Haemophilia B: impact on patients and economic burden of disease.

Abstract:

:Worldwide, haemophilia is the most common hereditary bleeding disorder. The incidence of haemophilia B, however, is considerably less than haemophilia A and consequently appears to have received less attention in the research literature. This article aims to summarise the available evidence documenting the patient and economic burden associated with haemophilia B and current methods of disease management. Both the immediate and long-term clinical consequences of haemophilia B can have significant implications for patients in terms of functional limitations and diminished health-related quality of life (HRQOL). Evidence demonstrates that primary prophylaxis is the optimal strategy for replacing missing clotting factor IX (FIX) and managing haemophilia B. Use of recombinant FIX (rFIX) over plasma-derived FIX (pd-FIX) is also generally preferred for safety reasons. Prophylaxis using currently available rFIX products, however, requires a demanding regimen of intravenous infusions 2-3 times a week which may have significant implications for adherence and ultimately the long-term efficacy of such regimens. Only limited assessments of the cost-effectiveness of prophylactic versus on-demand FIX treatment regimens have been conducted to date. Prophylaxis, however, is generally more costly as greater quantities of FIX are consumed. Any reduction in FIX replacement dosing frequency is expected to improve patient adherence and contribute to improved clinical outcomes, further supporting the cost-effectiveness of such interventions. Although a rare disease, as economic constraints for healthcare increase, generating further information regarding the key clinical, patient and economic outcomes associated with haemophilia B will be essential for supporting improvements in care for people with haemophilia B.

journal_name

Thromb Haemost

authors

Gater A,Thomson TA,Strandberg-Larsen M

doi

10.1160/TH11-03-0193

subject

Has Abstract

pub_date

2011-09-01 00:00:00

pages

398-404

issue

3

eissn

0340-6245

issn

2567-689X

pii

11-03-0193

journal_volume

106

pub_type

杂志文章,评审
  • A study of the possible role of mesothelium as a surface for flowing blood.

    abstract::Mouse mesothelium has been examined as a surface for supporting blood flow. We have examined ten pieces of intact mesothelium and ten pieces of damaged mesothelium following 10 min exposure to flowing blood in a Baumgartner chamber. Scanning electron microscopy of the intact specimens demonstrated no adhering blood pl...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Clarke JM,Pittilo RM,Machin SJ,Woolf N

    更新日期:1984-02-28 00:00:00

  • Low prevalence of factor V:Q506 in 41 patients with isolated pulmonary embolism.

    abstract::In 70-80% of cases, pulmonary embolism is the consequence of lower extremity deep vein thrombosis. It has been demonstrated that the most common coagulation defect predisposing to venous thrombosis, resistance to activated protein C (APC), is not associated with an increased risk for pulmonary embolism, but the eviden...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Martinelli I,Cattaneo M,Panzeri D,Mannucci PM

    更新日期:1997-03-01 00:00:00

  • Detection and neutralisation of heparin by a fluorescent ruthenium compound.

    abstract::Heparin and low-molecular-weight heparin (LMWH) are commonly monitored by determination of activated clotting times or chromogenic assays. Despite their wide use, these assays determine the biological activity and not the concentration of the anticoagulants. They may be inaccurate in some circumstances such as certain...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1160/TH09-03-0168

    authors: Szelke H,Harenberg J,Krämer R

    更新日期:2009-11-01 00:00:00

  • Evaluation of monoclonal antifibrin antibodies by their binding to human blood clots.

    abstract::The primary goal of this study was to develop a method for evaluating fibrin-specific antibodies as thrombus detecting agents. The apparatus and assay conditions were chosen by testing antibody 64C5, which binds to the amino terminus of the fibrin beta chain, for its ability to bind to human blood clots. Using 125I-la...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Liau CS,Haber E,Matsueda GR

    更新日期:1987-02-03 00:00:00

  • Angiogenesis: molecular mechanisms and functional interactions--2nd Kloster Seeon Meeting of the German Priority Research Grant "Angiogenesis".

    abstract::The German Priority Research Program "Angiogenesis" (www.angiogenese.de) hosts a biannual meeting in the Kloster Seeon in Southern Germany. The 2nd Kloster Seeon Meeting "Angiogenesis: Molecular Mechanisms and Functional Interactions" was held in September 2002. It included sessions on hypoxia, the biology of endothel...

    journal_title:Thrombosis and haemostasis

    pub_type:

    doi:

    authors: Augustin HG,Breier G

    更新日期:2003-01-01 00:00:00

  • Anfibatide, a novel GPIb complex antagonist, inhibits platelet adhesion and thrombus formation in vitro and in vivo in murine models of thrombosis.

