. Population PK parameter values for clearance and V1 were 284 mL h-170 kg-1 and 5450 mL70 kg-1. Perioperative PK parameters differ from those during non-surgical prophylactic treatment. SUMMARY

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2018 Journal of Thrombosis and Haemostasis

9. Performance of a recombinant fusion protein linking coagulation factor IX with recombinant albumin in one-stage clotting assays. (PubMed)

Performance of a recombinant fusion protein linking coagulation factor IX with recombinant albumin in one-stage clotting assays. Essentials Performance of the one-stage clotting (OSC) assay varies with the clotting activator used. Recombinant FIX-albumin fusion protein (rIX-FP) was reliably monitored with most OSC reagents. rIX-FP shows comparable reagent-dependent variability to other rFIX products in the OSC assay. Actin® FS and kaolin-based reagents underestimated rIX-FP activity by around (...) 50% in the OSC assay. SUMMARY: Background Measuring factor IX activity (FIX:C) with one-stage clotting (OSC) assays, based on the activated partial thromboplastin time (APTT), is the current mainstay of diagnostic techniques for hemophilia B. Assessing the performance of new recombinant FIX (rFIX) products in OSC assays is essential, as APTT reagents from different manufacturers yield different potency estimates for rFIX. Objectives To evaluate the extent to which choice of reagent composition

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2018 Journal of Thrombosis and Haemostasis

10. A Factor IX Gene Therapy Study (FIX-GT)

A Factor IX Gene Therapy Study (FIX-GT) A Factor IX Gene Therapy Study (FIX-GT) - Full Text View - ClinicalTrials.gov Hide glossary Glossary Study record managers: refer to the if submitting registration or results information. Search for terms x × Study Record Detail Saved Studies Save this study Warning You have reached the maximum number of saved studies (100). Please remove one or more studies before adding more. A Factor IX Gene Therapy Study (FIX-GT) (FIX-GT) The safety and scientific (...) ): University College, London Study Details Study Description Go to Brief Summary: Severe haemophilia B is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor; with severe haemophilia B, levels of clotting factor IX (FIX) (nine) are very low and affected individuals can suffer life threatening bleeding episodes. HB mainly affects boys and men (normally one in every 30,000 males). Current treatment

2017 Clinical Trials

11. Systemic delivery of factor IX messenger RNA for protein replacement therapy (PubMed)

Systemic delivery of factor IX messenger RNA for protein replacement therapy Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (FIX)-deficient mouse model of hemophilia B. Delivery of human FIX (hFIX) mRNA encapsulated in our LUNAR LNPs results in a rapid pulse of FIX protein (within 4-6 h (...) ) that remains stable for up to 4-6 d and is therapeutically effective, like the recombinant human factor IX protein (rhFIX) that is the current standard of care. Extensive cytokine and liver enzyme profiling showed that repeated administration of the mRNA-LUNAR complex does not cause any adverse innate or adaptive immune responses in immune-competent, hemophilic mice. The levels of hFIX protein that were produced also remained consistent during repeated administrations. These results suggest that delivery

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2017 Proceedings of the National Academy of Sciences of the United States of America

12. Overestimation of N9-GP (N-glycoPEGylated FIX) in one-stage factor IX clotting method due to silica-mediated precocious conversion to factor IXa. (PubMed)

Overestimation of N9-GP (N-glycoPEGylated FIX) in one-stage factor IX clotting method due to silica-mediated precocious conversion to factor IXa. Essentials Nonacog beta pegol (N9-GP) activity is overestimated in clot method using silica-based reagents. Mimicking contact activation phase with silica reveals N9-GP activation before recalcification. Localization of N9-GP to silica facilitates activation by factor XIa and plasma kallikrein. Silica-based reagents to be used with caution when (...) monitoring N9-GP therapy using clot method.Background Clinical laboratories routinely quantify factor IX (FIX) activity by measurement of the activated partial thromboplastin time (APTT) in a one-stage (OS) clotting assay. This assay can be performed with any of a plethora of differently composed APTT reagents, giving variable recovery when applied to nonacog beta pegol (N9-GP), an N-glycoPEGylated recombinant FIX. Objective To identify the cause of observed overestimations of N9-GP activity in an OS FIX

