A5005016720- Hemophilia Treatment and Research
- Blood Coagulation and Thrombosis Mechanisms
- Platelet Disorders and Treatments
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Virus-based gene therapy research
- Trauma, Hemostasis, Coagulopathy, Resuscitation
- Liver Disease and Transplantation
- Liver Disease Diagnosis and Treatment
- Blood groups and transfusion
- Venous Thromboembolism Diagnosis and Management
- CAR-T cell therapy research
- Viral Infectious Diseases and Gene Expression in Insects
- Antiplatelet Therapy and Cardiovascular Diseases
- Heparin-Induced Thrombocytopenia and Thrombosis
- COVID-19 Clinical Research Studies
- Atrial Fibrillation Management and Outcomes
- CRISPR and Genetic Engineering
- Vitamin K Research Studies
- Complement system in diseases
- Chronic Myeloid Leukemia Treatments
- Acute Kidney Injury Research
- Cardiovascular Issues in Pregnancy
- Muscle and Compartmental Disorders
- Hemostasis and retained surgical items
- Maternal and fetal healthcare
The Royal Free Hospital
2016-2025
University College London
2016-2025
Royal Free London NHS Foundation Trust
1997-2023
Innsbruck Medical University
2023
National Health Service
2023
Sonic Healthcare (Germany)
2022
Royal Victoria Hospital
2008
University of Ulster
2008
Pediatrics and Genetics
2007
University of Padua
2007
Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new therapy in patients with disorder.We infused single dose serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) peripheral vein six severe hemophilia B (FIX activity, <1% normal values). Study participants were enrolled sequentially one three cohorts (given high, intermediate, or...
In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up 16 months. We wanted determine the durability transgene expression, dose–response relationship, and level persistent or late toxicity.
FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses synthetic capsid and gain-of-function protein to normalize factor IX levels in patients with hemophilia B.
Abstract Introduction The factor VIII mimetic emicizumab (Hemlibra, Hoffman‐la Roche, Basel, Switzerland) has a novel mode of action that affects the laboratory monitoring patients receiving this treatment. Aim This guideline from United Kingdom Haemophilia Centre Doctors Organisation ( UKHCDO ) aims to provide advice for clinical and staff on appropriate use assays in with A treated emicizumab. Methodology was prepared by review available literature discussion revision authors. Results...
Background Antiplatelet therapy with aspirin and clopidogrel is known to confer protection against ischemic events. Increasing numbers of patients are presenting for surgery while taking these drugs. This may lead an increase in perioperative blood loss, particularly those who have a heightened response the Identifying preoperatively would allow us plan appropriate management. Methods The antiplatelet effect and/or was measured using two point-of-care monitors: platelet function analyzer...
The international normalised ratio is frequently raised in patients who have undergone major liver resection, and assumed to represent a potential bleeding risk. However, these an increased risk of venous thromboembolic events, despite conventional coagulation tests indicating hypocoagulability. This prospective, observational study undergoing hepatic resection analysed the serial changes early postoperative period. Thrombin generation parameters viscoelastic (thromboelastometry) remained...
This guideline updates the previous guidelines1, 2 published on behalf of British Society for Haematology (BSH) and United Kingdom Haemophilia Centre Doctors' Organisation (UKHCDO), focussing laboratory components diagnosis monitoring. Clinical aspects will be addressed in a separate guideline. was compiled according to BSH process at https://b-s-h.org.uk/media/16732/bsh-guidance-development-process-dec-5-18.pdf. The writing group, which comprised selected members Haemostasis Thrombosis Task...
Acquired haemophilia A (AHA) is an acquired bleeding disorder resulting from autoantibodies against Factor VIII (FVIII). Previous studies have reported differences in FVIII inhibitor kinetics (type I or type II) AHA compared to severe A. To characterise and evaluate the proportions displaying I, II indeterminate kinetics. Single-centre retrospective study of adults with AHA. Type were defined as linear inhibition ≥ 97% inactivation. non-linear inability completely neutralise FVIII. Inhibitor...
To investigate the effects of unfractionated heparin (UFH), low molecular weight (LMWH) and danaparoid (DPD) added to whole blood in vitro on standard heparinase-modified thromboelastogram (TEG) parameters compared with conventional assays coagulation. The UFH, LMWH DPD TEG were prothrombin time, activated partial thromboplastin thrombin time anti-activated factor X (anti-FXa) activity, at concentrations these anticoagulants ranging from 0.025 1 U/ml. In second part study, very (0.005–0.05...
Endogenous heparinoids impair coagulation, evidenced by thrombelastography in cirrhotic patients with bacterial infection, but it is not clear which glycosaminoglycans can be detected native and heparinase-modified thrombelastography. To assess the effects of different on parameters reversibility these heparinase-I-modified Twenty volunteers were enrolled. Solutions heparan sulphate, dermatan chondroitin-4-sulphate prepared at 'equivalent' concentrations, based composition anticoagulant...
To increase middle molecule clearances, high-flux dialyzers with increased internal filtration have been developed. However, dialyzer design and structure may affect thrombin generation platelet activation, thereby risking clotting reduced clearances.Coagulation parameters, platelet, white cell endothelial activation markers were measured prior to following dialysis sessions in 12 patients using two different designed for filtration.Prior dialysis, had evidence of coagulation factor VIII:C,...
Background: Bacterial infections have been proposed as a trigger for portal hypertensive bleeding in cirrhotic patients. Endogenous low molecular weight heparinoids previously detected vitro by heparinase‐modified thromboelastography, but it is not known what type of they are. The aim this study was to assay anti‐Xa concentrations detect heparin activity infected cirrhotics vivo. Methods: We evaluated 30 patients (15 with bacterial infection, 15 infected) and 9 non‐cirrhotic infection....
To determine changes in Factor VIII (FVIII) and von Willebrand (VWF) the first 3 days of puerperium. A prospective study assessing FVIII clotting activity, VWF activity antigen levels 95 women (with singleton uncomplicated pregnancies) during labour on 1, 2 There were no significant differences FVIII, VWF:Ag VWF:CB 1 puerperium compared with labour. was a decrease (P = 0.009) 0.04) day 3. Age, ethnicity, duration mode delivery did not have any effect levels. The pregnancy induced increase is...
Although factor XI (FXI) concentrate is an effective replacement therapy in severe FXI deficiency without inhibitors, some patients are unwilling to receive it because plasma-derived. We report on the use and monitoring of low dose, recombinant VIIa (rFVIIa, NovoSeven®), cover surgery (caesarean section, cholecystectomy abdominoplasty) four female (FXI:C 2-4 IU/dl, aged 32-51 years) who wished avoid exposure plasma. None our had inhibitors FXI. Our aim was find optimal dose rFVIIa by vitro...
In good risk patients (historic inhibitor peak < 200BU), the International Immune Tolerance Study demonstrated equal efficacy to induce tolerance between high (200iu/kg/day) and low dose (50iu/kg ×3 times/week) immune induction (ITI) regimens. However, trial stopped early on account of excessive bleed rate in ITI arm.United Kingdom Haemophilia Centre Doctors' Organization (UKHCDO) Paediatric Inhibitor working parties considered available data alongside bi-phenotypic antibody emicizumab...