    abstract::Platelet adhesion and aggregation at the sites of vascular injury are key events for thrombosis and haemostasis. It has been well demonstrated that interaction between glycoprotein (GP) Ibα and von Willebrand factor (VWF) initiates platelet adhesion and contributes to platelet aggregation, particularly at high shear. ...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1160/TH13-06-0490

    authors: Lei X,Reheman A,Hou Y,Zhou H,Wang Y,Marshall AH,Liang C,Dai X,Li BX,Vanhoorelbeke K,Ni H

    更新日期:2014-02-01 00:00:00

  • Thrombosis and inflammation as multicellular processes: significance of cell-cell interactions.

    abstract::Platelet activation as a result of vascular injury provokes endothelial cells to respond in a manner which limits or reverses the occlusive consequences of platelet accumulation. If the agonistic forces are strong, platelet accumulation is irreversible. In vitro data from our laboratory have repeatedly demonstrated th...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章,评审

    doi:

    authors: Marcus AJ,Safier LB,Broekman MJ,Islam N,Fliessbach JH,Hajjar KA,Kaminski WE,Jendraschak E,Silverstein RL,von Schacky C

    更新日期:1995-07-01 00:00:00

  • Once-daily subcutaneous dalteparin, a low molecular weight heparin, for the initial treatment of acute deep vein thrombosis.

    abstract::The aim of the study was to compare the efficacy and safety of once-daily subcutaneous injection of dalteparin, a low molecular weight heparin, with that of intravenous unfractionated heparin in the treatment of deep venous thrombosis (DVT). Patients were included if they had deep venous thrombosis distal to inguinal ...

    journal_title:Thrombosis and haemostasis

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:

    authors: Fiessinger JN,Lopez-Fernandez M,Gatterer E,Granqvist S,Kher A,Olsson CG,Söderberg K

    更新日期:1996-08-01 00:00:00

  • Purification of human brain tissue factor.

    abstract::Tissue factor (thromboplastin or Factor III), a glycoprotein cofactor, is required for Factor VII to express its catalytic activity, thereby initiating the extrinsic as well as intrinsic pathway of blood coagulation. Human brain tissue factor was purified 2,500-fold to 98% homogeneity from 2% Triton X-100 extraction o...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Kang S,Niemetz J

    更新日期:1988-06-16 00:00:00

  • Interaction of bovine platelets with bovine glomerular basement membrane.

    abstract::The interaction of bovine platelets with bovine glomerular basement membrane has been studied by aggregometry, transmission and scanning electron microscopy and measurement of [3H] serotonin release. In the absence of added calcium platelets adhere to basement membrane but fail to undergo the release reaction or aggre...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Davis JL,Chandler DK,Riquetti P,Rogers S,Kocan KM,Ownby CL,Hudson BG,Carraway KL

    更新日期:1979-12-21 00:00:00

  • Hypertrophic Cardiomyopathy in Patients with Atrial Fibrillation: Prevalence and Associated Stroke Risks in a Nationwide Cohort Study.

    abstract:BACKGROUND: There have been no prior nationwide reports on the prevalence of hypertrophic cardiomyopathy (HCM) among patients suffering from atrial fibrillation (AF). It is also unclear how much stroke risk is attributable to HCM compared with other stroke risks in patients with AF. This study assessed the prevalence o...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1055/s-0038-1676818

    authors: Jung H,Yang PS,Sung JH,Jang E,Yu HT,Kim TH,Uhm JS,Kim JY,Pak HN,Lee MH,Lip GYH,Joung B

    更新日期:2019-02-01 00:00:00

  • Plasma glycoprotein V levels in the general population: normal distribution, associated parameters and implications for clinical studies.

    abstract::Soluble glycoprotein V (sGPV) is a new plasma marker of thrombosis released from the platelet surface by thrombin. sGPV levels are increased in patients with atherothrombotic diseases, but the determinants of sGPV levels are unknown in the general population. Identification of these potential confounding factors is ne...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Aleil B,Meyer N,Wolff V,Kientz D,Wiesel ML,Gachet C,Cazenave JP,Lanza F

    更新日期:2006-10-01 00:00:00

  • Inhibition of ADP-induced P-selectin expression and platelet-leukocyte conjugate formation by clopidogrel and the P2Y12 receptor antagonist AR-C69931MX but not aspirin.

    abstract::Platelet-leukocyte interactions are recognised to have pro-inflammatory effects, which may be important in the pathophysiology of ischaemic heart disease. Clopidogrel and the novel intravenous antithrombotic agent AR-C69931MX act at the level of the platelet P2Y12 receptor, which is known to amplify platelet activatio...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Storey RF,Judge HM,Wilcox RG,Heptinstall S

    更新日期:2002-09-01 00:00:00

  • Ex vivo Improvement of a von Willebrand Disease Type 2A Phenotype Using an Allele-Specific Small-Interfering RNA.