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2016 Journal of Thrombosis and Haemostasis

13. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia

Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition) - Collins - 2013 - British Journal of Haematology - Wiley Online Library Search within Search term Search term The full text of this article hosted at iucr.org is unavailable due to technical difficulties. Guideline Free Access Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th (...) with different health care arrangements and resources. Methods The writing group reviewed publications known to them supplemented with papers identified through Pubmed, using index terms h(a)emophilia, factor VIII and IX, inhibitors, alloantibodies, rFVIIa, NovoSeven, FEIBA, aPCC, rituximab, management. The writing group produced the draft guideline which was reviewed and revised by members of the UKHCDO Advisory Board. The guideline was finally reviewed by a sounding board of approximately 50 UK

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2013 United Kingdom Haemophilia Centre Doctors' Organisation

14. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia

Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition) - Collins - 2013 - British Journal of Haematology - Wiley Online Library By continuing to browse this site, you agree to its use of cookies as described in our . Search within Search term Search term The full text of this article hosted at iucr.org is unavailable due to technical difficulties. Guideline Free Access (...) Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition) School of Medicine, Cardiff University, University Hospital of Wales, Wales, UK Royal Hospital for Sick Children, Glasgow, UK The London School of Medicine and Dentistry, Royal London Hospital, Barts, Queen Mary University, London, UK Great Ormond Street NHS Trust, London, UK Hampshire Hospital NHS Foundation Trust, Basingstoke & North Hampshire Hospital, Basingstoke, UK Royal Victoria Infirmary, Newcastle

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2012 British Committee for Standards in Haematology

15. Continuous infusion of recombinant activated factor VII: a review of data in congenital hemophilia with inhibitors and congenital factor VII deficiency. (PubMed)

Continuous infusion of recombinant activated factor VII: a review of data in congenital hemophilia with inhibitors and congenital factor VII deficiency. Continuous infusion (CI) of clotting factors as a replacement therapy for perioperative hemostatic protection has been performed for many years, including with factors VIII and IX and recombinant activated factor VII (rFVIIa). This approach provides steady factor levels without requiring frequent administration of bolus doses.To review safety (...) , efficacy, and dosing data regarding CI of rFVIIa for hemostatic management of patients with congenital hemophilia with inhibitors (CHwI) or congenital factor VII deficiency (C7D).A literature review identified instances of CI of rFVIIa in patients with CHwI or C7D undergoing surgery or experiencing bleeding episodes. Data regarding safety, efficacy, and dosing were extracted.The safety and efficacy of 50 mcg/kg/h CI of rFVIIa following a 90 mcg/kg bolus injection, vs a standard bolus injection regimen

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2018 Journal of blood medicine

16. Exon 2 Skipping Eliminates Gamma-Glutamyl Carboxylase Activity, Indicating a Partial Splicing Defect in a Patient with Vitamin K Clotting Factor Deficiency. (PubMed)

Exon 2 Skipping Eliminates Gamma-Glutamyl Carboxylase Activity, Indicating a Partial Splicing Defect in a Patient with Vitamin K Clotting Factor Deficiency. Mutations in the gamma-glutamyl carboxylase (GGCX), which is required for vitamin K-dependent (VKD) protein activation, can result in vitamin K clotting factor deficiency (VKCFD1). A recent report described a VKCFD1 patient with a homozygous carboxylase mutation that altered splicing and deleted exon 2 (Δ2GGCX). Only Δ2GGCX RNA was observed (...) in the patient.Loss of exon 2 deletes carboxylase sequences thought to be important for membrane topology and consequent function. Carboxylase activity is required for life, and we therefore tested whether the Δ2GGCX mutant is active.HEK 293 cells were edited using CRISPR-Cas9 to eliminate endogenous carboxylase. r-Wild type- and r-Δ2GGCX carboxylases were then expressed and tested for carboxylation of the VKD protein factor IX. A second approach monitored carboxylation biochemically, using r-carboxylases

