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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. Factor XI promotes hemostasis in factor IX deficient mice. Full Text available with Trip Pro

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

3. Emicizumab, the bispecific antibody to factors IX/IXa and X/Xa, potentiates coagulation function in factor XI-deficient plasma in vitro. Full Text available with Trip Pro

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

2018 Journal of Thrombosis and Haemostasis

4. 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

5. Factor IX Deficiency

, NOS , deficiency; PTC (plasma thromboplastin component) , deficiency; plasma thromboplastin component (PTC) , plasma thromboplastin; component, deficiency , Deficiency, PTC , Deficiency, plasma thromboplastin component , Deficiency, functional factor IX , Deficiency, factor IX Italian Deficit di Fattore IX , Disturbi congeniti del fattore IX , Emofilia B (Fattore IX) , Malattia di Christmas (fattore IX) , Malattia di Christmas , Deficienza del fattore IX , Emofilia B Dutch hemofilie B (factor IX (...) 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

2018 FP Notebook

6. Coagadex - human coagulation factor X. In patients with hereditary factor X deficiency

Coagadex - human coagulation factor X. In patients with hereditary factor X deficiency 30 Churchill Place ? Canary Wharf ? London E14 5EU ? United Kingdom An agency of the European Union Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact © European Medicines Agency, 2016. Reproduction is authorised provided the source is acknowledged. 28 January 2016 EMA/CHMP/139881/2016 Committee for Medicinal Products for Human Use (CHMP (...) Agency F1+2 Prothrombin Fragments F1 and F2 FACTOR X syn. for Coagadex FCT Fibrinogen Clotting Time FII Coagulation factor II FIX Coagulation factor IX FX Coagulation factor X FXa Activated coagulation factor X FX:Ag Factor X antigen FX:C Factor X activity FFP Fresh frozen plasma GC Gas chromatography GCP Good Clinical Practice GLP Good Laboratory Practice GMP Good Manufacturing Practice HAV Hepatitis A virus HBV Hepatitis B virus HBsAg Hepatitis B surface antigen HCV Hepatitis C virus HIV Human

2016 European Medicines Agency - EPARs

7. Factor IX from prothrombin complex concentrate augments low dose tissue factor-triggered thrombin generation in vitro. Full Text available with Trip Pro

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

8. Diagnosis and Management of Glycogen Stored Diseases type VI and IX a practice resource of ACMG

. Purpose: Glycogen storage disease (GSD) types VI and IX are rare diseases of variable clinical severity affecting primarily the liver. GSD VI is caused by deficient activity of hepatic glycogen phosphorylase, an enzyme encoded by the PYGL gene. GSD IX is caused by deficient activity of phosphorylase kinase (PhK), the enzymesubunitsofwhichareencodedbyvariousgenes:?(PHKA1, PHKA2), ß (PHKB), ? (PHKG1, PHKG2), and d (CALM1, CALM2, CALM3). Glycogen storage disease types VI and IX have a wide spectrum (...) phosphorylase causes GSD V (OMIM 232600), 4 also known as McArdle disease, and will not be discussed here. Glycogen storage disease type IX, liver form, (OMIM 306000) (GSD IX) is often clinically indistinguishable from GSD VI. It results from deficiency of liver phosphorylase kinase (PhK). Isolated muscle PhK deficiency that is caused by pathogenic variants inPHKA1 and has also been known as GSDIXd,hasalsobeendescribed 5–11 butwillnotbediscussed in further detail here. PhK is a protein kinase

2019 American College of Medical Genetics and Genomics

9. Continuous infusion of recombinant activated factor VII: a review of data in congenital hemophilia with inhibitors and congenital factor VII deficiency. Full Text available with Trip Pro

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

2018 Journal of blood medicine Controlled trial quality: uncertain

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

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

11. Elevated thrombin generation in patients with congenital disorder of glycosylation and combined coagulation factor deficiencies. (Abstract)