    abstract::Von Willebrand disease (VWD) is the most common inherited bleeding disorder and is mainly caused by dominant-negative mutations in the multimeric protein von Willebrand factor (VWF). These mutations may either result in quantitative or qualitative defects in VWF. VWF is an endothelial protein that is secreted to the c...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1055/s-0040-1715442

    authors: de Jong A,Dirven RJ,Boender J,Atiq F,Anvar SY,Leebeek FWG,van Vlijmen BJM,Eikenboom J

    更新日期:2020-11-01 00:00:00

  • The Effect of Age on von Willebrand Factor and Bleeding Symptoms in von Willebrand Disease.

    abstract::von Willebrand disease (VWD) is a quantitative or qualitative defect in von Willebrand factor (VWF) resulting in mucocutaneous bleeding symptoms and hemorrhage following hemostatic challenges, such as trauma or surgery. VWD-specific therapy, DDAVP (1-desamino-8-D-arginine vasopressin) and VWF concentrates, is necessar...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1055/s-0040-1713636

    authors: Seaman CD,Ragni MV

    更新日期:2020-08-01 00:00:00

  • Red cell distribution width (RDW) as a predictor of long-term mortality in patients undergoing percutaneous coronary intervention.

    abstract::Red cell distribution width (RDW) has been shown to be an independent predictor of mortality in patients with coronary artery disease and in patients with heart failure. The current study evaluated the prognostic utility of RDW in patients undergoing percutaneous coronary intervention (PCI). We evaluated 859 patients ...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1160/TH09-02-0127

    authors: Poludasu S,Marmur JD,Weedon J,Khan W,Cavusoglu E

    更新日期:2009-09-01 00:00:00

  • Factor VIII: C and factor VIII R: Ag in Argentine hemorrhagic fever.

    abstract::Factor VIII procoagulant activity (F VIII:C) and factor VIII related antigen (F VIII R: Ag) were investigated in 35 patients with Argentine hemorrhagic fever. Since the results obtained in the three clinical forms of the disease were not significantly different, they were tabulated altogether. F VIII:C was low in earl...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Molinas FC,Maiztegui JI

    更新日期:1981-08-28 00:00:00

  • Commercial antithrombin concentrate contains inactive L-forms of antithrombin.

    abstract::The preparation of antithrombin concentrate for clinical use requires a viral inactivation step. In most commercial preparations this is achieved by heat pasteurisation. This process would be expected to alter the conformation of antithrombin from the active native species to an inactive latent (L-form) state (1, 2). ...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Chang WS,Harper PL

    更新日期:1997-02-01 00:00:00

  • Activation and inactivation of human protein C by plasmin.

    abstract::Transient procoagulant states resulting in failure of recanalization or rethrombosis of the reperfused artery during thrombolytic therapy might be due to an inhibitory effect of plasmin on the anticoagulant properties of protein C. We therefore studied the effect of plasmin on protein C (PC) and activated protein C (A...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Váradi K,Philapitsch A,Santa T,Schwarz HP

    更新日期:1994-05-01 00:00:00

  • Multiplexed genetic profiling of human blood platelets using fluorescent microspheres.

    abstract::Human platelets have unique and reproducible mRNA profiles, with evidence for distinct profiles in haematopoietic stem cell disorders associated with thrombocytosis. Platelet transcript profiling is traditionally studied by microarray analysis, quantitative reverse transcription-PCR or serial analysis of gene expressi...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Gnatenko DV,Zhu W,Bahou WF

    更新日期:2008-11-01 00:00:00

  • Secreted ADP plays a central role in thrombin-induced phospholipase D activation in human platelets.

    abstract::Thrombin and other agonists that induce secretion and aggregation in human platelets also activate phospholipase D (PLD), but the signaling cascade leading to activation of PLD in human platelets is not yet clear. We have determined that apyrase, which scavenges ADP secreted during platelet activation, is able to bloc...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Martinson EA,Scheible S,Marx-Grunwitz A,Presek P

    更新日期:1998-12-01 00:00:00

  • Structure and function of the urokinase receptor.

    abstract::The binding of the urokinase plasminogen activator (uPA) to its receptor (uPAR) regulates cell adhesion, surface proteolysis, chemotaxis and cell extravasation in a number of experimental systems. Recent evidences have suggested that uPAR can by itself mediate chemotaxis of human monocytes and cause profound changes i...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章,评审

    doi:

    authors: Mondino A,Resnati M,Blasi F

    更新日期:1999-09-01 00:00:00

  • Increased plasma levels of plasminogen activator inhibitor-1 and soluble vascular cell adhesion molecule after triacylglycerol infusion in man.

    abstract::Increased plasma plasminogen activator inhibitor-1 (PAI-1) has been implicated in the development of vascular disease. In type 2 diabetes mellitus high PAI-1 levels are associated with increased plasma concentrations of free fatty acids (FFA) and triacylglycerol indicating an association or a causal relationship. To a...