2019 Journal of Thrombosis and Haemostasis

17. Single synonymous mutation in factor IX alters protein properties and underlies haemophilia B. (PubMed)

Single synonymous mutation in factor IX alters protein properties and underlies haemophilia B. Haemophilia B is caused by genetic aberrations in the F9 gene. The majority of these are non-synonymous mutations that alter the primary structure of blood coagulation factor IX (FIX). However, a synonymous mutation c.459G>A (Val107Val) was clinically reported to result in mild haemophilia B (FIX coagulant activity 15%-20% of normal). The F9 mRNA of these patients showed no skipping or retention (...) of introns and/or change in mRNA levels, suggesting that mRNA integrity does not contribute to the origin of the disease in affected individuals. The aim of this study is to elucidate the molecular mechanisms that can explain disease manifestations in patients with this synonymous mutation.We analyse the molecular mechanisms underlying the FIX deficiency through in silico analysis and reproducing the c.459G>A (Val107Val) mutation in stable cell lines. Conformation and non-conformation sensitive

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2016 Journal of Medical Genetics

18. Baboon envelope pseudotyped lentiviral vectors transduce efficiently human B cells and allow active factor IX B cell secretion in vivo in NOD/SCID mice. (PubMed)

transfer of BaEV-LV-transduced mature B cells into NOD/SCID/γc-/- (NSG) [non-obese diabetic (NOD), severe combined immuno-deficiency (SCID)] mice allowed differentiation into plasmablasts and plasma B cells, confirming a sustained high-level gene marking in vivo. As proof of principle, we assessed BaEV-LV for transfer of human factor IX (hFIX) into B cells. BaEV-LVs encoding FIX efficiently transduced hB cells and their transfer into NSG mice demonstrated for the first time secretion of functional hFIX (...) Baboon envelope pseudotyped lentiviral vectors transduce efficiently human B cells and allow active factor IX B cell secretion in vivo in NOD/SCID mice. Essentials B cells are attractive targets for gene therapy and particularly interesting for immunotherapy. A baboon envelope pseudotyped lentiviral vector (BaEV-LV) was tested for B-cell transduction. BaEV-LVs transduced mature and plasma human B cells with very high efficacy. BaEV-LVs allowed secretion of functional factor IX from B cells

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2016 Journal of Thrombosis and Haemostasis

19. A two-component system regulates gene expression of the type IX secretion component proteins via an ECF sigma factor (PubMed)

A two-component system regulates gene expression of the type IX secretion component proteins via an ECF sigma factor The periodontopathogen Porphyromonas gingivalis secretes potent pathogenic proteases, gingipains, via the type IX secretion system (T9SS). This system comprises at least 11 components; however, the regulatory mechanism of their expression has not yet been elucidated. Here, we found that the PorY (PGN_2001)-PorX (PGN_1019)-SigP (PGN_0274) cascade is involved in the regulation (...) deficient in a putative extracytoplasmic function (ECF) sigma factor, PGN_0274 (SigP), similar to the porX mutant. Electrophoretic gel shift assays showed that rSigP bound to the putative promoter regions of T9SS component-encoding genes. The SigP protein was lacking in the porX mutant. Co-immunoprecipitation and SPR analysis revealed the direct interaction between SigP and PorX. Together, these results indicate that the PorXY TCS regulates T9SS-mediated protein secretion via the SigP ECF sigma factor.

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2016 Scientific reports

20. CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse (PubMed)

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse The X-linked genetic bleeding disorder caused by deficiency of coagulator factor IX, hemophilia B, is a disease ideally suited for gene therapy with genome editing technology. Here, we identify a family with hemophilia B carrying a novel mutation, Y371D, in the human F9 gene. The CRISPR/Cas9 system was used to generate distinct genetically modified mouse models and confirmed

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2016 EMBO molecular medicine

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