Elevated thrombin generation in patients with congenital disorder of glycosylation and combined coagulation factor deficiencies. Congenital disorders of glycosylation are rare inherited diseases affecting many different proteins. The lack of glycosylation notably affects the hemostatic system and leads to deficiencies of both procoagulant and anticoagulant factors.To assess the hemostatic balance in patients with multiple coagulation disorders by using a thrombin generation assay.We performed (...) conventional coagulation assays and a thrombin generation assay on samples from patients with congenital disorder of glycosylation. The thrombin generation assay was performed before and after activation of the protein C system by the addition of soluble thrombomodulin.A total of 35 patients were included: 71% and 57% had low antithrombin and factor XI levels, respectively. Protein C and protein S levels were abnormally low in 29% and 26% of the patients, respectively, whereas only 11% displayed low factor

2019 Journal of Thrombosis and Haemostasis

12. Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX Full Text available with Trip Pro

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

2018 Cell reports

13. Population pharmacokinetics of factor IX in hemophilia B patients undergoing surgery. Full Text available with Trip Pro

: 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

2018 Journal of Thrombosis and Haemostasis

14. Performance of a recombinant fusion protein linking coagulation factor IX with recombinant albumin in one-stage clotting assays. Full Text available with Trip Pro

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

2018 Journal of Thrombosis and Haemostasis

15. Coagulation factor 9-deficient mice are protected against dextran sulfate sodium-induced colitis Full Text available with Trip Pro

from body-weight loss and had a reduced disease activity score. We detected decreased colonic myeloperoxidase activity and decreased CXCL1 levels in DSS-treated F9-deficient mice compared with wild-type (WT) littermate controls, indicating decreased neutrophil infiltration. Remarkably, we identified expression of coagulation factor IX (FIX) protein in small intestinal epithelial cells (MODE-K). In epithelial cell cultures, cellular FIX protein expression was increased following stimulation (...) Coagulation factor 9-deficient mice are protected against dextran sulfate sodium-induced colitis Patients with inflammatory bowel disease (IBD) are susceptible to thromboembolism. Interestingly, IBD occurs less frequently in patients with inherited bleeding disorders. Therefore, we analyzed whether F9-deficiency is protective against the onset of acute colitis in a genetic hemophilia B mouse model. In the 3.5% dextran sulfate sodium (DSS)-induced colitis model, F9-deficient mice were protected

2018 Biology open

16. Turoctocog alfa (Novoeight) - haemophilia A (congenital factor VIII deficiency)

Turoctocog alfa (Novoeight) - haemophilia A (congenital factor VIII deficiency) HAS - Medical, Economic and Public Health Assessment Division 1/18 The legally binding text is the original French version T TR RA AN NS SP PA AR RE EN NC CY Y C CO OM MM MI IT TT TE EE E Opinion 2 April 2014 NOVOEIGHT 250 IU, powder and solvent for solution for injection B/1 vial of powder + 1 prefilled syringe of 4 ml solvent + 1 vial adaptor (CIP:3400958573958) NOVOEIGHT 500 IU, powder and solvent for solution (...) with haemophilia A (congenital factor VIII deficiency)” HAS - Medical, Economic and Public Health Assessment Division 2/18 Actual Benefit The actual benefit of NOVOEIGHT is substantial. Improvement in Actual Benefit NOVOEIGHT does not provide any improvement in actual benefit (level V, non-existent) in the treatment and prophylaxis of haemophilia A compared with other available treatments. Therapeutic use NOVOEIGHT is one of the first-line factor VIII concentrates used in the treatment and prophylaxis

2014 Haute Autorite de sante

17. 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

2012 British Committee for Standards in Haematology

18. 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

19. Systemic delivery of factor IX messenger RNA for protein replacement therapy Full Text available with Trip Pro

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

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

20. Factor IX Deficiency

, NOS , deficiency; PTC (plasma thromboplastin component) , deficiency; plasma thromboplastin component (PTC) , plasma thromboplastin; component, deficiency , Deficiency, PTC , Deficiency, plasma thromboplastin component , Deficiency, functional factor IX , Deficiency, factor IX Italian Deficit di Fattore IX , Disturbi congeniti del fattore IX , Emofilia B (Fattore IX) , Malattia di Christmas (fattore IX) , Malattia di Christmas , Deficienza del fattore IX , Emofilia B Dutch hemofilie B (factor IX (...) 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

2015 FP Notebook

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