    journal_title:Thrombosis and haemostasis

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1160/TH03-07-0457

    authors: Krebs M,Geiger M,Polak K,Vales A,Schmetterer L,Wagner OF,Waldhäusl W,Binder BR,Roden M

    更新日期:2003-09-01 00:00:00

  • Expression of vascular endothelial growth factor in human monocyte/macrophages stimulated with lipopolysaccharide.

    abstract::Vascular endothelial growth factor (VEGF) is a mitogen for endothelial cells. We have studied the production of VEGF by human macrophages in response to lipopolysaccharide (LPS). Macrophages stimulated with LPS expressed VEGF mRNA and protein in concentration- and time-dependent manners. The LPS-induced expression of ...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Itaya H,Imaizumi T,Yoshida H,Koyama M,Suzuki S,Satoh K

    更新日期:2001-01-01 00:00:00

  • A sensitive immunochemical assay for measuring the concentration of the activated protein C-protein C inhibitor complex in plasma: use of a catcher antibody specific for the complexed/cleaved form of the inhibitor.

    abstract::Activated protein C (APC) is a serine proteinase that regulates blood coagulation. In plasma it is inhibited mainly by the protein C inhibitor (PCI). The plasma concentrations of APC-PCI complex is increased in hypercoagulative states such as deep venous thrombosis. Formation of the APC-PCI complex induces a drastic c...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Strandberg K,Kjellberg M,Knebel R,Lilja H,Stenflo J

    更新日期:2001-08-01 00:00:00

  • Influence of polymorphisms in the factor VII gene promoter on activated factor VII levels and on the risk of myocardial infarction in advanced coronary atherosclerosis.

    abstract::In this study, we investigate the influence of three factor VII (FVII) gene polymorphisms on activated FVII levels (FVIIa), and also on the risk of myocardial infarction (MI) in patients with advanced coronary atherosclerotic disease (CAD). The -323A2 allele in the promoter is known to be associated with low FVII leve...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:10.1160/TH04-02-0130

    authors: Bozzini C,Girelli D,Bernardi F,Ferraresi P,Olivieri O,Pinotti M,Martinelli N,Manzato F,Friso S,Villa G,Pizzolo F,Beltrame F,Corrocher R

    更新日期:2004-09-01 00:00:00

  • Prediction of pulmonary embolism extent by clinical findings, D-dimer level and deep vein thrombosis shown by ultrasound.

    abstract::Pulmonary embolism (PE) may encompass a wide spectrum of severity. To determine whether clinical findings, D-dimer (DD) concentration, and deep vein thrombosis (DVT) shown by lower-limb venous compression ultrasonography (US) might predict the scintigraphic extent of PE, we studied 104 hemodynamically stable consecuti...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Galle C,Papazyan JP,Miron MJ,Slosman D,Bounameaux H,Perrier A

    更新日期:2001-11-01 00:00:00

  • Bleeding in uremic patients after carbenicillin.

    abstract::Hemorrhagic diathesis was observed in patients with renal insufficiency after carbenicillin at serum levels greater than 300 mug/ml. Normal coagulation factors (F. I, II, V, VII, VIII, X), normal PTT, normal platelet counts, negative ethanol gelation test (fibrin monomers) were found as well as a prolongation of throm...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章

    doi:

    authors: Andrassy K,Weischedel E,Ritz E,Andrassy T

    更新日期:1976-08-31 00:00:00

  • F7 gene variants modulate protein levels in a large cohort of patients with factor VII deficiency. Results from a genotype-phenotype study.

    abstract::Congenital factor VII (FVII) deficiency is a rare bleeding disorder caused by mutations in F7 gene with autosomal recessive inheritance. A clinical heterogeneity with poor correlation with FVII:C levels has been described. It was the objective of this study to identify genetic defects and to evaluate their relationshi...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章,多中心研究

    doi:10.1160/TH17-02-0085

    authors: Quintavalle G,Riccardi F,Rivolta GF,Martorana D,Di Perna C,Percesepe A,Tagliaferri A,Ad-Hoc Study Group.

    更新日期:2017-08-01 00:00:00

  • The vascular biology of macrophage migration inhibitory factor (MIF). Expression and effects in inflammation, atherogenesis and angiogenesis.

    abstract::Macrophage migration inhibitory factor (MIF) is a pleiotropic cytokine with chemokine-like functions. MIF is a critical mediator of the host immune and inflammatory response. Dysregulated MIF expression has been demonstrated to contribute to various acute and chronic inflammatory conditions as well as cancer developme...

    journal_title:Thrombosis and haemostasis

    pub_type: 杂志文章,评审

    doi:10.1160/TH12-11-0831

    authors: Asare Y,Schmitt M,Bernhagen J

    更新日期:2013-03-01 00:00:00